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

Conditions

• Myeloid Chimerism

Eligibility Criteria

Sex

ALL

Age

2 Years - 80 Years

Healthy Volunteers

Accepted

Interventions

• CliniMACS CD34 Reagent — DEVICE
  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.

Study Details

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....

Key Dates

Start date

Oct 24, 2019

Status verified

Feb 2026

Primary completion

Jan 30, 2030

Completion

Dec 1, 2037

Study Design

Enrollment

32 participants (estimated)

Allocation

NON_RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Arms

• Other: 1
  patients with HLA-matched sibling donors
• Other: 2
  patients with haploidentical donors

Primary Outcome Measure

dichotomous positive/negative outcome where a positive response is defined by absence of graft rejection [ Time Frame: 5 years ]

Central Contacts

• Julia M Varga, R.N.
  (301) 402-3595
  [email protected]
• Courtney F Joseph, M.D.
  (301) 402-6496
  [email protected]

Locations (1)

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