Trial Review

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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT04617860




Registration number
NCT04617860
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-120102 in Patients With Huntington's Disease
Scientific title
A Multicenter, Open-label Extension Study to Evaluate the Safety, Pharmacodynamics, and Clinical Effects of WVE-120102 in Patients With Huntington's Disease
Secondary ID [1] 0 0
WVE-HDSNP2-002
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-120102

Experimental: WVE-120102 (Dose A) -


Treatment: Drugs: WVE-120102
WVE-120102 is a stereopure antisense oligonucleotide. 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 Adverse Events (TEAEs)
Timepoint [1] 0 0
Day 1 to Study Termination (maximum of 12 monthly doses)
Primary outcome [2] 0 0
Safety: Number of Patients With a Severe TEAE
Timepoint [2] 0 0
Day 1 to Study Termination (maximum of 12 monthly doses)
Primary outcome [3] 0 0
Safety: Number of Patients With Serious TEAEs
Timepoint [3] 0 0
Day 1 to Study Termination (maximum of 12 monthly doses)
Primary outcome [4] 0 0
Safety and Tolerability: Number of Patients Who Withdraw Due to TEAEs
Timepoint [4] 0 0
Day 1 to Study Termination (maximum of 12 monthly doses)

Eligibility
Key inclusion criteria
1. Patient successfully completed the Phase 1b/2a study with WVE-120102, WVE-HDSNP2-001.
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-120102, 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
N/A
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/Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Terminated
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
24/09/2019
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
29/04/2021
Sample size
Target
Accrual to date
Final
36
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
Ontario
Country [3] 0 0
Canada
State/province [3] 0 0
Quebec
Country [4] 0 0
Denmark
State/province [4] 0 0
Aarhus
Country [5] 0 0
Denmark
State/province [5] 0 0
Copenhagen
Country [6] 0 0
Denmark
State/province [6] 0 0
Odense
Country [7] 0 0
France
State/province [7] 0 0
Créteil
Country [8] 0 0
France
State/province [8] 0 0
Paris
Country [9] 0 0
Germany
State/province [9] 0 0
Muenster
Country [10] 0 0
Poland
State/province [10] 0 0
Gdansk
Country [11] 0 0
Poland
State/province [11] 0 0
Warsaw
Country [12] 0 0
United Kingdom
State/province [12] 0 0
Devon
Country [13] 0 0
United Kingdom
State/province [13] 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-HDSNP2-002 is an open-label extension (OLE) study to evaluate the safety, tolerability,
PK, PD, and clinical effects of WVE-120102 in adult patients with early manifest HD who carry
a targeted single nucleotide polymorphism, rs362331 (SNP2). To participate in the study,
patients must have completed the Phase 1b/2a clinical study WVE-HDSNP2-001.
Trial website
https://clinicaltrials.gov/ct2/show/NCT04617860
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/ct2/show/NCT04617860