Evaluation of efficacy of LOLA (L-ornithine L-aspartate) in adult-onset citrullinemia patients

JRCT ID: jRCTs031180195

Registered date:06/03/2019

Basic Information

• Recruitment status: Complete
• Health condition(s) or Problem(s) studied: Adult-onset citrullinemia (CTLN2)
• Date of first enrollment: 06/03/2019
• Target sample size: 10
• Study type: Interventional
• Intervention(s): Patients are started to administer LOLA at 3g TID (per os) and three days later, the dose is up to 6g TID (per os) if patients have any adverse events.

Outcome(s)

• Primary Outcome: Decrease in plasma levels of ammonia and /or citrulline at 12 weeks and 3.5 years after start of LOLA
• Secondary Outcome: Improvements of severity of hepatic encephalopathy (improvement of the grade of hepatic encephalopathy and neuropsychological tests)

Key inclusion & exclusion criteria

• Age minimum: >= 20age old
• Age maximum: Not applicable
• Gender: Both

Include criteria

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

Exclude criteria

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)

Related Information

• Primary Sponsor: Yazaki Masahide
• Source(s) of Monetary Support: Citrin foundation (Singapore)