Status:
COMPLETED
Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood Cells
Lead Sponsor:
Children's Hospital Los Angeles
Conditions:
Sickle Cell Disease
Thalassemia
Eligibility:
All Genders
Up to 21 years
Phase:
PHASE1
PHASE2
Brief Summary
This is a clinical trial of bone marrow transplantation for patients with the diagnosis of a genetic disease of blood cells that do not have an HLA-matched sibling donor. Genetic diseases of blood cel...
Detailed Description
This is a pilot clinical trial of hematopoietic stem cell transplantation for patients with the diagnosis of a genetic disease of blood cells that do not have an HLA-matched sibling donor. The stem ce...
Eligibility Criteria
Inclusion
- Lethal or sublethal genetic disease of blood cells, who lack a fully histocompatible sibling or other family donor
- Genetic diseases that would be candidates for this protocol includes those that have been shown to benefit from allogeneic HSCT: Red blood cell defects, Leukocyte defects/ Primary immune deficiencies, Platelets defects, Metabolic/storage disorders and Stem cell defects.
- Renal: creatinine clearance or glomerular filtration rate (GFR) ≥50 ml/min/1.73m2 and not requiring dialysis.
- Pulmonary: FEV1, FVC and DLCO (corrected for hemoglobin) ≥ 50% predicted. if unable to perform pulmonary function tests, then O2 saturation ≥ 92% in room air.
- Cardiac: Left ventricular ejection fraction at rest must be ≥ 40%, or shortening fraction ≥ 26%
- Hepatic: Bilirubin ≤3x upper limit of normal (ULN) and ALT and AST ≤ 5x for age (with the exception of isolated hyperbilirubinemia due to Gilbert's syndrome).
- Patients will be 0-21 years of age.
- Disease specific inclusion criteria (as applicable per protocol).
Exclusion
- Recipients should not have any of the general exclusion criteria, and disease specific exclusion criteria when applicable.
- Patient with histocompatible sibling
- End-organ failure that precludes the ability to tolerate the transplant procedure, including the conditioning regimen.
- Creatinine clearance or GFR \< 50 ml/min/1.73m2 or renal failure requiring dialysis.
- Congenital heart disease resulting in congestive heart failure.
- Severe residual CNS disease/impairment \[(other than hemiplegia alone) e.g. coma or intractable seizures\]
- Ventilatory failure
- Major congenital anomalies that adversely affect survival, e.g. CNS malformations
- Lansky score \< 40% or Karnofsky score \< 60%
- HIV seropositivity
- Diagnosis of Fanconi's anemia, Severe Combined Immunodeficiency (SCID)
- Positive pregnancy test (For female patients in child bearing period)
- Uncontrolled bacterial, viral, or fungal infections (currently taking medication yet clinical symptoms progress)
- Disease specific exclusion criteria (as applicable per protocol).
Key Trial Info
Start Date :
August 1 2008
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
August 1 2015
Estimated Enrollment :
25 Patients enrolled
Trial Details
Trial ID
NCT00730314
Start Date
August 1 2008
End Date
August 1 2015
Last Update
June 23 2016
Active Locations (1)
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1
Children Hospital Los Angeles
Los Angeles, California, United States, 90027