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

• Register: ClinicalTrials.gov
• Last refreshed on: 19 October 2017
• Main ID: NCT00595127
• Date of registration: 07/01/2008
• Prospective Registration: No
• Primary sponsor: Memorial Sloan Kettering Cancer Center
• Public title: Hematopoietic Stem Cell Transplantation for Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using Total Body Irradiation, Cyclophosphamide and Fludarabine
• Scientific title: A Pilot Trial of Hematopoietic Stem Cell Transplantation for the Treatment of Patients With Fanconi Anemia Lacking a Genotypically Identical Donor, Using Total Body Irradiation, Cyclophosphamide and Fludarabine
• Date of first enrolment: June 2001
• Target sample size: 21
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT00595127
• Study type: Interventional
• Phase: N/A

Countries of recruitment

United States

Contacts

• Name: Farid Boulad, MD
• Affiliation: Memorial Sloan Kettering Cancer Center

Key inclusion & exclusion criteria

Inclusion Criteria:

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.

Exclusion Criteria:

- 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



Age minimum: N/A
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Fanconi Anemia

Intervention(s)

Radiation: Total body irradiation (TBI)

Drug: Cyclophosphamide

Drug: Fludarabine

Primary Outcome(s)

Incidence & Quality of Engraftment & Hematopoietic Reconstitution [Time Frame: 8 years]

Secondary ID(s)

01-062

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Miltenyi Biotec, Inc.

National Cancer Institute (NCI)

Results

• Results available: Yes
• Date Posted: 10/08/2016
• URL: https://clinicaltrials.gov/ct2/show/results/NCT00595127