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

NCT00881556 · Status: TERMINATED · Phase: EARLY_PHASE1 · Type: INTERVENTIONAL · Enrollment: 3

Last updated 2021-08-17

No results posted yet for this study

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 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.

Conditions

  • Epidermolysis Bullosa

Interventions

DRUG

Palifermin

60 mcg/kg/day for 6 days

DRUG

Fludarabine

30 mg/m2 IV x 1 for 6 days

DRUG

Busulfan

4 mg/kg/day IV divided BID for 4 days

DRUG

Lorazepam

0.02-0.05 mg/kg for 5 days

DRUG

Alemtuzumab

20 mg/m2 IV for 5 days

DRUG

Tacrolimus

0.03mg/kg/24 hours as continuous infusion for 4 days

Sponsors & Collaborators

Principal Investigators

  • Angela Christiano, PhD · Columbia University

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Max Age
21 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2009-08-20
Primary Completion
2015-09-30
Completion
2015-09-30
FDA Drug
Yes

Countries

  • United States

Study Locations

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Entities

Drugs
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT00881556 on ClinicalTrials.gov