Phase 2 N=14 Treatment

Hematopoietic Stem Cell Transplantation in High Risk Patients With Fanconi Anemia

Fanconi Anemia

Bottom Line

View on ClinicalTrials.gov: NCT00258427 ↗

Enrolled (actual)

Serious AEs

35.7%

Results posted

Dec 2021

Primary outcome: Primary: Number of Participants Experiencing Graft Failure — 1; 0; 0; 0 Participants

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: anti-thymocyte globulin (Biological); filgrastim (Biological); busulfan (Drug); cyclophosphamide (Drug); fludarabine phosphate (Drug); methylprednisolone (Drug); Hematopoietic stem cell transplantation (Biological)
Age: Pediatric, Adult
Sex: All
Sponsor: Masonic Cancer Center, University of Minnesota
Primary completion: Oct 2020

Outcome Measures

Outcome	Result	p-value
PRIMARY Number of Participants Experiencing Graft Failure	1; 0; 0; 0	—
SECONDARY Number of Participants Experiencing Chronic Graft-Versus-Host Disease	0; 0; 0; 0	—
SECONDARY Number of Participants Experiencing Chronic Graft-Versus-Host Disease	0; 0; 0; 0	—
SECONDARY Number of Participants Experiencing Acute Graft-Versus-Host Disease	0; 0; 0; 0	—
SECONDARY Number of Participants Experiencing Acute Graft-Versus-Host Disease	0; 0; 0; 0	—
SECONDARY Number of Participants Experiencing Relapse	0; 2; 1; 0	—
SECONDARY Number of Participants Experiencing Overall Survival	1; 4; 1; 1	—
SECONDARY Number of Participants Experiencing Major Infections	3; 8; 2; 1	—

Summary

RATIONALE: A bone marrow or umbilical cord blood transplant may be able to replace blood-forming cells that were destroyed by chemotherapy. Giving combination chemotherapy before a donor stem cell transplant may make the transplant more likely to work. This may be an effective treatment for patients with high risk Fanconi's anemia. PURPOSE: This clinical trial is studying how well combination chemotherapy works in treating high risk patients who are undergoing a donor stem cell transplant for Fanconi's anemia.

Eligibility Criteria

Inclusion Criteria

Patients must be 45%
Hepatic: no clinical evidence of hepatic failure (e.g. coagulopathy, ascites, no cirrhosis)
Karnofsky performance status >70% or Lansky >50%
Women of child bearing potential must be using adequate birth control and have a negative pregnancy test.

Exclusion Criteria

Active CNS leukemia at time of HSCT.
Active uncontrolled infection within one week of hematopoietic stem cell transplant (HSCT).
Pregnant or lactating female.

Donor Inclusion Criteria:

Donor must be in good health based on review of systems and results of physical examination.
Donor must have a normal hemoglobin, white count, platelet count and partial thromboplastin time (PTT), and a negative diepoxybutane (DEB) test.
HIV-NAT negative, HTLV-1, HTLV-2 negative, Hepatitis B and C negative.
Female donors of childbearing potential must have a negative pregnancy test.
Unrelated donors must agree to peripheral blood stem cell (PBSC) donation

Donor Exclusion Criteria:

Donor is a lactating female.

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT00258427). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.