Status:
TERMINATED
Allogeneic Stem Cell Transplantation (ALLOSCT) in Recessive Dystrophic Epidermolysis Bullosa (RDEB)
Lead Sponsor:
Columbia University
Conditions:
Epidermolysis Bullosa
Eligibility:
All Genders
Up to 21 years
Phase:
EARLY_PHASE1
Brief Summary
Reduced Intensity Conditioning (RIC) and Allogeneic Stem Cell Transplantation (AlloSCT) from family-related donors and unrelated cord blood (UCB) donors will be safe and well tolerated in selected pat...
Detailed Description
Epidermolysis bullosa (EB), is a diverse group of genodermatoses, which is considered a rare and orphan disease and affects approximately 1 in 20,000 people in the United States for a cumulative total...
Eligibility Criteria
Inclusion
- Recessive Dystrophic Epidermolysis Bullosa (RDEB)
- Diagnosis of RDEB using molecular diagnosis and sequencing of mutations
- Skin biopsy to determine status of type VII collagen
- Age ≤21 years
- Patient must have adequate organ function as below:
- Adequate renal function defined as:
- Serum creatinine less than or equal to 1.5 x normal, or
- Creatinine clearance or radioisotope glomerular filtration rate (GFR) =40 ml/min/m2 or \> 60 ml/min/1.73 m2 or an equivalent GFR as determined by the institutional normal range
- 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:
- Uncorrected diffusing capacity of the lungs for carbon monoxide (DLCO) ≥35% by pulmonary function test
- For children who are uncooperative, no evidence of dyspnea at rest
Exclusion
- Karnofsky/Lansky Performance Score \<50%
- Pregnant or nursing
- Uncontrolled bacterial, viral or mold infection
- History or presence of skin squamous cell carcinoma
Key Trial Info
Start Date :
August 20 2009
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
September 1 2015
Estimated Enrollment :
3 Patients enrolled
Trial Details
Trial ID
NCT00881556
Start Date
August 20 2009
End Date
September 1 2015
Last Update
August 17 2021
Active Locations (3)
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1
The Children's Hospital
Aurora, Colorado, United States, 80045
2
Children's Memorial Hospital
Chicago, Illinois, United States, 60614
3
Morgan Stanley Children's Hospital of NYP
New York, New York, United States, 10032