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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/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
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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
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Secondary ID [1]
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2016-001654-18
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Secondary ID [2]
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CN001-016
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Duchenne Muscular Dystrophy
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Condition category
Condition code
Musculoskeletal
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Other muscular and skeletal disorders
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Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Neurological
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Other neurological disorders
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Intervention/exposure
Study type
Interventional
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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
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Baseline for the North Star Ambulatory Assessment (NSAA) Total Score
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Assessment method [1]
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The NSAA is a functional scale specifically designed for ambulant boys with Duchenne muscular dystrophy (DMD) that can provide information about motor function. The NSAA is a 17-item test of standing, ability to transition from lying to sitting, sitting to standing, and other mobility assessments. Each of the 17 items is evaluated on an ordinal scale of 0-2: 0 = unable to achieve independently, 1 = modified method but achieves goal independent of physical assistance from another, or 2 = normal with no obvious modification of activity. Total score range is 0 to 34. Higher scores reflect better performance.
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Timepoint [1]
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Baseline
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Primary outcome [2]
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Change From Baseline in the North Star Ambulatory Assessment (NSAA) Total Score at Week 48
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Assessment method [2]
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The NSAA is a functional scale specifically designed for ambulant boys with Duchenne muscular dystrophy (DMD) that can provide information about motor function. The NSAA is a 17-item test of standing, ability to transition from lying to sitting, sitting to standing, and other mobility assessments. Each of the 17 items is evaluated on an ordinal scale of 0-2: 0 = unable to achieve independently, 1 = modified method but achieves goal independent of physical assistance from another, or 2 = normal with no obvious modification of activity. Total score range is 0 to 34. Higher scores reflect better performance. A positive change from baseline indicates an improvement. Based on the mixed-effect model of repeated measures (MMRM).
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Timepoint [2]
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Baseline, Week 48
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Secondary outcome [1]
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Baseline Time for 4 Stair Climb
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Assessment method [1]
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The time to complete the 4 stair climb was measured at baseline.
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Timepoint [1]
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Baseline
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Secondary outcome [2]
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Change From Baseline at Week 48 in 4 Stair Climb Velocity (4SCV)
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Assessment method [2]
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4SCV was calculated as the ratio of the number of stairs climbed (4) divided by the number of seconds taken to complete the 4-stair climb. The results were converted into velocity (distance/time). A positive change from baseline indicates an improvement. Based on the mixed-effect model of repeated measures (MMRM).
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Timepoint [2]
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Baseline, Week 48
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Secondary outcome [3]
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Baseline for the Time to Stand From Supine
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Assessment method [3]
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The time required for a participant to stand from supine position. A longer time reflects a worse outcome.
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Timepoint [3]
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Baseline
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Secondary outcome [4]
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Change From Baseline at Week 48 in Stand From Supine Velocity
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Assessment method [4]
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The time required for a participant to stand from supine position. A longer time reflects a worse outcome. A negative change from baseline indicates an improvement. Based on the mixed-effect model of repeated measures (MMRM).
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Timepoint [4]
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Baseline, Week 48
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Secondary outcome [5]
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Baseline Time for 10 Meter Walk/Run
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Assessment method [5]
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The time required for a participant to run or walk a distance of 10 meters as quickly as possible. A longer time reflects a worse outcome.
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Timepoint [5]
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Baseline
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Secondary outcome [6]
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Change From Baseline at Week 48 in 10 M Walk/Run Velocity
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Assessment method [6]
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The time required for a participant to run or walk a distance of 10 meters as quickly as possible calculated as velocity (distance/time). A positive change from baseline indicates an improvement. Based on the mixed-effect model of repeated measures (MMRM).
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Timepoint [6]
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Baseline, Week 48
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Secondary outcome [7]
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Baseline for the Pediatric Outcome Data Collection Instrument (PODCI) Transfer and Basic Mobility Subscale
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Assessment method [7]
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The PODCI is designed to be completed by the parent/guardian of a child who has knowledge of the child's conditions. The Transfer and Basic Mobility scale is one of the subscales of the PODCI. The results are standardized into a scale of 0-100 with a higher score reflecting better performance.
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Timepoint [7]
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Baseline
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Secondary outcome [8]
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Change From Baseline at Week 48 in Pediatric Outcome Data Collection Instrument (PODCI) Transfer and Basic Mobility Subscale
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Assessment method [8]
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The PODCI is designed to be completed by the parent/guardian of a child who has knowledge of the child's conditions. The Transfer and Basic Mobility scale is one of the subscales of the PODCI. The results are standardized into a scale of 0-100 with a higher score reflecting better performance. A positive change from baseline indicates an improvement. Based on the mixed-effect model of repeated measures (MMRM).
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Timepoint [8]
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Baseline, Week 48
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Secondary outcome [9]
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Change From Baseline at Week 48 in Proximal Lower Extremity Flexor Strength
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Assessment method [9]
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Proximal lower extremity flexor (knee extension and knee flexion) strength was measured using manual myometry. A higher score reflects a better outcome. A positive change from baseline indicates an improvement.
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Timepoint [9]
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Baseline, Week 48
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Secondary outcome [10]
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Baseline for the 6 Minute Walk Distance (6MWD)
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Assessment method [10]
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The 6MWD measured the distance a participant was able to traverse while walking for 6 minutes. A longer distance reflects a better outcome.
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Timepoint [10]
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Baseline
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Secondary outcome [11]
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Change From Baseline at Week 48 in 6 Minute Walk Distance (6MWD)
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Assessment method [11]
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The 6MWD measured the distance a participant was able to traverse while walking for 6 minutes. A longer distance reflects a better outcome. A positive change from baseline indicates an improvement. Based on the mixed-effect model of repeated measures (MMRM).
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Timepoint [11]
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Baseline, Week 48
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Secondary outcome [12]
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Percentage of Participants for Each Clinical Global Impression of Change (CGI-C) Assessment Status at Week 48
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Assessment method [12]
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The CGI-C was used to assess the participant's overall condition on a 7-point scale, using the status markers "very much improved, much improved, slightly improved, no change, slightly worse, much worse or very much worse" at Week 48 as compared to baseline.
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Timepoint [12]
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Baseline, Week 48
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Secondary outcome [13]
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Change From Baseline at Week 48 in 95th Percentile Stride Velocity
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Assessment method [13]
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Stride velocity was recorded with the ActiMyo device in a subset of the overall study population. The ActiMyo device measures the daily movement and activity levels of the participant. The device consists of two sensors worn on each ankle. A higher velocity reflects a better outcome. A positive change from baseline indicates an improvement.
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Timepoint [13]
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Baseline, Week 48
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Secondary outcome [14]
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Number of Participants With Adverse Events (AEs)
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Assessment method [14]
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An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product and which does not necessarily have to have a causal relationship with the treatment. An adverse event can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a pharmaceutical product, whether or not considered related to the pharmaceutical product. Preexisting conditions which worsen during a study are also considered as adverse events.
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Timepoint [14]
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During DB period (48 weeks) and Whole study (up to approximately 34 months)
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Secondary outcome [15]
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Number of Participants With AEs Leading to Discontinuation
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Assessment method [15]
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An adverse event is any untoward medical occurrence in a subject administered a pharmaceutical product and which does not necessarily have to have a causal relationship with the treatment. An adverse event can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a pharmaceutical product, whether or not considered related to the pharmaceutical product. Preexisting conditions which worsen during a study are also considered as adverse events. Reported here is the number of participants with AEs that led to study discontinuation.
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Timepoint [15]
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During DB period (48 weeks) and Whole study (up to approximately 34 months)
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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
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Minimum age
6
Years
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Maximum age
11
Years
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Sex
Males
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Can healthy volunteers participate?
No
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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.
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Blinded (masking used)
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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
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 2/Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Completed
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
6/07/2017
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
28/04/2020
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Sample size
Target
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Accrual to date
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Final
166
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Recruitment in Australia
Recruitment state(s)
NSW,QLD,VIC
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Recruitment hospital [1]
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Children's Hospital Westmead; Paediatrics & Child Health - Westmead
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Recruitment hospital [2]
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Lady Cilento Children's Hospital; Neurosciences Department - South Brisbane
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Recruitment hospital [3]
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Royal Children's Hospital - Parkville
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Recruitment postcode(s) [1]
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2145 - Westmead
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Recruitment postcode(s) [2]
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4101 - South Brisbane
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Recruitment postcode(s) [3]
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3052 - Parkville
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Recruitment outside Australia
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United States of America
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Arizona
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United States of America
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California
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United States of America
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Connecticut
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Florida
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United States of America
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Georgia
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United States of America
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Illinois
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Country [7]
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United States of America
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Iowa
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United States of America
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Kansas
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Maryland
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Massachusetts
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Missouri
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Nevada
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Ohio
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United States of America
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Washington
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Country [15]
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Argentina
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State/province [15]
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Buenos Aires
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Belgium
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Gent
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Canada
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Ontario
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France
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Lyon
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France
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Nantes
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France
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Paris Cedex 12
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France
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Strasbourg
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Germany
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Essen
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Italy
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Emilia-Romagna
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Italy
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Lazio
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Italy
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Sicilia
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Japan
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Hyogo
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Japan
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Miyagi
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Japan
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Nagano
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Japan
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Osaka
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Japan
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Tokyo
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Netherlands
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Leiden
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Netherlands
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Nijmegen
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Spain
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Barcelona
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Spain
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Valencia
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Sweden
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Goeteborg
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United Kingdom
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Liverpool
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United Kingdom
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London
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
Hoffmann-La Roche
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Address
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Ethics approval
Ethics application status
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Summary
Brief summary
This is a multi-center, randomized, double-blind, placebo-controlled study to assess the
efficacy, safety and tolerability of two different weekly doses of RO7239361 in ambulatory
boys with Duchenne Muscular Dystrophy (DMD).
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Trial website
https://clinicaltrials.gov/ct2/show/NCT03039686
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Clinical Trials
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Address
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Hoffmann-La Roche
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Email
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Contact person for public queries
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT03039686
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