TerminatedNCT05938023Updated Feb 3, 2025

A Study of ATL1102 or Placebo in Participants with Non-ambulatory Duchenne Muscular Dystrophy

A Phase 2 interventional study of ATL1102 25mg and ATL1102 50mg in Duchenne Muscular Dystrophy, sponsored by Percheron Therapeutics. Terminated at 13 sites in 5 countries. Open to male participants aged 10 Years to 17 Years. Per ClinicalTrials.gov, last updated 2025-02-03.

Sponsored by Percheron Therapeutics · Phase 2, Interventional, and Treatment

Why this study was terminated

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.

Phase: Phase 2
Study type: Interventional
Enrollment: 48
Allocation: Randomized
Ages: 10 Years to 17 Years
Sex: Male

Study 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).

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 studied

Duchenne Muscular Dystrophy

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Keywords

Non-ambulatory
DMD

In context

Muscular Dystrophies

548 studies on the registry are indexed under Muscular Dystrophies; 89 are open to participants now.

This study's enrollment of 48 is above the median of 24 across 344 interventional studies indexed under Muscular Dystrophies.

Browse Muscular Dystrophies studies →

Lead sponsor

This is the only study on the registry with Percheron Therapeutics as lead sponsor.

Counted across the registry records on this site, refreshed daily.

Who can participate

Ages eligible: 10 Years to 17 Years
Sexes eligible: Male
Accepts healthy volunteers: No

Eligibility criteria

Key Inclusion Criteria:

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 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

Phase: Phase 2
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel assignment
Masking: Quadruple (Participant, Care provider, Investigator, Outcomes assessor)
Enrollment: 48 participants (actual)

Study arms

Experimental
ATL1102 25mg
ATL1102 25mg administered subcutaneously once weekly
Drug: ATL1102 25mg
Experimental
ATL1102 50mg
ATL1102 50mg administered subcutaneously once weekly
Drug: ATL1102 50mg
Placebo comparator
Placebo
Placebo is administered subcutaneously once weekly
Drug: Placebo

Interventions

DrugATL1102 25mg
Dose and scheduled as specified in the Arm description
DrugATL1102 50mg
Dose and scheduled as specified in the Arm description
DrugPlacebo
Dose and scheduled as specified in the Arm description

What researchers measure

Primary outcomes

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
Time frame: 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
Time frame: 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
Time frame: 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.
Time frame: 65 weeks

Secondary outcomes

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.
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).
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.
Time frame: 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
Time frame: 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
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).
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.
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).
An Adverse Event is any untoward medical occurrence in a participant and does not necessarily have to have a causal relationship with the intervention.
Time frame: 25 weeks
Maximum and minimum plasma concentration (Cmax and Cmin) for ATL1102 over multiple timepoints
Pharmacokinetic evaluation to evaluate dose response
Time frame: 65 weeks
Area under the plasma concentration time curve (AUC) for ATL1102 over multiple timepoints
Pharmacokinetic evaluation to evaluate dose concentration over time
Time frame: 65 weeks
Time to Cmax and Cmin for ATL1102 over multiple timepoints
Pharmacokinetic evaluation to evaluate concentration of ATL1102
Time frame: 65 weeks
The terminal half life for ATL1102
Pharmacokinetic evaluation to evaluate the time for the ATL1102 concentration to reduce by half
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).
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.
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).
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.
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).
The percent predicted for Forced Vital Capacity (FVC%p) will be calculated at multiple timepoints after respiratory function is assessed using spirometry tests
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).
The percent predicted for peak expiratory flow (PEF%p) will be calculated at multiple timepoints after respiratory function is assessed using spirometry tests
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).
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.
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).
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.
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).
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.
Time frame: 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
Time frame: 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
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).
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.
Time frame: 49 weeks

Other outcomes

Changes in lymphocyte populations to assess pharmacodynamic effects of ATL1102 from baseline to Week 57
Lymphocyte population (cells/L) including cells expressing CD49d will be evaluated at multiple timepoints during the study utilizing chip cytometry.
Time frame: 57 weeks

Study locations

13 sites

Royal Childrens Hospital
Melbourne, Australia
Queensland Children's Hospital
South Brisbane, Australia
The Children's Hospital at Westmead
Westmead, Australia
UMHAT Aleksandrovska Neurology clinic
Sofia, 1432, Bulgaria
University Children's Hospital
Belgrade, 11000, Serbia
Mother and Child Health Care Institute
Belgrade, 11070, Serbia
Eskisehir Osmangazi Universitesi Tip Fakultesi
Eskisehir, Turkey
Yeditepe University Hospital
Istanbul, Turkey
Marmara University Pendik Training and Research Hospital
Pendik, Turkey
Birmingham Heartlands Hospital
Birmingham, B9 5SS, United Kingdom
The General Infirmary at Leeds, Leeds Teaching Hospital NHS Trust
Leeds, LS2 9NS, United Kingdom
University College London (UCL) - Great Ormond Street Institute of Child Health (ICH)
London, WC1N 3JH, United Kingdom
The Robert Jones and Agnes Hunt Orthopaedic Hospital
Oswestry, SY10 7AG, United Kingdom

References and documents

Individual participant data

Plan to share: No

No publications or documents are linked to this record.

Updates

Tracking since Sep 25, 2026

No changes since tracking began. The registry record was last updated on Feb 3, 2025, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗

Registry details

Key details

Study ID: NCT05938023
Lead sponsor: Percheron Therapeutics
Responsible party: Sponsor
First posted: Jul 10, 2023
Start date: May 18, 2023
Primary completion: Nov 19, 2024
Completion: Jan 15, 2025
Last update: Feb 3, 2025

Study contacts

Thomas Voit

principal investigator · UCL Great Ormond Street Institute of Child Health

Oversight

Data monitoring committee: Yes
FDA-regulated drug: No
FDA-regulated device: No

View the source record on ClinicalTrials.gov ↗

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