NS-050/NCNP-03 in Boys With DMD (Meteor50)

NS Pharma

Status and phase: Enrolling; Phase 2; Phase 1

Conditions: Duchenne Muscular Dystrophy

Treatments: Drug: NS-050/NCNP-03; Drug: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT06053814

NS-050/NCNP-03-101

Details and patient eligibility

About

This is a Phase 1/2 study of Multiple-Ascending Dose (MAD) levels for 12 weeks of treatment followed by 24 weeks of open-label treatment with a selected dose of NS-050/NCNP-03 administered once weekly to ambulant boys with DMD, who have a DMD exon deletion amenable to exon 50 skipping.

Enrollment: 20 estimated patients

Sex: Male

Ages: 4 to 14 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Male ≥ 4 years and <15 years of age;
• Confirmed DMD exon deletion in the dystrophin gene that is amenable to skipping of exon 50 to restore the dystrophin mRNA reading frame;
• Able to walk independently without assistive devices;
• Able to complete the TTSTAND without assistance in <20 seconds;
• Stable dose of glucocorticoid for at least 3 months and the dose is expected to remain on a stable dose for the duration of the study.

Other inclusion criteria may apply.

Exclusion criteria

• Evidence of symptomatic cardiomyopathy;
• Current or previous treatment with anabolic steroids (e.g., oxendolone, oxandrolone) or products containing resveratrol or adenosine triphosphate within 3 months prior to first dose of study drug;
• Currently taking another investigational drug or has taken another investigational drug within 3 months prior to the first dose of study drug;
• Surgery within the 3 months prior to the first dose of study drug or planned during the study duration;
• Having taken any gene therapy.

Other exclusion criteria may apply.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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20 participants in 3 patient groups, including a placebo group

Part 1: NS-050/NCNP-03

Experimental group

Description:

Participants will be randomized and receive NS-050/NCNP-03 intravenous (IV) infusions once weekly for 2 weeks at each of MAD levels (1.95, 5, 10, 20, 40, and 80 mg/kg).

Treatment:

Drug: NS-050/NCNP-03

Part 1: Placebo

Placebo Comparator group

Description:

Participants will be randomized and receive NS-050/NCNP-03 placebo-matching IV infusions once weekly for 2 weeks at each of MAD levels.

Treatment:

Drug: Placebo

Part 2: NS-050/NCNP-03

Experimental group

Description:

Participants will receive NS-050/NCNP-03 IV infusions once weekly for 24 weeks at the dosage selected by the Data and Safety Monitoring Board (DSMB) at the conclusion of Part 1.

Treatment:

Drug: NS-050/NCNP-03