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Dose-Finding Study for Cyclophosphamide as Conditioning Regimens for Bone Marrow Transplantation From Related Donors in Patients With Fanconi Anemia


Phase 1
N/A
N/A
Not Enrolling
Both
Fanconi Anemia

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Trial Information

Dose-Finding Study for Cyclophosphamide as Conditioning Regimens for Bone Marrow Transplantation From Related Donors in Patients With Fanconi Anemia


OBJECTIVES:

- Decrease the conditioning-related toxicity of cyclophosphamide without decreasing the
engraftment rate to < 90% in patients undergoing allogeneic bone marrow transplantation
for Fanconi's anemia.

OUTLINE: This is a multicenter, dose-finding study of cyclophosphamide.

- Nonmyeloablative conditioning regimen: Patients receive cyclophosphamide IV on days -5
to -2.

Cohorts of 5-10 patients receive decreasing doses of cyclophosphamide until the optimal dose
(OD) is determined. The OD is defined as the dose at which ≥ 4 of 5 patients achieve
engraftment and < 1 of 10 patients experiences dose-limiting toxicity.

- Allogeneic bone marrow transplantation (BMT): Patients undergo allogeneic BMT on day 0.

- Graft-vs-host-disease (GVHD) prophylaxis: Patients receive cyclosporine orally or IV
twice daily beginning on day -1 and continuing until day 49, followed by a taper on
days 50-180 in the absence of GVHD. Patients also receive methotrexate IV on days 1, 3,
6, and 11.

After completion of study treatment, patients are followed periodically for 5 years.

PROJECTED ACCRUAL: A total of 27 patients will be accrued for this study.

Inclusion Criteria


DISEASE CHARACTERISTICS:

- Diagnosis of Fanconi's anemia by chromosome fragility with a diepoxybutane (DEB) or
mitomycin C test

- Hemoglobin ≤ 8.0 g/dL, absolute granulocyte count ≤ 1,000/mm^3, or platelet
count ≤ 50,000/mm^3

- No refractory anemia with excess blasts, refractory anemia with excess blasts in
transformation, or acute leukemia

- HLA-identical related donor available

PATIENT CHARACTERISTICS:

- Glomerular filtration rate ≥ 30% predicted for age

- No liver disease (e.g., active hepatitis or moderate to severe portal
fibrosis/cirrhosis by biopsy)

- No symptomatic cardiac insufficiency or symptomatic arrhythmia

- No other diseases that would severely limit the probability of survival

- No HIV seropositivity

- Not pregnant or nursing

- Fertile patients must use effective contraception

PRIOR CONCURRENT THERAPY:

- Not specified

Type of Study:

Interventional

Study Design:

Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Conditioning-related toxicity

Outcome Time Frame:

100 days post-transplant

Safety Issue:

Yes

Principal Investigator

Hans-Peter Kiem, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Fred Hutchinson Cancer Research Center

Authority:

United States: Federal Government

Study ID:

1288.00

NCT ID:

NCT00317876

Start Date:

June 1998

Completion Date:

Related Keywords:

  • Fanconi Anemia
  • Fanconi anemia
  • Anemia
  • Fanconi Anemia
  • Fanconi Syndrome

Name

Location

Fred Hutchinson Cancer Research CenterSeattle, Washington  98109
Seattle Cancer Care AllianceSeattle, Washington  98109