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

Part of paid clinical trials in Chicago, Illinois.

Sponsor
University of Illinois at Chicago
Study ID
NCT03121001
Phase
PHASE2
Status
Recruiting
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Conditions

Eligibility Criteria

Sex
ALL
Age
16 Years - 60 Years
Healthy Volunteers
Not accepted

Interventions

  • ATG — DRUG
    0.5 mg/kg IV on day -9, and 2 mg/kg on days -8 and day -7
  • fludarabine — DRUG
    30 mg/m2 IVPB daily for day -6 (6 days before stem cell infusion) through day -2
  • cyclophosphamide — DRUG
    14.5 mg/kg IV on days -6 and -5 and 50 mg/kg/d on days +3 and +4
  • Total body irradiation — RADIATION
    3 Gy on day -1
  • Stem cell infusion — PROCEDURE
    Stem cell product infused according to BMT unit policy on day 0.
  • Sirolimus — DRUG
    loading dose of 15 mg followed by 5 mg per day on day +5
  • mycophenolate mofetil — DRUG
    1 g every 8 h (until day 35) will be started on day 5

Study Details

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.

Key Dates

Start date
Mar 20, 2017
Status verified
Jan 2026
Primary completion
May 31, 2026
Completion
May 31, 2026

Study Design

Enrollment
50 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Subject treatment
    Patients will receive the following conditioning regimen: ATG, fludarabine (6 days before stem cell infusion), cyclophosphamide, and total body irradiation. The stem cell product will be infused according to BMT unit policy. Patients will also receive GVHD prophylaxis which will consist of cyclophosphamide, sirolimus, and mycophenolate mofetil according to the protocol. Post-transplant evaluation will be done as per standard care with study data collected at days 30, 60, 100, 180, 365, and annually thereafter.

Primary Outcome Measure

Estimate the number of patients who engraft by Day +60 [ Time Frame: Up to Day +60 ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
University of Illinois at ChicagoChicagoIllinois60612
Damiano Rondelli, MD
[email protected]
312-413-3547

Find similar trials in Chicago, IL

By condition
Sickle cell disease
By research site
University of Illinois at Chicago· Chicago, IL

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