Confirmation of effects of medium chain triglyceride therapy for citrin deficiency

Confirmation of effects of medium chain triglyceride therapy for citrin deficiency, especially in patients with symptomatic post neonatal intrahepatic cholestasis and that with adult-onset type II citrullinemia

Status

Recruiting

Phases

Phase 2

Study type

Interventional

Source

JPRN

Registry ID

JPRN-jRCTs041190017

Enrollment

Registered

2019-04-10

Start date

2012-07-31

Completion date

Unknown

Last updated

2026-06-29

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

citrin deficiency Endocrinology and Metabolism, Pediatrics

Interventions

Medium chain triglyceride 5-15ml 3 times a day

Eligibility

Sex/Gender

All

Inclusion criteria

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

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

Design outcomes

Primary

Measure	Time frame
Improvemet of clinical symptoms and QOL	—

Secondary

Measure	Time frame
1) plasma ammonia concentration 2) plasma citrulline concentration 3) plasma Fisher ratio 4) auxological data	—

Contacts

Public ContactChikahiko Numakura

Yamagata University Hospital

cnumakur@med.id.yamagata-u.ac.jp+81-23-628-5329

Outcome results

None listed

Source: JPRN (via WHO ICTRP) · Data processed: Jul 3, 2026