Duchenne Muscular DystrophyPhase 2/3NCT03039686
Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy
A Phase 2/3 study testing RO7239361 in people with Duchenne Muscular Dystrophy, run by Hoffmann-La Roche at 44 sites in 13 countries.
Trial 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). Read more about this study
- Who is it for?
- Ages 6 to 11 years, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy
- What is being tested?
- RO7239361
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 166 participants
- Where
- 44 sitesin 13 countries
- Study dates
- 6 Jul 2017to 28 Apr 2020
- Phase
- Phase 2/3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 to 11 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- 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
Who cannot join
- 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.
Trial locations and contacts
44 sites in 13 countries.
44 sites in 13 countries
United States17 sites
Rare Disease Research, LLC
Atlanta, Georgia, 30318
Kennedy Krieger Institute
Baltimore, Maryland, 21205
Rush University Medical Center - PPDS
Chicago, Illinois, 60612
Cincinnati Childrens Hospital Medical Center;Investigational Pharmacy
Cincinnati, Ohio, 45229
Nationwide Childrens Hospital; Research Institute at Nationwide Childrens Hospital
Columbus, Ohio, 43205
University of Florida
Gainesville, Florida, 32607
University of Iowa
Iowa City, Iowa, 52242
University of Kansas Medical Center
Kansas City, Kansas, 66160
Las Vegas Clinic
Las Vegas, Nevada, 89145
Yale University School of Medicine ; Pulmonary & Critical Care
New Haven, Connecticut, 06510
Nemours Children's Hospital
Orlando, Florida, 32827
Stanford University
Palo Alto, California, 94304
Neuromuscular Research Center
Phoenix, Arizona, 85028
University of California Davis Medical Center
Sacramento, California, 95817
Seattle Children's Hospital
Seattle, Washington, 98105
Saint Louis Children's Hospital
St Louis, Missouri, 63110
University of Massachusetts Memorial Childrens Medical Center; Department of Neurology
Worcester, Massachusetts, 01655
Japan5 sites
Hyogo College of Medicine Hospital
Hyōgo, 663-8501
Miyagi Children's Hospital
Miyagi, 989-3126
Shinshu University Hospital
Nagano, 390-8621
National Hospital Organization Osaka Toneyama Medical Center
Osaka, 560-8552
National Center of Neurology and Psychiatry
Tokyo, 187-8551
France4 sites
Hospices Civils de Lyon
Lyon, 69004
Hotel Dieu; Service Pharmacie Essais Cliniques
Nantes, 44093
Hopital Armand Trousseau; centre reference Maladies Neuro-musculaires Est parisien Neuropediatrie
Paris, 75571
Hopitaux Universitaires de Strasbourg
Strasbourg, 67091
Australia3 sites
Royal Children's Hospital
Parkville, Victoria, 3052
Lady Cilento Children's Hospital; Neurosciences Department
South Brisbane, Queensland, 4101
Children's Hospital Westmead; Paediatrics & Child Health
Westmead, New South Wales, 2145
Italy3 sites
Az. Osp. Universitaria Pol. G. Martino; Dip. Neuroscienze, Scienze Psichiatriche e Anest.
Messina, Sicily, 98125
Fondazione Serena Onlus - CENTRO CLINICO NEMO
Milano, Emilia-Romagna, 20162
Fondazione Policlinico Universitario A Gemelli; Servizio di Farmacia
Rome, Lazio, 00168
Canada2 sites
London Health Sciences Centre; Children's Hospital; Pediatrics
London, Ontario, N6A 5W9
Children'S Hospital of Eastern Ontario
Ottawa, Ontario, K1H 8L1
Netherlands2 sites
Leids Universitair Medisch Centrum
Leiden, 2333 ZA
Radboud University Nijmegen Medical Centre; Ophthalmology
Nijmegen, 6525 EX
Spain2 sites
Hospital Sant Joan De Deu
Esplugues de Llobregas, Barcelona, 08950
Hospital Universitari i Politecnic La Fe de Valencia; Servicio de Farmacia
Valencia, 46026
United Kingdom2 sites
Alder Hey Children s Hospital; Department of Pediatrics
Liverpool, L12 2AP
UCL Institute of Child Health & Great Ormond Street Hospital for Children
London, WC1N 1EH
Sweden1 site
Drottning Silvias Barn- och ungdomssjukhus; Kliniken for barnmedicin
Gothenburg, 41685
About this study
Official title: 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).
- Conditions
- Duchenne Muscular Dystrophy
- Being tested
- RO7239361, Placebo for RO7239361
- Keywords
- muscular dystrophy, Duchenne's Muscular Dystrophy, DMD
- Medical subject headings
- Muscular Dystrophy, Duchenne, Muscular Dystrophies
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RO7239361Drug
Take RO7239361 subcutaneously on specified days over a 48 week blinded period
Placebo for RO7239361Drug
Take placebo subcutaneously on specified days over a 48 week blinded period
| Study group | Role | What they receive |
|---|---|---|
| RO7239361 Low Dose | Experimental | 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. Receives: Drug: RO7239361 |
| RO7239361 High Dose | Experimental | 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. Receives: Drug: RO7239361 |
| Placebo | Placebo comparator | 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. Receives: Drug: Placebo for RO7239361 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Baseline for the North Star Ambulatory Assessment (NSAA) Total Score
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.
Measured: Baseline
Change From Baseline in the North Star Ambulatory Assessment (NSAA) Total Score at 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).
Measured: Baseline, Week 48
Secondary outcomes (15)
Baseline Time for 4 Stair Climb
The time to complete the 4 stair climb was measured at baseline.
Measured: Baseline
Change From Baseline at Week 48 in 4 Stair Climb Velocity (4SCV)
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).
Measured: Baseline, Week 48
Baseline for the Time to Stand From Supine
The time required for a participant to stand from supine position. A longer time reflects a worse outcome.
Measured: Baseline
Change From Baseline at Week 48 in Stand From Supine Velocity
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).
Measured: Baseline, Week 48
Baseline Time for 10 Meter Walk/Run
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.
Measured: Baseline
Change From Baseline at Week 48 in 10 M Walk/Run Velocity
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).
Measured: Baseline, Week 48
Baseline for the Pediatric Outcome Data Collection Instrument (PODCI) Transfer and Basic Mobility Subscale
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.
Measured: Baseline
Change From Baseline at Week 48 in Pediatric Outcome Data Collection Instrument (PODCI) Transfer and Basic Mobility Subscale
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).
Measured: Baseline, Week 48
Change From Baseline at Week 48 in Proximal Lower Extremity Flexor Strength
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.
Measured: Baseline, Week 48
Baseline for the 6 Minute Walk Distance (6MWD)
The 6MWD measured the distance a participant was able to traverse while walking for 6 minutes. A longer distance reflects a better outcome.
Measured: Baseline
Change From Baseline at Week 48 in 6 Minute Walk Distance (6MWD)
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).
Measured: Baseline, Week 48
Percentage of Participants for Each Clinical Global Impression of Change (CGI-C) Assessment Status at 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.
Measured: Baseline, Week 48
Change From Baseline at Week 48 in 95th Percentile Stride Velocity
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.
Measured: Baseline, Week 48
Number of Participants With Adverse Events (AEs)
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.
Measured: During DB period (48 weeks) and Whole study (up to approximately 34 months)
Number of Participants With AEs Leading to Discontinuation
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.
Measured: During DB period (48 weeks) and Whole study (up to approximately 34 months)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Feb 2017 (estimated)
- Study start6 Jul 2017
- Primary completion28 Apr 2020
- Study completion28 Apr 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Duchenne Muscular DystrophyStudies of the same condition, open and under way.
- More trials run by Hoffmann-La RocheThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 166 participants (actual)
Sponsor and investigators
- Lead sponsor
- Hoffmann-La Roche
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Trials, Study director, Hoffmann-La Roche
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03039686
- Sponsor's protocol ID
- CN001-016
- Other IDs
- 2016-001654-18, WN40227, 2016
- Status last verified
- Nov 2020
- First submitted
- 27 Jan 2017
- Last updated
- 21 Dec 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Muntoni F, Byrne BJ, McMillan HJ, Ryan MM, Wong BL, Dukart J, Bansal A, Cosson V, Dreghici R, Guridi M, Rabbia M, Staunton H, Tirucherai GS, Yen K, Yuan X, Wagner KR; Taldefgrobep Alfa Study Group. The Clinical Development of Taldefgrobep Alfa: An Anti-Myostatin Adnectin for the Treatment of Duchenne Muscular Dystrophy. Neurol Ther. 2024 Feb;13(1):183-219. doi: 10.1007/s40120-023-00570-w. Epub 2024 Jan 8.
PubMed 38190001
Study at a glance
From the registry record, last updated by the sponsor on 21 Dec 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double Blind, Placebo-Controlled, Study to Assess the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy
- ClinicalTrials.gov ID
- NCT03039686
- Acronym / sponsor ID
- CN001-016
- Conditions
- Duchenne Muscular Dystrophy
- Keywords
- muscular dystrophy, Duchenne's Muscular Dystrophy, DMD
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 166 participants (actual)
- Lead sponsor
- Hoffmann-La RocheIndustry (pharma, biotech, devices)
- Study start
- 6 Jul 2017 (actual)
- Primary completion
- 28 Apr 2020 (actual)
- Study completion
- 28 Apr 2020 (actual)
- First posted
- 1 Feb 2017 (estimated)
- Results
- Posted 21 Dec 2020
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| RO7239361 Low Dose | Experimental | Drug: RO7239361Participants 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. |
| RO7239361 High Dose | Experimental | Drug: RO7239361Participants 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 | Placebo comparator | Drug: Placebo for RO7239361Participants 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. |
Who may qualify
- Ages
- 6 to 11 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- 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)
Key exclusion criteria (6 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| RO7239361Take RO7239361 subcutaneously on specified days over a 48 week blinded period | Drug | RO7239361 High Dose, RO7239361 Low Dose |
| Placebo for RO7239361Take placebo subcutaneously on specified days over a 48 week blinded period | Drug | Placebo |
Primary outcome
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.
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).
15 secondary outcomes
Baseline Time for 4 Stair Climb
Time frame: Baseline
Change From Baseline at Week 48 in 4 Stair Climb Velocity (4SCV)
Time frame: Baseline, Week 48
Baseline for the Time to Stand From Supine
Time frame: Baseline
Change From Baseline at Week 48 in Stand From Supine Velocity
Time frame: Baseline, Week 48
Baseline Time for 10 Meter Walk/Run
Time frame: Baseline
Change From Baseline at Week 48 in 10 M Walk/Run Velocity
Time frame: Baseline, Week 48
Baseline for the Pediatric Outcome Data Collection Instrument (PODCI) Transfer and Basic Mobility Subscale
Time frame: Baseline
Change From Baseline at Week 48 in Pediatric Outcome Data Collection Instrument (PODCI) Transfer and Basic Mobility Subscale
Time frame: Baseline, Week 48
Change From Baseline at Week 48 in Proximal Lower Extremity Flexor Strength
Time frame: Baseline, Week 48
Baseline for the 6 Minute Walk Distance (6MWD)
Time frame: Baseline
Change From Baseline at Week 48 in 6 Minute Walk Distance (6MWD)
Time frame: 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
Change From Baseline at Week 48 in 95th Percentile Stride Velocity
Time frame: 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)
Number of Participants With AEs Leading to Discontinuation
Time frame: During DB period (48 weeks) and Whole study (up to approximately 34 months)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Dec 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 38190001Muntoni F, Byrne BJ, McMillan HJ, Ryan MM, Wong BL, Dukart J, Bansal A, Cosson V, Dreghici R, Guridi M, Rabbia M, Staunton H, Tirucherai GS, Yen K, Yuan X, Wagner KR; Taldefgrobep Alfa Study Group. The Clinical Development of Taldefgrobep Alfa: An Anti-Myostatin Adnectin for the Treatment of Duchenne Muscular Dystrophy. Neurol Ther. 2024 Feb;13(1):183-219. doi: 10.1007/s40120-023-00570-w. Epub 2024 Jan 8.
- FDA Safety Alerts and RecallsLink provided by the sponsor.
- Provides information about the Roche clinical trial NCT03039686 and molecule being investigated in DuchenneLink provided by the sponsor.
Related trials for Duchenne Muscular Dystrophy
RecruitingEarly Phase 1
Safety and Dystrophin Expression of SPOT-03 in Duchenne Muscular Dystrophy (DMD) Patients
Duchenne Muscular Dystrophy (DMD)
RecruitingPhase 2
Safety and Efficacy of KER-065 in Participants With Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy
Not yet recruitingPhase 2
A Phase 2 Trial to Investigate the Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Tegacorat in Male Participants 4 to <7 Years of Age With Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy (DMD)
Not yet recruitingObservational study
Multimodal Study About Absence of Dp140 in Becker Muscular Dystrophy: Brain-muscle Imaging, Cognition and Muscle Function
Becker Muscular Dystrophy
Enrolling by invitationObservational study
A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice
Duchenne Muscular Dystrophy
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Clinical Trials study director
Hoffmann-La Roche
44 sites in 13 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing RO7239361 in people with Duchenne Muscular Dystrophy, run by Hoffmann-La Roche at 44 sites in 13 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 21 Dec 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 44 sites in United States, Japan, France, Australia, Italy and 8 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 21 Dec 2020). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Baseline for the North Star Ambulatory Assessment (NSAA) Total Score (time frame: Baseline). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03039686, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 21 Dec 2020.