NCT04008368 Repeat Peripheral Blood Stem Cell Transplantation for Patients With Sickle Cell Disease and Falling Donor Myeloid Chimerism Levels
| NCT ID | NCT04008368 |
| Status | Recruiting |
| Phase | Phase 1, Phase 2 |
| Sponsor | National Heart, Lung, and Blood Institute (NHLBI) |
| Condition | Myeloid Chimerism |
| Study Type | INTERVENTIONAL |
| Enrollment | 32 participants |
| Start Date | 2019-10-24 |
| Primary Completion | 2030-01-30 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 1 is the earliest stage of human testing — safety and dosage are the primary focus. Visits are frequent and medical supervision is intensive. You will be among the first people to receive this treatment.
This trial targets 32 participants in total. It began in 2019-10-24 with a primary completion date of 2030-01-30.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
Background: Sickle cell disease can often be treated with blood stem cell transplants. But for some people the disease returns. This study will give a second transplant to people whose disease has returned but still have some donor cells in their body. Objective: To cure people s sickle cell disease by giving a second treatment that makes more room in their bone marrow for donor cells. Eligibility: People ages 4 and older with sickle cell disease who had a transplant but the disease returned, and their donor relatives. Donors can be 2 years of age or older. Design: Participants will be screened with medical history, physical exam, and blood tests. Recipients will also be screened with heart and breathing tests, x-rays, a bone marrow sample, and teeth and eye exams. They must have a caregiver. Donors will have 7-8 visits. They will take a drug for 5-6 days to prepare them for the donation. For the donation, blood is taken from a vein in the arm or groin. The stem cells are collected. The rest of the blood is returned. This may be repeated. Recipients will get a long IV line in their arm or chest for about 1-2 months. They will take drugs to help their body accept the donor cells. They will get the donor cells and red blood cell transfusions through the line. They will stay in the hospital about 30 days after the transfusion of donor cells. In first 3 months after the infusion, recipients will have many visits. Then they will have visits every 6 months to 1 year for 5 years. During those visits they will repeat some of the screening tests....
Eligibility Criteria
* INCLUSION CRITERIA: Inclusion criteria- recipient 1. Patient with history of SCD or beta thalassemia who previously underwent allogeneic hematopoietic stem cell transplantation (HSCT) 2. Patient with recurrent SCD defined as HbS greater than or equal to 50% for donors with sickle cell trait and greater than or equal to 10% for donors with HbAA with recurrent clinical manifestations (for example but not limited to recurrent painful crises, acute chest syndrome, priapism, or severe anemia) or patients with recurrent beta thalassemia defined as clinical manifestations such as transfusion-dependence or evidence of extramedullary hematopoiesis. The HbS requirement may be waived by the PI or designee for reasons such as the patient is requiring chronic transfusion therapy or otherwise meets clinical criteria for return of SCD. 3. Persistent donor chimerism levels 4. Age greater than or equal to 4 years 5. Negative beta-HCG 6. Ejection fraction greater than or equal to 35% 7. DLCO greater than or equal to 35% (Note: This criterion may be omitted in young children (e.g. near the age of 5 years old) or other individuals who may have difficulty understanding or complying with the instructions for accurate testing.) Inclusion- donor Related donor from original transplant, deemed suitable and eligible, and willing to donate, per clinical evaluations who are additionally willing to donate blood for research. Related donors will be evaluated in accordance with existing Standard NIH Policies and Procedures for determination of eligibility and suitability for clinical donation. Age \>= 2 years \<80 years old EXCLUSION CRITERIA: Exclusion criteria- recipient 1. ECOG performance status of 3 or more or if \<16 years of age Lansky score of 50 or lower. 2. Evidence of uncontrolled bacterial, viral, or fungal infections (currently taking medication and progression of clinical symptoms) within one month prior to signing the consent 3. Patients with fever or suspected minor infection should await resolution of symptoms before signing the consent 4. Major anticipated illness or organ failure incompatible with survival from PBSC transplant 5. Pregnant or breastfeeding 6. History of secondary malignancies (other than localized skin cancer) 7. Donor-specific HLA antibodies with mean fluorescent intensity \>= 2,000 Exclusion- donor None
Contact & Investigator
Courtney F Joseph, M.D.
PRINCIPAL INVESTIGATOR
National Heart, Lung, and Blood Institute (NHLBI)
Frequently Asked Questions
Who can join the NCT04008368 clinical trial?
This trial is open to participants of all sexes, aged 2 Years or older, up to 80 Years, studying Myeloid Chimerism. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT04008368 trial and what does that mean for participants?
Phase 1 trials are the first stage of human testing. The primary goal is to assess safety and determine appropriate dosage levels. Participants are closely monitored. These trials typically involve a small number of volunteers.
Is NCT04008368 currently recruiting?
Yes, NCT04008368 is actively recruiting participants. Contact the research team at julia.varga@nih.gov for enrollment information.
Where is the NCT04008368 trial being conducted?
This trial is being conducted at Bethesda, United States.
Who is sponsoring the NCT04008368 clinical trial?
NCT04008368 is sponsored by National Heart, Lung, and Blood Institute (NHLBI). The principal investigator is Courtney F Joseph, M.D. at National Heart, Lung, and Blood Institute (NHLBI). The trial plans to enroll 32 participants.