Gene therapy for rare metabolic disease passes early safety check
NCT ID NCT05506254
First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study follows 4 people with methylmalonic acidemia who previously received hLB-001 gene therapy. Researchers are checking for long-term side effects. The goal is to see if the treatment remains safe over time.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- hLB-001 gene therapy
- What this could lead to
- If successful, this follow-up could show that hLB-001 is safe over the long term, supporting its development as a treatment for methylmalonic acidemia.
- What could go wrong
- This is a very small, non-interventional study with only 4 participants, so results may not apply to others. It only monitors safety, not effectiveness.
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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
Locations
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Clinical Trial Site
Atlanta, Georgia, 30322, United States
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Clinical Trial Site
Pittsburgh, Pennsylvania, 15224, United States
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Clinical Trial Site
Nashville, Tennessee, 37232, United States
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Clinical Trial Site
Seattle, Washington, 98105, United States
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