Duchenne Muscular DystrophyPhase 2NCT05938023
A Study of ATL1102 or Placebo in Participants With Non-ambulatory Duchenne Muscular Dystrophy
A Phase 2 study testing ATL1102 25mg and ATL1102 50mg in people with Duchenne Muscular Dystrophy, run by Percheron Therapeutics at 13 sites in 5 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 10 to 17 years, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy
- What is being tested?
- ATL1102 25mg and ATL1102 50mg
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 48 participants
- Where
- 13 sitesin 5 countries
- Study dates
- 18 May 2023to 15 Jan 2025
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 10 to 17 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- 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.
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- 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.
Who cannot join
- 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.
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- 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
Trial locations and contacts
13 sites in 5 countries.
13 sites in 5 countries
United Kingdom4 sites
Birmingham Heartlands Hospital
Birmingham, B9 5SS
The General Infirmary at Leeds, Leeds Teaching Hospital NHS Trust
Leeds, LS2 9NS
University College London (UCL) - Great Ormond Street Institute of Child Health (ICH)
London, WC1N 3JH
The Robert Jones and Agnes Hunt Orthopaedic Hospital
Oswestry, SY10 7AG
Australia3 sites
Royal Childrens Hospital
Melbourne
Queensland Children's Hospital
South Brisbane
The Children's Hospital at Westmead
Westmead
Turkey (Türkiye)3 sites
Eskisehir Osmangazi Universitesi Tip Fakultesi
Eskişehir
Yeditepe University Hospital
Istanbul
Marmara University Pendik Training and Research Hospital
Pendik
Serbia2 sites
University Children's Hospital
Belgrade, 11000
Mother and Child Health Care Institute
Belgrade, 11070
About this study
Official 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
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).
Read the detailed description
This Phase IIb study is a two part, multicenter study to evaluate the efficacy, safety, pharmacokinetics and pharmacodynamics of ATL1102 and will enroll 45 non-ambulant boys with Duchenne Muscular Dystrophy (DMD) aged 10 to <18 years old.
During the 24 week randomised, double-blind, placebo-controlled treatment period (Part A) participants will be enrolled and randomised to receive either ATL1102 25mg, ATL1102 50mg or matched placebo in a 1:1:1 ratio given as a weekly subcutaneous injection.
Participants will then continue to the 24 week Open Labelled Treatment Period (Part B) and continue to receive ATL1102 25mg or ATL1102 50mg for a further 24 weeks. Participants on placebo in Part A will transition to ATL1102.
The study will consist of a 4 week screening period, 24 week randomised, double-blind, placebo-controlled treatment period (Part A), 24 week open label treatment period (Part B) and 16 week follow up period.
- Conditions
- Duchenne Muscular Dystrophy
- Being tested
- ATL1102 25mg, ATL1102 50mg, Placebo
- Keywords
- Non-ambulatory, DMD
- Medical subject headings
- Muscular Dystrophy, Duchenne
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ATL1102 25mgDrug
Dose and scheduled as specified in the Arm description
ATL1102 50mgDrug
Dose and scheduled as specified in the Arm description
PlaceboDrug
Dose and scheduled as specified in the Arm description
| Study group | Role | What they receive |
|---|---|---|
| ATL1102 25mg | Experimental | ATL1102 25mg administered subcutaneously once weekly Receives: Drug: ATL1102 25mg |
| ATL1102 50mg | Experimental | ATL1102 50mg administered subcutaneously once weekly Receives: Drug: ATL1102 50mg |
| Placebo | Placebo comparator | Placebo is administered subcutaneously once weekly Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in the Performance of Upper Limb (PUL) 2.0 score from baseline to Week 25 (blinded treatment period).
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
Measured: 25 weeks
Change in the Performance of Upper Limb (PUL) 2.0 score from Week 25 to Week 49 (open label treatment period).
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
Measured: 49 weeks
Change in the Performance of Upper Limb (PUL) 2.0 score from baseline to Week 49 (combined treatment period).
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
Measured: 49 weeks
Safety measured by the incidence and frequency of adverse events, serious adverse events and suspected unexpected adverse events from baseline to Week 65
An Adverse Event is any untoward medical occurrence in a participant and does not necessarily have to have a causal relationship with the intervention.
Measured: 65 weeks
Secondary outcomes (20)
Change in the grip strength of the hand from baseline to Week 25 using a handheld dynamometer tool (MyoGrip) (blinded treatment period).
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.
Measured: 25 weeks
Change in the pinch strength of the fingers from baseline to Week 25 using handheld dynamometer tool (MyoPinch) (blinded treatment period).
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.
Measured: 25 weeks
Change in the respiratory function assessed by Forced Vital Capacity (FVC) from baseline to Week 25 (blinded treatment period).
The percent predicted for Forced Vital Capacity (FVC%p) will be calculated at multiple timepoints after respiratory function is assessed using spirometry tests
Measured: 25 weeks
Change in the respiratory function assessed by peak expiratory flow (PEF) from baseline to Week 25 (blinded treatment period).
The percent predicted for peak expiratory flow (PEF%p) will be calculated at multiple timepoints after respiratory function is assessed using spirometry tests
Measured: 25 weeks
Change in the Paediatric Quality of Life (PedsQL™) questionnaire Duchenne Muscular Dystrophy (DMD) Module from baseline to Week 25 (blinded treatment period).
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.
Measured: 25 weeks
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).
An Adverse Event is any untoward medical occurrence in a participant and does not necessarily have to have a causal relationship with the intervention.
Measured: 25 weeks
Maximum and minimum plasma concentration (Cmax and Cmin) for ATL1102 over multiple timepoints
Pharmacokinetic evaluation to evaluate dose response
Measured: 65 weeks
Area under the plasma concentration time curve (AUC) for ATL1102 over multiple timepoints
Pharmacokinetic evaluation to evaluate dose concentration over time
Measured: 65 weeks
Time to Cmax and Cmin for ATL1102 over multiple timepoints
Pharmacokinetic evaluation to evaluate concentration of ATL1102
Measured: 65 weeks
The terminal half life for ATL1102
Pharmacokinetic evaluation to evaluate the time for the ATL1102 concentration to reduce by half
Measured: 65 weeks
Change in the grip strength of the hand from Week 25 to Week 49 using a handheld dynamometer tool (MyoGrip) (open label treatment period).
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.
Measured: 49 weeks
Change in the pinch strength of the fingers from Week 25 to Week 49 using handheld dynamometer tool (MyoPinch) (open label treatment period).
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.
Measured: 49 weeks
Change in the respiratory function assessed by Forced Vital Capacity (FVC) from Week 25 to Week 49 (open label treatment period).
The percent predicted for Forced Vital Capacity (FVC%p) will be calculated at multiple timepoints after respiratory function is assessed using spirometry tests
Measured: 49 weeks
Change in the respiratory function assessed by peak expiratory flow (PEF) from Week 25 to Week 49 (open label treatment period).
The percent predicted for peak expiratory flow (PEF%p) will be calculated at multiple timepoints after respiratory function is assessed using spirometry tests
Measured: 49 weeks
Change in the Paediatric Quality of Life (PedsQL) questionnaire Duchenne Muscular Dystrophy (DMD) Module from Week 25 to Week 49 (open label treatment period).
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.
Measured: 49 weeks
Change in the grip strength of the hand from baseline to Week 49 using a handheld dynamometer tool (MyoGrip) (combined treatment period).
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.
Measured: 49 weeks
Change in the pinch strength of the fingers from Baseline to Week 49 using handheld dynamometer tool (MyoPinch) (combined treatment period).
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.
Measured: 49 weeks
Change in the respiratory function assessed by Forced Vital Capacity (FVC) from Baseline to Week 49 (combined treatment period).
The percent predicted for Forced Vital Capacity (FVC%p) will be calculated at multiple timepoints after respiratory function is assessed using spirometry tests
Measured: 49 weeks
Change in the respiratory function assessed by peak expiratory flow (PEF) from baseline to Week 49 (combined treatment period).
The percent predicted for peak expiratory flow (PEF%p) will be calculated at multiple timepoints after respiratory function is assessed using spirometry tests
Measured: 49 weeks
Change in the Paediatric Quality of Life (PedsQL) questionnaire Duchenne Muscular Dystrophy (DMD) Module from Baseline to Week 49 (combined treatment period).
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.
Measured: 49 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Jul 2023
- Study start18 May 2023
- Primary completion19 Nov 2024
- Study completion15 Jan 2025
Why it stopped: The study failed to meet its primary or secondary efficacy endpoints. In consultation with investigators, the sponsor determined that it is not in the best interests of patients for the study to continue.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Duchenne Muscular DystrophyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- randomized, double-blind, placebo controlled treatment periods followed by open labelled treatment period
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 48 participants (actual)
Sponsor and investigators
- Lead sponsor
- Percheron Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Thomas Voit, Principal investigator, UCL Great Ormond Street Institute of Child Health
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05938023
- Sponsor's protocol ID
- 1102-DMD-Pre-CT03
- Status last verified
- Jan 2025
- First submitted
- 5 Jun 2023
- Last updated
- 3 Feb 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 Feb 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official 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
- ClinicalTrials.gov ID
- NCT05938023
- Acronym / sponsor ID
- 1102-DMD-Pre-CT03
- Conditions
- Duchenne Muscular Dystrophy
- Keywords
- Non-ambulatory, DMD
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: The study failed to meet its primary or secondary efficacy endpoints. In consultation with investigators, the sponsor determined that it is not in the best interests of patients for the study to continue.
- Enrolment
- 48 participants (actual)
- Lead sponsor
- Percheron TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 18 May 2023 (actual)
- Primary completion
- 19 Nov 2024 (actual)
- Study completion
- 15 Jan 2025 (actual)
- First posted
- 10 Jul 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Detailed description
This Phase IIb study is a two part, multicenter study to evaluate the efficacy, safety, pharmacokinetics and pharmacodynamics of ATL1102 and will enroll 45 non-ambulant boys with Duchenne Muscular Dystrophy (DMD) aged 10 to <18 years old.
During the 24 week randomised, double-blind, placebo-controlled treatment period (Part A) participants will be enrolled and randomised to receive either ATL1102 25mg, ATL1102 50mg or matched placebo in a 1:1:1 ratio given as a weekly subcutaneous injection.
Participants will then continue to the 24 week Open Labelled Treatment Period (Part B) and continue to receive ATL1102 25mg or ATL1102 50mg for a further 24 weeks. Participants on placebo in Part A will transition to ATL1102.
The study will consist of a 4 week screening period, 24 week randomised, double-blind, placebo-controlled treatment period (Part A), 24 week open label treatment period (Part B) and 16 week follow up period.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
- Model description
- randomized, double-blind, placebo controlled treatment periods followed by open labelled treatment period
| Arm | Type | Receives |
|---|---|---|
| ATL1102 25mg | Experimental | Drug: ATL1102 25mgATL1102 25mg administered subcutaneously once weekly |
| ATL1102 50mg | Experimental | Drug: ATL1102 50mgATL1102 50mg administered subcutaneously once weekly |
| Placebo | Placebo comparator | Drug: PlaceboPlacebo is administered subcutaneously once weekly |
Who may qualify
- Ages
- 10 to 17 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (9 listed)
- 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
Key exclusion criteria (13 listed)
- 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).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ATL1102 25mgDose and scheduled as specified in the Arm description | Drug | ATL1102 25mg |
| ATL1102 50mgDose and scheduled as specified in the Arm description | Drug | ATL1102 50mg |
| PlaceboDose and scheduled as specified in the Arm description | Drug | Placebo |
Primary outcome
Change in the Performance of Upper Limb (PUL) 2.0 score from baseline to Week 25 (blinded treatment period).
Time frame: 25 weeks
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
Change in the Performance of Upper Limb (PUL) 2.0 score from Week 25 to Week 49 (open label treatment period).
Time frame: 49 weeks
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
Change in the Performance of Upper Limb (PUL) 2.0 score from baseline to Week 49 (combined treatment period).
Time frame: 49 weeks
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
Safety measured by the incidence and frequency of adverse events, serious adverse events and suspected unexpected adverse events from baseline to Week 65
Time frame: 65 weeks
An Adverse Event is any untoward medical occurrence in a participant and does not necessarily have to have a causal relationship with the intervention.
20 secondary outcomes
Change in the grip strength of the hand from baseline to Week 25 using a handheld dynamometer tool (MyoGrip) (blinded treatment period).
Time frame: 25 weeks
Change in the pinch strength of the fingers from baseline to Week 25 using handheld dynamometer tool (MyoPinch) (blinded treatment period).
Time frame: 25 weeks
Change in the respiratory function assessed by Forced Vital Capacity (FVC) from baseline to Week 25 (blinded treatment period).
Time frame: 25 weeks
Change in the respiratory function assessed by peak expiratory flow (PEF) from baseline to Week 25 (blinded treatment period).
Time frame: 25 weeks
Change in the Paediatric Quality of Life (PedsQL™) questionnaire Duchenne Muscular Dystrophy (DMD) Module from baseline to Week 25 (blinded treatment period).
Time frame: 25 weeks
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).
Time frame: 25 weeks
Maximum and minimum plasma concentration (Cmax and Cmin) for ATL1102 over multiple timepoints
Time frame: 65 weeks
Area under the plasma concentration time curve (AUC) for ATL1102 over multiple timepoints
Time frame: 65 weeks
Time to Cmax and Cmin for ATL1102 over multiple timepoints
Time frame: 65 weeks
The terminal half life for ATL1102
Time frame: 65 weeks
Change in the grip strength of the hand from Week 25 to Week 49 using a handheld dynamometer tool (MyoGrip) (open label treatment period).
Time frame: 49 weeks
Change in the pinch strength of the fingers from Week 25 to Week 49 using handheld dynamometer tool (MyoPinch) (open label treatment period).
Time frame: 49 weeks
Change in the respiratory function assessed by Forced Vital Capacity (FVC) from Week 25 to Week 49 (open label treatment period).
Time frame: 49 weeks
Change in the respiratory function assessed by peak expiratory flow (PEF) from Week 25 to Week 49 (open label treatment period).
Time frame: 49 weeks
Change in the Paediatric Quality of Life (PedsQL) questionnaire Duchenne Muscular Dystrophy (DMD) Module from Week 25 to Week 49 (open label treatment period).
Time frame: 49 weeks
Change in the grip strength of the hand from baseline to Week 49 using a handheld dynamometer tool (MyoGrip) (combined treatment period).
Time frame: 49 weeks
Change in the pinch strength of the fingers from Baseline to Week 49 using handheld dynamometer tool (MyoPinch) (combined treatment period).
Time frame: 49 weeks
Change in the respiratory function assessed by Forced Vital Capacity (FVC) from Baseline to Week 49 (combined treatment period).
Time frame: 49 weeks
Change in the respiratory function assessed by peak expiratory flow (PEF) from baseline to Week 49 (combined treatment period).
Time frame: 49 weeks
Change in the Paediatric Quality of Life (PedsQL) questionnaire Duchenne Muscular Dystrophy (DMD) Module from Baseline to Week 49 (combined treatment period).
Time frame: 49 weeks
Results and safety
The study was stopped early (reason given: The study failed to meet its primary or secondary efficacy endpoints. In consultation with investigators, the sponsor determined that it is not in the best interests of patients for the study to continue.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Feb 2025.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Thomas Voit principal investigator
UCL Great Ormond Street Institute of Child Health
13 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing ATL1102 25mg and ATL1102 50mg in people with Duchenne Muscular Dystrophy, run by Percheron Therapeutics at 13 sites in 5 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 3 Feb 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 13 sites in United Kingdom, Australia, Turkey (Türkiye), Serbia, Bulgaria. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: The study failed to meet its primary or secondary efficacy endpoints. In consultation with investigators, the sponsor determined that it is not in the best interests of patients for the study to continue.). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in the Performance of Upper Limb (PUL) 2.0 score from baseline to Week 25 (blinded treatment period). (time frame: 25 weeks). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Feb 2025.