Experimental gene therapy targets rare muscle disease in first human test
NCT ID NCT05876780
First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This early-phase trial tests a single-dose gene therapy called SRP-9003 in 6 people with limb girdle muscular dystrophy type 2E/R4, a rare genetic muscle-weakening disease. The main goals are to check safety and see if the therapy can produce the missing beta-sarcoglycan protein in muscle. Both ambulatory and non-ambulatory participants are included.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- SRP-9003 (a gene therapy given as a single IV infusion)
- What this could lead to
- If successful, this could point toward a treatment that restores the missing protein in muscle cells, potentially slowing or stopping muscle weakness in LGMD2E/R4.
- What could go wrong
- This is a very early, small phase 1 trial with only 6 people. It primarily checks safety and protein levels, not whether it improves strength or daily life. Gene therapies can cause immune reactions or other side effects.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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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
-
Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
-
St. Jude Children's Research Hospital
Memphis, Tennessee, 38105, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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- New study maps key tests for LGMD to speed up drug development