NCT03121001

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

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

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 50 (estimated)
• Sponsor: University of Illinois at Chicago · Academic / Other
• Sex: All
• Age: 16 Years – 60 Years
• Healthy volunteers: Not accepted

Summary

The study is a Phase II clinical trial. Patients will receive intensity modulated total body irradiation (TBI) at a dose of 3 Gy with standard fludarabine/ i.v. cyclophosphamide conditioning prior to human leukocyte antigen (HLA)-haploidentical hematopoietic stem cell transplant (HSCT). The primary objective of the study is to determine the engraftment at Day +60 following HLA-haploidentical hematopoietic stem cell transplant protocol using immunosuppressive agents and low-dose total body irradiation (TBI) for conditioning and post-transplant cyclophosphamide in patients with sickle cell disease.

Conditions

• Sickle Cell Disease

Interventions

Type	Name	Description
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

Timeline

• Start date: 2017-03-20
• Primary completion: 2026-05-01
• Completion: 2026-05-01
• First posted: 2017-04-19
• Last updated: 2026-01-26

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study