Reduced Intensity Transplantation for Severe Sickle Cell Disease
Public ClinicalTrials.gov record NCT04362293. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
Reduced Intensity Related Donor Peripheral Blood Derived Hematopoietic Progenitor Cell Transplantation for Patients With Severe Sickle Cell Disease
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
This study is being done to test a transplant method that may have fewer side effects (or less toxic, less harmful) than conventional high dose chemotherapy conditioning-based transplants for children and young adults with Sickle Cell Disease (SCD). Patients less than or equal to 25 years old with SCD who would likely benefit from allogeneic hematopoietic cell transplantation (HCT) will be included in this study. Patients with a suitable HLA matched sibling donor (MSD) will be enrolled on the MSD arm while patients without an eligible MSD who have a suitable haploidentical (HAPLO) donor available will be enrolled on the HAPLO arm of the study. Primary Objective To assess the donor T-cell chimerism at 1-year post transplant in each respective arm (MSD, HAPLO) of the trial. Secondary Objectives * Assess the overall survival and 1-year, 2-year and 3-year post-transplant graft versus host disease (GVHD)-free SCD-free survival. * Estimate the primary and secondary graft rejection rate at 1-year, 2-year and 3-year post- transplant. * Estimate the incidence and severity of acute and chronic (GVHD). * Estimate the incidence of SCD recurrence after transplant * Assess the neutrophil and platelet recovery kinetics post-transplant. Exploratory Objectives * Record immune reconstitution parameters, including chimerism analysis, quantitative lymphocyte subsets, T cell receptor excision circle (TREC) analysis, V-beta spectratyping, and lymphocyte phenotype and function. * Conduct longitudinal examination of impact of HCT on patient health-related quality of life (HRQL) and adjustment, and parental adjustment. * Examine impact of HCT on patient cognitive and academic function. * Determine factors that influenced the decision to undergo HCT, explore perceptions of the HCT experience, and assess decisional satisfaction/regret. * Develop and evaluate an objective/quantitative imaging biomarker to assess organ (liver and heart) function/disease status and changes following HCT. * Develop and evaluate an objective/quantitative imaging biomarker to determine cerebral blood flow and oxygen extraction fraction following HCT.
Study identification
- NCT ID
- NCT04362293
- Recruitment status
- Active, not recruiting
- Study type
- Interventional
- Phase
- Phase 2
- Enrollment
- 46 participants
Conditions and interventions
Eligibility (public fields only)
- Age range
- 2 Years to 25 Years
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Apr 29, 2020
- Primary completion
- Jun 15, 2026
- Completion
- Jul 31, 2029
- Last update posted
- Aug 31, 2026
2020 – 2029
United States locations
- U.S. sites
- 1
- U.S. states
- 1
- U.S. cities
- 1
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| St. Jude Children's Research Hospital | Memphis | Tennessee | 38105 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT04362293, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Aug 31, 2026 · Synced Sep 10, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT04362293 live on ClinicalTrials.gov.