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Haploidentical Donor Hematopoietic Cell Transplant for Sickle Cell Disease

NCT07616154 · Status: NOT_YET_RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 45

Last updated 2026-06-01

No results posted yet for this study

Summary

The purpose of this study it to evaluate a reduced toxicity conditioning regimen for haploidentical donor HCT followed by a GVHD prophylaxis regimen comprising of post-transplant cyclophosphamide, sirolimus and abatacept with the goal to improve the GVHD-free rejection-free survival (GRFS) to greater than 90% after haploidentical donor HCT in children and young adults with SCD.

Primary Objective:

\- To assess the GVHD-free and rejection free survival (GRFS) after haploidentical donor HCT in children and young adults with SCD.

Secondary Objectives:

* Assess the overall survival (OS) and disease-free survival (DFS) after haploidentical donor HCT for SCD.
* Estimate incidence and severity of acute and chronic GVHD after haploidentical donor HCT for SCD.
* Assess the neutrophil and platelet engraftment kinetics after haploidentical donor HCT for SCD.

Conditions

Interventions

DRUG

Cyclophosphamide 50mg

IV

DRUG

Abatacept

IV

DRUG

Alemtuzumab

IV

DRUG

Hydroxyurea

IV

DRUG

Thiotepa

IV

DRUG

Filgrastim

IV

DRUG

Sirolimus

IV

DRUG

Azathioprine

IV

RADIATION

TBI

Radiaiton therapy

PROCEDURE

Hematopoietic Progenitor Cell Infusion

Hematopoietic Progenitor Cell Infusion

Sponsors & Collaborators

Principal Investigators

  • Akshay Sharma, MD · St. Jude Children's Research Hospital

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Max Age
22 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2026-09-30
Primary Completion
2034-09-30
Completion
2035-09-30
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Drugs
Diseases
Companies

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 NCT07616154 on ClinicalTrials.gov