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A Clinical Study of Glycerol Phenylbutyrate in Chinese Patients With Urea Cycle Disorders

NCT06904027 · Status: RECRUITING · Type: OBSERVATIONAL · Enrollment: 40

Last updated 2026-04-27

No results posted yet for this study

Summary

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

  • Urea Cycle Disorders

Interventions

DRUG

Glycerol phenylbutyrate Oral Liquid

Administration and dosage: The recommended total daily dose of glycerol phenylbutyrate is calculated based on the patient's body surface area, ranging from 4.5 mL/m2/d to 11.2 mL/m2/d: 1. Recommended initial dose in phenylbutyrate-naïve patients * Patients with body surface area (BSA) \< 1.3 m2: 8.5 mL/m2/day * Patients with body surface area (BSA) ≥ 1.3 m2: 7 mL/m2/day 2. Initial dose for patients switching from sodium phenylbutyrate to glycerol phenylbutyrate: Total daily dose of glycerol phenylbutyrate (ml) = total daily dose of sodium phenylbutyrate granules (g) ×0.81 Medication Schedule: The daily total dose should be divided into equal amounts and administered with each meal or feeding (such as three to six times daily).

Sponsors & Collaborators

  • Tongji Hospital

    lead OTHER

Principal Investigators

  • Xiaoping Luo, M.D. · Tongji Hospital

Eligibility

Min Age
0 Years
Max Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2025-12-09
Primary Completion
2026-09-30
Completion
2031-07-31

Countries

  • China

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06904027 on ClinicalTrials.gov