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

NCT ID: NCT02006472

Last Updated: 2021-07-19

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 408 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2014-02-28
• Study Completion Date: 2016-07-07

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.

Conditions

Huntington's Disease

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

Pridopidine 45 mg

Twice daily

Group Type: EXPERIMENTAL

Pridopidine

Intervention Type: DRUG

22.5 mg and 45 mg capsules

Placebo

Intervention Type: OTHER

Capsules matching drug

Pridopidine 67.5 mg

Twice daily

Group Type: EXPERIMENTAL

Pridopidine

Intervention Type: DRUG

22.5 mg and 45 mg capsules

Placebo

Intervention Type: OTHER

Capsules matching drug

Pridopidine 90 mg

Twice daily

Group Type: EXPERIMENTAL

Pridopidine

Intervention Type: DRUG

22.5 mg and 45 mg capsules

Placebo

Intervention Type: OTHER

Capsules matching drug

Pridopidine 112.5 mg

Twice daily

Group Type: EXPERIMENTAL

Pridopidine

Intervention Type: DRUG

22.5 mg and 45 mg capsules

Placebo

Intervention Type: OTHER

Capsules matching drug

Placebo

Twice daily

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: OTHER

Capsules matching drug

Interventions

Pridopidine

22.5 mg and 45 mg capsules

Intervention Type: DRUG

Placebo

Capsules matching drug

Intervention Type: OTHER

Other Intervention Names

TV7820

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

• Minimum Eligible Age: 21 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

European Huntington's Disease Network

NETWORK

Sponsor Role: collaborator

Huntington Study Group

NETWORK

Sponsor Role: collaborator

Prilenia

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Investigational Site 12199

La Jolla, California, United States

Investigational Site 12204

Los Angeles, California, United States

Investigational Site 12201

Englewood, Colorado, United States

Investigational Site 12196

Washington D.C., District of Columbia, United States

Investigational Site 12207

Chicago, Illinois, United States

Investigational Site 12202

Baltimore, Maryland, United States

Investigational Site 12206

Baltimore, Maryland, United States

Investigational Site 12200

Manhasset, New York, United States

Investigational Site 12203

New York, New York, United States

Investigational Site 12198

Rochester, New York, United States

Investigational Site 12211

Winston-Salem, North Carolina, United States

Investigational Site 12205

Cincinnati, Ohio, United States

Investigational Site 12209

Pittsburgh, Pennsylvania, United States

Investigational Site 12208

Salt Lake City, Utah, United States

Investigational Site 12210

Richmond, Virginia, United States

Investigational Site 12197

Kirkland, Washington, United States

Investigational Site 78055

Caulfield South, , Australia

Investigational Site 78056

Kew, , Australia

Investigational Site 78058

Subiaco, , Australia

Investigational Site 78057

Westmead, , Australia

Investigational Site 33021

Innsbruck, , Austria

Investigational Site 33027

Vienna, , Austria

Investigational Site 11035

Vancouver, British Columbia, Canada

Investigational Site 11037

Ottawa, Ontario, Canada

Investigational Site 11036

Toronto, Ontario, Canada

Investigational Site 39028

Aarhus, , Denmark

Investigational Site 39027

Copenhagen, , Denmark

Investigational Site 35123

Angers, , France

Investigational Site 35122

Créteil, , France

Investigational Site 35125

Lille, , France

Investigational Site 35124

Marseille, , France

Investigational Site 35121

Salouël, , France

Investigational Site 35165

Toulouse, , France

Investigational Site 32408

Berlin, , Germany

Investigational Site 32410

Bochum, , Germany

Investigational Site 32409

Münster, , Germany

Investigational Site 32407

Ulm, , Germany

Investigational Site 30083

Florence, , Italy

Investigational Site 30080

Milan, , Italy

Investigational Site 30082

Napoli, , Italy

Investigational Site 30081

Pozzilli, , Italy

Investigational Site 30084

San Giovanni Rotondo, , Italy

Investigational Site 38059

Leiden, , Netherlands

Investigational Site 53150

Gdansk, , Poland

Investigational Site 53149

Krakow, , Poland

Investigational Site 53148

Poznan, , Poland

Investigational Site 53151

Warsaw, , Poland

Investigational Site 50215

Kazan', , Russia

Investigational Site 50213

Moscow, , Russia

Investigational Site 50214

Nizhny Novgorod, , Russia

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 Kingdom

Countries

United States; Australia; Austria; Canada; Denmark; France; Germany; Italy; Netherlands; Poland; Russia; United Kingdom

References

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.

Reference Type: BACKGROUND

PMID: 33164941 (View on PubMed)

Darpo B, Geva M, Ferber G, Goldberg YP, Cruz-Herranz A, Mehra M, Kovacs R, Hayden MR. Pridopidine Does Not Significantly Prolong the QTc Interval at the Clinically Relevant Therapeutic Dose. Neurol Ther. 2023 Apr;12(2):597-617. doi: 10.1007/s40120-023-00449-w. Epub 2023 Feb 22.

Reference Type: DERIVED

PMID: 36811812 (View on PubMed)

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.

Reference Type: DERIVED

PMID: 30776019 (View on PubMed)

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.

Reference Type: DERIVED

PMID: 30563778 (View on PubMed)

Other Identifiers

2013-001888-23

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

TV7820-CNS-20002

Identifier Type: -

Identifier Source: org_study_id