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8 Study Matches

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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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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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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MT2015-29 : Myeloablative Allogeneic Hematopoietic Cell Transplantation Using a Related or Adult Unrelated Donor for the Treatment of Hematological Disorders

The primary research element is to determine whether a graft-versus-host disease (GVHD) prophylaxis regimen of post-transplant cyclophosphamide, tacrolimus and MMF will reduce the likelihood of chronic GVHD in patients receiving a standard hematopoietic myeloablative stem cell transplant. The treatment related components of this protocol are established clinical practices and are considered non-investigational. The primary endpoint is cumulative incidence of chronic GVHD requiring systemic immunosuppressive treatment at 1 year post-transplant.

Shernan Holtan
All
up to 60 Years old
Phase 2
This study is NOT accepting healthy volunteers
NCT03314974
STUDY00001087
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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.
Biological: HSCT with TBI Regimen, Biological: HSCT with Non-TBI Regimen
Acute Leukemia, Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, Lymphoma, Chronic Myelogenous Leukemia, Plasma Cell Leukemia, Myeloproliferative Neoplasms, Myelofibrosis, Myelodysplasia, Refractory Anemia, High Risk Anemia, Chronic Lymphocytic Leukemia, Small Lymphocytic Lymphoma, Marginal Zone B-Cell Lymphoma, Follicular Lymphoma, Lymphoplasmacytic Lymphoma, Mantle-Cell Lymphoma, Prolymphocytic Leukemia, Diffuse Large Cell Non Hodgkins Lymphoma, Lymphoblastic Lymphoma, Burkitt Lymphoma, High Grade Non-Hodgkin's Lymphoma, Adult, Multiple Myeloma, Juvenile Myelomonocytic Leukemia, Biphenotypic/Undifferentiated/Prolymphocytic Leukemias, MRD Positive Leukemia, Natural Killer Cell Malignancies, Acquired Bone Marrow Failure Syndromes
AML, ALL, MDS, NHL, CLL, CML, SLL, Clinics and Surgery Center (CSC)
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MT2017-30 :Haploidentical Donor T-cell Replete Allogeneic Hematopoietic Cell Transplant following Reducing Intensity Conditioning for Patients with Selected High Risk Non-Malignant Disease

Christen Ebens
All
up to 25 Years old
Phase 2
This study is NOT accepting healthy volunteers
NCT03367546
STUDY00001922
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Inclusion Criteria:

• Sickle Cell Disease (SCD) * If diagnosis of SCD must meet one or more of the following disease characteristics:
• Stroke, CNS hemorrhage or a neurologic event lasting longer than 24 hours, or abnormal cerebral MRI or cerebral arteriogram or MRI angiographic study and impaired neuropsychological testing
• Acute chest syndrome with a history of recurrent hospitalizations or exchange transfusions
• Recurrent vaso-occlusive pain 3 or more episodes per year for 3 years or more years or recurrent priapism,
• Impaired neuropsychological function and abnormal cerebral MRI scan
• Stage I or II sickle lung disease,
• Sickle nephropathy (moderate or severe proteinuria or a glomerular filtration rate [GFR] 30-50% of the predicted normal value)
• Bilateral proliferative retinopathy and major visual impairment in at least one eye
• Osteonecrosis of multiple joints with documented destructive changes
• Requirement for chronic transfusions
• RBC alloimmunization
• Transfusion Dependent Alpha- or Beta-Thalassemia
• Other Non-Malignant Hematologic Disorders: Transfusion dependent or involve other potential life-threatening cytopenias, including but not limited to Paroxysmal Nocturnal Hemoglobinuria, Glanzmann's Thrombasthenia, Severe Congenital Neutropenia and Shwachman-Diamond Syndrome
• cALD
• Diagnosis of ALD by abnormal plasma very long chain fatty acid (VLCFA) profile or ABCD1 gene mutation
• Cerebral disease on MRI
• Absence of a Major Functional Disability (cortical blindness, loss of communication, wheelchair dependence) on the ALD Neurologic Function Scale
• Other inherited metabolic disorders: Any other inherited metabolic disorder for which alloHCT is indicated and for whom, in the opinion of the treating physician, the patient's best treatment option is with a haploidentical donor following non-myeloablatve conditioning.
• Age, Performance Status, Consent
• Age: 0-55 years
• Performance Status: Karnofsky ≥ 70%, Lansky play score ≥ 70
• Consent: voluntary written consent (adult or parental/guardian)
• Adequate Organ Function
• Renal: Creatinine <2.0 mg/dl for adults or glomerular filtration rate > 50 ml/min for children
• Hepatic: Bilirubin and ALT <3 times the upper limit of institutional normal
• Cardiac: Absence of decompensated congestive heart failure, or uncontrolled arrhythmia and left ventricular ejection fraction > 40%.
Exclusion Criteria:

• Availability of a suitable HLA-matched related donor
• Uncontrolled infection
• Pregnant or breastfeeding
• HIV positive
Procedure: Blood and Marrow Transplant
Sickle Cell Disease, Thalassemia, High Risk Hematologic Disorders, Cerebral Adrenoleukodystrophy, Inherited Metabolic Disorders
SCD, cALD, Clinics and Surgery Center (CSC)
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MT2017-17: T cell receptor Alpha/Beta T Cell Depleted Hematopoietic Cell Transplantation in Patients with Fanconi Anemia (FA)

The primary objective is to determine the incidence of grade II-IV acute graft versus host disease (GVHD) by Day 100 using an alpha/beta T cell depleted peripheral blood stem cells (PBSC) and without routine GVHD prophylaxis.

Margaret MacMillan, MD
All
up to 65 Years old
Phase 2
This study is NOT accepting healthy volunteers
NCT03579875
STUDY00003182
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Patient Selection:
Inclusion Criteria:

• Diagnosis of Fanconi anemia
• Less than 65 years of age
• Karnofsky performance status of ≥ 70% or, for children < 16 years of age, Lansky Play Score ≥ 50
• Presence of at least one of the following risk factors:
• Severe aplastic anemia (SAA) defined as: Aplastic anemia is defined as having at least one of the following when not receiving growth factors or transfusions:
• platelet count <20 x 109/L
• absolute neutrophil count of <5 x 108/L
• hemoglobin <8 g/dL
• Myelodysplastic syndrome (MDS) or acute leukemia
• High risk genotype
• Adequate organ function defined as:
• Bilirubin, AST or ALT, ALP <5 x normal, Cardiac: left ventricle ejection fraction (LEFV) ≥45% by ECHO
• Pulmonary: DLCO, FEV1, FVC ≥ 40% predicted, and absence of O2 requirements. For children that are not able to cooperate with PFTs, a pulse oximetry with exercise should be attempted. If neither test can be obtained it should be clearly stated in the physician's note.
• Identification of a suitable donor for peripheral blood cells per match criteria found in Section 5.
• Females of childbearing potential and males with partners of child-bearing potential must agree to use of contraception for the duration of treatment and 4 months after the transplant
• Able to provide written voluntary consent prior to the performance of any research related tests or procedures with parental/guardian consent for minor (and assent as appropriate)
Exclusion Criteria:

• Pregnant or breastfeeding as the treatment used in this study are Pregnancy Category D. Females of childbearing potential must have a negative pregnancy test (serum or urine) within 14 days of study registration
• Active, uncontrolled infection within 1 week prior to starting study therapy
• Malignant solid tumor cancer within previous 2 years Donor Selection (Inclusion Criteria): meets one of the following match criteria:
• an HLA-A, B, DRB1 matched sibling donor (matched sibling)
• an HLA-A, B, DRB1 matched related donor (other than sibling)
• a related donor mismatched at 1 HLA-A, B, C and DRB1 antigen
• 7-8/8 HLA-A,B,C,DRB1 allele matched unrelated donor per current institutional guidelines Patients and donors are typed for HLA-A and B using serological or molecular techniques and for DRB1 using high resolution molecular typing. If a donor has been selected on the basis of HLA-A, B, C and DRB1 typing as above, preference will be made for donors matched at the HLA-C locus.
• Body weight of at least 40 kilograms and at least 12 years of age
• Willing and able to undergo mobilized peripheral blood apheresis
• In general good health as determined by the medical provider
• Adequate organ function defined as:
• Hematologic: hemoglobin, WBC, platelet within 10% of upper and lower limit of normal range of test (gender based for hemoglobin)
• Hepatic: ALT < 2 x upper limit of normal
• Renal: serum creatinine < 1.8 mg/dl
• Performance of a donor infectious disease screen panel including CMV Antibody, Hepatitis B Surface Antigen, Hepatitis B Core Antibody, Hepatitis C Antibody, HIV 1/2 Antibody, HTLVA 1/2 Antibody, Treponema, and Trypanosoma Cruzi (T. Cruzi) plus HBV, HCV, WNV, HIV by nucleic acid testing (NAT); and screening for evidence of and risks factors for infection with Zika virus, or per current standard institutional donor screen - must be negative for HIV and active hepatitis B
• Not pregnant - females of childbearing potential must have a negative pregnancy test within 7 days of mobilization start
• Voluntary written consent (parent/guardian and minor assent, if < 18 years) prior to the performance of any research related procedure
Drug: Total Body Irradiation (TBI) (Plan 1), Drug: Cyclophosphamide (CY) (Plan 1), Drug: Fludarabine (FLU), Drug: Methylprednisolone (MP), Device: Donor mobilized PBSC infusion, Drug: G-CSF, Drug: Cyclophosphamide (CY) (Plan 2), Drug: Rituximab, Drug: Busulfan
Fanconi Anemia, Severe Aplastic Anemia, Myelodysplastic Syndromes
Clinics and Surgery Center (CSC)
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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.

Evaluate the efficacy of treatment with bb1111 (also known as LentiGlobin BB305 Drug Product for Sickle Cell Disease) in subjects with sickle cell disease (SCD).

Ashish Gupta
All
2 Years to 50 Years old
Phase 3
This study is NOT accepting healthy volunteers
NCT04293185
STUDY00006923
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Inclusion Criteria:

• Have a diagnosis of SCD, with either βS/βS, βS/β0 or βS/β+ genotype.
• Be ≥2 and ≤50 years of age at time of consent.
• Weigh a minimum of 6 kg.
• Have a Karnofsky performance status of ≥60 (≥16 years of age) or a Lansky performance status of ≥60 (<16 years of age).
• Be treated and followed for at least the past 24 months prior to Informed Consent in medical center(s) that maintained detailed records on sickle cell disease history.
• In the setting of appropriate supportive care measures (e.g., pain management plan), have experienced at least 4 protocol-defined severe VOEs in the 24 months prior to informed consent as defined below. A protocol-defined severe VOE is: (a) an event of acute priapism: defined as a sustained, unwanted painful erection lasting more than 2 hours and requiring care at a medical facility (with or without hospitalization) or (b) an event that requires a ≥ 24-hour hospital or emergency room (ER) observation unit visit or at least 2 visits to a day unit or ER over 72 hours with both visits requiring intravenous treatment.
• Have either experienced HU failure at any point in the past or must have intolerance to HU (intolerance is defined as the patient being unable to continue to take HU per PI judgment).
• Female and male subjects of childbearing potential agree to use 1 method of highly effective contraception from Screening to at least 6 months after drug product infusion.
• Provision of written informed consent for this study by subject, or as applicable, subject's parent(s)/legal guardian(s).
Exclusion Criteria:

• Subjects for whom allogeneic hematopoietic stem cell transplantation is medically appropriate per PI judgment and a willing, human leukocyte antigen (HLA)-matched related hematopoietic stem cell donor is available.
• Severe cerebral vasculopathy, defined by any history of overt ischemic or hemorrhagic stroke, a history of abnormal transcranial Doppler (TCD) or TCD imaging (TCDI) (e.g. TCD velocity >200 cm/sec) requiring chronic transfusions,a Screening TCD or TCDI velocity > 200 cm/sec (central read), a Screening MRA showing > 50% stenosis or occlusion in the circle of Willis (central read), or a Screening MRA showing the presence of Moyamoya (central read).
• Positive for presence of human immunodeficiency virus type 1 or 2 (HIV-1 or HIV-2), hepatitis B, hepatitis C, human T-lymphotrophic virus-1 (HTLV-1) or -2 (HTLV-2), active syphilis.
• Clinically significant, active bacterial, viral, fungal, or parasitic infection
• Advanced liver disease, such as
• clear evidence of liver cirrhosis, active hepatitis or significant fibrosis (based on MRI or liver biopsy)
• liver iron concentration ≥15 mg/g unless liver biopsy shows no evidence of cirrhosis, active hepatitis or significant fibrosis
• Inadequate bone marrow function, as defined by an absolute neutrophil count of <1×10^9/L (<0.5×10^9/L for subjects on hydroxyurea treatment) or a platelet count <100×10^9/L.
• Any contraindications to the use of plerixafor during the mobilization of hematopoietic stem cells and any contraindications to the use of busulfan and any other medicinal products required during the myeloablative conditioning, including hypersensitivity to the active substances or to any of the excipients.
• Patients needing therapeutic anticoagulation treatment during the period of conditioning through platelet engraftment
• Unable to receive pRBC transfusion.
• Prior receipt of an allogeneic transplant.
• Prior receipt of gene therapy.
• Any prior or current malignancy or immunodeficiency disorder, except previously treated, non-life threatening, cured tumors such as squamous cell carcinoma of the skin.
• Immediate family member with a known or suspected Familial Cancer Syndrome.
• Female subject is breastfeeding, pregnant or will attempt to become pregnant from Screening to at least 6 months after drug product infusion.
• Any other condition that would render the subject ineligible for HSCT.
• Participation in another clinical study with an investigational drug within 30 days of screening.
• Presence of a chromosomal abnormality or genetic mutation in the bone marrow that may put the subject at an increased risk of myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML) per Investigator's judgment.
Genetic: bb1111
Sickle Cell Disease
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A Phase I, Multi-Center, Open Label, Single Ascending Dose Study to Evaluate the Safety, Tolerability, and Pharmakokinetics of CSL889 in Adult Patients with Stable Sickle Cell Disease

This study is being done to measure levels of CSL889 in the blood and see how well it is tolerated. The study will also look for changes in several blood tests related to sickle cell disease to see how the study drug might affect these measures.

Alexander Boucher
All
18 Years to 60 Years old
Phase 1
This study is NOT accepting healthy volunteers
NCT04285827
STUDY00012191
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Inclusion Criteria:

• Diagnosis of SCD characterized by HbSS or SCD characterized by the compound heterozygous state of the βS mutation with β0 thalassemia mutations (HbSβ0)
• Aged 18 to 60 years, inclusive
• Stable SCD for at least 30 days before CSL889 infusion (Part A) or subject hospitalized for uncomplicated VOC (Part B)
• Subject is either not taking hydroxyurea and / or L-glutamine, or subject has been taking hydroxyurea and / or L-glutamine for at least 30 days before Day 1 on a stable, well tolerated regimen that is planned to continue without change throughout the study
Exclusion Criteria:

• History of primary hemorrhagic stroke
• History or evidence of inherited bleeding diathesis or significant coagulopathy at risk for bleeding
• Weight >110 kg (242 lbs)
• Surgery within 30 days before Day 1 or any preplanned surgeries during the study (minor surgeries may be permitted under local anesthesia before screening, with permission of the medical monitor)
• Female subjects who are pregnant or breastfeeding
• Female subject of childbearing potential or fertile male subject either not using or not willing to use an acceptable method of contraception to avoid pregnancy during the study and for 30 days after receipt of CSL889.
• Treatment with any other drug / biologic that is newly approved for SCD during the conduct of this study within 90 days before Day 1.
• Treatment with another investigational product within 30 days or within 5 half-lives of the product (whichever is greater) before Day 1
• Vaccination within 30 days before Day 1, or planned vaccination during the study
• Body-mass index < 16 kg/m2 or weight < 50 kg (110 lbs)
• History of anaphylactic-type reactions, transfusion related reaction, asthma, or autoimmune disease
Biological: CSL889
Sickle Cell Disease
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