UMIN-CTR Clinical Trial
| Unique ID issued by UMIN | UMIN000044914 |
|---|---|
| Receipt number | R000051301 |
| Scientific Title | EVRYSDI Dry Syrup 60 mg General Drug Use Surveillance (All-Patient Surveillance) -Spinal Muscular Atrophy- |
| Date of disclosure of the study information | 2021/08/31 |
| Last modified on | 2025/01/22 11:45:56 |
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Basic information
Public title
EVRYSDI Dry Syrup 60 mg General Drug Use Surveillance (All-Patient Surveillance) -Spinal Muscular Atrophy-
Acronym
EVRYSDI Dry Syrup 60 mg General Drug Use Surveillance (All-Patient Surveillance) -Spinal Muscular Atrophy-
Scientific Title
EVRYSDI Dry Syrup 60 mg General Drug Use Surveillance (All-Patient Surveillance) -Spinal Muscular Atrophy-
Scientific Title:Acronym
EVRYSDI Dry Syrup 60 mg General Drug Use Surveillance (All-Patient Surveillance) -Spinal Muscular Atrophy-
Region
| Japan |
Condition
Condition
Spinal muscular atrophy
Classification by specialty
| Medicine in general | Neurology | Pediatrics |
Classification by malignancy
Others
Genomic information
NO
Objectives
Narrative objectives1
To determine the safety profile of EVRYSDI Dry Syrup 60 mg (hereinafter, the drug) by calculating the incidence of adverse drug reactions and the distribution of days to onset in actual clinical use.
Safety specification: retinal toxicity, embryonic and fetal toxicity, effects on male reproductive potential, epithelial histological damage, safety in patients with type IV SMA and patients with SMN2 gene-copy numbers over 5, effects on QT/QTc interval, safety in premature babies
Basic objectives2
Safety,Efficacy
Basic objectives -Others
Trial characteristics_1
Trial characteristics_2
Developmental phase
Assessment
Primary outcomes
1)name of institution, name of department, date CRF completed, name of physician completing CRF
2)patient's initials, age at initiation of treatment, gestational age (type I only), identification number, sex, pregnancy, height, weight, reason for use, time of SMA onset, time of SMA diagnosis, presence of genetic testing, SMN1 mutation defect, copy-number of SMN2 gene, presence of family history, previous disease, concurrent disease, prior medications for SMA, history of surgery
3)treatment status of the drug (daily dose, dosing schedule, treatment start date, treatment stop date), status at CRF completion (reason for discontinuation in patients who discontinued treatment)
4)presence of concomitant drug, drug name, treatment start date, treatment stop date
5)presence of adverse events, name of adverse events or laboratory abnormalities, date of onset, seriousness, action taken (with the drug, etc.), outcome, date of outcome, causal relationship (with the drug, other factors)
6)global improvement (12 months after initiation of treatment, 24 months after initiation of treatment)
7)presence of motor function assessment, assessment scale, assessment results (at the start of administration, 12 months after initiation of treatment, 24 months after initiation of treatment)
8) (type I only) ability to maintain sitting position for 5 seconds (at the start of administration, 12 months after the start of administration, 24 months after the start of administration), presence of use of respiratory equipment at the start of administration, presence of long-term respiratory care (12 months after the start of administration, 24 months after the start of administration), duration of respiratory equipment use, presence of tube feeding at the start of administration, presence and duration of tube feeding (12 months after the start of administration, 24 months after the start of administration)
Key secondary outcomes
Base
Study type
Observational
Study design
Basic design
Randomization
Randomization unit
Blinding
Control
Stratification
Dynamic allocation
Institution consideration
Blocking
Concealment
Intervention
No. of arms
Purpose of intervention
Type of intervention
Interventions/Control_1
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
| Not applicable |
Gender
Male and Female
Key inclusion criteria
Patients eligible for enrollment: all patients expected to receive the drug during the enrollment period
Patients eligible for case report form (CRF) collection: all patients eligible for enrollment who receive the drug
Data will be collected retrospectively from patients who receive the drug before conclusion of the agreement for this surveillance study, and CRFs will be collected for all patients who receive the drug.
Key exclusion criteria
None
Target sample size
330
Research contact person
Name of lead principal investigator
| 1st name | Shinya |
| Middle name | |
| Last name | Takemoto |
Organization
Chugai Pharmaceutical Co. L
Division name
Safety science 2 Dept.
Zip code
1038324
Address
1-1 Nihonbashi-muromachi 2-chome, Chuo-ku Tokyo, Japan
TEL
03-3281-6611
takemotosny@chugai-pharm.co.jp
Public contact
Name of contact person
| 1st name | Ayako |
| Middle name | |
| Last name | Murayama |
Organization
Chugai Pharmaceutical Co. Ltd.
Division name
Safety Science 2 Dept.
Zip code
1038324
Address
1-1 Nihonbashi-muromachi 2-chome, Chuo-ku Tokyo, Japan
TEL
03-3281-6611
Homepage URL
murayamaayk@chugai-pharm.co.jp
Sponsor or person
Institute
Chugai Pharmaceutical Co. Ltd.
Institute
Department
Personal name
Funding Source
Organization
Chugai Pharmaceutical Co. Ltd.
Organization
Division
Category of Funding Organization
Profit organization
Nationality of Funding Organization
Other related organizations
Co-sponsor
Name of secondary funder(s)
IRB Contact (For public release)
Organization
None
Address
None
Tel
None
None
Secondary IDs
Secondary IDs
NO
Study ID_1
Org. issuing International ID_1
Study ID_2
Org. issuing International ID_2
IND to MHLW
Institutions
Institutions
Other administrative information
Date of disclosure of the study information
| 2021 | Year | 08 | Month | 31 | 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
Open public recruiting
Date of protocol fixation
| 2021 | Year | 07 | Month | 08 | Day |
Date of IRB
| 2021 | Year | 07 | Month | 08 | Day |
Anticipated trial start date
| 2021 | Year | 08 | Month | 12 | Day |
Last follow-up date
| 2030 | Year | 08 | Month | 31 | Day |
Date of closure to data entry
Date trial data considered complete
Date analysis concluded
Other
Other related information
None
Management information
Registered date
| 2021 | Year | 07 | Month | 20 | Day |
Last modified on
| 2025 | Year | 01 | Month | 22 | Day |
Link to view the page
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000051301
