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Trial record 9 of 95 for:    Recruiting, Not yet recruiting, Available Studies | "Muscular Dystrophies"

An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular Dystrophy

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ClinicalTrials.gov Identifier: NCT03532542
Recruitment Status : Not yet recruiting
First Posted : May 22, 2018
Last Update Posted : May 22, 2018
Sponsor:
Information provided by (Responsible Party):
Sarepta Therapeutics

Brief Summary:
The main objective of this study is to evaluate the safety and tolerability of long-term treatment with 30 mg/kg of casimersen or golodirsen in patients with Duchenne muscular dystrophy (DMD).

Condition or disease Intervention/treatment Phase
Duchenne Muscular Dystrophy Drug: Casimersen Drug: Golodirsen Phase 3

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 150 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Long-term, Open-label Extension Study for Patients With Duchenne Muscular Dystrophy Enrolled in Clinical Trials Evaluating Casimersen or Golodirsen
Estimated Study Start Date : June 30, 2018
Estimated Primary Completion Date : June 30, 2024
Estimated Study Completion Date : June 30, 2024


Arm Intervention/treatment
Experimental: Casimersen
Patients amenable to exon 45 skipping who have been participating in a clinical trial evaluating casimersen will receive open-label casimersen intravenous (IV) infusions, weekly, at 30 mg/kg for up to 144 Weeks. In the previous clinical trials patients may have been receiving casimersen or matching placebo.
Drug: Casimersen
Casimersen solution for IV infusion
Other Name: SRP-4045

Experimental: Golodirsen
Patients amenable to exon 53 skipping who have been participating in a clinical trial evaluating golodirsen will receive open-label golodirsen intravenous (IV) infusions, weekly, at 30 mg/kg for up to 144 Weeks. In the previous clinical trials patients may have been receiving golodirsen or matching placebo.
Drug: Golodirsen
Golodirsen solution for IV infusion
Other Name: SRP-4053




Primary Outcome Measures :
  1. Number of Patients With Serious Adverse Events (SAEs) [ Time Frame: Up to 30 days after the last infusion of study drug (assessed up to 148 weeks)] ]


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Ages Eligible for Study:   7 Years to 21 Years   (Child, Adult)
Sexes Eligible for Study:   Male
Gender Based Eligibility:   Yes
Accepts Healthy Volunteers:   Yes
Criteria

Inclusion Criteria:

  • Completed a clinical trial evaluating casimersen or golodirsen, per protocol.
  • Is able to understand and comply with all the study requirements and, if under 18 years of age, had as (a) parent(s) or legal guardian(s) who is (are) able to understand and comply with all the study requirements.
  • Is willing to provide informed assent (if applicable) and has (a) parent(s) or legal guardian(s) who is (are) willing to provide written informed consent for the patient to participate in the study.
  • If sexually active, agrees to use a male condom during such activity for the entire duration of the study and for 90 days after the last dose of study drug. The sexual partner must also use a medically acceptable form of contraceptive (example, female oral contraceptives) during this time frame.

Exclusion Criteria:

  • A medical condition that could, in the Investigator's opinion, 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.
  • Any patient who, in the Investigator's opinion, seems unable/unwilling to comply with the study procedures.

Information from the National Library of Medicine

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): NCT03532542


Contacts
Contact: Medical information +1 888 727 3782 medinfo@sarepta.com

Sponsors and Collaborators
Sarepta Therapeutics
Investigators
Study Director: Jon Lu, MD, PhD Sarepta Therapeutics

Responsible Party: Sarepta Therapeutics
ClinicalTrials.gov Identifier: NCT03532542     History of Changes
Other Study ID Numbers: 4045-302
2017-004625-32 ( EudraCT Number )
First Posted: May 22, 2018    Key Record Dates
Last Update Posted: May 22, 2018
Last Verified: May 2018

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Sarepta Therapeutics:
Duchenne muscular dystrophy
Exon Skipping
DMD
Exon 53
Exon 45

Additional relevant MeSH terms:
Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked