Study of HLA-Haploidentical Stem Cell Transplantation to Treat Clinically Aggressive Sickle Cell Disease

PHASE2RecruitingINTERVENTIONAL
Enrollment

50

Participants

Timeline

Start Date

March 20, 2017

Primary Completion Date

November 30, 2025

Study Completion Date

November 30, 2025

Conditions
Sickle Cell Disease
Interventions
DRUG

ATG

0.5 mg/kg IV on day -9, and 2 mg/kg on days -8 and day -7

DRUG

fludarabine

30 mg/m2 IVPB daily for day -6 (6 days before stem cell infusion) through day -2

DRUG

cyclophosphamide

14.5 mg/kg IV on days -6 and -5 and 50 mg/kg/d on days +3 and +4

RADIATION

Total body irradiation

3 Gy on day -1

PROCEDURE

Stem cell infusion

Stem cell product infused according to BMT unit policy on day 0.

DRUG

Sirolimus

loading dose of 15 mg followed by 5 mg per day on day +5

DRUG

mycophenolate mofetil

1 g every 8 h (until day 35) will be started on day 5

Trial Locations (1)

60612

RECRUITING

University of Illinois at Chicago, Chicago

All Listed Sponsors
lead

University of Illinois at Chicago

OTHER