Low-Dose Total-Body Irradiation and Fludarabine Phosphate Followed by Unrelated Donor Stem Cell Transplant in Treating Patients With Fanconi Anemia

• Register: ClinicalTrials.gov
• Last refreshed on: 16 December 2017
• Main ID: NCT00093743
• Date of registration: 06/10/2004
• Prospective Registration: No
• Primary sponsor: Fred Hutchinson Cancer Research Center
• Public title: Low-Dose Total-Body Irradiation and Fludarabine Phosphate Followed by Unrelated Donor Stem Cell Transplant in Treating Patients With Fanconi Anemia
• Scientific title: Low-Dose Total Body Irradiation and Fludarabine Followed By Unrelated Donor Stem Cell Transplantation for Patients With Fanconi Anemia - A Multicenter Trial
• Date of first enrolment: January 2000
• Target sample size: 2
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT00093743
• Study type: Interventional
• Study design: Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1

Countries of recruitment

United States

Contacts

• Name: Hans-Peter Kiem
• Affiliation: Fred Hutchinson Cancer Research Center/University of Washington Cancer Consortium

Key inclusion & exclusion criteria

Inclusion Criteria:

- Any patient with marrow failure and increased chromosome fragility as determined in
the diepoxybutane (DEB) or mitomycin C test

- Any patient with Fanconi anemia (FA) with marrow failure meeting the following
criteria:

- Granulocyte count < 0.2 x 10^9/L

- Platelet count < 20 x 10^9/L

- Hemoglobin < 8 g/dl

- Corrected reticulocyte count <1%

- Any patient with FA as determined by DEB fragility, who has life-threatening marrow
failure involving a single hematopoietic lineage

- Any patient with FA and pre-existing cytogenetic abnormality including hematopoietic
malignancy (myelodysplastic syndromes [MDS] or acute myeloid leukemia [AML]) in
remission

- DONOR: Unrelated Donors who are prospectively: Matched for human lymphocyte antigen
(HLA)-DRB1 and DQB1 alleles (must be defined by high resolution typing); only a single
allele disparity will be allowed for HLA -A, B, or C as defined by high resolution
typing

- DONOR: HLA typing will be performed at the highest level of resolution available at
the time of transplant

Exclusion Criteria:

- Evidence for hematopoietic malignancy in relapse

- Heart or lung disease that would prevent compliance with conditioning and GvHD regimen
or would severely limit the probability of survival

- Human immunodeficiency virus (HIV) seropositive patients

- Females who are pregnant or breastfeeding, or unwilling to use contraceptive
techniques during and for the 12 months following treatment

- DONOR: Donors who by DEB testing are found to have FA

- DONOR: Donors who test positive in the lymphocytotoxic crossmatch assay

- DONOR: Donors who are HIV positive

- DONOR: Donors who for other medical or psychological reasons are not suitable as
donors



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

Health Condition(s) or Problem(s) studied

Adult Acute Myeloid Leukemia in Remission

Adult Acute Myeloid Leukemia With Inv(16)(p13;q22)

Adult Acute Myeloid Leukemia With t(16;16)(p13;q22)

Childhood Myelodysplastic Syndromes

Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities

Adult Acute Myeloid Leukemia With Del(5q)

Childhood Acute Myeloid Leukemia in Remission

Adult Acute Myeloid Leukemia With t(8;21)(q22;q22)

Fanconi Anemia

Adult Acute Myeloid Leukemia With t(15;17)(q22;q12)

Previously Treated Myelodysplastic Syndromes

Intervention(s)

Drug: fludarabine phosphate

Radiation: total-body irradiation

Drug: mycophenolate mofetil

Drug: cyclosporine

Procedure: allogeneic bone marrow transplantation

Procedure: allogeneic hematopoietic stem cell transplantation

Procedure: peripheral blood stem cell transplantation

Primary Outcome(s)

Engraftment, defined as donor chimerism (mixed or complete) [Time Frame: Day 180]

Engraftment, defined as donor chimerism (mixed or complete) [Time Frame: Day 56]

Engraftment, defined as donor chimerism (mixed or complete) [Time Frame: Day 28]

Acute GvHD defined using the Seattle criteria [Time Frame: Day 84]

Engraftment, defined as donor chimerism (mixed or complete) [Time Frame: Day 84]

Regimen toxicity assessed using the Bearman scale [Time Frame: Up to day 100]

Secondary ID(s)

NCI-2012-00593

P30CA015704

1444.00

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

National Heart, Lung, and Blood Institute (NHLBI)

National Cancer Institute (NCI)