PRECISION-HD1: Safety and Tolerability of WVE-120101 in Patients With Huntington's Disease
Sponsor
Wave Life Sciences Ltd. (Industry)
Overall Status
Terminated
CT.gov ID
NCT03225833
Collaborator
(none)
61
21
5
45.8
2.9
0.1
Study Details
Study Description
Brief 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 nucleotide polymorphism (SNP) rs362307 (SNP1).
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Phase 1/Phase 2 |
Study Design
Study Type:
Interventional
Actual Enrollment
:
61 participants
Allocation:
Randomized
Intervention Model:
Sequential Assignment
Masking:
Double (Participant, Investigator)
Primary Purpose:
Treatment
Official Title:
A Multicenter, Randomized, Double-blind, Placebo-controlled, Phase 1b/2a Study of WVE-120101 Administered Intrathecally in Patients With Huntington's Disease
Actual Study Start Date
:
Jul 17, 2017
Actual Primary Completion Date
:
May 11, 2021
Actual Study Completion Date
:
May 11, 2021
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: WVE-120101 (Dose A) or placebo
|
Drug: WVE-120101
WVE-120101 is a stereopure antisense oligonucleotide (ASO)
Drug: Placebo
0.9% Sodium Chloride
|
| Experimental: WVE-120101 (Dose B) or placebo
|
Drug: WVE-120101
WVE-120101 is a stereopure antisense oligonucleotide (ASO)
Drug: Placebo
0.9% Sodium Chloride
|
| Experimental: WVE-120101 (Dose C) or placebo
|
Drug: WVE-120101
WVE-120101 is a stereopure antisense oligonucleotide (ASO)
Drug: Placebo
0.9% Sodium Chloride
|
| Experimental: WVE-120101 (Dose D) or placebo
|
Drug: WVE-120101
WVE-120101 is a stereopure antisense oligonucleotide (ASO)
Drug: Placebo
0.9% Sodium Chloride
|
| Experimental: WVE-120101 (Dose E) or placebo
|
Drug: WVE-120101
WVE-120101 is a stereopure antisense oligonucleotide (ASO)
Drug: Placebo
0.9% Sodium Chloride
|
Outcome Measures
Primary Outcome Measures
- Safety: Number of Patients With Treatment-emergent Adverse Events (TEAEs) [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 [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 [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 [Day 1 to end of study (up to Day 182 [32 mg cohort]/ Day 210 [all other cohorts])]
Secondary Outcome Measures
- Pharmacokinetics (PK): Maximum Observed Concentration (Cmax) [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.]
Cmax of WVE-120101 in plasma
- PK: Time of Occurrence of Cmax (Tmax) [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.]
tmax of WVE-120101 in plasma
- PK: Area Under the Plasma Concentration-time Curve (AUClast) [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.]
AUClast from time 0 to the last quantifiable concentration of WVE-120101 in plasma
- PK: Terminal Elimination Half Life [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.]
Terminal elimination half life of WVE-120101 in plasma (t1/2)
- Pharmacodynamics [Day 1 to last observation - up to Day 140 (32 mg cohort) or Day 196 (all other cohorts)]
Percentage change from baseline in concentration of mutant huntingtin (mHTT) protein in CSF
- Clinical Effects: Total Functional Capacity (TFC) [Day 1 to last observation - up to Day 140 (32 mg cohort) or Day 196 (all other cohorts)]
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).
Eligibility Criteria
Criteria
Ages Eligible for Study:
25 Years
to 65 Years
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No
Key Inclusion Criteria:
-
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:
-
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
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Westmead Hospital | Sidney | New South Wales | Australia | 2145 |
| 2 | Royal Brisbane & Women's Hospital | Herston | Queensland | Australia | QLD 4006 |
| 3 | Royal Melbourne Hospital | Carlton | Victoria | Australia | 3053 |
| 4 | Monash Health | Clayton | Victoria | Australia | 3168 |
| 5 | Alfred Health | Melbourne | Victoria | Australia | 3004 |
| 6 | Calvary Health Care Bethlehem | Parkdale | Victoria | Australia | 3195 |
| 7 | North Metropolitan Health Service | Perth | Western Australia | Australia | 6910 |
| 8 | University of Alberta | Edmonton | Alberta | Canada | T6G 2B7 |
| 9 | Centre For Movement Disorders | Toronto | Ontario | Canada | M3B 2S7 |
| 10 | Center Hospitalier de l'Universite de Montreal | Montreal | Quebec | Canada | H2X0A9 |
| 11 | Aarhus Universitets Hospital | Aarhus | Denmark | 8200 | |
| 12 | Rigshospitalet | Copenhagen | Denmark | 2100 | |
| 13 | Odense University Hospital and University of Southern Denmark | Odense | Denmark | 5000 | |
| 14 | Hospital Henri Mondor | Créteil | France | 94010 | |
| 15 | Institut du Cerveau et de la Moelle Epinière | Paris | France | 75646 | |
| 16 | George-Huntington-Institut GmbH | Muenster | Germany | 48149 | |
| 17 | Szpital Sw. Wojciecha | Gdańsk | Poland | 80-462 | |
| 18 | Instytut Psychiatrii i Neurologii | Warsaw | Poland | 02-957 | |
| 19 | Royal Devon and Exeter Hospital NHS Trust | Exeter | Devon | United Kingdom | EX2 5DW |
| 20 | Queen Elizabeth University Hospital - PPDS | Glasgow | Glasgow City | United Kingdom | G12 0XH |
| 21 | Royal Liverpool University Hospital | Liverpool | United Kingdom | L7 8XP |
Sponsors and Collaborators
- Wave Life Sciences Ltd.
Investigators
- Study Director: Medical Director, MD, Wave Life Sciences
Study Documents (Full-Text)
More Information
Publications
None provided.Responsible Party:
Wave Life Sciences Ltd.
ClinicalTrials.gov Identifier:
NCT03225833
Other Study ID Numbers:
- WVE-HDSNP1-001
First Posted:
Jul 21, 2017
Last Update Posted:
Feb 10, 2022
Last Verified:
Feb 1, 2022
Studies a U.S. FDA-regulated Drug Product:
No
Studies a U.S. FDA-regulated Device Product:
No
Additional relevant MeSH terms:
Study Results
Participant Flow
| Recruitment Details | |
|---|---|
| Pre-assignment Detail |
| Arm/Group Title | Pooled Placebo | WVE-120101 (2 mg) | WVE-120101 (4 mg) | WVE-120101 (8 mg) | WVE-120101 (16 mg) | WVE-120101 (32 mg) |
|---|---|---|---|---|---|---|
| Arm/Group Description | Placebo: 0.9% Sodium Chloride | WVE-120101: WVE-120101 is a stereopure antisense oligonucleotide (ASO) | WVE-120101: WVE-120101 is a stereopure antisense oligonucleotide (ASO) | WVE-120101: WVE-120101 is a stereopure antisense oligonucleotide (ASO) | WVE-120101: WVE-120101 is a stereopure antisense oligonucleotide (ASO) | WVE-120101: WVE-120101 is a stereopure antisense oligonucleotide (ASO) |
| Period Title: Overall Study | ||||||
| STARTED | 16 | 9 | 9 | 9 | 8 | 10 |
| Single Dose Period Only | 1 | 1 | 2 | 0 | 0 | 4 |
| Multiple Dose Period Only | 0 | 0 | 0 | 0 | 0 | 1 |
| Single Dose and Multiple Dose Periods | 15 | 8 | 7 | 9 | 8 | 5 |
| COMPLETED | 11 | 8 | 7 | 9 | 7 | 0 |
| NOT COMPLETED | 5 | 1 | 2 | 0 | 1 | 10 |
Baseline Characteristics
| Arm/Group Title | Pooled Placebo | WVE-120101 (2 mg) | WVE-120101 (4 mg) | WVE-120101 (8 mg) | WVE-120101 (16 mg) | WVE-120101 (32 mg) | Total |
|---|---|---|---|---|---|---|---|
| Arm/Group Description | Placebo: 0.9% Sodium Chloride | WVE-120101: WVE-120101 is a stereopure antisense oligonucleotide (ASO) | WVE-120101: WVE-120101 is a stereopure antisense oligonucleotide (ASO) | WVE-120101: WVE-120101 is a stereopure antisense oligonucleotide (ASO) | WVE-120101: WVE-120101 is a stereopure antisense oligonucleotide (ASO) | WVE-120101: WVE-120101 is a stereopure antisense oligonucleotide (ASO) | Total of all reporting groups |
| Overall Participants | 16 | 9 | 9 | 9 | 8 | 10 | 61 |
| Age (Count of Participants) | |||||||
| <=18 years |
0
0%
|
0
0%
|
0
0%
|
0
0%
|
0
0%
|
0
0%
|
0
0%
|
| Between 18 and 65 years |
15
93.8%
|
9
100%
|
9
100%
|
9
100%
|
8
100%
|
10
100%
|
60
98.4%
|
| >=65 years |
1
6.3%
|
0
0%
|
0
0%
|
0
0%
|
0
0%
|
0
0%
|
1
1.6%
|
| Sex: Female, Male (Count of Participants) | |||||||
| Female |
7
43.8%
|
3
33.3%
|
5
55.6%
|
6
66.7%
|
1
12.5%
|
7
70%
|
29
47.5%
|
| Male |
9
56.3%
|
6
66.7%
|
4
44.4%
|
3
33.3%
|
7
87.5%
|
3
30%
|
32
52.5%
|
| Ethnicity (NIH/OMB) (Count of Participants) | |||||||
| Hispanic or Latino |
0
0%
|
0
0%
|
0
0%
|
0
0%
|
0
0%
|
0
0%
|
0
0%
|
| Not Hispanic or Latino |
16
100%
|
9
100%
|
9
100%
|
9
100%
|
8
100%
|
10
100%
|
61
100%
|
| Unknown or Not Reported |
0
0%
|
0
0%
|
0
0%
|
0
0%
|
0
0%
|
0
0%
|
0
0%
|
| Race (NIH/OMB) (Count of Participants) | |||||||
| American Indian or Alaska Native |
0
0%
|
0
0%
|
0
0%
|
0
0%
|
0
0%
|
0
0%
|
0
0%
|
| Asian |
0
0%
|
0
0%
|
0
0%
|
0
0%
|
0
0%
|
0
0%
|
0
0%
|
| Native Hawaiian or Other Pacific Islander |
0
0%
|
0
0%
|
0
0%
|
0
0%
|
0
0%
|
0
0%
|
0
0%
|
| Black or African American |
0
0%
|
0
0%
|
0
0%
|
0
0%
|
0
0%
|
0
0%
|
0
0%
|
| White |
16
100%
|
9
100%
|
9
100%
|
9
100%
|
8
100%
|
10
100%
|
61
100%
|
| More than one race |
0
0%
|
0
0%
|
0
0%
|
0
0%
|
0
0%
|
0
0%
|
0
0%
|
| Unknown or Not Reported |
0
0%
|
0
0%
|
0
0%
|
0
0%
|
0
0%
|
0
0%
|
0
0%
|
| Region of Enrollment (participants) [Number] | |||||||
| Canada |
4
25%
|
2
22.2%
|
2
22.2%
|
1
11.1%
|
0
0%
|
3
30%
|
12
19.7%
|
| Denmark |
0
0%
|
0
0%
|
0
0%
|
1
11.1%
|
1
12.5%
|
0
0%
|
2
3.3%
|
| Poland |
4
25%
|
7
77.8%
|
5
55.6%
|
1
11.1%
|
2
25%
|
0
0%
|
19
31.1%
|
| Australia |
4
25%
|
0
0%
|
0
0%
|
6
66.7%
|
3
37.5%
|
4
40%
|
17
27.9%
|
| France |
1
6.3%
|
0
0%
|
0
0%
|
0
0%
|
1
12.5%
|
2
20%
|
4
6.6%
|
| Germany |
2
12.5%
|
0
0%
|
0
0%
|
0
0%
|
1
12.5%
|
1
10%
|
4
6.6%
|
| United Kingdom |
1
6.3%
|
0
0%
|
2
22.2%
|
0
0%
|
0
0%
|
0
0%
|
3
4.9%
|
| Time since initial diagnosis (Years) [Mean (Standard Deviation) ] | |||||||
| Mean (Standard Deviation) [Years] |
7
(6.93)
|
4.9
(4.28)
|
3.4
(6.37)
|
3.2
(3.03)
|
6.6
(6.09)
|
8.7
(7.26)
|
5.4
(5.80)
|
| Age at Disease Onset (Years) [Mean (Standard Deviation) ] | |||||||
| Mean (Standard Deviation) [Years] |
40.75
(11.079)
|
37.33
(7.826)
|
42.89
(9.280)
|
46.11
(6.254)
|
44.88
(12.495)
|
44.60
(10.865)
|
43.16
(9.625)
|
| Diagnosis Stage (Count of Participants) | |||||||
| Stage 1 |
9
56.3%
|
7
77.8%
|
3
33.3%
|
3
33.3%
|
4
50%
|
7
70%
|
33
54.1%
|
| Stage 2 |
7
43.8%
|
2
22.2%
|
6
66.7%
|
6
66.7%
|
4
50%
|
3
30%
|
28
45.9%
|
Outcome Measures
| Title | Safety: Number of Patients With Treatment-emergent Adverse Events (TEAEs) |
|---|---|
| Description | All TEAEs reported or observed during the study, including TEAEs resulting from concurrent illnesses, reactions to concurrent medications, or progression of disease states |
| Time Frame | Day 1 to end of study (up to Day 182 [32 mg cohort]/ Day 210 [all other cohorts]) |
Outcome Measure Data
| Analysis Population Description |
|---|
| [Not Specified] |
| Arm/Group Title | Pooled Placebo | WVE-120101 (2 mg) | WVE-120101 (4 mg) | WVE-120101 (8 mg) | WVE-120101 (16 mg) | WVE-120101 (32 mg) |
|---|---|---|---|---|---|---|
| Arm/Group Description | Placebo: 0.9% Sodium Chloride | WVE-120101: WVE-120101 is a stereopure antisense oligonucleotide (ASO) | WVE-120101: WVE-120101 is a stereopure antisense oligonucleotide (ASO) | WVE-120101: WVE-120101 is a stereopure antisense oligonucleotide (ASO) | WVE-120101: WVE-120101 is a stereopure antisense oligonucleotide (ASO) | WVE-120101: WVE-120101 is a stereopure antisense oligonucleotide (ASO) |
| Measure Participants | 16 | 9 | 9 | 9 | 8 | 10 |
| Count of Participants [Participants] |
12
75%
|
8
88.9%
|
8
88.9%
|
9
100%
|
7
87.5%
|
9
90%
|
| Title | Safety: Severity of Adverse Events |
|---|---|
| Description | 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 |
| Time Frame | Day 1 to end of study (up to Day 182 [32 mg cohort]/ Day 210 [all other cohorts]) |
Outcome Measure Data
| Analysis Population Description |
|---|
| [Not Specified] |
| Arm/Group Title | Pooled Placebo | WVE-120101 (2 mg) | WVE-120101 (4 mg) | WVE-120101 (8 mg) | WVE-120101 (16 mg) | WVE-120101 (32 mg) |
|---|---|---|---|---|---|---|
| Arm/Group Description | Placebo: 0.9% Sodium Chloride | WVE-120101: WVE-120101 is a stereopure antisense oligonucleotide (ASO) | WVE-120101: WVE-120101 is a stereopure antisense oligonucleotide (ASO) | WVE-120101: WVE-120101 is a stereopure antisense oligonucleotide (ASO) | WVE-120101: WVE-120101 is a stereopure antisense oligonucleotide (ASO) | WVE-120101: WVE-120101 is a stereopure antisense oligonucleotide (ASO) |
| Measure Participants | 16 | 9 | 9 | 9 | 8 | 10 |
| Count of Participants [Participants] |
1
6.3%
|
2
22.2%
|
1
11.1%
|
1
11.1%
|
0
0%
|
5
50%
|
| Title | Safety: Number of Patients With Serious TEAEs |
|---|---|
| Description | 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. |
| Time Frame | Day 1 to end of study (up to Day 182 [32 mg cohort]/ Day 210 [all other cohorts]) |
Outcome Measure Data
| Analysis Population Description |
|---|
| [Not Specified] |
| Arm/Group Title | Pooled Placebo | WVE-120101 (2 mg) | WVE-120101 (4 mg) | WVE-120101 (8 mg) | WVE-120101 (16 mg) | WVE-120101 (32 mg) |
|---|---|---|---|---|---|---|
| Arm/Group Description | Placebo: 0.9% Sodium Chloride | WVE-120101: WVE-120101 is a stereopure antisense oligonucleotide (ASO) | WVE-120101: WVE-120101 is a stereopure antisense oligonucleotide (ASO) | WVE-120101: WVE-120101 is a stereopure antisense oligonucleotide (ASO) | WVE-120101: WVE-120101 is a stereopure antisense oligonucleotide (ASO) | WVE-120101: WVE-120101 is a stereopure antisense oligonucleotide (ASO) |
| Measure Participants | 16 | 9 | 9 | 9 | 8 | 10 |
| Count of Participants [Participants] |
0
0%
|
2
22.2%
|
1
11.1%
|
0
0%
|
0
0%
|
4
40%
|
| Title | Safety and Tolerability: Number of Patients Who Withdraw Due to TEAEs |
|---|---|
| Description | |
| Time Frame | Day 1 to end of study (up to Day 182 [32 mg cohort]/ Day 210 [all other cohorts]) |
Outcome Measure Data
| Analysis Population Description |
|---|
| [Not Specified] |
| Arm/Group Title | Pooled Placebo | WVE-120101 (2 mg) | WVE-120101 (4 mg) | WVE-120101 (8 mg) | WVE-120101 (16 mg) | WVE-120101 (32 mg) |
|---|---|---|---|---|---|---|
| Arm/Group Description | Placebo: 0.9% Sodium Chloride | WVE-120101: WVE-120101 is a stereopure antisense oligonucleotide (ASO) | WVE-120101: WVE-120101 is a stereopure antisense oligonucleotide (ASO) | WVE-120101: WVE-120101 is a stereopure antisense oligonucleotide (ASO) | WVE-120101: WVE-120101 is a stereopure antisense oligonucleotide (ASO) | WVE-120101: WVE-120101 is a stereopure antisense oligonucleotide (ASO) |
| Measure Participants | 16 | 9 | 9 | 9 | 8 | 10 |
| Count of Participants [Participants] |
0
0%
|
1
11.1%
|
2
22.2%
|
0
0%
|
0
0%
|
2
20%
|
| Title | Pharmacokinetics (PK): Maximum Observed Concentration (Cmax) |
|---|---|
| Description | Cmax of WVE-120101 in plasma |
| 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. |
Outcome Measure Data
| Analysis Population Description |
|---|
| The PK population consists of all treated patients in the safety population with at least 1 post-dose plasma or CSF WVE-120101 concentration measurement. The number of overall participants analyzed represents the number with at least 1 postdose plasma or CSF measurement at Day 1. The overall number of patients was different for Day 112, and represents patients with at least 1 postdose plasma or CSF measurement at Day 112. |
| Arm/Group Title | WVE-120101 (2 mg) | WVE-120101 (4 mg) | WVE-120101 (8 mg) | WVE-120101 (16 mg) | WVE-120101 (32 mg) |
|---|---|---|---|---|---|
| Arm/Group Description | WVE-120101: WVE-120101 is a stereopure antisense oligonucleotide (ASO) | WVE-120101: WVE-120101 is a stereopure antisense oligonucleotide (ASO) | WVE-120101: WVE-120101 is a stereopure antisense oligonucleotide (ASO) | WVE-120101: WVE-120101 is a stereopure antisense oligonucleotide (ASO) | WVE-120101: WVE-120101 is a stereopure antisense oligonucleotide (ASO) |
| Measure Participants | 9 | 9 | 9 | 8 | 9 |
| Day 1 |
7.70
(7.901)
|
23.54
(18.139)
|
32.82
(22.964)
|
184.48
(209.470)
|
229.01
(168.330)
|
| Day 112 |
13.296
(6.057)
|
14.27
(12.574)
|
| Title | PK: Time of Occurrence of Cmax (Tmax) |
|---|---|
| Description | tmax of WVE-120101 in plasma |
| 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. |
Outcome Measure Data
| Analysis Population Description |
|---|
| The PK population consists of all treated patients in the safety population with at least 1 post-dose plasma or CSF WVE-120101 concentration measurement. The number of overall participants analyzed represents the number with at least 1 postdose plasma or CSF measurement at Day 1. The overall number of patients was different for Day 112, and represents patients with at least 1 postdose plasma or CSF measurement at Day 112. |
| Arm/Group Title | WVE-120101 (2 mg) | WVE-120101 (4 mg) | WVE-120101 (8 mg) | WVE-120101 (16 mg) | WVE-120101 (32 mg) |
|---|---|---|---|---|---|
| Arm/Group Description | WVE-120101: WVE-120101 is a stereopure antisense oligonucleotide (ASO) | WVE-120101: WVE-120101 is a stereopure antisense oligonucleotide (ASO) | WVE-120101: WVE-120101 is a stereopure antisense oligonucleotide (ASO) | WVE-120101: WVE-120101 is a stereopure antisense oligonucleotide (ASO) | WVE-120101: WVE-120101 is a stereopure antisense oligonucleotide (ASO) |
| Measure Participants | 9 | 9 | 9 | 8 | 9 |
| Day 1 |
1.34
(1.103)
|
1.58
(1.081)
|
2.66
(1.391)
|
2.71
(3.068)
|
4.61
(7.054)
|
| Day 112 |
1.99
(0.934)
|
2.23
(1.175)
|
| Title | PK: Area Under the Plasma Concentration-time Curve (AUClast) |
|---|---|
| Description | AUClast from time 0 to the last quantifiable concentration of WVE-120101 in plasma |
| 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. |
Outcome Measure Data
| Analysis Population Description |
|---|
| The PK population consists of all treated patients in the safety population with at least 1 post-dose plasma or CSF WVE-120101 concentration measurement. The number of overall participants analyzed represents the number with at least 1 postdose plasma or CSF measurement at Day 1. The overall number of patients was different for Day 112, and represents patients with at least 1 postdose plasma or CSF measurement at Day 112. |
| Arm/Group Title | WVE-120101 (2 mg) | WVE-120101 (4 mg) | WVE-120101 (8 mg) | WVE-120101 (16 mg) | WVE-120101 (32 mg) |
|---|---|---|---|---|---|
| Arm/Group Description | WVE-120101: WVE-120101 is a stereopure antisense oligonucleotide (ASO) | WVE-120101: WVE-120101 is a stereopure antisense oligonucleotide (ASO) | WVE-120101: WVE-120101 is a stereopure antisense oligonucleotide (ASO) | WVE-120101: WVE-120101 is a stereopure antisense oligonucleotide (ASO) | WVE-120101: WVE-120101 is a stereopure antisense oligonucleotide (ASO) |
| Measure Participants | 9 | 9 | 9 | 8 | 9 |
| Day 1 |
35.20
(16.037)
|
90.77
(50.672)
|
255.14
(98.342)
|
1133.74
(551.997)
|
1968.31
(1188.173)
|
| Day 112 |
36.19
(20.135)
|
49.54
(34.735)
|
| Title | PK: Terminal Elimination Half Life |
|---|---|
| Description | Terminal elimination half life of WVE-120101 in plasma (t1/2) |
| 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. |
Outcome Measure Data
| Analysis Population Description |
|---|
| The t1/2 value was calculated using data from patients who had at least 3 postdose concentration values in the terminal phase. No patients had sufficient postdose samples at Day 112 to calculate t1/2 values. |
| Arm/Group Title | WVE-120101 (2 mg) | WVE-120101 (4 mg) | WVE-120101 (8 mg) | WVE-120101 (16 mg) | WVE-120101 (32 mg) |
|---|---|---|---|---|---|
| Arm/Group Description | WVE-120101: WVE-120101 is a stereopure antisense oligonucleotide (ASO) | WVE-120101: WVE-120101 is a stereopure antisense oligonucleotide (ASO) | WVE-120101: WVE-120101 is a stereopure antisense oligonucleotide (ASO) | WVE-120101: WVE-120101 is a stereopure antisense oligonucleotide (ASO) | WVE-120101: WVE-120101 is a stereopure antisense oligonucleotide (ASO) |
| Measure Participants | 0 | 0 | 3 | 3 | 6 |
| Median (Full Range) [hours] |
8.12
|
12.30
|
14.38
|
| Title | Pharmacodynamics |
|---|---|
| Description | Percentage change from baseline in concentration of mutant huntingtin (mHTT) protein in CSF |
| Time Frame | Day 1 to last observation - up to Day 140 (32 mg cohort) or Day 196 (all other cohorts) |
Outcome Measure Data
| Analysis Population Description |
|---|
| [Not Specified] |
| Arm/Group Title | Pooled Placebo | WVE-120101 (2 mg) | WVE-120101 (4 mg) | WVE-120101 (8 mg) | WVE-120101 (16 mg) | WVE-120101 (32 mg) |
|---|---|---|---|---|---|---|
| Arm/Group Description | Placebo: 0.9% Sodium Chloride | WVE-120101: WVE-120101 is a stereopure antisense oligonucleotide (ASO) | WVE-120101: WVE-120101 is a stereopure antisense oligonucleotide (ASO) | WVE-120101: WVE-120101 is a stereopure antisense oligonucleotide (ASO) | WVE-120101: WVE-120101 is a stereopure antisense oligonucleotide (ASO) | WVE-120101: WVE-120101 is a stereopure antisense oligonucleotide (ASO) |
| Measure Participants | 16 | 9 | 9 | 9 | 8 | 10 |
| Median (Inter-Quartile Range) [Percent change from baseline] |
-6.62
|
-5.20
|
-12.33
|
-8.58
|
-11.73
|
-9.13
|
| Title | Clinical Effects: Total Functional Capacity (TFC) |
|---|---|
| Description | 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). |
| Time Frame | Day 1 to last observation - up to Day 140 (32 mg cohort) or Day 196 (all other cohorts) |
Outcome Measure Data
| Analysis Population Description |
|---|
| [Not Specified] |
| Arm/Group Title | Pooled Placebo | WVE-120101 (2 mg) | WVE-120101 (4 mg) | WVE-120101 (8 mg) | WVE-120101 (16 mg) | WVE-120101 (32 mg) |
|---|---|---|---|---|---|---|
| Arm/Group Description | Placebo: 0.9% Sodium Chloride | WVE-120101: WVE-120101 is a stereopure antisense oligonucleotide (ASO) | WVE-120101: WVE-120101 is a stereopure antisense oligonucleotide (ASO) | WVE-120101: WVE-120101 is a stereopure antisense oligonucleotide (ASO) | WVE-120101: WVE-120101 is a stereopure antisense oligonucleotide (ASO) | WVE-120101: WVE-120101 is a stereopure antisense oligonucleotide (ASO) |
| Measure Participants | 16 | 9 | 9 | 9 | 8 | 10 |
| Median (Inter-Quartile Range) [% Change from Baseline] |
0.00
|
0.00
|
0.00
|
0.00
|
4.17
|
0.00
|
Adverse Events
| Time Frame | Day 1 to end of study (Day 196 [32 mg cohort] or Day 210 [all other cohorts]) | |||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Adverse Event Reporting Description | ||||||||||||
| Arm/Group Title | Pooled Placebo | WVE-120101 (2 mg) | WVE-120101 (4 mg) | WVE-120101 (8 mg) | WVE-120101 (16 mg) | WVE-120101 (32 mg) | ||||||
| Arm/Group Description | Placebo: 0.9% Sodium Chloride | WVE-120101: WVE-120101 is a stereopure antisense oligonucleotide (ASO) | WVE-120101: WVE-120101 is a stereopure antisense oligonucleotide (ASO) | WVE-120101: WVE-120101 is a stereopure antisense oligonucleotide (ASO) | WVE-120101: WVE-120101 is a stereopure antisense oligonucleotide (ASO) | WVE-120101: WVE-120101 is a stereopure antisense oligonucleotide (ASO) | ||||||
All Cause Mortality |
||||||||||||
| Pooled Placebo | WVE-120101 (2 mg) | WVE-120101 (4 mg) | WVE-120101 (8 mg) | WVE-120101 (16 mg) | WVE-120101 (32 mg) | |||||||
| Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | |
| Total | 0/16 (0%) | 0/9 (0%) | 0/9 (0%) | 0/9 (0%) | 0/8 (0%) | 0/10 (0%) | ||||||
Serious Adverse Events |
||||||||||||
| Pooled Placebo | WVE-120101 (2 mg) | WVE-120101 (4 mg) | WVE-120101 (8 mg) | WVE-120101 (16 mg) | WVE-120101 (32 mg) | |||||||
| Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | |
| Total | 0/16 (0%) | 2/9 (22.2%) | 1/9 (11.1%) | 0/9 (0%) | 0/8 (0%) | 4/10 (40%) | ||||||
| General disorders | ||||||||||||
| Gait disturbance | 0/16 (0%) | 0/9 (0%) | 0/9 (0%) | 0/9 (0%) | 0/8 (0%) | 1/10 (10%) | ||||||
| Injury, poisoning and procedural complications | ||||||||||||
| Fall | 0/16 (0%) | 0/9 (0%) | 0/9 (0%) | 0/9 (0%) | 0/8 (0%) | 1/10 (10%) | ||||||
| Skull Fracture | 0/16 (0%) | 0/9 (0%) | 1/9 (11.1%) | 0/9 (0%) | 0/8 (0%) | 0/10 (0%) | ||||||
| Subdural haematoma | 0/16 (0%) | 0/9 (0%) | 1/9 (11.1%) | 0/9 (0%) | 0/8 (0%) | 0/10 (0%) | ||||||
| Ankle fracture | 0/16 (0%) | 0/9 (0%) | 0/9 (0%) | 0/9 (0%) | 0/8 (0%) | 1/10 (10%) | ||||||
| Musculoskeletal and connective tissue disorders | ||||||||||||
| Muscular weakness | 0/16 (0%) | 0/9 (0%) | 0/9 (0%) | 0/9 (0%) | 0/8 (0%) | 1/10 (10%) | ||||||
| Neoplasms benign, malignant and unspecified (incl cysts and polyps) | ||||||||||||
| Adenocarcinoma of colon | 0/16 (0%) | 1/9 (11.1%) | 0/9 (0%) | 0/9 (0%) | 0/8 (0%) | 0/10 (0%) | ||||||
| Nervous system disorders | ||||||||||||
| Ataxia | 0/16 (0%) | 0/9 (0%) | 0/9 (0%) | 0/9 (0%) | 0/8 (0%) | 1/10 (10%) | ||||||
| Dysarthria | 0/16 (0%) | 0/9 (0%) | 0/9 (0%) | 0/9 (0%) | 0/8 (0%) | 1/10 (10%) | ||||||
| Psychiatric disorders | ||||||||||||
| Agitation | 0/16 (0%) | 0/9 (0%) | 0/9 (0%) | 0/9 (0%) | 0/8 (0%) | 1/10 (10%) | ||||||
| Disorientation | 0/16 (0%) | 0/9 (0%) | 0/9 (0%) | 0/9 (0%) | 0/8 (0%) | 1/10 (10%) | ||||||
| Aggression | 0/16 (0%) | 1/9 (11.1%) | 0/9 (0%) | 0/9 (0%) | 0/8 (0%) | 0/10 (0%) | ||||||
Other (Not Including Serious) Adverse Events |
||||||||||||
| Pooled Placebo | WVE-120101 (2 mg) | WVE-120101 (4 mg) | WVE-120101 (8 mg) | WVE-120101 (16 mg) | WVE-120101 (32 mg) | |||||||
| Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | Affected / at Risk (%) | # Events | |
| Total | 12/16 (75%) | 8/9 (88.9%) | 8/9 (88.9%) | 9/9 (100%) | 7/8 (87.5%) | 9/10 (90%) | ||||||
| Blood and lymphatic system disorders | ||||||||||||
| Leukopenia | 0/16 (0%) | 0/9 (0%) | 1/9 (11.1%) | 0/9 (0%) | 0/8 (0%) | 0/10 (0%) | ||||||
| Thrombocytopenia | 0/16 (0%) | 0/9 (0%) | 1/9 (11.1%) | 0/9 (0%) | 0/8 (0%) | 0/10 (0%) | ||||||
| Ear and labyrinth disorders | ||||||||||||
| Ear discomfort | 0/16 (0%) | 0/9 (0%) | 0/9 (0%) | 0/9 (0%) | 1/8 (12.5%) | 0/10 (0%) | ||||||
| Eye disorders | ||||||||||||
| Dry eye | 0/16 (0%) | 0/9 (0%) | 0/9 (0%) | 1/9 (11.1%) | 0/8 (0%) | 0/10 (0%) | ||||||
| Gastrointestinal disorders | ||||||||||||
| Vomiting | 0/16 (0%) | 1/9 (11.1%) | 0/9 (0%) | 1/9 (11.1%) | 1/8 (12.5%) | 0/10 (0%) | ||||||
| Nausea | 1/16 (6.3%) | 1/9 (11.1%) | 0/9 (0%) | 1/9 (11.1%) | 0/8 (0%) | 0/10 (0%) | ||||||
| Constipation | 0/16 (0%) | 0/9 (0%) | 0/9 (0%) | 0/9 (0%) | 0/8 (0%) | 1/10 (10%) | ||||||
| Diarrhoea | 1/16 (6.3%) | 0/9 (0%) | 0/9 (0%) | 0/9 (0%) | 0/8 (0%) | 0/10 (0%) | ||||||
| General disorders | ||||||||||||
| Gait disturbance | 0/16 (0%) | 0/9 (0%) | 0/9 (0%) | 0/9 (0%) | 0/8 (0%) | 2/10 (20%) | ||||||
| Administration site bruise | 0/16 (0%) | 0/9 (0%) | 0/9 (0%) | 0/9 (0%) | 1/8 (12.5%) | 0/10 (0%) | ||||||
| Administration site pain | 0/16 (0%) | 0/9 (0%) | 0/9 (0%) | 0/9 (0%) | 1/8 (12.5%) | 0/10 (0%) | ||||||
| Asthenia | 0/16 (0%) | 1/9 (11.1%) | 0/9 (0%) | 0/9 (0%) | 0/8 (0%) | 0/10 (0%) | ||||||
| Fatigue | 1/16 (6.3%) | 0/9 (0%) | 0/9 (0%) | 0/9 (0%) | 0/8 (0%) | 1/10 (10%) | ||||||
| Influenza like illness | 0/16 (0%) | 0/9 (0%) | 0/9 (0%) | 0/9 (0%) | 1/8 (12.5%) | 0/10 (0%) | ||||||
| Pain | 0/16 (0%) | 0/9 (0%) | 1/9 (11.1%) | 0/9 (0%) | 0/8 (0%) | 0/10 (0%) | ||||||
| Puncture site haemorrhage | 0/16 (0%) | 0/9 (0%) | 0/9 (0%) | 1/9 (11.1%) | 0/8 (0%) | 0/10 (0%) | ||||||
| Injection site pain | 1/16 (6.3%) | 0/9 (0%) | 0/9 (0%) | 0/9 (0%) | 0/8 (0%) | 0/10 (0%) | ||||||
| Vessel puncture site bruise | 1/16 (6.3%) | 0/9 (0%) | 0/9 (0%) | 0/9 (0%) | 0/8 (0%) | 0/10 (0%) | ||||||
| Vessel puncture site pain | 1/16 (6.3%) | 0/9 (0%) | 0/9 (0%) | 0/9 (0%) | 0/8 (0%) | 0/10 (0%) | ||||||
| Infections and infestations | ||||||||||||
| Viral upper respiratory tract infection | 1/16 (6.3%) | 2/9 (22.2%) | 1/9 (11.1%) | 1/9 (11.1%) | 1/8 (12.5%) | 0/10 (0%) | ||||||
| Urinary tract infection | 0/16 (0%) | 0/9 (0%) | 0/9 (0%) | 1/9 (11.1%) | 0/8 (0%) | 1/10 (10%) | ||||||
| Conjunctivitis | 0/16 (0%) | 0/9 (0%) | 1/9 (11.1%) | 0/9 (0%) | 0/8 (0%) | 0/10 (0%) | ||||||
| Ear infection | 0/16 (0%) | 0/9 (0%) | 0/9 (0%) | 0/9 (0%) | 1/8 (12.5%) | 0/10 (0%) | ||||||
| Lower respiratory tract infection | 0/16 (0%) | 0/9 (0%) | 0/9 (0%) | 1/9 (11.1%) | 0/8 (0%) | 0/10 (0%) | ||||||
| Respiratory tract infection | 0/16 (0%) | 0/9 (0%) | 1/9 (11.1%) | 0/9 (0%) | 0/8 (0%) | 0/10 (0%) | ||||||
| Influenza | 1/16 (6.3%) | 0/9 (0%) | 0/9 (0%) | 0/9 (0%) | 0/8 (0%) | 0/10 (0%) | ||||||
| Upper respiratory tract infection | 1/16 (6.3%) | 1/9 (11.1%) | 0/9 (0%) | 0/9 (0%) | 0/8 (0%) | 0/10 (0%) | ||||||
| Injury, poisoning and procedural complications | ||||||||||||
| Procedural pain | 3/16 (18.8%) | 0/9 (0%) | 2/9 (22.2%) | 3/9 (33.3%) | 1/8 (12.5%) | 2/10 (20%) | ||||||
| Fall | 0/16 (0%) | 1/9 (11.1%) | 1/9 (11.1%) | 1/9 (11.1%) | 2/8 (25%) | 1/10 (10%) | ||||||
| Post lumbar puncture syndrome | 2/16 (12.5%) | 1/9 (11.1%) | 2/9 (22.2%) | 0/9 (0%) | 1/8 (12.5%) | 0/10 (0%) | ||||||
| Contusion | 1/16 (6.3%) | 1/9 (11.1%) | 0/9 (0%) | 0/9 (0%) | 0/8 (0%) | 0/10 (0%) | ||||||
| Eye contusion | 0/16 (0%) | 0/9 (0%) | 0/9 (0%) | 0/9 (0%) | 0/8 (0%) | 1/10 (10%) | ||||||
| Hand fracture | 0/16 (0%) | 0/9 (0%) | 0/9 (0%) | 1/9 (11.1%) | 0/8 (0%) | 0/10 (0%) | ||||||
| Head injury | 0/16 (0%) | 0/9 (0%) | 1/9 (11.1%) | 0/9 (0%) | 0/8 (0%) | 0/10 (0%) | ||||||
| Post procedural discomfort | 0/16 (0%) | 0/9 (0%) | 1/9 (11.1%) | 0/9 (0%) | 0/8 (0%) | 0/10 (0%) | ||||||
| Procedural headache | 0/16 (0%) | 1/9 (11.1%) | 0/9 (0%) | 0/9 (0%) | 0/8 (0%) | 0/10 (0%) | ||||||
| Skull fracture | 0/16 (0%) | 0/9 (0%) | 1/9 (11.1%) | 0/9 (0%) | 0/8 (0%) | 0/10 (0%) | ||||||
| Thermal burn | 0/16 (0%) | 0/9 (0%) | 0/9 (0%) | 0/9 (0%) | 1/8 (12.5%) | 0/10 (0%) | ||||||
| Wound | 0/16 (0%) | 0/9 (0%) | 0/9 (0%) | 0/9 (0%) | 1/8 (12.5%) | 0/10 (0%) | ||||||
| Foot fracture | 1/16 (6.3%) | 0/9 (0%) | 0/9 (0%) | 0/9 (0%) | 0/8 (0%) | 0/10 (0%) | ||||||
| Post procedural contusion | 1/16 (6.3%) | 0/9 (0%) | 0/9 (0%) | 0/9 (0%) | 0/8 (0%) | 0/10 (0%) | ||||||
| Procedural nausea | 1/16 (6.3%) | 0/9 (0%) | 0/9 (0%) | 0/9 (0%) | 0/8 (0%) | 0/10 (0%) | ||||||
| Procedural vomiting | 1/16 (6.3%) | 0/9 (0%) | 0/9 (0%) | 0/9 (0%) | 0/8 (0%) | 0/10 (0%) | ||||||
| Investigations | ||||||||||||
| Blood creatine phosphokinase increased | 0/16 (0%) | 0/9 (0%) | 0/9 (0%) | 1/9 (11.1%) | 1/8 (12.5%) | 1/10 (10%) | ||||||
| CSF lymphocyte count increase | 0/16 (0%) | 0/9 (0%) | 0/9 (0%) | 0/9 (0%) | 0/8 (0%) | 1/10 (10%) | ||||||
| CSF protein increased | 0/16 (0%) | 0/9 (0%) | 0/9 (0%) | 0/9 (0%) | 1/8 (12.5%) | 0/10 (0%) | ||||||
| CSF white blood cell count increased | 0/16 (0%) | 0/9 (0%) | 1/9 (11.1%) | 0/9 (0%) | 0/8 (0%) | 0/10 (0%) | ||||||
| Haemoglobin decreased | 0/16 (0%) | 0/9 (0%) | 1/9 (11.1%) | 0/9 (0%) | 0/8 (0%) | 0/10 (0%) | ||||||
| Hepatic enzyme increased | 0/16 (0%) | 1/9 (11.1%) | 0/9 (0%) | 0/9 (0%) | 0/8 (0%) | 0/10 (0%) | ||||||
| Lymphocyte count decreased | 0/16 (0%) | 0/9 (0%) | 0/9 (0%) | 0/9 (0%) | 0/8 (0%) | 1/10 (10%) | ||||||
| Neutrophil count increased | 0/16 (0%) | 0/9 (0%) | 0/9 (0%) | 0/9 (0%) | 0/8 (0%) | 1/10 (10%) | ||||||
| Platelet count increased | 0/16 (0%) | 0/9 (0%) | 1/9 (11.1%) | 0/9 (0%) | 0/8 (0%) | 0/10 (0%) | ||||||
| White blood cell count increased | 1/16 (6.3%) | 0/9 (0%) | 0/9 (0%) | 0/9 (0%) | 0/8 (0%) | 1/10 (10%) | ||||||
| Blood bilirubin increased | 1/16 (6.3%) | 0/9 (0%) | 0/9 (0%) | 0/9 (0%) | 0/8 (0%) | 0/10 (0%) | ||||||
| Metabolism and nutrition disorders | ||||||||||||
| Increased appetite | 1/16 (6.3%) | 0/9 (0%) | 0/9 (0%) | 0/9 (0%) | 0/8 (0%) | 0/10 (0%) | ||||||
| Musculoskeletal and connective tissue disorders | ||||||||||||
| Back pain | 3/16 (18.8%) | 3/9 (33.3%) | 1/9 (11.1%) | 2/9 (22.2%) | 3/8 (37.5%) | 0/10 (0%) | ||||||
| Osteoarthritis | 0/16 (0%) | 1/9 (11.1%) | 1/9 (11.1%) | 0/9 (0%) | 0/8 (0%) | 0/10 (0%) | ||||||
| Arthralgia | 2/16 (12.5%) | 0/9 (0%) | 0/9 (0%) | 0/9 (0%) | 0/8 (0%) | 1/10 (10%) | ||||||
| Limb discomfort | 1/16 (6.3%) | 0/9 (0%) | 0/9 (0%) | 0/9 (0%) | 1/8 (12.5%) | 0/10 (0%) | ||||||
| Musculoskeletal pain | 0/16 (0%) | 1/9 (11.1%) | 0/9 (0%) | 0/9 (0%) | 0/8 (0%) | 0/10 (0%) | ||||||
| Musculoskeletal stiffness | 0/16 (0%) | 0/9 (0%) | 0/9 (0%) | 1/9 (11.1%) | 0/8 (0%) | 0/10 (0%) | ||||||
| Neck pain | 0/16 (0%) | 0/9 (0%) | 0/9 (0%) | 0/9 (0%) | 1/8 (12.5%) | 0/10 (0%) | ||||||
| Pain in extremity | 1/16 (6.3%) | 0/9 (0%) | 0/9 (0%) | 0/9 (0%) | 1/8 (12.5%) | 0/10 (0%) | ||||||
| Spinal pain | 0/16 (0%) | 0/9 (0%) | 0/9 (0%) | 0/9 (0%) | 1/8 (12.5%) | 0/10 (0%) | ||||||
| Muscle spasms | 2/16 (12.5%) | 0/9 (0%) | 0/9 (0%) | 0/9 (0%) | 0/8 (0%) | 0/10 (0%) | ||||||
| Muscular weakness | 0/16 (0%) | 0/9 (0%) | 0/9 (0%) | 0/9 (0%) | 0/8 (0%) | 1/10 (10%) | ||||||
| Nervous system disorders | ||||||||||||
| Headache | 5/16 (31.3%) | 0/9 (0%) | 1/9 (11.1%) | 2/9 (22.2%) | 2/8 (25%) | 4/10 (40%) | ||||||
| Dizziness | 0/16 (0%) | 1/9 (11.1%) | 0/9 (0%) | 1/9 (11.1%) | 1/8 (12.5%) | 4/10 (40%) | ||||||
| Dysarthria | 0/16 (0%) | 0/9 (0%) | 0/9 (0%) | 0/9 (0%) | 0/8 (0%) | 2/10 (20%) | ||||||
| Ataxia | 0/16 (0%) | 0/9 (0%) | 0/9 (0%) | 0/9 (0%) | 0/8 (0%) | 1/10 (10%) | ||||||
| Balance disorder | 0/16 (0%) | 0/9 (0%) | 0/9 (0%) | 0/9 (0%) | 1/8 (12.5%) | 1/10 (10%) | ||||||
| Chorea | 1/16 (6.3%) | 0/9 (0%) | 0/9 (0%) | 2/9 (22.2%) | 0/8 (0%) | 0/10 (0%) | ||||||
| Hyperreflexia | 0/16 (0%) | 1/9 (11.1%) | 0/9 (0%) | 1/9 (11.1%) | 0/8 (0%) | 0/10 (0%) | ||||||
| Amnesia | 0/16 (0%) | 0/9 (0%) | 0/9 (0%) | 0/9 (0%) | 0/8 (0%) | 1/10 (10%) | ||||||
| Dysgeusia | 0/16 (0%) | 0/9 (0%) | 0/9 (0%) | 0/9 (0%) | 1/8 (12.5%) | 0/10 (0%) | ||||||
| Hypoaesthesia | 1/16 (6.3%) | 1/9 (11.1%) | 0/9 (0%) | 0/9 (0%) | 0/8 (0%) | 0/10 (0%) | ||||||
| Hyporeflexia | 0/16 (0%) | 0/9 (0%) | 0/9 (0%) | 0/9 (0%) | 0/8 (0%) | 1/10 (10%) | ||||||
| Lethargy | 0/16 (0%) | 0/9 (0%) | 0/9 (0%) | 0/9 (0%) | 1/8 (12.5%) | 0/10 (0%) | ||||||
| Lumbosacral radiculopathy | 0/16 (0%) | 1/9 (11.1%) | 0/9 (0%) | 0/9 (0%) | 0/8 (0%) | 0/10 (0%) | ||||||
| Paraesthesia | 1/16 (6.3%) | 0/9 (0%) | 0/9 (0%) | 1/9 (11.1%) | 0/8 (0%) | 0/10 (0%) | ||||||
| Pleocytosis | 0/16 (0%) | 1/9 (11.1%) | 0/9 (0%) | 0/9 (0%) | 0/8 (0%) | 0/10 (0%) | ||||||
| Post-traumatic headache | 0/16 (0%) | 1/9 (11.1%) | 0/9 (0%) | 0/9 (0%) | 0/8 (0%) | 0/10 (0%) | ||||||
| Radicular pain | 0/16 (0%) | 0/9 (0%) | 1/9 (11.1%) | 0/9 (0%) | 0/8 (0%) | 0/10 (0%) | ||||||
| Syncope | 0/16 (0%) | 1/9 (11.1%) | 0/9 (0%) | 0/9 (0%) | 0/8 (0%) | 0/10 (0%) | ||||||
| Migraine | 1/16 (6.3%) | 0/9 (0%) | 0/9 (0%) | 0/9 (0%) | 0/8 (0%) | 0/10 (0%) | ||||||
| Sensory disturbance | 1/16 (6.3%) | 0/9 (0%) | 0/9 (0%) | 0/9 (0%) | 0/8 (0%) | 0/10 (0%) | ||||||
| Psychiatric disorders | ||||||||||||
| Anxiety | 2/16 (12.5%) | 0/9 (0%) | 0/9 (0%) | 1/9 (11.1%) | 1/8 (12.5%) | 0/10 (0%) | ||||||
| Irritability | 1/16 (6.3%) | 1/9 (11.1%) | 0/9 (0%) | 0/9 (0%) | 1/8 (12.5%) | 0/10 (0%) | ||||||
| Affect lability | 0/16 (0%) | 1/9 (11.1%) | 0/9 (0%) | 0/9 (0%) | 0/8 (0%) | 0/10 (0%) | ||||||
| Bradyphrenia | 0/16 (0%) | 0/9 (0%) | 0/9 (0%) | 0/9 (0%) | 0/8 (0%) | 1/10 (10%) | ||||||
| Delirium | 0/16 (0%) | 0/9 (0%) | 0/9 (0%) | 0/9 (0%) | 0/8 (0%) | 1/10 (10%) | ||||||
| Depression | 0/16 (0%) | 1/9 (11.1%) | 0/9 (0%) | 0/9 (0%) | 0/8 (0%) | 0/10 (0%) | ||||||
| Depression suicidal | 0/16 (0%) | 0/9 (0%) | 1/9 (11.1%) | 0/9 (0%) | 0/8 (0%) | 0/10 (0%) | ||||||
| Emotional disorder | 0/16 (0%) | 0/9 (0%) | 0/9 (0%) | 0/9 (0%) | 1/8 (12.5%) | 0/10 (0%) | ||||||
| Insomnia | 0/16 (0%) | 0/9 (0%) | 1/9 (11.1%) | 0/9 (0%) | 0/8 (0%) | 0/10 (0%) | ||||||
| Restlessness | 1/16 (6.3%) | 0/9 (0%) | 0/9 (0%) | 0/9 (0%) | 0/8 (0%) | 0/10 (0%) | ||||||
| Renal and urinary disorders | ||||||||||||
| Haematuria | 0/16 (0%) | 0/9 (0%) | 1/9 (11.1%) | 0/9 (0%) | 0/8 (0%) | 0/10 (0%) | ||||||
| Urinary retention | 0/16 (0%) | 0/9 (0%) | 0/9 (0%) | 0/9 (0%) | 0/8 (0%) | 1/10 (10%) | ||||||
| Reproductive system and breast disorders | ||||||||||||
| Ovarian cyst | 1/16 (6.3%) | 0/9 (0%) | 0/9 (0%) | 0/9 (0%) | 0/8 (0%) | 0/10 (0%) | ||||||
| Respiratory, thoracic and mediastinal disorders | ||||||||||||
| Dyspnoea | 0/16 (0%) | 0/9 (0%) | 0/9 (0%) | 1/9 (11.1%) | 0/8 (0%) | 0/10 (0%) | ||||||
| Epistaxis | 0/16 (0%) | 0/9 (0%) | 0/9 (0%) | 1/9 (11.1%) | 0/8 (0%) | 0/10 (0%) | ||||||
| Rhinorrhea | 1/16 (6.3%) | 0/9 (0%) | 0/9 (0%) | 0/9 (0%) | 0/8 (0%) | 0/10 (0%) | ||||||
| Skin and subcutaneous tissue disorders | ||||||||||||
| Rash vesicular | 0/16 (0%) | 0/9 (0%) | 0/9 (0%) | 1/9 (11.1%) | 0/8 (0%) | 0/10 (0%) | ||||||
| Alopecia | 1/16 (6.3%) | 0/9 (0%) | 0/9 (0%) | 0/9 (0%) | 0/8 (0%) | 0/10 (0%) | ||||||
| Dermatitis contact | 1/16 (6.3%) | 0/9 (0%) | 0/9 (0%) | 0/9 (0%) | 0/8 (0%) | 0/10 (0%) | ||||||
| Vascular disorders | ||||||||||||
| Hypotension | 0/16 (0%) | 0/9 (0%) | 0/9 (0%) | 0/9 (0%) | 1/8 (12.5%) | 1/10 (10%) | ||||||
| Hot flush | 0/16 (0%) | 0/9 (0%) | 0/9 (0%) | 1/9 (11.1%) | 0/8 (0%) | 0/10 (0%) | ||||||
| Hypertension | 1/16 (6.3%) | 1/9 (11.1%) | 0/9 (0%) | 0/9 (0%) | 0/8 (0%) | 0/10 (0%) | ||||||
| Flushing | 1/16 (6.3%) | 0/9 (0%) | 0/9 (0%) | 0/9 (0%) | 0/8 (0%) | 0/10 (0%) | ||||||
Limitations/Caveats
[Not Specified]
More Information
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
| Name/Title | Medical Director |
|---|---|
| Organization | Wave Life Sciences |
| Phone | 855-215-4687 |
| clinicaltrials@wavelifesci.com |
Responsible Party:
Wave Life Sciences Ltd.
ClinicalTrials.gov Identifier:
NCT03225833
Other Study ID Numbers:
- WVE-HDSNP1-001
First Posted:
Jul 21, 2017
Last Update Posted:
Feb 10, 2022
Last Verified:
Feb 1, 2022