Experimental cell shot aims to boost brain repair in kids with rare metabolic diseases
NCT ID NCT02254863
First seen Jun 24, 2026 · Last updated Jun 26, 2026 · Updated 1 time
Summary
This early-stage trial tests whether adding special cells (DUOC-01) into the spinal fluid is safe for children with inherited metabolic diseases that damage the brain. Participants are ages 1 week to 21 years and are already receiving a standard umbilical cord blood transplant. The study will monitor for side effects and begin to see if the added cells help protect or repair the brain.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
DUOC-01 (special cells made from umbilical cord blood, given into the spinal fluid)
What this could lead to
If it works, this could improve brain repair in children with inherited metabolic diseases who already receive a cord blood transplant.
What could go wrong
This is a very early Phase 1 trial with only 40 participants, so it is primarily checking safety, not yet proving benefit. There may be risks like infusion reactions or nerve damage.
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
Email: •••••@•••••
-
Contact
Email: •••••@•••••
Locations
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Duke University Medical Center
RECRUITINGDurham, North Carolina, 27705, United States
Contact
Contact
Contact Email: •••••@•••••