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531 Study Matches

MT2013-31:Allogeneic Hematopoietic Cell Transplantation for Inherited Metabolic Disorders and Severe Osteopetrosis following Conditioning with Busulfan (Therapeutic Drug Monitoring), Fludarabine +/- ATG

To evaluate the ability to achieve high-level donor hematopoietic engraftment (defined as neutrophil recovery by Day +42 post-transplant and ≥ 80% donor cells on the myeloid fraction of peripheral blood at Day +100 post-transplant) using related and unrelated BM, PBSC, or UCB grafts following a reduced intensity conditioning regimen based on targeted-exposure busulfan, fludarabine +/- serotherapy in patients with inherited metabolic disorders and severe osteopetrosis.

Paul Orchard
All
up to 55 Years old
Phase 2
This study is NOT accepting healthy volunteers
NCT02171104
1406M51542
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Inclusion Criteria:

• 0 through 55 years of age
• Adequate graft available
• Adequate organ function
• Eligible Diseases:
• Mucopolysaccharidosis Disorders:
• MPS IH (Hurler syndrome)
• MPS II (Hunter syndrome) if the patient has no or minimal evidence of symptomatic neurologic disease but is expected to have a neurologic phenotype
• MPS VI (Maroteaux-Lamy syndrome)
• MPS VII (Sly syndrome)
• Glycoprotein Metabolic Disorders:
• Alpha mannosidosis
• Fucosidosis
• Aspartylglucosaminuria
• Sphingolipidoses and Recessive Leukodystrophies:
• Globoid cell leukodystrophy
• Metachromatic leukodystrophy
• Niemann-Pick B patients (sphingomyelin deficiency)
• Niemann-Pick C subtype 2
• Peroxisomal Disorders:
• Adrenoleukodystrophy with cerebral involvement
• Zellweger syndrome
• Neonatal Adrenoleukodystrophy
• Infantile Refsum disease
• Acyl-CoA-Oxidase Deficiency
• D-Bifunctional enzyme deficiency
• Multifunctional enzyme deficiency
• Alpha-methylacyl-CoA Racmase Deficiency (AMACRD)
• Mitochondrial Neurogastrointestingal Encephalopathy (MNGIE)
• Severe Osteopetrosis (OP)
• Hereditary Leukoencephalopathy with axonal spheroids (HDLS; CSF1R mutation)
• Other Inherited Metabolic Disorders (IMD): Patients will also be considered who have other life-threatening, rare lysosomal, peroxisomal or other similar inherited disorders characterized by white matter disease or other neurologic manifestations for which there is rationale that transplantation would be of benefit, such as certain patients with Wolman's disease, GM1 gangliosidosis, I-cell disease, Tay-Sachs disease, Sandhoff disease or others.
• Voluntary written consent
Exclusion Criteria:

• Pregnancy - menstruating females must have a negative serum or urine pregnancy test within 14 days of study treatment start
• Prior myeloablative chemotherapy exposure within 4 months of the start of conditioning on this protocol (patients excluded for this reason may be eligible for other institutional protocols)
• Uncontrolled bacterial, fungal or viral infections including HIV (including active infection with Aspergillus or other mold within 30 days)
Biological: Stem Cell Transplantation, Drug: IMD Preparative Regimen, Drug: Osteopetrosis Only Preparative Regimen, Drug: Osteopetrosis Haploidentical Only Preparative Regimen, Drug: cALD SR-A (Standard-Risk, Regimen A), Drug: cALD SR-B (Standard-Risk, Regimen B), Drug: cALD HR-D (High-Risk, Regimen C), Drug: cALD HR-D (High-Risk, Regimen D)
Mucopolysaccharidosis Disorders, Hurler Syndrome, Hunter Syndrome, Maroteaux Lamy Syndrome, Sly Syndrome, Alpha-Mannosidosis, Fucosidosis, Aspartylglucosaminuria, Glycoprotein Metabolic Disorders, Sphingolipidoses, Recessive Leukodystrophies, Globoid Cell Leukodystrophy, Metachromatic Leukodystrophy, Niemann-Pick B, Niemann-Pick C Subtype 2, Sphingomyelin Deficiency, Peroxisomal Disorders, Adrenoleukodystrophy With Cerebral Involvement, Zellweger Syndrome, Neonatal Adrenoleukodystrophy, Infantile Refsum Disease, Acyl-CoA Oxidase Deficiency, D-Bifunctional Enzyme Deficiency, Multifunctional Enzyme Deficiency, Alpha-methylacyl-CoA Racmase Deficiency, Mitochondrial Neurogastrointestingal Encephalopathy, Severe Osteopetrosis, Hereditary Leukoencephalopathy With Axonal Spheroids (HDLS, CSF1R Mutation), Inherited Metabolic Disorders
Clinics and Surgery Center (CSC), allogeneic hematopoietic cell transplantation, bone marrow transplantation, IMD, AMACRD, MNGIE, HDLS, OP, ALD
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Idiopathic Pulmonary Fibrosis Prospective Outcomes (IPF-PRO) Registry (IPF/ILD-PRO)

Idiopathic Pulmonary Fibrosis Prospective Outcomes (IPF-PRO) Registry is a prospective registry that will collect information regarding the natural history, health care interactions, participant reported questionnaire data to assess quality of life of IPF participants, and the methods of treatment of participants with a diagnosis of idiopathic pulmonary fibrosis (IPF) established at the enrolling centers. In addition, blood samples will be collected and banked for future research projects.

Hyun Kim
All
30 Years and over
N/A
This study is NOT accepting healthy volunteers
NCT01915511
1408M52921
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Inclusion Criteria:

• Willing and able to provide informed consent
• Established a new diagnosis of IPF by the enrolling subspecialty center (as defined by ATS/ERS/JRS/ALAT criteria)
• Age 30 years or older, or
• Diagnosis of a non-IPF ILD of any duration, including, but not limited to Idiopathic Non-Specific Interstitial, Pneumonia (iNSIP), Unclassifiable Idiopathic Interstitial Pneumonias (IIPs), Interstitial Pneumonia with Autoimmune Features (IPAF), Autoimmune ILDs such as Rheumatoid Arthritis (RA-ILD) and Systemic Sclerosis (SSc-ILD), Chronic Hypersensitivity Pneumonitis (HP), Sarcoidosis or Exposure-related ILDs such as asbestosis with progressive phenotype
Exclusion Criteria:

• Malignancy, treated or untreated, other than skin or early stage prostate cancer, within the past 5 years
• Currently listed for lung transplantation at the time of enrollment
• Currently enrolled in a clinical trial at the time of enrollment in this registry
Idiopathic Pulmonary Fibrosis, Interstitial Lung Disease
Idiopathic pulmonary fibrosis, Pulmonary fibrosis, IPF, Registry, 1199.174, Interstitial Lung Disease, ILD, Interstitial Lung Disease with Progressive Phenotype, Clinics and Surgery Center (CSC)
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MT2013-06C : Treatment of graft Failure after HSCT

Troy Lund
All
Not specified
N/A
This study is NOT accepting healthy volunteers
NCT02161783
1404M49341
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Inclusion Criteria:

• Patients with primary or secondary graft failure, as defined below, may receive a second transplant:
• Primary graft failure is defined as not achieving an ANC ≥0.5x10^9/L for three consecutive days by day 35 - 42 following the first transplant.
• Secondary graft failure is defined as achieving an ANC ≥0.5x10^9/L for three consecutive days by day 35 - 42, but subsequently drops below 0.5x10^9/L without recovery.
• Loss of chimerism is defined as achieving an ANC ≥0.5x10^9/L for three consecutive, but with less than 10% CD15+ donor cells in the marrow or peripheral blood.
• Recipients should have acceptable organ function defined as:
• Renal: creatinine < 2.0 (adults) and creatinine clearance > 30. For creatinine clearance < 70, consultation with a BMT pharmacist is necessary for chemotherapy dose adjustments.
• Hepatic: bilirubin, AST/ALT, ALP < 10 x upper limit of normal
• Cardiac: left ventricular ejection fraction > 40%
Exclusion Criteria:

• Uncontrolled infection at the time of transplant.
• Patients with Fanconi Anemia or other DNA breakage syndromes.
Drug: Fludarabine, Drug: Cyclophosphamide, Radiation: Total Body Irradiation, Biological: Hematopoietic stem cell infusion
Primary Graft Failure, Secondary Graft Failure
Hematopoietic stem cell transplant, Clinics and Surgery Center (CSC)
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The Lysosomal Disease Network Longitudinal Study of the Mucopolysaccharidoses

The purpose of this study is to examine the changes in the central nervous system over time in both treated and untreated patients with MPS I, II, IV, VI, and VII in both structure and function (including emotional-social characteristics) through the use of brain MRI, neuropsychological testing, and gathering an updated medical history.

Chester Whitley, MD, PhD
All
6 Years and over
N/A
This study is NOT accepting healthy volunteers
NCT01870375
STUDY00014153
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Inclusion Criteria:

• Any MPS I, II, IV, VI or VII child or adult aged 6 years of age or older
Exclusion Criteria:

• Exclusion Criteria for Neuroimaging:
• Participants with:
• Pacemakers
• Any indwelling electronic device including programmable shunts
• Orthodontic braces unless they are not made of metal
• Other implanted metal in the body other than titanium
• Unable to stay still during MRI because of low cognitive function, behavioral dysregulation, or young age, if the patient is not a clinical patient having sedation/anesthesia
• Pregnancy
• Exclusion Criteria for Neuropsychological and Neurobehavioral Testing
• Participants who:
• Are too functionally impaired for testing
Mucopolysaccharidosis Type I, Mucopolysaccharidosis Type II, Mucopolysaccharidosis Type VI, Mucopolysaccharidosis Type IV, Mucopolysaccharidosis Type VII
Mucopolysaccharidosis, Longitudinal, Brain, Cognition, Quality-of-Life, Hurler syndrome, Hunter syndrome, Hurler-Scheie syndrome, Scheie syndrome, Maroteaux-Lamy syndrome, MPS I, MPS II, MPS VI, Mucopolysaccharidosis type I, Mucopolysaccharidosis type II, Mucopolysaccharidosis type VI, MPS IV, MPS VII, Mucopolysaccharidosis type IV, Mucopolysaccharidosis type VII, Morquio syndrome, Sly syndrome
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MT2013-09C : Umbilical Cord Blood Transplantation Using a Myeloablative Preparative Regimen for the Treatment of Hematological Diseases

This is a treatment protocol for an unrelated umbilical cord blood transplant (UCBT) using a myeloablative preparative regimen for the treatment of hematological diseases, including, but not limited to acute leukemias. There is no research element except the collection of routine clinical data.

Margaret MacMillan, MD
All
up to 55 Years old
N/A
This study is NOT accepting healthy volunteers
NCT01962636
1305M34181
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Inclusion Criteria:

• Eligible Disease Status
• Acute Myeloid Leukemia (AML): high risk CR1 (as evidenced by preceding MDS, high risk cytogenetics, ≥ 2 cycles to obtain CR, erythroblastic or megakaryocytic leukemia; CR2+. All patients must be in CR as defined by hematological recovery, AND <5% blasts by light microscopy within the bone marrow with a cellularity of ≥15%.
• Very high risk pediatric patients with AML: Patients <21 years, however, are eligible with (M2 marrow) with < 25% blasts in marrow after having failed one or more cycles of chemotherapy.
• Acute Lymphocytic Leukemia (ALL): high risk CR1 as defined by cytogenetics (such as t(9;22), t (1:19), t(4;11), other MLL rearrangements, hypodiploidy, or IKZF1 abnormalities), DNA index < 0.81, > 1 cycle to obtain CR or presence minimal residual disease (MRD). Patients in CR2+ are eligible. All patients must be in CR as defined by hematological recovery, AND <5% blasts by light microscopy within the bone marrow with a cellularity of ≥15%.
• Very high risk pediatric patients with ALL: patients <21 years are also considered high risk CR1 if they had M2 or M3 marrow at day 42 from the initiation of induction or M3 marrow at the end of induction. They are eligible once they achieved a complete remission.
• Chronic Myelogenous Leukemia excluding refractory blast crisis: To be eligible in first chronic phase (CP1) patient must have failed or be intolerant to imatinib mesylate.
• Plasma Cell Leukemia after initial therapy, who achieved at least a partial remission
• Advanced Myelofibrosis
• Myelodysplasia (MDS) IPSS INT-2 or High Risk (i.e. RAEB, RAEBt) or Refractory Anemia with severe pancytopenia or high risk cytogenetics: Blasts must be < 10% by a representative bone marrow aspirate morphology.
• Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma (CLL/SLL), Marginal Zone B-Cell Lymphoma or Follicular Lymphoma are eligible if there was disease progression/relapse within 12 of achieving a partial or complete remission. Patients who had remissions lasting > 12 months, are eligible after at least two prior therapies. Patients with bulky disease (nodal mass greater than 5 cm) should be considered for de-bulking chemotherapy before transplant.
• Lymphoplasmacytic Lymphoma, Mantle-Cell Lymphoma, Prolymphocytic Leukemia are eligible after initial therapy in CR1+ or PR1+.
• Large Cell NHL > CR2/> PR2: Patients in CR2/PR2 with initial short remission (<6 months) are eligible.
• Lymphoblastic Lymphoma, Burkitt's Lymphoma, and other high-grade NHL after initial therapy if stage III/IV in CR1/PR1 or after progression if stage I/II < 1 year.
• Multiple Myeloma beyond PR2: Patients with chromosome 13 abnormalities, first response lasting less than 6 months, or β-2 microglobulin > 3 mg/L, may be considered for this protocol after initial therapy.
• Myeloproliferative Syndromes
• Availability of suitable UCB unit(s)
• 0 to 55 years
• Voluntary written consent (adult or parental/guardian)
Exclusion Criteria:

• previous irradiation that precludes the safe administration of TBI - Radiation Oncology will evaluate all patients who have had previous radiation therapy
• chemotherapy refractory large cell and high grade NHL (ie progressive disease after > 2 salvage regimens)
• if ≤ 18 years old, prior myeloablative transplant within the last 6 months. If >18 years old prior myeloablative allotransplant or autologous transplant
• extensive prior therapy including > 12 months alkylator therapy or > 6 months alkylator therapy with extensive radiation
• pregnant or breastfeeding
• HIV positive
Drug: Fludarabine, Drug: Cyclophosphamide, Radiation: Total Body Irradiation, Drug: Cyclosporine A, Drug: Mycophenylate mofetil, Biological: Umbilical cord blood
Acute Myeloid Leukemia (AML), Acute Lymphocytic Leukemia (ALL), Chronic Myelogenous Leukemia, Plasma Cell Leukemia, Myelofibrosis, Myelodysplasia, Chronic Lymphocytic Leukemia, Small Lymphocytic Lymphoma, Marginal Zone B-Cell Lymphoma, Follicular Lymphoma, Lymphoplasmacytic Lymphoma, Mantle-Cell Lymphoma, Prolymphocytic Leukemia, Diffuse Large B Cell Lymphoma, Lymphoblastic Lymphoma, Burkitt's Lymphoma, Non-Hodgkin Lymphoma, Multiple Myeloma
Umbilical Cord Transplant, Acute Myeloid Leukemia (AML), Acute Lymphocytic Leukemia (ALL), Chronic Myelogenous Leukemia, Plasma Cell Leukemia, Myelofibrosis, Myelodysplasia, Chronic Lymphocytic Leukemia, Small Lymphocytic Lymphoma, Marginal Zone B-Cell Lymphoma, Follicular Lymphoma, Lymphoplasmacytic Lymphoma, Mantle-Cell Lymphoma, Prolymphocytic Leukemia, Large Cell Non-Hodgkin Lymphoma, Lymphoblastic Lymphoma, Burkitt's Lymphoma, Non-Hodgkin Lymphoma, Multiple Myeloma, Clinics and Surgery Center (CSC)
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Prospective Global Registry for the Study of Chronic Total Occlusion Intervention (PROGRESS-CTO)

All
Not specified
N/A
This study is NOT accepting healthy volunteers
NCT02061436
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Inclusion Criteria:

• Patients undergoing CTO PCI at each of the participating centers.
Exclusion Criteria:

• None
Coronary Artery Disease
chronic total occlusion, percutaneous coronary intervention
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MT2012-10C: Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies

Christen Ebens
All
up to 50 Years old
N/A
This study is NOT accepting healthy volunteers
NCT01652092
1207M17321
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Inclusion Criteria:

• Diagnosis of immunodeficiency or histiocytic disorder including the following:
• Severe combined immunodeficiency (SCID - all variants)
• Second bone marrow transplant (BMT) for SCID (after graft rejection)
• Omenn's Syndrome
• Reticular dysgenesis
• Wiskott-Aldrich syndrome
• Major histocompatibility complex (MHC) Class II deficiency (bare lymphocyte syndrome)
• Hyper IgM Syndrome (CD40 Ligand Deficiency)
• Common variable immunodeficiency (CVID) with severe phenotype
• Chronic Granulomatous Disease (CGD)
• Other severe Combined Immune Deficiencies (CID)
• Hemophagocytic Lymphohistiocytosis (HLH)
• X-linked Lymphoproliferative Disease (XLP)
• Chediak-Higashi Syndrome (CHS)
• Griscelli Syndrome
• Langerhans Cell Histiocytosis (LCH)
• Acceptable stem cell sources include:
• HLA identical or 1 antigen matched sibling donor eligible to donate bone marrow
• HLA identical or up to a 1 antigen mismatched unrelated BM donor
• Sibling donor cord blood with acceptable HLA match and cell dose as per current institutional standards
• Single unrelated umbilical cord blood unit with 0-2 antigen mismatch and minimum cell dose of >5 x 10^7 nucleated cells/kg as per current institutional guidelines
• Double unrelated umbilical cord blood units that are:
• up to 2 antigen mismatched to the patient
• up to 2 antigen mismatched to each other
• minimum cell dose of at least one single unit must be ≥ 3.5 x 10^7 nucleated cells/kg
• combined dose of both units must provide a total cell dose of ≥ 5 x 10^7 nucleated cells/kg
• Age: 0 to 50 years
• Adequate organ function and performance status. Exclusion Criteria
• pregnant or breastfeeding
• active, uncontrolled infection and/or HIV positive
• acute hepatitis or evidence of moderate or severe portal fibrosis or cirrhosis on biopsy
Drug: Alemtuzumab 0.3 mg, Drug: Cyclophosphamide, Drug: Busulfan, Biological: Stem Cell Transplantation, Drug: Fludarabine phosphate 40 mg, Drug: Melphalan, Drug: Alemtuzumab 0.2 mg, Drug: Busulfan, Drug: Fludarabine phosphate 30 mg, Drug: MESNA
SCID, Omenn's Syndrome, Reticular Dysgenesis, Wiskott-Aldrich Syndrome, Bare Lymphocyte Syndrome, Common Variable Immunodeficiency, Chronic Granulomatous Disease, CD40 Ligand Deficiency, Hyper IgM Syndrome, X-linked Lymphoproliferative Disease, Hemophagocytic Lymphohistiocytosis, Griscelli Syndrome, Chediak-Higashi Syndrome, Langerhan's Cell Histiocytosis
immunodeficiency disorder, histiocytic disorder, Clinics and Surgery Center (CSC)
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Synergistic Enteral Regimen for Treatment of the Gangliosidoses (SYNER-G) (Syner-G)

The Syner-G regimen research study seeks to evaluate the use of a combination of a medication called miglustat and a ketogenic diet for treatment of the gangliosidoses to learn if this combination will provide improved clinical outcomes compared to what we currently know about the natural course of the disease.

Jeanine Jarnes
All
up to 204 Months old
Phase 4
This study is NOT accepting healthy volunteers
NCT02030015
1311M46101
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Inclusion Criteria:

• Subjects must have a documented infantile or juvenile gangliosidosis disease.
• Age: 17 years or less at time of enrollment
• Subjects and their caregivers must be willing to work with a ketogenic diet team for management of the subject's ketogenic diet.
Exclusion Criteria:

• A desire to not participate
• Patients who are older than 17 years will not be enrolled in this study.
• Children with severe renal impairment will not be enrolled in this study.
• Post-pubertal females who are pregnant, or who are unwilling to use highly-effective methods to prevent pregnancy, will be excluded from this study.
• Breast-feeding females will be excluded from this study.
• Subjects who have an allergy to miglustat or any of the components within the drug product will be excluded from this study.
Drug: miglustat, Other: Ketogenic Diet
GM1 Gangliosidoses, GM2 Gangliosidoses, Tay-Sachs Disease, Sandhoff Disease
infantile Tay-Sachs disease, juvenile Tay-Sachs disease, infantile GM1 gangliosidosis, juvenile GM1 gangliosidosis, infantile GM2 gangliosidosis, juvenile GM2 gangliosidosis, Sandhoff disease, gangliosidoses, miglustat, ketogenic diet, SYNER-G regimen, Syner-G, Zavesca, Tay-Sachs disease, Tay Sachs disease
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University of Minnesota Transplant Registry

This registry is open for people of all ages who have either received or donated an organ at the University of Minnesota. This study aims to evaluate organ transplant outcomes among people who have been part of the transplant program.

All
Not specified
N/A
This study is NOT accepting healthy volunteers
NCT01062581
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Inclusion Criteria:

• Received a transplanted organ at the University of Minnesota
• Living donor who donates an organ at the University of Minnesota
Exclusion Criteria:

• Did not receive a transplant at the University of Minnesota
• Did not donate an organ at the University of Minnesota
Transplant Recipient, Transplant Donation
Recipient, Donor, Organ Transplant, Registry, University of Minnesota
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Pediatric Obesity Weight Evaluation Registry (POWER) Study (POWER)

All
up to 18 Years old
N/A
This study is NOT accepting healthy volunteers
NCT02121132
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Inclusion Criteria:

• age 18 years or younger
• overweight or obese patient
• initial medical evaluation in a pediatric weight management program between March 1, 2014-April 30, 2020.
Exclusion Criteria:

• no exclusion criteria
Overweight, Obesity
Overweight, Obesity
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MT2010-09 Primary Immune Deficiency Treatment Consortium (PIDTC). PROJECT 1: A Prospective Natural History Study of Diagnosis, Treatment and Outcomes of Children with SCID Disorders (RDCRN PIDTC #6901)

Christen Ebens
All
Not specified
N/A
This study is NOT accepting healthy volunteers
NCT01186913
1011M93312
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Inclusion Criteria:
Stratum A: Typical SCID (formerly referred to as Classic SCID)- -Subjects who meet the following inclusion criteria and the intention is to treat with allogeneic hematopoietic cell transplant (HCT) are eligible for enrollment into Stratum A (Typical SCID) of the study:
• Absence or very low number of T cells (CD3 T cells <300/microliter) AND
• No or very low T cell function (<10% of lower limit of normal) as measured by response to phytohemagglutinin (PHA) OR
• T cells of maternal origin present. Stratum B: Leaky SCID, Omenn Syndrome, Reticular Dysgenesis- -Subjects who meet the following criteria and the intention is to treat with HCT are eligible for enrollment into Stratum B: Leaky SCID:
• Maternal lymphocytes tested for and not detected AND
• Either one or both of the following (a,b) :
• a.) <50% of lower limit of normal T cell function as measured by response to PHA, OR response to anti-CD3/CD28 antibody
• b.) Absent or <30% of lower limit of normal proliferative responses to candida and tetanus toxoid antigens
• AND at least two of the following (a through e):
• a.) Reduced number of CD3 T cells
• age ≤2 years: <1500/microliter
• age >2 years and ≤4 years: <800/microliter
• age >4 years: <600/microliter
• b.) ≥80% of CD3+ or CD4+ T cells that are CD45RO+
• AND/OR >80% of CD3+ or CD4+ T cells are CD62L negative
• AND/OR >50% of CD3+ or CD4+T cells express HLA-DR (at <4 years of age)
• AND/OR are oligoclonal T cells
• c.) Hypomorphic mutation in IL2RG in a male, or homozygous hypomorphic mutation or compound heterozygosity with ≥1 hypomorphic mutation in an autosomal SCID-causing gene
• d.) Low T Cell Receptor Excision Circles (TRECs) and/or the percentage of CD4+/45RA+/CD31+ or CD4+/45RA+/CD62L+ cells is below the lower limit of normal.
• e.) Functional testing in vitro supporting impaired, but not absent, activity of the mutant protein, AND
• Does not meet criteria for Omenn Syndrome. Omenn Syndrome:
• Generalized skin rash
• Maternal lymphocytes tested for and not detected; --Note: If maternal engraftment was not assessed and ruled out, the subject is not eligible as Omenn Syndrome.
• ≥80% of CD3+ or CD4+ T cells are CD45RO+ AND/OR
• 80% of CD3+ or CD4+T cells are CD62L negative AND/OR
• 50% of CD3+ or CD4+ T cells express HLA-DR (at <2 years of age);
• Absent or low (< 30% lower limit of normal) T cell proliferation response to antigens (Candida, tetanus) to which the subject has been exposed NOTE: If proliferation to antigen was not performed, but at least 4 of the following 9 supportive criteria, at least one of which must be among those marked with an asterisk (*) below are present, the subject is eligible as Omenn Syndrome:
• Hepatomegaly
• Splenomegaly
• Lymphadenopathy
• Elevated IgE
• Elevated absolute eosinophil count
• *Oligoclonal T cells measured by CDR3 length or flow cytometry
• *Proliferation to PHA is reduced <50% of lower limit of normal or SI <30
• *Hypomorphic mutation in a SCID causing gene
• Low TRECS and/or the percentage of CD4+/45RA+/CD31+ or CD4+/45RA+/CD62L+ cells is below the lower limit of normal. Reticular Dysgenesis:
• Absence or very low number of T cells (CD3 <300/µL
• No or very low (<10% lower limit of normal) T cell response to PHA
• Severe neutropenia (absolute neutrophil count < 200 /µL) AND
• ≥2 of the following (a,b,c):
• a.) Sensori-neural deafness
• b.) Deficiency of marrow granulopoiesis on bone marrow examination
• c.) A pathogenic mutation in the adenylate kinase 2 (AK2) gene identified. Stratum C: Subjects who meet the following criteria and the intention is to treat with therapy other than allogeneic HCT, primarily PEG-ADA ERT or gene therapy with autologous modified (gene transduced) cells, are eligible for enrollment into Stratum C:
• ADA Deficient SCID with intention to treat with PEG-ADA ERT
• ADA Deficient SCID with intention to treat with gene therapy
• X-linked SCID with intention to treat with gene therapy
• Any SCID patient previously treated with a thymus transplant (includes intention to treat with HCT, as well as PEG-ADA ERT or gene therapy)
• Any SCID patient who received therapy for SCID deemed "non-standard" or "investigational", including in utero procedures.
Exclusion Criteria:
-Subjects who meet any of the following exclusion criteria are disqualified from enrollment in Strata A, B, or C of the study:
• Presence of an Human Immunodeficiency Virus (HIV) infection (by PCR) or other cause of secondary immunodeficiency
• Presence of DiGeorge syndrome
• MHC Class I and MHC Class II antigen deficiency, and
• Metabolic conditions that imitate SCID or related disorders such as folate transporter deficiency, severe zinc deficiency or transcobalamin deficiency.
Severe Combined Immunodeficiency (SCID), Leaky SCID, Omenn Syndrome, Reticular Dysgenesis, ADA SCID, XSCID
Severe Combined Immunodeficiency (SCID), natural history study, SCID treatment
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MT2013-34C: Hematopoietic Stem Cell Transplant for Dyskeratosis Congenita or Severe Aplastic Anemia: Standard of Care Considerations

Christen Ebens
All
up to 70 Years old
N/A
This study is NOT accepting healthy volunteers
NCT02162420
1404M50183
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Inclusion Criteria:

• Aged 0 - 70 years
• Acceptable hematopoeitic stem cell donor
• Dyskeratosis Congenita (DC) with evidence of BM failure defined as:
• requirement for red blood cell and/or platelet transfusions or
• requirement for G-CSF or GM-CSF or erythropoietin or
• refractory cytopenias having one of the following three
• platelets <50,000/uL or transfusion dependent
• absolute neutrophil count <500/uL without hematopoietic growth factor support
• hemoglobin <9g/uL or transfusion dependent
• Diagnosis of DC with a triad of mucocutaneous features:
• oral leukoplakia
• nail dystrophy
• abnormal reticular skin hyperpigmentation, or
• Diagnosis of DC with one of the following:
• short telomeres (under a research study)
• mutation in telomerase holoenzyme (DKC1, TERT, TERC, NOP10, NHP2, TCAB1)
• mutation in shelterin complex (TINF2)
• mutation in telomere-capping complex (CTC1)
• Severe Aplastic Anemia (SAA) primary transplant with evidence of BM failure:
• Refractory cytopenia defined by bone marrow cellularity <50% (with < 30% residual hematopoietic cells)
• Diagnosis of SAA with refractory cytopenias having one of the following three:
• platelets <20,000/uL or transfusion dependent
• absolute neutrophil count <500/uL without hematopoietic growth factor support
• absolute reticulocyte count <20,000/uL
• Severe Aplastic Anemia (SAA) requiring a 2nd transplant
• Graft failure as defined by blood/marrow chimerism of < 5%
• Early myelodysplastic features
• With or without clonal cytogenetic abnormalities
• Adequate organ function defined as:
• cardiac: left ventricular ejection fraction ≥ 35% with no evidence of decompensated heart failure
• pulmonary: DLCO ≥30% predicted, no supplemental oxygen requirement
• renal: Glomerular filtration rate (GFR) ≥30% predicted
• Voluntary written consent
Exclusion Criteria:

• Acute hepatitis or evidence of moderate or severe portal fibrosis or cirrhosis on biopsy
• Pregnant or lactating
• Uncontrolled infection
• Prior radiation therapy (applies to SAA patients only)
• Diagnosis of Fanconi anemia based on DEB
• Diagnosis of dyskeratosis congenita with advanced MDS or acute myeloid leukemia with >30% blasts
Drug: Alemtuzumab, Drug: Fludarabine, Drug: Cyclophosphamide, Radiation: Total Body Irradiation, Biological: Stem Cell Transplant, Drug: Anti-thymocyte globulin
Dyskeratosis Congenita, Aplastic Anemia
Clinics and Surgery Center (CSC), severe aplastic anemia, Hematopoietic Stem Cell Transplant
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COG ANBL00B1 - Neuroblastoma Biology Studies

This research trial studies biomarkers in tumor tissue samples from patients with newly diagnosed neuroblastoma or ganglio-neuroblastoma. Studying samples of tumor tissue from patients with cancer in the laboratory may help doctors identify and learn more about biomarkers related to cancer.

Emily Greengard
All
up to 30 Years old
N/A
This study is NOT accepting healthy volunteers
NCT00904241
0807M39682
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Inclusion Criteria:

• All newly diagnosed patients with suspected neuroblastoma, suspected ganglioneuroblastoma, or suspected ganglioneuroma/maturing subtype seen at Children's Oncology Group (COG) institutions are eligible for this study
• There will be no penalty under any circumstances for enrollment of a patient whose definitive institutional diagnosis, or central review diagnosis, is found to be a tumor other than neuroblastoma, ganglioneuroblastoma, or ganglioneuroma/ maturing subtype
• Patients may not have received chemotherapy prior to enrollment on ANBL00B1 and procurement of study-related tissues with the following exception:
• Patients that in the opinion of the treating physician are too ill to undergo pre-treatment tissue biopsy and require EMERGENT chemotherapy may be enrolled on ANBL00B1; documentation of the emergent nature of therapy initiation is required
• It is required that a good faith effort (documented by specimen tracking) be made to submit a neuroblastoma sample (tumor, metastasis, and/or tumor-involved bone marrow) of sufficient quality for MYCN analysis in the Neuroblastoma Reference Laboratory in order for any newly diagnosed patient to be enrolled on ANBL00B1; this should be obtained prior to initiation of therapy
• Exceptions
• In rare cases, patients may be deemed too ill to undergo pre-treatment tissue biopsy and require EMERGENT therapy; the following eligibility guidelines apply to these cases:
• For presumed INSS stage 4S patients: Efforts to submit tumor tissue (e.g., primary tumor, skin nodule, or metastatic site) within 96 hours of EMERGENT therapy initiation should be made; however, if the child is deemed too unstable for such a procedure they may still be enrolled as long as pre-treatment peripheral blood and serum have been submitted
• For all other INSS stages: tumor tissue should be obtained as soon as possible within 96 hours of EMERGENT therapy initiation; patients without tumor tissues submitted within this time-frame are not eligible for enrollment
• Note: it may not be possible to obtain all necessary tumor biomarkers for therapy stratification in such cases; if a patient enrolled on ANBL00B1 undergoes an additional diagnostic procedure within 96 hours of initiating therapy, additional tumor specimens may be submitted to obtain biomarkers used for risk classification; the decision to perform such procedures, and/or submit these specimens, is to be made by the managing clinicians and should reflect the clinical need to know the status of such biomarkers
• Patients enrolled on ANBL1232 in Group A (either A1 or A2) will not have a tumor biopsy or resection upfront; tumor tissue submission is therefore not required for these patients to enroll on ANBL00B1; a peripheral blood and serum sample is the only specimen required to be submitted for this group of patients; should they undergo a biopsy or resection at a later date tumor can be submitted for biomarker testing at this time
• All patients and/or their parents or legal guardians must sign a written informed consent
• All institutional, Food and Drug Administration (FDA), and National Cancer Institute (NCI) requirements for human studies must be met
Exclusion Criteria:

• Patients with relapsed neuroblastoma who were not enrolled on ANBL00B1 at original diagnosis are NOT eligible; samples should be submitted as part of the ABTR04B1 protocol
Other: Cytology Specimen Collection Procedure, Other: Laboratory Biomarker Analysis
Ganglioneuroblastoma, Localized Resectable Neuroblastoma, Localized Unresectable Neuroblastoma, Regional Neuroblastoma, Stage 4 Neuroblastoma, Stage 4S Neuroblastoma
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MT2011-11C: High Dose Chemotherapy and Autologous Peripheral Blood Stem Cell (PBSC) Rescue for Neuroblastoma

This therapy involves the use of consolidation chemotherapy, autologous stem cell rescue, post-transplant radiation therapy and a maintenance phase with Isotretinoin (Accutane, 13-cis-retinoic acid) therapy. If available, patients should also consider post-transplant therapy with cytokines and monoclonal antibody (ch14.18) on a COG or NANT trial. Ideally, patients should begin consolidation chemotherapy no later than 8 weeks after the start of Induction Cycle #6. (It is strongly recommended to begin consolidation within 4-6 weeks after starting Induction Cycle #6).

Ashish Gupta
All
up to 30 Years old
N/A
This study is NOT accepting healthy volunteers
NCT01526603
1112M07741
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Inclusion Criteria:

• Less than 30 years of age at diagnosis of neuroblastoma
• No evidence of disease progression: defined as increase in tumor size of >25% or new lesions
• Recovery from last induction course of chemotherapy (absolute neutrophil count > 500 and platelet > 20,000)
• No uncontrolled infection
• Minimum frozen peripheral blood stem cells (PBSCs) of 2 x 10^6 CD34 cells/kg for transplant are mandatory and 2 x 10^6 CD34 cells/kg for back-up are strongly recommended (thus, PBSC of 4 x 106 CD34 cells/kg is encouraged)
• Adequate organ function defined as:
• Hepatic: aspartate aminotransferase (AST) < 3 x upper limit of institutional normal 8 Cardiac: shortening fraction ≥ 27% or ejection fraction ≥ 50%, no clinical congestive heart failure 8 Renal: Creatinine clearance or glomerular filtration rate (GFR) > 60 mL/min/1.73m^2 If a creatinine clearance is performed at end induction and the result is < 100 ml/min/1.73m^2, a GFR must then be performed using a nuclear blood sampling method or iothalamate clearance method. Camera method is NOT allowed as measure of GFR prior to or during Consolidation therapy for patients with GFR or creatinine clearance of < 100 ml/min/1.73m^2 Exclusion Criteria
• Patients with progressive disease should consider participating in phase I studies since consolidation therapy using the regimen outlined in this document have not been determined to be useful.
• Patients who are delayed in consolidation chemotherapy beyond 8 weeks, and don't meet organ function criteria.
Drug: Carboplatin, Biological: Autologous stem cell infusion, Biological: Granulocyte colony stimulating factor, Radiation: Radiation therapy, Drug: Isotretinoin (13-cis-retinoic acid), Drug: Melphalan, Drug: Etoposide
Neuroblastoma
peripheral blood stem cell transplantation, autologous stem cell transplant
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Mapping Chemical and Microbiological Heterogeneity Throughout Explanted Lung Specimens

We propose to study explanted tissue of patients that are scheduled to undergo single or double lung transplant surgery as a late-stage disease therapeutic strategy. The primary endpoint will be to characterize the composition of the bacterial community in the lung. The secondary endpoint will be to describe bacterial gene expression in the lower airways.

Ryan Hunter
All
18 Years and over
N/A
This study is NOT accepting healthy volunteers
NCT02128711
1404M49426
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Inclusion Criteria:

• diagnosis of cystic fibrosis
• eligible for lung transplantation
• exhausted other available therapies without success
• informed consent
Exclusion Criteria:

• there are no exclusion criteria
Cystic Fibrosis
lung, cystic fibrosis, bacteria, positive diagnosis of CF,, Clinics and Surgery Center (CSC)
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The TrialNet Natural History Study of the Development of Type 1 Diabetes

Antoinette Moran
All
30 Months to 45 Years old
N/A
This study is NOT accepting healthy volunteers
NCT00097292
0305M47349
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Inclusion Criteria:

• Individuals 2.5 to 45 years old who have an immediate family member with type 1 diabetes (such as a child, parent, or sibling)
• Individuals 2.5-20 years old who have an extended family member with type 1 diabetes (such as a cousin, niece, nephew, aunt, uncle, grandparent, or half-sibling)
• Individuals 2.5-45 years old without a type 1 diabetes proband, who are known to have 1 or more islet antibody are eligible for screening if needed to determine eligibility for a clinical trial to delay or prevent disease progression.
Exclusion Criteria:
To be eligible a person must not:
• Have diabetes already
• Have a previous history of being treated with insulin or oral diabetes medications.
• Currently be using systemic immunosuppressive agents (topical and inhaled agents are acceptable)
• Have any known serious diseases
Diabetes Mellitus, Type 1
"at risk" for developing type 1 diabetes, T1DM, T1D, juvenile diabetes, Type 1 Diabetes TrialNet, TrialNet
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COG AREN03B2: Renal Tumors Classification, Biology and Banking Study.

• To classify patients (< 30 years old) with renal tumors by histological categorization, surgico-pathological stage, presence of metastases, age at diagnosis, tumor weight and loss of heterozygosity for chromosomes 1p and 16q, to thereby define eligibility for a series of therapeutic studies. • To maintain a biological samples bank to make specimens available to scientists to evaluate additional potential biological prognostic variables and for the conduct of other research by scientists.

Emily Greengard
All
up to 29 Years old
N/A
This study is NOT accepting healthy volunteers
NCT00898365
0708M15261
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Inclusion Criteria:

• Patients with the first occurrence of any tumor of the kidney identified on CT scan or MRI are eligible for this study; histologic diagnosis is not required prior to enrollment but is required for all patients once on study
• Eligible tumors include (but are not limited to):
• Nephroblastic tumors
• Nephroblastoma (Wilms' tumor) (favorable histology, anaplasia [diffuse, focal])
• Nephrogenic rests and nephroblastomatosis
• Cystic nephroma and cystic partially differentiated nephroblastoma
• Metanephric tumors (metanephric adenoma, metanephric adenofibroma, metanephric stromal tumor)
• Mesoblastic nephroma (cellular, classic, mixed)
• Clear cell sarcoma
• Rhabdoid tumor (any malignant rhabdoid tumor occurring outside the central nervous system [CNS])
• Renal epithelioid tumors of childhood (papillary renal cell carcinoma, medullary renal cell carcinoma, renal tumors associated with Xp11.2 translocations, oncocytic renal neoplasms after neuroblastoma)
• Angiolipoma
• Ossifying renal tumor of infancy
• Patients with the first occurrence of the following tumors are also eligible:
• Extrarenal nephroblastoma or extrarenal neprogenic rests
• Malignant rhabdoid tumor occurring anywhere outside the central nervous system
• Required specimens, reports, forms, and copies of imaging studies must be available or will become available for submission and the institution must intend on submitting them as described in the protocol procedures
• For ALL patients, (with exception of bilateral, bilaterally predisposed, multicentric, or unilateral tumor in solitary kidney planning to enroll without biopsy***), the following submissions are required:
• A complete set of recut hematoxylin and eosin (H & E) slides (including from sampled lymph nodes, if patient had upfront nephrectomy)
• * Tissue must be from diagnosis, prior to any renal tumor directed chemotherapy or radiation (only exception is for presumed favorable histology Wilms tumor [FHWT] patients discovered to have diffuse anaplastic Wilms tumor [DAWT] at delayed nephrectomy and plan to enroll at delayed nephrectomy)
• Representative formalin-fixed paraffin-embedded tissue block or if a block is unavailable, 10 unstained slides from a representative block of tumor, if available.
• Tissue must be from diagnosis, prior to any renal tumor directed chemotherapy or radiation (only exception is for presumed FHWT patients discovered to have DAWT at delayed nephrectomy and plan to enroll at delayed nephrectomy)
• Institutional pathology report, Specimen Transmittal Form, and Pre-Treatment Pathology Checklist
• Copies of images and institutional reports of CT and/or MRI abdomen and pelvis, and Pre Treatment Imaging Checklist
• Copies of images and institutional report of chest CT for all malignant tumors
• Institutional surgical report(s) and Pre-Treatment Surgical Checklist
• CRFs: Staging Checklist and Metastatic Disease Form (if metastatic disease is noted on imaging)
• Patients with bilateral, bilaterally predisposed, multicentric, or unilateral tumor in solitary kidney planning to enroll without biopsy via imaging only - these patients will not have central review or have a risk assignment issued, but may contribute to specimen banking for future research. However, if biopsy is done, tissue must be submitted as for other renal tumors, and initial risk assignment will require pathology and surgical rapid central reviews. The Specimen Transmittal Form and Pre Treatment Pathology Checklist are also needed.
• Please note: if the above required items are not received within 120 days of study enrollment, the patient will be considered off study
• All patients and/or their parents or legal guardians must sign a written informed consent
• All institutional, Food and Drug Administration (FDA), and National Cancer Institute (NCI) requirements for human studies must be met
Other: Cytology Specimen Collection Procedure, Other: Laboratory Biomarker Analysis
Adult Cystic Nephroma, Anaplastic Kidney Wilms Tumor, Angiolipoma, Cellular Congenital Mesoblastic Nephroma, Classic Congenital Mesoblastic Nephroma, Clear Cell Sarcoma of the Kidney, Congenital Mesoblastic Nephroma, Cystic Partially Differentiated Kidney Nephroblastoma, Diffuse Hyperplastic Perilobar Nephroblastomatosis, Extrarenal Rhabdoid Tumor, Kidney Medullary Carcinoma, Kidney Neoplasm, Kidney Oncocytoma, Kidney Wilms Tumor, Metanephric Adenofibroma, Metanephric Adenoma, Metanephric Stromal Tumor, Metanephric Tumor, Mixed Congenital Mesoblastic Nephroma, Ossifying Renal Tumor of Infancy, Papillary Renal Cell Carcinoma, Renal Cell Carcinoma, Renal Cell Carcinoma Associated With Xp11.2 Translocations/TFE3 Gene Fusions, Rhabdoid Tumor of the Kidney, Wilms Tumor
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A Natural History Study of the Gangliosidoses

This study's primary aims are to define and characterize disease progression for the infantile and juvenile forms of the gangliosidoses, and the late-onset forms of gangliosidosis, including their heterogeneity; and to observe treatment outcomes for any treatments tried. The secondary aims of this study are to understand the neurological involvement in late-onset gangliosidosis; and to collect data on disease progression that can be used for creation of an objective disease stage and severity index.

Jeanine Jarnes
All
Not specified
N/A
This study is NOT accepting healthy volunteers
NCT00668187
1007M85712
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Inclusion Criteria:

• Subjects must have a documented gangliosidosis disease.
• Subjects must be able to complete appropriate neuropsychological and neurobehavioral assessments.
• Late-onset gangliosidosis subjects must be able to tolerate a head MRI.
Exclusion Criteria:

• There are no exclusion criteria, beyond a desire not to participate.
Tay-Sachs Disease, Sandhoff Disease, Late Onset Tay-Sachs Disease, GM1 Gangliosidosis, GM2 Gangliosidosis
Tay-Sachs disease, Sandhoff disease, Late Onset Tay-Sachs disease, LOTS, hexosaminidase A deficiency, hexosaminidase A and B deficiency, infantile Tay-Sachs disease, adult-onset Tay-Sachs disease, prospective, natural history, GM1 gangliosidosis, gangliosidoses, β-galactosidase, β-galactosidase deficiency, hexosaminidase, hexosaminidase deficiency, Tay-Sachs, Sandhoff, juvenile Tay-Sachs, juvenile Tay-Sachs disease, late onset Tay-Sachs, juvenile Sandhoff, juvenile Sandhoff disease, GM2 gangliosidosis
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Longitudinal study of bone and endocrine disease in children with MPS I, II, and VI

This multi-centered, longitudinal study of male and female participants with MPS I, II, and VI has an overall objective to document the progression of skeletal disease and identify biomarkers through the use of bone imaging, range of motion tests, and biomarker analysis that either predict disease severity or could be used as therapeutic targets.

Brad Miller, MD, PhD
All
5 Years to 35 Years old
N/A
This study is NOT accepting healthy volunteers
NCT01521429
0906M68810
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Inclusion Criteria:

• Diagnosis of MPS I, II, or VI
• Ability to travel to study center for evaluations.
• Age ≥ 5 years and < 35 years: age at entry into study must be ≥5 years and ≤33 years to ensure a minimum of 2 study visits.
Exclusion Criteria:

• Pregnancy (will be determined at each study visit)
• Participation in any other study within the past 12 months which would result in increasing the child's radiation exposure above 500 mrem for the calendar year.
• Participants who cannot comply with study procedures or have other factors that would inhibit their participation as determined by the PI's discretion.
Mucopolysaccharidoses
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MT2011-09C Alkylator-Intense Conditioning Followed By Autologous Transplantation for Patients with High Risk or Relapsed Solid or CNS Tumors

Treatment guidelines for high risk or relapsed solid tumors consisting of a busulfan, melphalan, thiotepa conditioning followed by an autologous peripheral blood stem cell transplant and, if appropriate, disease specific radiation therapy at day 60+

Ashish Gupta
All
up to 70 Years old
N/A
This study is NOT accepting healthy volunteers
NCT01505569
1107M02641
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Inclusion Criteria:
All patients must have histological verification of malignancy at original diagnosis.
• Eligible Diseases
• Arm A: Solid Tumor
• Ewing's Family Tumors (ES/PNET/DSRCT) - metastatic at time of diagnosis and/or relapsed after therapy
• Renal Tumors - relapsed (all histology - Wilm's tumor) or at diagnosis (clear cell sarcoma and Rhabdoid tumor)
• Hepatoblastoma - metastatic at time of diagnosis and/or relapsed after therapy
• Rhabdomyosarcoma - metastatic at time of diagnosis and/or relapsed after therapy
• Soft Tissue Sarcoma - chemotherapy responsive metastatic disease or chemotherapy responsive relapsed disease
• Primary Malignant Brain Neoplasms <18 years of age - at diagnosis and/or relapse
• Retinoblastoma - disseminated at diagnosis and/or relapsed
• CNS Lymphoma - primary or secondary CNS lymphoma.
• Other High Risk Metastatic or Relapsed Solid Tumors - to be approved by 2 or more pediatric hematology/oncology and bone marrow transplant (BMT) physicians
• Arm B: Certain CNS tumors
• Medulloblastoma: Children less than 36 months (3 years) of age at time of definitive surgery (for histopathologic diagnosis) who have high risk Medulloblastoma, defined as any one of the following:
• > 1.5 cm2 residual disease following resection for any Medulloblastoma histology
• lumbar CSF cytology positive for tumor cells by analysis of fluid collected either before definitive surgery or at least 10 days after definitive surgery
• MRI evidence of (a) gross nodular seeding in the intracranial subarachnoid space or ventricular system distant from primary tumor site, M2; or (b) gross nodular seeding in the spinal subarachnoid space +/- evidence of intracranial seeding, M3; or (c) extraneural metastases, M4,
• Anaplastic Histologic Variant Medulloblastoma: less than 70 years of age, any metastatic stage, with total or sub-total resection.
• Infant Medulloblastoma: Children less than 8 months of age at the time of definitive surgery (for histopathologic diagnosis), any histology, any metastatic state, with total or sub-total resection.
• Supra-tentorial Primative Neuro-Ectodermal Tumor (PNET): Children less than 36 months (3 years) of age at time of definitive surgery (for histopathologic diagnosis) with or without metastatic disease
• Atypical Teratoid/Rhabdoid Tumor (AT/RT): less than 70 years of age with CNS AT/RT (with or without metastatic disease).
• Other High Risk CNS Tumors - to be approved by 2 or more physicians (at least one oncologist and one BMT physician).
• Arm C: Germ Cell Tumors
• Confirmation of germ cell tumor (GCT) histology (both seminoma and nonseminoma). Tumor may have originated in any primary site. NOTE: In rare circumstances, patients will be allowed to enroll even if a pathologic diagnosis may not have been established. This would require a clinical situation consistent with the diagnosis of GCT (testicular, peritoneal, retroperitoneal or mediastinal mass, elevated tumor marker levels {HCG ≥ 500; AFP ≥ 500} and typical pattern of metastases).
• One or more unfavorable prognostic features for achieving a CR with conventional-dose chemotherapy. Unfavorable prognostic features include:
• extragonadal primary site
• PD following an incomplete response (IR) to first-line therapy,
• PD after a conventional-dose salvage (cisplatin + ifosfamide -based) regimen
• Arm D: Certain CNS Tumor patients who can only undergo one transplant
• Medulloblastoma: Children less than 36 months (3 years) of age at time of definitive surgery (for histopathologic diagnosis) who have high risk Medulloblastoma, defined as any one of the following:
• > 1.5 cm2 residual disease following resection for any Medulloblastoma histology
• lumbar CSF cytology positive for tumor cells by analysis of fluid collected either before definitive surgery or at least 10 days after definitive surgery
• MRI evidence of (a) gross nodular seeding in the intracranial subarachnoid space or ventricular system distant from primary tumor site, M2; or (b) gross nodular seeding in the spinal subarachnoid space +/- evidence of intracranial seeding, M3; or (c) extraneural metastases, M4,
• Anaplastic Histologic Variant Medulloblastoma: less than 70 years of age, any metastatic stage, with total or sub-total resection.
• Infant Medulloblastoma: Children less than 8 months of age at the time of definitive surgery (for histopathologic diagnosis), any histology, any metastatic state, with total or sub-total resection.
• Supra-tentorial Primative Neuro-Ectodermal Tumor (PNET): Children less than 36 months (3 years) of age at time of definitive surgery (for histopathologic diagnosis) with or without metastatic disease
• Atypical Teratoid/Rhabdoid Tumor (AT/RT): less than 70 years of age with CNS AT/RT (with or without metastatic disease).
• Other High Risk CNS Tumors including choroid plexus carcinoma in children- to be approved by 2 or more physicians (at least one oncologist and one BMT physician).
• Arm E: Neuroblastoma ** Neuroblastoma (ICD-O morphology 9500/3) or ganglioneuroblastoma (nodular or intermixed) verified by histology or demonstration of clumps of tumor cells in bone marrow with elevated urinary catecholamine metabolites.
• Disease Status at Enrollment
• Arm A, Arm B and Arm D must have fit one of the following:
• no evidence of disease or
• stable, non-progressive disease (defined as non-progressive abnormalities on physical exam or CT and/or MRI) within 4 weeks of study entry
• Arm C: Evidence of progressive or recurrent GCT (measurable or non-measurable) following one or more cisplatin-based chemotherapy, defined as meeting at least one of the following criteria:
• Tumor biopsy of new or growing or unresectable lesions demonstrating viable non-teratomatous GCT. Patients with incomplete gross resection where viable GCT is found are considered eligible.
• Consecutive elevated serum tumor markers (HCG or AFP) that are increasing. Increase of an elevated LDH alone does not constitute progressive disease.
• Development of new or enlarging lesions in the setting of persistently elevated HCG or AFP, even if the HCG and AFP are not continuing to increase.
• Arm E: Patients with the following disease stages at diagnosis are eligible, if they meet the other specified criteria.
• Patients with newly diagnosed neuroblastoma with INSS Stage 4 are eligible with the following:
• MYCN amplification (> 4-fold increase in MYCN signals as compared to reference signals), regardless of age or additional biologic features or
• Age > 18 months (> 547 days) regardless of biologic features or
• Age 12-18 months (365-547 days) with any of the following 3 unfavorable biologic features (MYCN amplification, unfavorable pathology and/or DNA index = 1) or any biologic feature that is indeterminate/unsatisfactory/unknown.
• Patients with newly diagnosed neuroblastoma with INSS Stage 3 are eligible with the following:
• MYCN amplification (> 4-fold increase in MYCN signals as compared to reference signals), regardless of age or additional biologic features or
• Age > 18 months (> 547 days) with unfavorable pathology, regardless of MYCN status.
• Patients with newly diagnosed neuroblastoma with INSS Stage 2A/2B with MYCN amplification (> 4-fold increase in MYCN signals as compared to reference signals), regardless of age or additional biologic features.
• Patients with newly diagnosed neuroblastoma with INSS Stage 4S with MYCN amplification (> 4-fold increase in MYCN expression signals as compared to reference signals), regardless of additional biologic features.
• Patients ≥ 365 days initially diagnosed with neuroblastoma INSS Stage 1, 2, 4S who progressed to aStage 4 without interval chemotherapy.
• Age and Performance Status
• Age and Performance Status, Arm A
• Age: 0 - 70 years
• Performance status: Karnofsky Performance Status ≥ 50% for patients > 16 years of age or Lansky Play Score ≥ 50 for patients ≤ 16 years of age (Note: Neurologic deficits in patients with CNS tumors must be stable for a minimum of 1 week prior to study entry)
• Age and Performance Status, Arm B
• Age: see Eligible diseases, section 3.1, for age criteria
• Performance status: Karnofsky Performance Status ≥ 50% for patients > 16 years of age or Lansky Play Score ≥ 50 for patients ≤ 16 years of age (Note: Neurologic deficits in patients with CNS tumors must be stable for a minimum of 1 week prior to study entry)
• Age and Performance Status, Arm C
• Age: 0-70 years of age
• Performance status: Karnofsky Performance Status ≥ 70% for patients > 16 years of age or Lansky Play Score ≥ 70 for patients ≤ 16 years of age
• Age and Performance Status, Arm D
• Age: see Eligible diseases, section 3.1, for age criteria
• Performance status: Karnofsky Performance Status ≥ 50% for patients > 16 years of age or Lansky Play Score ≥ 50 for patients ≤ 16 years of age (Neurologic deficits in patients with CNS tumors must be stable for a minimum of 1 week prior to study entry)
• Age and Performance Status, Arm E
• Age: Patients must be ≤ 30 years of age at the time of initial diagnosis.
• Performance status: Karnofsky Performance Status ≥ 50% for patients > 16 years of age or Lansky Play Score ≥ 50 for patients ≤ 16 years of age
• Organ Function
• Organ Function, Arm A
• Hematologic: hemoglobin of >9 gm/dl and platelet count > 20,000/μl. Patients may receive transfusions as necessary.
• Renal: GFR ≥ 50 ml/min/1.73m2 or serum creatinine ≤ 2.5 x ULN for age
• Hepatic: AST or ALT ≤ 5 x ULN and bilirubin ≤ 5 x ULN
• Cardiac: ejection fraction ≥ 45% or no clinical evidence of heart failure
• Pulmonary: oxygen saturation > 92% at rest (on room air)
• Organ Function, Arm B (to begin first consolidation cycle)
• Timing: patients must be fully recovered from radiation, induction chemotherapy or surgery prior to receiving consolidation, with minimum elapsed time of 2 weeks.
• Hematologic: ANC > 750/μl, hemoglobin of >8 gm/dl (may receive PRBC transfusions) and platelet count > 75,000/μl (transfusion independent).
• Renal: GFR ≥ 50 ml/min/1.73m2
• Hepatic: AST or ALT ≤ 2.5 x ULN and bilirubin ≤ 1.5 x ULN
• Cardiac: ejection fraction ≥ 45% or no clinical evidence of heart failure
• Pulmonary: oxygen saturation > 94% at rest (on room air)
• Central Nervous System: patients with seizure history are allowed if on anti-convulsants and well controlled; patients must not be in status epilepticus, coma or require assisted ventilation
• Organ Function, Arm C (to begin TI chemotherapy)
• Hematologic: ANC ≥ 750/mm3, platelets ≥ 75,000/mm3
• Renal: GFR ≥ 50 ml/min/1.73m2 or serum creatinine ≤ 2.5 x ULN for age
• Hepatic: AST or ALT ≤ 2.5 x upper limits of normal (ULN), if hepatic involvement < 5 x ULN; bilirubin ≤ 2.0 x upper limits of normal (ULN)
• Arms A and C: Patients with a history of CNS tumor involvement are eligible if they have completed treatment for CNS disease (radiotherapy or surgery or chemotherapy), have recovered from or stabilization of the side effects associated with the therapy and have no evidence of progressive CNS disease at the time of enrollment
• Organ Function, Arm D
• Timing: patients must be fully recovered from radiation, induction chemotherapy or surgery prior to receiving consolidation, with minimum elapsed time of 2 weeks.
• Hematologic: ANC > 750/μl, hemoglobin of >8 gm/dl (may receive PRBC transfusions) and platelet count > 75,000/μl (transfusion independent).
• Renal: GFR ≥ 50 ml/min/1.73m2
• Hepatic: AST or ALT ≤ 2.5 x ULN and bilirubin ≤ 1.5 x ULN
• Cardiac: ejection fraction ≥ 45% or no clinical evidence of heart failure
• Pulmonary: oxygen saturation > 92% at rest (on room air)
• Central Nervous System: patients with seizure history are allowed if on anti-convulsants and well controlled; patients must not be in status epilepticus, coma or require assisted ventilation
• Organ Function, Arm E
• No evidence of disease progression: defined as increase in tumor size of >25% or new lesions.
• Timing: Recovery from last induction course of chemotherapy.
• Minimum frozen PBSC of 4 x 106 CD34 cells/kg as 2 aliquots; i.e. 2 x 106 CD34 cells/kg for each transplant are mandatory. A third aliquot of 2 x 106 CD34 cells/kg is strongly recommended for back-up.
• Hepatic: AST < 3 x upper normal
• Cardiac: Shortening fraction ≥ 27%, or ejection fraction ≥ 50%, no clinical congestive heart failure.
• Renal: Creatinine clearance or GFR > 60 ml/min/1.73m2 (If a creatinine clearance is performed at end of induction and the result is < 100 ml/min/1.73m2, a GFR must be performed using a nuclear blood sampling method or iothalamate clearance method. Camera method is NOT allowed as measure of GFR prior to or during Consolidation therapy for patients with GFR or creatinine clearance of < 100 ml/min/1.73m2.)
Exclusion Criteria:

• Arm A, B, C, and D:
• Pregnant or breastfeeding
• Active, uncontrolled infection and/or human immunodeficiency virus (HIV) positive constitute progressive disease.
• Concomitant enrollment on clinical study (such as COG study) that does not allow co-enrollment on this standard of care protocol (Arm B only)
• Arm E: Pregnant or breastfeeding
• Active, uncontrolled infection and/or HIV positive
• Known contraindication to PBSC collection. Examples of contraindications might be a weight or size less than that determined to be feasible at the collecting institution, or a physical condition that would limit the ability of the child to undergo apheresis catheter placement (if necessary) and/or the apheresis procedure.
• Patients that are 12-18 months of age with INSS Stage 4 and all 3 favorable biologic features (ie, non- amplified MYCN, favorable pathology, and DNA index > 1).
Drug: Ifosfamide, Drug: Etoposide, Drug: Mesna, Biological: G-CSF, Drug: Busulfan, Drug: Melphalan, Drug: Thiotepa, Biological: Autologous stem cell infusion, Radiation: Radiation, Drug: Carboplatin, Drug: Paclitaxel, Procedure: Leukapheresis, Drug: Anti-seizure prophylaxis, Drug: Ursodiol
Ewing's Family Tumors, Renal Tumors, Hepatoblastoma, Rhabdomyosarcoma, Soft Tissue Sarcoma, Primary Malignant Brain Neoplasms, Retinoblastoma, Medulloblastoma, Supra-tentorial Primative Neuro-Ectodermal Tumor (PNET), Atypical Teratoid/Rhabdoid Tumor (AT/RT), CNS Tumors, Germ Cell Tumors
autologous transplantation, high risk solid tumor, relapsed solid tumor, Clinics and Surgery Center (CSC)
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An Open-Label, Safety Study for Previously Treated Ataluren (PTC124) Patients with Nonsense Mutation Dystrophinopathy

a Phase 3, open-label, safety study of ataluren in patients who previously received ataluren at an investigational site in a prior PTC-sponsored clinical study or treatment plan.

Peter Karachunski
Male
Not specified
Phase 3
This study is NOT accepting healthy volunteers
NCT01247207
1011M92812
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Inclusion Criteria:

• Evidence of signed and dated informed consent/assent document(s) indicating that the participant (and/or his parent/legal guardian) has been informed of all pertinent aspects of the trial. Note: If the study candidate is considered a child under local regulation, a parent or legal guardian must provide written consent prior to initiation of study screening procedures and the study candidate may be required to provide written assent. The rules of the responsible Institutional Review Board/Independent Ethic Committee (IRB/IEC) regarding whether one or both parents must provide consent and the appropriate ages for obtaining consent and assent from the participant should be followed.
• History of exposure to ataluren in a prior PTC study or treatment plan and effected nmDBMD siblings of those participants (provided those participants have completed the placebo-controlled portion of the trial).
• Fertile men, who are sexually active with women of childbearing potential and who have not had a vasectomy, must agree to use a barrier method of birth control during the study and for up to 50 days after the last dose of study drug.
• Willingness and ability to comply with scheduled visits, drug administration and return plan, study procedures, laboratory tests, and study restrictions Note: Psychological, social, familial, or geographical factors that might preclude adequate study participation should be considered.
Exclusion Criteria:

• Exposure to another investigational drug within 1 month prior to start of study treatment.
• Eligibility for another ataluren clinical trial that is actively enrolling study participants.
• Positive for Hepatitis B core antibody or Hepatitis C antibody at screening for ataluren naïve participants (siblings) or participants who have a temporary treatment gap of 1 year before entering study.
• Known hypersensitivity to any of the ingredients or excipients of ataluren (refined polydextrose, polyethylene glycol 3350, poloxamer 407, mannitol 25C, crospovidone XL10, hydroxyethyl cellulose, colloidal silica, magnesium stearate).
• Ongoing intravenous (IV) aminoglycoside or IV vancomycin therapy.
• Ongoing uncontrolled medical/surgical condition, electrocardiogram (ECG) findings, or laboratory abnormality that, in the investigator's opinion, could adversely affect the safety of the participant or make it unlikely that follow-up would be completed.
Drug: Ataluren
Duchenne Muscular Dystrophy, Rare Diseases
Duchenne muscular dystrophy, Becker muscular dystrophy, Nonsense mutation, Premature stop codon, DMD, BMD, nmDBMD, DBMD, Ataluren, PTC124
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Determinants of Renal Structural Responses to Enzyme Replacement Therapy (ERT) in Fabry Disease Study (LDN6702)

The effect of enzyme replacement therapy on how well your kidneys are responding to enzyme replacement therapy (ERT) is not clear from blood and urine tests alone, but may be more clear in comparisons of kidney biopsies performed before and some time after ERT has been initiated, and this is what we are focusing our study efforts on. The purpose of this study is to obtain your permission to allow us to study the kidney biopsy tissues (collected for medical reasons) after the regular routine studies have been completed. Through our special research measurements and additional study, we hope to be able to see and measure very specific changes in the kidney tissues from Fabry patients taking ERT. We also hope that through these studies of what happens within the kidney before and after starting ERT, we are able to reveal valuable information about the importance of factors like your age that you started ERT, the amount or dosage of ERT, and any differences seen between males and females.

Michael Mauer
All
1 Year to 75 Years old
N/A
This study is NOT accepting healthy volunteers
NCT01581424
1205M14901
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Inclusion Criteria:

• Patients diagnosed with Fabry disease who have/have not received enzyme replacement therapy where a clinical decision has been made to obtain a kidney biopsy, a GFR, and urinary albumin studies or where patients have previously completed clinical trials which included measures of renal function and renal biopsies.
Exclusion Criteria:

• Patients with serum creatinine more than 2.5 mg/dL or known to have a renal disease other than Fabry.
Fabry Disease
Fabry disease, kidney function, kidney structure morphometry, Fabry kidney disease, Podocytes
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MT2014-10C : Allogeneic Hematopoietic Stem Cell Transplant for Patients with High Risk Hemoglobinopathies and Other Red Cell Transfusion Dependent Disorders

Ashish Gupta
All
up to 55 Years old
N/A
This study is NOT accepting healthy volunteers
NCT02179359
1407M52125
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Inclusion Criteria:

• Diagnosis of Sickle Cell Disease, Thalassemia, Diamond Blackfan Anemia or other non-malignant hematologic disorders for which a stem cell transplant is indicated
• Acceptable stem cell source identified
• Performance status of ≥ 70% (Karnofsky),or ≥ 70 (Lansky play score)
• Creatinine <2.0 mg/dl for adults or glomerular filtration rate > 50 ml/min for children
• Bilirubin, Aspartate Aminotransferase, Alkaline phosphatase <5 times the upper limit of institutional normal
• Absence of decompensated congestive heart failure, or uncontrolled arrhythmia and left ventricular ejection fraction > 40%
Exclusion Criteria:

• active, uncontrolled infection
• pregnant or breastfeeding
• HIV positive
Drug: Reduced Toxicity Ablative Regimen, Drug: Reduced Intensity Preparative Regimen, Drug: Myeloablative Preparative Regimen
Sickle Cell Disease, Transfusion Dependent Alpha- or Beta- Thalassemia, Diamond Blackfan Anemia, Paroxysmal Nocturnal Hemoglobinuria, Glanzmann Thrombasthenia, Severe Congenital Neutropenia, Shwachman-Diamond Syndrome, Non-Malignant Hematologic Disorders
Stem Cell Transplant, Clinics and Surgery Center (CSC)
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Transcranial Direct Current Stimulation and Cognitive Remediation Therapy for Psychosis

Ian Ramsay
All
18 Years to 64 Years old
N/A
This study is NOT accepting healthy volunteers
NCT02085421
1311M45305
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Inclusion Criteria:

• Meet diagnostic criteria for schizophrenia or schizoaffective disorder
• Are age 18-64
• Fluent in written and spoken English
• Have an outpatient status of at least 1 month prior to participation
• Has been on a stable dose of psychiatric medication for at least one month prior to participation
Exclusion Criteria:

• History of seizures or epilepsy
• Metallic cranial plates, screws, or implanted devices
• History of craniotomy
• History of stroke
• History of eczema on scalp
• Pre-existing sores or lesions at sites of tDCS electrode placement
• Non removable facial piercings
• Current or possibility of current pregnancy
• Has received a clinically meaningful dose of a targeted cognitive training intervention in the last 12 months
Device: tDCS
Psychosis
tdcs, neuromodulation, CRT, psychosis, schizophrenia
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MT2005-25 Hematopoietic Stem Cell Transplantation in the Treatment of Infant Leukemia

To determine the incidence of engraftment (defined as achieving donor derived neutrophil count >500/uL by day 42) in young children with leukemia or myelodysplastic syndrome undergoing a partially matched single unit umbilical cord blood transplant (UCBT) after a myeloablative preparative regimen consisting of busulfan, melphalan and fludarabine.

Christen Ebens
All
up to 3 Years old
Phase 2
This study is NOT accepting healthy volunteers
NCT00357565
0511M77206
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Inclusion Criteria:

• Matched sibling donor (HLA 8/8), if available, or a unrelated partially HLA matched single unit based on the following priority:
• 1st priority: 4/6 matched unit, cell dose >5 x 10-7 nucleated cells/kg
• 2nd priority: 5/6 matched unit, cell dose > 4 x 10-7 nucleated cells/kg
• 3rd priority: 6/6 matched unit, cell dose > 3 x 10-7 nucleated cells/kg
• Patients aged ≤ 3 years at diagnosis (not age of transplant) with hematological malignancy as detailed below:
• Acute myeloid leukemia: high risk CR1 as evidenced by:
• High risk cytogenetics t(4;11) or other MLL rearrangements; chromosome 5, 7, or 19 abnormalities; complex karyotype (>5 distinct changes); ≥ 2 cycles to obtain complete response (CR); CR2 or higher; Preceding myelodysplastic syndrome (MDS); All patients must be in CR or early relapse (i.e., <15% blasts in BM).
• Acute lymphocytic leukemia: high risk CR1 as evidenced by: High-risk cytogenetic: t(4;11) or other MLL rearrangements; hypodiploid; t(9;22); >1 cycle to obtain CR; CR2 or higher; All patients must be in CR as defined by hematological recovery, AND <5% blasts by light microscopy within the bone marrow with a cellularity of ≥15%.
• Myelodysplasia (MDS) IPSS Int-2 or High risk (i.e. RAEB, RAEBt) or refractory anemia with severe pancytopenia or high risk cytogenetics. Blasts must be < 10% by a representative bone marrow aspirate morphology.
• Persistent or rising minimal residual disease (MRD) after standard chemotherapy regimens: Patients with evidence of minimal residual disease at the completion of therapy or evidence of rising MRD while on therapy. MRD will be defined by either flow cytometry (>0.1% residual cells in the blast gate with immune phenotype of original leukemic clone), by molecular techniques (PCR or FISH) or conventional cytogenetics (g-banding).
• New Leukemia Subtypes: A major effort in the field of pediatric hematology is to identify patients who are of high risk for treatment failure so that patients can be appropriately stratified to either more (or less) intensive therapy. This effort is continually ongoing and retrospective studies identify new disease features or characteristics that are associated with treatment outcomes. Therefore, if new high risk features are identified after the writing of this protocol, patients can be enrolled with the approval of two members of the study committee.
• Recipients must have a Lansky score ≥ 50% and have acceptable organ function defined as:
• Renal: glomerial filtration rate > 60ml/min/1.73m^2
• Hepatic: bilirubin, AST/ALT, ALP < 5 x upper limit of normal,
• Pulmonary function: oxygen saturation >92%
• Cardiac: left ventricular ejection fraction > 45%.
• Voluntary written informed consent before performance of any study-related procedure not part of normal medical care.
Exclusion Criteria:

• Active infection at time of transplantation (including active infection with Aspergillus or other mold within 30 days).
• History of HIV infection or known positive serology
• Myeloablative transplant within the last 6 months.
• Evidence of active extramedullary disease (including central nervous system leukemia).
Biological: filgrastim, Drug: busulfan, Drug: cyclosporine, Drug: fludarabine phosphate, Drug: melphalan, Drug: mycophenolate mofetil, Procedure: umbilical cord blood transplantation
Leukemia, Myelodysplastic Syndromes, Childhood Acute Myeloid Leukemia in Remission, Recurrent Childhood Acute Myeloid Leukemia, Secondary Acute Myeloid Leukemia, Childhood Acute Lymphoblastic Leukemia in Remission, Previously Treated Myelodysplastic Syndrome, Secondary Myelodysplastic Syndrome, Refractory Anemia With Excess Blasts in Transformation, Refractory Anemia With Excess Blasts, Refractory Anemia, De Novo Myelodysplastic Syndrome, Childhood Myelodysplastic Syndrome
MDS, AML
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MT2012-11C: Second or Greater Allogeneic Hematopoietic Stem Cell Transplant Using Reduced Intensity Conditioning (RIC)

This is a treatment guideline for a second or greater allogeneic hematopoietic stem cell transplant (HSCT) using a reduced intensity conditioning (RIC) regimen. This regimen, consisting of busulfan, fludarabine, and low dose total body irradiation (TBI), is designed to promote engraftment in patients who failed to achieve an acceptable level of donor-derived engraftment following a previous allogeneic HSCT. While it will primarily be applied for the treatment of non-malignant diseases (NMD), on occasion it may be used to treat patients with malignant disorders as well.

Troy Lund
All
up to 55 Years old
N/A
This study is NOT accepting healthy volunteers
NCT01666080
1207M17641
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Inclusion Criteria:

• Diagnosis of any disease for which a second or greater hematopoietic stem cell transplant is needed due to insufficient donor chimerism following hematopoietic recovery after previous HSCT. Determination of "insufficiency of donor chimerism" will be made by the treating transplant physician. Occasionally donor derived engraftment may be present, but sustained aplasia or failed recovery of sufficient hematopoiesis requires administration of a second graft. This intervention may be used for both situations.
• Donor Availability: Patients considered for transplantation must have a sufficient graft as based on current criteria of the University of Minnesota Blood and Marrow Transplantation Program
• Transplantation using sufficiently matched related donors (such as matched siblings) or unrelated donors will be considered. Both granulocyte-colony stimulating factor (GCSF) stimulated peripheral blood grafts and bone marrow grafts will be considered, although bone marrow will be the priority.
• Cord blood grafts, both related and unrelated, are also eligible. As this protocol will use a reduced intensity regimen, this protocol will use the current recommendations of the University of Minnesota for choosing cord blood grafts. If a single cord blood unit cell dose is insufficient, double cord transplantation should be considered if sufficiently matched cord blood units are available. The priority of choosing cord blood donors is based on the current institutional recommendations.
• Exclusion of Metabolic Disorder or other Inherited Disorder Carrier Status from related donor and unrelated cord blood grafts as appropriate for primary disease. At the discretion of the treating transplant physician, an allograft from the previous donor may be used, if available.
• Age, Performance Status, Consent
• Age: 0 to 55 years
• Consent: voluntary written consent (adult or parental/guardian)
Exclusion Criteria:

• Previous irradiation that precludes the safe administration of an additional dose of 200 cGy of total body irradiation (TBI). Radiation Oncology will evaluate all patients who have had previous radiation therapy or TBI for approval to receive an additional 200 cGy of TBI
• Pregnant or breastfeeding
• Active, uncontrolled infection - infection that is stable or improving after 1 week of appropriate therapy (4 weeks for presumed or documented fungal infections) will be permitted
• HIV positive
• While it would be advantageous to begin therapy on this second transplant regimen > 6 months following a prior myeloablative regimen or >2 months after a reduced intensity regimen, it is recognized that there are circumstances where this may not be practical.
Drug: Busulfan, Drug: Fludarabine, Radiation: Total body irradiation, Biological: Stem cell transplant, Drug: Keppra
Hematologic Disorders, Hemoglobinopathies, Immunodeficiencies
second stem cell transplant, donor hematopoietic engraftment, hematopoietic stem cell transplantation, inherited metabolic disorder
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MT1999-20 Hematopoietic cell transplantation in children with juvenile myelomonocytic leukemia

To determine the probability of long-term disease-free survival in patients with JMML treated with busulfan (BU), cyclophosphamide (CY) and melphalan (L-PAM) followed by hematopoietic cell transplantation.

Margaret MacMillan, MD
All
up to 18 Years old
Phase 1/Phase 2
This study is NOT accepting healthy volunteers
NCT00167219
9911M24961
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Inclusion Criteria:

• Patients must have a diagnosis of JMML and fulfill these minimal criteria (International diagnostic criteria for JMML):
• Leukocytosis (> 13,000) with absolute monocytosis (> 1,000)
• The presence of immature myeloid cells in the peripheral blood
• Less than 30% marrow blasts
• Absence of t(9:22) or BCR-ABL transcript
• Adequate major organ function including:
• Cardiac: ejection fraction > 45%
• Hepatic: no clinical evidence of hepatic failure (e.g. coagulopathy, ascites)
• Karnofsky performance status > 70% or Lansky score > 50%
• Creatinine must be < 2 x normal for age
• Written informed consent.
Exclusion Criteria:

• Active uncontrolled infection within one week of HCT.
Biological: Stem Cell Transplant, Drug: Preparative Regimen
Juvenile Myelomonocytic Leukemia
Stem cell transplant, long term survival, retinoic acid
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COG ANBL1232 - Utilizing Response- and Biology-Based Risk Factors to Guide Therapy in Patients with Non-High- Risk Neuroblastoma

This phase III trial studies how well response and biology-based risk factor-guided therapy works in treating younger patients with non-high risk neuroblastoma. Sometimes a tumor may not need treatment until it progresses. In this case, observation may be sufficient. Measuring biomarkers in tumor cells may help plan when effective treatment is necessary and what the best treatment is. Response and biology-based risk factor-guided therapy may be effective in treating patients with non-high risk neuroblastoma and may help to avoid some of the risks and side effects related to standard treatment.

Emily Greengard
All
up to 18 Months old
Phase 3
This study is NOT accepting healthy volunteers
NCT02176967
1410M54605
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Inclusion Criteria:

• Patients must be:
• < 12 months (< 365 days) of age at diagnosis with INRG stage L1; or
• < 18 months (< 547 days) of age at diagnosis with INRG stage L2 or stage Ms neuroblastoma/ganglioneuroblastoma
• Enrollment on ANBL00B1 or APEC14B1 is required for all newly diagnosed patients
• Patients must have newly diagnosed v-myc avian myelocytomatosis viral oncogene neuroblastoma derived homolog (MYCN) non-amplified neuroblastoma (International Classification of Diseases for Oncology [ICD-O] morphology 9500/3) or MYCN non-amplified ganglioneuroblastoma verified by histology
• Patients must meet the specified criteria for one of the treatment groups defined below; genomic features include MYCN gene amplification, segmental chromosome aberrations (somatic copy number loss at 1p, 3p, 4p, or 11q or somatic copy number gain at 1q, 2p, or 17q) and deoxyribonucleic acid (DNA) index
• "Favorable" genomic features are defined by one or more whole-chromosome gains or hyperdiploid tumor (DNA index > 1) in the absence of segmental chromosome aberrations as defined above
• "Unfavorable" genomic features are defined by the presence of any segmental chromosome aberration (somatic copy number loss at 1p, 3p, 4p, or 11q or somatic copy number gain at 1q, 2p, or 17q) or diploid tumor (DNA index = 1); this includes copy neutral loss of heterozygosity (LOH)
• Only patients with MYCN non-amplified tumors are eligible for this study
• Group A: patients < 12 months (< 365 days) of age with newly diagnosed INRG stage L1 neuroblastoma/ganglioneuroblastoma who meet the following criteria:
• Greatest tumor diameter < 5 cm of adrenal or non-adrenal origin
• Patients with non-adrenal primaries are eligible, but must have positive uptake on metaiodobenzylguanidine (MIBG) scan or elevated catecholamine metabolites (urine or serum) to support the diagnosis of neuroblastoma
• No prior tumor resection or biopsy
• Group A will be further split into two subsets, which are mutually exclusive, for statistical purposes
• Group A1:
• > 6 months and < 12 months of age with an adrenal primary tumor < 5 cm in greatest diameter OR
• Patients less than 6 months of age with an adrenal primary tumor > 3.1 and < 5 cm in greatest diameter OR
• < 12 months of age with a non-adrenal primary site < 5 cm in greatest diameter
• Group A2: =< 6 months of age with an adrenal primary site and tumor =< 3.1 cm in greatest diameter.
• Group B: patients < 18 months (< 547 days) of age with newly diagnosed INRG stage L2 neuroblastoma/ganglioneuroblastoma who meet the following criteria:
• No life threatening symptoms or no impending neurologic or other organ function compromise (e.g. epidural or intraspinal tumors with existing or impending neurologic impairment, periorbital or calvarial-based lesions with existing or impending cranial nerve impairment, anatomic or mechanical compromise of critical organ function by tumor [abdominal compartment syndrome, urinary obstruction, etc.]); horner syndrome is not considered neurologic compromise
• No prior tumor resection, tumor biopsy ONLY
• Only patients with both favorable histology and favorable genomic features will remain on study as part of Group B; the institution will be notified of histologic and genomic results within 3 weeks of specimen submission on ANBL00B1 or APEC14B1
• Group C: patients < 18 months (< 547 days) of age with newly diagnosed INRG stage Ms neuroblastoma/ganglioneuroblastoma
• No prior radiotherapy or chemotherapy, with the exception of dexamethasone, which is allowed
• All patients and/or their parents or legal guardians must sign a written informed consent
• All institutional, Food and Drug Administration (FDA), and National Cancer Institute (NCI) requirements for human studies must be met
Exclusion Criteria:

• Patients with MYCN amplified tumors are not eligible
• Group B and C patients who do not enroll on ANBL1232 within 4 weeks of definitive diagnostic procedure
• Group A and C patients, not required to undergo tumor biopsy, who do not enroll on ANBL1232 within 4 weeks of confirmatory imaging study
Drug: Carboplatin, Other: Clinical Observation, Drug: Cyclophosphamide, Drug: Doxorubicin Hydrochloride, Drug: Etoposide, Other: Laboratory Biomarker Analysis, Other: Pharmacological Study
Ganglioneuroblastoma, Localized Resectable Neuroblastoma, Localized Unresectable Neuroblastoma, Neuroblastoma
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Nerve Function in Skin Diseases

This study will evaluate sensory perception responses as well as other skin characteristics in healthy individuals and in patients with known dermatological disease. All subjects will undergo testing with non-invasive neuroselective transcutaneous current stimulation to evaluate sensory perception thresholds.

Maria Hordinsky
All
18 Years to 70 Years old
Phase 1
This study is NOT accepting healthy volunteers
NCT02268448
0610M95566
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Inclusion Criteria:

• Age range 18-70 years old and ability to give informed consent and HIPAA authorization.
• Female subjects of childbearing potential must have a negative pregnancy test, and must agree to practice two methods of effective birth control during the study period as clonidine is an FDA pregnancy category C drug (including abstinence, oral or implant contraceptives or condoms).
• Diagnosis of AD via simplified UK Working Group Criteria and a baseline PSGA score of 2 or greater
• Willingness to adhere to study protocol
• Subjects taking hormone-containing medications must be on a stable dose for 6 months prior to study start to avoid any confounding influence on sensory and pain perception
Exclusion Criteria:

• Use of topical or oral anti-inflammatory medications for 2 weeks prior to the study start.
• Use of topical or oral anti-histamines for 2 weeks prior to the study start.
• Use of topical or oral anti-pruritic agents for 2 weeks prior to the study start.
• Use of oral neuromodulatory agents for 2 months prior to study start.
• Current use of chronic pain medications (including opioids, antidepressants and anti-epileptic drugs).
• Use of nicotine-containing products for the past 6 months prior to study start.
• History of radiation or chemotherapy.
• History of traumatic injury on prospective test sites.
• Unstable thyroid function within the past 6 months prior to study start to exclude thyroid-related neuropathy (Duyff et al, 2000).
• Known history of central or peripheral nervous system dysfunction.
• History of acute hepatitis, chronic liver disease or end stage liver disease.
• History of human immunodeficiency virus (HIV) or acquired immune deficiency syndrome.
• History of neuropathy associated with chronic obstructive pulmonary disease, diabetes mellitus, documented exposure to organophosphates or heavy metals or polychlorinated biphenyls.
• Known nutritional deficiency (vitamin B12, vitamin D, iron or zinc) within 3 months prior to the study start.
• Use of illicit drugs within the past 6 months prior to study start.
• History of daily use of power tools.
• Lyme disease, porphyria, rheumatoid arthritis, Hansen's disease (leprosy) or use of antineoplastic chemotherapeutic agents.
• Subject has any medical condition that, in the judgment of the Investigator, would jeopardize the subject's safety following exposure to the administered medications.
• Adults lacking capacity to consent
Drug: Clonidine, Drug: Naltrexone
Cutaneous Nerves CNS Itch
itch atopic dermatitis
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An Observational Registry of Abatacept in Patients with Juvenile Idiopathic Arthritis (BMS Protocol IM101240)

The objective of this study is to create an international registry with long-term follow-up to characterize and evaluate the safety of abatacept in juvenile idiopathic arthritis (JIA). The primary objective of the JIA registry is to describe the long-term safety of abatacept treatment for JIA by quantifying the incidence rates of serious infections, autoimmune disorders, and malignancies.

Colleen Correll
All
up to 17 Years old
N/A
This study is NOT accepting healthy volunteers
NCT01357668
1403M48721
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For more information regarding BMS clinical trial participation, please visit www.BMSStudyConnect.com
Inclusion Criteria:

• Diagnosis of JIA (any subtype)
• Age < 18 years at the time of enrollment unless currently or previously enrolled in an abatacept clinical trial and received abatacept
• Receiving Abatacept at the time of enrollment as per treating physician's decision or received abatacept in a clinical trial
• Parent or legally acceptable representative willing to participate in the study and sign the informed consent
Exclusion Criteria:

• Pregnant or nursing female at the time of enrollment
• Prior malignancies if the patient has not been malignancy free for at least 5 years.
• Any serious acute or chronic medical condition other than JIA, including chronic infection, which would compromise the patient's ability to participate in the study
• Known poor compliance with clinic visits (based on physician judgment)
Juvenile Idiopathic Arthritis
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