Half-Matched stem cell transplant offers hope for children with rare immune and metabolic diseases

NCT ID NCT04414046

Recruiting now Disease control Sponsor: Johns Hopkins All Children's Hospital Source: ClinicalTrials.gov ↗

First seen Jul 01, 2026 · Last updated Jul 02, 2026 · Updated 1 time

Summary

This study tests a new type of stem cell transplant for children with primary immune deficiencies or inherited metabolic disorders. The transplant uses stem cells from a half-matched family donor, which are specially processed to remove certain immune cells. The goal is to see if this method is safe and effective at restoring a healthy immune system and improving the child's condition.

What this could mean

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

Active substance: TCR alpha beta T-cell and CD19 B-cell depleted haploidentical stem cell transplant
What this could lead to: If successful, this approach could offer a safer, more effective transplant option for children with severe immune or metabolic diseases who lack a fully matched donor.
What could go wrong: This is a small, early-phase study, so results may not apply broadly. Risks include graft failure, infection, or graft-versus-host disease, and long-term outcomes are unknown.

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Conditions

The condition(s) this trial relates to.

inborn error of immunity inborn errors of metabolism metabolic disease Primary Immunodeficiency Diseases

As listed by the trial registrant

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

METABOLIC DISEASE PRIMARY IMMUNE DEFICIENCY DISORDERS

Contacts and locations

Locations

Johns Hopkins All Children's Hospital
RECRUITING

St. Petersburg, Florida, 33701, United States

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