Open-label Extension Study of Pridopidine (ACR16) in the Symptomatic Treatment of Huntington Disease (OPEN-HART)

A Multi-Center, North American, Open-Label Extension Study of Pridopidine (ACR16) in the Symptomatic Treatment of Huntington's Disease (Open-HART).

Huntington disease (HD) is a hereditary neurodegenerative disorder causing impairment in movement, behavioral dysfunction and dementia. The movement disorder is mainly characterized by chorea (involuntary movements) and a progressive loss of voluntary movement causing a substantial functional impairment over time. The study will assess the long-term safety of pridopidine and the treatment effects during long-term, open-label treatment.

Study Overview

• Status: Completed
• Conditions: Huntington Disease
• Intervention / Treatment: Drug: pridopidine

• Study Type: Interventional
• Enrollment (Actual): 134
• Phase: Phase 2

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Subject is able to, and has provided written Informed Consent prior to any study related procedure.
• Patient has completed the HART (ACR16C009) or the PRIDE-HD (TV7820- CNS-20002) studies and had remained on IMP during the full on-treatment part of the study (including de-escalated patients) or has transitioned from the Open-HART pre-virtualization study period.
• Willing and able to take oral medication and able to comply with the study specific procedures.
• Patient has a wireless internet connection at home (and/or applicable locations) at the first remote visit.

• Patient has the ability to transition from in-person study visits to virtual study visits. The first remote visit (RV1) will take place within approximately 30 days after the last in-person visit.

  • Additional criteria apply, please contact the investigator for more information

Exclusion Criteria:

• Ongoing treatment with tetrabenazine or deutetrabenazine, seizure threshold lowering medications, or certain antipsychotics and antidepressants.
• Newly instigated or changed treatment with neuroleptics/antipsychotics
• Use of tricyclic antidepressants or class I & III antiarrhythmics at any time during the study period.
• Severe intercurrent illness that, in the opinion of the Investigator (or qualified designee), may put the subject at risk when continuing participation in the study.
• Alcohol and/or drug abuse as defined by the Diagnostic and Statistical Manual - Fourth Edition - Text Revision criteria for substance abuse - this includes the illicit use of cannabis.
• Subjects with a known history of epilepsy or a history of febrile seizure(s) or seizure(s) of unknown cause.
• Females who are pregnant or lactating.
• Females who are of child bearing potential and not taking adequate contraceptive precautions (either oral, barrier or chemical contraceptives) are excluded from the trial. Females of child bearing potential taking acceptable contraceptive precautions can be included.

• Known allergy to any ingredients of the trial medication.

  • Additional criteria apply, please contact the investigator for more information

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: pridopidine; 45mg bid	Drug: pridopidine; 45mg bid

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Number of Patients With at Least One Adverse Event	From signing of the informed consent through the end of the follow-up period, which was defined as 30 days after the final study visit in an individual patient, an average of 2.8 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Unified Huntington's Disease Rating Scale (UHDRS) Total Motor Score (TMS)	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 31 assessments is rated on a scale of 0 (normal) to 4 (marked impairment) for a TMS range of 0-124.	Baseline and at Month 12, 24, 36, 48, 60, and 72

Collaborators and Investigators

• Sponsor: Prilenia
• Investigators: Principal Investigator: See Central Contact section for questions about, Study Officials

Publications and helpful links

General Publications

• McGarry A, Auinger P, Kieburtz K, Geva M, Mehra M, Abler V, Grachev ID, Gordon MF, Savola JM, Gandhi S, Papapetropoulos S, Hayden M. Additional Safety and Exploratory Efficacy Data at 48 and 60 Months from Open-HART, an Open-Label Extension Study of Pridopidine in Huntington Disease. J Huntingtons Dis. 2020;9(2):173-184. doi: 10.3233/JHD-190393.
• McGarry A, Kieburtz K, Abler V, Grachev ID, Gandhi S, Auinger P, Papapetropoulos S, Hayden M. Safety and Exploratory Efficacy at 36 Months in Open-HART, an Open-Label Extension Study of Pridopidine in Huntington's Disease. J Huntingtons Dis. 2017;6(3):189-199. doi: 10.3233/JHD-170241.

Study record dates

Study Major Dates

• Study Start (Actual): March 24, 2011
• Primary Completion (Actual): January 5, 2018
• Study Completion (Actual): January 5, 2018

Study Registration Dates

• First Submitted: February 28, 2011
• First Submitted That Met QC Criteria: March 1, 2011
• First Posted (Estimate): March 2, 2011

Study Record Updates

• Last Update Posted (Actual): February 9, 2022
• Last Update Submitted That Met QC Criteria: January 15, 2022
• Last Verified: January 1, 2022

More Information

Terms related to this study

• Keywords: Huntington Disease.
• Additional Relevant MeSH Terms: Mental Disorders; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Neurocognitive Disorders; Genetic Diseases, Inborn; Basal Ganglia Diseases; Movement Disorders; Neurodegenerative Diseases; Dyskinesias; Heredodegenerative Disorders, Nervous System; Dementia; Cognition Disorders; Chorea; Huntington Disease
• Other Study ID Numbers: ACR16C015

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No