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

This study is currently recruiting participants. (see Contacts and Locations)
Verified February 2017 by Summit Therapeutics
Sponsor:
Information provided by (Responsible Party):
Summit Therapeutics
ClinicalTrials.gov Identifier:
NCT02858362
First received: July 27, 2016
Last updated: February 22, 2017
Last verified: February 2017
  Purpose
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 Intervention Phase
Duchenne Muscular Dystrophy
Drug: ezutromid
Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: No masking
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

Resource links provided by NLM:


Further study details as provided by Summit Therapeutics:

Primary Outcome Measures:
  • 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 ]

Secondary Outcome Measures:
  • 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 ]

Estimated Enrollment: 40
Study Start Date: June 2016
Estimated Primary Completion Date: December 2017 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Open Label Treatment Arm
SMTC1100 (ezutromid) oral suspension
Drug: ezutromid
oral suspension
Other Name: SMTC1100

Detailed Description:

This is a Phase 2, open-label, study to assess the activity and safety of utrophin modulation with SMT C1100 (ezutromid) 2500 mg 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 a 30-day Safety Follow up Phase.

  Eligibility

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

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)
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02858362

Contacts
Contact: Michele Cioffi 617-401-2473 clinicaltrials@summitplc.com

Locations
United States, California
UCLA-David Geffen School of Medicine Recruiting
Los Angeles, California, United States
Contact: Michael Bonitati    310-825-3264    mbonitati@mednet.ucla.edu   
Principal Investigator: Perry Shieh, M.D.         
United States, Colorado
Children's Hosptial of Colorado Recruiting
Aurora, Colorado, United States
Contact: Hannah Johnson    720-777-3293    hannah.johnson@childrenscolorado.org   
Principal Investigator: Michele Yang, MD         
United States, Florida
Nemours Children's Clinic Recruiting
Orlando, Florida, United States
Contact: Ginny Rizzo, MS    407-650-7403    virginia.rizzo@nemours.org   
Principal Investigator: Richard Finkel, MD         
United States, Massachusetts
Boston Children's Hospital Recruiting
Boston, Massachusetts, United States
Contact: Timothy Harrington    857-218-4677    timothy.harrington@childrens.harvard.edu   
Contact: Grace Ordonez    617-919-7384    grace.ordonez@childrens.harvard.edu   
Principal Investigator: Basil Darras, MD         
United States, Ohio
Cincinnati Children's Hospital Medical Center Recruiting
Cincinnati, Ohio, United States
Principal Investigator: John Jefferies, MD         
United States, Oregon
Oregon Health and Science University Recruiting
Portland, Oregon, United States
Contact: Bryn McCarthy, RN    503-418-8297    mccarbry@ohsu.edu   
Principal Investigator: Erika Finanger, MD         
United States, Pennsylvania
Children's Hospital of Philadelphia Recruiting
Philadelphia, Pennsylvania, United States
Contact: Joshua Zigmont, RN, BSN    267-426-7161    zigmontj@email.chop.edu   
Principal Investigator: John Brandsema, MD         
United States, Tennessee
Vanderbilt University Medical Center Recruiting
Nashville, Tennessee, United States
Contact: Diana Davis, RN    615-322-8957    diana.davis@vanderbilt.edu   
Principal Investigator: W. Bryan Burnette, MD         
United States, Utah
University of Utah Hospital and Clinics Recruiting
Salt Lake City, Utah, United States
Contact: Bryant Gordon    801-585-5052    bgordon@genetics.utah.edu   
Principal Investigator: Russell Butterfield, MD         
United Kingdom
Heart of England NHS Foundation Trust - Heartlands Hospital Enrolling by invitation
Birmingham, United Kingdom
Bristol Children's Hospital Enrolling by invitation
Bristol, United Kingdom
Addenbrooke's Hospital Active, not recruiting
Cambridge, United Kingdom
Alder Hey Children's NHS Foundation Trust Enrolling by invitation
Liverpool, United Kingdom
Great Ormond Street Hospital for Children NHS Foundation Trust Active, not recruiting
London, United Kingdom
Royal Manchester Children's Hospital - Central Manchester University Hospitals NHS Foundation Trust Enrolling by invitation
Manchester, United Kingdom
The Freeman Hospital, Newcastle Upon Tyne Hospitals Enrolling by invitation
Newcastle, United Kingdom
Sponsors and Collaborators
Summit Therapeutics
Investigators
Study Director: Medical Monitor Summit (Oxford) Limited
  More Information

Responsible Party: Summit Therapeutics
ClinicalTrials.gov Identifier: NCT02858362     History of Changes
Other Study ID Numbers: SMTC11005
2015-004333-27 ( EudraCT Number )
Study First Received: July 27, 2016
Last Updated: February 22, 2017

Keywords provided by Summit Therapeutics:
DMD
Muscular Dystrophy
Utrophin
Duchenne
PhaseOut DMD

Additional relevant MeSH terms:
Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked

ClinicalTrials.gov processed this record on March 28, 2017