Experimental drug targets root cause of rare muscle-wasting disease
NCT ID NCT04800874
First seen Jul 21, 2026 · Last updated Jul 22, 2026 · Updated 1 time
Summary
This study tests an experimental drug, BBP-418, in people with limb girdle muscular dystrophy type 2I (LGMD2I), a rare genetic condition that causes progressive muscle weakness. The drug aims to fix a molecular defect by helping a faulty enzyme work better. The trial includes both people who can walk and those who cannot, and focuses on safety and how the body processes the drug.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- BBP-418, a drug that supplies extra substrate to help the mutant enzyme work better and improve muscle function
- What this could lead to
- If successful, BBP-418 could become the first approved treatment for LGMD2I, potentially slowing or improving muscle weakness.
- What could go wrong
- This is a small, early-phase trial with only 14 participants, so results may not apply broadly. Safety and effectiveness are still unproven.
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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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Virginia Commonwealth University
Richmond, Virginia, 23219, United States
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