Evaluation of efficacy of LOLA (L-ornithine L-aspartate) in adult-onset citrullinemia patients
- Conditions
- Adult-onset citrullinemia (CTLN2)
- Registration Number
- JPRN-jRCTs031180195
- Lead Sponsor
- Yazaki Masahide
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 10
1) Adult patients(more than 20 yo) with adult-onset citrullinemia patients at obtaining informed consent
2) Patients who were confirmed citrin-deficiency in the liver by western blot analysis or SLC25A13 gene analysis.
3) Patients who have raised plasma levels of ammonia (more than 70 micro g/dL) and/or citrulline (more than 40 nmol/mL).
4) Patients obtained written informed consent
1) Patients who previously underwent liver-transplant
2) Expectant and lactating patients
3) Patients with renal dysfunction (serum Cr more than 2.0 mg/dL or dialysis patients)
4) Patients who are receiving therapies for hepatic cancer (Patients who had made a completely recovery from hepatic cancer are not excluded)
5) Others (patients who are judged to be unqualified for this clinical study)
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Decrease in plasma levels of ammonia and /or citrulline at 12 weeks and 3.5 years after start of LOLA
- Secondary Outcome Measures
Name Time Method Improvements of severity of hepatic encephalopathy (improvement of the grade of hepatic encephalopathy and neuropsychological tests)