RecruitingEarly Phase 1

Fludarabine Based RIC for Bone Marrow Failure Syndromes

United States25 participantsStarted 2015-04

Plain-language summary

This is a pilot study to determine whether fludarabine-based reduced intensity conditioning (RIC) regimens facilitate successful donor engraftment of patients with acquired aplastic anemia (AA) and Inherited bone marrow failure (iBMF) syndromes undergoing Matched related donor bone marrow transplant (MRD-BMT).

Who can participate

Age range22 Years

SexALL

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Inclusion criteria

✓. Ages 0-22 years at time of enrollment
✓. Diseases:
✓. Lansky or Karnofsky performance \>60
✓. HLA matched related donor available.
✓. No active untreated infection
✓. Females of childbearing potential must have negative pregnancy test.

What they're measuring

Rate of graft failure

Timeframe: Up to 1 year post transplant

Time to neutrophil engraftment

Timeframe: Up to 1 year post transplant

Transplant-related mortality

Timeframe: Up to 100 days post transplant

Trial details

NCT IDNCT02928991

SponsorChildren's Hospital of Philadelphia

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2026-12

Contact for this trial

Megan Atkinson

215-590-2820 cttsbmtintake@chop.edu

View on ClinicalTrials.gov More Bone Marrow Failure Syndromes trials