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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT05938023
Registration number
NCT05938023
Ethics application status
Date submitted
5/06/2023
Date registered
10/07/2023
Date last updated
30/05/2024
Titles & IDs
Public title
A Study of ATL1102 or Placebo in Participants With Non-ambulatory Duchenne Muscular Dystrophy
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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
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Secondary ID [1]
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1102-DMD-Pre-CT03
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Duchenne Muscular Dystrophy
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Condition category
Condition code
Musculoskeletal
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Other muscular and skeletal disorders
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Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Neurological
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Other neurological disorders
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Intervention/exposure
Study type
Interventional
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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
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Change in the Performance of Upper Limb (PUL) 2.0 score from baseline to Week 25 (blinded treatment period).
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Assessment method [1]
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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
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Timepoint [1]
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25 weeks
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Primary outcome [2]
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Change in the Performance of Upper Limb (PUL) 2.0 score from Week 25 to Week 49 (open label treatment period).
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Assessment method [2]
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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
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Timepoint [2]
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49 weeks
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Primary outcome [3]
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Change in the Performance of Upper Limb (PUL) 2.0 score from baseline to Week 49 (combined treatment period).
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Assessment method [3]
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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
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Timepoint [3]
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49 weeks
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Primary outcome [4]
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Safety measured by the incidence and frequency of adverse events, serious adverse events and suspected unexpected adverse events from baseline to Week 65
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Assessment method [4]
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An Adverse Event is any untoward medical occurrence in a participant and does not necessarily have to have a causal relationship with the intervention.
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Timepoint [4]
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65 weeks
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Secondary outcome [1]
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Change in the grip strength of the hand from baseline to Week 25 using a handheld dynamometer tool (MyoGrip) (blinded treatment period).
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Assessment method [1]
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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.
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Timepoint [1]
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25 weeks
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Secondary outcome [2]
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Change in the pinch strength of the fingers from baseline to Week 25 using handheld dynamometer tool (MyoPinch) (blinded treatment period).
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Assessment method [2]
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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.
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Timepoint [2]
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25 weeks
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Secondary outcome [3]
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Change in the respiratory function assessed by Forced Vital Capacity (FVC) from baseline to Week 25 (blinded treatment period).
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Assessment method [3]
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The percent predicted for Forced Vital Capacity (FVC%p) will be calculated at multiple timepoints after respiratory function is assessed using spirometry tests
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Timepoint [3]
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25 weeks
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Secondary outcome [4]
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Change in the respiratory function assessed by peak expiratory flow (PEF) from baseline to Week 25 (blinded treatment period).
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Assessment method [4]
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The percent predicted for peak expiratory flow (PEF%p) will be calculated at multiple timepoints after respiratory function is assessed using spirometry tests
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Timepoint [4]
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25 weeks
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Secondary outcome [5]
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Change in the Paediatric Quality of Life (PedsQLâ„¢) questionnaire Duchenne Muscular Dystrophy (DMD) Module from baseline to Week 25 (blinded treatment period).
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Assessment method [5]
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Health related quality of life is assessed by percentage of change in the score collected in the Paediatric Quality of Life (PedsQLâ„¢) 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.
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Timepoint [5]
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25 weeks
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Secondary outcome [6]
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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).
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Assessment method [6]
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An Adverse Event is any untoward medical occurrence in a participant and does not necessarily have to have a causal relationship with the intervention.
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Timepoint [6]
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25 weeks
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Secondary outcome [7]
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Maximum and minimum plasma concentration (Cmax and Cmin) for ATL1102 over multiple timepoints
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Assessment method [7]
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Pharmacokinetic evaluation to evaluate dose response
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Timepoint [7]
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65 weeks
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Secondary outcome [8]
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Area under the plasma concentration time curve (AUC) for ATL1102 over multiple timepoints
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Assessment method [8]
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Pharmacokinetic evaluation to evaluate dose concentration over time
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Timepoint [8]
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65 weeks
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Secondary outcome [9]
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Time to Cmax and Cmin for ATL1102 over multiple timepoints
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Assessment method [9]
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Pharmacokinetic evaluation to evaluate concentration of ATL1102
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Timepoint [9]
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65 weeks
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Secondary outcome [10]
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The terminal half life for ATL1102
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Assessment method [10]
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Pharmacokinetic evaluation to evaluate the time for the ATL1102 concentration to reduce by half
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Timepoint [10]
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65 weeks
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Secondary outcome [11]
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Change in the grip strength of the hand from Week 25 to Week 49 using a handheld dynamometer tool (MyoGrip) (open label treatment period).
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Assessment method [11]
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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.
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Timepoint [11]
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49 weeks
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Secondary outcome [12]
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Change in the pinch strength of the fingers from Week 25 to Week 49 using handheld dynamometer tool (MyoPinch) (open label treatment period).
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Assessment method [12]
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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.
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Timepoint [12]
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49 weeks
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Secondary outcome [13]
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Change in the respiratory function assessed by Forced Vital Capacity (FVC) from Week 25 to Week 49 (open label treatment period).
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Assessment method [13]
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The percent predicted for Forced Vital Capacity (FVC%p) will be calculated at multiple timepoints after respiratory function is assessed using spirometry tests
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Timepoint [13]
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49 weeks
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Secondary outcome [14]
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Change in the respiratory function assessed by peak expiratory flow (PEF) from Week 25 to Week 49 (open label treatment period).
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Assessment method [14]
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The percent predicted for peak expiratory flow (PEF%p) will be calculated at multiple timepoints after respiratory function is assessed using spirometry tests
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Timepoint [14]
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49 weeks
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Secondary outcome [15]
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Change in the Paediatric Quality of Life (PedsQL) questionnaire Duchenne Muscular Dystrophy (DMD) Module from Week 25 to Week 49 (open label treatment period).
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Assessment method [15]
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Health related quality of life is assessed by percentage of change in the score collected in the Paediatric Quality of Life (PedsQLâ„¢) 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.
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Timepoint [15]
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49 weeks
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Secondary outcome [16]
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Change in the grip strength of the hand from baseline to Week 49 using a handheld dynamometer tool (MyoGrip) (combined treatment period).
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Assessment method [16]
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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.
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Timepoint [16]
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49 weeks
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Secondary outcome [17]
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Change in the pinch strength of the fingers from Baseline to Week 49 using handheld dynamometer tool (MyoPinch) (combined treatment period).
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Assessment method [17]
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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.
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Timepoint [17]
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49 weeks
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Secondary outcome [18]
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Change in the respiratory function assessed by Forced Vital Capacity (FVC) from Baseline to Week 49 (combined treatment period).
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Assessment method [18]
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The percent predicted for Forced Vital Capacity (FVC%p) will be calculated at multiple timepoints after respiratory function is assessed using spirometry tests
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Timepoint [18]
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49 weeks
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Secondary outcome [19]
0
0
Change in the respiratory function assessed by peak expiratory flow (PEF) from baseline to Week 49 (combined treatment period).
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Assessment method [19]
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The percent predicted for peak expiratory flow (PEF%p) will be calculated at multiple timepoints after respiratory function is assessed using spirometry tests
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Timepoint [19]
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49 weeks
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Secondary outcome [20]
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Change in the Paediatric Quality of Life (PedsQL) questionnaire Duchenne Muscular Dystrophy (DMD) Module from Baseline to Week 49 (combined treatment period).
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Assessment method [20]
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Health related quality of life is assessed by percentage of change in the score collected in the Paediatric Quality of Life (PedsQLâ„¢) 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.
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Timepoint [20]
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49 weeks
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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
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Minimum age
10
Years
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Maximum age
17
Years
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Sex
Males
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Can healthy volunteers participate?
No
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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
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Blinded (masking used)
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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
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 2
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Active, not recruiting
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
18/05/2023
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
5/03/2025
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Actual
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Sample size
Target
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Accrual to date
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Final
48
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Recruitment in Australia
Recruitment state(s)
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Recruitment hospital [1]
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Royal Childrens Hospital - Melbourne
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Recruitment hospital [2]
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Queensland Children's Hospital - South Brisbane
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Recruitment hospital [3]
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The Children's Hospital at Westmead - Westmead
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Recruitment postcode(s) [1]
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- Melbourne
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Recruitment postcode(s) [2]
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- South Brisbane
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Recruitment postcode(s) [3]
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- Westmead
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Recruitment outside Australia
Country [1]
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Bulgaria
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State/province [1]
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Sofia
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Country [2]
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Serbia
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State/province [2]
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Belgrade
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Country [3]
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Turkey
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State/province [3]
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Eskisehir
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Country [4]
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Turkey
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State/province [4]
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Istanbul
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Country [5]
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Turkey
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State/province [5]
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Pendik
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Country [6]
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United Kingdom
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State/province [6]
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Birmingham
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Country [7]
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United Kingdom
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State/province [7]
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Leeds
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Country [8]
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United Kingdom
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State/province [8]
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London
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Country [9]
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United Kingdom
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State/province [9]
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Oswestry
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
Percheron Therapeutics
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Address
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Country
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Ethics approval
Ethics application status
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Summary
Brief summary
This Phase IIb study is a two part, multicenter study to evaluate the efficacy, safety,
pharmacokinetics and pharmacodynamics of ATL1102 in non-ambulant boys with Duchenne Muscular
Dystrophy aged 10 to <18 years old. The study includes a randomised, double-blind,
placebo-controlled treatment period (Part A), followed by an open labelled treatment period
(Part B).
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Trial website
https://clinicaltrials.gov/ct2/show/NCT05938023
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Thomas Voit
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Address
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UCL Great Ormond Street Institute of Child Health
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Country
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Address
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Country
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Phone
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Fax
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Email
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT05938023
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