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MT2019-06: A Phase 3 Study Evaluating Gene Therapy by Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo with the LentiGlobin BB305 Lentiviral Vector in Subjects with Sickle Cell Disease.
The purpose of this study is to evaluate the safety and ability of a transplant with your own gene modified stem cells (autologous stem cell transplant) to treat sickle cell disease. The goal is to determine if a sufficient amount of hemoglobin that prevents red blood sickling can be produced after the gene modified stem cells are returned to your body. This study may provide information on the potential usefulness of bb1111 for treatment of sickle cell disease
Ashish Gupta
Not specified
STUDY00006923
Inclusion Criteria:
• must be 2 to 50 years old
• diagnosis of Sickle Cell Disease
• weigh a minimum of 6 kg (13.2 pounds)
• treated and followed for at least the past 24 months
• experienced at least 4 protocol-defined VOEs in the past 24 months
• experienced HU failure at any point in the past or must have intolerance to HU
• female and male subjects of childbearing potential agree to use 1 method of highly effective contraception from starting the study to at least 6 months after drug product infusion.
Exclusion Criteria:
• if allogeneic hematopoietic stem cell transplantation (allo-HSCT) is medically appropriate and a willing, human leukocyte antigen (HLA)-matched related hematopoietic stem cell donor is available
• unable to receive a transfusion
• prior allogeneic transplant or gene therapy
• prior or current malignancy or immunodeficiency disorder, except cured tumors such as squamous cell carcinoma of the skin
• women who are pregnant or breast feeding
• additional exclusion criteria (study staff will review)
Blood Disorders
SCD, Sickle Cell Disease
RARE-OB-16: Rare CFTR Mutation Cell Collection Protocol (RARE) (RARE)
We are doing the RARE study to learn more about Cystic Fibrosis (CF). CF is caused by mutations in a gene that produces a protein called the cystic fibrosis transmembrane conductance regulator (CFTR). In people with CF, the CFTR does not function correctly. Medications are being developed to help the CFTR function better, but those medications mostly benefit people with common CFTR mutations. There are more than 1,900 mutations of the CF gene. Some of these mutations are rare and found only in a few people. The goal of this research study is to collect specimens (blood, nasal cells, rectal cells) from people with rare CFTR mutations. Another purpose of this study is to create induced pluripotent stem cells or iPS cells. “Pluripotent” stem cells are cells that can be changed into almost any cell type of the body (such as lung or intestine). They can be kept alive and stored indefinitely. There are different kinds of pluripotent stem cells. Inducted pluripotent stem cells can be created from many different kinds of specimens (such as blood, nasal cells, rectal cells). This is different from embryonic stem cells, which can only be derived from embryos. The specimens collected during this study and iPS cells created from them will be stored for use in future research to learn more about CF and study the effect of new medications. This could identify new medications that may help people with rare CFTR mutations.
Joanne Billings
Not specified
1702M07621
Inclusion Criteria:
• at least 12 years old
• documented diagnosis of Cystic Fibrosis (CF)
• willing to travel (if needed) to a regional study site for cell collection
Exclusion Criteria:
• presence of a medical condition, abnormality, or laboratory value that would place the participant at risk (study staff will review)
Rare Diseases, Respiratory System
Clinics and Surgery Center (CSC), CF, Cystic Fibrosis
MT2021-01: PTCy + Sirolimus/VIC-1911 as GVHD prophylaxis in myeloablative PBSC transplantation
The primary objective of this study is to determine the optimal dose of VIC-1911 when given in combination with standard immunosuppressive therapy in adult patients undergoing myeloablative stem cell transplantation.
Punita Grover
18 years and over
STUDY00015049
Inclusion Criteria:
Diagnosis of
acute leukemia in complete remission, or
myelodysplasia with <5% blasts, or
myeloproliferative neoplasm/myelofibrosis with <5% marrow or circulating blasts
chemosensitive Hodgkin or non-Hodgkin lymphoma
Age 18 years or older
Performance status of ≥ 80% Karnofsky
Adequate organ function within 28 days of study registration defined as:
left ventricular ejection fraction ≥ 45%
pulmonary function with FEV1, FVC, and DLCO ≥ 50% predicted
AST and ALT < 2 times upper limit of normal
Total bilirubin <1.5 times the upper limit of normal. If the patient is suspected of having Gilbert syndrome, they require prior approval of the medical monitor
creatinine clearance ≥ 50cc/min
no active/uncontrolled infection
negative HIV, HBV and HCV
ferritin < 2000 ng/ml
Patients able to tolerate oral medication
Women of childbearing potential and men with partners of child-bearing potential must agree to use of contraception for the duration of treatment through 60 days after the last treatment of VIC-1911 or sirolimus
Able to provide written voluntary consent prior to the performance of any research related tests or procedures
Exclusion Criteria:
HCT-CI > 4 or unable to receive myeloablative TBI
Use of planned post-transplant maintenance therapy to begin prior to day +75. Patients may receive standard of care maintenance therapies starting at day
+75 or later
Patients with a history of hypersensitivity to any of the investigational products
Pregnant or breastfeeding as agents used in this study are Pregnancy Category
o C: Drugs which, owing to their pharmacological effects, have caused or may be suspected of causing, harmful effects on the human fetus or neonate without causing malformations, and Pregnancy category D: There is positive evidence of human fetal risk based on adverse reaction data from investigational or marketing experience or studies in humans, but potential benefits may warrant use of the drug in pregnant women despite potential risks. Females of childbearing potential must have a negative pregnancy test (serum or urine) within 28 days of study registration.
Women or men of childbearing potential unwilling to take adequate precautions to avoid unintended pregnancy from the start of protocol treatment through 60 days after the last treatment of VIC-1911 or sirolimus Clinics and Surgery Center (CSC)
MT2021-29: Evaluation of intravenous laronidase pharmacokinetics before and after hematopoietic cell transplantation in patients with mucopolysaccharidosis type IH
In this study, the researchers are collecting blood samples to learn more about laronidase treatment in children that receive a hematopoietic cell transplantation. The laronidase dose regimens used after a hematopoietic cell transplantation may differ from those administered before. This study will establish the basis for determining if there is a need to adjust laronidase dosing regimens after receiving a hematopoietic cell transplantation.
Silvia Illamola
Up to 18 years old
STUDY00016560
Inclusion Criteria:
• between 0 to 3 years of age
• meet protocol specific eligibility criteria for allogeneic HCT for MPS IH
• planning to receive laronidase both pre and post-transplant in an inpatient setting as part of standard-of-care treatment. Virtually all patients with MPSIH being considered for transplantation at the University of Minnesota are already receiving enzyme infusions, and it is standard practice to continue to give enzyme infusions to 8 weeks post-transplant. Therefore, participation will not modify the treatment course
Exclusion Criteria:
• patient's parent/ legal guardians are unable to provide informed consent.
Rare Diseases, Cancer
Hematopoietic Cell Transplantation
MT2014-10C : Allogeneic Hematopoietic Stem Cell Transplant for Patients with High Risk Hemoglobinopathies and Other Red Cell Transfusion Dependent Disorders
Ashish Gupta
Up to 55 years old
1407M52125
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 Clinics and Surgery Center (CSC)
MT2023-31: A multi-center, randomized, active controlled clinical trial to evaluate the efficacy and safety of OTL-203 in subjects with mucopolysaccharidosis type I, Hurler syndrome (MPS-IH) compared to standard of care with allogeneic hematopoietic stem cell transplantation (allo-HSCT) (HURCULES)
This research study is designed to compare a new gene therapy, known as OTL-203 (study drug), with a standard treatment called “allogeneic hematopoietic stem cell transplant” (allo-HSCT), to find out which is better for the treatment of MPS-IH.
Ashish Gupta
Up to 18 years old
STUDY00020015
Inclusion Criteria:
• at least 28 days old to no more than 30 months old
• confirmed laboratory diagnosis of MPS-IH
• evidence of altered GAG metabolism
• see link to clinicaltrials.gov for complete inclusion and exclusion criteria
Exclusion Criteria:
• previous allo-HSCT or gene therapy
• diagnosis of HIV, Hepatitis B, Hepatitis C, or Mycoplasma
• history of uncontrolled seizures
• contraindications for MRI scans
• study staff will review additional exclusion criteria
Rare Diseases
Hurler syndrome, MPS-IH, mucopolysaccharidosis type I
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
Up to 3 years old
0511M77206
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).MT2022-27: TRANSPIRE: Lung Injury in a Longitudinal Cohort of Pediatric HSCT Patients
Primary Purpose: To identify risk factors and mechanisms of lung injury, test novel diagnostic strategies and treatments to reduce morbidity and mortality from lung injury after transplant.
Samuel Goldfarb
Up to 24 years old
SITE00001589
Inclusion Criteria:
• up to 24 years old
• undergoing allogeneic or autologous HSCT
Breathing, Lung & Sleep Health
Diffuse Alveolar Hemorrhage, Hematopoietic Stem Cell Transplant (HSCT), Interstitial Pneumonitis
MT2021-08: Phase II, Open-Label, Prospective Study of T Cell Receptor Alpha/Beta Depletion (A/B TCD) Peripheral Blood Stem Cell (PBSC) Transplantation for Children and Adults with Hematological Malignancies
The research aspect of this trial is the use of a new machine to remove specific lymphocytes from the donor’s peripheral blood stem cells (PBSCs). This is called T cell receptor alpha/beta T cell depletion. This machine does such a good job at removing the unwanted donor T cells, and as a result we think patients will need fewer drugs to suppress their immune system.
Margaret MacMillan, MD
Not specified
STUDY00016450
Inclusion Criteria:
• hematological cancer needing stem cell transplant
• 60 years old or younger
Exclusion Criteria:
• pregnant or breast feeding
• active infection
• positive for HIV, Hepatitis B or C
• brain metastasis
Cancer
Hematologic Malignancy, Leukemia, Stem Cell Transplant, Clinics and Surgery Center (CSC)
MT2013-34C: Hematopoietic Stem Cell Transplant for Dyskeratosis Congenita or Severe Aplastic Anemia: Standard of Care Considerations
Christen Ebens
0 years to 70 years old
1404M50183
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 Clinics and Surgery Center (CSC)
MT2012-10C: Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies
The primary purpose of this study is to record outcomes and patient characteristics in the Cancer Center’s and BMT databases for patients who are undergoing an allogeneic (donor) hematopoietic stem cell transplant. The data will be analyzed for transplant “milestones” such as time to blood count recovery (engraftment) and how patients are doing at 3 months and 6 months after the transplant. Participation in this study will not alter treatment or medical care. All information for this study will be collected from medical records.
Christen Ebens
Not specified
1207M17321
Inclusion Criteria:
• up to 50 years old
• diagnosis of immunodeficiency or histiocytic disorder
• see link to clinicaltrials.gov for complete inclusion criteria
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
Immune Diseases, Rare Diseases
Clinics and Surgery Center (CSC), Allogeneic Hematopoietic Stem Cell Transplant, Primary Immune Deficiencies
MT2013-06C : Treatment of graft Failure after HSCT
Troy Lund
1404M49341
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. Clinics and Surgery Center (CSC)
MT2015-29 : Myeloablative Allogeneic Hematopoietic Cell Transplantation Using a Related or Adult Unrelated Donor for the Treatment of Hematological Disorders
Punita Grover
Up to 60 years old
STUDY00001087
-
Inclusion Criteria:
Age: ≤ 60 years of age
Performance Status: Karnofsky ≥ 70%, Lansky play score ≥ 70
Consent: Voluntary written consent (adult or legally authorized representative; or parental/guardian)
Adequate Organ Function:
Renal: Creatinine <2x upper limit of normal. Patients above this limit must have creatinine clearance ≥ 40 ml/min/1.73m2 as determined by an age-appropriate method, such as cystatin C GFR.
Hepatic: Bilirubin, AST, alkaline phosphatase <4 times the upper limit of institutional normal
Pulmonary: Diffusion capacity of oxygen, corrected for hemoglobin, > 50% of predicted. For pediatric patients not able to undergo PFTs or diffusion testing: O2 sat of >95% on room air
Cardiac: Absence of decompensated congestive heart failure, or uncontrolled arrhythmia and left ventricular ejection fraction > 45%. For children not able to cooperate with MUGA or echocardiography, such should be clearly stated in the physician's documentation
HIV Status: HIV infection with undetectable viral load. All HIV+ patients must be evaluated by Infectious Disease (ID) and a HIV management plan establish prior to transplantation
Other Inclusion Criteria:
Women of child bearing potential and sexually active males with partners of child bearing potential must agree to use adequate birth control for the duration of treatment.
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
Eligible Diseases and Status: Patients are eligible unless their treatment is to be guided by a higher priority protocol.
Acute Leukemias: Must be in remission by morphology (≤5% blasts). Also a small percentage of blasts that is equivocal between marrow regeneration vs. early relapse are acceptable provided there are no associated cytogenetic markers consistent with relapse.
Acute Myeloid Leukemia (AML) and related precursor neoplasms: 2nd or greater complete remission (CR); first complete remission (CR1) in patients > 60 years old; CR1 in ≤ 60 years old that is NOT considered as favorable-risk.
Favorable risk AML is defined as having one of the following:
t(8,21) without cKIT mutation
inv(16) or t(16;16) without cKIT mutation
Normal karyotype with mutated NPM1 and wild type FLT-ITD
Normal karyotype with double mutated CEBPA
Acute prolymphocytic leukemia (APL) in first molecular remission at the end of consolidation
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 lymphoblastic leukemia (ALL)/lymphoma: second or greater CR; CR1 unable to tolerate consolidation chemotherapy due to chemotherapy-related toxicities; CR1 high-risk ALL.
High risk ALL is defined as having one of the following:
Evidence of high risk cytogenetics, e.g. t(9;22), t(1;19), t(4;11), other MLL rearrangements, IKZF1
30 years of age or older at diagnosis
White blood cell counts of greater than 30,000/mcL (B-ALL) or greater than 100,000/mcL (T-ALL) at diagnosis
CNS leukemia involvement during the course of disease
Slow cytologic response (>10% lymphoblasts in bone marrow on Day 14 of induction therapy)
Evidence of persistent immonophenotypic or molecular minimal residual disease (MRD) at the end of induction and consolidation therapy
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 achieve 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 one or more tyrosine kinase inhibitors.
Plasma Cell Leukemia after initial therapy, in patients who have achieved at least a partial remission
Myeloproliferative Neoplasms/Myelofibrosis, either primary as a result of polycythemia vera or essential thrombocythemia, with disease risk of intermediate or high-risk according to DIPSS criteria. Blasts must be <10% by bone marrow aspirate morphology.
Myelodysplasia (MDS) IPSS INT-2 or High Risk (i.e. RAEB, RAEBt) or Refractory Anemia with severe pancytopenia, transfusion dependence, or high risk cytogenetics or molecular features. 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 debulking chemotherapy before transplant.
Lymphoplasmacytic Lymphoma, Mantle-Cell Lymphoma, Prolymphocytic Leukemia are eligible after initial therapy in CR1+ or PR1+.
Diffuse large Cell NHL > CR/> PR: Patients in CR/PR with initial short remission (<6 months) are eligible, or those who have failed/or are not eligible for autologous transplant.
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.
Juvenile myelomonocytic leukemia
Biphenotypic/Undifferentiated/Prolymphocytic Leukemias in first or subsequent CR.
MRD positive leukemia (AML, ALL or accelerated/blast phase CML). Selected patients in morphologic CR, but with positive immunophenotypic (flow cytometry) or molecular evidence of MRD may be eligible if recent chemotherapy has not resulted in MRD negative status.
Natural Killer Cell Malignancies
Acquired Bone Marrow Failure Syndromes except for Fanconi Anemia or Dyskeratosis Congenita
Other Leukemia Subtypes: A major effort in the field of 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 features are identified after the writing of this protocol, patients can be enrolled with the approval of two members of the study committee.
Exclusion Criteria:
Chemotherapy refractory large cell and high grade NHL (i.e., progressive disease after > 2 salvage regimens)
CML in blast crisis
Large cell lymphoma, mantle cell lymphoma and Hodgkin disease that is progressing on salvage therapy.
Evidence of progressive disease by imaging modalities or biopsy - persistent PET activity, though possibly related to lymphoma, is not an exclusion criterion in the absence of CT changes indicating progression.
Active central nervous system malignancy
if ≤ 18 years old, prior myeloablative transplant within the last 6 months. If >18 years old prior myeloablative allotransplant or autologous transplant
Active HIV infection or known HIV positive serology
active uncontrolled infection
Pregnant or breastfeeding. The agents used in this study include Pregnancy Category D: known to cause harm to a fetus. Females of childbearing potential must have a negative pregnancy test prior to starting therapy. Clinics and Surgery Center (CSC)
MT2015-25: Tandem Myeloablative Consolidation Therapy and Autologous Stem Cell Rescue for High-Risk Neuroblastoma
Ashish Gupta
Up to 30 years old
1601M82901
Less than 30 years of age at diagnosis of neuroblastoma
End of Induction disease evaluation demonstrating CR, PR, MR or SD
Hematopoietic Recovery from last induction course of chemotherapy
No uncontrolled infection
Minimum frozen PBSCs of 2 x 10^6 CD34 cells/kg for each transplant are mandatory and a PBSC of 2 x 10^6 CD34 cells/kg for back-up are strongly recommended (thus, PBSC of no less than 6 x 10^6 CD34 cells/kg is encouraged). These must all be collected prior to the initiation of consolidation.
Adequate organ function defined as:
Hepatic: AST and ALT < 3 x upper limit of institutional normal; ALT ≤ 3 x ULN for age; total bilirubin ≤ 1.5 x ULN for age, if baseline was normal, > 1.0 1.5 x baseline if baseline was abnormal
Cardiac: shortening fraction ≥ 27% or ejection fraction ≥ 45%, no clinical congestive heart failure
Pulmonary: no evidence of dyspnea at rest and norequirement for supplemental oxygen
Renal: Creatinine clearance or 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
Recovery from acute toxicities of last cycle of induction chemotherapy
Appropriate written consent - adult or parent/guardian if patient is < 18 years of age and minor information sheet if patient is > 8 years of age
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
Up to 55 years old
1207M17641
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.MT2022-52: Allogeneic Hematopoietic Stem Cell Transplantation Using Reduced Intensity Conditioning (RIC) with Post-Transplant Cytoxan (PTCy) for the Treatment of Hematological Diseases
Stem cell transplants (sometimes referred to as a bone marrow transplants) have been done for over 40 years but research continues to further refine the method to reduce side effects without affecting transplant success. The purpose of this study is to improve on transplant outcomes while reducing the potential side effects based on what has been learned from previous transplant studies using a reduced intensity preparative regimen. Information collected during this study (transplant outcomes and side effects) will be compared with the outcomes of the previous reduced intensity conditioning transplant study that enrolled more than 300 patients since 2002.
Mark Juckett
18 years and over
STUDY00017906
Inclusion Criteria:
• up to 75 years of age
• have a matched related donor
• additional criteria for diagnosis, and physical status (study staff will review)
Exclusion Criteria:
• women who are pregnant or breast feeding
• active central nervous system malignancy
• untreated active infection
• additional criteria for exclusion (study staff will review)
Cancer
Clinics and Surgery Center (CSC), Bone Marrow Transplant, Leukemia, Stem Cell Transplant
MT2016-11 :Autologous Stem Cell Transplant In Patients with Hodgkin Lymphoma (HL) and Non-Hodgkin Lymphomas (NHL)
The treatment (chemotherapy and transplant procedures) is considered standard clinical care that are usually given to the patients with this disease. The research aspect of this study is to collect data on the patients who are being treated on this plan. Patients will be followed throughout the course of their clinical care and for three years after their transplant.
Veronika Bachanova, MD
Not specified
1611M99805
Inclusion Criteria:
• up to 75 years of age
• diagnosis of Hodgkin Lymphoma, Non-Hodgkin Lymphoma, Mature B cell Lymphoma, or Mature T cell Lymphoma
• at least 4 weeks from previous chemotherapy; 6 weeks from nitrosoureas
• women of child bearing potential and sexually active males with partners of child bearing potential must agree to use adequate birth control for the duration of treatment
• see link to clinicaltrials.gov for complete criteria
Exclusion Criteria:
• women who are pregnant or breastfeeding
• chemotherapy resistant disease
• unrelated active infection
Cancer
Clinics and Surgery Center (CSC), Hodgkin Lymphoma, Non-Hodgkin Lymphoma
2020IS043; MT2020-06; A PHASE 1/2 STUDY TO EVALUATE THE SAFETY, TOLERABILITY, AND EFFICACY OF JSP191 FOR HEMATOPOIETIC CELL TRANSPLANTATION CONDITIONING TO ACHIEVE ENGRAFTMENT AND IMMUNE RECONSTITUTION IN SUBJECTS WITH SCID
This study is looking at whether giving a new type of experimental medicine, called JSP191, can prepare the body to help the stem cell transplant work better, so the immune system can grow and fight infections. The study doctor and Sponsor also want to see how safe and well tolerated this experimental medicine is. They will study whether it is safe to give to patients and look at how much medication to give and what side effects may occur. During this study, the optimal dose of JSP191 will be determined and additional patients will be enrolled in this study using that dose level.
Christen Ebens
Not specified
STUDY00010559
Inclusion Criteria:
• at least 3 months old
• diagnosis of typical Severe Combined Immunodeficiency (SCID)
• patient with human leukocyte antigen (HLA) matched related or unrelated donors
Exclusion Criteria:
• acute or uncontrolled infections
• patients receiving any other investigational agents, or concurrent biological, chemotherapy, or radiation therapy
• patients with active malignancies
• active Graft-versus-host disease (GVHD) within 6 months prior to enrollment, or on immunosuppressive therapy for GVHD
Immune Diseases, Rare Diseases
SCID, Severe combined immunodeficiency
MT2022-41 A Phase 1/2 Study Evaluating the Safety and Efficacy of a Single Dose of Autologous CD34+ Base Edited Hematopoietic Stem Cells (BEAM-101) in Patients with Sickle Cell Disease and Severe Vaso-Occlusive Crises (BEACON Trial) (BEACON)
BEAM-101 is an experimental new therapy being developed for treating people with SCD and vaso-occlusive crises. The goal of this study is to see if BEAM-101 is safe and effective for people in the study. The study sponsor and study doctors would also like to see if individuals who are treated with BEAM-101 require fewer blood transfusions and experience fewer vasoocclusive crises requiring hospitalization, compared to before they received BEAM-101. This study will also measure the levels of fetal hemoglobin along with measures that assess quality of life and ability to function following treatment with BEAM-101.
Ashish Gupta
18 years and over
STUDY00017341
Inclusion Criteria:
• 18 to 35 years old
• documented diagnosis of sickle cell disease with specific genotypes (study staff will review)
• disease is severe
Exclusion Criteria:
• HbF levels >20%, obtained at the time of screening on or off hydroxyurea therapy
• previous transplant
• history of an overt stroke
Rare Diseases, Blood Disorders
Clinics and Surgery Center (CSC), SCD, Sickle Cell Disease
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
Up to 55 years old
1406M51542
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) Clinics and Surgery Center (CSC)
MT-2018-20: COG AALL1631 - International Phase 3 Trial in Philadelphia Chromosome-Positive Acute Lymphoblastic Leukemia (Ph+ALL) Testing Imatinib in Combination With Two Different Cytotoxic Chemotherapy Backbones
This randomized phase III trial studies how well imatinib mesylate and combination chemotherapy work in treating patients (> 1 year and < 21 years) with newly diagnosed Philadelphia chromosome positive acute lymphoblastic leukemia. Imatinib mesylate may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Drugs used in chemotherapy, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving imatinib mesylate and combination chemotherapy may work better in treating patients with Philadelphia chromosome positive acute lymphoblastic leukemia.
Peter Gordon
Not specified
SITE00000271
Inclusion Criteria:
• >= 1 year (365 days) and =< 21 years at ALL diagnosis
• Diagnosis: Ph+ (BCR-ABL1 fusion): newly diagnosed de novo ALL (B-ALL or T-ALL) or mixed phenotypic acute leukemia (MPAL meeting 2016 WHO definition) with definitive evidence of BCR-ABL1 fusion by karyotype, FISH and/or molecular methodologies. OR
• Diagnosis: ABL-class fusion: newly diagnosed B-ALL with definitive evidence of ABLclass fusions. ABL-class fusions are defined as those involving the following genes: ABL1, ABL2, CSF1R, PDGFRB, PDGFRA.
• see link to clinicaltrials.gov for complete Inclusion Criteria
Exclusion Criteria:
• known history of chronic myelogenous leukemia (CML)
• ALL developing after a previous cancer treated with cytotoxic chemotherapy
• Down syndrome
• patients with congenital long QT syndrome, history of ventricular arrhythmias or heart block
Cancer
Acute Lymphoblastic Leukemia, ALL, B Acute Lymphoblastic Leukemia, T Acute Lymphoblastic