Gene therapy recipients monitored for years in MMA Follow-Up

NCT ID NCT05506254

First seen Nov 01, 2025 · Last updated May 17, 2026 · Updated 28 times

Summary

This study follows 4 people with methylmalonic acidemia (MMA) who previously received the experimental gene therapy hLB-001. Researchers are checking for any long-term side effects or health problems that may appear years after treatment. The goal is to understand the lasting safety of this therapy, not to test a new treatment.

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Contacts and locations

Locations

  • Clinical Trial Site

    Atlanta, Georgia, 30322, United States

  • Clinical Trial Site

    Pittsburgh, Pennsylvania, 15224, United States

  • Clinical Trial Site

    Nashville, Tennessee, 37232, United States

  • Clinical Trial Site

    Seattle, Washington, 98105, United States

Conditions

Explore the condition pages connected to this study.