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A Phase 2, to Evaluating the Safety and Efficacy of Pridopidine Vs Placebo for Symptomatic Treatment in Patients With Huntington's Disease

Primary Purpose

Huntington's Disease

Status
Completed
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
Pridopidine
Placebo
Sponsored by
Prilenia
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Huntington's Disease focused on measuring Huntington's Disease, Pridopidine, Pride-HD

Eligibility Criteria

21 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Investigational Site 12199
  • Investigational Site 12204
  • Investigational Site 12201
  • Investigational Site 12196
  • Investigational Site 12207
  • Investigational Site 12202
  • Investigational Site 12206
  • Investigational Site 12200
  • Investigational Site 12203
  • Investigational Site 12198
  • Investigational Site 12211
  • Investigational Site 12205
  • Investigational Site 12209
  • Investigational Site 12208
  • Investigational Site 12210
  • Investigational Site 12197
  • Investigational Site 78055
  • Investigational Site 78056
  • Investigational Site 78058
  • Investigational Site 78057
  • Investigational Site 33021
  • Investigational Site 33027
  • Investigational Site 11035
  • Investigational Site 11037
  • Investigational Site 11036
  • Investigational Site 39028
  • Investigational Site 39027
  • Investigational Site 35123
  • Investigational Site 35122
  • Investigational Site 35125
  • Investigational Site 35124
  • Investigational Site 35121
  • Investigational Site 35165
  • Investigational Site 32408
  • Investigational Site 32410
  • Investigational Site 32409
  • Investigational Site 32407
  • Investigational Site 30083
  • Investigational Site 30080
  • Investigational Site 30082
  • Investigational Site 30081
  • Investigational Site 30084
  • Investigational Site 38059
  • Investigational Site 53150
  • Investigational Site 53149
  • Investigational Site 53148
  • Investigational Site 53151
  • Investigational Site 50215
  • Investigational Site 50213
  • Investigational Site 50214
  • Investigational Site 34058
  • Investigational Site 34054
  • Investigational Site 34059
  • Investigational Site 34056
  • Investigational Site 34060
  • Investigational Site 34055
  • Investigational Site 34061
  • Investigational Site 34057

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm 5

Arm Type

Experimental

Experimental

Experimental

Experimental

Placebo Comparator

Arm Label

Pridopidine 45 mg

Pridopidine 67.5 mg

Pridopidine 90 mg

Pridopidine 112.5 mg

Placebo

Arm Description

Twice daily

Twice daily

Twice daily

Twice daily

Twice daily

Outcomes

Primary Outcome Measures

Change From Baseline in Unified Huntington's Disease Rating Scale-Total Motor Score (UHDRS-TMS) at Week 26
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

Number of Patients With Adverse Events

Full Information

First Posted
December 5, 2013
Last Updated
July 16, 2021
Sponsor
Prilenia
Collaborators
European Huntington's Disease Network, Huntington Study Group
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1. Study Identification

Unique Protocol Identification Number
NCT02006472
Brief Title
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

2. Study Status

Record Verification Date
May 2021
Overall Recruitment Status
Completed
Study Start Date
February 28, 2014 (Actual)
Primary Completion Date
December 16, 2015 (Actual)
Study Completion Date
July 7, 2016 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Prilenia
Collaborators
European Huntington's Disease Network, Huntington Study Group

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Huntington's Disease
Keywords
Huntington's Disease, Pridopidine, Pride-HD

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
408 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Pridopidine 45 mg
Arm Type
Experimental
Arm Description
Twice daily
Arm Title
Pridopidine 67.5 mg
Arm Type
Experimental
Arm Description
Twice daily
Arm Title
Pridopidine 90 mg
Arm Type
Experimental
Arm Description
Twice daily
Arm Title
Pridopidine 112.5 mg
Arm Type
Experimental
Arm Description
Twice daily
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Twice daily
Intervention Type
Drug
Intervention Name(s)
Pridopidine
Other Intervention Name(s)
TV7820
Intervention Description
22.5 mg and 45 mg capsules
Intervention Type
Other
Intervention Name(s)
Placebo
Intervention Description
Capsules matching drug
Primary Outcome Measure Information:
Title
Change From Baseline in Unified Huntington's Disease Rating Scale-Total Motor Score (UHDRS-TMS) at Week 26
Description
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.
Time Frame
26 weeks
Secondary Outcome Measure Information:
Title
Number of Patients With Adverse Events
Time Frame
52 weeks
Other Pre-specified Outcome Measures:
Title
Change From Baseline in Total Functional Capacity (TFC) at Week 52
Description
The TFC is one subscale of the Unified Huntington's Disease Rating Scale (UHDRS), comprising 5 functional domains associated with disability (occupation, finances, domestic chores, activities of daily living, and care level), with scores on each item ranging from 0 to either 2 or 3. The TFC total score is the sum of the 5 TFC items and can range from 0 to 13, with greater scores indicating higher functioning. Negative change from baseline indicates worsening.
Time Frame
52 weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
21 Years
Accepts Healthy Volunteers
No
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
Facility Information:
Facility Name
Investigational Site 12199
City
La Jolla
State/Province
California
Country
United States
Facility Name
Investigational Site 12204
City
Los Angeles
State/Province
California
Country
United States
Facility Name
Investigational Site 12201
City
Englewood
State/Province
Colorado
Country
United States
Facility Name
Investigational Site 12196
City
Washington
State/Province
District of Columbia
Country
United States
Facility Name
Investigational Site 12207
City
Chicago
State/Province
Illinois
Country
United States
Facility Name
Investigational Site 12202
City
Baltimore
State/Province
Maryland
Country
United States
Facility Name
Investigational Site 12206
City
Baltimore
State/Province
Maryland
Country
United States
Facility Name
Investigational Site 12200
City
Manhasset
State/Province
New York
Country
United States
Facility Name
Investigational Site 12203
City
New York
State/Province
New York
Country
United States
Facility Name
Investigational Site 12198
City
Rochester
State/Province
New York
Country
United States
Facility Name
Investigational Site 12211
City
Winston-Salem
State/Province
North Carolina
Country
United States
Facility Name
Investigational Site 12205
City
Cincinnati
State/Province
Ohio
Country
United States
Facility Name
Investigational Site 12209
City
Pittsburgh
State/Province
Pennsylvania
Country
United States
Facility Name
Investigational Site 12208
City
Salt Lake City
State/Province
Utah
Country
United States
Facility Name
Investigational Site 12210
City
Richmond
State/Province
Virginia
Country
United States
Facility Name
Investigational Site 12197
City
Kirkland
State/Province
Washington
Country
United States
Facility Name
Investigational Site 78055
City
Caulfield South
Country
Australia
Facility Name
Investigational Site 78056
City
Kew
Country
Australia
Facility Name
Investigational Site 78058
City
Subiaco
Country
Australia
Facility Name
Investigational Site 78057
City
Westmead
Country
Australia
Facility Name
Investigational Site 33021
City
Innsbruck
Country
Austria
Facility Name
Investigational Site 33027
City
Wien
Country
Austria
Facility Name
Investigational Site 11035
City
Vancouver
State/Province
British Columbia
Country
Canada
Facility Name
Investigational Site 11037
City
Ottawa
State/Province
Ontario
Country
Canada
Facility Name
Investigational Site 11036
City
Toronto
State/Province
Ontario
Country
Canada
Facility Name
Investigational Site 39028
City
Aarhus
Country
Denmark
Facility Name
Investigational Site 39027
City
Copenhagen
Country
Denmark
Facility Name
Investigational Site 35123
City
Angers cedex 9
Country
France
Facility Name
Investigational Site 35122
City
Creteil
Country
France
Facility Name
Investigational Site 35125
City
Lille
Country
France
Facility Name
Investigational Site 35124
City
Marseille Cedex 5
Country
France
Facility Name
Investigational Site 35121
City
Salouel
Country
France
Facility Name
Investigational Site 35165
City
Toulouse
Country
France
Facility Name
Investigational Site 32408
City
Berlin
Country
Germany
Facility Name
Investigational Site 32410
City
Bochum
Country
Germany
Facility Name
Investigational Site 32409
City
Muenster
Country
Germany
Facility Name
Investigational Site 32407
City
Ulm
Country
Germany
Facility Name
Investigational Site 30083
City
Firenze
Country
Italy
Facility Name
Investigational Site 30080
City
Milano
Country
Italy
Facility Name
Investigational Site 30082
City
Napoli
Country
Italy
Facility Name
Investigational Site 30081
City
Pozzilli
Country
Italy
Facility Name
Investigational Site 30084
City
San Giovanni Rotondo
Country
Italy
Facility Name
Investigational Site 38059
City
Leiden
Country
Netherlands
Facility Name
Investigational Site 53150
City
Gdansk
Country
Poland
Facility Name
Investigational Site 53149
City
Krakow
Country
Poland
Facility Name
Investigational Site 53148
City
Poznan
Country
Poland
Facility Name
Investigational Site 53151
City
Warsaw
Country
Poland
Facility Name
Investigational Site 50215
City
Kazan
Country
Russian Federation
Facility Name
Investigational Site 50213
City
Moscow
Country
Russian Federation
Facility Name
Investigational Site 50214
City
Nizhny Novgorod
Country
Russian Federation
Facility Name
Investigational Site 34058
City
Birmingham
Country
United Kingdom
Facility Name
Investigational Site 34054
City
Cambridge
Country
United Kingdom
Facility Name
Investigational Site 34059
City
Cardiff
Country
United Kingdom
Facility Name
Investigational Site 34056
City
Headington
Country
United Kingdom
Facility Name
Investigational Site 34060
City
London
Country
United Kingdom
Facility Name
Investigational Site 34055
City
Manchester
Country
United Kingdom
Facility Name
Investigational Site 34061
City
Newcastle-Upon-Tyne
Country
United Kingdom
Facility Name
Investigational Site 34057
City
Sheffield
Country
United Kingdom

12. IPD Sharing Statement

Citations:
PubMed Identifier
33164941
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.
Results Reference
background
PubMed Identifier
30776019
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.
Results Reference
derived
PubMed Identifier
30563778
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.
Results Reference
derived

Learn more about this trial

A Phase 2, to Evaluating the Safety and Efficacy of Pridopidine Vs Placebo for Symptomatic Treatment in Patients With Huntington's Disease

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