Duchenne Muscular Dystrophy
Conditions
Keywords
DMD, Duchenne, Eteplirsen, Dystrophy, Dystrophin, Exon Skipping, Exon 51, Ambulatory, Pediatric
Brief 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.
Interventions
DRUGEteplirsen
Eteplirsen IV infusion once weekly.
Sponsors
Sarepta Therapeutics, Inc.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
MALE
Age
2 Years to 5 Years
Healthy volunteers
No
Inclusion criteria
* Participant successfully completes 96 weeks of treatment in Study 4658-102.
Exclusion criteria
* 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/
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants Experiencing Adverse Events (AEs) | 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 | 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) | 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. |
Countries
Belgium, France, Italy, United Kingdom
Participant flow
Recruitment details
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.
Participants by arm
| Arm | Count |
|---|---|
| Eteplirsen Participants received eteplirsen via IV infusions, once weekly, for up to 162 weeks. | 15 |
| Total | 15 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Study Terminated by Sponsor | 12 |
| Overall Study | Transitioned to Another Sarepta Study | 2 |
| Overall Study | Withdrawal by Subject | 1 |
Baseline characteristics
| Characteristic | Eteplirsen |
|---|---|
| Age, Continuous | 3.7 years STANDARD_DEVIATION 1.1 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 0 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 7 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 8 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 6 Participants |
| Race (NIH/OMB) White | 9 Participants |
| Sex: Female, Male Female | 0 Participants |
| Sex: Female, Male Male | 15 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 15 |
| other Total, other adverse events | 14 / 15 |
| serious Total, serious adverse events | 1 / 15 |
Outcome results
Primary
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.
Time frame: Up to 162 weeks
Population: Safety Set: all participants who received eteplirsen.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Eteplirsen | Number of Participants Experiencing Adverse Events (AEs) | 15 Participants |
Primary
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.
Time frame: Up to 162 weeks
Population: Safety Set: all participants who received eteplirsen.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Eteplirsen | Number of Participants Experiencing Adverse Events of Special Interest (AESIs) | 0 Participants |
Primary
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.
Time frame: Up to 162 weeks
Population: Safety Set: all participants who received eteplirsen.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Eteplirsen | Number of Participants Experiencing Death Due to Adverse Events | 0 Participants |