Trial Outcomes & Findings for A Study to Evaluate the Safety and Efficacy of PTC518 in Participants With Huntington's Disease (HD) (NCT NCT05358717)
NCT ID: NCT05358717
Last Updated: 2026-01-13
Results Overview
An adverse event (AE) was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. A TEAE was defined as an AE that had an onset date or date of worsening on or after the first dose of study drug. A summary of other non-serious AEs and all serious adverse events (SAEs), regardless of causality is located in the 'Reported AE section'.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
159 participants
Primary outcome timeframe
Baseline up to Month 18
Results posted on
2026-01-13
Participant Flow
Participants were randomized to receive placebo, PTC518 5 milligrams (mg), or PTC518 10 mg. While the Independent Data Monitoring Committee authorized opening of the 20 mg dosing arm, the sponsor elected not to open PTC518 20 mg dosing arm for enrollment.
Participant milestones
| Measure |
Placebo
Participants received placebo matched to PTC518 tablets once daily (QD) orally for 12 months.
|
PTC518 5 mg
Participants received 5 mg tablet of PTC518 QD orally for 12 months.
|
PTC518 10 mg
Participants received 10 mg tablet of PTC518 QD orally for 12 months.
|
|---|---|---|---|
|
Overall Study
STARTED
|
53
|
52
|
54
|
|
Overall Study
Received at Least 1 Dose of Study Drug
|
53
|
52
|
54
|
|
Overall Study
COMPLETED
|
51
|
47
|
51
|
|
Overall Study
NOT COMPLETED
|
2
|
5
|
3
|
Reasons for withdrawal
| Measure |
Placebo
Participants received placebo matched to PTC518 tablets once daily (QD) orally for 12 months.
|
PTC518 5 mg
Participants received 5 mg tablet of PTC518 QD orally for 12 months.
|
PTC518 10 mg
Participants received 10 mg tablet of PTC518 QD orally for 12 months.
|
|---|---|---|---|
|
Overall Study
Adverse Event
|
0
|
1
|
1
|
|
Overall Study
Death
|
1
|
1
|
0
|
|
Overall Study
Withdrawal by Subject
|
1
|
1
|
2
|
|
Overall Study
Study Paused by Sponsor in United States
|
0
|
2
|
0
|
Baseline Characteristics
A Study to Evaluate the Safety and Efficacy of PTC518 in Participants With Huntington's Disease (HD)
Baseline characteristics by cohort
| Measure |
Placebo
n=53 Participants
Participants received placebo matched to PTC518 tablets once daily (QD) orally for 12 months.
|
PTC518 5 mg
n=52 Participants
Participants received 5 mg tablet of PTC518 QD orally for 12 months.
|
PTC518 10 mg
n=54 Participants
Participants received 10 mg tablet of PTC518 QD orally for 12 months.
|
Total
n=159 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Continuous
|
48.6 years
STANDARD_DEVIATION 9.59 • n=210 Participants
|
49.2 years
STANDARD_DEVIATION 9.04 • n=19 Participants
|
47.8 years
STANDARD_DEVIATION 9.10 • n=123 Participants
|
48.5 years
STANDARD_DEVIATION 9.20 • n=123 Participants
|
|
Sex: Female, Male
Female
|
33 Participants
n=210 Participants
|
26 Participants
n=19 Participants
|
29 Participants
n=123 Participants
|
88 Participants
n=123 Participants
|
|
Sex: Female, Male
Male
|
20 Participants
n=210 Participants
|
26 Participants
n=19 Participants
|
25 Participants
n=123 Participants
|
71 Participants
n=123 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
2 Participants
n=210 Participants
|
3 Participants
n=19 Participants
|
1 Participants
n=123 Participants
|
6 Participants
n=123 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
51 Participants
n=210 Participants
|
49 Participants
n=19 Participants
|
53 Participants
n=123 Participants
|
153 Participants
n=123 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=210 Participants
|
0 Participants
n=19 Participants
|
0 Participants
n=123 Participants
|
0 Participants
n=123 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=210 Participants
|
0 Participants
n=19 Participants
|
0 Participants
n=123 Participants
|
0 Participants
n=123 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=210 Participants
|
1 Participants
n=19 Participants
|
0 Participants
n=123 Participants
|
1 Participants
n=123 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=210 Participants
|
0 Participants
n=19 Participants
|
0 Participants
n=123 Participants
|
0 Participants
n=123 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=210 Participants
|
0 Participants
n=19 Participants
|
0 Participants
n=123 Participants
|
0 Participants
n=123 Participants
|
|
Race (NIH/OMB)
White
|
52 Participants
n=210 Participants
|
47 Participants
n=19 Participants
|
52 Participants
n=123 Participants
|
151 Participants
n=123 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=210 Participants
|
0 Participants
n=19 Participants
|
0 Participants
n=123 Participants
|
0 Participants
n=123 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=210 Participants
|
4 Participants
n=19 Participants
|
2 Participants
n=123 Participants
|
7 Participants
n=123 Participants
|
PRIMARY outcome
Timeframe: Baseline up to Month 18Population: Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
An adverse event (AE) was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. A TEAE was defined as an AE that had an onset date or date of worsening on or after the first dose of study drug. A summary of other non-serious AEs and all serious adverse events (SAEs), regardless of causality is located in the 'Reported AE section'.
Outcome measures
| Measure |
Placebo
n=53 Participants
Participants received placebo matched to PTC518 tablets once daily (QD) orally for 12 months.
|
PTC518 5 mg
n=52 Participants
Participants received 5 mg tablet of PTC518 QD orally for 12 months.
|
PTC518 10 mg
n=54 Participants
Participants received 10 mg tablet of PTC518 QD orally for 12 months.
|
|---|---|---|---|
|
Number of Participants With Treatment-emergent Adverse Events (TEAEs)
|
46 Participants
|
43 Participants
|
49 Participants
|
PRIMARY outcome
Timeframe: Baseline, Month 3Population: ITT Population included all randomized participants who took at least 1 dose of the study drug.
Least square (LS) mean and 95% confidence interval (CI) were calculated using mixed-model repeated measures (MMRM). The blood tHTT protein value was log-transformed before fitting the MMRM model. The LS means from MMRM model were back-transformed to present geometric mean percent changes.
Outcome measures
| Measure |
Placebo
n=53 Participants
Participants received placebo matched to PTC518 tablets once daily (QD) orally for 12 months.
|
PTC518 5 mg
n=52 Participants
Participants received 5 mg tablet of PTC518 QD orally for 12 months.
|
PTC518 10 mg
n=54 Participants
Participants received 10 mg tablet of PTC518 QD orally for 12 months.
|
|---|---|---|---|
|
Percent Change From Baseline in Geometric Mean Blood tHTT Protein at Month 3
|
-1.40 percent change
95% Confidence Interval -7.25 • Interval -7.25 to 4.82
|
-17.36 percent change
95% Confidence Interval -22.36 • Interval -22.36 to -12.05
|
-28.67 percent change
95% Confidence Interval -32.74 • Interval -32.74 to -24.36
|
SECONDARY outcome
Timeframe: Baseline, Month 12Population: ITT Population included all randomized participants who took at least 1 dose of the study drug.
LS mean and standard error (SE) were calculated using MMRM.
Outcome measures
| Measure |
Placebo
n=53 Participants
Participants received placebo matched to PTC518 tablets once daily (QD) orally for 12 months.
|
PTC518 5 mg
n=52 Participants
Participants received 5 mg tablet of PTC518 QD orally for 12 months.
|
PTC518 10 mg
n=54 Participants
Participants received 10 mg tablet of PTC518 QD orally for 12 months.
|
|---|---|---|---|
|
Percent Change From Baseline in Caudate Volume as Assessed Via Volumetric Magnetic Resonance Imaging (vMRI) at Month 12
|
4.42 percent change
Standard Error 0.413 • Interval 0.413 to
|
4.99 percent change
Standard Error 0.413 • Interval 0.413 to
|
5.74 percent change
Standard Error 0.391 • Interval 0.391 to
|
SECONDARY outcome
Timeframe: Baseline, Month 12Population: ITT Population included all randomized participants who took at least 1 dose of the study drug.
cUHDRS includes the Total Functional Capacity (range, 0-13; higher score means better functioning), Total Motor Score (range, 0-124; higher score means worse motor severity), Symbol Digit Modality Test (range, 0-110, correctly paired numbers-symbols in 90 seconds; higher score means better cognitive performance), and Stroop Word Reading (range, 0-no max value, correctly read colour words in 45 seconds; higher score means better cognitive performance) scores. The total score for the cUHDRS ranges from approximately 3 to 18, where higher scores indicated better functioning. LS mean and SE were calculated using analysis of covariance (ANCOVA) model.
Outcome measures
| Measure |
Placebo
n=53 Participants
Participants received placebo matched to PTC518 tablets once daily (QD) orally for 12 months.
|
PTC518 5 mg
n=52 Participants
Participants received 5 mg tablet of PTC518 QD orally for 12 months.
|
PTC518 10 mg
n=54 Participants
Participants received 10 mg tablet of PTC518 QD orally for 12 months.
|
|---|---|---|---|
|
Change From Baseline in Composite Unified Huntington's Disease Rating Scale (cUHDRS) Scores at Month 12
|
-0.64 units on a scale
Standard Error 0.168 • Interval 0.168 to
|
-0.61 units on a scale
Standard Error 0.176 • Interval 0.176 to
|
-0.92 units on a scale
Standard Error 0.164 • Interval 0.164 to
|
SECONDARY outcome
Timeframe: Baseline, Month 12Population: ITT Population included all randomized participants who took at least 1 dose of the study drug.
LS mean and 95% CI were calculated using MMRM. The blood tHTT protein value was log-transformed before fitting the MMRM model. The LS means from MMRM model were back-transformed to present geometric mean percent changes.
Outcome measures
| Measure |
Placebo
n=53 Participants
Participants received placebo matched to PTC518 tablets once daily (QD) orally for 12 months.
|
PTC518 5 mg
n=52 Participants
Participants received 5 mg tablet of PTC518 QD orally for 12 months.
|
PTC518 10 mg
n=54 Participants
Participants received 10 mg tablet of PTC518 QD orally for 12 months.
|
|---|---|---|---|
|
Percent Change From Baseline in Geometric Mean Blood tHTT Protein at Month 12
|
8.19 percent change
95% Confidence Interval 1.99 • Interval 1.99 to 14.76
|
-14.18 percent change
95% Confidence Interval -19.25 • Interval -19.25 to -8.79
|
-28.44 percent change
95% Confidence Interval -32.45 • Interval -32.45 to -24.18
|
SECONDARY outcome
Timeframe: Baseline, Month 12Population: ITT Population included all randomized participants who took at least 1 dose of the study drug.
LS mean and 95% CI were calculated using MMRM. The CSF mHTT protein value was log-transformed before fitting the MMRM model. The LS means from MMRM model were back-transformed to present geometric mean percent changes.
Outcome measures
| Measure |
Placebo
n=53 Participants
Participants received placebo matched to PTC518 tablets once daily (QD) orally for 12 months.
|
PTC518 5 mg
n=52 Participants
Participants received 5 mg tablet of PTC518 QD orally for 12 months.
|
PTC518 10 mg
n=54 Participants
Participants received 10 mg tablet of PTC518 QD orally for 12 months.
|
|---|---|---|---|
|
Percent Change From Baseline in Cerebrospinal Fluid (CSF) Mutant Huntingtin Protein (mHTT) at Month 12
|
-15.64 percent change
95% Confidence Interval -26.71 • Interval -26.71 to -2.9
|
-23.68 percent change
95% Confidence Interval -33.97 • Interval -33.97 to -11.78
|
-23.28 percent change
95% Confidence Interval -32.84 • Interval -32.84 to -12.35
|
SECONDARY outcome
Timeframe: Baseline, Month 12Population: ITT Population included all randomized participants who took at least 1 dose of the study drug.
LS mean and 95% CI were calculated using MMRM. The blood mHTT protein value was log-transformed before fitting the MMRM model. The LS means from MMRM model were back-transformed to present geometric mean percent changes.
Outcome measures
| Measure |
Placebo
n=53 Participants
Participants received placebo matched to PTC518 tablets once daily (QD) orally for 12 months.
|
PTC518 5 mg
n=52 Participants
Participants received 5 mg tablet of PTC518 QD orally for 12 months.
|
PTC518 10 mg
n=54 Participants
Participants received 10 mg tablet of PTC518 QD orally for 12 months.
|
|---|---|---|---|
|
Percent Change From Baseline in Blood mHTT Protein at Month 12
|
13.89 percent change
95% Confidence Interval 6.33 • Interval 6.33 to 21.98
|
-14.11 percent change
95% Confidence Interval -19.99 • Interval -19.99 to -7.79
|
-28.20 percent change
95% Confidence Interval -32.89 • Interval -32.89 to -23.2
|
Adverse Events
Placebo
Serious events: 4 serious events
Other events: 32 other events
Deaths: 1 deaths
PTC518 5 mg
Serious events: 1 serious events
Other events: 34 other events
Deaths: 1 deaths
PTC518 10 mg
Serious events: 2 serious events
Other events: 41 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Placebo
n=53 participants at risk
Participants received placebo matched to PTC518 tablets QD orally for 12 months.
|
PTC518 5 mg
n=52 participants at risk
Participants received 5 mg tablet of PTC518 QD orally for 12 months.
|
PTC518 10 mg
n=54 participants at risk
Participants received 10 mg tablet of PTC518 QD orally for 12 months.
|
|---|---|---|---|
|
Blood and lymphatic system disorders
Bone marrow oedema
|
0.00%
0/53 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
0.00%
0/52 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
1.9%
1/54 • Number of events 1 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
|
Cardiac disorders
Atrial fibrillation
|
1.9%
1/53 • Number of events 1 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
0.00%
0/52 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
0.00%
0/54 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
|
Ear and labyrinth disorders
Vestibular disorder
|
1.9%
1/53 • Number of events 1 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
0.00%
0/52 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
0.00%
0/54 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
|
Gastrointestinal disorders
Inguinal hernia
|
1.9%
1/53 • Number of events 1 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
0.00%
0/52 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
0.00%
0/54 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
|
General disorders
Drowning
|
0.00%
0/53 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
1.9%
1/52 • Number of events 1 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
0.00%
0/54 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
|
Injury, poisoning and procedural complications
Femur fracture
|
0.00%
0/53 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
0.00%
0/52 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
1.9%
1/54 • Number of events 1 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
|
Injury, poisoning and procedural complications
Road traffic accident
|
1.9%
1/53 • Number of events 1 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
0.00%
0/52 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
0.00%
0/54 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
|
Injury, poisoning and procedural complications
Wrist fracture
|
1.9%
1/53 • Number of events 1 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
0.00%
0/52 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
0.00%
0/54 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
Other adverse events
| Measure |
Placebo
n=53 participants at risk
Participants received placebo matched to PTC518 tablets QD orally for 12 months.
|
PTC518 5 mg
n=52 participants at risk
Participants received 5 mg tablet of PTC518 QD orally for 12 months.
|
PTC518 10 mg
n=54 participants at risk
Participants received 10 mg tablet of PTC518 QD orally for 12 months.
|
|---|---|---|---|
|
Blood and lymphatic system disorders
Lymphopenia
|
0.00%
0/53 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
1.9%
1/52 • Number of events 1 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
5.6%
3/54 • Number of events 3 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
|
Gastrointestinal disorders
Constipation
|
0.00%
0/53 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
0.00%
0/52 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
5.6%
3/54 • Number of events 3 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
|
Gastrointestinal disorders
Diarrhoea
|
5.7%
3/53 • Number of events 3 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
7.7%
4/52 • Number of events 5 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
9.3%
5/54 • Number of events 6 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
|
Gastrointestinal disorders
Nausea
|
1.9%
1/53 • Number of events 1 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
5.8%
3/52 • Number of events 5 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
1.9%
1/54 • Number of events 5 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
|
Gastrointestinal disorders
Vomiting
|
3.8%
2/53 • Number of events 2 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
3.8%
2/52 • Number of events 3 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
5.6%
3/54 • Number of events 3 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
|
General disorders
Fatigue
|
5.7%
3/53 • Number of events 3 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
5.8%
3/52 • Number of events 3 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
1.9%
1/54 • Number of events 1 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
|
Infections and infestations
COVID-19
|
5.7%
3/53 • Number of events 3 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
3.8%
2/52 • Number of events 2 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
7.4%
4/54 • Number of events 4 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
|
Infections and infestations
Influenza
|
17.0%
9/53 • Number of events 12 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
13.5%
7/52 • Number of events 7 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
16.7%
9/54 • Number of events 12 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
|
Infections and infestations
Nasopharyngitis
|
17.0%
9/53 • Number of events 14 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
11.5%
6/52 • Number of events 8 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
14.8%
8/54 • Number of events 15 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
|
Infections and infestations
Upper respiratory tract infection
|
0.00%
0/53 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
5.8%
3/52 • Number of events 4 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
0.00%
0/54 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
|
Injury, poisoning and procedural complications
Fall
|
9.4%
5/53 • Number of events 7 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
15.4%
8/52 • Number of events 10 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
20.4%
11/54 • Number of events 16 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
|
Injury, poisoning and procedural complications
Post lumbar puncture syndrome
|
5.7%
3/53 • Number of events 4 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
9.6%
5/52 • Number of events 5 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
5.6%
3/54 • Number of events 6 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
|
Injury, poisoning and procedural complications
Skin abrasion
|
1.9%
1/53 • Number of events 1 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
3.8%
2/52 • Number of events 2 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
7.4%
4/54 • Number of events 6 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
7.5%
4/53 • Number of events 5 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
17.3%
9/52 • Number of events 10 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
14.8%
8/54 • Number of events 12 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
|
Nervous system disorders
Dizziness
|
1.9%
1/53 • Number of events 1 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
5.8%
3/52 • Number of events 4 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
3.7%
2/54 • Number of events 2 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
|
Nervous system disorders
Headache
|
13.2%
7/53 • Number of events 9 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
15.4%
8/52 • Number of events 30 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
24.1%
13/54 • Number of events 19 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
|
Nervous system disorders
Paraesthesia
|
0.00%
0/53 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
1.9%
1/52 • Number of events 1 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
5.6%
3/54 • Number of events 4 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
|
Nervous system disorders
Tension headache
|
1.9%
1/53 • Number of events 1 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
1.9%
1/52 • Number of events 1 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
5.6%
3/54 • Number of events 3 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
|
Psychiatric disorders
Depressed mood
|
5.7%
3/53 • Number of events 3 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
0.00%
0/52 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
1.9%
1/54 • Number of events 1 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
1.9%
1/53 • Number of events 1 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
5.8%
3/52 • Number of events 3 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
9.3%
5/54 • Number of events 5 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
|
Vascular disorders
Hypertension
|
0.00%
0/53 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
0.00%
0/52 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
5.6%
3/54 • Number of events 3 • Baseline up to Month 18
Safety Population included all randomized participants who received at least 1 dose of the study drug, with participants grouped according to the treatment they received.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee The Sponsor can review results and/or communications prior to public release and can embargo communications regarding trial results for a period that is up to 180 days from the time submitted to the sponsor for review. The sponsor may consult with the PI to require changes to the communication or extend the embargo.
- Publication restrictions are in place
Restriction type: OTHER