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NCT03077542 · ClinicalTrials.gov registry record · Phase 1
Nonmyeloablative Haploidentical Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease
A Phase 1 study of Sickle Cell Disease, sponsored by National Heart, Lung, and Blood Institute (NHLBI).
- Active
- Registry status
- Phase 1
- Development phase
- 57
- Enrollment target
- 1
- Study location
NCT03077542: Active Phase 1 study of Sickle Cell Disease, sponsored by National Heart, Lung, and Blood Institute (NHLBI).
NCT03077542 is a Phase 1 study of Sickle Cell Disease that is active but no longer recruiting, run by National Heart, Lung, and Blood Institute (NHLBI). The registered enrollment target is 57 participants, below the 293-participant average among 213 other Sickle Cell Disease trials with a reported enrollment target (81% lower). The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03077542, a Phase 1 study of Sickle Cell Disease, is active but no longer recruiting, sponsored by National Heart, Lung, and Blood Institute (NHLBI).
- ACTIVE NOT RECRUITING
- Registry status
- Phase 1
- Development phase
- 57 participants
- Enrollment target
- 1
- Study location
Study Summary
Background: Peripheral blood stem cell transplantation procedures are used for people with sickle cell disease. Researchers want to improve the success and reduce the complications for these procedures. This might allow more people to have a transplant. Objective: To see if a new transplant regime is effective, safe and well tolerated in people with sickle cell disease. Eligibility: Adults at least 18 years old with sickle cell disease and certain complications. A relative who is a half tissue match. Design: Participants will be screened with medical history, physical exam, and blood tests. Recipients will also have: * Heart, lung, and mental health tests * Chest x-rays * Bone marrow taken from the pelvic bone * Eyes and teeth checked Recipients will have a large central line inserted into a vein for up to 6 months. Donors will have their veins tested and have an IV inserted for 1 day or on rare occasions 2 days. Donors will get a drug to activate bone marrow. It will be injected for about 6 days. Donors will have at least 1 five-hour procedure where bone marrow stem cells will be collected. Blood will be taken from a vein in one arm or in rare cases from a groin vein and put through a machine. Some blood will be saved and the rest will be returned. Stem cells will be taken from the saved blood in a lab and frozen until ready to give to the recipient. Recipients will have: * Stems cells collected and frozen * Hygiene lessons * Bone density scans * Low-dose radiation * Drugs for their immune system * Donor cells infused through their central line * Transfusions After about 30 days, recipients will leave the hospital. They must stay near NIH for 3 months after the transplant and have frequent visits. After returning home, they will have 8 visits over 5 years, then be contacted yearly.
Primary Outcome
The percentage of sickle cell participants at 100 days post-transplant who have not rejected their grafts and who are without severe graft-versus-host disease (GVHD). Severe GVHD is defined as grade 3 or higher for acute GVHD and moderate to severe for chronic GVHD according to NIH Consensus Criteria. Stem cell graft rejection is defined as
Conditions Studied
Interventions
- DRUG Cyclophosphamide
- DRUG Sirolimus
- DRUG Alemtuzumab
- DRUG Pentostatin
- PROCEDURE haploidentical stem cell transplant
Study Locations (1)
Maryland
- National Institutes of Health Clinical Center - Bethesda
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 57 participants |
| Start Date | 2017-04-06 |
| Est. Completion | 2026-08-31 |
| Phase | Phase 1 |
What the registry record for NCT03077542 still lists
NCT03077542 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 57 participants, a relatively small participant target, below the 293-participant average among 213 other Sickle Cell Disease trials with a reported enrollment target (81% lower).
The record links to 1 condition, with Sickle Cell Disease appearing as the primary indexed condition, and to 5 interventions - of which Cyclophosphamide is the first listed.
NCT03077542 reports a single indexed study location in Maryland.
Frequently Asked Questions
What is clinical trial NCT03077542 about?
NCT03077542 is a clinical study titled "Nonmyeloablative Haploidentical Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease". Background: Peripheral blood stem cell transplantation procedures are used for people with sickle cell disease. Researchers want to improve the success and reduce the complications for these procedures. This might allow more people to have a transplant. Objective: To see if a new transplant regim...
What is the current status of trial NCT03077542?
This trial is currently active not recruiting. It is a Phase 1 study. The enrollment target is 57 participants. The study started on 2017-04-06. Estimated completion is 2026-08-31.
What conditions does trial NCT03077542 study?
This clinical trial studies the following conditions: Sickle Cell Disease.
What interventions are being tested in trial NCT03077542?
The interventions under investigation include: Cyclophosphamide (DRUG), Sirolimus (DRUG), Alemtuzumab (DRUG), Pentostatin (DRUG), haploidentical stem cell transplant (PROCEDURE).
Who is sponsoring clinical trial NCT03077542?
This trial is sponsored by National Heart, Lung, and Blood Institute (NHLBI), which has 501 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT03077542 being conducted?
This trial has 1 study location across Maryland. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
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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 NCT03077542's enrollment target sits among peer trials
57 97th of 213 higher than 116 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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