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Completed

NCT Number: NCT03218995

Study of Eteplirsen in Young Participants With Duchenne Muscular Dystrophy (DMD) Amenable to Exon 51 Skipping

This is a multicenter, open-label, dose-escalation study to evaluate the safety, tolerability, and PK of once-weekly IV infusions of eteplirsen in approximately 12 male participants, ages 6 months to 48 months (inclusive), who have genotypically confirmed DMD with a deletion mutation amenable to exon 51 skipping.

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Key information

Conditions

Duchenne Muscular Dystrophy Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Genetic Diseases, X-Linked Muscular Diseases Muscular Disorders, Atrophic Muscular Dystrophies Muscular Dystrophy, Duchenne Musculoskeletal Diseases Nervous System Diseases Neuromuscular Diseases

Age range

6 month–48 month

Sex eligibility

Male

Study type

Interventional

Phase

Phase 2

Primary location

Universitair ziekenhuis Gent, Ghent, Belgium

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Eteplirsen

Drug

Infusion for intravenous use.

Other names: AVI-4658, EXONDYS 51®

Primary outcomes

  1. 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.

  2. 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

  3. 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.

  4. 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.

  5. 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.

Secondary outcomes

  1. 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)

  2. 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)

  3. 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)

  4. 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.

Sponsors and collaborators

Lead sponsor

Sarepta Therapeutics, Inc.

Industry

Registry information

Official study title

An Open-Label Safety, Tolerability, and Pharmacokinetics Study of Eteplirsen in Young Patients With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping

Important dates

Study start
2017
Primary completion
2021
Study completion
2021
First posted
Jul 17, 2017
Registry last updated
Dec 9, 2021

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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