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A Blood Stem Cell Transplant for Sickle Cell Disease
NCT03249831 · View on ClinicalTrials.gov ↗
Study Summary
Blood stem cells can produce red blood cells (which carry oxygen), white blood cells of the immune system (which fight infections) and platelets (which help the blood clot). Patients with sickle cell disease produce abnormal red blood cells. A blood stem cell transplant from a donor is a treatment option for patients with severe sickle cell disease. The donor can be healthy or have the sickle cell trait. The blood stem cell transplant will be given to the patient as an intravenous infusion (IV). The donor blood stem cells will then make normal red blood cells - as well as other types of blood cells - in the patient. When blood cells from two people co-exist in the patient, this is called mixed chimerism. Most children are successfully treated with blood stem cells from a sibling (brother/sister) who completely shares their tissue type (full-matched donor). However, transplant is not an option for patients who (1) have serious medical problems, and/or (2) do not have a full-matched donor. Most patients will have a relative who shares half of their tissue type (e.g. parent, child, and brother/sister) and can be a donor (half-matched or haploidentical donor). Adult patients with severe sickle cell disease were successfully treated with a half-matched transplant in a clinical study. Researchers would like to make half-matched transplant an option for more patients by (1) improving transplant success and (2) reducing transplanted-related complications. This research transplant is being tested in this Pilot study for the first time. It is different from a standard transplant because: 1. Half-matched related donors will be used, and 2. A new combination of drugs (chemotherapy) that does not completely wipe out the bone marrow cells (non-myeloablative treatment) will be used to prepare the patient for transplant, and 3. Most of the donor CD4+ T cells (a type of immune cells) will be removed (depleted) before giving the blood stem cell transplant to the patient to impro
Conditions Studied
Interventions
- DRUG Cyclophosphamide
- DRUG Tacrolimus
- DRUG Mycophenolate mofetil
- DRUG Pentostatin
- DRUG Rabbit anti-thymocyte globulin
Study Locations (1)
California
- City of Hope Medical Center — Duarte
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 3 participants |
| Start Date | 2019-01-04 |
| Est. Completion | 2027-01-25 |
| Phase | Phase 1 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT03249831
The ClinicalTrials.gov registry entry for NCT03249831 describes a study currently listed as active not recruiting. It is categorized as Phase 1, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 3 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is City of Hope Medical Center, which has 771 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 5 conditions, with Sickle Cell Disease appearing as the primary indexed condition, and to 5 interventions — of which Cyclophosphamide is the first listed. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT03249831 reports 1 study location spanning 1 distinct geographic area — top geographies include California. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
Frequently Asked Questions
What is clinical trial NCT03249831 about?
NCT03249831 is a clinical study titled "A Blood Stem Cell Transplant for Sickle Cell Disease". Blood stem cells can produce red blood cells (which carry oxygen), white blood cells of the immune system (which fight infections) and platelets (which help the blood clot). Patients with sickle cell disease produce abnormal red blood cells. A blood stem cell transplant from a donor is a treatment ...
What is the current status of trial NCT03249831?
This trial is currently active not recruiting. It is a Phase 1 study. The enrollment target is 3 participants. The study started on 2019-01-04. Estimated completion is 2027-01-25.
What conditions does trial NCT03249831 study?
This clinical trial studies the following conditions: Sickle Cell Disease, Thalassemia, Hemoglobinopathies, Anemia, Sickle Cell, Sickle Cell Disorder. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT03249831?
The interventions under investigation include: Cyclophosphamide (DRUG), Tacrolimus (DRUG), Mycophenolate mofetil (DRUG), Pentostatin (DRUG), Rabbit anti-thymocyte globulin (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT03249831?
This trial is sponsored by City of Hope Medical Center, which has 771 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT03249831 being conducted?
This trial has 1 study location across California. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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