Condition: Urea Cycle Disorders · Sponsor: Tongji Hospital
Urea cycle disorders (UCD) are rare diseases in China, would lead to high mortality and disability, which require long-term management due to the recurrent symptoms. This multi-center, prospective, single-arm study was designed to assess the efficacy and safety of Glycerol Phenylbutyrate for Chinese pediatric patients with UCD, to provide the additional references and treatment options for Chinese UCD patients, and enhance the clinical management of UCD in China. This study primarily observes patients with UCD who are on long-term treatment with glyceryl phenylbutyrate, the total planned observation period is 5 years.
This description comes directly from the study's public registry record.
Xiaoping Luo, M.D. · 027-83662640 · xpluo@tjh.tjmu.edu.cn
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| Peking University First Hospital | Beijing, Beijing Municipality, China | Recruiting |
| Guangzhou Women and Children's Medical Center | Guangzhou, Guangdong, China | Recruiting |
| Tongji Hosipital | Wuhan, Hubei, China | Recruiting |
| Xinhua Hospital, Shanghai Jiao Tong University School of Medicine | Shanghai, Shanghai Municipality, China | Recruiting |
| Children's Hospital of Zhejiang University School of Medicine | Hangzhou, Zhejiang, China | Recruiting |
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Source record: clinicaltrials.gov/study/NCT06904027