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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT03039686




Registration number
NCT03039686
Ethics application status
Date submitted
27/01/2017
Date registered
1/02/2017
Date last updated
21/12/2020

Titles & IDs
Public title
Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy
Scientific title
A Randomized, Double Blind, Placebo-Controlled, Study to Assess the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy
Secondary ID [1] 0 0
2016-001654-18
Secondary ID [2] 0 0
CN001-016
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Duchenne Muscular Dystrophy 0 0
Condition category
Condition code
Musculoskeletal 0 0 0 0
Other muscular and skeletal disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Neurological 0 0 0 0
Other neurological disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - RO7239361
Treatment: Drugs - Placebo for RO7239361

Experimental: RO7239361 Low Dose - Participants received low dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received low dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.

Experimental: RO7239361 High Dose - Participants received high dose RO7239361 SC on specified days of the 48-week DB period. Following the DB period participants received high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.

Placebo comparator: Placebo - Participants received matching placebo solution subcutaneously (SC) on specified days of the 48-week double-blind (DB) period. Following the DB period participants received low dose or high dose RO7239361 on specified days for up to 192 weeks during the open-label period followed by 24 weeks of follow-up.


Treatment: Drugs: RO7239361
Take RO7239361 subcutaneously on specified days over a 48 week blinded period

Treatment: Drugs: Placebo for RO7239361
Take placebo subcutaneously on specified days over a 48 week blinded period
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Baseline for the North Star Ambulatory Assessment (NSAA) Total Score
Timepoint [1] 0 0
Baseline
Primary outcome [2] 0 0
Change From Baseline in the North Star Ambulatory Assessment (NSAA) Total Score at Week 48
Timepoint [2] 0 0
Baseline, Week 48
Secondary outcome [1] 0 0
Baseline Time for 4 Stair Climb
Timepoint [1] 0 0
Baseline
Secondary outcome [2] 0 0
Change From Baseline at Week 48 in 4 Stair Climb Velocity (4SCV)
Timepoint [2] 0 0
Baseline, Week 48
Secondary outcome [3] 0 0
Baseline for the Time to Stand From Supine
Timepoint [3] 0 0
Baseline
Secondary outcome [4] 0 0
Change From Baseline at Week 48 in Stand From Supine Velocity
Timepoint [4] 0 0
Baseline, Week 48
Secondary outcome [5] 0 0
Baseline Time for 10 Meter Walk/Run
Timepoint [5] 0 0
Baseline
Secondary outcome [6] 0 0
Change From Baseline at Week 48 in 10 M Walk/Run Velocity
Timepoint [6] 0 0
Baseline, Week 48
Secondary outcome [7] 0 0
Baseline for the Pediatric Outcome Data Collection Instrument (PODCI) Transfer and Basic Mobility Subscale
Timepoint [7] 0 0
Baseline
Secondary outcome [8] 0 0
Change From Baseline at Week 48 in Pediatric Outcome Data Collection Instrument (PODCI) Transfer and Basic Mobility Subscale
Timepoint [8] 0 0
Baseline, Week 48
Secondary outcome [9] 0 0
Change From Baseline at Week 48 in Proximal Lower Extremity Flexor Strength
Timepoint [9] 0 0
Baseline, Week 48
Secondary outcome [10] 0 0
Baseline for the 6 Minute Walk Distance (6MWD)
Timepoint [10] 0 0
Baseline
Secondary outcome [11] 0 0
Change From Baseline at Week 48 in 6 Minute Walk Distance (6MWD)
Timepoint [11] 0 0
Baseline, Week 48
Secondary outcome [12] 0 0
Percentage of Participants for Each Clinical Global Impression of Change (CGI-C) Assessment Status at Week 48
Timepoint [12] 0 0
Baseline, Week 48
Secondary outcome [13] 0 0
Change From Baseline at Week 48 in 95th Percentile Stride Velocity
Timepoint [13] 0 0
Baseline, Week 48
Secondary outcome [14] 0 0
Number of Participants With Adverse Events (AEs)
Timepoint [14] 0 0
During DB period (48 weeks) and Whole study (up to approximately 34 months)
Secondary outcome [15] 0 0
Number of Participants With AEs Leading to Discontinuation
Timepoint [15] 0 0
During DB period (48 weeks) and Whole study (up to approximately 34 months)

Eligibility
Key inclusion criteria
* Diagnosed with DMD by confirmed medical history and genetic testing
* Able to walk without assistance
* Minimum North Star Ambulatory Assessment score of 15 at screening
* Able to walk up 4 stairs in 8 seconds or less
* Weigh at least 15 kg (33 lbs)
* Taking corticosteroids for DMD
Minimum age
6 Years
Maximum age
11 Years
Sex
Males
Can healthy volunteers participate?
No
Key exclusion criteria
* Any behavior or mental issue that will affect the ability to complete the required study procedures
* Previously or currently taking medications like androgens or human growth hormone
* Use of a ventilator during the day
* Unable to have blood samples collected or receive an injection under the skin
* Concomitant or previous participation at any time in a gene therapy study

Other protocol defined Inclusion/Exclusion Criteria could apply.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
6/07/2017
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
28/04/2020
Sample size
Target
Accrual to date
Final
166
Recruitment in Australia
Recruitment state(s)
NSW,QLD,VIC
Recruitment hospital [1] 0 0
Children's Hospital Westmead; Paediatrics & Child Health - Westmead
Recruitment hospital [2] 0 0
Lady Cilento Children's Hospital; Neurosciences Department - South Brisbane
Recruitment hospital [3] 0 0
Royal Children's Hospital - Parkville
Recruitment postcode(s) [1] 0 0
2145 - Westmead
Recruitment postcode(s) [2] 0 0
4101 - South Brisbane
Recruitment postcode(s) [3] 0 0
3052 - Parkville
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Connecticut
Country [4] 0 0
United States of America
State/province [4] 0 0
Florida
Country [5] 0 0
United States of America
State/province [5] 0 0
Georgia
Country [6] 0 0
United States of America
State/province [6] 0 0
Illinois
Country [7] 0 0
United States of America
State/province [7] 0 0
Iowa
Country [8] 0 0
United States of America
State/province [8] 0 0
Kansas
Country [9] 0 0
United States of America
State/province [9] 0 0
Maryland
Country [10] 0 0
United States of America
State/province [10] 0 0
Massachusetts
Country [11] 0 0
United States of America
State/province [11] 0 0
Missouri
Country [12] 0 0
United States of America
State/province [12] 0 0
Nevada
Country [13] 0 0
United States of America
State/province [13] 0 0
Ohio
Country [14] 0 0
United States of America
State/province [14] 0 0
Washington
Country [15] 0 0
Argentina
State/province [15] 0 0
Buenos Aires
Country [16] 0 0
Belgium
State/province [16] 0 0
Gent
Country [17] 0 0
Canada
State/province [17] 0 0
Ontario
Country [18] 0 0
France
State/province [18] 0 0
Lyon
Country [19] 0 0
France
State/province [19] 0 0
Nantes
Country [20] 0 0
France
State/province [20] 0 0
Paris Cedex 12
Country [21] 0 0
France
State/province [21] 0 0
Strasbourg
Country [22] 0 0
Germany
State/province [22] 0 0
Essen
Country [23] 0 0
Italy
State/province [23] 0 0
Emilia-Romagna
Country [24] 0 0
Italy
State/province [24] 0 0
Lazio
Country [25] 0 0
Italy
State/province [25] 0 0
Sicilia
Country [26] 0 0
Japan
State/province [26] 0 0
Hyogo
Country [27] 0 0
Japan
State/province [27] 0 0
Miyagi
Country [28] 0 0
Japan
State/province [28] 0 0
Nagano
Country [29] 0 0
Japan
State/province [29] 0 0
Osaka
Country [30] 0 0
Japan
State/province [30] 0 0
Tokyo
Country [31] 0 0
Netherlands
State/province [31] 0 0
Leiden
Country [32] 0 0
Netherlands
State/province [32] 0 0
Nijmegen
Country [33] 0 0
Spain
State/province [33] 0 0
Barcelona
Country [34] 0 0
Spain
State/province [34] 0 0
Valencia
Country [35] 0 0
Sweden
State/province [35] 0 0
Goeteborg
Country [36] 0 0
United Kingdom
State/province [36] 0 0
Liverpool
Country [37] 0 0
United Kingdom
State/province [37] 0 0
London

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Hoffmann-La Roche
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Clinical Trials
Address 0 0
Hoffmann-La Roche
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

No information has been provided regarding IPD availability


What supporting documents are/will be available?




Results publications and other study-related documents

No documents have been uploaded by study researchers.

Results are available at https://clinicaltrials.gov/study/NCT03039686