EVRYSDI Dry Syrup 60 mg/5 mg Tablet General Drug Use Surveillance (All-Patient Surveillance) -Spinal Muscular Atrophy-

EVRYSDI Dry Syrup 60 mg/5 mg Tablet General Drug Use Surveillance (All-Patient Surveillance) -Spinal Muscular Atrophy- - EVRYSDI Dry Syrup 60 mg/5 mg Tablet General Drug Use Surveillance (All-Patient Surveillance) -Spinal Muscular Atrophy-

Status

Recruiting

Phases

Unknown

Study type

Observational

Source

JPRN

Registry ID

JPRN-UMIN000044914

Enrollment

330

Registered

2021-08-31

Start date

2021-08-12

Completion date

Unknown

Last updated

2026-06-29

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Spinal muscular atrophy

Interventions

None listed

Eligibility

Sex/Gender

All

Inclusion criteria

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.

Exclusion criteria

Exclusion criteria: None

Design outcomes

Primary

Measure	Time frame
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)	—

Measure

Time frame

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)

—

Countries

Japan

Contacts

Public ContactAyako Murayama

Chugai Pharmaceutical Co. Ltd. Safety Science 2 Dept.

murayamaayk@chugai-pharm.co.jp03-3281-6611

Outcome results