Phase 2
Completed N=16
Safety and Dose Finding Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)
Source: ClinicalTrials.gov NCT02740972 ↗Enrolled (actual)
16
Serious AEs
0.0%
Results posted
Jul 2021
Primary outcomePrimary: Incidence of Adverse Events as Assessed by CTCAE v4.0. — 3; 4; 4; 5 Participants
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.
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 | — |
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. Informational only — not medical advice.