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.
Disclaimer
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This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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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
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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