Study to Assess the Safety, Tolerability, and Efficacy of Viltolarsen in Ambulant and Non-Ambulant Boys With DMD (Galactic53)
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. |
ClinicalTrials.gov Identifier: NCT04956289 |
Recruitment Status :
Completed
First Posted : July 9, 2021
Last Update Posted : July 19, 2023
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Sponsor:
NS Pharma, Inc.
Collaborator:
Nippon Shinyaku Co., Ltd.
Information provided by (Responsible Party):
NS Pharma, Inc.
Tracking Information | |||||
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First Submitted Date ICMJE | June 30, 2021 | ||||
First Posted Date ICMJE | July 9, 2021 | ||||
Last Update Posted Date | July 19, 2023 | ||||
Actual Study Start Date ICMJE | July 1, 2021 | ||||
Actual Primary Completion Date | June 20, 2023 (Final data collection date for primary outcome measure) | ||||
Current Primary Outcome Measures ICMJE |
Number of participants with treatment related Adverse Events as assessed by CTCAE v4.03 [ Time Frame: baseline to up to 48 weeks of treatment ] | ||||
Original Primary Outcome Measures ICMJE | Same as current | ||||
Change History | |||||
Current Secondary Outcome Measures ICMJE |
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Original Secondary Outcome Measures ICMJE |
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Current Other Pre-specified Outcome Measures | Not Provided | ||||
Original Other Pre-specified Outcome Measures | Not Provided | ||||
Descriptive Information | |||||
Brief Title ICMJE | Study to Assess the Safety, Tolerability, and Efficacy of Viltolarsen in Ambulant and Non-Ambulant Boys With DMD (Galactic53) | ||||
Official Title ICMJE | A Phase 2 Open-label Study to Assess the Safety, Tolerability, and Efficacy of Viltolarsen in Ambulant and Non-Ambulant Boys With Duchenne Muscular Dystrophy (DMD) Compared to Natural History Controls | ||||
Brief Summary | This is a phase II, open-label study where weekly doses of 80 mg/kg viltolarsen is administered intravenously over a 48-week treatment period to ambulant and non-ambulant DMD patients over the age of 8 years. | ||||
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 |
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Condition ICMJE | Duchenne Muscular Dystrophy | ||||
Intervention ICMJE | Drug: Viltolarsen
Received during weekly intravenous infusions
Other Name: NS-065/NCNP-01
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Study Arms ICMJE | Experimental: Viltolarsen
Patients amenable to exon 53 skipping will receive viltolarsen intravenous (IV) infusions, weekly, at 80 mg/kg for up to 48 weeks.
Intervention: Drug: Viltolarsen
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Publications * | Not Provided | ||||
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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Recruitment Information | |||||
Recruitment Status ICMJE | Completed | ||||
Actual Enrollment ICMJE |
20 | ||||
Original Estimated Enrollment ICMJE | Same as current | ||||
Actual Study Completion Date ICMJE | July 13, 2023 | ||||
Actual Primary Completion Date | June 20, 2023 (Final data collection date for primary outcome measure) | ||||
Eligibility Criteria ICMJE | Inclusion Criteria:
Other inclusion criteria may apply Exclusion Criteria:
Other exclusion criteria may apply |
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Sex/Gender ICMJE |
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Ages ICMJE | 8 Years and older (Child, Adult, Older Adult) | ||||
Accepts Healthy Volunteers ICMJE | No | ||||
Contacts ICMJE | Contact information is only displayed when the study is recruiting subjects | ||||
Listed Location Countries ICMJE | China, Italy, Russian Federation, Spain, Turkey, United States | ||||
Removed Location Countries | |||||
Administrative Information | |||||
NCT Number ICMJE | NCT04956289 | ||||
Other Study ID Numbers ICMJE | NS-065/NCNP-01-211 | ||||
Has Data Monitoring Committee | Not Provided | ||||
U.S. FDA-regulated Product |
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IPD Sharing Statement ICMJE | Not Provided | ||||
Current Responsible Party | NS Pharma, Inc. | ||||
Original Responsible Party | Same as current | ||||
Current Study Sponsor ICMJE | NS Pharma, Inc. | ||||
Original Study Sponsor ICMJE | Same as current | ||||
Collaborators ICMJE | Nippon Shinyaku Co., Ltd. | ||||
Investigators ICMJE | Not Provided | ||||
PRS Account | NS Pharma, Inc. | ||||
Verification Date | July 2023 | ||||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |