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

P

Percheron Therapeutics

Status and phase

Active, not recruiting
Phase 2

Conditions

Duchenne Muscular Dystrophy

Treatments

Drug: Placebo
Drug: ATL1102 50mg
Drug: ATL1102 25mg

Study type

Interventional

Funder types

Industry

Identifiers

NCT05938023
1102-DMD-Pre-CT03

Details and patient eligibility

About

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).

Full 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.

Enrollment

48 patients

Sex

Male

Ages

10 to 17 years old

Volunteers

No Healthy Volunteers

Inclusion and exclusion 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

Trial design

Primary purpose

Treatment

Allocation

Randomized

Interventional model

Parallel Assignment

Masking

Quadruple Blind

48 participants in 3 patient groups, including a placebo group

ATL1102 25mg
Experimental group
Description:
ATL1102 25mg administered subcutaneously once weekly
Treatment:
Drug: ATL1102 25mg
ATL1102 50mg
Experimental group
Description:
ATL1102 50mg administered subcutaneously once weekly
Treatment:
Drug: ATL1102 50mg
Placebo
Placebo Comparator group
Description:
Placebo is administered subcutaneously once weekly
Treatment:
Drug: Placebo

Trial contacts and locations

13

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Central trial contact

Andrew McKenzie; Susan Turner

Data sourced from clinicaltrials.gov

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