A safety and efficacy study of ATL1102 in patients with Duchenne muscular dystrophy | DuchenneXchange

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A safety and efficacy study of ATL1102 in patients with Duchenne muscular dystrophy

key information

study id #: ACTRN12618000970246

condition: Duchenne Muscular Dystrophy

status: recruiting

purpose:

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.

Read more: ACTRN12618000970246

 

intervention: ATL1102 (25mg/week)

study details

start date: August 28, 2018

estimated completion: June 17, 2019

phase of development: Phase 2

size / enrollment: 09

study description:
One cohort will be investigated. ATL1102 25mg/week, administered SC once weekly for 24 weeks in 25kg-60kg patients, (0.42-1mg/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.

primary outcomes:

  • Safety and tolerability as assessed by safety data on adverse events, injection site reactions and laboratory assessments.
    Mild injection site erythema, increased liver enzymes and decreased platelet count.
    These will be assessed visually and by biochemistry (bilirubin & gamma glutamyl transferase) and haematology respectively.
    [Assessed at screening, baseline (week 1, day 1) and during treatment weeks 1, 3, 5, 7, 8, 10, 12, 14, 16, 18, 20, 22, 24, and post treatment weeks 28 and 32]
  • Lymphocyte modulation assessed by haematological parameters lymphocytes, CD4 and CD8 T cells.
    [Assessed at baseline, (week 1, day 1), and during treatment weeks 5, 8, 12, 24 and post treatment at week 28]

secondary outcomes:

  • Performance of upper limb assessment will be assessed via the Myo-Pinch device (pinch strength)
    [Assessed at baseline (week 1, day 1) and during treatment weeks 5, 8, 12, and 24]
  • Performance of upper limb assessment will be assessed via the Myo-Grip device (grip strength)
    [Assessed at baseline (week 1, day 1) and during treatment weeks 5, 8, 12, and 24]
  • Performance of upper limb assessment will be assessed via the score as determined by the MoviPlate device (hand function) scores
    [Assessed at baseline (week 1, day 1) and during treatment weeks 5, 8, 12, and 24]

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.

study contacts

sponsor: Antisense Therapeutics Limited

contacts: Prof Monique Ryan, The Royal Children's Hospital, +61 3 9345 5661, monique.ryan@rch.org.au

investigators: Dr Ian Woodcock, The Royal Children's Hospital, +61 3 9345 5661, Ian.Woodcock@rch.org.au

locations: Australia