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This is a Phase 1b/2a multicenter, randomized, double-blind, placebo-controlled study to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of WVE-003 in adult patients with early-manifest HD who carry the targeted single nucleotide polymorphism (SNP) - SNP3.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| SAD: Pooled Placebo | Placebo Comparator | Placebo |
|
| SAD: 30mg WVE-003 | Experimental | Single Ascending Dose - 30mg WVE-003 |
|
| SAD: 60mg WVE-003 | Experimental | Single Ascending Dose - 60mg WVE-003 |
|
| SAD: 90mg WVE-003 | Experimental | Single Ascending Dose - 90mg WVE-003 |
|
| MD: Placebo | Placebo Comparator | Placebo |
|
| MD: 30mg WVE-003 | Experimental | Multiple Dose - 30mg WVE-003 |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| SAD: 30mg WVE-003 | Drug | Single ascending dose of 30mg WVE-003, an allele-selective stereopure antisense oligonucleotide (ASO) |
|
| Measure | Description | Time Frame |
|---|---|---|
| Safety: Proportion of Patients With Treatment Emergent Adverse Events (TEAEs) Related to Study Drug | The primary outcome for this study was safety and is reported as the proportion of patients with TEAEs related to study drug. | Day 1 through Week 24 (single ascending dose Period 1); Day 1 through Week 28 (multi dose Period 2) |
| Measure | Description | Time Frame |
|---|---|---|
| Pharmacokinetics of WVE-003 in Plasma | Parameter analyzed: AUC0-6 = area under the concentration-time curve from time 0 to 6 hrs | Day 1 (single ascending dose Period 1); Day 1 and Day 113 (multi dose Period 2) |
| Pharmacokinetics of WVE-003 in Plasma |
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Inclusion Criteria:
Exclusion Criteria:
Malignancy or received treatment for malignancy, other than treated basal cell or squamous cell carcinoma of the skin, within the previous 5 years
Received any other study drug, including an investigational oligonucleotide, within the past 1 year or 5 half-lives of the drug, whichever is longer, with the exception of the following:
a. Received WVE-120101 or WVE-120102 within the last 3 months
Implantable CNS device that may interfere with ability to administer study drug via lumbar puncture or undergo MRI scan
Inability to undergo brain MRI (with or without sedation)
Bone, spine, bleeding, or other disorder that exposes the patient to risk of injury or unsuccessful lumbar puncture
Previously received tominersen
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| Name | Affiliation | Role |
|---|---|---|
| Medical Director, MD | Wave Life Sciences | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Westmead Hospital | Westmead | New South Wales | 2145 | Australia | ||
| Monash Health |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 39027419 | Derived | Iwamoto N, Liu Y, Frank-Kamenetsky M, Maguire A, Tseng WC, Taborn K, Kothari N, Akhtar A, Bowman K, Shelke JD, Lamattina A, Hu XS, Jang HG, Kandasamy P, Liu F, Longo K, Looby R, Meena, Metterville J, Pan Q, Purcell-Estabrook E, Shimizu M, Prakasha PS, Standley S, Upadhyay H, Yang H, Yin Y, Zhao A, Francis C, Byrne M, Dale E, Verdine GL, Vargeese C. Preclinical evaluation of stereopure antisense oligonucleotides for allele-selective lowering of mutant HTT. Mol Ther Nucleic Acids. 2024 Jun 11;35(3):102246. doi: 10.1016/j.omtn.2024.102246. eCollection 2024 Sep 10. | |
| 36463457 |
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| ID | Title | Description |
|---|---|---|
| FG000 | SAD: Pooled Placebo | SAD: Single dose of placebo. |
| FG001 | SAD: 30mg WVE-003* | SAD: Single ascending dose of 30mg WVE-003. * |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Period 1 (Single Ascending Dose) |
|
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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 | Dec 26, 2022 |
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| SAD: 60mg WVE-003 | Drug | Single ascending dose of 60mg WVE-003, an allele-selective stereopure antisense oligonucleotide (ASO) |
|
| SAD: 90mg WVE-003 | Drug | Single ascending dose of 90mg WVE-003, an allele-selective stereopure antisense oligonucleotide (ASO) |
|
| SAD: Pooled Placebo | Drug | Single dose of placebo |
|
| MD: 30mg WVE-003 | Drug | Three doses of 30mg WVE-003 Q8WK an allele-selective stereopure, antisense oligonucleotide (ASO) |
|
| MD: Placebo | Drug | Three doses of placebo Q8WK |
|
Parameter analyzed: Cmax = maximum observed concentration.
| Day 1 (single ascending dose Period 1); Day 1 and Day 113 (multi dose Period 2) |
| Concentration of WVE-003 in Cerebrospinal Fluid (CSF) | WVE-003 concentration in cerebrospinal fluid (CSF) is reported in ng/mL. | 28 days post-dose during Period 1 (P1:Day29); 28 days post last dose during Period 2 (P2: Day141) |
| Clayton |
| Victoria |
| 3168 |
| Australia |
| Royal Melbourne Hospital | Melbourne | Victoria | 3050 | Australia |
| University of Alberta Hospital | Edmonton | Alberta | T6G 2G3 | Canada |
| The Ottawa Hospital | Ottawa | Ontario | K1H 8L6 | Canada |
| Centre Hospitalier de l-Universite de Montreal | Montreal | Quebec | H2X019 | Canada |
| Rigshospitalet | Copenhagen | 2100 | Denmark |
| Hopital Henri Mondor - Hospital | Créteil | 94010 | France |
| Institut du Cerveau et de la Moelle Epiniere | Paris | 75646 | France |
| Katholisches Klinikum Bochum gGmbH | Bochum | 44791 | Germany |
| George-Huntington-Institut GmbH | Münster | 48149 | Germany |
| kbo-Isar-Amper-Klinikum Taufkirchen (Vils) | Taufkirchen | 84416 | Germany |
| Centro Ricerche Cliniche Di Verona | Verona | Italy |
| Leiden University Medical Center | Leiden | 2333 ZA | Netherlands |
| Maastricht University Medical Center | Maastricht | 6229 HX | Netherlands |
| Szpital Sw. Wojciecha | Gdansk | 80-462 | Poland |
| Instytut Psychiatrii I Neurologii | Warsaw | 02-957 | Poland |
| Hospital de la Sanata Creu i Sant Pau | Barcelona | 08041 | Spain |
| Hospital Universitario Ramón y Cajal | Madrid | 28034 | Spain |
| Royal Devon and Exeter Hospital NHS Trust | Exeter | Devon | EX2 5DW | United Kingdom |
| Royal Hospital for Children, Pharmacy Aseptic Unit | Glasgow | Glasgow City | G51 4TF | United Kingdom |
| Cardiff University, Schools of Medicine and Biosciences | Cardiff | Wales | CF14 4XW | United Kingdom |
| Royal Liverpool University Hospital | Liverpool | L7 8XP | United Kingdom |
| Derived |
| Estevez-Fraga C, Tabrizi SJ, Wild EJ. Huntington's Disease Clinical Trials Corner: November 2022. J Huntingtons Dis. 2022;11(4):351-367. doi: 10.3233/JHD-229006. |
| FG002 | SAD: 60mg WVE-003* | SAD: Single ascending dose of 60mg WVE-003. * |
| FG003 | SAD: 90mg WVE-003 | SAD: Single ascending dose of 90mg WVE-003. |
| FG004 | MD: Placebo | MD: 3 doses of placebo Q8W. 7 Pts from Period 1 rolled over into Period 2. |
| FG005 | MD: 30mg WVE-003 | MD: 3 doses of 30mg WVE-003 Q8W. 16 Pts rolled over from Period 1 into Period 2. |
| COMPLETED | 1 Pt was randomized to WVE-003 60mg SAD group but received 30mg instead. This Pt is counted in the 30mg arm, but appears in the 60mg arm in the study flow diagram. |
|
| NOT COMPLETED |
|
|
| Period 2 (Multidose) |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | SAD: Pooled Placebo | Placebo SAD: Pooled Placebo: Single dose of placebo |
| BG001 | SAD: 30mg WVE-003 | Single Ascending Dose - 30mg WVE-003 SAD: 30mg WVE-003: Single ascending dose of 30mg WVE-003, an allele-selective stereopure antisense oligonucleotide (ASO) |
| BG002 | SAD: 60mg WVE-003 | Single Ascending Dose - 60mg WVE-003 SAD: 60mg WVE-003: Single ascending dose of 60mg WVE-003, an allele-selective stereopure antisense oligonucleotide (ASO) |
| BG003 | SAD: 90mg WVE-003 | Single Ascending Dose - 90mg WVE-003 SAD: 90mg WVE-003: Single ascending dose of 90mg WVE-003, an allele-selective stereopure antisense oligonucleotide (ASO) |
| BG004 | MD: Placebo | Placebo MD: Placebo: Three doses of placebo Q8WK |
| BG005 | MD: 30mg WVE-003 | Multiple Dose - 30mg WVE-003 MD: 30mg WVE-003: Three doses of 30mg WVE-003 Q8WK an allele-selective stereopure antisense oligonucleotide (ASO |
| BG006 | 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, Continuous | 47 participants were enrolled into Period 1, and of those 23 participants were rolled over into Period 2. | Mean | Standard Deviation | years |
| |||||||||
| Sex: Female, Male | 47 participants were enrolled into Period 1, and of those 23 participants were rolled over into Period 2. | Count of Participants | Participants |
| ||||||||||
| Ethnicity (NIH/OMB) | 47 participants were enrolled into Period 1, and of those 23 participants were rolled over into Period 2. | Count of Participants | Participants |
| ||||||||||
| Race (NIH/OMB) | 47 participants were enrolled into Period 1, and of those 23 participants were rolled over into Period 2. | Count of Participants | 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: Proportion of Patients With Treatment Emergent Adverse Events (TEAEs) Related to Study Drug | The primary outcome for this study was safety and is reported as the proportion of patients with TEAEs related to study drug. | Posted | Count of Participants | Participants | Day 1 through Week 24 (single ascending dose Period 1); Day 1 through Week 28 (multi dose Period 2) |
|
|
| ||||||||||||||||||||||||||||||||||||||||||
| Secondary | Pharmacokinetics of WVE-003 in Plasma | Parameter analyzed: AUC0-6 = area under the concentration-time curve from time 0 to 6 hrs | Please note that the PK analysis only includes participants treated with WVE-003 (all placebo participants have been set as '0'). | Posted | Mean | Standard Deviation | AUC 0-6 (hr*ng/mL) | Day 1 (single ascending dose Period 1); Day 1 and Day 113 (multi dose Period 2) |
| ||||||||||||||||||||||||||||||||||||||||||
| Secondary | Pharmacokinetics of WVE-003 in Plasma | Parameter analyzed: Cmax = maximum observed concentration. | Please note that the PK analysis only includes participants treated with WVE-003 (all placebo participants have been set as '0'). | Posted | Mean | Standard Deviation | Cmax (ng/mL) | Day 1 (single ascending dose Period 1); Day 1 and Day 113 (multi dose Period 2) |
| ||||||||||||||||||||||||||||||||||||||||||
| Secondary | Concentration of WVE-003 in Cerebrospinal Fluid (CSF) | WVE-003 concentration in cerebrospinal fluid (CSF) is reported in ng/mL. | Please note that the PK analysis only includes participants treated with WVE-003 (all placebo participants have been set as '0'). | Posted | Mean | Standard Deviation | ng/mL | 28 days post-dose during Period 1 (P1:Day29); 28 days post last dose during Period 2 (P2: Day141) |
|
Day 1 through Week 24 (single ascending dose Period 1); Day 1 through Week 28 (multi dose Period 2)
This study protocol collected all treatment-emergent adverse events and serious adverse events, whether they were treatment related or not. 5 months after a patient completed their final safety visit, an SAE was reported that sponsor assessed to be not-related to WVE-003.
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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 | SAD: Pooled Placebo | Placebo SAD: Pooled Placebo: Single dose of placebo | 0 | 16 | 1 | 16 | 13 | 16 |
| EG001 | SAD: 30mg WVE-003 | Single Ascending Dose - 30mg WVE-003 SAD: 30mg WVE-003: Single ascending dose of 30mg WVE-003, a stereopure antisense oligonucleotide (ASO) | 0 | 13 | 0 | 13 | 9 | 13 |
| EG002 | SAD: 60mg WVE-003 | Single Ascending Dose - 60mg WVE-003 SAD: 60mg WVE-003: Single ascending dose of 60mg WVE-003, a stereopure antisense oligonucleotide (ASO) | 0 | 10 | 1 | 10 | 8 | 10 |
| EG003 | SAD: 90mg WVE-003 | Single Ascending Dose - 90mg WVE-003 SAD: 90mg WVE-003: Single ascending dose of 90mg WVE-003, a stereopure antisense oligonucleotide (ASO) | 0 | 8 | 0 | 8 | 8 | 8 |
| EG004 | MD: Placebo | Placebo MD: Placebo: Three doses of placebo Q8WK | 0 | 7 | 0 | 7 | 7 | 7 |
| EG005 | MD: 30mg WVE-003 | Multiple Dose - 30mg WVE-003 MD: 30mg WVE-003: Three doses of 30mg WVE-003 Q8WK a stereopure antisense oligonucleotide (ASO | 0 | 16 | 0 | 16 | 13 | 16 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Post lumbar puncture syndrome | Injury, poisoning and procedural complications | Systematic Assessment |
| ||
| Ataxia | Nervous system disorders | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Eosinophilia | Blood and lymphatic system disorders | Systematic Assessment |
| ||
| Abdominal pain | Gastrointestinal disorders | Systematic Assessment |
| ||
| Diarrhoea | Gastrointestinal disorders | Systematic Assessment |
| ||
| Toothache | Gastrointestinal disorders | Systematic Assessment |
| ||
| Fatigue | General disorders | Systematic Assessment |
| ||
| Pain | General disorders | Systematic Assessment |
| ||
| Non-cardiac chest pain | General disorders | Systematic Assessment |
| ||
| Nasopharyngitis | Infections and infestations | Systematic Assessment |
| ||
| Influenza | Infections and infestations | Systematic Assessment |
| ||
| Upper respiratory tract infection | Infections and infestations | Systematic Assessment |
| ||
| Fall | Injury, poisoning and procedural complications | Systematic Assessment |
| ||
| Skin laceration | Injury, poisoning and procedural complications | Systematic Assessment |
| ||
| Contusion | Injury, poisoning and procedural complications | Systematic Assessment |
| ||
| Ligament sprain | Injury, poisoning and procedural complications | Systematic Assessment |
| ||
| Post lumbar puncture syndrome | Injury, poisoning and procedural complications | Systematic Assessment |
| ||
| Procedural pain | Injury, poisoning and procedural complications | Systematic Assessment |
| ||
| CSF white blood cell count increased | Investigations | Systematic Assessment |
| ||
| Blood creatine phosphokinase increased | Investigations | Systematic Assessment |
| ||
| CSF protein increased | Investigations | Systematic Assessment |
| ||
| Hyperkalaemia | Metabolism and nutrition disorders | Systematic Assessment |
| ||
| Arthralgia | Musculoskeletal and connective tissue disorders | Systematic Assessment |
| ||
| Back pain | Musculoskeletal and connective tissue disorders | Systematic Assessment |
| ||
| Headache | Nervous system disorders | Systematic Assessment |
| ||
| Balance disorder | Nervous system disorders | Systematic Assessment |
| ||
| Coordination abnormal | Nervous system disorders | Systematic Assessment |
| ||
| Dizziness | Nervous system disorders | Systematic Assessment |
| ||
| Dyskinesia | Nervous system disorders | Systematic Assessment |
| ||
| Hyperaesthesia | Nervous system disorders | Systematic Assessment |
| ||
| Hyporeflexia | Nervous system disorders | Systematic Assessment |
| ||
| Paraesthesia | Nervous system disorders | Systematic Assessment |
| ||
| Paresis | Nervous system disorders | Systematic Assessment |
| ||
| Pleocytosis | Nervous system disorders | Systematic Assessment |
| ||
| Presyncope | Nervous system disorders | Systematic Assessment |
| ||
| Intracranial hypotension | Nervous system disorders | Systematic Assessment |
| ||
| Attention deficit hyperactivity disorder | Psychiatric disorders | Systematic Assessment |
| ||
| Depression | Psychiatric disorders | Systematic Assessment |
| ||
| Sleep disorder | Psychiatric disorders | Systematic Assessment |
| ||
| Amenorrhoea | Reproductive system and breast disorders | Systematic Assessment |
| ||
| Eczema | Skin and subcutaneous tissue disorders | Systematic Assessment |
| ||
| Tachycardia | Cardiac disorders | Systematic Assessment |
| ||
| Eye pain | Eye disorders | Systematic Assessment |
| ||
| Nausea | Gastrointestinal disorders | Systematic Assessment |
| ||
| Constipation | Gastrointestinal disorders | Systematic Assessment |
| ||
| Dental caries | Gastrointestinal disorders | Systematic Assessment |
| ||
| Vomiting | Gastrointestinal disorders | Systematic Assessment |
| ||
| Influenza like illness | General disorders | Systematic Assessment |
| ||
| Pyrexia | General disorders | Systematic Assessment |
| ||
| Vessel puncture site haematoma | General disorders | Systematic Assessment |
| ||
| Puncture site pain | General disorders | Systematic Assessment |
| ||
| COVID-19 | Infections and infestations | Systematic Assessment |
| ||
| Urinary tract infection | Infections and infestations | Systematic Assessment |
| ||
| Infected seroma | Infections and infestations | Systematic Assessment |
| ||
| Skin abrasion | Injury, poisoning and procedural complications | Systematic Assessment |
| ||
| Concussion | Injury, poisoning and procedural complications | Systematic Assessment |
| ||
| Head injury | Injury, poisoning and procedural complications | Systematic Assessment |
| ||
| Injury | Injury, poisoning and procedural complications | Systematic Assessment |
| ||
| Procedural complication | Injury, poisoning and procedural complications | Systematic Assessment |
| ||
| Upper limb fracture | Injury, poisoning and procedural complications | Systematic Assessment |
| ||
| Eye injury | Injury, poisoning and procedural complications | Systematic Assessment |
| ||
| Limb injury | Injury, poisoning and procedural complications | Systematic Assessment |
| ||
| Lip injury | Injury, poisoning and procedural complications | Systematic Assessment |
| ||
| Road traffic accident | Injury, poisoning and procedural complications | Systematic Assessment |
| ||
| CSF red blood cell count positive | Investigations | Systematic Assessment |
| ||
| C-reactive protein increased | Investigations | Systematic Assessment |
| ||
| Myalgia | Musculoskeletal and connective tissue disorders | Systematic Assessment |
| ||
| Pain in extremity | Musculoskeletal and connective tissue disorders | Systematic Assessment |
| ||
| Musculoskeletal chest pain | Musculoskeletal and connective tissue disorders | Systematic Assessment |
| ||
| Muscle contracture | Musculoskeletal and connective tissue disorders | Systematic Assessment |
| ||
| Sciatica | Nervous system disorders | Systematic Assessment |
| ||
| Tension headache | Nervous system disorders | Systematic Assessment |
| ||
| Depressed mood | Psychiatric disorders | Systematic Assessment |
| ||
| Disorientation | Psychiatric disorders | Systematic Assessment |
| ||
| Irritability | Psychiatric disorders | Systematic Assessment |
| ||
| Anxiety | Psychiatric disorders | Systematic Assessment |
| ||
| Compulsions | Psychiatric disorders | Systematic Assessment |
| ||
| Insomnia | Psychiatric disorders | Systematic Assessment |
| ||
| Intermenstrual bleeding | Reproductive system and breast disorders | Systematic Assessment |
| ||
| Cough | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
| ||
| Rash | Skin and subcutaneous tissue disorders | Systematic Assessment |
| ||
| Rash erythematous | Skin and subcutaneous tissue disorders | Systematic Assessment |
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| Dermatitis contact | Skin and subcutaneous tissue disorders | Systematic Assessment |
| ||
| Skin irritation | Skin and subcutaneous tissue disorders | Systematic Assessment |
| ||
| Hypotension | Vascular disorders | Systematic Assessment |
| ||
| Haematoma | Vascular disorders | Systematic Assessment |
|
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Medical Director, MD | Wave Life Sciences USA, Inc | +1 617-949-2900 | info@wavelifesci.com |
| Jun 6, 2025 |
| Prot_SAP_000.pdf |
Not provided
| 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 |
Not provided
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| ID | Term |
|---|---|
| D064346 | Sagittal Abdominal Diameter |
| ID | Term |
|---|---|
| D049628 | Body Size |
| D001837 | Body Weights and Measures |
| D001824 | Body Constitution |
| D010808 | Physical Examination |
| D019937 | Diagnostic Techniques and Procedures |
| D003933 | Diagnosis |
| D000886 | Anthropometry |
| D008919 | Investigative Techniques |
| D010829 | Physiological Phenomena |
Not provided
Not provided
|
| Period 2 |
|
|
|
| Period 2 |
|
|
|
| Period 2 |
|
|
|
| Period 2 |
|
|
Multiple Dose - 30mg WVE-003
MD: 30mg WVE-003: Three doses of 30mg WVE-003 Q8WK an allele-selective stereopure antisense oligonucleotide (ASO
|
|
Multiple Dose - 30mg WVE-003
MD: 30mg WVE-003: Three doses of 30mg WVE-003 Q8WK an allele-selective stereopure antisense oligonucleotide (ASO
|
|
Multiple Dose - 30mg WVE-003 MD: 30mg WVE-003: Three doses of 30mg WVE-003 Q8WK an allele-selective stereopure antisense oligonucleotide (ASO |
|
|
| Unknown or Not Reported |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
| Title | Measurements |
|---|---|
|
| Title | Measurements |
|---|---|
|