Experimental cell shot aims to boost brain repair in kids with rare metabolic diseases

NCT ID NCT02254863

Recruiting now Disease control Sponsor: Joanne Kurtzberg, MD Source: ClinicalTrials.gov ↗

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.

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Conditions

The condition(s) this trial relates to.

adrenoleukodystrophy alpha-mannosidosis Brain Diseases, Metabolic, Inborn inborn errors of metabolism Krabbe disease metachromatic leukodystrophy metachromatic leukodystrophy, juvenile form mucopolysaccharidosis type 2 mucopolysaccharidosis type 3 neuronal ceroid lipofuscinosis 3 Neuronal Ceroid-Lipofuscinoses Niemann-Pick disease Niemann-Pick disease type A Pelizaeus-Merzbacher spectrum disorder Sandhoff disease Tay-Sachs disease

As listed by the trial registrant

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

ADRENOLEUKODYSTROPHY ALPHA-MANNOSIDOSIS BATTEN DISEASE BRAIN DISEASES, METABOLIC, INBORN LEUKODYSTROPHY, GLOBOID CELL LEUKODYSTROPHY, METACHROMATIC MUCOPOLYSACCHARIDOSIS II NEIMANN PICK DISEASE PELIZAEUS-MERZBACHER DISEASE SANDHOFF DISEASE SANFILIPPO MUCOPOLYSACCHARIDOSES TAY-SACHS DISEASE

Contacts and locations

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Study contacts

Contact

Email: •••••@•••••
Contact

Email: •••••@•••••

Locations

Duke University Medical Center
RECRUITING

Durham, North Carolina, 27705, United States

Contact Email: •••••@•••••

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