"A Phase 3 study of Viltolarsen in boys with Duchenne Muscular Dystrophy (DMD)"

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Ns Pharma Inc.

Participant type

Pediatric, Patients

Age range

0-17 years

Gender

Male

Therapeutic area

Diseases [C] - Musculoskeletal Diseases [C05]

Trial duration

7 Jun 2021 → 3 Nov 2025

Decision date (initial)

2024-02-27

Transition trial

Yes

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

NS Pharma, Inc.

External identifiers

EU CT number

2023-507146-91-00

EudraCT number

2021-000122-10

WHO UTN

U1111-1298-6244

ClinicalTrials.gov

NCT04768062

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Others, Therapy, Efficacy

To evaluate the safety and tolerability of viltolarsen administered
intravenously (IV) at weekly doses of 80 mg/kg in boys who have
completed the NS-065/NCNP-01-301 study

Secondary objectives 1

1. Secondary Objective: To compare the efficacy of viltolarsen administered IV at weekly doses of 80 mg/kg in boys who have completed the NS-065/NCNP-01-301 study over a 96-week treatment period versus natural history controls using strength and endurance outcomes.

Conditions and MedDRA coding

Duchenne Muscular Dystrophy (DMD)

Regulatory references

EMA paediatric investigation plan (PIP)

EMEA-002853-PIP01-20

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 3

1. 1. Patient has completed the NS-065/NCNP-01-301 study;
2. 2. Patient's parent(s) or legal guardian(s) has (have) provided written informed consent and Health Insurance Portability and Accountability Act authorization, where applicable, prior to any study-related procedures; patients will be asked to give written or verbal assent according to local requirements;
3. 3. Patient and parent(s)/guardian(s) are willing and able to comply with scheduled visits, investigational product (IP) administration plan, and study procedures.

Exclusion criteria 4

1. 1. Patient had an adverse event in Study NS-065/NCNP-01-301 that, in the opinion of the investigator and/or the sponsor, precludes safe use of viltolarsen for the patient in this study;
2. 2. Patient had a treatment which was made for the purpose of dystrophin or dystrophin-related protein induction after completion of Study NS-065/NCNP-01-301;
3. 3. Patient took any other investigational drug(s) during or after completion of Study NS-065/NCNP-01-301;
4. 4. Patient is judged by the investigator and/or the sponsor not to be appropriate to participate in the extension study for any reason.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. - Vital signs - Physical examination - Clinical laboratory tests o Hematology and clinical chemistry o Urinalysis o Urine cytology - Antibodies to dystrophin and viltolarsen - 12-lead electrocardiogram (ECG) - Renal ultrasound - Treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)

Secondary endpoints 1

1. Secondary: - Time to Stand Test (TTSTAND) - Time to Run/Walk 10 Meters Test (TTRW) - Six-minute Walk Test (6MWT) - North Star Ambulatory Assessment (NSAA) - Time to Climb 4 Stairs Test (TTCLIMB) - Quantitative muscle strength measured by hand-held dynamometer (elbow extension, elbow flexion, knee extension, and knee flexion on the dominant side only)

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Ns Pharma Inc.

Sponsor organisation

Ns Pharma Inc.

Address

140 East Ridgewood Avenue Suite 280s

City

Paramus

Postcode

07652-3914

Country

United States

Scientific contact point

Organisation

Ns Pharma Inc.

Contact name

Regulatory Submissions

Public contact point

Organisation

Ns Pharma Inc.

Contact name

Regulatory Submissions

Third parties 14

Locations

6 EU/EEA countries · 9 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 54 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

12 submissions — initial application plus substantial / non-substantial modifications