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Trial registered on ANZCTR

Registration number
Ethics application status
Date submitted
Date registered
Date last updated
Date data sharing statement initially provided
Date results information initially provided
Type of registration
Prospectively registered

Titles & IDs
Public title
A safety and efficacy study of ATL1102 in patients with Duchenne Muscular Dystrophy
Scientific title
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.
Secondary ID [1] 294185 0
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Duchenne Muscular Dystrophy 306822 0
Condition category
Condition code
Human Genetics and Inherited Disorders 305928 305928 0 0
Other human genetics and inherited disorders
Musculoskeletal 307228 307228 0 0
Other muscular and skeletal disorders

Study type
Description of intervention(s) / exposure
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.
Intervention code [1] 300482 0
Treatment: Drugs
Comparator / control treatment
No control group.
Control group

Primary outcome [1] 304960 0
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.
Timepoint [1] 304960 0
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.
Secondary outcome [1] 343743 0
Lymphocyte modulation assessed by haematological parameters lymphocytes, CD4 and CD8 T cells
Timepoint [1] 343743 0
Assessed at baseline, (week 1, day 1), and during treatment weeks 5, 8, 12, 24 and post treatment at week 28.
Secondary outcome [2] 347820 0
Performance of upper limb assessment will be assessed via the Myo-Pinch device (pinch strength),
Timepoint [2] 347820 0
Assessed at baseline (week 1, day 1) and during treatment weeks 5, 8, 12, and 24.
Secondary outcome [3] 347821 0
Performance of upper limb assessment will be assessed via the Myo-Grip device (grip strength).
Timepoint [3] 347821 0
Assessed at baseline (week 1, day 1) and during treatment weeks 5, 8, 12, and 24.
Secondary outcome [4] 375371 0
Performance of upper limb assessment will be assessed via the score as determined by the MoviPlate device (hand function) scores.
Timepoint [4] 375371 0
Assessed at baseline (week 1, day 1) and during treatment weeks 5, 8, 12, and 24.

Key inclusion criteria
Adolescent males aged 10 to 18 years with a diagnosis of non-ambulatory Duchenne Muscular Dystrophy for at least 3 months.
Minimum age
10 Years
Maximum age
18 Years
Can healthy volunteers participate?
Key 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 design
Purpose of the study
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?

Intervention assignment
Single group
Other design features
Phase 2
Type of endpoint/s
Statistical methods / analysis
A clinically important reduction in lymphocyte count from baseline to end of treatment was judged to be 25%, equating to a reduction of 0.47 (=0.75x1.89 x109/L) in mean lymphocyte count. A level of significance was set to 0.05 with a 2-sided paired t-test, resulting in a sample size of at least 9 participants being required to achieve a power of 80%.
Quantitative safety, efficacy, PK and PD data will be summarised by descriptive statistics (arithmetic mean, SD, SEM, median, minimum, and maximum) for both cohorts as well as overall and over time. Summaries will be presented for the change from baseline, when appropriate. Qualitative variables will be summarised by frequency, percentage of patients, and Clopper-Pearson 95% CI for the cohort, overall ,and over time. For key efficacy variables, the paired t-test will be used to test the change from baseline to end of treatment.

Recruitment status
Date of first participant enrolment
Date of last participant enrolment
Date of last data collection
Sample size
Accrual to date
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 10231 0
The Royal Childrens Hospital - Parkville
Recruitment postcode(s) [1] 21895 0
3052 - Parkville

Funding & Sponsors
Funding source category [1] 298819 0
Commercial sector/Industry
Name [1] 298819 0
Antisense Therapeutics Limited
Country [1] 298819 0
Primary sponsor type
Commercial sector/Industry
Antisense Therapeutics Limited
6 Wallace Avenue
Toorak, Victoria, 3142
Secondary sponsor category [1] 298015 0
Name [1] 298015 0
Address [1] 298015 0
Country [1] 298015 0

Ethics approval
Ethics application status
Ethics committee name [1] 299768 0
The Royal Children's Hospital Human Research Ethics Committee
Ethics committee address [1] 299768 0
50 Flemington Road
Parkville Victoria 3052
Ethics committee country [1] 299768 0
Date submitted for ethics approval [1] 299768 0
Approval date [1] 299768 0
Ethics approval number [1] 299768 0

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.
Trial website
Trial related presentations / publications
Public notes

Principal investigator
Name 81502 0
Dr Ian Woodcock
Address 81502 0
The Royal Children's Hospital
50 Flemington Road
Parkville, Vic 3052
Country 81502 0
Phone 81502 0
+61 3 9345 5661
Fax 81502 0
Email 81502 0
Contact person for public queries
Name 81503 0
Prof Susan Turner
Address 81503 0
Percheron Therapeutics Limited
Level 30. 35 Collins Street
Country 81503 0
Phone 81503 0
+61 398278999
Fax 81503 0
Email 81503 0
Contact person for scientific queries
Name 81504 0
Dr Ian Woodcock
Address 81504 0
The Royal Children's Hospital
50 Flemington Road
Parkville, Vic 3052
Country 81504 0
Phone 81504 0
+61 3 9345 5661
Fax 81504 0
Email 81504 0

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No/undecided IPD sharing reason/comment
Privacy and intellectual property considerations

What supporting documents are/will be available?

No Supporting Document Provided
Current supporting documents:

Results publications and other study-related documents

Documents added manually
TypeIs Peer Reviewed?DOICitations or Other DetailsAttachment
Study results articleYes Woodcock IR, Tachas G, Desem N, Houweling PJ, Kean... [More Details]

Documents added automatically
SourceTitleYear of PublicationDOI
EmbaseA phase 2 open-label study of the safety and efficacy of weekly dosing of ATL1102 in patients with non-ambulatory Duchenne muscular dystrophy and pharmacology in mdx mice.2024
EmbaseEmerging therapies for Duchenne muscular dystrophy.2022
EmbaseTherapeutic approaches to preserve the musculature in Duchenne Muscular Dystrophy: The importance of the secondary therapies.2022
EmbaseCardiorespiratory management of Duchenne muscular dystrophy: emerging therapies, neuromuscular genetics, and new clinical challenges.2022
Dimensions AIVLA-4 Expression and Activation in B Cell Malignancies: Functional and Clinical Aspects2020
Dimensions AITargeting integrins in drug-resistant acute myeloid leukaemia2023