Study of Eteplirsen in Young Participants With Duchenne Muscular Dystrophy (DMD) Amenable to Exon 51 Skipping
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ClinicalTrials.gov Identifier: NCT03218995 |
Recruitment Status :
Completed
First Posted : July 17, 2017
Results First Posted : December 9, 2021
Last Update Posted : December 9, 2021
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Condition or disease | Intervention/treatment | Phase |
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Duchenne Muscular Dystrophy | Drug: Eteplirsen | Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 15 participants |
Allocation: | N/A |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | An Open-Label Safety, Tolerability, and Pharmacokinetics Study of Eteplirsen in Young Patients With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping |
Actual Study Start Date : | August 16, 2017 |
Actual Primary Completion Date : | March 10, 2021 |
Actual Study Completion Date : | March 10, 2021 |

Arm | Intervention/treatment |
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Experimental: Eteplirsen
Eteplirsen will be administered once every 7 days by intravenous (IV) infusion starting on Day 1 for up to 96 weeks. The starting dose will be 2 milligrams/kilogram (mg/kg) eteplirsen, with escalation to 4, 10, 20, and 30 mg/kg for 10 weeks, and then participants will continue to receive eteplirsen at 30 mg/kg for the duration of the study.
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Drug: Eteplirsen
Infusion for intravenous use.
Other Names:
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- Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and TEAEs Leading to Discontinuation From Study Drug [ Time Frame: Baseline up to Week 100 ]TEAEs were defined as adverse events (AEs) with an onset following administration of the first dose of study drug. An AE was any untoward medical occurrence in a participant, which does not necessarily have a causal relationship with the study drug. Abnormalities presented at Baseline were considered AEs if they reoccurred after resolution or worsen during the AE collection period. An SAE was defined as any AE that, in the view of either the Investigator or Sponsor, resulted in any of the following outcomes as fatal, life-threatening, required hospitalization or prolongation of existing hospitalization, persistent or significant disability/incapacity, a congenital anomaly/birth defect, or an important medical event. A summary of all SAEs and Other AEs (nonserious) regardless of causality is located in the 'Adverse Events' Section.
- Number of Participants With at Least 1 Potentially Clinically Significant Clinical Safety Laboratory Abnormality [ Time Frame: Baseline up to Week 100 ]
Clinical laboratory parameters that were evaluated included
- Any Grade ≥2 (moderate) or serious event without an alternative etiology that the Investigator deemed was related to study drug
- Two consecutive drug-related serum creatinine levels ≥2*upper limit of normal (ULN) without an alternative etiology
- Creatine kinase (CK) levels >50,000 units/liter (U/L)
- A confirmed, unexplained, increase in gamma glutamyl transferase (GGT) >3*ULN and either an increase in bilirubin >2*ULN or nascent prothrombin time >2*ULN concurrently, without an alternative etiology
- Number of Participants With at Least 1 Markedly Abnormal Vital Sign [ Time Frame: Baseline up to Week 100 ]The vital sign parameters that were evaluated included blood pressure, heart rate, respiration, and temperature. A summary of all SAEs and Other AEs (nonserious) regardless of causality is located in the 'Adverse Events' Section.
- Abnormal Changes From Baseline or Worsening of Physical Examination Findings [ Time Frame: Baseline up to Week 100 ]Data not collected during the study for this Outcome Measure. A summary of all SAEs and Other AEs (nonserious) regardless of causality is located in the 'Adverse Events' Section.
- Number of Participants With at Least 1 Markedly Abnormal Electrocardiogram (ECG) and Echocardiogram (ECHO) [ Time Frame: Weeks 8, 12, 24, 36, 48, 60, 72, 84, 96 ]The ECG was manually reviewed and interpreted by medically qualified personnel using a central vendor according to pre-specified criteria. The Investigator determined if the findings in the centrally read ECG report were clinically significant. Clinical significance was defined as any variation in ECG findings that had medical relevance resulting in an alteration in medical care. The ECHO was reviewed and interpreted by medically qualified personnel using a central vendor according to pre-specified criteria. The Investigator determined if the findings in the ECHO report were clinically significant. Clinical significance was defined as any variation in ECHO findings that had medical relevance resulting in an alteration in medical care. A summary of all SAEs and Other AEs (nonserious) regardless of causality is located in the 'Adverse Events' Section.
- Maximum Plasma Concentration (Cmax) of Eteplirsen [ Time Frame: Pre-infusion, immediately prior to end of infusion, and approximately 1-3 hours and 6-8 hours after completion of infusion during Weeks 2 (2 mg/kg dose level), 6 (10 mg/kg dose level), 8 (20 mg/kg dose level), and 10 and 24 (30 mg/kg dose level) ]
- Time to Reach Maximum Plasma Concentration (Tmax) of Eteplirsen [ Time Frame: Pre-infusion, immediately prior to end of infusion, and approximately 1-3 hours and 6-8 hours after completion of infusion during Weeks 2 (2 mg/kg dose level), 6 (10 mg/kg dose level), 8 (20 mg/kg dose level), and 10 and 24 (30 mg/kg dose level) ]
- Area Under Concentration-Time Curve From Time 0 to the Last Quantifiable Concentration (AUClast) of Eteplirsen in Plasma [ Time Frame: Pre-infusion, immediately prior to end of infusion, and approximately 1-3 hours and 6-8 hours after completion of infusion during Weeks 2 (2 mg/kg dose level), 6 (10 mg/kg dose level), 8 (20 mg/kg dose level), and 10 and 24 (30 mg/kg dose level) ]
- Amount of Drug Eliminated in Urine [ Time Frame: Pre-infusion, immediately prior to end of infusion, and approximately 1-3 hours and 6-8 hours after completion of infusion during Weeks 2 (2 mg/kg dose level), 6 (10 mg/kg dose level), 8 (20 mg/kg dose level), and 10 and 24 (30 mg/kg dose level) ]Amount of unchanged drug excreted in urine from time 0 to 4 hours after completion of dosing is reported.

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
Ages Eligible for Study: | 6 Months to 48 Months (Child) |
Sexes Eligible for Study: | Male |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Male between 6 months to 48 months of age (inclusive)
- Diagnosis of DMD with a deletion mutation amenable to exon 51 skipping
- Parent(s) or legal guardian(s) who is willing to provide written informed consent
Exclusion Criteria:
- Received treatment that might have an effect on muscle strength or function within 12 weeks prior to dosing
- Received previous or current treatment with any experimental treatment
- Clinically significant illness other than DMD
- Clinically significant laboratory abnormality
- Any other condition that could interfere with the participation in the study.

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03218995
Belgium | |
Universitair ziekenhuis Gent | |
Gent, Belgium, 9000 | |
France | |
Armand-Trousseau Hospital | |
Paris, France, 75012 | |
Italy | |
Site Fondazione Policlinico Universitario Agostino Gemelli | |
Roma, Italy | |
United Kingdom | |
UCL Great Ormond Street Institute of Child Health | |
London, United Kingdom, WC1N 1EH |
Study Chair: | Medical Director | Sarepta Therapeutics, Inc. |
Documents provided by Sarepta Therapeutics, Inc.:
Responsible Party: | Sarepta Therapeutics, Inc. |
ClinicalTrials.gov Identifier: | NCT03218995 |
Other Study ID Numbers: |
4658-102 |
First Posted: | July 17, 2017 Key Record Dates |
Results First Posted: | December 9, 2021 |
Last Update Posted: | December 9, 2021 |
Last Verified: | November 2021 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | No |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
Product Manufactured in and Exported from the U.S.: | Yes |
DMD Duchenne Eteplirsen |
dystrophy dystrophin exon 51 |
Muscular Dystrophies Muscular Dystrophy, Duchenne Muscular Disorders, Atrophic Muscular Diseases Musculoskeletal Diseases |
Neuromuscular Diseases Nervous System Diseases Genetic Diseases, Inborn Genetic Diseases, X-Linked |