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PoC Study to Assess Activity and Safety of SMT C1100 (Ezutromid) in Boys With DMD (PhaseOut DMD)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT02858362
Recruitment Status : Completed
First Posted : August 8, 2016
Last Update Posted : May 6, 2019
Information provided by (Responsible Party):
Summit Therapeutics

Brief Summary:
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)

Condition or disease Intervention/treatment Phase
Duchenne Muscular Dystrophy Drug: ezutromid Phase 2

Detailed Description:

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.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 40 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 2 Clinical Study to Assess the Activity and Safety of Utrophin Modulation With SMT C1100 in Ambulatory Paediatric Male Subjects With Duchenne Muscular Dystrophy
Actual Study Start Date : June 2016
Actual Primary Completion Date : April 11, 2018
Actual Study Completion Date : August 27, 2018

Arm Intervention/treatment
Experimental: Open Label Treatment Arm
SMTC1100 (ezutromid) oral suspension
Drug: ezutromid
oral suspension
Other Name: SMTC1100

Primary Outcome Measures :
  1. Change in MRI leg muscle parameters [ Time Frame: Baseline, Weeks 12, 24, 36 and 48 ]
  2. SMTC1100 (ezutromid) plasma concentrations [ Time Frame: Pre-dose and post-dose at Weeks 1, 4, 8, 12, 24, 36 and 48 ]

Secondary Outcome Measures :
  1. Change in utrophin membrane staining via quantifiable imaging of immunostained biopsy sections [ Time Frame: Baseline and at either Week 24 or 48 ]
  2. 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 ]
  3. Treatment emergent adverse events (AEs) and safety laboratory abnormalities [ Time Frame: Through study completion of 48 weeks ]

Information from the National Library of Medicine

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Ages Eligible for Study:   5 Years to 10 Years   (Child)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Male
  • 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

Exclusion Criteria:

  • 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)

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT02858362

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United States, California
UCLA-David Geffen School of Medicine
Los Angeles, California, United States
United States, Colorado
Children's Hosptial of Colorado
Aurora, Colorado, United States
United States, Florida
Nemours Children's Clinic
Orlando, Florida, United States
United States, Massachusetts
Boston Children's Hospital
Boston, Massachusetts, United States
United States, Ohio
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
United States, Oregon
Oregon Health and Science University
Portland, Oregon, United States
United States, Pennsylvania
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
United States, Tennessee
Vanderbilt University Medical Center
Nashville, Tennessee, United States
United States, Utah
University of Utah Hospital and Clinics
Salt Lake City, Utah, United States
United Kingdom
Heart of England NHS Foundation Trust - Heartlands Hospital
Birmingham, United Kingdom
Bristol Children's Hospital
Bristol, United Kingdom
Addenbrooke's Hospital
Cambridge, United Kingdom
Alder Hey Children's NHS Foundation Trust
Liverpool, United Kingdom
Great Ormond Street Hospital for Children NHS Foundation Trust
London, United Kingdom
Royal Manchester Children's Hospital - Central Manchester University Hospitals NHS Foundation Trust
Manchester, United Kingdom
The Freeman Hospital, Newcastle Upon Tyne Hospitals
Newcastle, United Kingdom
Sponsors and Collaborators
Summit Therapeutics
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Study Director: Medical Monitor Summit (Oxford) Limited

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Responsible Party: Summit Therapeutics Identifier: NCT02858362     History of Changes
Other Study ID Numbers: SMTC11005
2015-004333-27 ( EudraCT Number )
First Posted: August 8, 2016    Key Record Dates
Last Update Posted: May 6, 2019
Last Verified: May 2019
Keywords provided by Summit Therapeutics:
Muscular Dystrophy
PhaseOut DMD
Additional relevant MeSH terms:
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Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked