New hope for kids with rare metabolic disease: 5-Year study launches
NCT ID NCT06904027
First seen Nov 01, 2025 · Last updated May 16, 2026 · Updated 28 times
Summary
This study looks at how well a medicine called glycerol phenylbutyrate works for children with urea cycle disorders, a rare condition that causes dangerous ammonia buildup in the blood. About 40 children aged 0-18 in China will take the medicine and be followed for up to 5 years. The goal is to see if it safely controls ammonia levels and helps manage the disease long-term.
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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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