New hope for kids with rare metabolic disease: 5-Year trial launches
NCT ID NCT06904027
First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study is testing a drug called glycerol phenylbutyrate in 40 Chinese children with urea cycle disorders, a rare genetic condition that causes dangerous ammonia buildup. The drug aims to help control ammonia levels over 5 years. Researchers will monitor safety and effectiveness to see if it offers a better long-term management option.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- glycerol phenylbutyrate oral liquid
- What this could lead to
- If successful, this could provide a safe and effective long-term treatment option for Chinese children with urea cycle disorders, helping to control ammonia levels and reduce hospitalizations.
- What could go wrong
- This is a single-arm study with no placebo group, so results may be less definitive. The small size (40 patients) and 5-year duration mean it may not capture rare side effects or apply to all patients.
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
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Children's Hospital of Zhejiang University School of Medicine
RECRUITINGHangzhou, Zhejiang, China
-
Guangzhou Women and Children's Medical Center
RECRUITINGGuangzhou, Guangdong, 510000, China
-
Peking University First Hospital
RECRUITINGBeijing, Beijing Municipality, 100034, China
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Tongji Hosipital
RECRUITINGWuhan, Hubei, 430030, China
-
Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
RECRUITINGShanghai, Shanghai Municipality, 200092, China
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