Huntington's DiseasePhase 1/2PRECISION-HD1NCT03225833

Safety and Tolerability of WVE-120101 in Patients With Huntington's Disease

A Phase 1/2 study testing WVE-120101 in people with Huntington's Disease, run by Wave Life Sciences USA, Inc. at 21 sites in 7 countries.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record

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Trial summary

PRECISION-HD1 is a Phase 1b/2a multicenter, randomized, double-blind, placebo-controlled study to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of single and multiple doses of WVE-120101 in adult patients with early manifest Huntington's disease (HD) who carry a targeted single… Read more about this study

Who is it for?: Ages 25 to 65 years
Conditions the study is for: Huntington's Disease
What is being tested?: WVE-120101
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 61 participants
Where: 21 sitesin 7 countries
Study dates: 17 Jul 2017to 11 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 25 to 65 years
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

Prescreened with targeted SNP on the same allele as the pathogenic CAG expansion
Ambulatory, male or female patients aged ≥25 - ≤65 years
Clinical diagnostic motor features of HD, defined as Unified Huntington's Disease Rating Scale (UHDRS) Diagnostic Confidence Score = 4
Early manifest HD, Stage I or Stage II based on UHDRS Total Functional Capacity Scores ≥7 and ≤13

Who cannot join

Malignancy or received treatment for malignancy, other than treated basal cell or squamous cell carcinoma of the skin, within the previous 5 years.
Received investigational drug or implantable device in prior 3 months or investigational oligonucleotide in prior 6 months or 5 half-lives of the oligonucleotide, whichever is longer
Clinically significant medical condition, unstable psychiatric symptoms, substance abuse, or pregnancy
Inability to undergo brain MRI
Bone, spine, bleeding, or other disorder that exposes the patient to risk of injury or unsuccessful lumbar puncture

Trial locations and contacts

21 sites in 7 countries.

Country

City, postcode or hospital

21 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

Other trials in Australia

Canada3 sites

University of Alberta
Edmonton, Alberta, T6G 2B7
Center Hospitalier de l'Universite de Montreal
Montreal, Quebec, H2X0A9
Centre For Movement Disorders
Toronto, Ontario, M3B 2S7

Other trials in Canada

Denmark3 sites

Aarhus Universitets Hospital
Aarhus, 8200
Rigshospitalet
Copenhagen, 2100
Odense University Hospital and University of Southern Denmark
Odense, 5000

Other trials in Denmark

United Kingdom3 sites

Royal Devon and Exeter Hospital NHS Trust
Exeter, Devon, EX2 5DW
Queen Elizabeth University Hospital - PPDS
Glasgow, Glasgow City, G12 0XH
Royal Liverpool University Hospital
Liverpool, L7 8XP

Other trials in United Kingdom

France2 sites

Hospital Henri Mondor
Créteil, 94010
Institut du Cerveau et de la Moelle Epinière
Paris, 75646

Other trials in France

Poland2 sites

Szpital Sw. Wojciecha
Gdansk, 80-462
Instytut Psychiatrii i Neurologii
Warsaw, 02-957

Other trials in Poland

Germany1 site

George-Huntington-Institut GmbH
Münster, 48149

Other trials in Germany

About this study

Official title: A Multicenter, Randomized, Double-blind, Placebo-controlled, Phase 1b/2a Study of WVE-120101 Administered Intrathecally in Patients With Huntington's Disease

Conditions: Huntington's Disease
Being tested: WVE-120101, Placebo
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)
PlaceboDrug
0.9% Sodium Chloride

Study groups in NCT03225833
Study group	Role	What they receive
WVE-120101 (Dose A) or placebo	Experimental	Not described Receives: Drug: WVE-120101; Drug: Placebo
WVE-120101 (Dose B) or placebo	Experimental	Not described Receives: Drug: WVE-120101; Drug: Placebo
WVE-120101 (Dose C) or placebo	Experimental	Not described Receives: Drug: WVE-120101; Drug: Placebo
WVE-120101 (Dose D) or placebo	Experimental	Not described Receives: Drug: WVE-120101; Drug: Placebo
WVE-120101 (Dose E) or placebo	Experimental	Not described Receives: Drug: WVE-120101; Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

Safety: Number of Patients With Treatment-emergent Adverse Events (TEAEs)
All TEAEs reported or observed during the study, including TEAEs resulting from concurrent illnesses, reactions to concurrent medications, or progression of disease states
Measured: Day 1 to end of study (up to Day 182 [32 mg cohort]/ Day 210 [all other cohorts])
Safety: Severity of Adverse Events
Number of patients who experienced a severe treatment-emergent adverse event. Severity was evaluated using the National Cancer Institute Common Terminology Criteria for Adverse Events version 4.0
Measured: Day 1 to end of study (up to Day 182 [32 mg cohort]/ Day 210 [all other cohorts])
Safety: Number of Patients With Serious TEAEs
A serious TEAE is defined as any event that results in death, is immediately life threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, or is a congenital anomaly/birth defect not present at Prescreening.
Measured: Day 1 to end of study (up to Day 182 [32 mg cohort]/ Day 210 [all other cohorts])
Safety and Tolerability: Number of Patients Who Withdraw Due to TEAEs
Measured: Day 1 to end of study (up to Day 182 [32 mg cohort]/ Day 210 [all other cohorts])

Secondary outcomes (6)

Pharmacokinetics (PK): Maximum Observed Concentration (Cmax)
Cmax of WVE-120101 in plasma
Measured: Patients participating in Period 1 (SAD) had PK samples collected on Day 1 predose through 24-48 hours postdose. Patients participating in Period 2 (MAD) had PK samples collected predose on Day 112 and through 4 hours postdose.
PK: Time of Occurrence of Cmax (Tmax)
tmax of WVE-120101 in plasma
Measured: Patients participating in Period 1 (SAD) had PK samples collected on Day 1 predose through 24-48 hours postdose. Patients participating in Period 2 (MAD) had PK samples collected predose on Day 112 and through 4 hours postdose.
PK: Area Under the Plasma Concentration-time Curve (AUClast)
AUClast from time 0 to the last quantifiable concentration of WVE-120101 in plasma
Measured: Patients participating in Period 1 (SAD) had PK samples collected on Day 1 predose through 24-48 hours postdose. Patients participating in Period 2 (MAD) had PK samples collected predose on Day 112 and through 4 hours postdose.
PK: Terminal Elimination Half Life
Terminal elimination half life of WVE-120101 in plasma (t1/2)
Measured: Patients participating in Period 1 (SAD) had PK samples collected on Day 1 predose through 24-48 hours postdose. Patients participating in Period 2 (MAD) had PK samples collected predose on Day 112 and through 4 hours postdose.
Pharmacodynamics
Percentage change from baseline in concentration of mutant huntingtin (mHTT) protein in CSF
Measured: Day 1 to last observation - up to Day 140 (32 mg cohort) or Day 196 (all other cohorts)
Clinical Effects: Total Functional Capacity (TFC)
Percentage change from baseline to the last measured time point in the Total Functional Capacity score, administered as part of the Unified Huntington's Disease Rating Scale (UHDRS). Total Functional Capacity is scored 13 (normal) to 0 (severe disability).
Measured: Day 1 to last observation - up to Day 140 (32 mg cohort) or Day 196 (all other cohorts)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

17 Jul 201711 May 2021

First posted21 Jul 2017
Study start17 Jul 2017
Primary completion11 May 2021
Study completion11 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.
Publications (2)Papers the sponsor has linked to this study.
Other trials for Huntington's 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: Randomised
Intervention model: Sequential
Masking: Double blind
Who is masked: Participant, Investigator
Enrolment: 61 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT03225833
Sponsor's protocol ID: WVE-HDSNP1-001
Acronym: PRECISION-HD1
Status last verified: Feb 2022
First submitted: 17 Jul 2017
Last updated: 10 Feb 2022
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes
FDA-regulated drug: No
FDA-regulated device: No

Publications

Rodrigues FB, Wild EJ. Huntington's Disease Clinical Trials Corner: April 2020. J Huntingtons Dis. 2020;9(2):185-197. doi: 10.3233/JHD-200002.
PubMed 32250312
Rodrigues FB, Wild EJ. Huntington's Disease Clinical Trials Corner: February 2018. J Huntingtons Dis. 2018;7(1):89-98. doi: 10.3233/JHD-189001.
PubMed 29480210

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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All Huntington's Disease trials

Study at a glance

From the registry record, last updated by the sponsor on 10 Feb 2022. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Multicenter, Randomized, Double-blind, Placebo-controlled, Phase 1b/2a Study of WVE-120101 Administered Intrathecally in Patients With Huntington's Disease
ClinicalTrials.gov ID: NCT03225833
Acronym / sponsor ID: PRECISION-HD1 / WVE-HDSNP1-001
Conditions: Huntington's Disease
Phase: Phase 1/2
Study type: Interventional
Recruitment status: TerminatedReason stopped: Lack of Efficacy
Enrolment: 61 participants (actual)
Lead sponsor: Wave Life Sciences USA, Inc.Industry (pharma, biotech, devices)
Study start: 17 Jul 2017 (actual)
Primary completion: 11 May 2021 (actual)
Study completion: 11 May 2021 (actual)
First posted: 21 Jul 2017 (actual)
Results: Posted 10 Feb 2022

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Sequential
Masking: Double blind (participant, investigator)
Number of arms: 5

Arm	Type	Receives
WVE-120101 (Dose A) or placebo	Experimental	Drug: WVE-120101, Drug: Placebo
WVE-120101 (Dose B) or placebo	Experimental	Drug: WVE-120101, Drug: Placebo
WVE-120101 (Dose C) or placebo	Experimental	Drug: WVE-120101, Drug: Placebo
WVE-120101 (Dose D) or placebo	Experimental	Drug: WVE-120101, Drug: Placebo
WVE-120101 (Dose E) or placebo	Experimental	Drug: WVE-120101, Drug: Placebo

Who may qualify

Ages: 25 to 65 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (4 listed)

Prescreened with targeted SNP on the same allele as the pathogenic CAG expansion
Ambulatory, male or female patients aged ≥25 - ≤65 years
Clinical diagnostic motor features of HD, defined as Unified Huntington's Disease Rating Scale (UHDRS) Diagnostic Confidence Score = 4
Early manifest HD, Stage I or Stage II based on UHDRS Total Functional Capacity Scores ≥7 and ≤13

Key exclusion criteria (5 listed)

Malignancy or received treatment for malignancy, other than treated basal cell or squamous cell carcinoma of the skin, within the previous 5 years.
Received investigational drug or implantable device in prior 3 months or investigational oligonucleotide in prior 6 months or 5 half-lives of the oligonucleotide, whichever is longer
Clinically significant medical condition, unstable psychiatric symptoms, substance abuse, or pregnancy
Inability to undergo brain MRI
Bone, spine, bleeding, or other disorder that exposes the patient to risk of injury or unsuccessful lumbar puncture

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)	Drug	WVE-120101 (Dose A) or placebo, WVE-120101 (Dose B) or placebo, WVE-120101 (Dose C) or placebo, WVE-120101 (Dose D) or placebo, WVE-120101 (Dose E) or placebo
Placebo0.9% Sodium Chloride	Drug	WVE-120101 (Dose A) or placebo, WVE-120101 (Dose B) or placebo, WVE-120101 (Dose C) or placebo, WVE-120101 (Dose D) or placebo, WVE-120101 (Dose E) or placebo

Primary outcome

Safety: Number of Patients With Treatment-emergent Adverse Events (TEAEs)
Time frame: Day 1 to end of study (up to Day 182 [32 mg cohort]/ Day 210 [all other cohorts])
All TEAEs reported or observed during the study, including TEAEs resulting from concurrent illnesses, reactions to concurrent medications, or progression of disease states
Safety: Severity of Adverse Events
Time frame: Day 1 to end of study (up to Day 182 [32 mg cohort]/ Day 210 [all other cohorts])
Number of patients who experienced a severe treatment-emergent adverse event. Severity was evaluated using the National Cancer Institute Common Terminology Criteria for Adverse Events version 4.0
Safety: Number of Patients With Serious TEAEs
Time frame: Day 1 to end of study (up to Day 182 [32 mg cohort]/ Day 210 [all other cohorts])
A serious TEAE is defined as any event that results in death, is immediately life threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, or is a congenital anomaly/birth defect not present at Prescreening.
Safety and Tolerability: Number of Patients Who Withdraw Due to TEAEs
Time frame: Day 1 to end of study (up to Day 182 [32 mg cohort]/ Day 210 [all other cohorts])

6 secondary outcomes

Pharmacokinetics (PK): Maximum Observed Concentration (Cmax)
Time frame: Patients participating in Period 1 (SAD) had PK samples collected on Day 1 predose through 24-48 hours postdose. Patients participating in Period 2 (MAD) had PK samples collected predose on Day 112 and through 4 hours postdose.
PK: Time of Occurrence of Cmax (Tmax)
Time frame: Patients participating in Period 1 (SAD) had PK samples collected on Day 1 predose through 24-48 hours postdose. Patients participating in Period 2 (MAD) had PK samples collected predose on Day 112 and through 4 hours postdose.
PK: Area Under the Plasma Concentration-time Curve (AUClast)
Time frame: Patients participating in Period 1 (SAD) had PK samples collected on Day 1 predose through 24-48 hours postdose. Patients participating in Period 2 (MAD) had PK samples collected predose on Day 112 and through 4 hours postdose.
PK: Terminal Elimination Half Life
Time frame: Patients participating in Period 1 (SAD) had PK samples collected on Day 1 predose through 24-48 hours postdose. Patients participating in Period 2 (MAD) had PK samples collected predose on Day 112 and through 4 hours postdose.
Pharmacodynamics
Time frame: Day 1 to last observation - up to Day 140 (32 mg cohort) or Day 196 (all other cohorts)
Clinical Effects: Total Functional Capacity (TFC)
Time frame: Day 1 to last observation - up to Day 140 (32 mg cohort) or Day 196 (all other cohorts)

Results and safety

Results posted to the registry on 10 Feb 2022, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Pooled Placebo	16	11
WVE-120101 (2 mg)	9	8
WVE-120101 (4 mg)	9	7
WVE-120101 (8 mg)	9	9
WVE-120101 (16 mg)	8	7
WVE-120101 (32 mg)	10	0

Primary outcome: Safety: Number of Patients With Treatment-emergent Adverse Events (TEAEs)

Time frame: Day 1 to end of study (up to Day 182 [32 mg cohort]/ Day 210 [all other cohorts]). Count of participants. Unit: Participants

Measure	Pooled Placebo n = 16	WVE-120101 (2 mg) n = 9	WVE-120101 (4 mg) n = 9	WVE-120101 (8 mg) n = 9	WVE-120101 (16 mg) n = 8	WVE-120101 (32 mg) n = 10
Value	12	8	8	9	7	9

Primary outcome: Safety: Severity of Adverse Events

Time frame: Day 1 to end of study (up to Day 182 [32 mg cohort]/ Day 210 [all other cohorts]). Count of participants. Unit: Participants

Measure	Pooled Placebo n = 16	WVE-120101 (2 mg) n = 9	WVE-120101 (4 mg) n = 9	WVE-120101 (8 mg) n = 9	WVE-120101 (16 mg) n = 8	WVE-120101 (32 mg) n = 10
Value	1	2	1	1	0	5

Primary outcome: Safety: Number of Patients With Serious TEAEs

Time frame: Day 1 to end of study (up to Day 182 [32 mg cohort]/ Day 210 [all other cohorts]). Count of participants. Unit: Participants

Measure	Pooled Placebo n = 16	WVE-120101 (2 mg) n = 9	WVE-120101 (4 mg) n = 9	WVE-120101 (8 mg) n = 9	WVE-120101 (16 mg) n = 8	WVE-120101 (32 mg) n = 10
Value	0	2	1	0	0	4

Primary outcome: Safety and Tolerability: Number of Patients Who Withdraw Due to TEAEs

Time frame: Day 1 to end of study (up to Day 182 [32 mg cohort]/ Day 210 [all other cohorts]). Count of participants. Unit: Participants

Measure	Pooled Placebo n = 16	WVE-120101 (2 mg) n = 9	WVE-120101 (4 mg) n = 9	WVE-120101 (8 mg) n = 9	WVE-120101 (16 mg) n = 8	WVE-120101 (32 mg) n = 10
Value	0	1	2	0	0	2

6 secondary outcomes are reported in the full results.

Adverse events

Time frame: Day 1 to end of study (Day 196 [32 mg cohort] or Day 210 [all other cohorts]). Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Pooled Placebo	0 of 16 (0%)	12 of 16 (75%)	0 of 16
WVE-120101 (2 mg)	2 of 9 (22.2%)	8 of 9 (88.9%)	0 of 9
WVE-120101 (4 mg)	1 of 9 (11.1%)	8 of 9 (88.9%)	0 of 9
WVE-120101 (8 mg)	0 of 9 (0%)	9 of 9 (100%)	0 of 9
WVE-120101 (16 mg)	0 of 8 (0%)	7 of 8 (87.5%)	0 of 8
WVE-120101 (32 mg)	4 of 10 (40%)	9 of 10 (90%)	0 of 10

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 10 Feb 2022.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 32250312Rodrigues FB, Wild EJ. Huntington's Disease Clinical Trials Corner: April 2020. J Huntingtons Dis. 2020;9(2):185-197. doi: 10.3233/JHD-200002.
Derived from this study: PubMed 29480210Rodrigues FB, Wild EJ. Huntington's Disease Clinical Trials Corner: February 2018. J Huntingtons Dis. 2018;7(1):89-98. doi: 10.3233/JHD-189001.

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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

21 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's Disease, run by Wave Life Sciences USA, Inc. at 21 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 4 inclusion and 5 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 10 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 21 sites in Australia, Canada, Denmark, United Kingdom, France 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 10 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 Adverse Events (TEAEs) (time frame: Day 1 to end of study (up to Day 182 [32 mg cohort]/ Day 210 [all other cohorts])). 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?

On the ClinicalTrials.gov record for NCT03225833, which also lists 2 linked publications.

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 10 Feb 2022.