Health Condition 1: M638- Disorders of muscle in diseases classified elsewhere
Conditions
Interventions
Intervention1: 2 O Methyl Phosphorothioate Antisense Oligo Ribonucleotide: The Oligo Ribonucleotide is a Tailored made based on individual Gene Sequencing ASO administration based on individual body w
Sponsors
Muscular Dystrophy Patients Welfare Society
Eligibility
Inclusion criteria
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
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 1 To evaluate efficacy safety tolerability of the investigational product for patients suffering from DMD and Identify the increase in Dystrophin production in treated patients 2 To determine the Pharmacokinetics after subcutaneous administrationTimepoint: 18 Weeks | — |
Secondary
| Measure | Time frame |
|---|---|
| Improvement of Muscular Function by using 6 minute walk testTimepoint: 18 Weeks | — |
Countries
India
Contacts
Public ContactNirmalya Roy
Institute of Child Health
Outcome results
None listed