Recruiting

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

Plain-English translation of NCT03653338 on ClinicalTrials.gov ↗ · Source last updated August 2025 · Translation generated June 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 new approach to bone marrow transplants for people with severe blood disorders like sickle cell disease and thalassemia. The key innovation is using donor cells that have been specially processed in the laboratory—specifically, certain immune cells are removed before transplant to reduce dangerous side effects while still allowing the new bone marrow to take hold. This treatment aims to help patients who don't have perfectly matched donors available.

Many patients with severe sickle cell disease and other serious blood disorders desperately need a bone marrow transplant to be cured, but they don't have a perfectly matched sibling or unrelated donor. This trial exists to make transplants possible for these patients by using less-perfect donor matches while removing problematic immune cells beforehand to make the transplant safer and more likely to succeed.

Do you qualify?

You likely qualify if…

you are between 5 and 40 years old at the time of enrollment
you have been diagnosed with severe sickle cell disease (types SS or Sβ0-thalassemia) with serious complications like recurrent pain crises, stroke, or need for frequent blood transfusions, OR you have beta-thalassemia major or Diamond-Blackfan anemia requiring regular transfusions
you do not have a perfectly matched related donor or an 8/8 matched unrelated donor, but you do have at least a partial match (4/8 from a relative or 6/8 from an unrelated donor)
your heart, lungs, kidneys, and liver are working well enough to safely undergo the transplant procedure, as confirmed by specific tests
you have completed a search for cord blood donors without finding a suitable option, or available cord blood has too few cells

You likely don't qualify if…

you are currently living with untreated HIV infection
you have severe organ damage (such as cirrhosis of the liver, severe heart failure, or severe lung disease) that would make the transplant too risky
your kidney function is too poor (creatinine clearance below 45 ml/min/1.73m)
you have a life-threatening infection that is not controlled

What participation looks like

If you join this trial, you will receive chemotherapy and other conditioning treatments to prepare your body for the transplant, followed by an infusion of processed bone marrow cells from your donor. The donor cells will have had certain immune cells removed in the laboratory to reduce the risk of a serious complication called graft-versus-host disease. After the transplant, you'll need close monitoring in the hospital and regular follow-up visits to watch for how well the new bone marrow is working and to manage any side effects. The exact timeline and frequency of visits will be explained by your doctor.

AI-generated summary from trial data · Jun 3, 2026 · Not medical advice

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

What is this trial?

Do you qualify?

What participation looks like

Trial locations(1 site)