Muscular Dystrophy, DuchennePhase 2NCT02286947
Safety Study of Eteplirsen to Treat Advanced Stage Duchenne Muscular Dystrophy
A Phase 2 study testing Eteplirsen in people with Muscular Dystrophy, Duchenne, run by Sarepta Therapeutics, Inc. at 9 sites in United States.
Trial summary
The primary objective of this study is to explore safety and tolerability of eteplirsen in participants with advanced stage Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping. Read more about this study
- Who is it for?
- Ages 7 to 21 years, men only
- Conditions the study is for
- Muscular Dystrophy, Duchenne
- 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
- 24 participants
- Where
- 9 sitesUnited States
- Study dates
- Nov 2014to 23 Mar 2018
- 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 7 to 21 years
- Men only
- No healthy volunteers
- Child, Adult
Who may be able to join
- Male 7 - 21 years of age
- Diagnosis of DMD with a mutation that is amenable to exon 51 skipping, confirmed by a genetic report
- Stable dose of oral corticosteroids for at least 24 weeks or has not received corticosteroids for at least 24 weeks
- Non-ambulatory, or incapable of walking ≥300 meters on the 6-Minute Walk Test (6MWT).
- Score of ≤4 on the Brooke Score for Arms and Shoulders.
- Stable cardiac and pulmonary function
- Use of contraceptives for sexually active males throughout the study
- Willing to provide consent and comply with the study
Who cannot join
- Use of any pharmacologic treatment (other than corticosteroids) within 12 weeks that may have an effect on muscle strength or function (e.g., growth hormone, anabolic steroids).
- Previous treatment with SMT C1100/BMN 195 at any time.
- Previous treatment with drisapersen (PRO051) within the last 6 months.
- Participation in any other DMD interventional clinical study within 12 weeks
- Major change in physiotherapy regimen within the past 3 months
- Major surgery within 3 months
Show all 12Show fewer
- Presence of other clinically significant illness
- Use of an aminoglycoside antibiotic within 12 weeks or the need for this antibiotic or statin during study
- Forced vital capacity % predicted [FVC % predicted] <40%, or requiring daytime ventilation.
- Require antiarrhythmic and/or antidiuretic therapy for heart failure.
- Have a left ventricular ejection fraction (LVEF) of <40%.
- Prior or ongoing medical condition that could adversely affect the safety of the patient, make it unlikely that the course of treatment would be completed, or impair the assessment of study results.
Trial locations and contacts
9 sites in 1 country.
9 sites in 1 country
United States9 sites
Kennedy Krieger Institute
Baltimore, Maryland, 21205
Massachusetts General Hospital
Boston, Massachusetts, 02114
Nationwide Children's Hospital
Columbus, Ohio, 43205
University of Iowa Children's Hospital
Iowa City, Iowa, 52242
Ronald Reagan UCLA Medical Center
Los Angeles, California, 90095
University of Rochester Medical Center
Rochester, New York, 14642
University of California, Davis Medical Center
Sacramento, California, 95817
Seattle Children's Hospital
Seattle, Washington, 98105
St. Louis Children's Hospital
St Louis, Missouri, 63110
About this study
Official title: An Open-Label, Multi-Center Study to Evaluate the Safety and Tolerability of Eteplirsen in Patients With Advanced Stage Duchenne Muscular Dystrophy
The primary objective of this study is to explore safety and tolerability of eteplirsen in participants with advanced stage Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping.
Read the detailed description
This is an open-label, multi-center study to explore the safety and tolerability of eteplirsen injection in participants with advanced stage DMD with confirmed genetic mutations amenable to treatment by exon 51 skipping.
Participants will be evaluated for inclusion during a Screening/Baseline period of up to 4 weeks. Eligible participants will receive once weekly intravenous (IV) infusions of 30 mg/kg eteplirsen for 96 weeks, followed by a safety extension (not to exceed 48 weeks).
Safety will be regularly assessed throughout the study via the collection of adverse events (AEs), laboratory tests, electrocardiograms (ECGs), echocardiograms (ECHOs), vital signs, and physical examinations.
- Conditions
- Muscular Dystrophy, Duchenne
- Being tested
- Eteplirsen
- Keywords
- DMD, exon 51, dystrophin, dystrophy, eteplirsen, 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.
EteplirsenDrug
Also called AVI-4658, EXONDYS 51®
Eteplirsen solution for IV infusion
| Study group | Role | What they receive |
|---|---|---|
| Eteplirsen 30 mg/kg | Experimental | Participants will receive eteplirsen 30 mg/kg/week intravenous (IV) infusions, weekly, for up to 96 weeks. Receives: Drug: Eteplirsen |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Treatment Emergent Adverse Events
An adverse event (AE) was any untoward medical occurrence in a participant that did not necessarily have a causal relationship with the study drug. A serious adverse event (SAE) was an AE resulting in any of the following outcomes: death; Life-threatening event; Required or prolonged inpatient hospitalization; persistent or significant disability/incapacity; congenital anomaly. Treatment-emergent AEs were events that developed or worsened during the on-treatment period (defined as time from first dose of study drug and up to 28 days after last dose of study drug (up to 100 weeks) that were absent before treatment or that worsened relative to pretreatment state. AEs included both serious and non-serious adverse events.
Measured: From first dose of drug up to 100 weeks
Secondary outcomes (5)
Number of Participants With Potentially Clinically Significant Laboratory Abnormalities
Laboratory parameters included hematology, clinical chemistry, urinalysis and coagulation. Data is only reported for parameters in which at least 1 participant had potentially clinically significant abnormal findings.
Incr=increase; LLN=lower limit of normal; ULN=upper limit of normal; GGT=gamma glutamyl transferase
Measured: Baseline up to 100 weeks
Number of Participants With Potentially Clinically Significant Abnormalities in Vital Signs
Vital sign parameters included systolic blood pressure (SBP), diastolic blood pressure (DBP), heart rate (HR), and body temperature. Data is only reported for parameters in which at least 1 participant had potentially clinically significant abnormal vital sign findings.
Measured: Baseline up to 100 weeks
Number of Participants With at Least One Potentially Clinically Significant Abnormalities in Physical Examinations
Physical examinations, full and brief, were performed by the Investigator, a physician Sub-Investigator, or a Nurse Practitioner (if licensed in the state or province to perform physical examinations). Full physical examinations included examination of general appearance; head, ears, eyes, nose, and throat; heart; lungs; chest; abdomen; skin; lymph nodes; and musculoskeletal and neurological systems. Brief physical examinations included examination of general appearance; head, ears, eyes, nose, and throat; heart; lungs; chest; abdomen; and skin.
Measured: Baseline up to 100 weeks
Number of Participants With Abnormalities in Electrocardiograms (ECGs)
Twelve-lead ECGs and Holter ECGs were performed at a consistent time of day throughout the study. Electrocardiograms were performed only after the patient was in the supine position, resting, and quiet for a minimum of 15 minutes. The ECG was manually reviewed and interpreted by medically qualified personnel using a central vendor according to prespecified criteria. The Investigator reviewed the results of the centrally read ECG report and determined if the findings were clinically significant. Data is only reported for parameters in which at least 1 participant had potentially clinically significant abnormal ECG findings.
msec=milliseconds; QTcF=QT interval corrected with Fridericia's method
Measured: Baseline up to 100 weeks
Number of Participants With Abnormalities in Echocardiograms (ECHO)
Standard, 2-dimensional ECHOs were performed at a consistent time of day throughout the study. The ECHO was reviewed and interpreted by medically qualified personnel using a central vendor according to prespecified criteria. Ejection fraction was noted. The Investigator reviewed the results of the ECHO report and determined if the findings were clinically significant.
LEVF=left ventricular ejection fraction
Measured: Baseline up to 100 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Nov 2014 (estimated)
- Study startNov 2014
- Primary completion21 Apr 2017
- Study completion23 Mar 2018
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 Muscular Dystrophy, DuchenneStudies 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
- 24 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
- NCT02286947
- Sponsor's protocol ID
- 4658-204
- Status last verified
- Mar 2020
- First submitted
- 30 Oct 2014
- Last updated
- 30 Mar 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 30 Mar 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label, Multi-Center Study to Evaluate the Safety and Tolerability of Eteplirsen in Patients With Advanced Stage Duchenne Muscular Dystrophy
- ClinicalTrials.gov ID
- NCT02286947
- Acronym / sponsor ID
- 4658-204
- Conditions
- Muscular Dystrophy, Duchenne
- Keywords
- DMD, exon 51, dystrophin, dystrophy, eteplirsen, Duchenne
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 24 participants (actual)
- Lead sponsor
- Sarepta Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- Nov 2014 (actual)
- Primary completion
- 21 Apr 2017 (actual)
- Study completion
- 23 Mar 2018 (actual)
- First posted
- 10 Nov 2014 (estimated)
- Results
- Posted 20 Feb 2019
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The primary objective of this study is to explore safety and tolerability of eteplirsen in participants with advanced stage Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping.
Detailed description
This is an open-label, multi-center study to explore the safety and tolerability of eteplirsen injection in participants with advanced stage DMD with confirmed genetic mutations amenable to treatment by exon 51 skipping.
Participants will be evaluated for inclusion during a Screening/Baseline period of up to 4 weeks. Eligible participants will receive once weekly intravenous (IV) infusions of 30 mg/kg eteplirsen for 96 weeks, followed by a safety extension (not to exceed 48 weeks).
Safety will be regularly assessed throughout the study via the collection of adverse events (AEs), laboratory tests, electrocardiograms (ECGs), echocardiograms (ECHOs), vital signs, and physical examinations.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Eteplirsen 30 mg/kg | Experimental | Drug: EteplirsenParticipants will receive eteplirsen 30 mg/kg/week intravenous (IV) infusions, weekly, for up to 96 weeks. |
Who may qualify
- Ages
- 7 to 21 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (8 listed)
- Male 7 - 21 years of age
- Diagnosis of DMD with a mutation that is amenable to exon 51 skipping, confirmed by a genetic report
- Stable dose of oral corticosteroids for at least 24 weeks or has not received corticosteroids for at least 24 weeks
- Non-ambulatory, or incapable of walking ≥300 meters on the 6-Minute Walk Test (6MWT).
- Score of ≤4 on the Brooke Score for Arms and Shoulders.
Key exclusion criteria (12 listed)
- Use of any pharmacologic treatment (other than corticosteroids) within 12 weeks that may have an effect on muscle strength or function (e.g., growth hormone, anabolic steroids).
- Previous treatment with SMT C1100/BMN 195 at any time.
- Previous treatment with drisapersen (PRO051) within the last 6 months.
- Participation in any other DMD interventional clinical study within 12 weeks
- Major change in physiotherapy regimen within the past 3 months
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®Eteplirsen solution for IV infusion | Drug | Eteplirsen 30 mg/kg |
Primary outcome
Number of Participants With Treatment Emergent Adverse Events
Time frame: From first dose of drug up to 100 weeks
An adverse event (AE) was any untoward medical occurrence in a participant that did not necessarily have a causal relationship with the study drug. A serious adverse event (SAE) was an AE resulting in any of the following outcomes: death; Life-threatening event; Required or prolonged inpatient hospitalization; persistent or significant disability/incapacity; congenital anomaly. Treatment-emergent AEs were events that developed or worsened during the on-treatment period (defined as time from first dose of study drug and up to 28 days after last dose of study drug (up to 100 weeks) that were absent before treatment or that worsened relative to pretreatment state. AEs included both serious and non-serious adverse events.
5 secondary outcomes
Number of Participants With Potentially Clinically Significant Laboratory Abnormalities
Time frame: Baseline up to 100 weeks
Number of Participants With Potentially Clinically Significant Abnormalities in Vital Signs
Time frame: Baseline up to 100 weeks
Number of Participants With at Least One Potentially Clinically Significant Abnormalities in Physical Examinations
Time frame: Baseline up to 100 weeks
Number of Participants With Abnormalities in Electrocardiograms (ECGs)
Time frame: Baseline up to 100 weeks
Number of Participants With Abnormalities in Echocardiograms (ECHO)
Time frame: Baseline up to 100 weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Mar 2020.
- 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.
9 sites in 1 country: 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 Muscular Dystrophy, Duchenne, run by Sarepta Therapeutics, Inc. at 9 sites in United States.
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 8 inclusion and 12 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 30 Mar 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 9 sites in United States. 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 20 Feb 2019). 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 Adverse Events (time frame: From first dose of drug up to 100 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 30 Mar 2020.