Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy

November 25, 2020 updated by: Hoffmann-La Roche

A Randomized, Double Blind, Placebo-Controlled, Study to Assess the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy

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).

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
166

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Argentina
      • Buenos Aires, Argentina, C1204AAD
        • Instituto Centenario
  • Australia
    • New South Wales
      • Westmead, New South Wales, Australia, 2145
        • Children's Hospital Westmead; Paediatrics & Child Health
    • Queensland
      • South Brisbane, Queensland, Australia, 4101
        • Lady Cilento Children's Hospital; Neurosciences Department
    • Victoria
      • Parkville, Victoria, Australia, 3052
        • Royal Children's Hospital
  • Belgium
      • Gent, Belgium, 9000
        • UZ Gent
  • Canada
    • Ontario
      • London, Ontario, Canada, N6A 5W9
        • London Health Sciences Centre; Children's Hospital; Pediatrics
      • Ottawa, Ontario, Canada, K1H 8L1
        • Children's Hospital of Eastern Ontario
  • France
      • Lyon, France, 69004
        • Hospices Civils de Lyon
      • Nantes, France, 44093
        • Hotel Dieu; Service Pharmacie Essais Cliniques
      • Paris Cedex 12, France, 75571
        • Hopital Armand Trousseau; centre reference Maladies Neuro-musculaires Est parisien Neuropediatrie
      • Strasbourg, France, 67091
        • Hopitaux Universitaires de Strasbourg
  • Germany
      • Essen, Germany, 45122
        • Universitatsklinikum Essen; Innere Klinik
  • Italy
    • Emilia-Romagna
      • Milano, Emilia-Romagna, Italy, 20162
        • Fondazione Serena Onlus - Centro Clinico Nemo
    • Lazio
      • Rome, Lazio, Italy, 00168
        • Fondazione Policlinico Universitario A Gemelli; Servizio di Farmacia
    • Sicilia
      • Messina, Sicilia, Italy, 98125
        • Az. Osp. Universitaria Pol. G. Martino; Dip. Neuroscienze, Scienze Psichiatriche e Anest.
  • Japan
      • Hyogo, Japan, 663-8501
        • Hyogo College of Medicine Hospital
      • Miyagi, Japan, 989-3126
        • Miyagi Children's Hospital
      • Nagano, Japan, 390-8621
        • Shinshu University Hospital
      • Osaka, Japan, 560-8552
        • National Hospital Organization Osaka Toneyama Medical Center
      • Tokyo, Japan, 187-8551
        • National Center of Neurology and Psychiatry
  • Netherlands
      • Leiden, Netherlands, 2333 ZA
        • Leids Universitair Medisch Centrum
      • Nijmegen, Netherlands, 6525 EX
        • Radboud University Nijmegen Medical Centre; Ophthalmology
  • Spain
      • Valencia, Spain, 46026
        • Hospital Universitari i Politecnic La Fe de Valencia; Servicio de Farmacia
    • Barcelona
      • Esplugues De Llobregas, Barcelona, Spain, 08950
        • Hospital Sant Joan de Déu
  • Sweden
      • Goeteborg, Sweden, 41685
        • Drottning Silvias Barn- och ungdomssjukhus; Kliniken för barnmedicin
  • United Kingdom
      • Liverpool, United Kingdom, L12 2AP
        • Alder Hey Children s Hospital; Department of Pediatrics
      • London, United Kingdom, WC1N 1EH
        • UCL Institute of Child Health & Great Ormond Street Hospital for Children
  • United States
    • Arizona
      • Phoenix, Arizona, United States, 85028
        • Neuromuscular Research Center
    • California
      • Palo Alto, California, United States, 94304
        • Stanford University
      • Sacramento, California, United States, 95817
        • University of California Davis Medical Center
    • Connecticut
      • New Haven, Connecticut, United States, 06510
        • Yale University School of Medicine ; Pulmonary & Critical Care
    • Florida
      • Gainesville, Florida, United States, 32607
        • University of Florida
      • Orlando, Florida, United States, 32827
        • Nemours Children's Hospital
    • Georgia
      • Atlanta, Georgia, United States, 30318
        • Rare Disease Research, LLC
    • Illinois
      • Chicago, Illinois, United States, 60612
        • Rush University Medical Center - PPDS
    • Iowa
      • Iowa City, Iowa, United States, 52242
        • University of Iowa
    • Kansas
      • Kansas City, Kansas, United States, 66160
        • University of Kansas Medical Center
    • Maryland
      • Baltimore, Maryland, United States, 21205
        • Kennedy Krieger Institute
    • Massachusetts
      • Worcester, Massachusetts, United States, 01655
        • University of Massachusetts Memorial Childrens Medical Center; Department of Neurology
    • Missouri
      • Saint Louis, Missouri, United States, 63110
        • Saint Louis Children's Hospital
    • Nevada
      • Las Vegas, Nevada, United States, 89145
        • Las Vegas Clinic
    • Ohio
      • Cincinnati, Ohio, United States, 45229
        • Cincinnati Childrens Hospital Medical Center;Investigational Pharmacy
      • Columbus, Ohio, United States, 43205
        • Nationwide Childrens Hospital; Research Institute at Nationwide Childrens Hospital
    • Washington
      • Seattle, Washington, United States, 98105
        • Seattle Children's Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

6 years to 11 years (Child)

Accepts Healthy Volunteers

No

Genders Eligible for Study

Male

Description

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

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
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.
Drug: RO7239361
Take RO7239361 subcutaneously on specified days over a 48 week blinded period
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.
Drug: RO7239361
Take RO7239361 subcutaneously on specified days over a 48 week blinded period
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.
Drug: Placebo for RO7239361
Take placebo subcutaneously on specified days over a 48 week blinded period

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Baseline for the North Star Ambulatory Assessment (NSAA) Total Score
Time Frame: Baseline
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.
Baseline
Change From Baseline in the North Star Ambulatory Assessment (NSAA) Total Score at Week 48
Time Frame: Baseline, Week 48
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).
Baseline, Week 48

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Baseline Time for 4 Stair Climb
Time Frame: Baseline
The time to complete the 4 stair climb was measured at baseline.
Baseline
Change From Baseline at Week 48 in 4 Stair Climb Velocity (4SCV)
Time Frame: Baseline, Week 48
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).
Baseline, Week 48
Baseline for the Time to Stand From Supine
Time Frame: Baseline
The time required for a participant to stand from supine position. A longer time reflects a worse outcome.
Baseline
Change From Baseline at Week 48 in Stand From Supine Velocity
Time Frame: Baseline, Week 48
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).
Baseline, Week 48
Baseline Time for 10 Meter Walk/Run
Time Frame: Baseline
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.
Baseline
Change From Baseline at Week 48 in 10 M Walk/Run Velocity
Time Frame: Baseline, Week 48
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).
Baseline, Week 48
Baseline for the Pediatric Outcome Data Collection Instrument (PODCI) Transfer and Basic Mobility Subscale
Time Frame: Baseline
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.
Baseline
Change From Baseline at Week 48 in Pediatric Outcome Data Collection Instrument (PODCI) Transfer and Basic Mobility Subscale
Time Frame: Baseline, Week 48
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).
Baseline, Week 48
Change From Baseline at Week 48 in Proximal Lower Extremity Flexor Strength
Time Frame: Baseline, Week 48
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.
Baseline, Week 48
Baseline for the 6 Minute Walk Distance (6MWD)
Time Frame: Baseline
The 6MWD measured the distance a participant was able to traverse while walking for 6 minutes. A longer distance reflects a better outcome.
Baseline
Change From Baseline at Week 48 in 6 Minute Walk Distance (6MWD)
Time Frame: Baseline, Week 48
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).
Baseline, Week 48
Percentage of Participants for Each Clinical Global Impression of Change (CGI-C) Assessment Status at Week 48
Time Frame: Baseline, Week 48
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.
Baseline, Week 48
Change From Baseline at Week 48 in 95th Percentile Stride Velocity
Time Frame: Baseline, Week 48
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.
Baseline, Week 48
Number of Participants With Adverse Events (AEs)
Time Frame: During DB period (48 weeks) and Whole study (up to approximately 34 months)
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.
During DB period (48 weeks) and Whole study (up to approximately 34 months)
Number of Participants With AEs Leading to Discontinuation
Time Frame: During DB period (48 weeks) and Whole study (up to approximately 34 months)
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.
During DB period (48 weeks) and Whole study (up to approximately 34 months)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Hoffmann-La Roche

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
July 6, 2017

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
April 28, 2020

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
April 28, 2020

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
January 27, 2017

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
January 31, 2017

First Posted (Estimate)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
February 1, 2017

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
December 21, 2020

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
November 25, 2020

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
November 1, 2020

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • CN001-016
  • 2016-001654-18 (EudraCT Number)
  • WN40227 (Other Identifier: Hoffman-La Roche)

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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