New hope for kids with devastating brain diseases: experimental cell therapy aims to halt progression

NCT ID NCT02254863

Summary

This early-stage study is testing the safety of adding a special type of cell, derived from umbilical cord blood, to a standard cord blood transplant for children with rare inherited metabolic diseases that damage the brain. The goal is to see if delivering these cells directly into the spinal fluid can help protect the nervous system faster and slow disease progression. The study will enroll up to 40 children and young adults up to age 21 who have one of several specific disorders, like Krabbe disease or Tay-Sachs.

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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.