Huntington Disease
Conditions
Keywords
Huntington Disease.
Brief summary
Huntington disease (HD) is a hereditary neurodegenerative disorder causing impairment in movement, behavioral dysfunction and dementia. The movement disorder is mainly characterized by chorea (involuntary movements) and a progressive loss of voluntary movement causing a substantial functional impairment over time. The study will assess the long-term safety of pridopidine and the treatment effects during long-term, open-label treatment.
Interventions
DRUGpridopidine
45mg bid
Sponsors
Prilenia
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
Inclusion criteria
* Subject is able to, and has provided written Informed Consent prior to any study related procedure. * Patient has completed the HART (ACR16C009) or the PRIDE-HD (TV7820- CNS-20002) studies and had remained on IMP during the full on-treatment part of the study (including de-escalated patients) or has transitioned from the Open-HART pre-virtualization study period. * Willing and able to take oral medication and able to comply with the study specific procedures. * Patient has a wireless internet connection at home (and/or applicable locations) at the first remote visit. * Patient has the ability to transition from in-person study visits to virtual study visits. The first remote visit (RV1) will take place within approximately 30 days after the last in-person visit. * Additional criteria apply, please contact the investigator for more information
Exclusion criteria
* Ongoing treatment with tetrabenazine or deutetrabenazine, seizure threshold lowering medications, or certain antipsychotics and antidepressants. * Newly instigated or changed treatment with neuroleptics/antipsychotics * Use of tricyclic antidepressants or class I & III antiarrhythmics at any time during the study period. * Severe intercurrent illness that, in the opinion of the Investigator (or qualified designee), may put the subject at risk when continuing participation in the study. * Alcohol and/or drug abuse as defined by the Diagnostic and Statistical Manual - Fourth Edition - Text Revision criteria for substance abuse - this includes the illicit use of cannabis. * Subjects with a known history of epilepsy or a history of febrile seizure(s) or seizure(s) of unknown cause. * Females who are pregnant or lactating. * Females who are of child bearing potential and not taking adequate contraceptive precautions (either oral, barrier or chemical contraceptives) are excluded from the trial. Females of child bearing potential taking acceptable contraceptive precautions can be included. * Known allergy to any ingredients of the trial medication. * Additional criteria apply, please contact the investigator for more information
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Number of Patients With at Least One Adverse Event | From signing of the informed consent through the end of the follow-up period, which was defined as 30 days after the final study visit in an individual patient, an average of 2.8 years |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Unified Huntington's Disease Rating Scale (UHDRS) Total Motor Score (TMS) | Baseline and at Month 12, 24, 36, 48, 60, and 72 | TMS was defined as the sum of all UHDRS motor domains ratings. The motor section of the UHDRS assesses motor features of Huntington's Disease (HD) with standardized ratings of oculo-motor function, dysarthria, chorea, dystonia, gait, and postural stability. Each of 31 assessments is rated on a scale of 0 (normal) to 4 (marked impairment) for a TMS range of 0-124. |
Participant flow
Recruitment details
The study recruited adult patients with Huntington's Disease (HD) who had completed studies ACR16C009 (NCT00724048) or TV7820-CNS-20002 (NCT02006472). The first patient was recruited on 24 Mar 2011, and the last patient completed the trial on 5 Jan 2018.
Participants by arm
| Arm | Count |
|---|---|
| Pridopidine Pridopidine 45 mg twice daily (bid), taken once in the morning and once in the afternoon. | 134 |
| Total | 134 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Adverse Event | 21 |
| Overall Study | Death | 8 |
| Overall Study | Lost to Follow-up | 2 |
| Overall Study | Non-compliance with study drug | 2 |
| Overall Study | Physician Decision | 13 |
| Overall Study | Protocol Violation | 3 |
| Overall Study | Various | 17 |
| Overall Study | Withdrawal by parent/guardian | 5 |
| Overall Study | Withdrawal by Subject | 23 |
Baseline characteristics
| Characteristic | Pridopidine |
|---|---|
| Age, Continuous | 52.4 years STANDARD_DEVIATION 9.98 |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants |
| Race (NIH/OMB) Black or African American | 3 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 | 2 Participants |
| Race (NIH/OMB) White | 129 Participants |
| Region of Enrollment Canada | 50 participants |
| Region of Enrollment United States | 84 participants |
| Sex: Female, Male Female | 67 Participants |
| Sex: Female, Male Male | 67 Participants |
| Weight | 74.7 kg STANDARD_DEVIATION 18.6 |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 8 / 134 |
| other Total, other adverse events | 119 / 134 |
| serious Total, serious adverse events | 31 / 134 |
Outcome results
Primary
Number of Patients With at Least One Adverse Event
Time frame: From signing of the informed consent through the end of the follow-up period, which was defined as 30 days after the final study visit in an individual patient, an average of 2.8 years
Population: Safety analysis set, including all enrolled patients who received at least 1 dose of study drug.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Pridopidine | Number of Patients With at Least One Adverse Event | 119 Participants |
Secondary
Unified Huntington's Disease Rating Scale (UHDRS) Total Motor Score (TMS)
TMS was defined as the sum of all UHDRS motor domains ratings. The motor section of the UHDRS assesses motor features of Huntington's Disease (HD) with standardized ratings of oculo-motor function, dysarthria, chorea, dystonia, gait, and postural stability. Each of 31 assessments is rated on a scale of 0 (normal) to 4 (marked impairment) for a TMS range of 0-124.
Time frame: Baseline and at Month 12, 24, 36, 48, 60, and 72
Population: Full analysis set, comprising all enrolled patients who received at least 1 dose of study drug and had a baseline and at least 1 post-baseline UHDRS-TMS assessment.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Pridopidine | Unified Huntington's Disease Rating Scale (UHDRS) Total Motor Score (TMS) | Month 12 | 41.5 score on a scale | Standard Deviation 18.7 |
| Pridopidine | Unified Huntington's Disease Rating Scale (UHDRS) Total Motor Score (TMS) | Baseline | 38.3 score on a scale | Standard Deviation 15.2 |
| Pridopidine | Unified Huntington's Disease Rating Scale (UHDRS) Total Motor Score (TMS) | Month 24 | 41.7 score on a scale | Standard Deviation 20.7 |
| Pridopidine | Unified Huntington's Disease Rating Scale (UHDRS) Total Motor Score (TMS) | Month 36 | 44.7 score on a scale | Standard Deviation 19.8 |
| Pridopidine | Unified Huntington's Disease Rating Scale (UHDRS) Total Motor Score (TMS) | Month 48 | 45.3 score on a scale | Standard Deviation 19.7 |
| Pridopidine | Unified Huntington's Disease Rating Scale (UHDRS) Total Motor Score (TMS) | Month 60 | 45.7 score on a scale | Standard Deviation 20.6 |
| Pridopidine | Unified Huntington's Disease Rating Scale (UHDRS) Total Motor Score (TMS) | Month 72 | 50.0 score on a scale | Standard Deviation 23.2 |