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Phase 2
MT2017-17:T Cell Receptor Alpha/Beta T Cell Depleted Hematopoietic Cell Transplantation in Patients With Inherited Bone Marrow Failure (BMF) Disorders
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 ma…
Fanconi AnemiaSevere Aplastic AnemiaMyelodysplastic Syndromes+5 more
Masonic Cancer Center, University of MinnesotaNCT03579875
The Natural History of Acquired and Inherited Bone Marrow Failure Syndromes
Background: Bone marrow failure diseases are rare. Much is known about the diseases at the time of diagnosis, but long-term data about the effects of the dise…
Severe Aplastic AnemiaTelomere Biology DisordersInherited Bone Marrow Failure Syndromes
National Heart, Lung, and Blood Institute (NHLBI)NCT05012111
Phase 1
Nucleoside Therapy in Patients With Telomere Biology Disorders
The goal of this clinical trial is to learn if a combination therapy of deoxycytidine (dC) plus deoxythymidine (dT) is safe in patients with telomere biology d…
Telomere Biology DisordersDyskeratosis CongenitaRevesz Syndrome+4 more
Suneet AgarwalNCT06817590