Half-Matched stem cell transplant offers hope for children with rare immune and metabolic diseases
NCT ID NCT04414046
First seen Jul 01, 2026 · Last updated Jul 01, 2026
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.
Disclaimer
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the original study
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Johns Hopkins All Children's Hospital
RECRUITINGSt. Petersburg, Florida, 33701, United States
Contact
Contact Email: •••••@•••••