Olson Clinical Trial #4

Telomere diseases SCT Trial (IRB 17‐014074, NCT01659606): Radiation‐ and alkylator‐free hematopoietic cell transplantation for bone marrow failure due to dyskeratosis congenita / telomere disease (Agarwal, Boston Children’s Hospital)

Type: Multicenter Prospective Clinical Trial (Olson Site PI)

Status: Awaiting opening of expansion phase

Description: In this study the efficacy of a reduced intensity conditioning regimen will be evaluated in allogeneic hematopoietic cell transplantation (HCT) for bone marrow failure (BMF) due to dyskeratosis congenita (DC) and related telomere diseases. The objective is to determine whether it is feasible to use conditioning agents with decreased anticipated short- and long-term organ toxicity, while maintaining donor hematopoeisis at an acceptable level.

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