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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT04617847




Registration number
NCT04617847
Ethics application status
Date submitted
30/10/2020
Date registered
5/11/2020
Date last updated
9/02/2022

Titles & IDs
Public title
Open-label Extension Study to Evaluate the Safety and Tolerability of WVE-120101 in Patients With Huntington's Disease
Scientific title
A Multicenter, Open-label Extension Study to Evaluate the Safety, Pharmacodynamics, and Clinical Effects of WVE-120101 in Patients With Huntington's Disease
Secondary ID [1] 0 0
WVE-HDSNP1-002
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Huntington Disease 0 0
Condition category
Condition code

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - WVE-120101

Experimental: WVE-120101 (Dose A) -


Treatment: Drugs: WVE-120101
WVE-120101 is a stereopure antisense oligonucleotide (ASO). It is administered monthly via intrathecal injection.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Safety: Number of Patients With Treatment-emergent AEs (TEAEs)
Timepoint [1] 0 0
First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment)
Primary outcome [2] 0 0
Safety: Number of Patients With a Severe TEAE
Timepoint [2] 0 0
First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment
Primary outcome [3] 0 0
Safety: Number of Patients With Serious TEAEs
Timepoint [3] 0 0
First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment
Primary outcome [4] 0 0
Safety and Tolerability: Number of Patients Who Withdraw Due to TEAEs
Timepoint [4] 0 0
First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment

Eligibility
Key inclusion criteria
Key

* 1. Patient successfully completed the Phase 1b/2a study with WVE-120101, WVE-HDSNP1-001.

Key
Minimum age
No limit
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* 1. Received an investigational drug other than WVE-120101, including an investigational oligonucleotide, within the past 1 year or 5 half-lives of the drug, whichever is longer.
* 2. Inability to undergo brain MRI (with or without sedation).
* 3. Clinically significant medical finding on the physical examination other than HD that, in the judgment of the Investigator, will make the patient unsuitable for participation in and/or completion of the study procedures.

Study design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Stopped early
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
13/04/2020
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
3/05/2021
Sample size
Target
Accrual to date
Final
27
Recruitment in Australia
Recruitment state(s)
NSW,QLD,VIC,WA
Recruitment hospital [1] 0 0
Westmead Hospital - Sydney
Recruitment hospital [2] 0 0
Royal Brisbane & Women's Hospital - Herston
Recruitment hospital [3] 0 0
Royal Melbourne Hospital - Carlton
Recruitment hospital [4] 0 0
Monash Health - Clayton
Recruitment hospital [5] 0 0
Alfred Health - Melbourne
Recruitment hospital [6] 0 0
Calvary Health Care Bethlehem - Parkdale
Recruitment hospital [7] 0 0
North Metropolitan Health Service - Perth
Recruitment postcode(s) [1] 0 0
2145 - Sydney
Recruitment postcode(s) [2] 0 0
QLD 4006 - Herston
Recruitment postcode(s) [3] 0 0
3053 - Carlton
Recruitment postcode(s) [4] 0 0
3168 - Clayton
Recruitment postcode(s) [5] 0 0
3004 - Melbourne
Recruitment postcode(s) [6] 0 0
3195 - Parkdale
Recruitment postcode(s) [7] 0 0
6910 - Perth
Recruitment outside Australia
Country [1] 0 0
Canada
State/province [1] 0 0
Alberta
Country [2] 0 0
Canada
State/province [2] 0 0
Quebec
Country [3] 0 0
Denmark
State/province [3] 0 0
Aarhus
Country [4] 0 0
Denmark
State/province [4] 0 0
Copenhagen
Country [5] 0 0
France
State/province [5] 0 0
Créteil
Country [6] 0 0
France
State/province [6] 0 0
Paris
Country [7] 0 0
Germany
State/province [7] 0 0
Muenster
Country [8] 0 0
Poland
State/province [8] 0 0
Gdansk
Country [9] 0 0
Poland
State/province [9] 0 0
Warsaw
Country [10] 0 0
United Kingdom
State/province [10] 0 0
Devon
Country [11] 0 0
United Kingdom
State/province [11] 0 0
Glasgow City

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Wave Life Sciences Ltd.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
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.
Trial website
https://clinicaltrials.gov/study/NCT04617847
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Medical Director, MD
Address 0 0
Wave Life Sciences
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT04617847