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History of Changes for Study: NCT00478244
Combination Chemotherapy and Allogeneic Hematopoietic Stem Cell Transplant in Treating Pediatric Patients With Epidermolysis Bullosa at High Risk of Developing Squamous Cell Carcinoma of the Skin
Latest version (submitted December 3, 2017) on ClinicalTrials.gov
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Study Record Versions
Version A B Submitted Date Changes
1 May 23, 2007 None (earliest Version on record)
2 June 4, 2007 Outcome Measures, Conditions, Study Description, Eligibility, Study Design, Study Status and Study Identification
3 July 17, 2007 Contacts/Locations, Sponsor/Collaborators and Study Status
4 September 20, 2007 Study Status
5 October 25, 2007 Arms and Interventions and Study Status
6 November 14, 2007 Sponsor/Collaborators, Contacts/Locations and Study Status
7 December 25, 2007 Study Status
8 April 1, 2008 Study Status
9 April 25, 2008 Contacts/Locations, Sponsor/Collaborators and Study Status
10 May 23, 2008 Arms and Interventions and Study Status
11 July 23, 2008 Study Design and Study Status
12 July 26, 2008 Eligibility, Study Design, Study Description and Study Status
13 February 6, 2009 Study Status
14 July 7, 2009 Arms and Interventions, Study Status, Contacts/Locations, Outcome Measures, Study Identification, Study Design, Conditions, Eligibility, Oversight and Sponsor/Collaborators
15 January 25, 2010 Study Identification, Arms and Interventions, Study Description, Study Status, Eligibility and Outcome Measures
16 July 29, 2010 Outcome Measures, Conditions, Study Description, Study Status, Eligibility, Arms and Interventions and Study Identification
17 January 11, 2011 Study Status
18 August 23, 2011 Sponsor/Collaborators, Study Status, Contacts/Locations and Eligibility
19 January 31, 2012 Recruitment Status, Study Status, Study Design and Contacts/Locations
20 February 6, 2012 Study Status
21 January 4, 2013 Outcome Measures, Study Status, Results, Eligibility and Study Identification
22 December 3, 2017 Study Status and Baseline Characteristics
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Study NCT00478244
Submitted Date:  May 23, 2007 (v1)

Open or close this module Study Identification
Unique Protocol ID: CDR0000546620
Brief Title: Combination Chemotherapy and Allogeneic Hematopoietic Stem Cell Transplant in Treating Pediatric Patients With Epidermolysis Bullosa at High Risk of Developing Squamous Cell Carcinoma of the Skin
Official Title: Allogeneic Hematopoietic Cell Transplantation to Correct the Biochemical Defect and Create Tolerance to Donor Tissue in Subjects With Epidermolysis Bullosa
Secondary IDs: UMN-MT2006-15
UMN-0702M01504
Open or close this module Study Status
Record Verification: May 2007
Overall Status: Recruiting
Study Start: April 2007
Primary Completion:
Study Completion:
First Submitted: May 23, 2007
First Submitted that
Met QC Criteria:
May 23, 2007
First Posted: May 24, 2007 [Estimate]
Last Update Submitted that
Met QC Criteria:
May 23, 2007
Last Update Posted: May 24, 2007 [Estimate]
Open or close this module Sponsor/Collaborators
Sponsor: University of Minnesota
Responsible Party:
Collaborators:
Open or close this module Oversight
U.S. FDA-regulated Drug:
U.S. FDA-regulated Device:
Data Monitoring:
Open or close this module Study Description
Brief Summary:

RATIONALE: Drugs used in chemotherapy work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing. Giving more than one drug (combination chemotherapy) may kill more tumor cells. Giving chemotherapy before a donor allogeneic bone marrow, peripheral blood, or umbilical cord blood stem cell transplant helps stop the growth of tumor cells. It also helps stop the patient's immune system from rejecting the donor's stem cells. When the healthy stem cells from a donor are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells. Giving cyclosporine together with mycophenolate mofetil before, during, and after transplant may stop this from happening.

PURPOSE: This clinical trial studies giving combination chemotherapy together with allogeneic hematopoietic stem cell transplant in treating pediatric patients with epidermolysis bullosa at high risk of developing squamous cell carcinoma of the skin.

Detailed Description:

OBJECTIVES:

Primary

  • Estimate the incidence of detectable donor-derived collagen type VII by day 100 in pediatric patients with epidermolysis bullosa at high risk of developing squamous cell carcinoma of the skin treated with busulfan, cyclophosphamide, and fludarabine phosphate followed by hematopoietic stem cell transplantation from a healthy, unaffected related donor.

Secondary

  • Determine the incidence of transplant-related mortality at 180 days in patients treated with this regimen.
  • Determine the incidence of chimerism at 21, 100, 180, 365, and 730 days in patients treated with this regimen.
  • Determine the incidence of neutrophil recovery at day 42 and platelet recovery at day 180 in patients treated with this regimen.
  • Determine the incidence of acute graft-versus-host disease (GVHD) grade II-IV and grade III-IV at day 100 in patients treated with this regimen.
  • Determine the incidence of chronic GVHD at 1-year in patients treated with this regimen.
  • Determine the probability of survival at 1 and 2 years in patients treated with this regimen.
  • Determine the incidence of donor skin-derived epidermal cell survival 90 days after epidermal cell transplant in the patient subpopulation for whom epidermal transplant was performed.

OUTLINE: This is an open-label, pilot study.

  • Conditioning regimen: Patients receive busulfan IV over 2 hours every 6 hours on days -9 to -4, fludarabine phosphate IV over 1 hour on days -5 to -3, and high-dose cyclophosphamide IV over 1 hour on days -5 to -2.
  • Stem cell transplantation: Patients undergo allogeneic hematopoietic stem cell transplantation (may include bone marrow, peripheral blood stem cells, or umbilical cord blood).
  • Graft-vs-host disease prophylaxis: Patients receive cyclosporine IV over 2 hours or orally every 8 hours on days -3 to 180 and mycophenolate mofetil IV or orally on days -3 to 30.
  • Epidermal cell transplantation: Patients with confirmed chimerism at day 100, but with persistent evidence of epidermolysis bullosa, undergo donor-derived epidermal cell transplantation.

After completion of study treatment, patients are followed periodically for at least 5 years.

PROJECTED ACCRUAL: A total of 10 patients will be accrued for this study.

Open or close this module Conditions
Conditions: Non-Melanomatous Skin Cancer
Keywords: squamous cell carcinoma of the skin
Open or close this module Study Design
Study Type: Interventional
Primary Purpose: Prevention
Study Phase: Not Applicable
Interventional Study Model:
Number of Arms:
Masking: None (Open Label)
Enrollment: 10
Open or close this module Arms and Interventions
Intervention Details:
Drug: busulfan
Drug: cyclophosphamide
Drug: cyclosporine
Drug: fludarabine phosphate
Drug: mycophenolate mofetil
Procedure: allogeneic bone marrow transplantation
Procedure: allogeneic hematopoietic stem cell transplantation
Procedure: biological therapy
Procedure: bone marrow ablation with stem cell support
Procedure: bone marrow transplantation
Procedure: chemotherapy
Procedure: graft versus host disease prophylaxis/therapy
Procedure: high-dose chemotherapy
Procedure: peripheral blood stem cell transplantation
Procedure: supportive care/therapy
Procedure: umbilical cord blood transplantation
Open or close this module Outcome Measures
Primary Outcome Measures:
1. Incidence of detectable donor-derived collagen type VII by day 100
Secondary Outcome Measures:
1. Incidence of transplant-related mortality at 180 days
2. Incidence of chimerism at 21, 100, 180, 365, and 730 days
3. Incidence of neutrophil recovery at day 42 and platelet recovery at 180 days
4. Incidence of acute graft-versus-host disease (GVHD) at day 100
5. Incidence of chronic GVHD at 1-year
6. Probability of survival and disease-free survival at 1 and 2 years
7. Incidence of donor skin-derived epidermal cell survival 90 days after epidermal cell transplantation
Open or close this module Eligibility
Minimum Age:
Maximum Age: 7 Years
Sex: All
Gender Based:
Accepts Healthy Volunteers: No
Criteria:

DISEASE CHARACTERISTICS:

  • Diagnosis of epidermolysis bullosa (EB)
    • Documented collagen type VII deficiency by the following methods:
      • Antigenic mapping (LH7.2 antibody)
      • Ultrastructure analysis of anchoring fibrils
      • DNA mutation analysis.
  • No squamous cell carcinoma of the skin
  • Healthy related hematopoietic stem cell donor available and meeting 1 of the following criteria:
    • HLA-A, B, DRB1- identical sibling donor bone marrow and/or umbilical cord blood (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

PATIENT CHARACTERISTICS:

  • Lansky performance status 60-100%
  • Glomerular filtration rate > 60 mL/min
  • Bilirubin < 5 times upper limit of normal (ULN)
  • AST and ALT < 5 times ULN
  • Alkaline phosphatase < 5 times ULN
  • Oxygen saturation > 92%
  • LVEF ≥ 45%
  • No active infection at time of transplantation (including active infection with Aspergillus or other mold within the past 30 days)
  • No HIV positivity

PRIOR CONCURRENT THERAPY:

  • No prior transplantation with donor skin
Open or close this module Contacts/Locations
Study Officials: John E. Wagner, MD
Study Chair
Masonic Cancer Center, University of Minnesota
Locations: United States, Minnesota
University of Minnesota Cancer Center
[Recruiting]
Minneapolis, Minnesota, United States, 55455
Contact:Contact: Clinical Trials Office - University of Minnesota Cancer Center 888-226-2376
Open or close this module IPDSharing
Plan to Share IPD:
Open or close this module References
Links:
Available IPD/Information:

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