Study to Assess the Safety, Tolerability, and Efficacy of... | NCT04956289

Status Module

Name	Class
Nippon Shinyaku Co., Ltd.	INDUSTRY

Label	Type	Description	Intervention Names
Viltolarsen	Experimental	Patients amenable to exon 53 skipping will receive viltolarsen intravenous (IV) infusions, weekly, at 80 mg/kg for up to 48 weeks.	Drug: Viltolarsen

Name	Type	Description	Arm Group Labels	Other Names
Viltolarsen	Drug	Received during weekly intravenous infusions	Viltolarsen

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Number of Participants With Treatment Emergent Adverse Events	No statistical analysis was performed for this endpoint. The information has been introduced in the section "Adverse Events".	baseline to up to 48 weeks of treatment
Number of Participants With Treatment Emergent Adverse Events by Maximum Severity	No statistical analysis was performed for this endpoint. The information has been introduced in the section "Adverse Events".	baseline to up to 48 weeks of treatment
Number of Participants With Treatment Emergent Adverse Events by Highest Intervention Level Regarding Investigational Product	No statistical analysis was performed for this endpoint. The information has been introduced in the section "Adverse Events".	baseline to up to 48 weeks of treatment
Number of Participants With Treatment Emergent Adverse Events by Worst Outcome	No statistical analysis was performed for this endpoint. The information has been introduced in the section "Adverse Events".	baseline to up to 48 weeks of treatment
Number of Participants With Investigational Product-related Treatment Emergent Adverse Events	No statistical analysis was performed for this endpoint. The information has been introduced in the section "Adverse Events".	baseline to up to 48 weeks of treatment
Number of Participants With Investigational Product-related Treatment Emergent Adverse Events by Maximum Severity	No statistical analysis was performed for this endpoint. The information has been introduced in the section "Adverse Events".

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

Patient (if age 18 years or older) or 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 younger than age 18 years will be asked to give written or verbal assent according to local requirements;
Patient has a confirmed diagnosis of DMD defined as:
1. Patient is male with clinical signs compatible with DMD; and
2. Patient has a confirmed DMD mutation(s) in the dystrophin gene that is amenable to skipping of exon 53 to restore the dystrophin messenger ribonucleic acid reading frame including determination of unambiguously defined exon boundaries (using techniques such as multiplex ligation-dependent probe amplification, comparative genomic hybridization array, or other techniques with similar capability);
Patient is more than 8 years of age at time of first infusion in the study;
Patient has a Brooke scale rating of 3 or better OR an upright FVC 30% or greater at Screening;
Patient, if sexually active, is willing to abstain from sexual intercourse or employ a barrier or medical method of contraception during and for 3 months following completion of IP administration;
Patient and patient's parent(s)/guardian(s) (if patient is <18 years of age) and/or caregiver(s) are willing and able to comply with scheduled visits, IP administration plan, and study procedures;
Patient must be on a stable dose of glucocorticoid (GC) or not treated with GC for at least 3 months prior to the first dose of IP and is expected to remain on stable dose of GC treatment or off GC for the duration of the study.

Other inclusion criteria may apply

Exclusion Criteria:

Patient has had an acute illness within 4 weeks prior to the first dose of IP;
Patient has evidence of symptomatic cardiomyopathy (New York Heart Association Class III or higher);
Patient requires ventilation support while awake during the day;
Patient has an allergy or hypersensitivity to IP or any of its constituents;
Patient has severe behavioral or cognitive problems that preclude participation in the study, in the opinion of the investigator;
Patient has a previous or ongoing medical condition, medical history, physical findings, or laboratory abnormalities that could affect patient 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 has had surgery within 3 months prior to the first anticipated administration of IP or has known plans to have surgery during the Treatment Period;
Patient has positive test results for hepatitis B antigen, hepatitis C antibody, or human immunodeficiency virus antibody at Screening;
Patient has been diagnosed with asthma that requires chronic treatment with a long-acting beta agonist;
Patient has relevant history of or current drug or alcohol abuse or use of any tobacco/marijuana products by smoking or vaping within 3 months prior to treatment with IP;
Patient is currently taking any other investigational drug or has taken any other investigational drug within 3 months prior to the first dose of IP or within 5 times the half-life of a medication, whichever is longer;
Patient has taken any gene therapy;
Patient is currently taking any other exon skipping agent or has taken any other exon skipping agent within 3 months prior to the first dose of IP;
Patient has hydronephrosis, hydroureter, renal or urinary tract calculi, or ureteral stenosis by renal ultrasound;
Patient was previously enrolled in an interventional study of viltolarsen.

Other exclusion criteria may apply

Facility	Status	City	State	ZIP	Country	Contacts
Children's Hospital of Richmond at VCU		Richmond	Virginia	23219	United States
The Third Medical Center of PLA General Hospital

Results Section

Participant Flow Module

Arm/Group Information

ID	Title	Description
FG000	Viltolarsen 80mg/kg	Participants who confirmed DMD with genetic deletions amenable to exon 53 skipping were administered an intravenous infusion of Viltolarsen 80 mg/kg once a week for up to 48 weeks.

Baseline Characteristics Module

Outcome Measures Module

Adverse Events Module

More Info Module

Type	Includes Protocol	Includes SAP	Includes ICF	Document Label	Document Date	Document Uploaded Date	Document File Name
Prot_SAP	Yes	Yes	No	Study Protocol and Statistical Analysis Plan	Mar 21, 2022

ID	Title	Description
BG000	Viltolarsen 80mg/kg	Participants who confirmed DMD with genetic deletions amenable to exon 53 skipping were administered an intravenous infusion of Viltolarsen 80 mg/kg once a week for up to 48 weeks.

Units	Counts
Participants	BG00020

Type

Title

Description

Population Description

Reporting Status

Anticipated Posting Date

Parameter Type

Dispersion Type

Unit of Measure

Calculate Percentage

Time Frame

Units Analyzed

Denominator Units Selected

Arm/Group Information

Denominators

Classes

Analyses

Primary

Number of Participants With Treatment Emergent Adverse Events

No statistical analysis was performed for this endpoint. The information has been introduced in the section "Adverse Events".

The Safety Population consisted of all patients who received at least 1 dose of IP (Investigational Product). This was the primary analysis population for the evaluation of safety. It includes all patients treated with viltolarsen both ambulant (10) and non-ambulant (10).

Posted

Count of Participants

Participants

baseline to up to 48 weeks of treatment

ID	Title	Description
OG000	Viltolarsen 80mg/kg	Participants who confirmed DMD with genetic deletions amenable to exon 53 skipping were administered an intravenous infusion of Viltolarsen 80 mg/kg once a week for up to 48 weeks.

Units	Counts
Participants	OG00020

Title

Denominators

Categories

Title	Measurements
	OG00019

Primary

Number of Participants With Treatment Emergent Adverse Events by Maximum Severity

No statistical analysis was performed for this endpoint. The information has been introduced in the section "Adverse Events".

Posted

Count of Participants

Participants

baseline to up to 48 weeks of treatment

ID	Title	Description
OG000	Viltolarsen 80mg/kg	Participants who confirmed DMD with genetic deletions amenable to exon 53 skipping were administered an intravenous infusion of Viltolarsen 80 mg/kg once a week for up to 48 weeks.

Units	Counts
Participants	OG000

Primary

Number of Participants With Treatment Emergent Adverse Events by Highest Intervention Level Regarding Investigational Product

No statistical analysis was performed for this endpoint. The information has been introduced in the section "Adverse Events".

Posted

Count of Participants

Participants

baseline to up to 48 weeks of treatment

ID	Title	Description
OG000	Viltolarsen 80mg/kg	Participants who confirmed DMD with genetic deletions amenable to exon 53 skipping were administered an intravenous infusion of Viltolarsen 80 mg/kg once a week for up to 48 weeks.

Units	Counts
Participants	OG000

Primary

Number of Participants With Treatment Emergent Adverse Events by Worst Outcome

No statistical analysis was performed for this endpoint. The information has been introduced in the section "Adverse Events".

Posted

Count of Participants

Participants

baseline to up to 48 weeks of treatment

ID	Title	Description
OG000	Viltolarsen 80mg/kg	Participants who confirmed DMD with genetic deletions amenable to exon 53 skipping were administered an intravenous infusion of Viltolarsen 80 mg/kg once a week for up to 48 weeks.

Units	Counts
Participants	OG000

Primary

Number of Participants With Investigational Product-related Treatment Emergent Adverse Events

No statistical analysis was performed for this endpoint. The information has been introduced in the section "Adverse Events".

Posted

Count of Participants

Participants

baseline to up to 48 weeks of treatment

ID	Title	Description
OG000	Viltolarsen 80mg/kg	Participants who confirmed DMD with genetic deletions amenable to exon 53 skipping were administered an intravenous infusion of Viltolarsen 80 mg/kg once a week for up to 48 weeks.

Units	Counts
Participants	OG000

Primary

Number of Participants With Investigational Product-related Treatment Emergent Adverse Events by Maximum Severity

No statistical analysis was performed for this endpoint. The information has been introduced in the section "Adverse Events".

Posted

Count of Participants

Participants

baseline to up to 48 weeks of treatment

ID	Title	Description
OG000	Viltolarsen 80mg/kg	Participants who confirmed DMD with genetic deletions amenable to exon 53 skipping were administered an intravenous infusion of Viltolarsen 80 mg/kg once a week for up to 48 weeks.

Units	Counts
Participants	OG000

Primary

Number of Participants With Investigational Product-related Treatment Emergent Adverse Events by Highest Intervention Level Regarding Investigational Product

No statistical analysis was performed for this endpoint. The information has been introduced in the section "Adverse Events".

Posted

Count of Participants

Participants

baseline to up to 48 weeks of treatment

ID	Title	Description
OG000	Viltolarsen 80mg/kg	Participants who confirmed DMD with genetic deletions amenable to exon 53 skipping were administered an intravenous infusion of Viltolarsen 80 mg/kg once a week for up to 48 weeks.

Units	Counts
Participants	OG000

Primary

Number of Participants With Investigational Product-related Treatment Emergent Adverse Events by Worst Outcome

No statistical analysis was performed for this endpoint. The information has been introduced in the section "Adverse Events".

Posted

Count of Participants

Participants

baseline to up to 48 weeks of treatment

ID	Title	Description
OG000	Viltolarsen 80mg/kg	Participants who confirmed DMD with genetic deletions amenable to exon 53 skipping were administered an intravenous infusion of Viltolarsen 80 mg/kg once a week for up to 48 weeks.

Units	Counts
Participants	OG000

Primary

Number of Participants With Adverse Event of Special Interest

No statistical analysis was performed for this endpoint. The information has been introduced in the section "Adverse Events".

Posted

Count of Participants

Participants

baseline to up to 48 weeks of treatment

ID	Title	Description
OG000	Viltolarsen 80mg/kg	Participants who confirmed DMD with genetic deletions amenable to exon 53 skipping were administered an intravenous infusion of Viltolarsen 80 mg/kg once a week for up to 48 weeks.

Units	Counts
Participants	OG000

Other Adverse Events

Term	Organ System	Source Vocabulary	Assessment Type	Statistical Information
COVID-19	Infections and infestations	MedDRA version 26.0	Systematic Assessment	EG0006 events6 affected20 at risk
Nasopharyngitis	Infections and infestations	MedDRA version 26.0	Systematic Assessment	EG0003 events3 affected20 at risk
Upper respiratory tract infection	Infections and infestations	MedDRA version 26.0	Systematic Assessment	EG0003 events3 affected20 at risk
Influenza	Infections and infestations	MedDRA version 26.0	Systematic Assessment	EG0002 events2 affected20 at risk
Rhinitis	Infections and infestations	MedDRA version 26.0	Systematic Assessment	EG0002 events2 affected20 at risk
Otitis media	Infections and infestations	MedDRA version 26.0	Systematic Assessment	EG0001 events1 affected20 at risk
Pharyngitis	Infections and infestations	MedDRA version 26.0	Systematic Assessment	EG0001 events1 affected20 at risk
Diarrhoea	Gastrointestinal disorders	MedDRA version 26.0	Systematic Assessment	EG00013 events5 affected20 at risk
Food poisoning	Gastrointestinal disorders	MedDRA version 26.0	Systematic Assessment	EG0008 events2 affected20 at risk
Abdominal pain upper	Gastrointestinal disorders	MedDRA version 26.0	Systematic Assessment	EG0002 events2 affected20 at risk
Toothache	Gastrointestinal disorders	MedDRA version 26.0	Systematic Assessment	EG0001 events1 affected20 at risk
Vomiting	Gastrointestinal disorders	MedDRA version 26.0	Systematic Assessment	EG0001 events1 affected20 at risk
Headache	Nervous system disorders	MedDRA version 26.0	Systematic Assessment	EG0004 events4 affected20 at risk
Tension headache	Nervous system disorders	MedDRA version 26.0	Systematic Assessment	EG0002 events1 affected20 at risk
Pyrexia	General disorders	MedDRA version 26.0	Systematic Assessment	EG0003 events2 affected20 at risk
Gait inability	General disorders	MedDRA version 26.0	Systematic Assessment	EG0001 events1 affected20 at risk
Non-cardiac chest pain	General disorders	MedDRA version 26.0	Systematic Assessment	EG0001 events1 affected20 at risk
Joint injury	Injury, poisoning and procedural complications	MedDRA version 26.0	Systematic Assessment	EG0002 events2 affected20 at risk
Contusion	Injury, poisoning and procedural complications	MedDRA version 26.0	Systematic Assessment	EG0001 events1 affected20 at risk
Ligament sprain	Injury, poisoning and procedural complications	MedDRA version 26.0	Systematic Assessment	EG0001 events1 affected20 at risk
Haematuria	Renal and urinary disorders	MedDRA version 26.0	Systematic Assessment	EG0006 events4 affected20 at risk
Nephrolithiasis	Renal and urinary disorders	MedDRA version 26.0	Systematic Assessment	EG0001 events1 affected20 at risk
Pain in extremity	Musculoskeletal and connective tissue disorders	MedDRA version 26.0	Systematic Assessment	EG0002 events2 affected20 at risk
Back pain	Musculoskeletal and connective tissue disorders	MedDRA version 26.0	Systematic Assessment	EG0001 events1 affected20 at risk
Myalgia	Musculoskeletal and connective tissue disorders	MedDRA version 26.0	Systematic Assessment	EG0001 events1 affected20 at risk
Angina pectoris	Cardiac disorders	MedDRA version 26.0	Systematic Assessment	EG0001 events1 affected20 at risk
Tachycardia	Cardiac disorders	MedDRA version 26.0	Systematic Assessment	EG0001 events1 affected20 at risk
Protein urine	Investigations	MedDRA version 26.0	Systematic Assessment	EG0001 events1 affected20 at risk
Urine cytology abnormal	Investigations	MedDRA version 26.0	Systematic Assessment	EG0001 events1 affected20 at risk
Nasal congestion	Respiratory, thoracic and mediastinal disorders	MedDRA version 26.0	Systematic Assessment	EG0001 events1 affected20 at risk
Oropharyngeal pain	Respiratory, thoracic and mediastinal disorders	MedDRA version 26.0	Systematic Assessment	EG0001 events1 affected20 at risk
Blister	Skin and subcutaneous tissue disorders	MedDRA version 26.0	Systematic Assessment	EG0001 events1 affected20 at risk
Rash	Skin and subcutaneous tissue disorders	MedDRA version 26.0	Systematic Assessment	EG0001 events1 affected20 at risk
Drug hypersensitivity	Immune system disorders	MedDRA version 26.0	Systematic Assessment	EG0001 events1 affected20 at risk
Hypertension	Vascular disorders	MedDRA version 26.0	Systematic Assessment	EG0001 events1 affected20 at risk

ID	Term
D009136	Muscular Dystrophies
D020966	Muscular Disorders, Atrophic
D009135	Muscular Diseases
D009140	Musculoskeletal Diseases
D009468	Neuromuscular Diseases
D009422	Nervous System Diseases
D040181	Genetic Diseases, X-Linked
D030342	Genetic Diseases, Inborn
D009358	Congenital, Hereditary, and Neonatal Diseases and Abnormalities