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Lack of efficacy
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WVE-HDSNP1-002 is an open-label extension (OLE) study to evaluate the safety, tolerability, PK, PD, and clinical effects of WVE-120101 in adult patients with early manifest HD who carry a targeted single nucleotide polymorphism, rs362307 (SNP1). To participate in the study, patients must have completed the Phase 1b/2a clinical study WVE-HDSNP1-001.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| WVE-120101 (Dose A) | Experimental |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| WVE-120101 | Drug | WVE-120101 is a stereopure antisense oligonucleotide (ASO). It is administered monthly via intrathecal injection. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Safety: Number of Patients With Treatment-emergent AEs (TEAEs) | First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment) | |
| Safety: Number of Patients With a Severe TEAE | First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment | |
| Safety: Number of Patients With Serious TEAEs | First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment | |
| Safety and Tolerability: Number of Patients Who Withdraw Due to TEAEs | First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment |
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Key Inclusion Criteria:
Key Exclusion Criteria:
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| 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 |
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In the original OLE study protocol, patients who were in the 2 and 4 mg dose cohorts in the Phase 1b/2a study were to be enrolled into the 4 or 8 mg dose group in this study. Patients in the 8 and 16 mg dose cohorts in WVE-HDSNP1-001 continued to receive that dose. As of Amendment 1.0 of this protocol, all new patients were to be enrolled at a dose level of at least 16 mg. All current patents were dose modified to 16 or 32 mg.
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| ID | Title | Description |
|---|---|---|
| FG000 | 4 mg WVE-120101 | Enrolled at 4 mg WVE-120101 dose level |
| FG001 | 16 mg WVE-120101 | Enrolled at 16 mg WVE-120101 dose level |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| 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 31, 2020 |
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| Herston |
| Queensland |
| QLD 4006 |
| Australia |
| Royal Melbourne Hospital | Carlton | Victoria | 3053 | Australia |
| Monash Health | Clayton | Victoria | 3168 | Australia |
| Alfred Health | Melbourne | Victoria | 3004 | Australia |
| Calvary Health Care Bethlehem | Parkdale | Victoria | 3195 | Australia |
| North Metropolitan Health Service | Perth | Western Australia | 6910 | Australia |
| University of Alberta | Edmonton | Alberta | T6G 2B7 | Canada |
| Centre Hospitalier de l-Universite de Montreal | Montreal | Quebec | H2X019 | Canada |
| Aarhus Universitets Hospital | Aarhus | 8200 | Denmark |
| Rigshospitalet | Copenhagen | 2100 | Denmark |
| Hospital Henri Mondor | Créteil | 94010 | France |
| Institut du Cerveau et de la Moelle Epinière | Paris | 75646 | France |
| George-Huntington-Institut GmbH | Münster | 48149 | Germany |
| Szpital Sw. Wojciecha | Gdansk | 80-462 | Poland |
| Instytut Psychiatrii i Neurologii | Warsaw | 02-957 | Poland |
| Royal Devon and Exeter Hospital NHS Trust | Exeter | Devon | EX2 5DW | United Kingdom |
| Queen Elizabeth University Hospital - PPDS | Glasgow | Glasgow City | G12 0XH | United Kingdom |
| Dose Modified to 16 mg WVE-120101 |
|
| Dose Modified to 32 mg WVE-120101 |
|
| COMPLETED |
|
| NOT COMPLETED |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | 4 mg WVE-120101 | Enrolled at 4 mg WVE-120101 dose level |
| BG001 | 16 mg WVE-120101 | Enrolled at 12 mg WVE-120101 dose level |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Demographics at the time of enrollment in the WVE-HDSNP1-001 study. | Count of Participants | Participants |
| |||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||||
| Region of Enrollment | Number | participants |
|
| 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 AEs (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 | First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment) |
|
|
| |||||||||||||||||||||||||||||||||
| Primary | Safety: Number of Patients With a Severe TEAE | 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 | First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment |
|
| ||||||||||||||||||||||||||||||||||
| 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 | First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment |
|
| ||||||||||||||||||||||||||||||||||
| Primary | Safety and Tolerability: Number of Patients Who Withdraw Due to 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 | First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment |
|
|
First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment)
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | 4 mg WVE-120101 | Patients who received 4 mg WVE-120101 at any point in the study | 0 | 3 | 0 | 3 | 2 | 3 |
| EG001 | 16 mg WVE-120101 | Patients who received 16 mg WVE-120101 at any point in the study | 2 | 27 | 3 | 27 | 16 | 27 |
| EG002 | 32 mg WVE-120101 | Patients who received 32 mg WVE-120101 at any point in the study | 0 | 5 | 1 | 5 | 2 | 5 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Lung Cancer Metastatic | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | Systematic Assessment |
| ||
| Neoplasm | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | Systematic Assessment |
| ||
| Balance disorder | Nervous system disorders | Systematic Assessment |
| ||
| Headache | Nervous system disorders | Systematic Assessment |
| ||
| Head Injury | Injury, poisoning and procedural complications | Systematic Assessment |
| ||
| Completed Suicide | Psychiatric disorders | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Fall | Injury, poisoning and procedural complications | Systematic Assessment |
| ||
| Procedural Pain | Injury, poisoning and procedural complications | Systematic Assessment |
| ||
| CSF Protein Increased | Investigations | Systematic Assessment |
| ||
| CSF white blood cell count increased | Investigations | Systematic Assessment |
| ||
| Lymphocyte count increased | Investigations | Systematic Assessment |
| ||
| Back pain | Musculoskeletal and connective tissue disorders | Systematic Assessment |
| ||
| Pain in extremity | Musculoskeletal and connective tissue disorders | Systematic Assessment |
| ||
| Dizziness | Nervous system disorders | Systematic Assessment |
| ||
| Headache | Nervous system disorders | Systematic Assessment |
| ||
| Dysarthria | Nervous system disorders | Systematic Assessment |
| ||
| Restless legs syndrome | Nervous system disorders | Systematic Assessment |
| ||
| Balance disorder | Nervous system disorders | Systematic Assessment |
|
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Medical Director | Wave Life Sciences | 855-215-4687 | clinicaltrials@wavelifesci.com |
| Dec 13, 2021 |
| Prot_SAP_000.pdf |
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| ID | Term |
|---|---|
| D006816 | Huntington Disease |
| 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 |
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| >=65 years |
|
| Male |
|
| Not Hispanic or Latino |
|
| Unknown or Not Reported |
|
| Asian |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
| Denmark |
|
| Poland |
|
| Australia |
|
| France |
|
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|
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