Status:
ACTIVE_NOT_RECRUITING
Stem Cell Transplant in Sickle Cell Disease and Thalassemia
Lead Sponsor:
Columbia University
Conditions:
Sickle Cell Disease
Beta Thalassemia
Eligibility:
All Genders
1-30 years
Phase:
PHASE2
Brief Summary
The primary purpose of this study is to see if giving lower doses of chemotherapy (moderately ablative) will result in successful bone marrow replacement without as severe side-effects but with perman...
Detailed Description
Sickle cell disease is a genetic disorder in which a mutation in the beta chain of human hemoglobin results in abnormal blood hemoglobin, causing red blood cells to sickle under stress with resulting ...
Eligibility Criteria
Inclusion
- Inclusion Criteria:
- Sickle Cell Disease:
- Diagnosis of Homozygous Hemoglobin S Disease or Heterozygous Hemoglobin Sickle Cell (SC) or S 0/+ thalassemia, or Sickle/variant resulting in Chronic Hemolytic Anemia with hemoglobin (HgB) ≤10 mg/dL
- Age ≤30
- Matched sibling donor and asymptomatic, or 8/8 human leukocyte antigen (HLA) matched unrelated adult donor
- Patient must have adequate organ function as below:
- Adequate renal function defined as serum creatinine ≤1.5 x normal, or Creatinine clearance or radioisotope glomerular filtration rate (GFR) \>100 ml/min/1.73 m2 or \>70ml/min/1.73m2 for patients \>16 years old
- Adequate liver function defined as serum glutamic oxaloacetic transaminase (SGOT) (aspartate aminotransferase (AST)) or serum glutamic-pyruvic transaminase (SGPT) (alanine aminotransferase (ALT)) \< 5.0 x normal
- Adequate Cardiac Function defined as shortening fraction of ≥28% by echocardiogram, or ejection fraction of ≥48% by radionuclide angiogram or echocardiogram
- Adequate pulmonary function defined as corrected Diffusing capacity of the lungs for carbon monoxide (DLCO) ≥40% by pulmonary function test, or for children who are unable to perform DLCO maneuver ≥85% O2 saturation, no evidence of dyspnea at rest
- Exclusion criteria:
- General
- Karnofsky/Lansky Performance Score \<60%
- Demonstrated lack of compliance with medical care
- Pregnant or nursing
- Evidence of uncontrolled bacterial, viral or fungal infections (currently taking medication and progression of clinical symptoms) within 1 month prior to starting the conditioning regimen. Patients with fever or suspected minor infection should await resolution of symptoms before starting the conditioning regimen.
- Histologic Exam of Liver (liver biopsy) with bridging fibrosis or cirrhosis.
Exclusion
Key Trial Info
Start Date :
September 1 2004
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
February 1 2025
Estimated Enrollment :
53 Patients enrolled
Trial Details
Trial ID
NCT00408447
Start Date
September 1 2004
End Date
February 1 2025
Last Update
June 13 2024
Active Locations (1)
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1
Morgan Stanley Children's Hospital, New York-Presbyterian, Columbia University
New York, New York, United States, 10032