None listed
Conditions
Brief summary
Current DMD therapies are aimed at increasing dystrophin levels and reducing inflammation. Improved anti-inflammatory therapies are needed to safely treat this pathology and delay disease progression. This study will be conducted in a single-centre and assess the safety, efficacy and PK of ATL1102 in non-ambulatory patients with DMD.
Interventions
One cohort will be investigated. ATL1102 25mg/week, administered SC once weekly for 24 weeks in 25kg-65kg patients, (0.4-1mg/kg/w dose).
All doses of ATL1102 will be administered subcutaneously by a nurse in the hospital and by a nurse at home.
Monitoring of adherence to the intervention is by diary card and by return of the used and unused vials of investigational product and reconciliation at the hospital.
Sponsors
Antisense Therapeutics Limited
Study design
Allocation
Non-randomised trial
Intervention model
Single group
Primary purpose
Treatment
Masking
Open (masking not used)
Eligibility
Sex/Gender
Male
Age
10 Years to 18 Years
Healthy volunteers
No
Inclusion criteria
Adolescent males aged 10 to 18 years with a diagnosis of non-ambulatory Duchenne Muscular Dystrophy for at least 3 months.
Exclusion criteria
Ambulatory patients with DMD who are able to complete at least 75 meters during a 6-minute walk test, or have abnormal haematology values or a history of bleeding or coagulation abnormalities.
Outcome results
None listed