Recruiting

Bone marrow transplant using specially processed donor cells for severe blood diseases

Plain-English translation of NCT03653338 on ClinicalTrials.gov ↗ · Source last updated July 2026 · Translation generated July 2026 · How we translate trials

Phase 1/2 — A combined trial that checks safety and dosing while also starting to look at whether the treatment works.

What is this trial?

This trial is testing a bone marrow transplant approach for people with severe sickle cell disease and other serious blood disorders. The transplant uses donor cells that have been specially processed in the lab to remove certain immune cells that could cause problems after transplant. The goal is to help patients who don't have a perfectly matched donor available by using donors that are only partially matched.

Many patients with sickle cell disease and other life-threatening blood disorders need a bone marrow transplant to be cured, but they can't find a perfectly matched donor. This trial exists to test a new method that removes harmful immune cells from partially matched donor cells, which could help more patients get access to life-saving transplants while reducing dangerous side effects.

Do you qualify?

You likely qualify if…

you are between 5 and 40 years old
you have been diagnosed with sickle cell disease (SS or S-beta-zero-thalassemia) with serious complications like frequent pain crises, chest syndrome episodes, stroke, or organ damage
you have beta-thalassemia major or Diamond-Blackfan anemia and need regular blood transfusions due to iron buildup
you have searched for a perfectly matched or well-matched bone marrow donor and none was available
your kidneys, liver, heart, and lungs are working well enough to handle the transplant procedure

You likely don't qualify if…

you have a perfectly matched sibling or unrelated bone marrow donor already available
you have human immunodeficiency virus (HIV) or active untreated infections
your heart, lung, kidney, or liver function is too severely damaged
you are unable to give informed consent or have a guardian unable to consent for you

What participation looks like

You would receive conditioning therapy (medicines to prepare your body for the transplant), then receive an infusion of specially processed donor bone marrow cells. The donor cells will have been treated in the lab to remove most immune cells while keeping some beneficial cells frozen for later use if needed. You would need close follow-up care in the hospital and then as an outpatient to monitor how well the transplant is working and manage any side effects.

AI-generated summary from trial data · Jul 14, 2026 · Not medical advice

Bone marrow transplant using specially processed donor cells for severe blood diseases

What is this trial?

Do you qualify?

What participation looks like

Trial locations(1 site)