Phase 3
N=83
TBI Dose De-escalation for Fanconi Anemia
Fanconi Anemia
Bottom Line
View on ClinicalTrials.gov: NCT00352976 ↗Enrolled (actual)
83
Serious AEs
4.8%
Results posted
Nov 2021
Primary outcome: Primary: Number of Participant With Neutrophil Recovery — 78 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Cyclophosphamide (Drug); Fludarabine (Drug); Total Body Irradiation (Procedure); Bone Marrow Transplantation (Procedure); Mycophenolate Mofetil (Drug); Sirolimus (Drug)
- Age
- Pediatric, Adult, Older Adult
- Sex
- All
- Sponsor
- Masonic Cancer Center, University of Minnesota
- Primary completion
- Oct 2020
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 | — |
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.
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). 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.