Allogeneic Stem Cell Transplantation (ALLOSCT) in Recessive Dystrophic Epidermolysis Bullosa (RDEB)

Stopped early · Early Phase 1

Conditions studied: Epidermolysis Bullosa

In brief

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 patients with RDEB. To determine the event-free survival (EFS) and overall survival (OS) following RIC consisting of busulfan/fludarabine/alemtuzumab (BFA) and AlloSCT in selected patients with RDEB.

Key facts

Study ID

NCT00881556

Run by

Columbia University

People needed

3

Starts

2009-08-20

Expected to finish

2015-09-01

Last updated by the study team

2021-08-17

Who can join

Age: any, up to 21. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• Karnofsky/Lansky Performance Score <50%
• Pregnant or nursing
• Uncontrolled bacterial, viral or mold infection
• History or presence of skin squamous cell carcinoma

Where it is running

• The Children's Hospital — Aurora, Colorado, United States
• Children's Memorial Hospital — Chicago, Illinois, United States
• Morgan Stanley Children's Hospital of NYP — New York, New York, United States

Full record on ClinicalTrials.gov

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