Confirmation of effects of medium chain triglyceride therapy for citrin deficiency

Phase 2

Recruiting

• Conditions: citrin deficiency; Endocrinology and Metabolism, Pediatrics; D006111

• Registration Number: JPRN-jRCTs041190017
• Lead Sponsor: Mitsui Tetsuo

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2019-04-10
• Last Update Posted: 17 October 2023

Recruitment & Eligibility

• Status: Recruiting
• Sex: All
• Target Recruitment: 50

Inclusion Criteria

1) Patients with symptomatic post NICCD
2) Patients with adult-onset type II citrullinemia
3) Patients with biallelic mutation of SLC25A13 gene

Exclusion Criteria

1) Patients with sodium pyruvate therapy
2) liver transplanted patients
3) Judged inappropriate for this study by the physicians.

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Improvemet of clinical symptoms and QO

Secondary Outcome Measures

Name	Time	Method
1) plasma ammonia concentration<br>2) plasma citrulline concentration<br>3) plasma Fisher ratio<br>4) auxological data