Phase 2 Muscular Dystrophy Trial, Recruiting

Written by an AI model from ClinicalTrials.gov eligibility criteria and checked on a sample basis. Please confirm against the original criteria.

Who might be able to join this trial:

Core Phase (first 48 weeks):

• Boys aged 2 years old up to (but not including) 6 years old at the time of screening
• A parent or legal guardian must provide written consent, and the child must provide written agreement if required by local rules
• People who have been confirmed through genetic testing to have Duchenne Muscular Dystrophy (DMD)
• People who are either taking a stable dose of steroid tablets (with no significant dose changes for at least 3 months before starting the study drug), or people not currently on steroids who agree not to start steroids during the first 48 weeks of the study

Extension Phase (continuing beyond 48 weeks):

• People who completed the full Core Phase (48 weeks) and attended the final Core Phase visit
• A parent or legal guardian must provide written consent again, and the child must provide written agreement if required by local rules
• People who are on a stable steroid tablet dose with no significant changes, or — for those who were not on steroids during the Core Phase — steroids may be started based on the treating doctor's judgement

Who may not be able to join:

Core Phase:

• People who have taken another experimental drug within the 3 months before starting the study drug
• People who have used a dystrophin restoration treatment (such as Ataluren or exon-skipping therapies) within the 6 months before starting the study drug
• People who have received gene therapy (such as AAV micro-dystrophin delivery) within the 12 months before starting the study drug
• People who have used any medication other than steroids that could affect muscle strength or function within the 3 months before starting the study drug (vitamins D, calcium, and general supplements are allowed)
• People who have had surgery affecting muscle strength or function within the 3 months before starting the study drug, or who have surgery planned at any point during the study
• People with other significant health conditions that, in the treating doctor's opinion, could affect safety, make it unlikely they would complete the study, or affect the study results
• People with uncontrolled neurological diseases or significant uncontrolled health conditions unrelated to DMD, based on the treating doctor's judgement
• People whose blood test results show platelet count, white blood cell count, or haemoglobin below the normal range at screening (borderline results may be retested once)
• People with current or past liver disease or liver problems, including abnormally high bilirubin levels in the blood (unless due to a condition called Gilbert disease)
• People with poor kidney function, measured by a specific blood protein called Cystatin C being more than twice the upper normal limit (borderline results may be retested once)
• People with very high fasting triglyceride (blood fat) levels above 300 mg/dL at screening (borderline results may be retested once)
• People who test positive for hepatitis B, hepatitis C, or HIV at screening
• People with a specific heart electrical reading (QTcF interval) above 450 milliseconds, or with certain heart rhythm risk factors such as heart failure, low potassium, or a family history of a condition called Long QT Syndrome
• People with psychiatric illness or social circumstances that, in the treating doctor's opinion, would prevent them from understanding or completing the required muscle function tests and study procedures
• People with a known allergy or sensitivity to any ingredient in the study drug
• People with sorbitol intolerance, sorbitol malabsorption, or a hereditary form of fructose intolerance
• People who weigh less than 10 kg at screening

Extension Phase:

• People whose blood test results show platelet count, white blood cell count, or haemoglobin below the normal range at the end of the Core Phase (borderline results may be retested once)
• People with current liver disease or liver problems, including abnormally high bilirubin levels (unless due to Gilbert disease)
• People with poor kidney function based on Cystatin C blood levels more than twice the upper normal limit (borderline results may be retested once)
• People with very high fasting triglyceride levels above 300 mg/dL (borderline results may be retested once)
• People with other significant health conditions that, in the treating doctor's opinion, could affect safety, make it unlikely treatment would be completed, or affect the study results
• People with psychiatric illness or social circumstances that, in the treating doctor's opinion, would prevent them from understanding or completing the required muscle function tests and study procedures

Important: Always verify eligibility with the trial site directly before applying.