Phase 3
Completed N=83
TBI Dose De-escalation for Fanconi Anemia
Source: ClinicalTrials.gov NCT00352976 ↗Enrolled (actual)
83
Serious AEs
4.8%
Results posted
Nov 2021
Primary outcomePrimary: Number of Participant With Neutrophil Recovery — 78 Participants
◆ Published Evidence
Highly cited
111citations · ~10 / year
Alternative donor hematopoietic cell transplantation for Fanconi anemia.
Summary
This is a single arm, total body irradiation (TBI) trial. All patients will be prescribed TBI 300 cGy with the goal of evaluating secondary endpoints.
Linked Publications
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Alternative donor hematopoietic cell transplantation for Fanconi anemia.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participant With Neutrophil Recovery |
78 | — |
| SECONDARY Number of Participants Experiencing Grade ≥3 Regimen Related Toxicity |
71 | — |
| SECONDARY Number of Participants With Secondary Graft Failure at 100 Days |
4 | — |
| SECONDARY Number of Participants Experiencing Acute Graft-versus-host Disease (GVHD) |
15 | — |
| SECONDARY Number of Participants Experiencing Chronic GVHD |
6 | — |
| SECONDARY Number of Participants Experiencing Overall Survival |
70 | — |
| SECONDARY Number of Participants Experiencing Infections by Day 100 |
53 | — |
| SECONDARY Number of Participants Experiencing Infections by Day 180 |
55 | — |
| SECONDARY Number of Participants Experiencing Infections by Day 365 |
56 | — |
| SECONDARY Average Immunoglobulin G (IgG) Levels as a Measure of Immune Reconstitution After Transplant, by 100 Days |
550.6 | — |
| SECONDARY Average IgG Levels as a Measure of Immune Reconstitution After Transplant, by 180 Days |
652.9 | — |
| SECONDARY Average IgG Levels as a Measure of Immune Reconstitution After Transplant by 365 Days |
724.0 | — |
| SECONDARY Average IgA Levels as a Measure of Immune Reconstitution After Transplant by 100 Days |
88.0 | — |
| SECONDARY Average IgA Levels as a Measure of Immune Reconstitution After Transplant by 180 Days |
98.7 | — |
| SECONDARY Average IgA Levels as a Measure of Immune Reconstitution After Transplant by 365 Days |
107.2 | — |
| SECONDARY Average IgM Levels as a Measure of Immune Reconstitution After Transplant by 100 Days |
71.1 | — |
| SECONDARY Average IgM Levels as a Measure of Immune Reconstitution After Transplant by 180 Days |
77.0 | — |
| SECONDARY Average IgM Levels as a Measure of Immune Reconstitution After Transplant by 365 Days |
82.8 | — |
Eligibility Criteria
Inclusion Criteria
Meeting the definition of standard risk or high risk Fanconi anemia as defined in the next two sections:
- Standard risk patients must be 18 years
- Very high risk patients must have Advanced MDS (≥ 5% blast) or acute leukemia after initial hematopoietic stem cell transplant (HSCT)
- Patients must have an appropriate source of stem cells. Patients and donors will be typed for HLA-A, B, C and DRB1 using high resolution molecular typing.
- Adequate major organ function including:
- Cardiac: ejection fraction >45%
- Hepatic: bilirubin, AST or ALT, ALP 70% or Lansky >50 (if < 16 years of age)
- Women of child-bearing age must be using adequate birth control and have a negative pregnancy test.
- Written consent.
Exclusion Criteria
- Available HLA-genotypically identical related donor in standard risk patients.
- Active central nervous system (CNS) leukemia at time of study enrollment.
- History of squamous cell carcinoma of the head/neck/cervix within previous 2 years.
- Prior radiation therapy that prevents further total body irradiation (TBI).
Data sourced from ClinicalTrials.gov (NCT00352976) and the linked publication. 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.