New cell booster may help cord blood transplants reach the brain in kids with rare diseases

NCT ID NCT02254863

First seen Oct 31, 2025 · Last updated May 16, 2026 · Updated 29 times

Summary

This early-stage study tests whether adding special cells (DUOC-01) to a standard cord blood transplant is safe for children with inherited metabolic diseases that damage the brain. About 40 participants, from 1 week to 21 years old, will receive the cells via a spinal injection. The goal is to see if this approach can help protect or repair the brain without causing serious side effects.

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Contacts and locations

Study contacts

  • Contact

    Email: •••••@•••••

  • Contact

    Email: •••••@•••••

Locations

  • Duke University Medical Center

    RECRUITING

    Durham, North Carolina, 27705, United States

    Contact

    Contact

    Contact Email: •••••@•••••

Conditions

Explore the condition pages connected to this study.