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

NCT05938023 | Terminated Phase 2 DMC

• 1 other identifier: 1102-DMD-Pre-CT03
• Study Type: interventional
• Target: 48
• Locations: 5 countries
• Sites: 13

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).

Conditions

Duchenne Muscular Dystrophy

Keywords

Non-ambulatory; DMD

Outcome Measures

Primary Outcomes (4)

• 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

  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

  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

  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.

  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).

  25 weeks

• Change in the pinch strength of the fingers from baseline to Week 25 using handheld dynamometer tool (MyoPinch) (blinded treatment period).

  25 weeks

• Change in the respiratory function assessed by Forced Vital Capacity (FVC) from baseline to Week 25 (blinded treatment period).

  25 weeks

• Change in the respiratory function assessed by peak expiratory flow (PEF) from baseline to Week 25 (blinded treatment period).

  25 weeks

• Change in the Paediatric Quality of Life (PedsQL™) questionnaire Duchenne Muscular Dystrophy (DMD) Module from baseline to Week 25 (blinded treatment period).

  25 weeks

Other Outcomes (1)

• Changes in lymphocyte populations to assess pharmacodynamic effects of ATL1102 from baseline to Week 57

  57 weeks

Study Arms (3)

ATL1102 25mg

EXPERIMENTAL

ATL1102 25mg administered subcutaneously once weekly

Drug: ATL1102 25mg

ATL1102 50mg

EXPERIMENTAL

ATL1102 50mg administered subcutaneously once weekly

Drug: ATL1102 50mg

Placebo

PLACEBO COMPARATOR

Placebo is administered subcutaneously once weekly

Drug: Placebo

Interventions

ATL1102 25mg DRUG

Dose and scheduled as specified in the Arm description

ATL1102 25mg

ATL1102 50mg DRUG

Dose and scheduled as specified in the Arm description

ATL1102 50mg

Placebo DRUG

Dose and scheduled as specified in the Arm description

Placebo

Eligibility Criteria

• Age: 10 Years - 17 Years
• Gender Eligibility Details: DMD is a disease that predominantly affects males
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• 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
• 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

Sponsors & Collaborators

• Percheron Therapeutics: lead

Study Sites (13)

Royal Childrens Hospital

Melbourne, Australia

Location

Queensland Children's Hospital

South Brisbane, Australia

Location

The Children's Hospital at Westmead

Westmead, Australia

Location

UMHAT Aleksandrovska Neurology clinic

Sofia, Sofia, 1432, Bulgaria

Location

University Children's Hospital

Belgrade, 11000, Serbia

Location

Mother and Child Health Care Institute

Belgrade, 11070, Serbia

Location

Eskisehir Osmangazi Universitesi Tip Fakultesi

Eskişehir, Turkey (Türkiye)

Location

Yeditepe University Hospital

Istanbul, Turkey (Türkiye)

Location

Marmara University Pendik Training and Research Hospital

Pendik, Turkey (Türkiye)

Location

Birmingham Heartlands Hospital

Birmingham, B9 5SS, United Kingdom

Location

The General Infirmary at Leeds, Leeds Teaching Hospital NHS Trust

Leeds, LS2 9NS, United Kingdom

Location

University College London (UCL) - Great Ormond Street Institute of Child Health (ICH)

London, WC1N 3JH, United Kingdom

Location

The Robert Jones and Agnes Hunt Orthopaedic Hospital

Oswestry, SY10 7AG, United Kingdom

Location

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Thomas Voit

  UCL Great Ormond Street Institute of Child Health

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: randomized, double-blind, placebo controlled treatment periods followed by open labelled treatment period

Study Record Dates

• First Submitted: June 5, 2023
• First Posted: July 10, 2023
• Study Start: May 18, 2023
• Primary Completion: November 19, 2024
• Study Completion: January 15, 2025
• Last Updated: February 3, 2025

Record last verified: 2025-01

Data Sharing

• IPD Sharing: Will not share

Locations

GB (4); AU (3); SE (2); BU (1); TU (3)