Phase 2
Completed N=14
Hematopoietic Stem Cell Transplantation in High Risk Patients With Fanconi Anemia
Source: ClinicalTrials.gov NCT00258427 ↗Enrolled (actual)
14
Serious AEs
35.7%
Results posted
Dec 2021
Primary outcomePrimary: Number of Participants Experiencing Graft Failure — 1; 0; 0; 0 Participants
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.
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 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 | — |
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.
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. Informational only — not medical advice.