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TerminatedNCT04617847Updated Feb 9, 2022Results posted

Open-label Extension Study to Evaluate the Safety and Tolerability of WVE-120101 in Patients With Huntington's Disease

A Phase 1/2 interventional study of WVE-120101 in Huntington Disease, sponsored by Wave Life Sciences USA, Inc.. Terminated at 18 sites in 7 countries. Per ClinicalTrials.gov, last updated 2022-02-09.

Sponsored by Wave Life Sciences USA, Inc. · Phase 1/2, Interventional, and Treatment

Why this study was terminated
Lack of efficacy

From the registry’s dates

  • Registered 6 months after the study started (first participant enrolled Apr 2020, registered Oct 2020).
Phase
Phase 1/2
Study type
Interventional
Enrollment
27
Allocation
Not applicable
Sex
All
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Study 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.

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Conditions studied

  • Huntington Disease

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In context

Huntington Disease

290 studies on the registry are indexed under Huntington Disease; 49 are open to participants now.

This study's enrollment of 27 is below the median of 40 across 206 interventional studies indexed under Huntington Disease.

Browse Huntington Disease studies →

Lead sponsor

Wave Life Sciences USA, Inc. is the lead sponsor of 9 studies on the registry; 3 are open to participants now.

Counted across the registry records on this site, refreshed daily.

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Who can participate

Ages eligible
Child (0–17), Adult (18–64), Older adult (65+)
Sexes eligible
All
Accepts healthy volunteers
No

Eligibility criteria

Key Inclusion Criteria:

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

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.
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Study design

Phase
Phase 1 / Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
27 participants (actual)

Study arms

  • Experimental
    WVE-120101 (Dose A)

    Drug: WVE-120101

Interventions

  • DrugWVE-120101

    WVE-120101 is a stereopure antisense oligonucleotide (ASO). It is administered monthly via intrathecal injection.

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What researchers measure

Primary outcomes

  1. 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)

  2. 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

  3. 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

  4. 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

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Results

Posted Feb 9, 2022

Participant flow

Participant flow — Overall Study
Milestone4 mg WVE-12010116 mg WVE-120101
Started324
Dose modified to 16 mg wve-12010131
Dose modified to 32 mg wve-12010105
Completed00
Not completed324
Withdrew: Withdrawal by subject10
Withdrew: Adverse event02
Withdrew: Death01
Withdrew: Termination of study by sponsor221

Outcome measures

PrimarySafety: 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)
Reported as:
Count of participants · Participants
Safety: Number of Patients With Treatment-emergent AEs (TEAEs)
Participants4 mg WVE-12010116 mg WVE-12010132 mg WVE-120101
Safety: Number of Patients With Treatment-emergent AEs (TEAEs)2172
PrimarySafety: 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
Reported as:
Count of participants · Participants
Safety: Number of Patients With a Severe TEAE
Participants4 mg WVE-12010116 mg WVE-12010132 mg WVE-120101
Safety: Number of Patients With a Severe TEAE050
PrimarySafety: Number of Patients With Serious TEAEs
Time frame:
First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment
Reported as:
Count of participants · Participants
Safety: Number of Patients With Serious TEAEs
Participants4 mg WVE-12010116 mg WVE-12010132 mg WVE-120101
Safety: Number of Patients With Serious TEAEs031
PrimarySafety 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
Reported as:
Count of participants · Participants
Safety and Tolerability: Number of Patients Who Withdraw Due to TEAEs
Participants4 mg WVE-12010116 mg WVE-12010132 mg WVE-120101
Safety and Tolerability: Number of Patients Who Withdraw Due to TEAEs010

Adverse events

Collected over First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment). Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
4 mg WVE-1201010/3 (0%)0/3 (0%)2/3 (66.7%)
16 mg WVE-1201012/27 (7.4%)3/27 (11.1%)16/27 (59.3%)
32 mg WVE-1201010/5 (0%)1/5 (20%)2/5 (40%)
Most frequent serious events
Most frequent serious events
Event4 mg WVE-12010116 mg WVE-12010132 mg WVE-120101
Balance disorderNervous system disorders0/30/271/5
Lung Cancer MetastaticNeoplasms benign, malignant and unspecified (incl cysts and polyps)0/31/270/5
NeoplasmNeoplasms benign, malignant and unspecified (incl cysts and polyps)0/31/270/5
HeadacheNervous system disorders0/31/270/5
Head InjuryInjury, poisoning and procedural complications0/31/270/5
Completed SuicidePsychiatric disorders0/31/270/5
Most frequent other events
Showing 10 of 12
Most frequent other events
Event4 mg WVE-12010116 mg WVE-12010132 mg WVE-120101
CSF Protein IncreasedInvestigations1/31/270/5
CSF white blood cell count increasedInvestigations1/30/270/5
Lymphocyte count increasedInvestigations1/30/270/5
DizzinessNervous system disorders0/37/270/5
Restless legs syndromeNervous system disorders0/30/271/5
Balance disorderNervous system disorders0/30/271/5
HeadacheNervous system disorders0/35/270/5
FallInjury, poisoning and procedural complications0/33/270/5
Procedural PainInjury, poisoning and procedural complications0/33/270/5
Back painMusculoskeletal and connective tissue disorders0/33/270/5

Baseline characteristics

Age, Categorical
Age, Categorical(Participants)4 mg WVE-12010116 mg WVE-120101Total
<=18 years000
Between 18 and 65 years32427
>=65 years000
Sex: Female, Male
Sex: Female, Male(Participants)4 mg WVE-12010116 mg WVE-120101Total
Female21517
Male1910
Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)4 mg WVE-12010116 mg WVE-120101Total
Hispanic or Latino000
Not Hispanic or Latino32427
Unknown or Not Reported000
Race (NIH/OMB)
Race (NIH/OMB)(Participants)4 mg WVE-12010116 mg WVE-120101Total
American Indian or Alaska Native000
Asian000
Native Hawaiian or Other Pacific Islander000
Black or African American000
White32427
More than one race000
Unknown or Not Reported000
Region of Enrollment
Region of Enrollment(participants)4 mg WVE-12010116 mg WVE-120101Total
Canada336
Denmark022
Poland099
Australia088
France022
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Study locations

18 sites
  • Westmead Hospital
    Sydney, New South Wales 2145, Australia
  • Royal Brisbane & Women's Hospital
    Herston, Queensland QLD 4006, Australia
  • Royal Melbourne Hospital
    Carlton, Victoria 3053, Australia
  • Monash Health
    Clayton, Victoria 3168, Australia
  • Alfred Health
    Melbourne, Victoria 3004, Australia
  • Calvary Health Care Bethlehem
    Parkdale, Victoria 3195, Australia
  • North Metropolitan Health Service
    Perth, Western Australia 6910, Australia
  • University of Alberta
    Edmonton, Alberta T6G 2B7, Canada
  • Centre Hospitalier de l-Universite de Montreal
    Montreal, Quebec H2X019, Canada
  • Aarhus Universitets Hospital
    Aarhus, 8200, Denmark
  • Rigshospitalet
    Copenhagen, 2100, Denmark
  • Hospital Henri Mondor
    Créteil, 94010, France
  • Institut du Cerveau et de la Moelle Epinière
    Paris, 75646, France
  • George-Huntington-Institut GmbH
    Münster, 48149, Germany
  • Szpital Sw. Wojciecha
    Gdansk, 80-462, Poland
  • Instytut Psychiatrii i Neurologii
    Warsaw, 02-957, Poland
  • Royal Devon and Exeter Hospital NHS Trust
    Exeter, Devon EX2 5DW, United Kingdom
  • Queen Elizabeth University Hospital - PPDS
    Glasgow, Glasgow City G12 0XH, United Kingdom
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References and documents

Study documents

  • Protocol and statistical analysis plan · Mar 31, 2020

Documents are hosted by the registry — open the source record to download them.

Individual participant data

Plan to share: No

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Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Feb 9, 2022, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
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Registry details

Key details

Study ID
NCT04617847
Lead sponsor
Wave Life Sciences USA, Inc.
Responsible party
Sponsor
First posted
Nov 5, 2020
Start date
Apr 13, 2020
Primary completion
May 3, 2021
Completion
May 3, 2021
Results posted
Feb 9, 2022
Last update
Feb 9, 2022

Study contacts

Medical Director, MD
study director · Wave Life Sciences

Oversight

Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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This study is terminated, as verified in Jan 2022. You cannot join it, but the record below documents what was studied.

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