Open-label Extension Study to Evaluate the Safety and Tol... | NCT04617860

NCT04617860

Status Module

Sponsor/Collaborators Module

Oversight Module

Description Module

Conditions Module

Design Module

Interventional Study Design

Biospecimen

No data available

No data is available for this block.

Arms/Interventions Module

Label	Type	Description	Intervention Names
WVE-120102 (Dose A)	Experimental		Drug: WVE-120102

Name	Type	Description	Arm Group Labels	Other Names
WVE-120102	Drug	WVE-120102 is a stereopure antisense oligonucleotide. It is administered monthly via intrathecal injection.	WVE-120102 (Dose A)

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Safety: Number of Patients With Treatment-emergent Adverse Events (TEAEs)		Day 1 to Study Termination (maximum of 12 monthly doses)
Safety: Number of Patients With a Severe TEAE		Day 1 to Study Termination (maximum of 12 monthly doses)
Safety: Number of Patients With Serious TEAEs		Day 1 to Study Termination (maximum of 12 monthly doses)
Safety and Tolerability: Number of Patients Who Withdraw Due to TEAEs		Day 1 to Study Termination (maximum of 12 monthly doses)

Eligibility Module

Contacts/Locations Module

Name	Affiliation	Role
Medical Director, MD	WAVE Life Sciences	Study Director

Facility	Status	City	State	ZIP	Country	Contacts
Westmead Hospital		Sydney	New South Wales	2145	Australia
Royal Brisbane & Women's Hospital

References Module

No data available

No data is available for this block.

IPD Sharing Statement Module

No data available

No data is available for this block.

Results Section

Participant Flow Module

ID	Title	Description
FG000	4 mg WVE-120102	Enrolled at 4 mg WVE-120102 dose level
FG001	8 mg WVE-120102	Enrolled at 8 mg WVE-120102 dose level

Baseline Characteristics Module

Outcome Measures Module

Adverse Events Module

More Info Module

Annotation Section

No data available

No data is available for this block.

Document Section

Large Document Module

Uploaded Document Information

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 19, 2020

Derived Section

Miscellaneous Info Module

Submission Tracking

Condition Browse Module

Intervention Browse Module

No data available

No data is available for this block.

Masking Info

Type of Units Analyzed

Arm/Group Information

ID	Title	Description
BG000	4 mg WVE-120102	Enrolled at 4 mg WVE-120102 dose level
BG001	8 mg WVE-120102	Enrolled at 8 mg WVE-120102 dose level
BG002	16 mg WVE-120102	Enrolled at 16 mg WVE-120102 dose level
BG003	Total	Total of all reporting groups

Units	Counts
Participants	BG0008 BG00110 BG00218 BG00336

Baseline Measures

Title

Description

Population Description

Parameter Type

Dispersion Type

Unit of Measure

Calculate Percentage

Denominator Units Selected

Denominators

Classes

Age, Categorical

Count of Participants

Participants

Title

Denominators

Categories

Title	Measurements
<=18 years	BG0000 BG0010 BG0020 BG003

Sex: Female, Male

Count of Participants

Participants

Title

Denominators

Categories

Title	Measurements
Female	BG0003 BG0016 BG002

Ethnicity (NIH/OMB)

Count of Participants

Participants

Title

Denominators

Categories

Title	Measurements
Hispanic or Latino	BG0000 BG0010 BG002

Race (NIH/OMB)

Count of Participants

Participants

Title

Denominators

Categories

Title	Measurements
American Indian or Alaska Native	BG0000 BG0010 BG002

Region of Enrollment

Number

participants

Title

Denominators

Categories

Canada

Title	Measurements
	BG0008 BG0011 BG002

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

Safety: Number of Patients With Treatment-emergent Adverse Events (TEAEs)

Patients treated at more than one dose level (e.g., initial dose and after dose modification) are included in each applicable dose group. Adverse events are counted in the dose the patient was receiving at the time of onset.

Posted

Count of Participants

Participants

Day 1 to Study Termination (maximum of 12 monthly doses)

ID	Title	Description
OG000	4 mg WVE-120102	Received 4 mg WVE-120102 at any point in the study
OG001	8 mg WVE-120102	Received 8 mg WVE-120102 at any point in the study
OG002	16 mg WVE-120102	Received 16 mg WVE-120102 at any point in the study
OG003	32 mg WVE-120102	Received 32 mg WVE-120102 at any point in the study

Units	Counts
Participants	OG0008 OG00118 OG00235 OG003

Title

Denominators

Categories

Title	Measurements
	OG0007 OG00114 OG00234 OG003

Primary

Safety: Number of Patients With a Severe TEAE

Posted

Count of Participants

Participants

Day 1 to Study Termination (maximum of 12 monthly doses)

ID	Title	Description
OG000	4 mg WVE-120102	Received 4 mg WVE-120102 at any point in the study
OG001	8 mg WVE-120102	Received 8 mg WVE-120102 at any point in the study
OG002	16 mg WVE-120102	Received 16 mg WVE-120102 at any point in the study
OG003	32 mg WVE-120102	Received 32 mg WVE-120102 at any point in the study

Units	Counts

Primary

Safety: Number of Patients With Serious TEAEs

Patients treated at more than one dose level (e.g. initial dose and after dose modification) are included in each applicable dose group. Adverse events are counted in the dose the patient was receiving at the time of onset.

Posted

Count of Participants

Participants

Day 1 to Study Termination (maximum of 12 monthly doses)

ID	Title	Description
OG000	4 mg WVE-120102	Received 4 mg WVE-120102 at any point in the study
OG001	8 mg WVE-120102	Received 8 mg WVE-120102 at any point in the study
OG002	16 mg WVE-120102	Received 16 mg WVE-120102 at any point in the study
OG003	32 mg WVE-120102	Received 32 mg WVE-120102 at any point in the study

Units	Counts

Primary

Safety and Tolerability: Number of Patients Who Withdraw Due to TEAEs

Posted

Count of Participants

Participants

Day 1 to Study Termination (maximum of 12 monthly doses)

ID	Title	Description
OG000	4 mg WVE-120102	Received 4 mg WVE-120102 at any point in the study
OG001	8 mg WVE-120102	Received 8 mg WVE-120102 at any point in the study
OG002	16 mg WVE-120102	Received 16 mg WVE-120102 at any point in the study
OG003	32 mg WVE-120102	Received 32 mg WVE-120102 at any point in the study

Units

Arm/Groups

ID	Title	Description	Deaths (At Risk)	Serious Events (Affected)	Serious Events (At Risk)	Other Events (Affected)	Other Events (At Risk)
EG000	4 mg WVE-120102	Received 4 mg WVE-120102 at any point in the study	8	0	8	7	8
EG001	8 mg WVE-120102	Received 8 mg WVE-120102 at any point in the study	18	1	18	14	18
EG002	16 mg WVE-120102	Received 16 mg WVE-120102 at any point in the study	35	5	35	34	35
EG003	32 mg WVE-120102	Received 32 mg WVE-120102 at any point in the study	17	3	17	17	17

Serious Adverse Events

Term	Organ System	Source Vocabulary	Assessment Type	Statistical Information
Pyrexia	General disorders	MedDRA 8.2	Systematic Assessment	EG0000 affected8 at risk EG0010 affected18 at risk EG0021 affected35 at risk EG0030 affected17 at risk
Meningitis	Infections and infestations	MedDRA 8.2	Systematic Assessment	EG0000 affected8 at risk EG0011 affected18 at risk EG0021 affected35 at risk EG003
Meningitis asceptic	Infections and infestations	MedDRA 8.2	Systematic Assessment	EG0000 affected8 at risk EG0010 affected18 at risk EG0022 affected35 at risk EG003
Confusional state	Psychiatric disorders	MedDRA 8.2	Systematic Assessment	EG0000 affected8 at risk EG0010 affected18 at risk EG0020 affected35 at risk EG003
Amnesia	Nervous system disorders	MedDRA 8.2	Systematic Assessment	EG0000 affected8 at risk EG0010 affected18 at risk EG0020 affected35 at risk EG003
Nervous system disorder	Nervous system disorders	MedDRA 8.2	Systematic Assessment	EG0000 affected8 at risk EG0010 affected18 at risk EG0020 affected35 at risk EG003
Aggression	Psychiatric disorders	MedDRA 8.2	Systematic Assessment	EG0000 affected8 at risk EG0010 affected18 at risk EG0021 affected35 at risk EG003
Agitation	Psychiatric disorders	MedDRA 8.2	Systematic Assessment	EG0000 affected8 at risk EG0010 affected18 at risk EG0020 affected35 at risk EG003

Other Adverse Events

Term	Organ System	Source Vocabulary	Assessment Type	Statistical Information
Tinnitus	Ear and labyrinth disorders	MedDRA 8.2	Systematic Assessment	EG0000 affected8 at risk EG0010 affected18 at risk EG0020 affected35 at risk EG0031 affected17 at risk
Nausea	Gastrointestinal disorders	MedDRA 8.2	Systematic Assessment	EG0000 affected8 at risk EG0010 affected18 at risk EG0023 affected35 at risk EG003
Vomiting	Gastrointestinal disorders	MedDRA 8.2	Systematic Assessment	EG0000 affected8 at risk EG0010 affected18 at risk EG0023 affected35 at risk EG003
Dysphagia	Gastrointestinal disorders	MedDRA 8.2	Systematic Assessment	EG0001 affected8 at risk EG0010 affected18 at risk EG0020 affected35 at risk EG003
Paresthesia oral	Gastrointestinal disorders	MedDRA 8.2	Systematic Assessment	EG0001 affected8 at risk EG0010 affected18 at risk EG0020 affected35 at risk EG003
Pyrexia	General disorders	MedDRA 8.2	Systematic Assessment	EG0000 affected8 at risk EG0010 affected18 at risk EG0026 affected35 at risk EG003
Fatigue	General disorders	MedDRA 8.2	Systematic Assessment	EG0000 affected8 at risk EG0010 affected18 at risk EG0026 affected35 at risk EG003
Pain	General disorders	MedDRA 8.2	Systematic Assessment	EG0001 affected8 at risk EG0010 affected18 at risk EG0023 affected35 at risk EG003
Gait disturbance	General disorders	MedDRA 8.2	Systematic Assessment	EG0000 affected8 at risk EG0010 affected18 at risk EG0022 affected35 at risk EG003
Injection site erythema	General disorders	MedDRA 8.2	Systematic Assessment	EG0001 affected8 at risk EG0010 affected18 at risk EG0020 affected35 at risk EG003
Injection site hypersensitivity	General disorders	MedDRA 8.2	Systematic Assessment	EG0000 affected8 at risk EG0010 affected18 at risk EG0020 affected35 at risk EG003
Nasopharyngitis	Infections and infestations	MedDRA 8.2	Systematic Assessment	EG0000 affected8 at risk EG0010 affected18 at risk EG0022 affected35 at risk EG003
Cystitis	Infections and infestations	MedDRA 8.2	Systematic Assessment	EG0000 affected8 at risk EG0011 affected18 at risk EG0020 affected35 at risk EG003
Oral candidiasis	Infections and infestations	MedDRA 8.2	Systematic Assessment	EG0000 affected8 at risk EG0011 affected18 at risk EG0020 affected35 at risk EG003
Upper respiratory tract infection	Infections and infestations	MedDRA 8.2	Systematic Assessment	EG0001 affected8 at risk EG0010 affected18 at risk EG0020 affected35 at risk EG003
Urinary tract infection	Infections and infestations	MedDRA 8.2	Systematic Assessment	EG0000 affected8 at risk EG0011 affected18 at risk EG0020 affected35 at risk EG003
Fall	Injury, poisoning and procedural complications	MedDRA 8.2	Systematic Assessment	EG0002 affected8 at risk EG0013 affected18 at risk EG0025 affected35 at risk EG003
Procedural pain	Injury, poisoning and procedural complications	MedDRA 8.2	Systematic Assessment	EG0002 affected8 at risk EG0010 affected18 at risk EG0024 affected35 at risk EG003
Procedural headache	Injury, poisoning and procedural complications	MedDRA 8.2	Systematic Assessment	EG0001 affected8 at risk EG0010 affected18 at risk EG0022 affected35 at risk EG003
Skin laceration	Injury, poisoning and procedural complications	MedDRA 8.2	Systematic Assessment	EG0001 affected8 at risk EG0010 affected18 at risk EG0021 affected35 at risk EG003
Contusion	Injury, poisoning and procedural complications	MedDRA 8.2	Systematic Assessment	EG0001 affected8 at risk EG0010 affected18 at risk EG0020 affected35 at risk EG003
Post procedural discomfort	Injury, poisoning and procedural complications	MedDRA 8.2	Systematic Assessment	EG0000 affected8 at risk EG0011 affected18 at risk EG0020 affected35 at risk EG003
Procedural dizziness	Injury, poisoning and procedural complications	MedDRA 8.2	Systematic Assessment	EG0001 affected8 at risk EG0010 affected18 at risk EG0020 affected35 at risk EG003
Scapula fracture	Injury, poisoning and procedural complications	MedDRA 8.2	Systematic Assessment	EG0000 affected8 at risk EG0011 affected18 at risk EG0020 affected35 at risk EG003
CSF protein increased	Investigations	MedDRA 8.2	Systematic Assessment	EG0000 affected8 at risk EG0013 affected18 at risk EG0027 affected35 at risk EG003
CSF lymphocyte count increased	Investigations	MedDRA 8.2	Systematic Assessment	EG0000 affected8 at risk EG0012 affected18 at risk EG0025 affected35 at risk EG003
CSF test abnormal	Investigations	MedDRA 8.2	Systematic Assessment	EG0000 affected8 at risk EG0010 affected18 at risk EG0020 affected35 at risk EG003
Blood bilirubin increased	Investigations	MedDRA 8.2	Systematic Assessment	EG0000 affected8 at risk EG0011 affected18 at risk EG0020 affected35 at risk EG003
CSF white blood cell count	Investigations	MedDRA 8.2	Systematic Assessment	EG0000 affected8 at risk EG0010 affected18 at risk EG0022 affected35 at risk EG003
Lymphocyte count increased	Investigations	MedDRA 8.2	Systematic Assessment	EG0000 affected8 at risk EG0010 affected18 at risk EG0022 affected35 at risk EG003
Complement factor increased	Investigations	MedDRA 8.2	Systematic Assessment	EG0000 affected8 at risk EG0010 affected18 at risk EG0020 affected35 at risk EG003
Dermatologic examination abnormal	Investigations	MedDRA 8.2	Systematic Assessment	EG0000 affected8 at risk EG0010 affected18 at risk EG0020 affected35 at risk EG003
Neurological examination abnormal	Investigations	MedDRA 8.2	Systematic Assessment	EG0000 affected8 at risk EG0010 affected18 at risk EG0020 affected35 at risk EG003
Decreased appetite	Metabolism and nutrition disorders	MedDRA 8.2	Systematic Assessment	EG0000 affected8 at risk EG0010 affected18 at risk EG0021 affected35 at risk EG003
Increased appetite	Metabolism and nutrition disorders	MedDRA 8.2	Systematic Assessment	EG0000 affected8 at risk EG0010 affected18 at risk EG0020 affected35 at risk EG003
Polydipsia	Metabolism and nutrition disorders	MedDRA 8.2	Systematic Assessment	EG0000 affected8 at risk EG0010 affected18 at risk EG0020 affected35 at risk EG003
Back pain	Musculoskeletal and connective tissue disorders	MedDRA 8.2	Systematic Assessment	EG0001 affected8 at risk EG0011 affected18 at risk EG0026 affected35 at risk EG003
Pain in extremity	Musculoskeletal and connective tissue disorders	MedDRA 8.2	Systematic Assessment	EG0000 affected8 at risk EG0010 affected18 at risk EG0025 affected35 at risk EG003
Muscular weakness	Musculoskeletal and connective tissue disorders	MedDRA 8.2	Systematic Assessment	EG0000 affected8 at risk EG0010 affected18 at risk EG0022 affected35 at risk EG003
Neck pain	Musculoskeletal and connective tissue disorders	MedDRA 8.2	Systematic Assessment	EG0000 affected8 at risk EG0010 affected18 at risk EG0021 affected35 at risk EG003
Myalgia	Musculoskeletal and connective tissue disorders	MedDRA 8.2	Systematic Assessment	EG0000 affected8 at risk EG0010 affected18 at risk EG0022 affected35 at risk EG003
Headache	Nervous system disorders	MedDRA 8.2	Systematic Assessment	EG0002 affected8 at risk EG0016 affected18 at risk EG00214 affected35 at risk EG003
Dizziness	Nervous system disorders	MedDRA 8.2	Systematic Assessment	EG0000 affected8 at risk EG0010 affected18 at risk EG0024 affected35 at risk EG003
Amnesia	Nervous system disorders	MedDRA 8.2	Systematic Assessment	EG0000 affected8 at risk EG0010 affected18 at risk EG0020 affected35 at risk EG003
Ataxia	Nervous system disorders	MedDRA 8.2	Systematic Assessment	EG0000 affected8 at risk EG0010 affected18 at risk EG0020 affected35 at risk EG003
Balance disorder	Nervous system disorders	MedDRA 8.2	Systematic Assessment	EG0000 affected8 at risk EG0010 affected18 at risk EG0020 affected35 at risk EG003
Dyskinesia	Nervous system disorders	MedDRA 8.2	Systematic Assessment	EG0001 affected8 at risk EG0010 affected18 at risk EG0021 affected35 at risk EG003
Memory impairment	Nervous system disorders	MedDRA 8.2	Systematic Assessment	EG0000 affected8 at risk EG0010 affected18 at risk EG0022 affected35 at risk EG003
Presyncope	Nervous system disorders	MedDRA 8.2	Systematic Assessment	EG0001 affected8 at risk EG0010 affected18 at risk EG0020 affected35 at risk EG003
Somnolence	Nervous system disorders	MedDRA 8.2	Systematic Assessment	EG0000 affected8 at risk EG0010 affected18 at risk EG0020 affected35 at risk EG003
Disturbance in attention	Nervous system disorders	MedDRA 8.2	Systematic Assessment	EG0000 affected8 at risk EG0011 affected18 at risk EG0020 affected35 at risk EG003
Dysarthria	Nervous system disorders	MedDRA 8.2	Systematic Assessment	EG0000 affected8 at risk EG0010 affected18 at risk EG0020 affected35 at risk EG003
Hypotonia	Nervous system disorders	MedDRA 8.2	Systematic Assessment	EG0000 affected8 at risk EG0010 affected18 at risk EG0020 affected35 at risk EG003
Migraine	Nervous system disorders	MedDRA 8.2	Systematic Assessment	EG0000 affected8 at risk EG0010 affected18 at risk EG0020 affected35 at risk EG003
Confusional state	Psychiatric disorders	MedDRA 8.2	Systematic Assessment	EG0000 affected8 at risk EG0010 affected18 at risk EG0023 affected35 at risk EG003
Apathy	Psychiatric disorders	MedDRA 8.2	Systematic Assessment	EG0000 affected8 at risk EG0011 affected18 at risk EG0021 affected35 at risk EG003
Behavior disorder	Psychiatric disorders	MedDRA 8.2	Systematic Assessment	EG0000 affected8 at risk EG0010 affected18 at risk EG0022 affected35 at risk EG003
Insomnia	Psychiatric disorders	MedDRA 8.2	Systematic Assessment	EG0000 affected8 at risk EG0011 affected18 at risk EG0021 affected35 at risk EG003
Irritability	Psychiatric disorders	MedDRA 8.2	Systematic Assessment	EG0000 affected8 at risk EG0012 affected18 at risk EG0020 affected35 at risk EG003
Restlessness	Psychiatric disorders	MedDRA 8.2	Systematic Assessment	EG0000 affected8 at risk EG0010 affected18 at risk EG0022 affected35 at risk EG003
Hallucination, visual	Psychiatric disorders	MedDRA 8.2	Systematic Assessment	EG0000 affected8 at risk EG0011 affected18 at risk EG0020 affected35 at risk EG003
Paranoia	Psychiatric disorders	MedDRA 8.2	Systematic Assessment	EG0000 affected8 at risk EG0010 affected18 at risk EG0020 affected35 at risk EG003
Persecutory delusion	Psychiatric disorders	MedDRA 8.2	Systematic Assessment	EG0000 affected8 at risk EG0011 affected18 at risk EG0020 affected35 at risk EG003
Urinary retention	Renal and urinary disorders	MedDRA 8.2	Systematic Assessment	EG0000 affected8 at risk EG0010 affected18 at risk EG0020 affected35 at risk EG003
Breast tenderness	Reproductive system and breast disorders	MedDRA 8.2	Systematic Assessment	EG0000 affected8 at risk EG0011 affected18 at risk EG0020 affected35 at risk EG003
Oropharyngeal pain	Respiratory, thoracic and mediastinal disorders	MedDRA 8.2	Systematic Assessment	EG0000 affected8 at risk EG0011 affected18 at risk EG0023 affected35 at risk EG003
Cough	Respiratory, thoracic and mediastinal disorders	MedDRA 8.2	Systematic Assessment	EG0000 affected8 at risk EG0011 affected18 at risk EG0020 affected35 at risk EG003
Dysnpoea	Respiratory, thoracic and mediastinal disorders	MedDRA 8.2	Systematic Assessment	EG0001 affected8 at risk EG0010 affected18 at risk EG0020 affected35 at risk EG003
Erythema	Skin and subcutaneous tissue disorders	MedDRA 8.2	Systematic Assessment	EG0000 affected8 at risk EG0011 affected18 at risk EG0020 affected35 at risk EG003
Hypotension	Vascular disorders	MedDRA 8.2	Systematic Assessment	EG0000 affected8 at risk EG0010 affected18 at risk EG0020 affected35 at risk EG003

First MCP Posted Date

ID	Term
D006816	Huntington Disease

Ancestor Terms

ID	Term
D001480	Basal Ganglia Diseases
D001927	Brain Diseases
D002493	Central Nervous System Diseases
D009422	Nervous System Diseases
D003704	Dementia
D002819	Chorea
D020820	Dyskinesias
D009069	Movement Disorders
D020271	Heredodegenerative Disorders, Nervous System
D019636	Neurodegenerative Diseases
D030342	Genetic Diseases, Inborn
D009358	Congenital, Hereditary, and Neonatal Diseases and Abnormalities
D003072	Cognition Disorders
D019965	Neurocognitive Disorders
D001523	Mental Disorders