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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.
Register: JPRN
Last refreshed on: 25 February 2020
Main ID:  JPRN-jRCTs041190055
Date of registration: 24/07/2019
Prospective Registration: Yes
Primary sponsor: Ito Tetsuya
Public title: Carglumic Acid Administration for OTC Deficienscy
Scientific title: A Clinical Study of Carglumic Acid in Patients with Late-Onset Ornithine Transcarbamylase Deficiency
Date of first enrolment: 24/07/2019
Target sample size: 2
Recruitment status: Recruiting
URL:  https://jrct.niph.go.jp/latest-detail/jRCTs041190055
Study type:  Interventional
Study design:  non-randomized controlled trial, open(masking not used), active control, single assignment, treatment purpose  
Phase:  4
Countries of recruitment
Japan
Contacts
Name: Tetsuya    Ito
Address:  1-98, Dengakugakubo, Kutsukake-cho, toyoake, Aichi 470-1192 Aichi Japan
Telephone: +81-562-93-2111
Email: itotetsu@fujita-hu.ac.jp
Affiliation:  Fujita Health University Hospital
Name: Tetsuya    Ito
Address:  1-98, Dengakugakubo, Kutsukake-cho, toyoake, Aichi 470-1192 Aichi Japan
Telephone: +81-562-93-9251
Email: itotetsu@fujita-hu.ac.jp
Affiliation:  Fujita Health University Hospital
Key inclusion & exclusion criteria
Inclusion criteria: 1. Chronic phase patients with genetically diagnosed ornitine transcarbamylase (OTC) deficiency.
2. Patients with the blood ammonia levels>60ug/dL(neonate:>200ug/dL, infant:>100ug/dL) under the treatment with Low-protain daiet, phenylbutyrate and citrurine administration. Or patients whoes blood ammonia level was two times higher than the average of before 6 months at outpatients clinic.
3. Male and female, below 75 years old at informed concent is obtained.
4. Patients who obtained written informed consent form the patient or legal representative.

Exclusion criteria: 1. Other types of urea cycle disorders or patients with organic acidemias.
2. Hyperammonemia due to acquired amino acid metabolism abnormality such as vilal hepatites, port-systemic shunt, biliary atresia or hepatic failure.
3. Completely defected patients of OTC activity.
4. Liver transplanted patients.
5. Patients who reseved dialysis within two days before the treatment.
6. Past histry of symptomatic worsening after drug administration, occurrence of adverse event or hypersensitivity.
7. Patients with severe hepatic, renal or cardiac disease
8. Patients who is pregnant or may be pregnant, or lactating.
9. Patients with drug hypersensitivity.
10. Patients who are involved in other clinical trials or had been involved within the last 3 months.
11. Patients who are determined as disqualified by attending physician.


Age minimum: Not applicable
Age maximum: < 75age old
Gender: Both
Health Condition(s) or Problem(s) studied
ornitine transcarbamylase deficiency patients
Intervention(s)
administraton of carglumic acid
Primary Outcome(s)
change of blood glutamine levels
Secondary Outcome(s)
change of daily protein intake
Secondary ID(s)
Source(s) of Monetary Support
Recordati Rare Diseases Japan
Secondary Sponsor(s)
Ethics review
Status: Approval
Approval date: 19/07/2019
Contact:
f-irb@fujita-hu.ac.jp
Fujita Health University Certified Clinical Research Review Board
+81-562-93-2865
f-irb@fujita-hu.ac.jp
Results
Results available:
Date Posted:
Date Completed:
URL:
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