Phase 1/2 study of NS-065/NCNP-01 in patients with Duchenne muscular dystrophy
Phase 1
- Conditions
- Duchenne muscular dystrophy
- Registration Number
- JPRN-jRCT2080223236
- Lead Sponsor
- IPPON SHINYAKU CO., LTD.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Sex
- Male
- Target Recruitment
- 16
Inclusion Criteria
1) Ambulant or non-ambulant patient
2) Able to treat with skipping of exon 53
Exclusion Criteria
Has participated in other clinical trial that might recover dystrophin protein by the readthrough or the exon-skipping therapy, and/or upregulate the dystrophin-associated proteins such as utrophin.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method