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 (C11005) - PoC Study to Assess Activity and Safety of SMT C1100 in Boys with DMD

Summit (Oxford) Limited0 sites43 target enrollmentNovember 4, 2015

Overview

Phase: Phase 1
Intervention: Not specified
Conditions: Not specified
Sponsor: Summit (Oxford) Limited
Enrollment: 43
Status: Active, not recruiting
Last Updated: 7 years ago

Overview Investigators Eligibility Criteria Outcomes Similar Trials

Overview

Brief Summary

No summary available.

Registry

who.int

Start Date

November 4, 2015

End Date

September 11, 2018

Last Updated

7 years ago

Study Type

Interventional clinical trial of medicinal product

Investigators

Sponsor

Summit (Oxford) Limited

Eligibility Criteria

Inclusion Criteria

•Patients will be required to satisfy the following criteria at the screening visit:
•1\. Be able to provide written informed consent/assent as per local requirements.
•2\. Be male.
•3\.Be aged \=5 years \<10 years of age (from 5th birthday to 10th birthday)\-not applicable for cohort 3
•4\. Have phenotypic evidence of dystrophinopathy based on the onset of characteristic clinical symptoms or signs (e.g., proximal muscle weakness, waddling gait, and Gowers' manoeuvre), an elevated serum creatinine kinase level, and ongoing difficulty with walking.
•5\. Have prior confirmation of the DMD diagnosis through:Documentation of the presence of a mutation in the dystrophin gene as determined by gene sequencing from a laboratory certified by the College of American Pathologists, the Clinical Laboratory Improvement Act/Amendment or an equivalent organization. Or Documentation of the absence of dystrophin in the muscle (via biopsy).
•6\. Be willing and able to comply with two muscle biopsy procedures (not applicable for cohort 3\)
•7\. Be able to undergo MRI examination.
•8\. Patients must have used stable systemic corticosteroids (prednisone, prednisolone or deflazacort) for a minimum of 6 months immediately prior to the start of the Treatment Phase , with no significant change in dosage or dosing regimen (not related to body weight change) and a reasonable expectation that dosage and dosing regimen will not change significantly for the duration of the study.
•9\. Have the ability to walk at least 300 meters unassisted during the screening 6MWD and be below the protocol\-specified threshold for 80%\-predicted 6MWD (not applicable for cohort 3\)

Exclusion Criteria

•Patients will be excluded from the study if they satisfy the following
•criteria at the screening visit
•1\. Have physical exam findings that in the investigator’s opinion should be exclusionary e.g., lower limb injury that may affect 6MWD performance.
•2\. Have any change (initiation, change in type of drug, dose modification, schedule modification, interruption, discontinuation or reinitiation) in prophylaxis/treatment for congestive heart failure (CHF) within 3 months prior to the start of study treatment.
•3\. Have uncontrolled clinical symptoms and signs of CHF (American College of Cardiology/American Heart Association Stage C or Stage D).
•4\.Have abnormal GLDH at baseline (\>1\.5 x ULN)
•5\.Have abnormal coagulation times at baseline (\>1\.5 x ULN)
•6\. Have an abnormal ECG e.g., a QTcF \>500ms, left bundle\-branch block or any other major conduction defect.
•7\. Use beta blockers (however, if during the course of the study they are clinically indicated they can be initiated; not applicable for cohort 3 )
•8\. Use herbal supplements and be unwilling to stop these for the duration of the study.