CompletedPhase 2ACTRN12618000970246

A safety and efficacy study of ATL1102 in patients with Duchenne Muscular Dystrophy

A Phase 2 open label, study to determine the safety, efficacy and pharmacokinetic profile of weekly dosing of ATL1102 in patients with non-ambulatory Duchenne Muscular Dystrophy.

Sponsor

Antisense Therapeutics Limited

Enrollment

9 participants

Start Date

Aug 28, 2018

Study Type

Interventional

Conditions

Duchenne Muscular Dystrophy

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.

Eligibility

Sex: MalesMin Age: 10 YearssMax Age: 18 Yearss

Inclusion Criteria1

Adolescent males aged 10 to 18 years with a diagnosis of non-ambulatory Duchenne Muscular Dystrophy for at least 3 months.

Exclusion Criteria1

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.

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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

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.