FA: Hematopoietic Stem Cell Transplant for Fanconi Anemia

Sponsor

Medical College of Wisconsin (Other)

Overall Status

Completed

CT.gov ID

NCT01071239

Collaborator

Memorial Sloan Kettering Cancer Center (Other)

Enrollment

Location

Arm

Duration (Months)

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

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 or Disease	Intervention/Treatment	Phase
Fanconi Anemia	Device: CliniMACs device Drug: Busulfan Drug: Fludarabine Drug: Cyclophosphamide Drug: ATG	Phase 2

Detailed Description

We are currently recruiting patients.

Study Design

Study Type:

Interventional

Actual Enrollment :

1 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (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

Study Start Date :

Apr 1, 2009

Actual Primary Completion Date :

Aug 30, 2016

Actual Study Completion Date :

Aug 30, 2016

Arms and Interventions

Arm	Intervention/Treatment
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 Names: Milteny Biotec CliniMACS device Drug: Busulfan Chemotherapy administered as a part of the HSCT conditioning regimen. Drug: Fludarabine Chemotherapy administered as a part of the HSCT conditioning regimen. Drug: Cyclophosphamide Chemotherapy administered as a part of the HSCT conditioning regimen. Drug: ATG Chemotherapy administered as a part of the HSCT conditioning regimen.

Arm

Intervention/Treatment

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 Names:

Milteny Biotec CliniMACS device

Drug: Busulfan

Chemotherapy administered as a part of the HSCT conditioning regimen.

Drug: Fludarabine

Chemotherapy administered as a part of the HSCT conditioning regimen.

Drug: Cyclophosphamide

Chemotherapy administered as a part of the HSCT conditioning regimen.

Drug: ATG

Chemotherapy administered as a part of the HSCT conditioning regimen.

Outcome Measures

Primary Outcome Measures

To measure the incidence and quality of engraftment and hematopoietic reconstitution. [1, 3, 6 and 12 months post transplant date]
To measure the incidence and quality of engraftment and hematopoietic reconstitution.

Secondary Outcome Measures

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]
The incidence of early transplant related mortality and incidence and severity of acute and chronic GVHD

Eligibility Criteria

Criteria

Ages Eligible for Study:

N/A and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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.