RecruitingPhase 1Phase 2NCT04008368

Repeat Peripheral Blood Stem Cell Transplantation for Patients With Sickle Cell Disease and Falling Donor Myeloid Chimerism Levels

Repeat Peripheral Blood Stem Cell Transplantation for Patients With Sickle Cell Disease or Beta-Thalassemia and Falling Donor Myeloid Chimerism Levels


Sponsor

National Heart, Lung, and Blood Institute (NHLBI)

Enrollment

32 participants

Start Date

Oct 24, 2019

Study Type

INTERVENTIONAL

Conditions

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

Min Age: 2 YearsMax Age: 80 Years

Plain Language Summary

Simplified for easier understanding

This study offers a repeat stem cell transplant (using peripheral blood stem cells) for patients with Sickle Cell Disease (SCD) or beta thalassemia who previously received a stem cell transplant but are now showing signs that the disease is returning. This can happen when the donor's cells are being gradually replaced by the patient's own diseased cells — a situation called falling donor chimerism. A successful stem cell transplant can effectively cure sickle cell disease and thalassemia, but sometimes the donor cells don't fully take hold long-term. When disease symptoms return, a second transplant from the same related donor may restore the benefit. This study tests whether this 'boost' transplant is safe and effective. **You may be eligible if...** - You have a history of SCD or beta thalassemia and previously had an allogeneic stem cell transplant - Your disease has recurred with clinical symptoms (e.g., painful crises, anemia, transfusion dependence) - You are 4 years of age or older - Your heart ejection fraction is at least 35% - Your lung function (DLCO) is at least 35% - Your related donor from the original transplant is willing and eligible to donate again **You may NOT be eligible if...** - You have a very poor performance status (ECOG 3 or higher) - You have an active uncontrolled infection - You are pregnant or breastfeeding - You have had a secondary cancer (other than skin cancer) - You have strong antibodies against the donor's tissue type (HLA) **Talk to your doctor** about your transplant history, current disease status, and whether your original donor is available and suitable for a repeat donation.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DEVICECliniMACS CD34 Reagent

Haploidentical recipients will receive CD34-selected cells using Miltenyi CliniMACS(R) CD34+ cell selection kits. The target CD34+ cell dose is at least 10 x 106/kg, and the minimum CD34+ cell dose is 5 x 106/kg. All of the cells collected during the apheresis procedure will be given. The cells will be cryopreserved and stored until the day of transplant.


Locations(1)

National Institutes of Health Clinical Center

Bethesda, Maryland, United States

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NCT04008368