Alpha/Beta TCD HCT in Patients With Inherited BMF Disorders

Recruiting Phase 2 Interventional Study
Fanconi Anemia Severe Aplastic Anemia Myelodysplastic Syndromes T Cell Receptor Alpha/Beta Depletion Telomere Biology Disorder Bone Marrow Failure Dyskeratosis Congenita Telomere Biology Disorders
No Placebo Group Every participant receives an active treatment — no one gets a placebo. Prior Safety Data This treatment has already been tested in at least one earlier human trial.
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At a Glance
Age
Up to 65
Sex
Any
Trial phase
Phase 2
Study type
Interventional
Purpose
Treatment
Participants needed
48 (estimated)
Sponsor
Masonic Cancer Center, University of Minnesota · Other
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About This Trial

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.

Trial Locations
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Eligibility Criteria
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…
Contacts

Margaret MacMillan, MD, Msc, FRCPC

612-626-2961

macmi002@umn.edu

CONTACT