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Completed

NCT Number: NCT01071239

Hematopoietic Stem Cell Transplant for Fanconi Anemia

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.

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Key information

Conditions

Fanconi Anemia Anemia Anemia, Aplastic Anemia, Hypoplastic, Congenital Bone Marrow Diseases Bone Marrow Failure Disorders Congenital Bone Marrow Failure Syndromes Congenital, Hereditary, and Neonatal Diseases and Abnormalities DNA Repair-Deficiency Disorders Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Metabolic Diseases Nutritional and Metabolic Diseases

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Medical College of Wisconsin

Milwaukee, Wisconsin, 53226, United States

About this study

We are currently recruiting patients.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

CliniMACs device

Device

Donor Peripheral blood progenitor cells will use CD34+ selection with the use of the CliniMACs device

Other names: Milteny Biotec CliniMACS device

busulfan

Drug

Chemotherapy administered as a part of the HSCT conditioning regimen.

Fludarabine

Drug

Chemotherapy administered as a part of the HSCT conditioning regimen.

Cyclophosphamide

Drug

Chemotherapy administered as a part of the HSCT conditioning regimen.

ATG

Drug

Chemotherapy administered as a part of the HSCT conditioning regimen.

Primary outcomes

  1. To measure the incidence and quality of engraftment and hematopoietic reconstitution.

    Time frame: 1, 3, 6 and 12 months post transplant date

    To measure the incidence and quality of engraftment and hematopoietic reconstitution.

Secondary outcomes

  1. 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

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

Sponsors and collaborators

Lead sponsor

Medical College of Wisconsin

Other

Collaborators

  • Memorial Sloan Kettering Cancer Center

Registry information

Official study 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

Acronym: FA

Important dates

Study start
2009
Primary completion
2016
Study completion
2016
First posted
Feb 19, 2010
Registry last updated
Sep 11, 2019

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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