ICH GCP - EU Clinical trials Registry

Summary
EudraCT Number:	2021-000122-10
Sponsor's Protocol Code Number:	NS-065/NCNP-01-302
National Competent Authority:	Netherlands - Competent Authority
Clinical Trial Type:	EEA CTA
Trial Status:
Date on which this record was first entered in the EudraCT database:	2021-04-08
Trial results

Index
A. PROTOCOL INFORMATION
B. SPONSOR INFORMATION
C. APPLICANT IDENTIFICATION
D. IMP IDENTIFICATION
D.8 INFORMATION ON PLACEBO
E. GENERAL INFORMATION ON THE TRIAL
F. POPULATION OF TRIAL SUBJECTS
G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
P. END OF TRIAL

A. Protocol Information

A.1

Member State Concerned

Netherlands - Competent Authority

A.2

EudraCT number

2021-000122-10

A.3

Full title of the trial

A Phase 3, Multi-center, Open-label Extension Study to Assess the Safety and Efficacy of Viltolarsen in Ambulant Boys with Duchenne Muscular Dystrophy (DMD)

A.3.1

Title of the trial for lay people, in easily understood, i.e. non-technical, language

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

A.3.2

Name or abbreviated title of the trial where available

RACER-53X

A.4.1

Sponsor's protocol code number

NS-065/NCNP-01-302

A.5.4

Other Identifiers

Name:

US IND

Number:

127474

A.7

Trial is part of a Paediatric Investigation Plan

No

A.8

EMA Decision number of Paediatric Investigation Plan

B. Sponsor Information
B.Sponsor: 1
B.1.1	Name of Sponsor	NS Pharma, Inc.
B.1.3.4	Country	United States
B.3.1 and B.3.2	Status of the sponsor	Commercial
B.4 Source(s) of Monetary or Material Support for the clinical trial:
B.4.1	Name of organisation providing support	NS Pharma, Inc.
B.4.2	Country	United States
B.4.1	Name of organisation providing support	Nippon Shinyaku Co., Ltd
B.4.2	Country	Japan
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Medpace
B.5.2	Functional name of contact point	Kyle Haas
B.5.3	Address:
B.5.3.1	Street Address	Medpace Way
B.5.3.2	Town/ city	Cincinnati
B.5.3.3	Post code	5375
B.5.3.4	Country	United States
B.5.4	Telephone number	+151357999111270
B.5.6	E-mail	regsubmissions@medpace.com

D. IMP Identification
D.IMP: 1
D.1.2 and D.1.3	IMP Role	Test
D.2	Status of the IMP to be used in the clinical trial
D.2.1	IMP to be used in the trial has a marketing authorisation	No
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	Yes
D.2.5.1	Orphan drug designation number	EU/3/20/2282
D.3 Description of the IMP
D.3.1	Product name	Viltolarsen
D.3.2	Product code	NS-065/NCNP-01
D.3.4	Pharmaceutical form	Solution for infusion
D.3.4.1	Specific paediatric formulation	Yes
D.3.7	Routes of administration for this IMP	Intravenous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	VILTOLARSEN
D.3.9.1	CAS number	2055732-84-6
D.3.9.2	Current sponsor code	NS-065/NCNP-01
D.3.9.4	EV Substance Code	SUB195543
D.3.10	Strength
D.3.10.1	Concentration unit	mg/ml milligram(s)/millilitre
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	50
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	Yes
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	No
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	No
D.3.11.5	Radiopharmaceutical medicinal product	No
D.3.11.6	Immunological medicinal product (such as vaccine, allergen, immune serum)	No
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	No
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another type of medicinal product	No

D.8 Information on Placebo

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

Duchenne Muscular Dystrophy (DMD)

E.1.1.1

Medical condition in easily understood language

Muscular Dystrophy

E.1.1.2

Therapeutic area

Diseases [C] - Musculoskeletal Diseases [C05]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	20.0
E.1.2	Level	PT
E.1.2	Classification code	10013801
E.1.2	Term	Duchenne muscular dystrophy
E.1.2	System Organ Class	10010331 - Congenital, familial and genetic disorders

E.1.3

Condition being studied is a rare disease

Yes

E.2 Objective of the trial

E.2.1

Main objective of the trial

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

E.2.2

Secondary objectives of the trial

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.

Exploratory Objectives:
- To evaluate health-related quality of life impact of viltolarsen treatment on patient's DMD
- To evaluate preservation of ambulation of patients with DMD

E.2.3

Trial contains a sub-study

No

E.3

Principal inclusion criteria

1. Patient has completed the NS-065/NCNP-01-301 study;
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. Patient and parent(s)/guardian(s) are willing and able to comply with scheduled visits, investigational product (IP) administration plan, and study procedures.

E.4

Principal exclusion criteria

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. 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. Patient took any other investigational drug(s) during or after completion of Study NS-065/NCNP-01-301;
4. Patient is judged by the investigator and/or the sponsor not to be appropriate to participate in the extension study for any reason.

E.5 End points

E.5.1

Primary end point(s)

- 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)

E.5.1.1

Timepoint(s) of evaluation of this end point

At 96 weeks treatment

E.5.2

Secondary end point(s)

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)

Exploratory:
- Pediatric Outcome Data Collection Instrument (PODCI)
- Personal Adjustment and Role Skills Scale, 3rd edition (PARS III) Questionnaire
-Loss of ambulation

E.5.2.1

Timepoint(s) of evaluation of this end point

At 96 weeks treatment

E.6 and E.7 Scope of the trial

E.6

Scope of the trial

E.6.1

Diagnosis

No

E.6.2

Prophylaxis

No

E.6.3

Therapy

Yes

E.6.4

Safety

Yes

E.6.5

Efficacy

Yes

E.6.6

Pharmacokinetic

Yes

E.6.7

Pharmacodynamic

No

E.6.8

Bioequivalence

No

E.6.9

Dose response

No

E.6.10

Pharmacogenetic

No

E.6.11

Pharmacogenomic

No

E.6.12

Pharmacoeconomic

No

E.6.13

Others

No

E.7

Trial type and phase

E.7.1

Human pharmacology (Phase I)

No

E.7.1.1

First administration to humans

No

E.7.1.2

Bioequivalence study

No

E.7.1.3

Other

No

E.7.1.3.1

Other trial type description

E.7.2

Therapeutic exploratory (Phase II)

No

E.7.3

Therapeutic confirmatory (Phase III)

Yes

E.7.4

Therapeutic use (Phase IV)

No

E.8 Design of the trial

E.8.1

Controlled

No

E.8.1.1

Randomised

No

E.8.1.2

Open

Yes

E.8.1.3

Single blind

No

E.8.1.4

Double blind

No

E.8.1.5

Parallel group

No

E.8.1.6

Cross over

No

E.8.1.7

Other

No

E.8.2

Comparator of controlled trial

E.8.2.1

Other medicinal product(s)

No

E.8.2.2

Placebo

No

E.8.2.3

Other

No

E.8.3

The trial involves single site in the Member State concerned

No

E.8.4

The trial involves multiple sites in the Member State concerned

Yes

E.8.4.1

Number of sites anticipated in Member State concerned

2

E.8.5

The trial involves multiple Member States

Yes

E.8.5.1

Number of sites anticipated in the EEA

20

E.8.6 Trial involving sites outside the EEA

E.8.6.1

Trial being conducted both within and outside the EEA

Yes

E.8.6.2

Trial being conducted completely outside of the EEA

No

E.8.6.3

If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned

Australia

Canada

Chile

China

Greece

Hong Kong

Italy

Japan

Korea, Republic of

Mexico

Netherlands

New Zealand

Norway

Russian Federation

Spain

Taiwan

Turkey

Ukraine

United Kingdom

United States

E.8.7

Trial has a data monitoring committee

Yes

E.8.8

Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial

The end of study date is defined as the date when the last patient at the site is assessed or receives an intervention for evaluation in the study (i.e., last patient last visit).

E.8.9 Initial estimate of the duration of the trial

E.8.9.1

In the Member State concerned years

2

E.8.9.1

In the Member State concerned months

9

E.8.9.1

In the Member State concerned days

0

E.8.9.2

In all countries concerned by the trial years

2

E.8.9.2

In all countries concerned by the trial months

9

E.8.9.2

In all countries concerned by the trial days

0

F. Population of Trial Subjects

F.1 Age Range

F.1.1

Trial has subjects under 18

Yes

F.1.1

Number of subjects for this age range:

74

F.1.1.1

In Utero

No

F.1.1.2

Preterm newborn infants (up to gestational age < 37 weeks)

No

F.1.1.3

Newborns (0-27 days)

No

F.1.1.4

Infants and toddlers (28 days-23 months)

No

F.1.1.5

Children (2-11years)

Yes

F.1.1.5.1

Number of subjects for this age range:

74

F.1.1.6

Adolescents (12-17 years)

No

F.1.2

Adults (18-64 years)

No

F.1.3

Elderly (>=65 years)

No

F.2 Gender

F.2.1

Female

No

F.2.2

Male

Yes

F.3 Group of trial subjects

F.3.1

Healthy volunteers

No

F.3.2

Patients

Yes

F.3.3

Specific vulnerable populations

Yes

F.3.3.1

Women of childbearing potential not using contraception

No

F.3.3.2

Women of child-bearing potential using contraception

No

F.3.3.3

Pregnant women

No

F.3.3.4

Nursing women

No

F.3.3.5

Emergency situation

No

F.3.3.6

Subjects incapable of giving consent personally

Yes

F.3.3.6.1

Details of subjects incapable of giving consent

Assent only to be provided, where appropriate, due to patient age

F.3.3.7

Others

No

F.4 Planned number of subjects to be included

F.4.1

In the member state

6

F.4.2

For a multinational trial

F.4.2.1

In the EEA

28

F.4.2.2

In the whole clinical trial

74

F.5

Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)

None

G. Investigator Networks to be involved in the Trial

N. Review by the Competent Authority or Ethics Committee in the country concerned

N.

Competent Authority Decision

Authorised

N.

Date of Competent Authority Decision

2021-04-02

N.

Ethics Committee Opinion of the trial application

N.

Ethics Committee Opinion: Reason(s) for unfavourable opinion

N.

Date of Ethics Committee Opinion

P. End of Trial
P.	End of Trial Status

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