Glycerol phenylbutyrate for Chinese children and teens with urea cycle disorders

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is a multicenter, prospective, single-arm observational study enrolling up to 40 Chinese participants aged 0–18 with diagnosed urea cycle disorder subtypes who plan to start glycerol phenylbutyrate and have not used it in the prior three months. Patients are followed for a total of five years with visits at 1 and 3 months, then every six months, collecting ammonia levels, clinical labs, adverse events, hyperammonemic crises, medication dosing, growth measures, and neurocognitive outcomes. The study documents real-world safety, dosing, biochemical control, and clinical events while participants receive glycerol phenylbutyrate during routine care. Data will provide longitudinal descriptive outcomes to inform clinical management in Chinese pediatric UCD patients.

Why it matters

Who can join: the full rules

Inclusion Criteria:

1. Male or female aged 0-18 years;
2. Subject and/or subject's legally authorized representative willing to follow the therapeutic regimen, dietary management and visit plan of the study, and voluntarily signing informed consent form;
3. Patients with the following subtypes of UCD: Carbamoyl phosphate synthetase I deficiency, Ornithine translocase deficiency, citrullinemia type I, argininosuccinic aciduria, argininemia, and hyperornithinemia-hyperammonemia-homocitrullinuria (HHH) syndrome;
4. Patients planned to use glycerol phenylbutyrate who have not used it in past 3 months (including at the time of 3 months);
5. Men with fertility and women of childbearing potential (with menstruation) who are willing to take effective contraceptive measures during the period from the date of signing the informed consent to 1 months after the last dose of the study drug, such as abstinence, condoms, intra-uterine contraceptive devices, and double barrier methods (such as condoms + contraceptive diaphragms). Pregnancy test results must be negative for women of childbearing age within ≤ 7 days before the initial administration of study drug.

Exclusion Criteria:

1. Hypersensitivity to any of the active ingredient, including phenylbutyrate (PBA), phenylacetate acid (PAA) and phenylacetyl glutamine (PAGN), or excipients;
2. Use of any drug known to significantly affect renal clearance (such as probenecid) or increase protein catabolism (such as corticosteroids) or other drugs known to increase blood ammonia levels (such as valproate) within 24 h before the first administration;
3. Use of other nitrogen-scavenging agent at the same time after enrollment, such as sodium phenylbutyrate and sodium benzoate;
4. Pregnant or breastfeeding females.
5. Other reasons, in the opinion of the investigator, that may affect the patient's compliance and safety in participating in the study.

Where it's running

• 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

Study contacts

• Principal investigator: Xiaoping Luo, M.D. — Tongji Hospital
• Study coordinator: Xiaoping Luo, M.D.
• Email: xpluo@tjh.tjmu.edu.cn
• Phone: 027-83662640

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Other studies for the same condition or treatment. Recruiting studies are listed first.

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.