MedTrace Logo

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

NCT03121001 · Status: RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 50

Last updated 2026-01-26

No results posted yet for this study

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

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

Sponsors & Collaborators

  • University of Illinois at Chicago

    lead OTHER

Principal Investigators

  • Damiano Rondelli, MD · University of Illinois at Chicago

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
16 Years
Max Age
60 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2017-03-20
Primary Completion
2026-05-31
Completion
2026-05-31
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Drugs
Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03121001 on ClinicalTrials.gov