NCT04008368

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

Recruiting now Phase 1, PHASE2 Last updated 5 February 2026

What this trial tests

Phase 1, PHASE2 trial testing CliniMACS CD34 Reagent in Myeloid Chimerism in 32 participants. Currently enrolling.

Timeline

24 October 2019

Primary endpoint
30 January 2030

1 December 2037

Quick facts

Lead sponsor	National Heart, Lung, and Blood Institute (NHLBI)
Phase	Phase 1, PHASE2
Status	Recruiting now
Study type	INTERVENTIONAL
Allocation	non randomized
Design	parallel
Masking	none
Primary purpose	treatment
Enrollment	32
Start date	24 October 2019
Primary completion	30 January 2030
Estimated completion	1 December 2037
Sites	1 location across United States

Drugs / interventions tested

• CliniMACS CD34 Reagent

Conditions studied

• Myeloid Chimerism — all drugs for Myeloid Chimerism →

Sponsor

National Heart, Lung, and Blood Institute (NHLBI)

Who can join

Adults 2 to 80, any sex, with Myeloid Chimerism. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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....

Publications & conference data

1 peer-reviewed publication reference this trial (live from Europe PMC):

1. Nonmyeloablative pentostatin-cyclophosphamide preconditioning improves rates of engraftment in adults undergoing haploidentical HCT for sickle cell disease.
   Limerick E, Hsieh M, Queen J, Grecco ML, et al · · 2026 · PMID 41871072 · DOI 10.1371/journal.pone.0332282