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出境医 / 临床实验 / A Study to Evaluate Safety, Tolerability, and Efficacy of Eteplirsen in Patients With Duchenne Muscular Dystrophy

A Study to Evaluate Safety, Tolerability, and Efficacy of Eteplirsen in Patients With Duchenne Muscular Dystrophy

Study Description
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 DMD patients who have successfully completed the 96-week eteplirsen study: Study 4658-102 (NCT03218995)

Condition or disease Intervention/treatment Phase
Duchenne Muscular Dystrophy Drug: Eteplirsen Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated 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 Efficacy Study of Eteplirsen in Patients With Duchenne Muscular Dystrophy Who Have Completed Study 4658-102
Actual Study Start Date : June 26, 2019
Estimated Primary Completion Date : November 2026
Estimated Study Completion Date : February 2027
Arms and Interventions
Arm Intervention/treatment
Experimental: Eteplirsen
Patients will receive eteplirsen via intravenous (IV) infusions, once weekly, for up to 284 weeks.
 Drug: Eteplirsen
Eteplirsen IV infusion once weekly.
Outcome Measures
Primary Outcome Measures :
  1. Incidence of Adverse Events (AEs) [ Time Frame: Up to 288 weeks ]
  2. Incidence of Death Due to Adverse Events [ Time Frame: Up to 288 weeks ]
  3. Incidence of Adverse Events of Special Interest (AESIs) [ Time Frame: Up to 288 weeks ]
    AESIs will be defined as any AE that is of scientific and medical concern specific to study treatment, for which ongoing and rapid communication by the Investigator to the sponsor is appropriate. AESIs will include findings potentially indicative of hepatic and renal abnormalities, hypersensitivity, and thrombocytopenia.


Eligibility Criteria
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Ages Eligible for Study:   2 Years to 5 Years   (Child)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Patient successfully completes 96 weeks of treatment in Study 4658-102.

Exclusion Criteria:

  • Patient has a prior or ongoing medical condition that, in the Investigator's opinion, could adversely affect the safety of the patient, or make it unlikely that the course of treatment or follow-up would be completed, or impair the assessment of study results.

Other inclusion/exclusion criteria apply.

Contacts and Locations

Locations
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Belgium
UZ-Gent
Gent, Belgium, 9000
France
Hopital Trousseau, Bâtiment lemariey
Paris, France, 75021
Italy
Fondazione Policlinico Universitario Agostino Gemelli, UOC Neuropsichiatria Infantile
Rome, Italy, 00168
United Kingdom
UCL Great Ormond Street Institute of Child Health, Dubowitz Neuromuscular Centre
London, England, United Kingdom, WC1N 1EH
Sponsors and Collaborators
Sarepta Therapeutics, Inc.
Investigators
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Study Director: Medical Director Sarepta Therapeutics, Inc.
Tracking Information
First Submitted Date  ICMJE June 11, 2019
First Posted Date  ICMJE June 14, 2019
Last Update Posted Date April 14, 2020
Actual Study Start Date  ICMJE June 26, 2019
Estimated Primary Completion Date November 2026   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: April 13, 2020)
  • Incidence of Adverse Events (AEs) [ Time Frame: Up to 288 weeks ]
  • Incidence of Death Due to Adverse Events [ Time Frame: Up to 288 weeks ]
  • Incidence of Adverse Events of Special Interest (AESIs) [ Time Frame: Up to 288 weeks ]
    AESIs will be defined as any AE that is of scientific and medical concern specific to study treatment, for which ongoing and rapid communication by the Investigator to the sponsor is appropriate. AESIs will include findings potentially indicative of hepatic and renal abnormalities, hypersensitivity, and thrombocytopenia.
Original Primary Outcome Measures  ICMJE
 (submitted: June 11, 2019)
  • Incidence of Adverse Events (AEs) [ Time Frame: Up to 288 weeks ]
  • Incidence of Death Due to Adverse Events [ Time Frame: Up to 288 weeks ]
  • Incidence of Adverse Events of Special Interest (AESIs) [ Time Frame: Up to 288 weeks ]
    AESIs will be defined as any AE that is of scientific and medical concern specific to study treatment, for which ongoing and rapid communication by the Investigator to the sponsor is appropriate. AESIs will include infusion-related reactions, hypersensitivity, and renal events.
Change History
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Study to Evaluate Safety, Tolerability, and Efficacy of Eteplirsen in Patients With Duchenne Muscular Dystrophy
Official Title  ICMJE An Open-Label Safety, Tolerability, and Efficacy Study of Eteplirsen in Patients With Duchenne Muscular Dystrophy Who Have Completed Study 4658-102
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 DMD patients who have successfully completed the 96-week eteplirsen study: Study 4658-102 (NCT03218995)
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Duchenne Muscular Dystrophy
Intervention  ICMJE Drug: Eteplirsen
Eteplirsen IV infusion once weekly.
Study Arms  ICMJE Experimental: Eteplirsen
Patients will receive eteplirsen via intravenous (IV) infusions, once weekly, for up to 284 weeks.
Intervention: Drug: Eteplirsen
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Enrolling by invitation
Estimated Enrollment  ICMJE
 (submitted: June 11, 2019) 		15
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE February 2027
Estimated Primary Completion Date November 2026   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Patient successfully completes 96 weeks of treatment in Study 4658-102.

Exclusion Criteria:

  • Patient has a prior or ongoing medical condition that, in the Investigator's opinion, could adversely affect the safety of the patient, or make it unlikely that the course of treatment or follow-up would be completed, or impair the assessment of study results.

Other inclusion/exclusion criteria apply.
Sex/Gender  ICMJE
Sexes Eligible for Study: Male
Ages  ICMJE 2 Years to 5 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Belgium,   France,   Italy,   United Kingdom
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03985878
Other Study ID Numbers  ICMJE 4658-102-OLE
2019-000337-39 ( EudraCT Number )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Sarepta Therapeutics, Inc.
Study Sponsor  ICMJE Sarepta Therapeutics, Inc.
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Medical Director Sarepta Therapeutics, Inc.
PRS Account Sarepta Therapeutics, Inc.
Verification Date April 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP

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