New hope for kids with rare metabolic disorder: 5-Year study launches
NCT ID NCT06904027
First seen Nov 01, 2025 · Last updated May 21, 2026 · Updated 29 times
Summary
This study tests a medicine called glycerol phenylbutyrate in 40 Chinese children with urea cycle disorders, rare genetic conditions that cause dangerous ammonia buildup. The goal is to see if the drug safely controls ammonia levels over 5 years. Participants must follow a strict diet and visit the doctor regularly.
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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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Children's Hospital of Zhejiang University School of Medicine
RECRUITINGHangzhou, Zhejiang, China
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Guangzhou Women and Children's Medical Center
RECRUITINGGuangzhou, Guangdong, 510000, China
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Peking University First Hospital
RECRUITINGBeijing, Beijing Municipality, 100034, China
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Tongji Hosipital
RECRUITINGWuhan, Hubei, 430030, China
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
RECRUITINGShanghai, Shanghai Municipality, 200092, China
Contact Phone: •••-•••-•••• Email: •••••@•••••
Conditions
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