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Status:

ACTIVE_NOT_RECRUITING

Haploidentical PBMC Transplant for Severe Congenital Anemias

Lead Sponsor:

National Heart, Lung, and Blood Institute (NHLBI)

Conditions:

Sickle Cell Anemia

Eligibility:

All Genders

2-80 years

Phase:

PHASE1

PHASE2

Brief Summary

Background: Bone marrow transplantation (BMT), which involves transplanting a donor's marrow stem cells, is capable of curing some congenital anemias. BMT usually involves high-intensity treatment wi...

Detailed Description

Nonmyeloablative allogeneic peripheral blood stem cell (PBSC) transplants are currently being investigated in phase I/II trials assessing engraftment, efficacy, and toxicity at a number of transplant ...

Eligibility Criteria

Inclusion

  • INCLUSION CRITERIA:
  • Recipients (must fulfill one disease category in 5.1.1 and all of 5.1.2)
  • 1.1 Disease specific
  • 1.1.1 Patients with sickle cell disease (HB SS, SC, or SBeta(0)-thal) at high risk for disease-related morbidity or mortality, defined by having severe end-organ damage (A, B, C, or D) or potentially modifiable complication(s) not ameliorated by hydroxyurea (E):
  • A. Stroke defined as a clinically significant neurologic event that is accompanied by an infarct on cerebral MRI or cerebral arteriopathy requiring chronic transfusion therapy; OR
  • B. Sickle cell-related renal insufficiency defined by a creatinine level greater than or equal to 1.5 times the upper limit of normal and kidney biopsy consistent with sickle cell nephropathy OR nephrotic syndrome OR creatinine clearance less than 50mL/min OR requiring peritoneal or hemodialysis; OR
  • C. Pulmonary hypertension defined as tricuspid regurgitant jet velocity (TRV) of greater than or equal to 2.5 m/s at baseline (without vaso-occlusive crisis); OR
  • D. Sickle hepatopathy defined as EITHER ferritin greater than 1000mcg/L OR direct bilirubin greater than 0.4 mg/dL AND platelet count less than 250,000/microL at baseline (without vaso-occlusive crisis)
  • E. Any one of the below complications:
  • Complication: Vaso-occlusive crises;
  • Eligible for hydroxyurea\*: At least 3 hospital admissions in the last year.
  • Eligible for HSCT: More than 1 hospital admission per year while on maximal tolerated dose of hydroxyurea\*
  • Complication: Acute chest syndrome
  • Eligible for hydroxyurea\*: 2 prior ACS
  • Eligible for HSCT: any ACS while on hydroxyurea\*
  • \*hydroxyurea at maximum tolerated dose for at least 6 months
  • 1.1.2 Patients with thalassemia who have grade 2 or 3 iron overload, determined by the presence of 2 or more of the following:
  • portal fibrosis by liver biopsy
  • inadequate chelation history (defined as failure to maintain adequate compliance with chelation with deferoxamine initiated within 18 months of the first transfusion and administered subcutaneously for 8-10 hours at least 5 days each week)
  • hepatomegaly of greater than 2cm below the costochondral margin
  • 1.2 Non-disease specific:
  • 1.2.1 Age greater than or equal to 18 years
  • 1.2.2 Haploidentical relative donor available
  • 1.2.3 Ability to comprehend and willing to sign an informed consent
  • 1.2.4 Negative Beta-HCG
  • EXCLUSION CRITERIA:
  • Recipient (any of the following would exclude the subject from participating)
  • 2.1 6/6 HLA-matched with or without an ABO minor mismatched sibling donor
  • 2.2 ECOG performance status of 3 or more
  • 2.3 Evidence of uncontrolled bacterial, viral, or fungal infections (currently taking medication and progression of clinical symptoms) within one month prior to starting the conditioning regimen. Patients with fever or suspected minor infection should await resolution of symptoms before starting the conditioning regimen.
  • 2.4 Major anticipated illness or organ failure incompatible with survival from PBSC transplant
  • 2.5 Pregnant or lactating
  • 2.6 Major ABO mismatch
  • INCLUSION CRITERIA:
  • Donor
  • 3.1 Haploidentical relative donor
  • 3.2 Weight greater than or equal to 20 kg (insofar that the weight difference between recipient and donor does not exceed a reasonable likelihood of being able to obtain an adequate cell dose from the donor within two aphereses)
  • 3.3 Fit to receive filgrastim (G-CSF) and to give peripheral blood stem cells (blood counts and blood pressure within DTM standards)
  • 3.4 No history of congestive heart failure or unstable angina, and no history of stroke)
  • 3.4 Ability to comprehend and willing to sign an informed consent; assent obtained from minors
  • EXCLUSION CRITERIA:
  • Donor: (any of the following would exclude the donor from participating)
  • 4.1 Pregnant or lactating
  • 4.2 HIV positive
  • 4.3 Hemoglobin S greater than or equal to 50 percent, or beta
  • thalassemia intermedia

Exclusion

    Key Trial Info

    Start Date :

    December 14 2009

    Trial Type :

    INTERVENTIONAL

    Allocation :

    ACTUAL

    End Date :

    September 10 2026

    Estimated Enrollment :

    23 Patients enrolled

    Trial Details

    Trial ID

    NCT00977691

    Start Date

    December 14 2009

    End Date

    September 10 2026

    Last Update

    January 17 2025

    Active Locations (1)

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    Page 1 of 1 (1 locations)

    1

    National Institutes of Health Clinical Center, 9000 Rockville Pike

    Bethesda, Maryland, United States, 20892