Allogeneic Hematopoietic Stem Cell Transplant For Epidermolysis Bullosa

• Register: ClinicalTrials.gov
• Last refreshed on: 19 October 2017
• Main ID: NCT00478244
• Date of registration: 23/05/2007
• Prospective Registration: No
• Primary sponsor: Masonic Cancer Center, University of Minnesota
• Public title: Allogeneic Hematopoietic Stem Cell Transplant For Epidermolysis Bullosa
• Scientific title: Allogeneic Hematopoietic Cell Transplantation to Correct the Biochemical Defect and Create Tolerance to Donor Tissue in Subjects With Epidermolysis Bullosa
• Date of first enrolment: April 2007
• Target sample size: 7
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/show/NCT00478244
• Study type: Interventional
• Phase: N/A

Countries of recruitment

United States

Contacts

• Name: John E. Wagner, MD
• Affiliation: Masonic Cancer Center, University of Minnesota

Key inclusion & exclusion criteria

Inclusion criteria:

- Diagnosis of epidermolysis bullosa (EB)

- Documented collagen type VII deficiency by:

- Antigenic mapping (LH7.2 antibody)

- Ultrastructure analysis of anchoring fibrils

- DNA mutation analysis

- Performance status: >50% Lansky; >50% Karnofsky

- Adequate organ function

- Renal: glomerular filtration rate > 60ml/min/1.73m2 patients aged = 10 years

- Hepatic: bilirubin, aspartate aminotransferase/alanine aminotransferase
(AST/ALT), Alkaline phosphatase (ALP) < 5 x upper limit of normal 4.2.3
Pulmonary: oxygen saturation >92% 4.2.4 Cardiac: left ventricular ejection
fraction > 45%.

- Healthy related hematopoietic stem cell donor available and meeting 1 of the following
criteria:

- HLA-A, B, DRB1-identical sibling bone marrow and/or umbilical cord blood donor
(first priority)

- HLA-A, B, DRB1-matched or partially matched related donor (second priority)

- Donor may be a carrier but must be unaffected by EB

- 8/8 HLA A, B, C, DRB1 allele level matched unrelated marrow donor (third
priority)

- 7/8 HLA-A, B, C, DRB1 allele level matched unrelated marrow donor or 4/6 HLA-A, B
(antigen level), DRB1 (allele level) matched unrelated cord blood donor (fourth
priority)

Exclusion criteria:

- Active infection at time of transplantation (including active infection with
Aspergillus or other mold within 30 days)

- Squamous cell carcinoma of the skin

- History of human immunodeficiency virus (HIV) infection

- Prior transplantation with donor skin



Age minimum: N/A
Age maximum: 25 Years
Gender: All

Health Condition(s) or Problem(s) studied

Epidermolysis Bullosa

Intervention(s)

Drug: cyclophosphamide

Drug: busulfan

Drug: fludarabine phosphate

Procedure: hematopoietic bone marrow transplantation

Primary Outcome(s)

Number of Patients With Detectable Collagen Type VII [Time Frame: Day 100 Post Transplant]

Secondary Outcome(s)

Number of Patients With Platelet Engraftment [Time Frame: Day 180 Post Transplant]

Number of Patients With Transplant-Related Mortality [Time Frame: Day 180 Post Transplant]

Number of Patients With Donor Derived Cells in Skin [Time Frame: Day 90 Post Transplant]

Number of Patients With >70% Donor Chimerism [Time Frame: Days 21, 100, 180, 365 and 730 Post Transplant]

Number of Patients With Acute Graft-Versus-Host Disease (GVHD) [Time Frame: Day 100 Post Transplant]

Number of Patients With Chronic Graft-Versus-Host Disease (cGVHD) [Time Frame: Day 365 Post Transplant]

Overall Survival [Time Frame: 1 year and 2 years Post Transplant]

Number of Patients With Neutrophil Engraftment [Time Frame: Day 42 Post Transplant]

Number of Patients With Resistance to Blister Formation [Time Frame: Month 1 through Month 24 Inclusive]

Secondary ID(s)

CDR0000546620

MT2006-15

UMN-0702M01504

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 11/02/2013
• URL: https://clinicaltrials.gov/ct2/show/results/NCT00478244