Trial Outcomes & Findings for A Study Evaluating if Pridopidine is Safe, Efficacious, and Tolerable in Patients With Huntington's Disease (NCT NCT02494778)
NCT ID: NCT02494778
Last Updated: 2021-09-17
Results Overview
From signature of the informed consent form through the end of the study, which was defined as Week 106
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
248 participants
Primary outcome timeframe
106 weeks
Results posted on
2021-09-17
Participant Flow
Participant milestones
| Measure |
Pridopidine 45 mg BID
Pridopidine (45 mg) administered as oral capsules, taken twice daily (bid)
|
|---|---|
|
Overall Study
STARTED
|
248
|
|
Overall Study
COMPLETED
|
27
|
|
Overall Study
NOT COMPLETED
|
221
|
Reasons for withdrawal
| Measure |
Pridopidine 45 mg BID
Pridopidine (45 mg) administered as oral capsules, taken twice daily (bid)
|
|---|---|
|
Overall Study
Death
|
3
|
|
Overall Study
Other
|
173
|
|
Overall Study
Lack of Efficacy
|
4
|
|
Overall Study
Adverse Event
|
18
|
|
Overall Study
Withdrawal by Subject
|
20
|
|
Overall Study
Lost to Follow-up
|
2
|
|
Overall Study
Noncompliant with study drug admin.
|
1
|
Baseline Characteristics
A Study Evaluating if Pridopidine is Safe, Efficacious, and Tolerable in Patients With Huntington's Disease
Baseline characteristics by cohort
| Measure |
Pridopidine 45 mg BID
n=248 Participants
Pridopidine (45 mg) administered as oral capsules, taken twice daily (bid)
|
|---|---|
|
Age, Continuous
|
50.6 years
STANDARD_DEVIATION 11.61 • n=5 Participants
|
|
Sex: Female, Male
Female
|
129 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
119 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
227 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
19 Participants
n=5 Participants
|
|
Region of Enrollment
Canada
|
4 participants
n=5 Participants
|
|
Region of Enrollment
Austria
|
18 participants
n=5 Participants
|
|
Region of Enrollment
Netherlands
|
9 participants
n=5 Participants
|
|
Region of Enrollment
United States
|
34 participants
n=5 Participants
|
|
Region of Enrollment
Poland
|
28 participants
n=5 Participants
|
|
Region of Enrollment
Italy
|
34 participants
n=5 Participants
|
|
Region of Enrollment
United Kingdom
|
28 participants
n=5 Participants
|
|
Region of Enrollment
France
|
18 participants
n=5 Participants
|
|
Region of Enrollment
Australia
|
9 participants
n=5 Participants
|
|
Region of Enrollment
Germany
|
32 participants
n=5 Participants
|
|
Region of Enrollment
Russia
|
34 participants
n=5 Participants
|
|
CYP2D6 metaboliser genotype
Poor metaboliser
|
8 Participants
n=5 Participants
|
|
CYP2D6 metaboliser genotype
Extensive metaboliser
|
214 Participants
n=5 Participants
|
|
CYP2D6 metaboliser genotype
Intermediate metaboliser
|
25 Participants
n=5 Participants
|
|
CYP2D6 metaboliser genotype
Ultra-rapid metaboliser
|
1 Participants
n=5 Participants
|
|
Neuroleptic use
Yes
|
94 Participants
n=5 Participants
|
|
Neuroleptic use
No
|
154 Participants
n=5 Participants
|
|
Number of CAG repeats
|
44.8 CAG repeats
STANDARD_DEVIATION 4.01 • n=5 Participants
|
PRIMARY outcome
Timeframe: 106 weeksPopulation: All patients enrolled who received at least one dose of study drug
From signature of the informed consent form through the end of the study, which was defined as Week 106
Outcome measures
| Measure |
Pridopidine 45 mg BID
n=248 Participants
Pridopidine (45 mg) administered as oral capsules, taken twice daily (bid)
|
|---|---|
|
Percentage of Participants With Adverse Events
|
193 Participants
|
SECONDARY outcome
Timeframe: Week 52; end of treatment (EOT) which was planned to occur at Week 104Population: All patients enrolled who received at least 1 dose of study drug and had at least 1 post-baseline UHDRS-TMS assessment.
Q-motor assessments were based on the application of force transducers and 3-dimensional position sensors. The reported parameter is the Pro-Sup-Inter-Onset-interval-SD-Hand, measured in seconds. Positive change from baseline indicates worsening.
Outcome measures
| Measure |
Pridopidine 45 mg BID
n=146 Participants
Pridopidine (45 mg) administered as oral capsules, taken twice daily (bid)
|
|---|---|
|
Change From Baseline in Quantitative Motor (Q-motor) Measurements, Pro-Sup-Inter-Onset-interval-SD-Hand
Change from baseline to EOT
|
-0.1 second
Standard Deviation 0.01
|
|
Change From Baseline in Quantitative Motor (Q-motor) Measurements, Pro-Sup-Inter-Onset-interval-SD-Hand
Change from baseline to Week 52
|
0.0 second
Standard Deviation 0.05
|
SECONDARY outcome
Timeframe: Week 52; end of treatment (EOT) which was planned to occur at Week 104Population: All patients enrolled who received at least 1 dose of study drug and had at least 1 post-baseline UHDRS-TMS assessment.
Q-motor assessments were based on the application of force transducers and 3-dimensional position sensors. The reported parameter is the Pro-Sup-Peak-Force-CV-Hand, measured in %. Positive change from baseline indicates worsening.
Outcome measures
| Measure |
Pridopidine 45 mg BID
n=146 Participants
Pridopidine (45 mg) administered as oral capsules, taken twice daily (bid)
|
|---|---|
|
Change From Baseline in Quantitative Motor (Q-motor) Measurements, Pro-Sup-Peak-Force-CV-Hand
Change from baseline to Week 52
|
-2.9 percentage
Standard Deviation 9.31
|
|
Change From Baseline in Quantitative Motor (Q-motor) Measurements, Pro-Sup-Peak-Force-CV-Hand
Change from baseline to EOT
|
-5.9 percentage
Standard Deviation 8.05
|
Adverse Events
Pridopidine 45 mg BID
Serious events: 31 serious events
Other events: 191 other events
Deaths: 3 deaths
Serious adverse events
| Measure |
Pridopidine 45 mg BID
n=248 participants at risk
Pridopidine (45 mg) administered as oral capsules, taken twice daily (bid)
|
|---|---|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Basal cell carcinoma
|
0.40%
1/248 • Number of events 1 • From signature of the informed consent form through the end of the study, which was defined as Week 106
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Malignant melanoma in situ
|
0.40%
1/248 • Number of events 1 • From signature of the informed consent form through the end of the study, which was defined as Week 106
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Prostate cancer
|
0.40%
1/248 • Number of events 1 • From signature of the informed consent form through the end of the study, which was defined as Week 106
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Squamous cell carcinoma
|
0.40%
1/248 • Number of events 1 • From signature of the informed consent form through the end of the study, which was defined as Week 106
|
|
General disorders
Death
|
0.40%
1/248 • Number of events 1 • From signature of the informed consent form through the end of the study, which was defined as Week 106
|
|
General disorders
Gait disturbance
|
0.40%
1/248 • Number of events 1 • From signature of the informed consent form through the end of the study, which was defined as Week 106
|
|
Psychiatric disorders
Suicidal ideation
|
1.2%
3/248 • Number of events 3 • From signature of the informed consent form through the end of the study, which was defined as Week 106
|
|
Psychiatric disorders
Depression
|
0.40%
1/248 • Number of events 1 • From signature of the informed consent form through the end of the study, which was defined as Week 106
|
|
Psychiatric disorders
Hallucination, auditory
|
0.40%
1/248 • Number of events 1 • From signature of the informed consent form through the end of the study, which was defined as Week 106
|
|
Psychiatric disorders
Paranoia
|
0.40%
1/248 • Number of events 1 • From signature of the informed consent form through the end of the study, which was defined as Week 106
|
|
Injury, poisoning and procedural complications
Fall
|
1.6%
4/248 • Number of events 4 • From signature of the informed consent form through the end of the study, which was defined as Week 106
|
|
Injury, poisoning and procedural complications
Subdural haematoma
|
0.81%
2/248 • Number of events 2 • From signature of the informed consent form through the end of the study, which was defined as Week 106
|
|
Injury, poisoning and procedural complications
Ankle fracture
|
0.40%
1/248 • Number of events 1 • From signature of the informed consent form through the end of the study, which was defined as Week 106
|
|
Injury, poisoning and procedural complications
Brain contusion
|
0.40%
1/248 • Number of events 1 • From signature of the informed consent form through the end of the study, which was defined as Week 106
|
|
Injury, poisoning and procedural complications
Contusion
|
0.40%
1/248 • Number of events 1 • From signature of the informed consent form through the end of the study, which was defined as Week 106
|
|
Injury, poisoning and procedural complications
Craniocerebral injury
|
0.40%
1/248 • Number of events 1 • From signature of the informed consent form through the end of the study, which was defined as Week 106
|
|
Injury, poisoning and procedural complications
Extradural haematoma
|
0.40%
1/248 • Number of events 1 • From signature of the informed consent form through the end of the study, which was defined as Week 106
|
|
Injury, poisoning and procedural complications
Facial bones fracture
|
0.40%
1/248 • Number of events 1 • From signature of the informed consent form through the end of the study, which was defined as Week 106
|
|
Injury, poisoning and procedural complications
Head injury
|
0.40%
1/248 • Number of events 1 • From signature of the informed consent form through the end of the study, which was defined as Week 106
|
|
Injury, poisoning and procedural complications
Skull fracture
|
0.40%
1/248 • Number of events 1 • From signature of the informed consent form through the end of the study, which was defined as Week 106
|
|
Injury, poisoning and procedural complications
Skull fractured base
|
0.40%
1/248 • Number of events 1 • From signature of the informed consent form through the end of the study, which was defined as Week 106
|
|
Injury, poisoning and procedural complications
Tibia fracture
|
0.40%
1/248 • Number of events 1 • From signature of the informed consent form through the end of the study, which was defined as Week 106
|
|
Injury, poisoning and procedural complications
Toxicity to varios agents
|
0.40%
1/248 • Number of events 1 • From signature of the informed consent form through the end of the study, which was defined as Week 106
|
|
Investigations
Weight decreased
|
0.40%
1/248 • Number of events 1 • From signature of the informed consent form through the end of the study, which was defined as Week 106
|
|
Congenital, familial and genetic disorders
Huntington's disease
|
0.40%
1/248 • Number of events 1 • From signature of the informed consent form through the end of the study, which was defined as Week 106
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonia aspiration
|
0.40%
1/248 • Number of events 1 • From signature of the informed consent form through the end of the study, which was defined as Week 106
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary embolism
|
0.40%
1/248 • Number of events 1 • From signature of the informed consent form through the end of the study, which was defined as Week 106
|
|
Blood and lymphatic system disorders
Anaemia
|
0.40%
1/248 • Number of events 1 • From signature of the informed consent form through the end of the study, which was defined as Week 106
|
|
Nervous system disorders
Chorea
|
1.2%
3/248 • Number of events 3 • From signature of the informed consent form through the end of the study, which was defined as Week 106
|
|
Nervous system disorders
Dystonia
|
0.40%
1/248 • Number of events 1 • From signature of the informed consent form through the end of the study, which was defined as Week 106
|
|
Nervous system disorders
Haemorrhage intracranial
|
0.40%
1/248 • Number of events 1 • From signature of the informed consent form through the end of the study, which was defined as Week 106
|
|
Nervous system disorders
Peripheral nerve palsy
|
0.40%
1/248 • Number of events 1 • From signature of the informed consent form through the end of the study, which was defined as Week 106
|
|
Nervous system disorders
Subarachnoid haemorrhage
|
0.40%
1/248 • Number of events 1 • From signature of the informed consent form through the end of the study, which was defined as Week 106
|
|
Nervous system disorders
Syncope
|
0.40%
1/248 • Number of events 1 • From signature of the informed consent form through the end of the study, which was defined as Week 106
|
|
Nervous system disorders
Transient ischaemic attack
|
0.40%
1/248 • Number of events 1 • From signature of the informed consent form through the end of the study, which was defined as Week 106
|
|
Gastrointestinal disorders
Dysphagia
|
0.40%
1/248 • Number of events 1 • From signature of the informed consent form through the end of the study, which was defined as Week 106
|
|
Gastrointestinal disorders
Gastrointestinal polyp haemorrhage
|
0.40%
1/248 • Number of events 1 • From signature of the informed consent form through the end of the study, which was defined as Week 106
|
|
Hepatobiliary disorders
Drug-induced liver injury
|
0.40%
1/248 • Number of events 1 • From signature of the informed consent form through the end of the study, which was defined as Week 106
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal pain
|
0.40%
1/248 • Number of events 1 • From signature of the informed consent form through the end of the study, which was defined as Week 106
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
0.40%
1/248 • Number of events 1 • From signature of the informed consent form through the end of the study, which was defined as Week 106
|
|
Metabolism and nutrition disorders
Cachexia
|
0.40%
1/248 • Number of events 1 • From signature of the informed consent form through the end of the study, which was defined as Week 106
|
|
Infections and infestations
Pneumonia
|
0.81%
2/248 • Number of events 2 • From signature of the informed consent form through the end of the study, which was defined as Week 106
|
|
Infections and infestations
Diverticulitis
|
0.40%
1/248 • Number of events 1 • From signature of the informed consent form through the end of the study, which was defined as Week 106
|
|
Infections and infestations
Urinary tract infection
|
0.40%
1/248 • Number of events 1 • From signature of the informed consent form through the end of the study, which was defined as Week 106
|
|
Infections and infestations
Urosepsis
|
0.40%
1/248 • Number of events 1 • From signature of the informed consent form through the end of the study, which was defined as Week 106
|
Other adverse events
| Measure |
Pridopidine 45 mg BID
n=248 participants at risk
Pridopidine (45 mg) administered as oral capsules, taken twice daily (bid)
|
|---|---|
|
Injury, poisoning and procedural complications
Fall
|
29.8%
74/248 • Number of events 163 • From signature of the informed consent form through the end of the study, which was defined as Week 106
|
|
Injury, poisoning and procedural complications
Contusion
|
5.2%
13/248 • Number of events 20 • From signature of the informed consent form through the end of the study, which was defined as Week 106
|
|
Investigations
Weight decreased
|
5.2%
13/248 • Number of events 13 • From signature of the informed consent form through the end of the study, which was defined as Week 106
|
|
Nervous system disorders
Chorea
|
9.3%
23/248 • Number of events 30 • From signature of the informed consent form through the end of the study, which was defined as Week 106
|
|
Nervous system disorders
Headache
|
5.6%
14/248 • Number of events 19 • From signature of the informed consent form through the end of the study, which was defined as Week 106
|
|
Psychiatric disorders
Insomnia
|
8.1%
20/248 • Number of events 27 • From signature of the informed consent form through the end of the study, which was defined as Week 106
|
|
Psychiatric disorders
Anxiety
|
6.5%
16/248 • Number of events 17 • From signature of the informed consent form through the end of the study, which was defined as Week 106
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
5.6%
14/248 • Number of events 15 • From signature of the informed consent form through the end of the study, which was defined as Week 106
|
|
Infections and infestations
Nasopharyngitis
|
11.7%
29/248 • Number of events 36 • From signature of the informed consent form through the end of the study, which was defined as Week 106
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee Data and results are owned by the sponsor. Results can be used by the institution for internal noncommercial research, education and patient care, and as required under applicable laws and regulations. Other uses require prior written consent of the sponsor.
- Publication restrictions are in place
Restriction type: OTHER