Inborn errors of bile acid metabolism
Conditions
Interventions
Continuous administration of RM1319
Sponsors
ReqMed Company, Ltd.
THERAVIA (formarly Laboratoires CTRS)
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Patients who meet all of the following criteria 1. Patients who meet 1) or 2) 1) Inborn errors of bile acid metabolism 2) Patients with acute symptoms of inborn errors of bile acid metabolism and strongly suspected inborn errors of bile acid metabolism by special biochemical tests such as bile acid analysis 2. For patients with above 1), if the patients are currently undergoing treatment with primary bile acid (chenodeoxycholic acid), the dosage and administration should be generally constant for at least 12 weeks until the registration of this study 3. Patients not older than 75 years old at the time of informed consent (male and female) 4. Patients who have consented to carry out all the observation items planned in the clinical trial 5. Patients who have consented to follow the instructions of the investigator or sub-investigator 6. For patients aged 20 years or older at the time of informed consent, patients who understand the purpose, content of this study and side effects, and agree to participate in this study and have signed the consent form by themselves or by a legal representative, if necessary, with a fair attendance. 7. Japanese patients living in Japan
Exclusion criteria
Exclusion criteria: Patients with any of the following criteria will be excluded from this study. 1. Among patients who have experienced hypersensitivity or serious side effects after administration of cholic acid or components contained in this investigational drug, patients who are determined to be unsuitable by the investigator or sub-investigator 2. Patients with galactose intolerance, Lapp lactase deficiency, or glucose/galactose malabsorption 3. Among patients with hypertriglyceridemia or family history, patients who are unable to confirm the effective dose even though they are administering a high dose of chenodeoxycholic acid 4. Patients who have participated in other clinical studies or clinical trials within the past 12 weeks 5. Patients with alcohol dependence or drug dependence 6. Pregnant or breast-feeding women. Among women of childbearing potential, who wish to become pregnant during this study or who cannot consent appropriate contraception (intrauterine contraceptive device/spermicide, wearing a condom by a partner, etc.) Check by urinary HCG test is mandatory at registration and specified timing during this study) 7. Other patients who are determined to be unsuitable by the investigator or sub-investigator
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Amount (concentration) of abnormal metabolites in urine (spot urine) which are characteristic of each disease type at 26 weeks after the first administration of RM1319 (interim evaluation) | — |
Secondary
| Measure | Time frame |
|---|---|
| 1. Amount (concentration) of abnormal metabolites in urine and serum that are characteristic of each disease type at each visit during the investigational drug evaluation period and continuous administration period 2. Clinical test values (serum) at each visit during the investigational drug evaluation period and continuous administration period: AST, ALT, D-Bil, PT, Vitamin D 3. Ultrasonic image findings and liver stiffness (Elastrography examination such as FibroScan) at base line of administration (Visit1), 26 weeks after the first administration, every 6 months during the continuous administration period at the time of completion (including withdrawal or interruption). | — |
Countries
Japan
Contacts
Public ContactMaki Urata
ReqMed Company, Ltd. Pharmaceutical Division
Outcome results
None listed