Trial Outcomes & Findings for A Study to Evaluate Safety, Tolerability, and Efficacy of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) Who Have Completed Study 4658-102 (NCT03218995) (NCT NCT03985878)
NCT ID: NCT03985878
Last Updated: 2023-08-18
Results Overview
A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' section.
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
15 participants
Primary outcome timeframe
Up to 162 weeks
Results posted on
2023-08-18
Participant Flow
In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
Participant milestones
| Measure |
Eteplirsen
Participants received eteplirsen via intravenous (IV) infusions, once weekly, for up to 162 weeks.
|
|---|---|
|
Overall Study
STARTED
|
15
|
|
Overall Study
Received At Least 1 Dose Of Study Drug
|
15
|
|
Overall Study
COMPLETED
|
0
|
|
Overall Study
NOT COMPLETED
|
15
|
Reasons for withdrawal
| Measure |
Eteplirsen
Participants received eteplirsen via intravenous (IV) infusions, once weekly, for up to 162 weeks.
|
|---|---|
|
Overall Study
Transitioned to Another Sarepta Study
|
2
|
|
Overall Study
Study Terminated by Sponsor
|
12
|
|
Overall Study
Withdrawal by Subject
|
1
|
Baseline Characteristics
A Study to Evaluate Safety, Tolerability, and Efficacy of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) Who Have Completed Study 4658-102 (NCT03218995)
Baseline characteristics by cohort
| Measure |
Eteplirsen
n=15 Participants
Participants received eteplirsen via IV infusions, once weekly, for up to 162 weeks.
|
|---|---|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
7 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
8 Participants
n=5 Participants
|
|
Age, Continuous
|
3.7 years
STANDARD_DEVIATION 1.10 • n=5 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
15 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
9 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
6 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Up to 162 weeksPopulation: Safety Set: all participants who received eteplirsen.
A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' section.
Outcome measures
| Measure |
Eteplirsen
n=15 Participants
Participants received eteplirsen via IV infusions, once weekly, for up to 162 weeks.
|
|---|---|
|
Number of Participants Experiencing Adverse Events (AEs)
|
15 Participants
|
PRIMARY outcome
Timeframe: Up to 162 weeksPopulation: Safety Set: all participants who received eteplirsen.
A summary of all deaths regardless of causality is located in the 'Reported Adverse Events' section.
Outcome measures
| Measure |
Eteplirsen
n=15 Participants
Participants received eteplirsen via IV infusions, once weekly, for up to 162 weeks.
|
|---|---|
|
Number of Participants Experiencing Death Due to Adverse Events
|
0 Participants
|
PRIMARY outcome
Timeframe: Up to 162 weeksPopulation: Safety Set: all participants who received eteplirsen.
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.
Outcome measures
| Measure |
Eteplirsen
n=15 Participants
Participants received eteplirsen via IV infusions, once weekly, for up to 162 weeks.
|
|---|---|
|
Number of Participants Experiencing Adverse Events of Special Interest (AESIs)
|
0 Participants
|
Adverse Events
Eteplirsen
Serious events: 1 serious events
Other events: 14 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Eteplirsen
n=15 participants at risk
Participants received eteplirsen via IV infusions, once weekly, for up to 162 weeks.
|
|---|---|
|
Infections and infestations
Influenza
|
6.7%
1/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
Other adverse events
| Measure |
Eteplirsen
n=15 participants at risk
Participants received eteplirsen via IV infusions, once weekly, for up to 162 weeks.
|
|---|---|
|
Infections and infestations
Nasopharyngitis
|
53.3%
8/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Infections and infestations
Corona virus infection
|
46.7%
7/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Infections and infestations
Rhinitis
|
40.0%
6/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Infections and infestations
Enterobiasis
|
20.0%
3/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Infections and infestations
Influenza
|
13.3%
2/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Infections and infestations
Upper respiratory tract infection
|
20.0%
3/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Infections and infestations
Gastroenteritis
|
13.3%
2/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Infections and infestations
Urinary tract infection
|
13.3%
2/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Infections and infestations
Bronchitis
|
6.7%
1/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Infections and infestations
Foot and mouth disease
|
6.7%
1/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Infections and infestations
Genital infection fungal
|
6.7%
1/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Infections and infestations
Helminthic infection
|
6.7%
1/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Infections and infestations
Herpes zoster
|
6.7%
1/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Infections and infestations
Hordeolum
|
6.7%
1/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Infections and infestations
Laryngitis
|
6.7%
1/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Infections and infestations
Lower respiratory tract infection
|
6.7%
1/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Infections and infestations
Molluscum contagiosum
|
6.7%
1/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Infections and infestations
Onychomycosis
|
6.7%
1/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Infections and infestations
Oral herpes
|
6.7%
1/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Infections and infestations
Otitis media
|
6.7%
1/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Infections and infestations
Pharyngitis
|
6.7%
1/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Infections and infestations
Respiratory tract infection
|
6.7%
1/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Infections and infestations
Tonsillitis
|
6.7%
1/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Infections and infestations
Tooth infection
|
6.7%
1/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Infections and infestations
Tooth abscess
|
6.7%
1/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Infections and infestations
Viral infection
|
6.7%
1/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Infections and infestations
Varicella
|
6.7%
1/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
80.0%
12/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhoea
|
60.0%
9/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
13.3%
2/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
13.3%
2/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
13.3%
2/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Respiratory, thoracic and mediastinal disorders
Productive cough
|
6.7%
1/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Gastrointestinal disorders
Constipation
|
33.3%
5/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Gastrointestinal disorders
Vomiting
|
33.3%
5/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Gastrointestinal disorders
Abdominal pain upper
|
26.7%
4/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Gastrointestinal disorders
Diarrhoea
|
26.7%
4/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Gastrointestinal disorders
Abdominal pain
|
20.0%
3/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Gastrointestinal disorders
Dental caries
|
13.3%
2/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Gastrointestinal disorders
Abdominal pain lower
|
6.7%
1/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Gastrointestinal disorders
Enteritis
|
6.7%
1/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Gastrointestinal disorders
Gastrointestinal disorder
|
6.7%
1/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Gastrointestinal disorders
Toothache
|
6.7%
1/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
General disorders
Pyrexia
|
60.0%
9/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
General disorders
Influenza like illness
|
20.0%
3/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
General disorders
Gait disturbance
|
13.3%
2/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
General disorders
Catheter site pain
|
6.7%
1/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
General disorders
Catheter site swelling
|
6.7%
1/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
General disorders
Chest pain
|
6.7%
1/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
General disorders
Device dislocation
|
6.7%
1/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
General disorders
Hyperpyrexia
|
6.7%
1/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
General disorders
Infusion site extravasation
|
6.7%
1/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
General disorders
Oedema
|
6.7%
1/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
General disorders
Oedema peripheral
|
6.7%
1/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Injury, poisoning and procedural complications
Contusion
|
33.3%
5/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Injury, poisoning and procedural complications
Fall
|
33.3%
5/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Injury, poisoning and procedural complications
Head injury
|
20.0%
3/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Injury, poisoning and procedural complications
Excoriation
|
13.3%
2/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Injury, poisoning and procedural complications
Face injury
|
13.3%
2/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Injury, poisoning and procedural complications
Scratch
|
13.3%
2/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Injury, poisoning and procedural complications
Skin abrasion
|
13.3%
2/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Injury, poisoning and procedural complications
Arthropod bite
|
6.7%
1/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Injury, poisoning and procedural complications
Eye contusion
|
6.7%
1/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Injury, poisoning and procedural complications
Human bite
|
6.7%
1/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Injury, poisoning and procedural complications
Laceration
|
6.7%
1/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Injury, poisoning and procedural complications
Ligament sprain
|
6.7%
1/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Injury, poisoning and procedural complications
Muscle strain
|
6.7%
1/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Injury, poisoning and procedural complications
Subcutaneous haematoma
|
6.7%
1/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Injury, poisoning and procedural complications
Traumatic haematoma
|
6.7%
1/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Skin and subcutaneous tissue disorders
Rash maculo-papular
|
26.7%
4/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Skin and subcutaneous tissue disorders
Ecchymosis
|
13.3%
2/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Skin and subcutaneous tissue disorders
Rash
|
13.3%
2/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
6.7%
1/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Skin and subcutaneous tissue disorders
Keloid scar
|
6.7%
1/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Skin and subcutaneous tissue disorders
Rash papular
|
6.7%
1/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Skin and subcutaneous tissue disorders
Skin lesion
|
6.7%
1/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Nervous system disorders
Headache
|
40.0%
6/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Nervous system disorders
Cognitive disorder
|
6.7%
1/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Nervous system disorders
Motor dysfunction
|
6.7%
1/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Nervous system disorders
Psychomotor hyperactivity
|
6.7%
1/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
20.0%
3/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
6.7%
1/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
6.7%
1/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Ear and labyrinth disorders
Ear pain
|
13.3%
2/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Ear and labyrinth disorders
Tympanic membrane hyperaemia
|
6.7%
1/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Eye disorders
Chalazion
|
13.3%
2/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Eye disorders
Eye pain
|
6.7%
1/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Eye disorders
Eye pruritus
|
6.7%
1/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Metabolism and nutrition disorders
Hypocalcaemia
|
6.7%
1/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Metabolism and nutrition disorders
Vitamin D deficiency
|
6.7%
1/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Metabolism and nutrition disorders
Weight gain poor
|
6.7%
1/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Cardiac disorders
Angina pectoris
|
6.7%
1/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Cardiac disorders
Dilatation ventricular
|
6.7%
1/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Cardiac disorders
Tachycardia
|
6.7%
1/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Psychiatric disorders
Initial insomnia
|
6.7%
1/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Psychiatric disorders
Irritability
|
6.7%
1/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Renal and urinary disorders
Chromaturia
|
6.7%
1/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Renal and urinary disorders
Haematuria
|
6.7%
1/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Renal and urinary disorders
Myoglobinuria
|
6.7%
1/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Blood and lymphatic system disorders
Neutropenia
|
6.7%
1/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
|
Investigations
Urine albumin/creatinine ratio abnormal
|
6.7%
1/15 • Up to 162 weeks
Safety Set: all participants who received eteplirsen. In an effort to reduce clinical trial burden on participants while ensuring continued treatment if desired, pending commercial availability of eteplirsen, participants were either transitioned to a post-trial access program or another Sarepta study, or they declined further treatment.
|
Additional Information
Medical Director
Sarepta Therapeutics, Inc.
Phone: 1-888-SAREPTA (1-888-727-3782)
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee There are agreements between the Principal Investigators and the Sponsor (or its agents) that restrict the PIs rights to discuss or publish trial results after the trial is completed.
- Publication restrictions are in place
Restriction type: OTHER