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

Huntington's Disease Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02006472
• Other study ID #: TV7820-CNS-20002
• Secondary ID: 2013-001888-23
• Status: Completed
• Phase: Phase 2
• Start date: February 28, 2014
• Est. completion date: July 7, 2016

Study information

• Verified date: May 2021
• Source: Prilenia
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

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 information / eligibility

• Status: Completed
• Enrollment: 408
• Est. completion date: July 7, 2016
• Est. primary completion date: December 16, 2015
• Accepts healthy volunteers: No
• Gender: All
• Age group: 21 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Huntington Disease
• Huntington's Disease

Intervention

Drug:
• Pridopidine
22.5 mg and 45 mg capsules

Other:
• Placebo
Capsules matching drug

Locations

Country	Name	City	State
Australia	Investigational Site 78055	Caulfield South
Australia	Investigational Site 78056	Kew
Australia	Investigational Site 78058	Subiaco
Australia	Investigational Site 78057	Westmead
Austria	Investigational Site 33021	Innsbruck
Austria	Investigational Site 33027	Wien
Canada	Investigational Site 11037	Ottawa	Ontario
Canada	Investigational Site 11036	Toronto	Ontario
Canada	Investigational Site 11035	Vancouver	British Columbia
Denmark	Investigational Site 39028	Aarhus
Denmark	Investigational Site 39027	Copenhagen
France	Investigational Site 35123	Angers cedex 9
France	Investigational Site 35122	Creteil
France	Investigational Site 35125	Lille
France	Investigational Site 35124	Marseille Cedex 5
France	Investigational Site 35121	Salouel
France	Investigational Site 35165	Toulouse
Germany	Investigational Site 32408	Berlin
Germany	Investigational Site 32410	Bochum
Germany	Investigational Site 32409	Muenster
Germany	Investigational Site 32407	Ulm
Italy	Investigational Site 30083	Firenze
Italy	Investigational Site 30080	Milano
Italy	Investigational Site 30082	Napoli
Italy	Investigational Site 30081	Pozzilli
Italy	Investigational Site 30084	San Giovanni Rotondo
Netherlands	Investigational Site 38059	Leiden
Poland	Investigational Site 53150	Gdansk
Poland	Investigational Site 53149	Krakow
Poland	Investigational Site 53148	Poznan
Poland	Investigational Site 53151	Warsaw
Russian Federation	Investigational Site 50215	Kazan
Russian Federation	Investigational Site 50213	Moscow
Russian Federation	Investigational Site 50214	Nizhny Novgorod
United Kingdom	Investigational Site 34058	Birmingham
United Kingdom	Investigational Site 34054	Cambridge
United Kingdom	Investigational Site 34059	Cardiff
United Kingdom	Investigational Site 34056	Headington
United Kingdom	Investigational Site 34060	London
United Kingdom	Investigational Site 34055	Manchester
United Kingdom	Investigational Site 34061	Newcastle-Upon-Tyne
United Kingdom	Investigational Site 34057	Sheffield
United States	Investigational Site 12202	Baltimore	Maryland
United States	Investigational Site 12206	Baltimore	Maryland
United States	Investigational Site 12207	Chicago	Illinois
United States	Investigational Site 12205	Cincinnati	Ohio
United States	Investigational Site 12201	Englewood	Colorado
United States	Investigational Site 12197	Kirkland	Washington
United States	Investigational Site 12199	La Jolla	California
United States	Investigational Site 12204	Los Angeles	California
United States	Investigational Site 12200	Manhasset	New York
United States	Investigational Site 12203	New York	New York
United States	Investigational Site 12209	Pittsburgh	Pennsylvania
United States	Investigational Site 12210	Richmond	Virginia
United States	Investigational Site 12198	Rochester	New York
United States	Investigational Site 12208	Salt Lake City	Utah
United States	Investigational Site 12196	Washington	District of Columbia
United States	Investigational Site 12211	Winston-Salem	North Carolina

Sponsors (3)

Lead Sponsor	Collaborator
Prilenia	European Huntington's Disease Network, Huntington Study Group

Countries where clinical trial is conducted

United States,  Australia,  Austria,  Canada,  Denmark,  France,  Germany,  Italy,  Netherlands,  Poland,  Russian Federation,  United Kingdom, 

References & Publications (1)

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. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Change From Baseline in Total Functional Capacity (TFC) at Week 52	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.	52 weeks
Primary	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.	26 weeks
Secondary	Number of Patients With Adverse Events		52 weeks