First gene therapy trial launches for rare childhood disease MLIV
NCT ID NCT07398872
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This early-stage trial tests a gene therapy called AAV9.hMCOLN1co in one child with Mucolipidosis Type IV (MLIV), a rare genetic disorder. The therapy is given as a single injection into the spinal fluid. The main goal is to check safety, but researchers will also look for any signs of benefit. The child must be between 1.5 and 8 years old and have a confirmed MCOLN1 gene mutation.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
AAV9.hMCOLN1co gene therapy
What this could lead to
If it works, this could point toward a treatment that slows or stops the progression of MLIV, a severe genetic disorder.
What could go wrong
This is a very early Phase 1 trial with only 1 participant, so it is too soon to know if it is safe or effective. Gene therapies can have unexpected side effects.
Disclaimer
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the original study
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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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Children's Hospital Zhejiang University School of Medicine
Hangzhou, Zhejiang, China