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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05938023




Registration number
NCT05938023
Ethics application status
Date submitted
5/06/2023
Date registered
10/07/2023

Titles & IDs
Public title
A Study of ATL1102 or Placebo in Participants with Non-ambulatory Duchenne Muscular Dystrophy
Scientific title
A Multicentre, Randomised, Double-blind, Placebo-controlled and Open Label Extension Study to Assess the Efficacy, Safety, and Pharmacokinetic Profile of of ATL1102 in Non-ambulatory Participants with Duchenne Muscular Dystrophy
Secondary ID [1] 0 0
1102-DMD-Pre-CT03
Universal Trial Number (UTN)
Trial acronym
Linked study record

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

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - ATL1102 25mg
Treatment: Drugs - ATL1102 50mg
Treatment: Drugs - Placebo

Experimental: ATL1102 25mg - ATL1102 25mg administered subcutaneously once weekly

Experimental: ATL1102 50mg - ATL1102 50mg administered subcutaneously once weekly

Placebo comparator: Placebo - Placebo is administered subcutaneously once weekly


Treatment: Drugs: ATL1102 25mg
Dose and scheduled as specified in the Arm description

Treatment: Drugs: ATL1102 50mg
Dose and scheduled as specified in the Arm description

Treatment: Drugs: Placebo
Dose and scheduled as specified in the Arm description
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Change in the Performance of Upper Limb (PUL) 2.0 score from baseline to Week 25 (blinded treatment period).
Assessment method [1] 0 0
The PUL is an assessment used to evaluate the upper limb strength for individuals with DMD where a higher score indicates a better outcome with a minimum of 0 and a maximum score of 42
Timepoint [1] 0 0
25 weeks
Primary outcome [2] 0 0
Change in the Performance of Upper Limb (PUL) 2.0 score from Week 25 to Week 49 (open label treatment period).
Assessment method [2] 0 0
The PUL is an assessment used to evaluate the upper limb strength for individuals with DMD where a higher score indicates a better outcome with a minimum of 0 and a maximum score of 42
Timepoint [2] 0 0
49 weeks
Primary outcome [3] 0 0
Change in the Performance of Upper Limb (PUL) 2.0 score from baseline to Week 49 (combined treatment period).
Assessment method [3] 0 0
The PUL is an assessment used to evaluate the upper limb strength for individuals with DMD where a higher score indicates a better outcome with a minimum of 0 and a maximum score of 42
Timepoint [3] 0 0
49 weeks
Primary outcome [4] 0 0
Safety measured by the incidence and frequency of adverse events, serious adverse events and suspected unexpected adverse events from baseline to Week 65
Assessment method [4] 0 0
An Adverse Event is any untoward medical occurrence in a participant and does not necessarily have to have a causal relationship with the intervention.
Timepoint [4] 0 0
65 weeks
Secondary outcome [1] 0 0
Change in the grip strength of the hand from baseline to Week 25 using a handheld dynamometer tool (MyoGrip) (blinded treatment period).
Assessment method [1] 0 0
The handheld dynamometer tool (MyoGrip) use strain gauge technology to measure the hand strength in Kilograms exerted by the participants with higher recordings indicating greater hand strength.
Timepoint [1] 0 0
25 weeks
Secondary outcome [2] 0 0
Change in the pinch strength of the fingers from baseline to Week 25 using handheld dynamometer tool (MyoPinch) (blinded treatment period).
Assessment method [2] 0 0
The handheld dynamometer tool (MyoPinch) use strain gauge technology to measure the pinch strength of the fingers in Kilograms exerted by the participants with higher recordings indicating greater hand strength.
Timepoint [2] 0 0
25 weeks
Secondary outcome [3] 0 0
Change in the respiratory function assessed by Forced Vital Capacity (FVC) from baseline to Week 25 (blinded treatment period).
Assessment method [3] 0 0
The percent predicted for Forced Vital Capacity (FVC%p) will be calculated at multiple timepoints after respiratory function is assessed using spirometry tests
Timepoint [3] 0 0
25 weeks
Secondary outcome [4] 0 0
Change in the respiratory function assessed by peak expiratory flow (PEF) from baseline to Week 25 (blinded treatment period).
Assessment method [4] 0 0
The percent predicted for peak expiratory flow (PEF%p) will be calculated at multiple timepoints after respiratory function is assessed using spirometry tests
Timepoint [4] 0 0
25 weeks
Secondary outcome [5] 0 0
Change in the Paediatric Quality of Life (PedsQL™) questionnaire Duchenne Muscular Dystrophy (DMD) Module from baseline to Week 25 (blinded treatment period).
Assessment method [5] 0 0
Health related quality of life is assessed by percentage of change in the score collected in the Paediatric Quality of Life (PedsQL™) Duchenne Muscular Dystrophy (DMD) Module for participants and parents at multiple timepoints. A higher score indicates a better health related quality of life with a minimum of 0 and a maximum score of 100.
Timepoint [5] 0 0
25 weeks
Secondary outcome [6] 0 0
Safety measured by the incidence and frequency of adverse events, serious adverse events and suspected unexpected adverse events from baseline to Week 25 (blinded treatment period).
Assessment method [6] 0 0
An Adverse Event is any untoward medical occurrence in a participant and does not necessarily have to have a causal relationship with the intervention.
Timepoint [6] 0 0
25 weeks
Secondary outcome [7] 0 0
Maximum and minimum plasma concentration (Cmax and Cmin) for ATL1102 over multiple timepoints
Assessment method [7] 0 0
Pharmacokinetic evaluation to evaluate dose response
Timepoint [7] 0 0
65 weeks
Secondary outcome [8] 0 0
Area under the plasma concentration time curve (AUC) for ATL1102 over multiple timepoints
Assessment method [8] 0 0
Pharmacokinetic evaluation to evaluate dose concentration over time
Timepoint [8] 0 0
65 weeks
Secondary outcome [9] 0 0
Time to Cmax and Cmin for ATL1102 over multiple timepoints
Assessment method [9] 0 0
Pharmacokinetic evaluation to evaluate concentration of ATL1102
Timepoint [9] 0 0
65 weeks
Secondary outcome [10] 0 0
The terminal half life for ATL1102
Assessment method [10] 0 0
Pharmacokinetic evaluation to evaluate the time for the ATL1102 concentration to reduce by half
Timepoint [10] 0 0
65 weeks
Secondary outcome [11] 0 0
Change in the grip strength of the hand from Week 25 to Week 49 using a handheld dynamometer tool (MyoGrip) (open label treatment period).
Assessment method [11] 0 0
The handheld dynamometer tool (MyoGrip) use strain gauge technology to measure the hand strength in Kilograms exerted by the participants with higher recordings indicating greater hand strength.
Timepoint [11] 0 0
49 weeks
Secondary outcome [12] 0 0
Change in the pinch strength of the fingers from Week 25 to Week 49 using handheld dynamometer tool (MyoPinch) (open label treatment period).
Assessment method [12] 0 0
The handheld dynamometer tool (MyoPinch) use strain gauge technology to measure the pinch strength of the fingers in Kilograms exerted by the participants with higher recordings indicating greater hand strength.
Timepoint [12] 0 0
49 weeks
Secondary outcome [13] 0 0
Change in the respiratory function assessed by Forced Vital Capacity (FVC) from Week 25 to Week 49 (open label treatment period).
Assessment method [13] 0 0
The percent predicted for Forced Vital Capacity (FVC%p) will be calculated at multiple timepoints after respiratory function is assessed using spirometry tests
Timepoint [13] 0 0
49 weeks
Secondary outcome [14] 0 0
Change in the respiratory function assessed by peak expiratory flow (PEF) from Week 25 to Week 49 (open label treatment period).
Assessment method [14] 0 0
The percent predicted for peak expiratory flow (PEF%p) will be calculated at multiple timepoints after respiratory function is assessed using spirometry tests
Timepoint [14] 0 0
49 weeks
Secondary outcome [15] 0 0
Change in the Paediatric Quality of Life (PedsQL) questionnaire Duchenne Muscular Dystrophy (DMD) Module from Week 25 to Week 49 (open label treatment period).
Assessment method [15] 0 0
Health related quality of life is assessed by percentage of change in the score collected in the Paediatric Quality of Life (PedsQL™) Duchenne Muscular Dystrophy (DMD) Module for participants and parents at multiple timepoints. A higher score indicates a better health related quality of life with a minimum of 0 and a maximum score of 100.
Timepoint [15] 0 0
49 weeks
Secondary outcome [16] 0 0
Change in the grip strength of the hand from baseline to Week 49 using a handheld dynamometer tool (MyoGrip) (combined treatment period).
Assessment method [16] 0 0
The handheld dynamometer tool (MyoGrip) use strain gauge technology to measure the hand strength in Kilograms exerted by the participants with higher recordings indicating greater hand strength.
Timepoint [16] 0 0
49 weeks
Secondary outcome [17] 0 0
Change in the pinch strength of the fingers from Baseline to Week 49 using handheld dynamometer tool (MyoPinch) (combined treatment period).
Assessment method [17] 0 0
The handheld dynamometer tool (MyoPinch) use strain gauge technology to measure the pinch strength of the fingers in Kilograms exerted by the participants with higher recordings indicating greater hand strength.
Timepoint [17] 0 0
49 weeks
Secondary outcome [18] 0 0
Change in the respiratory function assessed by Forced Vital Capacity (FVC) from Baseline to Week 49 (combined treatment period).
Assessment method [18] 0 0
The percent predicted for Forced Vital Capacity (FVC%p) will be calculated at multiple timepoints after respiratory function is assessed using spirometry tests
Timepoint [18] 0 0
49 weeks
Secondary outcome [19] 0 0
Change in the respiratory function assessed by peak expiratory flow (PEF) from baseline to Week 49 (combined treatment period).
Assessment method [19] 0 0
The percent predicted for peak expiratory flow (PEF%p) will be calculated at multiple timepoints after respiratory function is assessed using spirometry tests
Timepoint [19] 0 0
49 weeks
Secondary outcome [20] 0 0
Change in the Paediatric Quality of Life (PedsQL) questionnaire Duchenne Muscular Dystrophy (DMD) Module from Baseline to Week 49 (combined treatment period).
Assessment method [20] 0 0
Health related quality of life is assessed by percentage of change in the score collected in the Paediatric Quality of Life (PedsQL™) Duchenne Muscular Dystrophy (DMD) Module for participants and parents at multiple timepoints. A higher score indicates a better health related quality of life with a minimum of 0 and a maximum score of 100.
Timepoint [20] 0 0
49 weeks

Eligibility
Key inclusion criteria
Key

* Has a clinical diagnosis of DMD confirmed by validated genetic testing
* Is considered to be non-ambulatory, defined as unable to walk 10 meters without assistance or help at Screening.
* Male aged 10 to less than 18 years, at the time of Screening.
* Body weight of at least 25 kg at Screening.
* If receiving corticosteroid therapy, therapy was initiated at least six months prior to the baseline visit and a stable daily dose for at least 3 months prior to baseline
* Participant has a Performance of Upper Limb Module for DMD 2.0 (PUL 2.0) Entry Item A score =2.
* Able to perform spirometry and has sufficient Respiratory function defined as reproducible percent predicted FVC =50%.
* Has adequate cardiac function defined as left ventricular ejection fraction (LVEF) =45% by echocardiogram and if receiving cardiac medication, must be currently on a stable regimen and doses of cardiac therapy (at least 3 months prior to baseline Day 1)
* Participant and their parent/guardian/carer are willing and able to comply with scheduled visits, study medication administration and study procedures.

Key
Minimum age
10 Years
Maximum age
17 Years
Sex
Males
Can healthy volunteers participate?
No
Key exclusion criteria
* Participation in another clinical trial (non-interventional) or administration of any investigational product or experimental product within 12 weeks or 5 half-lives (whichever is longer) preceding Day 1.
* Exposure to more than 3 investigational products within the 12 months prior to Day 1.
* History of clinically significant bleeding or coagulation abnormalities or clinically significant abnormal coagulation parameters.
* Currently receiving antiplatelet or anticoagulant therapy or has taken medication with an antiplatelet or anticoagulant effect within 4 weeks prior Day 1
* Any evidence of clinically significant structural or functional heart abnormality (cardiomyopathy that is managed by ACEi or beta blockers is acceptable provided the LVEF inclusion criterion is met).
* Known history of or a positive test for hepatitis B surface antigen (HBsAg), hepatitis C (HCV) antibodies, human immunodeficiency virus (HIV) antibodies at Screening.
* Evidence of renal impairment and/or cystatin C >1.4 mg/L.
* Received a live vaccine (including intranasal influenza vaccine) within 4 weeks prior to Day 1 or planned live vaccination during the study period.
* Asthma (if requiring regular medication), bronchitis/chronic obstructive pulmonary disease (COPD), bronchiectasis, emphysema, pneumonia or the presence of any non-DMD respiratory illness that affects PEF and FVC or other respiratory measures.
* Requires day-time assisted mechanical or non-invasive ventilation (NIV) (night time NIV is permitted).
* Chronic use (daily intake >14 days), within one month of Day 1, of beta-2 agonists or any use of other bronchodilating medication (e.g., inhaled steroids, sympathomimetics, anticholinergics).
* Used carnitine, creatine, glutamine, oxatomide, idebenone or other forms of coenzyme Q10 or vitamin E or any other nutritional or antioxidant supplements or herbal medicines or anabolic steroids other than standard corticosteroids or puberty testosterone supplementation within 4 weeks of Day 1.
* Has an increased risk for opportunistic infections or systemic medical conditions resulting in significantly compromised immune system function

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled 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
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Stopped early
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
18/05/2023
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
15/01/2025
Sample size
Target
Accrual to date
Final
48
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Royal Childrens Hospital - Melbourne
Recruitment hospital [2] 0 0
Queensland Children's Hospital - South Brisbane
Recruitment hospital [3] 0 0
The Children's Hospital at Westmead - Westmead
Recruitment postcode(s) [1] 0 0
- Melbourne
Recruitment postcode(s) [2] 0 0
- South Brisbane
Recruitment postcode(s) [3] 0 0
- Westmead
Recruitment outside Australia
Country [1] 0 0
Bulgaria
State/province [1] 0 0
Sofia
Country [2] 0 0
Serbia
State/province [2] 0 0
Belgrade
Country [3] 0 0
Turkey
State/province [3] 0 0
Eskisehir
Country [4] 0 0
Turkey
State/province [4] 0 0
Istanbul
Country [5] 0 0
Turkey
State/province [5] 0 0
Pendik
Country [6] 0 0
United Kingdom
State/province [6] 0 0
Birmingham
Country [7] 0 0
United Kingdom
State/province [7] 0 0
Leeds
Country [8] 0 0
United Kingdom
State/province [8] 0 0
London
Country [9] 0 0
United Kingdom
State/province [9] 0 0
Oswestry

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Percheron Therapeutics
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Public notes

Contacts
Principal investigator
Name 0 0
Thomas Voit
Address 0 0
UCL Great Ormond Street Institute of Child Health
Country 0 0
Phone 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT05938023