Trial Outcomes & Findings for Open-label Extension Study to Evaluate the Safety and Tolerability of WVE-120101 in Patients With Huntington's Disease (NCT NCT04617847)
NCT ID: NCT04617847
Last Updated: 2022-02-09
Results Overview
Recruitment status
TERMINATED
Study phase
PHASE1/PHASE2
Target enrollment
27 participants
Primary outcome timeframe
First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment)
Results posted on
2022-02-09
Participant Flow
In the original OLE study protocol, patients who were in the 2 and 4 mg dose cohorts in the Phase 1b/2a study were to be enrolled into the 4 or 8 mg dose group in this study. Patients in the 8 and 16 mg dose cohorts in WVE-HDSNP1-001 continued to receive that dose. As of Amendment 1.0 of this protocol, all new patients were to be enrolled at a dose level of at least 16 mg. All current patents were dose modified to 16 or 32 mg.
Participant milestones
| Measure |
4 mg WVE-120101
Enrolled at 4 mg WVE-120101 dose level
|
16 mg WVE-120101
Enrolled at 16 mg WVE-120101 dose level
|
|---|---|---|
|
Overall Study
STARTED
|
3
|
24
|
|
Overall Study
Dose Modified to 16 mg WVE-120101
|
3
|
1
|
|
Overall Study
Dose Modified to 32 mg WVE-120101
|
0
|
5
|
|
Overall Study
COMPLETED
|
0
|
0
|
|
Overall Study
NOT COMPLETED
|
3
|
24
|
Reasons for withdrawal
| Measure |
4 mg WVE-120101
Enrolled at 4 mg WVE-120101 dose level
|
16 mg WVE-120101
Enrolled at 16 mg WVE-120101 dose level
|
|---|---|---|
|
Overall Study
Withdrawal by Subject
|
1
|
0
|
|
Overall Study
Adverse Event
|
0
|
2
|
|
Overall Study
Death
|
0
|
1
|
|
Overall Study
Termination of Study by Sponsor
|
2
|
21
|
Baseline Characteristics
Open-label Extension Study to Evaluate the Safety and Tolerability of WVE-120101 in Patients With Huntington's Disease
Baseline characteristics by cohort
| Measure |
4 mg WVE-120101
n=3 Participants
Enrolled at 4 mg WVE-120101 dose level
|
16 mg WVE-120101
n=24 Participants
Enrolled at 12 mg WVE-120101 dose level
|
Total
n=27 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
3 Participants
n=5 Participants
|
24 Participants
n=7 Participants
|
27 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
2 Participants
n=5 Participants
|
15 Participants
n=7 Participants
|
17 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
1 Participants
n=5 Participants
|
9 Participants
n=7 Participants
|
10 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
3 Participants
n=5 Participants
|
24 Participants
n=7 Participants
|
27 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
3 Participants
n=5 Participants
|
24 Participants
n=7 Participants
|
27 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
Canada
|
3 participants
n=5 Participants
|
3 participants
n=7 Participants
|
6 participants
n=5 Participants
|
|
Region of Enrollment
Denmark
|
0 participants
n=5 Participants
|
2 participants
n=7 Participants
|
2 participants
n=5 Participants
|
|
Region of Enrollment
Poland
|
0 participants
n=5 Participants
|
9 participants
n=7 Participants
|
9 participants
n=5 Participants
|
|
Region of Enrollment
Australia
|
0 participants
n=5 Participants
|
8 participants
n=7 Participants
|
8 participants
n=5 Participants
|
|
Region of Enrollment
France
|
0 participants
n=5 Participants
|
2 participants
n=7 Participants
|
2 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment)Population: Patients treated at more than one dose level (e.g., initial dose and after dose modification) are included in each applicable dose group. Adverse events are counted in the dose the patient was receiving at the time of onset.
Outcome measures
| Measure |
4 mg WVE-120101
n=3 Participants
Patients who received 4 mg WVE-120101 at any point in the study
|
16 mg WVE-120101
n=27 Participants
Patients who received 16 mg WVE-120101 at any point in the study
|
32 mg WVE-120101
n=5 Participants
Patients who received 32 mg WVE-120101 at any point in the study
|
|---|---|---|---|
|
Safety: Number of Patients With Treatment-emergent AEs (TEAEs)
|
2 Participants
|
17 Participants
|
2 Participants
|
PRIMARY outcome
Timeframe: First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatmentPopulation: Patients treated at more than one dose level (e.g., initial dose and after dose modification) are included in each applicable dose group. Adverse events are counted in the dose the patient was receiving at the time of onset.
Outcome measures
| Measure |
4 mg WVE-120101
n=3 Participants
Patients who received 4 mg WVE-120101 at any point in the study
|
16 mg WVE-120101
n=27 Participants
Patients who received 16 mg WVE-120101 at any point in the study
|
32 mg WVE-120101
n=5 Participants
Patients who received 32 mg WVE-120101 at any point in the study
|
|---|---|---|---|
|
Safety: Number of Patients With a Severe TEAE
|
0 Participants
|
5 Participants
|
0 Participants
|
PRIMARY outcome
Timeframe: First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatmentPopulation: Patients treated at more than one dose level (e.g., initial dose and after dose modification) are included in each applicable dose group. Adverse events are counted in the dose the patient was receiving at the time of onset.
Outcome measures
| Measure |
4 mg WVE-120101
n=3 Participants
Patients who received 4 mg WVE-120101 at any point in the study
|
16 mg WVE-120101
n=27 Participants
Patients who received 16 mg WVE-120101 at any point in the study
|
32 mg WVE-120101
n=5 Participants
Patients who received 32 mg WVE-120101 at any point in the study
|
|---|---|---|---|
|
Safety: Number of Patients With Serious TEAEs
|
0 Participants
|
3 Participants
|
1 Participants
|
PRIMARY outcome
Timeframe: First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatmentPopulation: Patients treated at more than one dose level (e.g., initial dose and after dose modification) are included in each applicable dose group. Adverse events are counted in the dose the patient was receiving at the time of onset.
Outcome measures
| Measure |
4 mg WVE-120101
n=3 Participants
Patients who received 4 mg WVE-120101 at any point in the study
|
16 mg WVE-120101
n=27 Participants
Patients who received 16 mg WVE-120101 at any point in the study
|
32 mg WVE-120101
n=5 Participants
Patients who received 32 mg WVE-120101 at any point in the study
|
|---|---|---|---|
|
Safety and Tolerability: Number of Patients Who Withdraw Due to TEAEs
|
0 Participants
|
1 Participants
|
0 Participants
|
Adverse Events
4 mg WVE-120101
Serious events: 0 serious events
Other events: 2 other events
Deaths: 0 deaths
16 mg WVE-120101
Serious events: 3 serious events
Other events: 16 other events
Deaths: 2 deaths
32 mg WVE-120101
Serious events: 1 serious events
Other events: 2 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
4 mg WVE-120101
n=3 participants at risk
Patients who received 4 mg WVE-120101 at any point in the study
|
16 mg WVE-120101
n=27 participants at risk
Patients who received 16 mg WVE-120101 at any point in the study
|
32 mg WVE-120101
n=5 participants at risk
Patients who received 32 mg WVE-120101 at any point in the study
|
|---|---|---|---|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Lung Cancer Metastatic
|
0.00%
0/3 • First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment)
|
3.7%
1/27 • First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment)
|
0.00%
0/5 • First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment)
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Neoplasm
|
0.00%
0/3 • First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment)
|
3.7%
1/27 • First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment)
|
0.00%
0/5 • First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment)
|
|
Nervous system disorders
Balance disorder
|
0.00%
0/3 • First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment)
|
0.00%
0/27 • First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment)
|
20.0%
1/5 • First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment)
|
|
Nervous system disorders
Headache
|
0.00%
0/3 • First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment)
|
3.7%
1/27 • First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment)
|
0.00%
0/5 • First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment)
|
|
Injury, poisoning and procedural complications
Head Injury
|
0.00%
0/3 • First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment)
|
3.7%
1/27 • First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment)
|
0.00%
0/5 • First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment)
|
|
Psychiatric disorders
Completed Suicide
|
0.00%
0/3 • First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment)
|
3.7%
1/27 • First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment)
|
0.00%
0/5 • First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment)
|
Other adverse events
| Measure |
4 mg WVE-120101
n=3 participants at risk
Patients who received 4 mg WVE-120101 at any point in the study
|
16 mg WVE-120101
n=27 participants at risk
Patients who received 16 mg WVE-120101 at any point in the study
|
32 mg WVE-120101
n=5 participants at risk
Patients who received 32 mg WVE-120101 at any point in the study
|
|---|---|---|---|
|
Injury, poisoning and procedural complications
Fall
|
0.00%
0/3 • First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment)
|
11.1%
3/27 • First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment)
|
0.00%
0/5 • First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment)
|
|
Injury, poisoning and procedural complications
Procedural Pain
|
0.00%
0/3 • First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment)
|
11.1%
3/27 • First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment)
|
0.00%
0/5 • First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment)
|
|
Investigations
CSF Protein Increased
|
33.3%
1/3 • First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment)
|
3.7%
1/27 • First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment)
|
0.00%
0/5 • First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment)
|
|
Investigations
CSF white blood cell count increased
|
33.3%
1/3 • First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment)
|
0.00%
0/27 • First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment)
|
0.00%
0/5 • First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment)
|
|
Investigations
Lymphocyte count increased
|
33.3%
1/3 • First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment)
|
0.00%
0/27 • First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment)
|
0.00%
0/5 • First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment)
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
0.00%
0/3 • First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment)
|
11.1%
3/27 • First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment)
|
0.00%
0/5 • First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment)
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
0.00%
0/3 • First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment)
|
7.4%
2/27 • First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment)
|
0.00%
0/5 • First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment)
|
|
Nervous system disorders
Dizziness
|
0.00%
0/3 • First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment)
|
25.9%
7/27 • First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment)
|
0.00%
0/5 • First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment)
|
|
Nervous system disorders
Headache
|
0.00%
0/3 • First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment)
|
18.5%
5/27 • First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment)
|
0.00%
0/5 • First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment)
|
|
Nervous system disorders
Dysarthria
|
0.00%
0/3 • First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment)
|
7.4%
2/27 • First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment)
|
0.00%
0/5 • First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment)
|
|
Nervous system disorders
Restless legs syndrome
|
0.00%
0/3 • First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment)
|
0.00%
0/27 • First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment)
|
20.0%
1/5 • First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment)
|
|
Nervous system disorders
Balance disorder
|
0.00%
0/3 • First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment)
|
0.00%
0/27 • First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment)
|
20.0%
1/5 • First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment)
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place