A Phase 2, to Evaluating the Safety and Efficacy of Pridopidine Vs Placebo for Symptomatic Treatment in Patients With Huntington's Disease
- Registration Number
- NCT02006472
- Lead Sponsor
- Prilenia
- Brief Summary
This is a multicenter, multinational, randomized, parallel-group, double-blind, placebo-controlled, dose range finding study to compare the efficacy and safety of different doses of pridopidine versus placebo in the treatment of motor impairment in Huntington's Disease (HD).
- Detailed Description
Originally, the study was designed to assess the effect of pridopidine on motor function at 26 weeks. Due to the recognition that the primary target of pridopidine is the Sigma-1 receptor, the trial was extended from 26 to 52 weeks to evaluate the effect of pridopidine on Total Functional Capacity (TFC). A minimum of 52 weeks are needed for the placebo group to decline and allow a window to assess an effect on TFC (a prespecified endpoint). Approximately 20% of patients completed 26 weeks of the study before IRB approvals for this extension, and did not continue into the 2nd treatment period up to 52 weeks.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 408
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Diagnosis of HD based on the presence of >/= 36 CAG repeats
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Male or female age ≥21 years, with an onset of HD after 18 years' old.
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Females of childbearing potential must be compliant in using adequate birth control throughout the duration of the study
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Body weight ≥50 kg
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Sum of >= 25 points on the UHDRS-TMS and UHDRS Independence Score <=90%
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Able and willing to provide written informed consent prior to any study related procedure.
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Willing to provide a blood sample for genetic analyses
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Willing and able to take oral medication and able to comply with the study specific procedures.
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Ambulatory, being able to travel to the study center, and judged by the investigator as likely to be able to continue to travel for the duration of the study.
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Availability and willingness of a caregiver, informant or family member to accompany the patient to the clinic at study, and the suitability of the caregiver should be judged by the Investigator.
- Other criteria apply, please contact the investigator for more information.
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Patients with clinically significant heart disease at the screening visit
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Treatment with tetrabenazine within 6 weeks of study screening
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Patients with a history of epilepsy or of seizures within the last 5 years
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Have other serious medical illnesses in the opinion of the investigator may put the patient at risk when participating in the study or may influence the results of the study or affect the patient's ability to take part in the study
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Patients receiving medications (within the last 6 weeks prior to screening) that have been proven to prolong QT interval or who may require such medications during the course of the study such as but not limited to non allowed anti psychotic medications, tricyclic antidepressants and/or Class I antiarrhythmics
- Other criteria apply, please contact the investigator for more information
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Pridopidine 45 mg Placebo Twice daily Pridopidine 67.5 mg Placebo Twice daily Pridopidine 90 mg Placebo Twice daily Pridopidine 112.5 mg Placebo Twice daily Placebo Placebo Twice daily Pridopidine 45 mg Pridopidine Twice daily Pridopidine 67.5 mg Pridopidine Twice daily Pridopidine 112.5 mg Pridopidine Twice daily Pridopidine 90 mg Pridopidine Twice daily
- Primary Outcome Measures
Name Time Method Change From Baseline in Unified Huntington's Disease Rating Scale-Total Motor Score (UHDRS-TMS) at Week 26 26 weeks 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 15 assessments is rated on a scale of 0 (normal) to 4 (marked impairment) for a TMS range of 0-124. Negative change from baseline values indicate improvement.
- Secondary Outcome Measures
Name Time Method Number of Patients With Adverse Events 52 weeks
Trial Locations
- Locations (58)
Investigational Site 12200
🇺🇸Manhasset, New York, United States
Investigational Site 12209
🇺🇸Pittsburgh, Pennsylvania, United States
Investigational Site 11036
🇨🇦Toronto, Ontario, Canada
Investigational Site 12202
🇺🇸Baltimore, Maryland, United States
Investigational Site 33027
🇦🇹Wien, Austria
Investigational Site 12207
🇺🇸Chicago, Illinois, United States
Investigational Site 12201
🇺🇸Englewood, Colorado, United States
Investigational Site 12205
🇺🇸Cincinnati, Ohio, United States
Investigational Site 33021
🇦🇹Innsbruck, Austria
Investigational Site 11037
🇨🇦Ottawa, Ontario, Canada
Investigational Site 78055
🇦🇺Caulfield South, Australia
Investigational Site 12206
🇺🇸Baltimore, Maryland, United States
Investigational Site 12203
🇺🇸New York, New York, United States
Investigational Site 12211
🇺🇸Winston-Salem, North Carolina, United States
Investigational Site 11035
🇨🇦Vancouver, British Columbia, Canada
Investigational Site 78056
🇦🇺Kew, Australia
Investigational Site 12208
🇺🇸Salt Lake City, Utah, United States
Investigational Site 12197
🇺🇸Kirkland, Washington, United States
Investigational Site 35125
🇫🇷Lille, France
Investigational Site 12210
🇺🇸Richmond, Virginia, United States
Investigational Site 53149
🇵🇱Krakow, Poland
Investigational Site 34059
🇬🇧Cardiff, United Kingdom
Investigational Site 50214
🇷🇺Nizhny Novgorod, Russian Federation
Investigational Site 34060
🇬🇧London, United Kingdom
Investigational Site 35165
🇫🇷Toulouse, France
Investigational Site 78057
🇦🇺Westmead, Australia
Investigational Site 34061
🇬🇧Newcastle-Upon-Tyne, United Kingdom
Investigational Site 32409
🇩🇪Muenster, Germany
Investigational Site 50215
🇷🇺Kazan, Russian Federation
Investigational Site 53150
🇵🇱Gdansk, Poland
Investigational Site 50213
🇷🇺Moscow, Russian Federation
Investigational Site 32407
🇩🇪Ulm, Germany
Investigational Site 30080
🇮🇹Milano, Italy
Investigational Site 34054
🇬🇧Cambridge, United Kingdom
Investigational Site 34055
🇬🇧Manchester, United Kingdom
Investigational Site 53148
🇵🇱Poznan, Poland
Investigational Site 38059
🇳🇱Leiden, Netherlands
Investigational Site 30081
🇮🇹Pozzilli, Italy
Investigational Site 39027
🇩🇰Copenhagen, Denmark
Investigational Site 39028
🇩🇰Aarhus, Denmark
Investigational Site 30083
🇮🇹Firenze, Italy
Investigational Site 30082
🇮🇹Napoli, Italy
Investigational Site 30084
🇮🇹San Giovanni Rotondo, Italy
Investigational Site 35124
🇫🇷Marseille Cedex 5, France
Investigational Site 32410
🇩🇪Bochum, Germany
Investigational Site 12199
🇺🇸La Jolla, California, United States
Investigational Site 12204
🇺🇸Los Angeles, California, United States
Investigational Site 12198
🇺🇸Rochester, New York, United States
Investigational Site 12196
🇺🇸Washington, District of Columbia, United States
Investigational Site 78058
🇦🇺Subiaco, Australia
Investigational Site 35121
🇫🇷Salouel, France
Investigational Site 35123
🇫🇷Angers cedex 9, France
Investigational Site 35122
🇫🇷Creteil, France
Investigational Site 53151
🇵🇱Warsaw, Poland
Investigational Site 34056
🇬🇧Headington, United Kingdom
Investigational Site 34058
🇬🇧Birmingham, United Kingdom
Investigational Site 34057
🇬🇧Sheffield, United Kingdom
Investigational Site 32408
🇩🇪Berlin, Germany