Hematopoietic Stem Cell Transplant for Fanconi Anemia

Study Overview
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Brief Summary
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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.

Detailed Description
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We are currently recruiting patients.

Official Title
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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

Conditions
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Fanconi Anemia

Intervention / Treatment
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* Device: CliniMACs device
* Drug: Busulfan
* Drug: Fludarabine
* Drug: Cyclophosphamide
* Drug: ATG


Participation Criteria
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Eligibility Criteria
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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.
Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Bone marrow processing<br>Bone Marrow processing using the CliniMACs device | Device: CliniMACs device<br>* Donor Peripheral blood progenitor cells will use CD34+ selection with the use of the CliniMACs device<br>* Other names: Milteny Biotec CliniMACS device;Drug: Busulfan<br>* Chemotherapy administered as a part of the HSCT conditioning regimen.<br>Drug: Fludarabine<br>* Chemotherapy administered as a part of the HSCT conditioning regimen.<br>Drug: Cyclophosphamide<br>* Chemotherapy administered as a part of the HSCT conditioning regimen.<br>Drug: ATG<br>* Chemotherapy administered as a part of the HSCT conditioning regimen.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| To measure the incidence and quality of engraftment and hematopoietic reconstitution. | To measure the incidence and quality of engraftment and hematopoietic reconstitution. | 1, 3, 6 and 12 months post transplant date | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| The incidence of early transplant related mortality and incidence and severity of acute and chronic GVHD | The incidence of early transplant related mortality and incidence and severity of acute and chronic GVHD | weekly for the first 30 days and then 3, 6, and 12 months post transplant date | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Fanconi Anemia, Hematopoietic stem cell transplant