Actively Recruiting
A Clinical Study of Glycerol Phenylbutyrate in Chinese Patients With Urea Cycle Disorders
Led by Tongji Hospital · Updated on 2026-04-27
40
Participants Needed
5
Research Sites
290 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
A Clinical Study of Glycerol Phenylbutyrate in Chinese Patients With Urea Cycle Disorders
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male or female aged 0-18 years
- Willingness to follow the treatment, dietary management, and visit schedule, and signing informed consent
- Diagnosis with one of the following UCD subtypes: carbamoyl phosphate synthetase I deficiency, ornithine translocase deficiency, citrullinemia type I, argininosuccinic aciduria, argininemia, or HHH syndrome
- Planned use of glycerol phenylbutyrate with no use in the past 3 months (including the past 3 months)
- For men with fertility and women of childbearing potential: willingness to use effective contraception from consent signing to 1 month after last dose, and negative pregnancy test within 7 days before starting study drug
You will not qualify if you...
- Allergy to phenylbutyrate, phenylacetate acid, phenylacetyl glutamine, or excipients
- Use of drugs affecting renal clearance, protein catabolism, or increasing blood ammonia within 24 hours before first dose
- Use of other nitrogen-scavenging agents after enrollment
- Pregnant or breastfeeding females
- Any other condition judged by the investigator to affect compliance or safety
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 5 locations
1
Peking University First Hospital
Beijing, Beijing Municipality, China, 100034
Actively Recruiting
2
Guangzhou Women and Children's Medical Center
Guangzhou, Guangdong, China, 510000
Actively Recruiting
3
Tongji Hosipital
Wuhan, Hubei, China, 430030
Actively Recruiting
4
Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
Shanghai, Shanghai Municipality, China, 200092
Actively Recruiting
5
Children's Hospital of Zhejiang University School of Medicine
Hangzhou, Zhejiang, China
Actively Recruiting
Research Team
X
Xiaoping Luo, M.D.
CONTACT
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
1
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