DMD Trial with Exon Skipping Technique
Phase 1
- Conditions
- Health Condition 1: M638- Disorders of muscle in diseases classified elsewhere
- Registration Number
- CTRI/2022/03/041060
- Lead Sponsor
- Muscular Dystrophy Patients Welfare Society
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Yet Recruiting
- Sex
- Not specified
- Target Recruitment
- 0
Inclusion Criteria
1 Written informed to be sign
2 Should of out frame deletion
3 Not ventilator dependent
4 Life expectancy of at least 6 months
5 FVC 70
6 Within 6 months no treatment with investigational medicine
Exclusion Criteria
1 Max presence of dystrophin 5 percentage
2 If FVC less than 60 percentage
3 If Creatinine clearance less than 70 percentage
4 If EF less than 30 percentage
5 If history of respiratory insufficiency
6 Severe Cognitive dysfunction
7 If any immune deficiency
8 If any known bleeding disorder
9 Receipt of Pharmacologic treatment apart from corticosteroids
10 Surgery within 6 months
11 Acute illness within 4 weeks
12 If have psychiatric disorder
13 Experimental treatment within 4 weeks
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method