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Nonmyeloablative Haploidentical Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease

StatusActive, Not Recruiting
PhasePhase 1/2
Started
View on ClinicalTrials.gov ↗

Record History

The 50 most recent of 144 record versions are accounted for below. See every version on ClinicalTrials.gov ↗

  1. Minor Secondary endpoints revised (10)v144
  2. Notable Results first posted: Aug 7, 2024v143 Show 5 more changesShow less
    • Primary endpoint revised
    • Enrollment target: 55 to 57
    • Interventions: Hydroxyurea (Drug), campath (Drug), cyclophosphamide (Drug), haploidentical stem cell transplant (Procedure), pentostatin (Drug), sirolimus… to Alemtuzumab (Drug), Cyclophosphamide (Drug), Filgrastim (Drug), Hydroxyurea (Drug), Pentostatin (Drug), Sirolimus (Drug), haploidentical…
    • Secondary endpoints: 1 to 10
    • Study arms: 1 to 2
  3. Minor 2 versions Re-verified since Oct 27, 2023, no change to tracked fieldsv141-v142
  4. Minor Trial status: Recruiting to Active, Not Recruiting
    • Enrollment target: estimated 98 to confirmed 55
    • Primary completion date changed from estimate of Sep 30, 2025 to Jul 27, 2023
    • Site details revised
    v140
  5. Minor 45 versions Re-verified since May 12, 2022, no change to tracked fieldsv95-v139
  • Stopped: an ongoing trial was terminated, suspended or withdrawn.
  • Notable: the trial’s plan changed such as a big move in its timeline or size, a new primary endpoint, sponsor or phase, or recruiting opened or resumed.
  • Minor: routine progress and housekeeping, such as sites added or enrollment closed.
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.
Trial Details
NCT Number NCT03077542
Lead Sponsor National Heart, Lung, and Blood Institute (NHLBI)
Conditions
(from sponsor)
Sickle Cell Disease
Related terms Anemia, Sickle Cell, Graft vs Host Disease
Enrollment 57 participants
Start Date
Primary Completion (estimated)
Study Completion (estimated)
Updated on ClinicalTrials.gov