Huntington DiseasePhase 1/2NCT04617847
Open-label Extension Study to Evaluate the Safety and Tolerability of WVE-120101 in Patients With Huntington's Disease
A Phase 1/2 study testing WVE-120101 in people with Huntington Disease, run by Wave Life Sciences USA, Inc. at 18 sites in 7 countries.
Trial 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). Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Huntington Disease
- What is being tested?
- WVE-120101
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 27 participants
- Where
- 18 sitesin 7 countries
- Study dates
- 13 Apr 2020to 3 May 2021
- Phase
- Phase 1/2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 1. Patient successfully completed the Phase 1b/2a study with WVE-120101, WVE-HDSNP1-001.
Who cannot join
- 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.
Trial locations and contacts
18 sites in 7 countries.
18 sites in 7 countries
Australia7 sites
Royal Melbourne Hospital
Carlton, Victoria, 3053
Monash Health
Clayton, Victoria, 3168
Royal Brisbane & Women's Hospital
Herston, Queensland, QLD 4006
Alfred Health
Melbourne, Victoria, 3004
Calvary Health Care Bethlehem
Parkdale, Victoria, 3195
North Metropolitan Health Service
Perth, Western Australia, 6910
Westmead Hospital
Sydney, New South Wales, 2145
Canada2 sites
University of Alberta
Edmonton, Alberta, T6G 2B7
Centre Hospitalier de l-Universite de Montreal
Montreal, Quebec, H2X019
Denmark2 sites
Aarhus Universitets Hospital
Aarhus, 8200
Rigshospitalet
Copenhagen, 2100
France2 sites
Hospital Henri Mondor
Créteil, 94010
Institut du Cerveau et de la Moelle Epinière
Paris, 75646
Poland2 sites
Szpital Sw. Wojciecha
Gdansk, 80-462
Instytut Psychiatrii i Neurologii
Warsaw, 02-957
United Kingdom2 sites
Royal Devon and Exeter Hospital NHS Trust
Exeter, Devon, EX2 5DW
Queen Elizabeth University Hospital - PPDS
Glasgow, Glasgow City, G12 0XH
About this study
Official title: A Multicenter, Open-label Extension Study to Evaluate the Safety, Pharmacodynamics, and Clinical Effects of WVE-120101 in Patients With Huntington's Disease
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.
- Conditions
- Huntington Disease
- Being tested
- WVE-120101
- Medical subject headings
- Huntington Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
WVE-120101Drug
WVE-120101 is a stereopure antisense oligonucleotide (ASO). It is administered monthly via intrathecal injection.
| Study group | Role | What they receive |
|---|---|---|
| WVE-120101 (Dose A) | Experimental | Not described Receives: Drug: WVE-120101 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety: Number of Patients With Treatment-emergent AEs (TEAEs)
Measured: First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment)
Safety: Number of Patients With a Severe TEAE
Measured: First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment
Safety: Number of Patients With Serious TEAEs
Measured: 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
Measured: First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Nov 2020
- Study start13 Apr 2020
- Primary completion3 May 2021
- Study completion3 May 2021
Why it stopped: Lack of efficacy
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Huntington DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 27 participants (actual)
Sponsor and investigators
- Lead sponsor
- Wave Life Sciences USA, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Director, MD, Study director, Wave Life Sciences
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04617847
- Sponsor's protocol ID
- WVE-HDSNP1-002
- Status last verified
- Jan 2022
- First submitted
- 30 Oct 2020
- Last updated
- 9 Feb 2022
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 Feb 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Open-label Extension Study to Evaluate the Safety, Pharmacodynamics, and Clinical Effects of WVE-120101 in Patients With Huntington's Disease
- ClinicalTrials.gov ID
- NCT04617847
- Acronym / sponsor ID
- WVE-HDSNP1-002
- Conditions
- Huntington Disease
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Lack of efficacy
- Enrolment
- 27 participants (actual)
- Lead sponsor
- Wave Life Sciences USA, Inc.Industry (pharma, biotech, devices)
- Study start
- 13 Apr 2020 (actual)
- Primary completion
- 3 May 2021 (actual)
- Study completion
- 3 May 2021 (actual)
- First posted
- 5 Nov 2020 (actual)
- Results
- Posted 9 Feb 2022
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| WVE-120101 (Dose A) | Experimental | Drug: WVE-120101 |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (1 listed)
- 1. Patient successfully completed the Phase 1b/2a study with WVE-120101, WVE-HDSNP1-001.
Key exclusion criteria (3 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| WVE-120101WVE-120101 is a stereopure antisense oligonucleotide (ASO). It is administered monthly via intrathecal injection. | Drug | WVE-120101 (Dose A) |
Primary outcome
Safety: Number of Patients With Treatment-emergent AEs (TEAEs)
Time frame: First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment)
Safety: Number of Patients With a Severe TEAE
Time frame: First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment
Safety: Number of Patients With Serious TEAEs
Time frame: 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
Time frame: First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Feb 2022.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Medical Director, MD study director
Wave Life Sciences
18 sites in 7 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing WVE-120101 in people with Huntington Disease, run by Wave Life Sciences USA, Inc. at 18 sites in 7 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 9 Feb 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 18 sites in Australia, Canada, Denmark, France, Poland and 2 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 9 Feb 2022). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety: Number of Patients With Treatment-emergent AEs (TEAEs) (time frame: First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment)). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Feb 2022.