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

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