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

Phase 2

Completed

• Conditions: Huntington's Disease
• Interventions: Drug: Pridopidine; Other: Placebo

• 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.

Study Timeline

• Study First Submitted: 2013-12-05
• Study First Submitted QC: 2013-12-05
• Study First Posted: 2013-12-10
• Study Start: 2014-02-28
• Primary Completion: 2015-12-16
• Study Completion: 2016-07-07
• Disposition First Submitted: 2018-05-08
• Disposition First Submitted QC: 2018-05-08
• Disposition First Posted: 2018-05-11
• Results First Submitted: 2021-03-14
• Status Verified: 2021-05
• Results First Submitted QC: 2021-07-16
• Last Update Submitted: 2021-07-16
• Results First Posted: 2021-07-19
• Last Update Posted: 2021-07-19

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 408

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

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 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, 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

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