Duchenne Muscular DystrophyPhase 2NCT03985878
A Study to Evaluate Safety, Tolerability, and Efficacy of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) Who Have Completed Study 4658-102 (NCT03218995)
A Phase 2 study testing Eteplirsen in people with Duchenne Muscular Dystrophy, run by Sarepta Therapeutics, Inc. at 4 sites in 4 countries.
Trial summary
The purpose of this extension study is to evaluate the ongoing safety and tolerability of additional treatment with eteplirsen administered once weekly by intravenous (IV) infusion in male participants with DMD who have successfully completed the 96-week eteplirsen Study 4658-102. Read more about this study
- Who is it for?
- Ages 2 to 5 years, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy
- What is being tested?
- Eteplirsen
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 4 sitesin 4 countries
- Study dates
- 26 Jun 2019to 31 Aug 2022
- Phase
- Phase 2
- 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 2 to 5 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- Participant successfully completes 96 weeks of treatment in Study 4658-102.
Who cannot join
- Participant has a prior or ongoing medical condition that, in the Investigator's opinion, could adversely affect the safety of the participant, or make it unlikely that the course of treatment or follow-up would be completed, or impair the assessment of study results.
- Other inclusion/exclusion criteria apply.
Trial locations and contacts
4 sites in 4 countries.
4 sites in 4 countries
Italy1 site
Fondazione Policlinico Universitario Agostino Gemelli, UOC Neuropsichiatria Infantile
Rome, 00168
United Kingdom1 site
UCL Great Ormond Street Institute of Child Health, Dubowitz Neuromuscular Centre
London, England, WC1N 1EH
About this study
Official title: An Open-Label Safety, Tolerability, and Efficacy Study of Eteplirsen in Patients With Duchenne Muscular Dystrophy Who Have Completed Study 4658-102
The purpose of this extension study is to evaluate the ongoing safety and tolerability of additional treatment with eteplirsen administered once weekly by intravenous (IV) infusion in male participants with DMD who have successfully completed the 96-week eteplirsen Study 4658-102.
- Conditions
- Duchenne Muscular Dystrophy
- Being tested
- Eteplirsen
- Keywords
- DMD, Duchenne, Eteplirsen, Dystrophy, Dystrophin, Exon Skipping, Exon 51, Ambulatory, Pediatric
- 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.
EteplirsenDrug
Also called AVI-4658, EXONDYS 51, EXONDYS
Eteplirsen IV infusion once weekly.
| Study group | Role | What they receive |
|---|---|---|
| Eteplirsen | Experimental | Participants will receive eteplirsen via IV infusions, once weekly, for up to 284 weeks. Receives: Drug: Eteplirsen |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants Experiencing Adverse Events (AEs)
A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' section.
Measured: Up to 162 weeks
Number of Participants Experiencing Death Due to Adverse Events
A summary of all deaths regardless of causality is located in the 'Reported Adverse Events' section.
Measured: Up to 162 weeks
Number of Participants Experiencing Adverse Events of Special Interest (AESIs)
AESIs were defined as any AE that was of scientific and medical concern specific to study treatment, for which ongoing and rapid communication by the Investigator to the sponsor was appropriate. AESIs included findings potentially indicative of hepatic and renal abnormalities, hypersensitivity, and thrombocytopenia. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' section.
Measured: Up to 162 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Jun 2019
- Study start26 Jun 2019
- Primary completion31 Aug 2022
- Study completion31 Aug 2022
Why it stopped: Participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment. There are no safety concerns with Eteplirsen.
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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 15 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
- NCT03985878
- Sponsor's protocol ID
- 4658-102-OLE
- Other IDs
- 2019-000337-39, 2019
- Status last verified
- Jul 2023
- First submitted
- 11 Jun 2019
- Last updated
- 18 Aug 2023
- 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 18 Aug 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label Safety, Tolerability, and Efficacy Study of Eteplirsen in Patients With Duchenne Muscular Dystrophy Who Have Completed Study 4658-102
- ClinicalTrials.gov ID
- NCT03985878
- Acronym / sponsor ID
- 4658-102-OLE
- Conditions
- Duchenne Muscular Dystrophy
- Keywords
- DMD, Duchenne, Eteplirsen, Dystrophy, Dystrophin, Exon Skipping, Exon 51, Ambulatory
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment. There are no safety concerns with Eteplirsen.
- Enrolment
- 15 participants (actual)
- Lead sponsor
- Sarepta Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- 26 Jun 2019 (actual)
- Primary completion
- 31 Aug 2022 (actual)
- Study completion
- 31 Aug 2022 (actual)
- First posted
- 14 Jun 2019 (actual)
- Results
- Posted 18 Aug 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this extension study is to evaluate the ongoing safety and tolerability of additional treatment with eteplirsen administered once weekly by intravenous (IV) infusion in male participants with DMD who have successfully completed the 96-week eteplirsen Study 4658-102.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Eteplirsen | Experimental | Drug: EteplirsenParticipants will receive eteplirsen via IV infusions, once weekly, for up to 284 weeks. |
Who may qualify
- Ages
- 2 to 5 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (1 listed)
- Participant successfully completes 96 weeks of treatment in Study 4658-102.
Key exclusion criteria (2 listed)
- Participant has a prior or ongoing medical condition that, in the Investigator's opinion, could adversely affect the safety of the participant, or make it unlikely that the course of treatment or follow-up would be completed, or impair the assessment of study results.
- Other inclusion/exclusion criteria apply.
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 |
|---|---|---|
| EteplirsenAlso: AVI-4658, EXONDYS 51, EXONDYSEteplirsen IV infusion once weekly. | Drug | Eteplirsen |
Primary outcome
Number of Participants Experiencing Adverse Events (AEs)
Time frame: Up to 162 weeks
A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' section.
Number of Participants Experiencing Death Due to Adverse Events
Time frame: Up to 162 weeks
A summary of all deaths regardless of causality is located in the 'Reported Adverse Events' section.
Number of Participants Experiencing Adverse Events of Special Interest (AESIs)
Time frame: Up to 162 weeks
AESIs were defined as any AE that was of scientific and medical concern specific to study treatment, for which ongoing and rapid communication by the Investigator to the sponsor was appropriate. AESIs included findings potentially indicative of hepatic and renal abnormalities, hypersensitivity, and thrombocytopenia. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' section.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Aug 2023.
- 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.
4 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Eteplirsen in people with Duchenne Muscular Dystrophy, run by Sarepta Therapeutics, Inc. at 4 sites in 4 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 1 inclusion and 2 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 18 Aug 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in Belgium, France, Italy, United Kingdom. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 18 Aug 2023). 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 Experiencing Adverse Events (AEs) (time frame: Up to 162 weeks). The study lists 3 primary outcomes in all. 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 18 Aug 2023.