Duchenne Muscular DystrophyPhase 3NCT03532542
An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular Dystrophy
A Phase 3 study testing Casimersen and Golodirsen in people with Duchenne Muscular Dystrophy, run by Sarepta Therapeutics, Inc. at 50 sites in 13 countries.
Trial summary
The main objective of this study is to evaluate the safety and tolerability of long-term treatment with casimersen or golodirsen in patients with Duchenne muscular dystrophy (DMD). Read more about this study
- Who is it for?
- Ages 7 to 23 years, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy
- What is being tested?
- Casimersen and Golodirsen
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 171 participants
- Where
- 50 sitesin 13 countries
- Study dates
- 2 Aug 2018to 26 Jul 2023
- Phase
- Phase 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 7 to 23 years
- Men only
- No healthy volunteers
- Child, Adult
Who may be able to join
- Completed a clinical trial evaluating casimersen or golodirsen, per protocol.
- Is between 7 and 23 years of age, inclusive, at enrollment.
- Other inclusion/exclusion criteria apply.
Who cannot join
None listed.
Trial locations and contacts
50 sites in 13 countries.
50 sites in 13 countries
United States25 sites
Rare Disease Research, LLC
Atlanta, Georgia, 30318
Kennedy Krieger Institute
Baltimore, Maryland, 21205
Boston Children's Hospital
Boston, Massachusetts, 02115
Ann and Robert H Lurie Childrens Hospital of Chicago
Chicago, Illinois, 60611
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Abigail Wexner Research Institute at Nationwide Children's Hospital
Columbus, Ohio, 43205
Children's Medical Center Dallas
Dallas, Texas, 75207
UF Health: University of Florida Clinical Research Center
Gainesville, Florida, 32610
NW Florida Clinical Research Group, LLC
Gulf Breeze, Florida, 32561
Texas Children's Hospital
Houston, Texas, 77030
University of Iowa Childrens Hospital
Iowa City, Iowa, 52242
University of Kansas Medical Center
Kansas City, Kansas, 66160
Las Vegas Clinic
Las Vegas, Nevada, 89145
Children's Hospital Los Angeles
Los Angeles, California, 90027
University of California Los Angeles
Los Angeles, California, 90095
Children's Hospital of Wisconsin, Corporate Center Suite 540
Milwaukee, Wisconsin, 53226
Children's Hospital of The King's Daughters
Norfolk, Virginia, 23510
Stanford Neuroscience Health Center
Palo Alto, California, 94304
Neuromuscular Research Center
Phoenix, Arizona, 85028
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, 15224
Shriners Hospital for Children
Portland, Oregon, 97239
University of Rochester Medical Center - Department of Neurology
Rochester, New York, 14642
University of Utah - PPDS
Salt Lake City, Utah, 84132
Rady Children's Hospital- San Diego
San Diego, California, 92123
St. Louis Children's Hospital
St Louis, Missouri, 63110
United Kingdom4 sites
Leeds General Infirmary, Leeds Teaching Hospitals NHS Trust
Leeds, LS1 3EX
Alder Hey Children's NHS Foundation Trust
Liverpool, L12 2AP
Great Ormond Street Hospital (GOSH)
London, WC1N 1EH
Royal Victoria Infirmary
Newcastle upon Tyne, NE1 4LP
Italy3 sites
Azienda Ospedaliero-Universitaria di Ferrara - Arcispedale Sant' Anna
Cona, 44124
UOSD Centro Traslazionale di Miologia e Patologie Neurogenerative
Genoa, Liguria, 16147
Fondazione Policlinico Universitario A Gemelli
Milan, 20123
Spain3 sites
Hospital de La Santa Creu i Sant Pau
Barcelona, Catalonia, 08041
Hospital Sant Joan de Deu
Barcelona, 8950
Hospital Universitari i Politecnic La Fe de Valencia
Valencia, 46026
Canada2 sites
Alberta Children's Hospital
Calgary, Alberta, T3B6A8
Children's Hospital of Eastern Ontario
Ottawa, Ontario, K1H 8L1
Czechia2 sites
Fakultni nemocnice Brno, Klinika detske neurologie LF MU a FN Brno
Brno, 613 00
Fakultni nemocnice v Motole
Prague, 150 06
France2 sites
Hotel Dieu- CHU Nantes
Nantes, 44093
Hôpital Armand Trousseau
Paris, 75013
Germany2 sites
Universitatsklinikum Essen
Essen, North Rhine-Westphalia, 45147
Universitatsklinikum Freiberg
Freiburg im Breisgau, Baden-Wurttemberg, 70106
Poland2 sites
Uniwersyteckie Centrum Kliniczne, Klinica Neurologii Rozwojowej
Gdansk, 80-952
Samodzielny Publiczny Centralny Szpital Kliniczny
Warsaw, Masovian Voivodeship, 02-097
Bulgaria1 site
University Multiprofile Hospital for active treatment Aleksandrovska EAD Clinic of Neurological Diseases
Sofia, 1431
Israel1 site
Schneider Children's Medical Center of Israel
Petah Tikva, 4920235
Sweden1 site
Provningsenhet Barn, Drottning Silvias Och Ungdomssjukhus
Gothenburg, 41685
About this study
Official title: Long-term, Open-label Extension Study for Patients With Duchenne Muscular Dystrophy Enrolled in Clinical Trials Evaluating Casimersen or Golodirsen
The main objective of this study is to evaluate the safety and tolerability of long-term treatment with casimersen or golodirsen in patients with Duchenne muscular dystrophy (DMD).
- Conditions
- Duchenne Muscular Dystrophy
- Being tested
- Casimersen, Golodirsen
- Keywords
- Duchenne muscular dystrophy, Exon Skipping, DMD, Exon 53, Exon 45, Duchenne
- Medical subject headings
- Muscular Dystrophy, Duchenne
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CasimersenDrug
Also called SRP-4045, AMONDYS 45
Casimersen solution for IV infusion
GolodirsenDrug
Also called SRP-4053, VYONDYS 53
Golodirsen solution for IV infusion
| Study group | Role | What they receive |
|---|---|---|
| Casimersen | Experimental | Patients amenable to exon 45 skipping who have completed a clinical trial evaluating casimersen will receive open-label casimersen intravenous (IV) infusions, weekly, at 30 mg/kg for up to 144 Weeks. Receives: Drug: Casimersen |
| Golodirsen | Experimental | Patients amenable to exon 53 skipping who have completed a clinical trial evaluating golodirsen will receive open-label golodirsen intravenous (IV) infusions, weekly, at 30 mg/kg for up to 144 Weeks. Receives: Drug: Golodirsen |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Treatment-emergent Serious Adverse Events (TESAEs)
A treatment-emergent adverse event (TEAE) was any untoward medical occurrence in a clinical study participant that did not necessarily have a causal relationship with the study drug. A TEAE could, therefore, be any unfavorable and unintended symptom, sign, disease, condition, or test abnormality that occurred during or after administration of the study drug, whether or not considered related to the study drug. A TESAE was any TEAE that resulted in any of the following outcomes: death, a life-threatening event, required or prolonged inpatient hospitalization, persistent or significant disability/incapacity, or an important medical event (that is, may have jeopardized the participant and may have required medical or surgical intervention to prevent one of the previously mentioned outcomes). A summary of serious and all other non-serious TEAEs regardless of causality is located in the Reported Adverse Events module.
Measured: Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 May 2018
- Study start2 Aug 2018
- Primary completion26 Jul 2023
- Study completion26 Jul 2023
Why it stopped: The trial was stopped to reduce the clinical trial burden on participants while ensuring continued treatment via a post-trial access program with commercially available casimersen and golodirsen. Study was not terminated due to safety concerns.
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.
- Other trials for Duchenne Muscular DystrophyStudies of the same condition, open and under way.
- More trials run by Sarepta Therapeutics, Inc.The 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 3
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 171 participants (actual)
Sponsor and investigators
- Lead sponsor
- Sarepta Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Director, Study director, Sarepta Therapeutics, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03532542
- Sponsor's protocol ID
- 4045-302
- Other IDs
- 2017-004625-32, 2017
- Status last verified
- Sep 2024
- First submitted
- 10 May 2018
- Last updated
- 19 Sep 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 19 Sep 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Long-term, Open-label Extension Study for Patients With Duchenne Muscular Dystrophy Enrolled in Clinical Trials Evaluating Casimersen or Golodirsen
- ClinicalTrials.gov ID
- NCT03532542
- Acronym / sponsor ID
- 4045-302
- Conditions
- Duchenne Muscular Dystrophy
- Keywords
- Duchenne muscular dystrophy, Exon Skipping, DMD, Exon 53, Exon 45, Duchenne
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: The trial was stopped to reduce the clinical trial burden on participants while ensuring continued treatment via a post-trial access program with commercially available casimersen and golodirsen. Study was not terminated due to safety concerns.
- Enrolment
- 171 participants (actual)
- Lead sponsor
- Sarepta Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- 2 Aug 2018 (actual)
- Primary completion
- 26 Jul 2023 (actual)
- Study completion
- 26 Jul 2023 (actual)
- First posted
- 22 May 2018 (actual)
- Results
- Posted 19 Sep 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The main objective of this study is to evaluate the safety and tolerability of long-term treatment with casimersen or golodirsen in patients with Duchenne muscular dystrophy (DMD).
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Casimersen | Experimental | Drug: CasimersenPatients amenable to exon 45 skipping who have completed a clinical trial evaluating casimersen will receive open-label casimersen intravenous (IV) infusions, weekly, at 30 mg/kg for up to 144 Weeks. |
| Golodirsen | Experimental | Drug: GolodirsenPatients amenable to exon 53 skipping who have completed a clinical trial evaluating golodirsen will receive open-label golodirsen intravenous (IV) infusions, weekly, at 30 mg/kg for up to 144 Weeks. |
Who may qualify
- Ages
- 7 to 23 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (3 listed)
- Completed a clinical trial evaluating casimersen or golodirsen, per protocol.
- Is between 7 and 23 years of age, inclusive, at enrollment.
- Other inclusion/exclusion criteria apply.
Key exclusion criteria (0 listed)
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 |
|---|---|---|
| CasimersenAlso: SRP-4045, AMONDYS 45Casimersen solution for IV infusion | Drug | Casimersen |
| GolodirsenAlso: SRP-4053, VYONDYS 53Golodirsen solution for IV infusion | Drug | Golodirsen |
Primary outcome
Number of Participants With Treatment-emergent Serious Adverse Events (TESAEs)
Time frame: Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
A treatment-emergent adverse event (TEAE) was any untoward medical occurrence in a clinical study participant that did not necessarily have a causal relationship with the study drug. A TEAE could, therefore, be any unfavorable and unintended symptom, sign, disease, condition, or test abnormality that occurred during or after administration of the study drug, whether or not considered related to the study drug. A TESAE was any TEAE that resulted in any of the following outcomes: death, a life-threatening event, required or prolonged inpatient hospitalization, persistent or significant disability/incapacity, or an important medical event (that is, may have jeopardized the participant and may have required medical or surgical intervention to prevent one of the previously mentioned outcomes). A summary of serious and all other non-serious TEAEs regardless of causality is located in the Reported Adverse Events module.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Sep 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
Medical Director study director
Sarepta Therapeutics, Inc.
50 sites in 13 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Casimersen and Golodirsen in people with Duchenne Muscular Dystrophy, run by Sarepta Therapeutics, Inc. at 50 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 3 inclusion and 0 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: stopped early, as of the sponsor's last update on 19 Sep 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 50 sites in United States, United Kingdom, Italy, Spain, Belgium 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. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
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 19 Sep 2024). 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?
Number of Participants With Treatment-emergent Serious Adverse Events (TESAEs) (time frame: Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 19 Sep 2024.