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NCT03249831 · ClinicalTrials.gov registry record · Phase 1
A Blood Stem Cell Transplant for Sickle Cell Disease
A Phase 1 study of Sickle Cell Disease and Thalassemia, sponsored by City of Hope Medical Center.
- Active
- Registry status
- Phase 1
- Development phase
- 3
- Enrollment target
- 1
- Study location
NCT03249831: Active Phase 1 study of Sickle Cell Disease and Thalassemia, sponsored by City of Hope Medical Center.
NCT03249831 is a Phase 1 study of Sickle Cell Disease and Thalassemia that is active but no longer recruiting, run by City of Hope Medical Center. The registered enrollment target is 3 participants, below the 293-participant average among 213 other Sickle Cell Disease trials with a reported enrollment target (99% lower). The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03249831, a Phase 1 study of Sickle Cell Disease and Thalassemia, is active but no longer recruiting, sponsored by City of Hope Medical Center.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 1
- Development phase
- 3 participants
- Enrollment target
- 1
- Study location
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 |
What the registry record for NCT03249831 still lists
NCT03249831 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 3 participants, a relatively small participant target, below the 293-participant average among 213 other Sickle Cell Disease trials with a reported enrollment target (99% lower).
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.
NCT03249831 reports a single indexed study location in California.
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.
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).
Who is sponsoring clinical trial NCT03249831?
This trial is sponsored by City of Hope Medical Center, which has 582 total clinical trials registered on ClinicalTrials.gov.
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.
Learn More About Clinical Trials
Similar trials for Sickle Cell Disease
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT03249831's enrollment target sits among peer trials
3 212th of 213 higher than 2 of 213 other Sickle Cell Disease trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Sickle Cell Disease trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
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