Clinical trial NCT02006472: A Phase 2, to Evaluating the Safety and Efficacy of Pridopidine Vs Placebo for Symptomatic Treatment in Patients With Huntington's Disease
Official title: A Phase 2, Dose-Finding, Randomized, Parallel-Group, Double-Blind, Placebo-Controlled Study, Evaluating the Safety and Efficacy of Pridopidine 45, 67.5, 90, and 112.5 mg Twice-Daily vs Placebo for Symptomatic Treatment in Patients With Huntington's Disease
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Huntington's Disease
Keywords: Huntington's Disease
Pridopidine
Pride-HD
Lead sponsor: Prilenia
Sponsor class: INDUSTRY
Collaborators: name: European Huntington's Disease Network
name: Huntington Study Group
First posted: 2013-12-10
Enrollment: 408
Sex: ALL
Minimum age (years): 21
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

Eligibility criteria: Inclusion Criteria:

* Diagnosis of HD based on the presence of \>/= 36 CAG repeats
* Male or female age ≥21 years, with an onset of HD after 18 years' old.
* Females of childbearing potential must be compliant in using adequate birth control throughout the duration of the study
* Body weight ≥50 kg
* Sum of \>= 25 points on the UHDRS-TMS and UHDRS Independence Score \<=90%
* Able and willing to provide written informed consent prior to any study related procedure.
* Willing to provide a blood sample for genetic analyses
* Willing and able to take oral medication and able to comply with the study specific procedures.
* 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.
* 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.

Exclusion Criteria:

* Patients with clinically significant heart disease at the screening visit
* Treatment with tetrabenazine within 6 weeks of study screening
* Patients with a history of epilepsy or of seizures within the last 5 years
* 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
* 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

Primary outcomes:
measure: Change From Baseline in Unified Huntington's Disease Rating Scale-Total Motor Score (UHDRS-TMS) at Week 26
timeFrame: 26 weeks
Secondary outcomes:
measure: Number of Patients With Adverse Events
timeFrame: 52 weeks
Other outcomes:
measure: Change From Baseline in Total Functional Capacity (TFC) at Week 52
timeFrame: 52 weeks
Locations:
city: La Jolla
country: United States
facility: Investigational Site 12199
state: California
city: Los Angeles
country: United States
facility: Investigational Site 12204
state: California
city: Englewood
country: United States
facility: Investigational Site 12201
state: Colorado
city: Washington
country: United States
facility: Investigational Site 12196
state: District of Columbia
city: Chicago
country: United States
facility: Investigational Site 12207
state: Illinois
city: Baltimore
country: United States
facility: Investigational Site 12202
state: Maryland
city: Baltimore
country: United States
facility: Investigational Site 12206
state: Maryland
city: Manhasset
country: United States
facility: Investigational Site 12200
state: New York
city: New York
country: United States
facility: Investigational Site 12203
state: New York
city: Rochester
country: United States
facility: Investigational Site 12198
state: New York
city: Winston-Salem
country: United States
facility: Investigational Site 12211
state: North Carolina
city: Cincinnati
country: United States
facility: Investigational Site 12205
state: Ohio
city: Pittsburgh
country: United States
facility: Investigational Site 12209
state: Pennsylvania
city: Salt Lake City
country: United States
facility: Investigational Site 12208
state: Utah
city: Richmond
country: United States
facility: Investigational Site 12210
state: Virginia
city: Kirkland
country: United States
facility: Investigational Site 12197
state: Washington
city: Caulfield South
country: Australia
facility: Investigational Site 78055
city: Kew
country: Australia
facility: Investigational Site 78056
city: Subiaco
country: Australia
facility: Investigational Site 78058
city: Westmead
country: Australia
facility: Investigational Site 78057
city: Innsbruck
country: Austria
facility: Investigational Site 33021
city: Wien
country: Austria
facility: Investigational Site 33027
city: Vancouver
country: Canada
facility: Investigational Site 11035
state: British Columbia
city: Ottawa
country: Canada
facility: Investigational Site 11037
state: Ontario
city: Toronto
country: Canada
facility: Investigational Site 11036
state: Ontario
city: Aarhus
country: Denmark
facility: Investigational Site 39028
city: Copenhagen
country: Denmark
facility: Investigational Site 39027
city: Angers cedex 9
country: France
facility: Investigational Site 35123
city: Creteil
country: France
facility: Investigational Site 35122
city: Lille
country: France
facility: Investigational Site 35125
city: Marseille Cedex 5
country: France
facility: Investigational Site 35124
city: Salouel
country: France
facility: Investigational Site 35121
city: Toulouse
country: France
facility: Investigational Site 35165
city: Berlin
country: Germany
facility: Investigational Site 32408
city: Bochum
country: Germany
facility: Investigational Site 32410
city: Muenster
country: Germany
facility: Investigational Site 32409
city: Ulm
country: Germany
facility: Investigational Site 32407
city: Firenze
country: Italy
facility: Investigational Site 30083
city: Milano
country: Italy
facility: Investigational Site 30080
city: Napoli
country: Italy
facility: Investigational Site 30082
city: Pozzilli
country: Italy
facility: Investigational Site 30081
city: San Giovanni Rotondo
country: Italy
facility: Investigational Site 30084
city: Leiden
country: Netherlands
facility: Investigational Site 38059
city: Gdansk
country: Poland
facility: Investigational Site 53150
city: Krakow
country: Poland
facility: Investigational Site 53149
city: Poznan
country: Poland
facility: Investigational Site 53148
city: Warsaw
country: Poland
facility: Investigational Site 53151
city: Kazan
country: Russian Federation
facility: Investigational Site 50215
city: Moscow
country: Russian Federation
facility: Investigational Site 50213
city: Nizhny Novgorod
country: Russian Federation
facility: Investigational Site 50214
city: Birmingham
country: United Kingdom
facility: Investigational Site 34058
city: Cambridge
country: United Kingdom
facility: Investigational Site 34054
city: Cardiff
country: United Kingdom
facility: Investigational Site 34059
city: Headington
country: United Kingdom
facility: Investigational Site 34056
city: London
country: United Kingdom
facility: Investigational Site 34060
city: Manchester
country: United Kingdom
facility: Investigational Site 34055
city: Newcastle-Upon-Tyne
country: United Kingdom
facility: Investigational Site 34061
city: Sheffield
country: United Kingdom
facility: Investigational Site 34057
Study officials:
References:
citation: McGarry A, Leinonen M, Kieburtz K, Geva M, Olanow CW, Hayden M. Effects of Pridopidine on Functional Capacity in Early-Stage Participants from the PRIDE-HD Study. J Huntingtons Dis. 2020;9(4):371-380. doi: 10.3233/JHD-200440.
citation: Rodrigues FB, Quinn L, Wild EJ. Huntington's Disease Clinical Trials Corner: January 2019. J Huntingtons Dis. 2019;8(1):115-125. doi: 10.3233/JHD-190001.
citation: Reilmann R, McGarry A, Grachev ID, Savola JM, Borowsky B, Eyal E, Gross N, Langbehn D, Schubert R, Wickenberg AT, Papapetropoulos S, Hayden M, Squitieri F, Kieburtz K, Landwehrmeyer GB; European Huntington's Disease Network; Huntington Study Group investigators. Safety and efficacy of pridopidine in patients with Huntington's disease (PRIDE-HD): a phase 2, randomised, placebo-controlled, multicentre, dose-ranging study. Lancet Neurol. 2019 Feb;18(2):165-176. doi: 10.1016/S1474-4422(18)30391-0. Epub 2018 Dec 15.