Safety and Dose Finding Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)
Completed · Phase 2 · Has a placebo group
Conditions studied: Duchenne Muscular Dystrophy
In brief
The main objective of this study is to evaluate the safety of a high (80mg/kg) and low (40mg/kg) dose of NS-065/NCNP-01 delivered as an intravenous infusion in patients with Duchenne Muscular Dystrophy (DMD) amendable to exon 53 skipping. Additional objectives include tolerability, muscle function and strength, pharmacokinetics and pharmacodynamics.
Key facts
- Study ID
- NCT02740972
- Run by
- NS Pharma, Inc.
- People needed
- 16
- Starts
- 2016-12-01
- Expected to finish
- 2018-04-01
- Last updated by the study team
- 2021-12-07
Who can join
Age: 4 and older, up to 9. Sex: male. Healthy volunteers: not accepted.
You may qualify if…
- Male ≥ 4 years and <10 years of age
- Confirmed DMD mutation(s) in the dystrophin gene that is amenable to skipping of exon 53 to restore the dystrophin mRNA reading frame;
- Able to walk independently without assistive devices;
- Ability to complete the time to stand, time to run/walk and time to climb assessments;
- Stable dose of glucocorticoid for at least 3 months
You may not qualify if…
- Acute illness within 4 weeks prior to the first dose of study medication;
- Evidence of symptomatic cardiomyopathy. [Note: Asymptomatic cardiac abnormality on investigation would not be exclusionary];
- Severe allergy or hypersensitivity to medications;
- Severe behavioral or cognitive problems that preclude participation in the study, in the opinion of the Investigator;
- Previous or ongoing medical condition, medical history, physical findings or laboratory abnormalities that could affect safety, make it unlikely that treatment and follow-up will be correctly completed or impair the assessment of study results, in the opinion of the Investigator;
- Patient is taking any other investigational drug currently or within 3 months prior to the start of study treatment; or
- Patient has had surgery within the 3 months prior to the first anticipated administration of study medication or surgery is planned for anytime during the duration of the study;
- Patient has previously participated in this study or any other study during which NS-065/NCNP-01 was administered.
Where it is running
- UC Davis — Sacramento, California, United States
- University of Florida Health — Gainesville, Florida, United States
- Lurie Children's Hospital — Chicago, Illinois, United States
- Washington University — St Louis, Missouri, United States
- Duke University Medical Center — Durham, North Carolina, United States
- Children's Hospital of Richmond at VCU — Richmond, Virginia, United States
- Alberta Children's Hospital — Calgary, Alberta, Canada
Full record on ClinicalTrials.gov
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