Ph I/II Study of Allogeneic SCT for Clinically Aggressive Sickle Cell Disease (SCD)

PHASE1/PHASE2Active, not recruitingINTERVENTIONAL
Enrollment

45

Participants

Timeline

Start Date

November 11, 2011

Primary Completion Date

September 23, 2028

Study Completion Date

October 31, 2029

Conditions
Sickle Cell Disease
Interventions
PROCEDURE

Allogeneic Non-Myeloablative Stem Cell Transplantation

"Alemtuzumab-based non-myeloablative allogeneic hematopoietic stem cell transplantation using immune-suppressive agents and low-dose total body irradiation (TBI) without standard chemotherapy.~Transplant regimen Day -7 to -3: Alemtuzumab (1mg/kg, total dose) divided over the 5 days, IVPB over 2 hours daily Day -3 until 100% chimerism obtained: Sirolimus dosed for target trough level of 10-15 ng/mL Day -2: Total body irradiation with 300cGy Day 0: Stem cell infusion"

DRUG

Alemtuzumab

In this protocol, patients will be given alemtuzumab 1mg/kg divided equally over five days with the maximum dose of 20mg per day.

DRUG

Sirolimus

On day -1, patients will receive a loading dose of 12 mg followed by 4 mg per day. Subsequent dosing will be based on clinical toxicity, GVHD concurrent medications, medical conditions, prior drug levels, drug-drug interactions, and blood levels with target of 3 to 12 ng/mL.

Trial Locations (1)

60612

University of Illinois at Chicago, Chicago

All Listed Sponsors
lead

University of Illinois at Chicago

OTHER

NCT01499888 - Ph I/II Study of Allogeneic SCT for Clinically Aggressive Sickle Cell Disease (SCD) | Biotech Hunter | Biotech Hunter