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4 recruiting studies matching your filters

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

Estimated

~$10 – $675

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

Estimated

~$10 – $8,750

Suneet AgarwalNCT06817590

Phase 2

Regenerative Medicine to Restore Hematopoiesis and Immune Function in Immunodeficiencies and Inherited Bone Marrow Failures

Phase II prospective trial to assess the rates of donor engraftment using reduced intensity conditioning (RIC) hematopoietic stem cell transplant (HSCT) and po…

Primary Immune Deficiency DisorderImmune Deficiency DiseaseBone Marrow Failure+7 more

Estimated

~$10 – $6,250

Sidney Kimmel Comprehensive Cancer Center at Johns HopkinsNCT04232085

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

Estimated

~$10 – $6,250

Masonic Cancer Center, University of MinnesotaNCT03579875