Status:
COMPLETED
TBI Dose De-escalation for Fanconi Anemia
Lead Sponsor:
Masonic Cancer Center, University of Minnesota
Conditions:
Fanconi Anemia
Eligibility:
All Genders
Phase:
PHASE2
PHASE3
Brief 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.
Detailed Description
Study Treatment: Patients will receive voriconazole (antifungal therapy) by mouth beginning 1 month prior to conditioning therapy, if possible. 1) The subject is to receive total body irradiation (300...
Eligibility Criteria
Inclusion
- 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 of age with a diagnosis of Fanconi anemia with aplastic anemia (AA), myelodysplastic syndrome without excess blasts, or high risk genotype as defined below:
- Aplastic anemia is defined as having at least one of the following when not receiving growth factors or transfusions:
- platelet count \<20 \* 10\^9/L
- ANC \<5 \* 10\^8/L
- Hemoglobin \<8 g/dL
- Myelodysplastic syndrome (MDS) with multilineage dysplasia with or without chromosomal anomalies
- High risk genotype (e.g. IVS-4 or exon 14 FANCC mutations, or BRCA1 or 2 mutations)
- High risk patients must have one or more of the following high risk features:
- Advanced MDS (≥ 5% blast) or acute leukemia
- Require additional HSCT for graft failure
- History at any time of systemic fungal or gram negative infection
- Severe renal disease with a creatinine clearance \<40 mL/min
- Age \> 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 \<5 x normal
- Karnofsky performance status \>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
- 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).
Key Trial Info
Start Date :
May 18 2006
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
October 9 2020
Estimated Enrollment :
83 Patients enrolled
Trial Details
Trial ID
NCT00352976
Start Date
May 18 2006
End Date
October 9 2020
Last Update
November 24 2021
Active Locations (1)
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1
University of Minnesota Medical Center
Minneapolis, Minnesota, United States, 55455