New stem cell transplant option for patients without a perfect donor match

NCT ID NCT02356653

Recruiting now Disease control Sponsor: Children's Hospital of Philadelphia Source: ClinicalTrials.gov ↗

First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This expanded access program offers a stem cell transplant using donor cells that have been specially processed to remove certain immune cells, reducing the risk of graft-versus-host disease. It is for patients with serious blood disorders, immune deficiencies, or metabolic diseases who lack a fully matched sibling donor and cannot join other clinical trials. Up to 100 participants will receive the transplant, and researchers will track survival, graft success, and complications.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: Donor stem cells with T-cell depletion (CliniMACs)
What this could lead to: If successful, this approach could provide a treatment option for patients with life-threatening blood or immune disorders who lack a fully matched donor.
What could go wrong: This is an early-phase expanded access protocol, not a controlled trial. Risks include graft failure, graft-versus-host disease, and infection. Results may not apply broadly.

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Conditions

The condition(s) this trial relates to.

acute leukemia Bone Marrow Failure Disorders bone marrow failure syndrome chronic leukemia hemoglobinopathy immune dysregulation-polyendocrinopathy-enteropathy-X-linked syndrome immunodeficiency disease inborn error of immunity inborn errors of metabolism leukemia lymphoma myelodysplastic syndrome

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

BONE MARROW FAILURE SYNDROMES IMMUNODEFICIENCIES IMMUNODYSREGULATION POLYENDOCRINOPATHY ENTEROPATHY X-LINKED SYNDROME INBORN ERRORS OF METABOLISM LEUKEMIA

Contacts and locations

Locations

Children's Hospital of Philadelphia
RECRUITING

Philadelphia, Pennsylvania, 19104, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.