Phase 2
N=16
Safety and Dose Finding Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)
Duchenne Muscular Dystrophy
Bottom Line
View on ClinicalTrials.gov: NCT02740972 ↗Enrolled (actual)
16
Serious AEs
0.0%
Results posted
Jul 2021
Primary outcome: Primary: Incidence of Adverse Events as Assessed by CTCAE v4.0. — 3; 4; 4; 5 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- NS-065/NCNP-01 (Drug); Placebo (Drug)
- Age
- Pediatric · 4+ yrs
- Sex
- Male
- Sponsor
- NS Pharma, Inc.
- Primary completion
- Mar 2018
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Incidence of Adverse Events as Assessed by CTCAE v4.0. |
3; 4; 4; 5; 7; 15 | — |
| PRIMARY Dystrophin Production by Western Blot |
5.7; 5.9; 5.8; 5.4; 3.7; 4.5 | — |
| SECONDARY Dystrophin Production by RT-PCR for mRNA - Percentage of Exons Skipped - Molarity |
17.4; 43.9; 30.6 | — |
| SECONDARY Dystrophin Production by Mass Spectrometry |
2.1; 4.2; 3.1 | — |
| SECONDARY Dystrophin Production by Immunofluorescence |
14.2; 34.8; 24.5 | — |
| SECONDARY Change From Baseline in Muscle Strength as Measured by Quantitative Muscle Testing (QMT). |
-0.2029; -0.8513; -0.4808; -0.4173; -1.0867; -0.7041 | — |
| SECONDARY Change From Baseline in Distance Traveled in the Six-Minute Walk Test (6MWT). |
15.6; 44.0; 28.9 | — |
| SECONDARY Change From Baseline in Time to Climb 4 Stairs (TTCLIMB). |
-0.34; 0.00; -0.17 | — |
| SECONDARY Change From Baseline in Time to Climb 4 Stairs (TTCLIMB) Velocity |
0.07; -0.00; 0.32 | — |
| SECONDARY Change From Baseline in Time to Run/Walk 10 Meters (TTRW). |
-0.65; -0.66; -0.66 | — |
| SECONDARY Change From Baseline in Time to Run/Walk 10 Meters (TTRW) Velocity. |
0.21; 0.24; 0.23 | — |
| SECONDARY Change From Baseline in Time to Stand (TTSTAND) |
0.05; -0.44; -0.19 | — |
| SECONDARY Change From Baseline in Time to Stand (TTSTAND) Velocity |
0.02; 0.02; 0.02 | — |
| SECONDARY Change From Baseline in North Star Ambulatory Assessment (NSAA) Score. |
0.5; 1.1; 0.8 | — |
Summary
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.
Eligibility Criteria
Inclusion Criteria
- 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
Exclusion Criteria
- 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.
Data sourced from ClinicalTrials.gov (NCT02740972). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.