Fludarabine Based RIC for Bone Marrow Failure Syndromes

EARLY_PHASE1RecruitingINTERVENTIONAL
Enrollment

25

Participants

Timeline

Start Date

April 30, 2015

Primary Completion Date

December 31, 2026

Study Completion Date

December 31, 2027

Conditions
Bone Marrow Failure Syndromes
Interventions
OTHER

MRD-BMT with Fludarabine-based RIC for Acquired AA

"Fludarabine: Dose: 30mg/m2/day (\<10kg will receive 1mg/kg/day) Days: -7, -6, -5, -4, -3~Cyclophosphamide: Dose: 60mg/kg/day Days: -5, -4~Thymoglobulin: Dose: 3mg/kg/day Days: -4, -3, -2~Bone marrow infusion: Day 0"

OTHER

MRD-BMT with Fludarabine-based RIC for iBMF with trilineage aplasia

"Fludarabine: Dose: 30mg/m2/day (\<10kg will receive 1mg/kg/day) Days: -7, -6, -5, -4, -3~Cyclophosphamide: Dose: 10 mg/kg/day Days: -6, -5, -4, -3~Thymoglobulin: Dose: 3mg/kg/day Days: -4, -3, -2~Bone marrow infusion: Day 0"

OTHER

MRD-BMT with Fludarabine-based RIC for iBMF without trilineage aplasia

"Fludarabine: Dose: 30mg/m2/day (\<10kg will receive 1mg/kg/day) Days: -6, -5, -4, -3, -2~Busulfan: Dose: every 6 hours for a total of 12 doses with dosing adjustments to achieve a steady state concentration of 900-1200ng/mL OR daily for a total of 3 doses targeting AUC 3600-6000 (micromole/liter)\*minute Days: -7, -6, -5, -4~Thymoglobulin: Dose: 3mg/kg/day Days: -10, -9, -8~Bone marrow infusion: Day 0"

Trial Locations (1)

19104

RECRUITING

Children's Hospital of Philadelphia, Philadelphia

All Listed Sponsors
lead

Children's Hospital of Philadelphia

OTHER