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PoC Study to Assess Activity and Safety of SMT C1100 (Ezutromid) in Boys With DMD
study id #: NCT02858362
condition: Duchenne Muscular Dystrophy
A Phase 2 Clinical Study to Assess the Activity and Safety of Utrophin Modulation with SMT C1100 (ezutromid) in Ambulatory Paediatric Male Subjects with Duchenne Muscular Dystrophy (DMD)
mechanism of action: Utophin modulator to promote utophin production
last updated: May 15, 2019
start date: June 2016
estimated completion: August 27, 2018
phase of development: Phase 2
size / enrollment: 40
This is a Phase 2, open-label, study to assess the activity and safety of utrophin modulation with SMT C1100 (ezutromid) administered orally bid in ambulatory paediatric male subjects with DMD. Approximately 40 subjects with DMD will be enrolled in this study.
This study will be conducted in a multi-centre setting in both the United Kingdom and the United States of America and comprises of a Screening and Baseline Phase of up to 28 days, a 48-week open label Treatment Phase, and either a 30-day Safety Follow up Phase or an optional extension phase where study treatment is provided until discontinuation of the program or regulatory approvals as applicable.
- Change in MRI leg muscle parameters [Time Frame: Baseline, Weeks 12, 24, 36 and 48]
- SMTC1100 (ezutromid) plasma concentrations [Time Frame: Pre-dose and post-dose at Weeks 1, 4, 8, 12, 24, 36 and 48]
- Change in utrophin membrane staining via quantifiable imaging of immunostained biopsy sections [Time Frame: Baseline and at either Week 24 or 48]
- Change in muscle regenerating fibres by measuring via muscle biopsy a combination of fibre size and neonatal myosin positivity [Time Frame: Baseline and at either Week 24 or 48]
- Treatment emergent adverse events (AEs) and safety laboratory abnormalities [Time Frame: Through study completion of 48 weeks]
• Age >=5 and >=10 years (from 5th birthday to 10th birthday)
• DMD diagnosis
• Willing and able to comply with study procedures, including 2 muscle biopsy procedures
• Able to undergo MRI
• Have used at least 6 months stable dose systemic corticosteroids
• Ability to walk 300 metres unassisted and below 80% predicted 6MWD
• Uncontrolled congestive heart failure (CHF) or recent change in CHF prophylaxis/treatment
• Use of beta blockers, herbal supplements, BCRP substrates, SNRIs, SSRIs, tricyclic antidepressants, or ADHD treatments such as methylphenidate or PEA.
• Use of over the counter, herbal or prescription CYP2B6, CYP1A1 or CYP1A2 inhibitors, inducers or substrates.
• Exposure to other investigational drug or DMD interventional agent within 3 months (except FOR-DMD Study participants are permitted)
• Require daytime ventilator assistance
• Be dairy or lactose intolerant
• Be a smoker, use other tobacco or nicotine products or be exposed to daily passive smoking
• Use of an approved DMD medication or anticipate use during the study (other than steroids)
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