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Safety and Dose Finding Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)
study id #: NCT02740972
condition: Duchenne Muscular Dystrophy
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.
intervention: NS-065/NCNP-01, Placebo
mechanism of action: Exon-skipping to promote dystrophin production
start date: December 2016
estimated completion: March 2018
phase of development: Phase 2
size / enrollment: 16
This is a Phase II, multiple center, 2-period, randomized, placebo-controlled, dose finding study of NS-065/NCNP-01 administered by infusion once weekly for 24 weeks to ambulant boys ages 4-<10 years with DMD. Two dose level cohorts will be enrolled. Period 1 of this study will be conducted in a double-blind fashion. Randomized patients will receive weekly IV infusions of NS-065/NCNP-01 or placebo for the first 4 weeks of their participation (Period 1) and NS-065/NCNP-01 by IV infusion for weeks 5-24 (20 weeks of active treatment - Period 2). Analysis of safety data from Period 1 of the 40mg/kg dose cohort will be completed prior to enrolling patients in the 80mg/kg dose cohort.
Patients completing the 24-week study will be eligible for an open-label extension study.
Clinical efficacy will be assessed at regularly scheduled study visits, including functional tests such as the six-minute walk test (6MWT), time to stand (TTSTAND), time to run/walk 10 meters (TTRW), time to climb 4 stairs (TTCLIMB) and quantitative muscle testing (QMT). All patients will undergo a muscle biopsy of the bicep at baseline and a second muscle biopsy at Week 24.
Safety will be assessed through the collection of adverse events (AEs), blood and urine laboratory tests, electrocardiograms (ECGs), vital signs, and physical examinations throughout the study.
Serial blood samples will be taken at four of the study visits to assess the pharmacokinetics of the study drug.
- Incidence of Adverse Events as assessed by CTCAE v4.0. [ Time Frame: 24 weeks of treatment phase ]
- Dystrophin protein in muscle as measured by western blot. [ Time Frame: 20-24 weeks of treatment ]
- Drug concentration in plasma. [ Time Frame: 20-24 weeks of treatment ]
- Induction of dystrophin messenger ribonucleic acid (mRNA) in muscle as measured by real time polymerase chain reaction (RT-PCR) for mRNA analysis. [ Time Frame: 20-24 weeks of treatment ]
- Induction of dystrophin protein in muscle as measured by mass spectrometry and immunofluorescence staining methods for protein analysis. [ Time Frame: 20-24 weeks of treatment ]
- Muscle strength as measured by Quantitative Muscle Testing (QMT). [ Time Frame: 20-24 weeks of treatment ]
- Change in distance traveled in the Six-Minute Walk Test (6MWT). [ Time Frame: baseline to 20-24 weeks of treatment ]
- Change in Time to Climb 4 Stairs (TTCLIMB). [ Time Frame: baseline to 20-24 weeks of treatment ]
- Change in Time to Run/Walk 10 meters (TTRW). [ Time Frame: baseline to 20-24 weeks of treatment ]
- Change in Time to Stand (TTSTAND) [ Time Frame: baseline to 20-24 weeks of treatment ]
- North Star Ambulatory Assessment (NSAA) results. [ Time Frame: baseline to 20-24 weeks of treatment ]
• 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
• 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.
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