Brain gene therapy trial offers hope for kids with rare movement disorder
NCT ID NCT02852213
First seen Nov 01, 2025
Summary
This early-stage trial tests a gene therapy called AAV2-hAADC in 42 children with AADC deficiency, a rare genetic disorder that causes severe movement problems and developmental delays. The therapy delivers a working gene directly into brain areas that control movement, aiming to restore missing enzyme activity. Researchers will check for side effects and measure improvements in motor function and symptom severity.
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This is a summary of
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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Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Nationwide Children's Hospital
RECRUITINGColumbus, Ohio, 43205, United States
Contact Email: •••••@•••••
Contact
Contact Email: •••••@•••••
Contact
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The Ohio State University Medical Center
RECRUITINGColumbus, Ohio, 43221, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
Contact
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University of California San Francisco, Benioff Children's Hospital
RECRUITINGSan Francisco, California, 94143, United States
Contact Email: •••••@•••••
Contact
Contact
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
AAV2-hAADC (a gene therapy that delivers a working copy of the AADC gene into brain cells)
What this could lead to
If this works, it could point toward a treatment that improves motor function and reduces severe symptoms in children with AADC deficiency, potentially offering long-term disease control.
What could go wrong
This is an early Phase 1 trial with only 42 participants, so results may not apply to all patients. Risks include side effects from brain surgery and gene transfer, such as bleeding, infection, or involuntary movements.
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.