Study Results
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View full resultsBasic Information
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COMPLETED
PHASE1/PHASE2
47 participants
INTERVENTIONAL
2021-09-06
2024-05-24
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
SEQUENTIAL
TREATMENT
DOUBLE
Study Groups
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SAD: Pooled Placebo
Placebo
SAD: Pooled Placebo
Single dose of placebo
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)
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)
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)
MD: Placebo
Placebo
MD: Placebo
Three doses of placebo Q8WK
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)
Interventions
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SAD: 30mg WVE-003
Single ascending dose of 30mg WVE-003, an allele-selective stereopure antisense oligonucleotide (ASO)
SAD: 60mg WVE-003
Single ascending dose of 60mg WVE-003, an allele-selective stereopure antisense oligonucleotide (ASO)
SAD: 90mg WVE-003
Single ascending dose of 90mg WVE-003, an allele-selective stereopure antisense oligonucleotide (ASO)
SAD: Pooled Placebo
Single dose of placebo
MD: 30mg WVE-003
Three doses of 30mg WVE-003 Q8WK an allele-selective stereopure, antisense oligonucleotide (ASO)
MD: Placebo
Three doses of placebo Q8WK
Eligibility Criteria
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Inclusion Criteria
2. Ambulatory, male or female patients aged ≥25 to ≤60 years
3. Clinical diagnostic motor features of HD, defined as Unified Huntington's Disease Rating Scale (UHDRS) Diagnostic Confidence Score = 4
4. UHDRS Total Functional Capacity Scores ≥9 and ≤13
Exclusion Criteria
2. 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
3. Implantable CNS device that may interfere with ability to administer study drug via lumbar puncture or undergo MRI scan
4. Inability to undergo brain MRI (with or without sedation)
5. Bone, spine, bleeding, or other disorder that exposes the patient to risk of injury or unsuccessful lumbar puncture
6. Previously received tominersen
25 Years
60 Years
ALL
No
Sponsors
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Wave Life Sciences Ltd.
INDUSTRY
Responsible Party
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Principal Investigators
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Medical Director, MD
Role: STUDY_DIRECTOR
Wave Life Sciences
Locations
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Westmead Hospital
Westmead, New South Wales, Australia
Monash Health
Clayton, Victoria, Australia
Royal Melbourne Hospital
Melbourne, Victoria, Australia
University of Alberta Hospital
Edmonton, Alberta, Canada
The Ottawa Hospital
Ottawa, Ontario, Canada
Centre Hospitalier de l-Universite de Montreal
Montreal, Quebec, Canada
Rigshospitalet
Copenhagen, , Denmark
Hopital Henri Mondor - Hospital
Créteil, , France
Institut du Cerveau et de la Moelle Epiniere
Paris, , France
Katholisches Klinikum Bochum gGmbH
Bochum, , Germany
George-Huntington-Institut GmbH
Münster, , Germany
kbo-Isar-Amper-Klinikum Taufkirchen (Vils)
Taufkirchen, , Germany
Centro Ricerche Cliniche Di Verona
Verona, , Italy
Leiden University Medical Center
Leiden, , Netherlands
Maastricht University Medical Center
Maastricht, , Netherlands
Szpital Sw. Wojciecha
Gdansk, , Poland
Instytut Psychiatrii I Neurologii
Warsaw, , Poland
Hospital de la Sanata Creu i Sant Pau
Barcelona, , Spain
Hospital Universitario Ramón y Cajal
Madrid, , Spain
Royal Devon and Exeter Hospital NHS Trust
Exeter, Devon, United Kingdom
Royal Hospital for Children, Pharmacy Aseptic Unit
Glasgow, Glasgow City, United Kingdom
Cardiff University, Schools of Medicine and Biosciences
Cardiff, Wales, United Kingdom
Royal Liverpool University Hospital
Liverpool, , United Kingdom
Countries
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References
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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.
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.
Provided Documents
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Document Type: Study Protocol and Statistical Analysis Plan
Other Identifiers
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WVE-003-001
Identifier Type: -
Identifier Source: org_study_id
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