Phase 3 study of K-828-SP
Phase 3
Completed
- Conditions
- spinal muscular atrophy
- Registration Number
- JPRN-jRCT2080223395
- Lead Sponsor
- Kowa Company, Ltd.
- Brief Summary
There were not significant safety issues on the long-term study of sodium valproate for childhood onset spinal muscular atrophy. Apparent improvements in motor function were not observed.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- completed
- Sex
- All
- Target Recruitment
- 14
Inclusion Criteria
(1) Patients with SMN 1 gene mutation proven by gene testing
(2) Patients of SMA types 1, 2 and 3
(3) Patients under age 15 when agreement was obtained
etc
Exclusion Criteria
1) Patients who have had an operation for spine scoliosis
2) Patient with severe liver dysfunction, renal dysfunction
3) Patients with urea cycle abnormalities
etc
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method safety<br>efficacy<br>Efficacy: HFMSE<br>Safety: AE and ADR<br>Efficacy: change from baseline<br>Safety: incidence rate
- Secondary Outcome Measures
Name Time Method safety<br>efficacy<br>Efficacy: HFME etc.<br>Safety: vital signs and laboratory tests<br>Efficacy: change from baseline<br>Safety: change from baseline