UMIN-CTR Clinical Trial
| Unique ID issued by UMIN | UMIN000041118 |
|---|---|
| Receipt number | R000046953 |
| Scientific Title | Phase III study combined with post-marketing clinical study of RM1319 in patients with inborn errors of bile acid metabolism, including children |
| Date of disclosure of the study information | 2020/07/15 |
| Last modified on | 2023/09/11 11:31:24 |
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Basic information
Public title
Phase III study combined with post-marketing clinical study of RM1319 in patients with inborn error of bile acid metabolism, including children
Acronym
Clinical study for patients with inborn errors of bile acid metabolism
Scientific Title
Phase III study combined with post-marketing clinical study of RM1319 in patients with inborn errors of bile acid metabolism, including children
Scientific Title:Acronym
Phase III study combined with post-marketing clinical study of RM1319 in patients with inborn errors of bile acid metabolism, including children
Region
| Japan |
Condition
Condition
Inborn errors of bile acid metabolism
Classification by specialty
| Hepato-biliary-pancreatic medicine | Endocrinology and Metabolism | Pediatrics |
Classification by malignancy
Others
Genomic information
NO
Objectives
Narrative objectives1
To confirm the efficacy and safety of RM1319 for Japanese patients with inborn errors of bile acid metabolism. And also, to investigate the concentration of cholic acid in urine and serum during investigational drug administration
Basic objectives2
Efficacy
Basic objectives -Others
Trial characteristics_1
Confirmatory
Trial characteristics_2
Pragmatic
Developmental phase
Phase III
Assessment
Primary outcomes
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)
Key secondary outcomes
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).
Base
Study type
Interventional
Study design
Basic design
Single arm
Randomization
Non-randomized
Randomization unit
Blinding
Open -no one is blinded
Control
Uncontrolled
Stratification
NO
Dynamic allocation
NO
Institution consideration
Institution is not considered as adjustment factor.
Blocking
NO
Concealment
No need to know
Intervention
No. of arms
1
Purpose of intervention
Treatment
Type of intervention
| Medicine |
Interventions/Control_1
Continuous administration of RM1319
Interventions/Control_2
Interventions/Control_3
Interventions/Control_4
Interventions/Control_5
Interventions/Control_6
Interventions/Control_7
Interventions/Control_8
Interventions/Control_9
Interventions/Control_10
Eligibility
Age-lower limit
| Not applicable |
Age-upper limit
| 75 | years-old | > |
Gender
Male and Female
Key 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
Key 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
Target sample size
5
Research contact person
Name of lead principal investigator
| 1st name | Tadashi |
| Middle name | |
| Last name | Matsumoto |
Organization
ReqMed Company, Ltd.
Division name
CEO
Zip code
194-0022
Address
1-7-23 Morino, Machida, Tokyo
TEL
042-732-2207
matsumoto@reqmed.co.jp
Public contact
Name of contact person
| 1st name | Maki |
| Middle name | |
| Last name | Urata |
Organization
ReqMed Company, Ltd.
Division name
Pharmaceutical Division
Zip code
194-0022
Address
1-7-23 Morino, Machida, Tokyo
TEL
042-732-2207
Homepage URL
urata@reqmed.co.jp
Sponsor or person
Institute
ReqMed Company, Ltd.
Institute
Department
Personal name
Funding Source
Organization
none
Organization
Division
Category of Funding Organization
Self funding
Nationality of Funding Organization
Other related organizations
Co-sponsor
THERAVIA (formarly Laboratoires CTRS)
Name of secondary funder(s)
IRB Contact (For public release)
Organization
Kurume University Hospital IRB
Address
67 Asahi-cho, Kurume-City, Fukuoka
Tel
0942-31-7200
kcrc_jimu@kurume-u.ac.jp
Secondary IDs
Secondary IDs
NO
Study ID_1
Org. issuing International ID_1
Study ID_2
Org. issuing International ID_2
IND to MHLW
2020-0468
Institutions
Institutions
久留米大学病院（福岡県）
順天堂大学医学部附属順天堂医院（東京都）
埼玉医科大学付属病院（埼玉県）
京都府立医科大学病院（京都府）
岐阜市民病院(岐阜県）
Other administrative information
Date of disclosure of the study information
| 2020 | Year | 07 | Month | 15 | Day |
Related information
URL releasing protocol
Publication of results
Unpublished
Result
URL related to results and publications
Number of participants that the trial has enrolled
Results
Results date posted
Results Delayed
Results Delay Reason
Date of the first journal publication of results
Baseline Characteristics
Participant flow
Adverse events
Outcome measures
Plan to share IPD
IPD sharing Plan description
Progress
Recruitment status
Completed
Date of protocol fixation
| 2020 | Year | 04 | Month | 20 | Day |
Date of IRB
| 2020 | Year | 06 | Month | 15 | Day |
Anticipated trial start date
| 2020 | Year | 07 | Month | 01 | Day |
Last follow-up date
| 2023 | Year | 06 | Month | 30 | Day |
Date of closure to data entry
| 2023 | Year | 09 | Month | 15 | Day |
Date trial data considered complete
| 2023 | Year | 09 | Month | 30 | Day |
Date analysis concluded
| 2023 | Year | 12 | Month | 31 | Day |
Other
Other related information
Management information
Registered date
| 2020 | Year | 07 | Month | 15 | Day |
Last modified on
| 2023 | Year | 09 | Month | 11 | Day |
Link to view the page
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000046953
