Huntington's Disease
Conditions
Keywords
Huntington's disease, pridopidine
Brief summary
The purpose of the study is to collect and assess long term data on the safety, tolerability, and efficacy of pridopidine in patients with Huntington's disease (HD).
Interventions
DRUGPridopidine
45 mg BID
Sponsors
Prilenia
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
21 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Pride HD completion within the last 6 months, including 2 week follow up period or patients who transitioned from the Open HART study or patients who complete future safety and efficacy clinical trials of pridopidine. In addition, patients who have already completed their defined study period under Open PRIDE HD global or local amendments and have discontinued treatment with pridopidine will be allowed to re enter the Open PRIDE HD study. * Women of child bearing potential or male participants: Adequate contraception and birth control * Good general health * other criteria apply, please contact the investigator for more information
Exclusion criteria
* Similar baseline criteria for ECG, vital signs, cardiovascular system, and renal function to PRIDE HD; * Similar concomitant medication restrictions to PRIDE HD. * other criteria apply, please contact the investigator for more information
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of Participants With Adverse Events | 106 weeks | From signature of the informed consent form through the end of the study, which was defined as Week 106 |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change From Baseline in Quantitative Motor (Q-motor) Measurements, Pro-Sup-Inter-Onset-interval-SD-Hand | Week 52; end of treatment (EOT) which was planned to occur at Week 104 | 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. |
| Change From Baseline in Quantitative Motor (Q-motor) Measurements, Pro-Sup-Peak-Force-CV-Hand | Week 52; end of treatment (EOT) which was planned to occur at Week 104 | 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. |
Countries
Australia, Austria, Canada, France, Germany, Italy, Netherlands, Poland, Russia, United Kingdom, United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Pridopidine 45 mg BID Pridopidine (45 mg) administered as oral capsules, taken twice daily (bid) | 248 |
| Total | 248 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Adverse Event | 18 |
| Overall Study | Death | 3 |
| Overall Study | Lack of Efficacy | 4 |
| Overall Study | Lost to Follow-up | 2 |
| Overall Study | Noncompliant with study drug admin. | 1 |
| Overall Study | Other | 173 |
| Overall Study | Withdrawal by Subject | 20 |
Baseline characteristics
| Characteristic | Pridopidine 45 mg BID |
|---|---|
| Age, Continuous | 50.6 years STANDARD_DEVIATION 11.61 |
| CYP2D6 metaboliser genotype Extensive metaboliser | 214 Participants |
| CYP2D6 metaboliser genotype Intermediate metaboliser | 25 Participants |
| CYP2D6 metaboliser genotype Poor metaboliser | 8 Participants |
| CYP2D6 metaboliser genotype Ultra-rapid metaboliser | 1 Participants |
| Neuroleptic use No | 154 Participants |
| Neuroleptic use Yes | 94 Participants |
| Number of CAG repeats | 44.8 CAG repeats STANDARD_DEVIATION 4.01 |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 2 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 19 Participants |
| Race (NIH/OMB) White | 227 Participants |
| Region of Enrollment Australia | 9 participants |
| Region of Enrollment Austria | 18 participants |
| Region of Enrollment Canada | 4 participants |
| Region of Enrollment France | 18 participants |
| Region of Enrollment Germany | 32 participants |
| Region of Enrollment Italy | 34 participants |
| Region of Enrollment Netherlands | 9 participants |
| Region of Enrollment Poland | 28 participants |
| Region of Enrollment Russia | 34 participants |
| Region of Enrollment United Kingdom | 28 participants |
| Region of Enrollment United States | 34 participants |
| Sex: Female, Male Female | 129 Participants |
| Sex: Female, Male Male | 119 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 3 / 248 |
| other Total, other adverse events | 191 / 248 |
| serious Total, serious adverse events | 31 / 248 |
Outcome results
Primary
Percentage of Participants With Adverse Events
From signature of the informed consent form through the end of the study, which was defined as Week 106
Time frame: 106 weeks
Population: All patients enrolled who received at least one dose of study drug
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Pridopidine 45 mg BID | Percentage of Participants With Adverse Events | 193 Participants |
Secondary
Change From Baseline in Quantitative Motor (Q-motor) Measurements, Pro-Sup-Inter-Onset-interval-SD-Hand
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.
Time frame: Week 52; end of treatment (EOT) which was planned to occur at Week 104
Population: All patients enrolled who received at least 1 dose of study drug and had at least 1 post-baseline UHDRS-TMS assessment.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Pridopidine 45 mg BID | 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 |
| Pridopidine 45 mg 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 |
Secondary
Change From Baseline in Quantitative Motor (Q-motor) Measurements, Pro-Sup-Peak-Force-CV-Hand
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.
Time frame: Week 52; end of treatment (EOT) which was planned to occur at Week 104
Population: All patients enrolled who received at least 1 dose of study drug and had at least 1 post-baseline UHDRS-TMS assessment.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Pridopidine 45 mg 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 |
| Pridopidine 45 mg BID | 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 |