Hematopoietic Stem Cell Transplantation for Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using Total Body Irradiation, Cyclophosphamide and Fludarabine

Completed · Not applicable

Conditions studied: Fanconi Anemia

In brief

The purpose of this research study is to: (1) determine if the combination of low dose total body irradiation, low dose cyclophosphamide and the addition of fludarabine, and a serum to suppress the immune system can allow selected stem cells to take and grow; (2) determine if selected stem cells from the blood or marrow can take and not cause graft-versus-host disease (GvHD), and; (3) evaluate the side effects of the combination of low dose radiation and chemotherapy drugs used for these transplants.

Key facts

Study ID

NCT00595127

Run by

Memorial Sloan Kettering Cancer Center

People needed

21

Starts

2001-06-01

Expected to finish

2013-05-01

Last updated by the study team

2016-08-10

Who can join

Age: any. Sex: any. Healthy volunteers: accepted.

You may qualify if…

• Diagnosis:
• Research participants must be have a diagnosis of Fanconi anemia (confirmed by mitomycin or diepoxybutane [DEB] chromosomal breakage testing).
• Hematologic Diagnosis and Status:
• Research participants must have one of the following hematologic diagnoses:
• Severe Aplastic Anemia (SAA)/Severe Isolated Single lineage Cytopenia
• Myelodysplastic Syndrome(MDS)
• Acute leukemia.
• HLA-compatible Unrelated volunteer donors:
• Research participants who do not have a related HLA-matched donor but have an unrelated donor who is either matched at all A, B and DRB1 loci or who is mismatched at 1/6 loci (A, B, or DRB1) as tested by DNA analysis, will be eligible for entry on this protocol.
• HLA-mismatched Related donors:
• Research participants who do not have a related or unrelated HLA-compatible donor must have a healthy family member who is at least HLA-haplotype identical to the recipient. First degree related donors must have a normal DEB test.
• The donor must be healthy and willing and able (1) to receive a 5 day course of G-CSF and undergo 2 daily leukaphereses, or (2) to undergo general anesthesia and bone marrow donation. In order to undergo a Tcell depletion, a donor should be able to have a volume of 15 ml/Kg of the research participant's body weight harvested safely.
• HLA-compatible Cord Blood Units:
• Research participants who do not have a related HLA-matched donor but have an unrelated placental cord blood unit which matched at all A, B and DRB1 loci or who is mismatched at 1/6 or 2/6 loci (A, B, or DRB1) as tested by DNA analysis, will be eligible for entry on this protocol.
• Research participants may be of either gender or any ethnic background.
• Research participants must have a Karnofsky adult, or Lansky pediatric performance scale status > 70%.
• At the time of referral for transplantation, research participants must be in good clinical condition without co-existing medical problems that would significantly increase the risk of the transplant procedure. Research participants must be free of infections at the time of transplant. Research participants must have a life expectancy that is greater than 8 weeks.
• Research participants must have adequate physical function measured by cardiac, Hepatic, Renal, Pulmonary.
• Research participants must be available for follow-up evaluations at 30, 60, 180 days post BMT and yearly for 5 years.

You may not qualify if…

• Active CNS leukemic involvement
• Female research participants who are pregnant or breast-feeding
• Active viral, bacterial or fungal infection
• Research participant seropositive for HIV-I/II; HTLV -I/II

Where it is running

• Memorial Sloan Kettering Cancer Center — New York, New York, United States

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.