Safety Study of Eteplirsen to Treat Advanced Stage Duchenne Muscular Dystrophy

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02286947
• Date of registration: 30/10/2014
• Prospective Registration: Yes
• Primary sponsor: Sarepta Therapeutics, Inc.
• Public title: Safety Study of Eteplirsen to Treat Advanced Stage Duchenne Muscular Dystrophy
• Scientific title: An Open-Label, Multi-Center Study to Evaluate the Safety and Tolerability of Eteplirsen in Patients With Advanced Stage Duchenne Muscular Dystrophy
• Date of first enrolment: November 2014
• Target sample size: 24
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02286947
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

United States

Contacts

• Name: Medical Director
• Affiliation: Sarepta Therapeutics, Inc.

Key inclusion & exclusion criteria

Inclusion Criteria:

- 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

Exclusion Criteria:

- 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

- 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.



Age minimum: 7 Years
Age maximum: 21 Years
Gender: Male

Health Condition(s) or Problem(s) studied

Muscular Dystrophy, Duchenne

Intervention(s)

Drug: Eteplirsen

Primary Outcome(s)

Number of Participants With Treatment Emergent Adverse Events [Time Frame: From first dose of drug up to 100 weeks]

Secondary Outcome(s)

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 Abnormalities in Electrocardiograms (ECGs) [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 Echocardiograms (ECHO) [Time Frame: Baseline up to 100 weeks]

Secondary ID(s)

4658-204

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 20/02/2019
• URL: https://clinicaltrials.gov/ct2/show/results/NCT02286947