Hematopoietic Stem Cell Transplant for Fanconi Anemia (FA)
This study is currently recruiting participants.
Verified January 2013 by Medical College of Wisconsin
Sponsor:
Medical College of Wisconsin
Collaborator:
Memorial Sloan-Kettering Cancer Center
Information provided by (Responsible Party):
David A. Margolis, Medical College of Wisconsin
ClinicalTrials.gov Identifier:
NCT01071239
First received: May 20, 2009
Last updated: January 4, 2013
Last verified: January 2013
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Purpose
The trial proposed is a single arm phase II treatment protocol designed to examine engraftment, toxicity, graft-versus-host disease, and ultimate disease-free survival following a novel cytoreductive regimen including busulfan, cyclophosphamide and fludarabine and anti-thymocyte globulin (ATG- a non-chemotherapy drug whose role is to kill your immune system) for the treatment of patients with Fanconi anemia who have severe aplastic anemia (SAA), or myelodysplastic syndrome (MDS) or acute myelogenous leukemia (AML), lacking HLA-genotypically identical donors using stem cell transplants derived from (1) HLA-compatible unrelated donors or (2) HLA haplotype-mismatched related donors.
| Condition | Intervention | Phase |
|---|---|---|
|
Fanconi Anemia |
Device: CliniMACs device |
Phase 2 |
| Study Type: | Interventional |
| Study Design: | Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment |
| Official Title: | A Phase II Trial of Hematopoietic Stem Cell Transplantation for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using a Chemotherapy Only Cytoreduction With Busulfan, Cyclophosphamide and Fludarabine |
Resource links provided by NLM:
Genetics Home Reference related topics:
Fanconi anemia
MedlinePlus related topics:
Anemia
U.S. FDA Resources
Further study details as provided by Medical College of Wisconsin:
Primary Outcome Measures:
- To measure the incidence and quality of engraftment and hematopoietic reconstitution. [ Time Frame: 1, 3, 6 and 12 months post transplant date ] [ Designated as safety issue: Yes ]
Secondary Outcome Measures:
- The incidence of early transplant related mortality and incidence and severity of acute and chronic GVHD [ Time Frame: weekly for the first 30 days and then 3, 6, and 12 months post transplant date ] [ Designated as safety issue: Yes ]
| Estimated Enrollment: | 25 |
| Study Start Date: | April 2009 |
| Estimated Study Completion Date: | May 2019 |
| Estimated Primary Completion Date: | May 2013 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
|
Experimental: Bone marrow processing
Bone Marrow processing using the CliniMACs device
|
Device: CliniMACs device
Donor Peripheral blood progenitor cells will use CD34+ selection with the use of the CliniMACs device
Other Name: Milteny Biotec CliniMACS device
|
Detailed Description:
We are currently recruiting patients.
Eligibility| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion Criteria:
- Fanconi Anemia (confirmed by mitomycin C or DEB chromosomal breakage testing and one of the following hematological diagnoses: Severe Aplastic Anemia, Myelodysplastic Syndrome, Acute Myelogenous Leukemia
- Karnofsky or Lansy performance scale > or = to 70%.
- Must have adequate cardiac, hepatic, renal and pulmonary function.
- Must have 7/8 or 8/8 available unrelated donor.
Exclusion Criteria:
- Pregnant or breastfeeding.
- Active CNS leukemic involvement
- Active uncontrolled viral, bacterial or fungal infection
- Positive for HIV.
Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01071239
Contacts
| Contact: David A Margolis, MD | 414-955-4170 | dmargoli@mcw.edu |
| Contact: Kristine M Allmendinger-Goertz, BA | 414-266-2137 | kallmend@mcw.edu |
Locations
| United States, Wisconsin | |
| Medical College of Wisconsin | Recruiting |
| Milwaukee, Wisconsin, United States, 53226 | |
| Principal Investigator: David A Margolis, MD | |
Sponsors and Collaborators
Medical College of Wisconsin
Memorial Sloan-Kettering Cancer Center
Investigators
| Principal Investigator: | David A Margolis, MD | Medical College of Wisconsin |
More Information
No publications provided
| Responsible Party: | David A. Margolis, Professor of Pediatrics and BMT Program Director, Medical College of Wisconsin |
| ClinicalTrials.gov Identifier: | NCT01071239 History of Changes |
| Other Study ID Numbers: | FA 08/89 |
| Study First Received: | May 20, 2009 |
| Last Updated: | January 4, 2013 |
| Health Authority: | United States: Food and Drug Administration |
Keywords provided by Medical College of Wisconsin:
|
Fanconi Anemia Hematopoietic stem cell transplant |
Additional relevant MeSH terms:
|
Anemia Fanconi Anemia Fanconi Syndrome Hematologic Diseases Anemia, Hypoplastic, Congenital Anemia, Aplastic Bone Marrow Diseases |
Genetic Diseases, Inborn DNA Repair-Deficiency Disorders Metabolic Diseases Kidney Diseases Urologic Diseases Renal Tubular Transport, Inborn Errors Metabolism, Inborn Errors |
ClinicalTrials.gov processed this record on May 19, 2013