Alpha/Beta TCD HCT in Patients With Inherited BMF Disorders (NCT03579875) | Clinical Trial Compass
RecruitingPhase 2
Alpha/Beta TCD HCT in Patients With Inherited BMF Disorders
United States48 participantsStarted 2018-11-13
Plain-language summary
This is a phase II trial of T cell receptor alpha/beta depletion (α/β TCD) peripheral blood stem cell (PBSC) transplantation in patients with inherited bone marrow failure (BMF) disorders to eliminate the need for routine graft-versus-host disease (GVHD) immune suppression leading to earlier immune recovery and potentially a reduction in the risk of severe infections after transplantation.
Who can participate
Age range65 Years
SexALL
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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Patient Selection:
Inclusion Criteria:
For FA patients:
* Diagnosis of Fanconi anemia
* Age \<65 years of age
* Has one of the following risk factors:
* Severe aplastic anemia (SAA)
* Myelodysplastic features
* High risk genotype
* Immunodeficiency associated with history of recurrent infections
* Karnofsky performance status ≥ 70% if ≥ 16 years of age or Lansky play score ≥ 50% for patients \<16 years of age
* Adequate pulmonary, cardiac and liver function
* Voluntary written consent (minor assent if appropriate) prior to the performance of any study related procedures not part of standard medical care
For TBD patients:
• Diagnosis of TBD
* Age \<70 years of age
* Has one of the following risk factors:
* Severe aplastic anemia (SAA)
* Myelodysplastic features
* Karnofsky performance status ≥ 70% if ≥ 16 years of age or Lansky play score
≥ 50% for patients \<16 years of age
* Adequate pulmonary, cardiac and liver function
* Voluntary written consent (minor assent if appropriate) prior to the performance of any study related procedures not part of standard medical care
Exclusion Criteria:
* Pregnant or breastfeeding as the treatment used in this study are Pregnancy Category D. Females of childbearing potential must have a negative pregnancy test (serum or urine) within 14 days of study registration
* Active, uncontrolled infection within 1 week prior to starting study therapy
* Malignant solid tumor cancer within previous 2 years
Donor Selection (I…
What they're measuring
1
Grade II-IV acute graft versus host disease (GVHD)
Timeframe: Day 100
Trial details
NCT IDNCT03579875
SponsorMasonic Cancer Center, University of Minnesota