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

• ClinicalTrials.gov Identifier: NCT01306929
• Recruitment Status: Completed
• First Posted: March 2, 2011
• Results First Posted: February 9, 2022
• Last Update Posted: February 9, 2022
• Sponsor: Prilenia
• Information provided by (Responsible Party): Prilenia

Study Description

Brief Summary:

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.

Condition or disease	Intervention/treatment	Phase
Huntington Disease	Drug: pridopidine	Phase 2

Study Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 134 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: A Multi-Center, North American, Open-Label Extension Study of Pridopidine (ACR16) in the Symptomatic Treatment of Huntington's Disease (Open-HART).
• Actual Study Start Date: March 24, 2011
• Actual Primary Completion Date: January 5, 2018
• Actual Study Completion Date: January 5, 2018

Arms and Interventions

Arm	Intervention/treatment
Experimental: pridopidine; 45mg bid	Drug: pridopidine; 45mg bid

Outcome Measures

Primary Outcome Measures :

1. Number of Patients With at Least One Adverse Event [ Time Frame: 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 :

1. Unified Huntington's Disease Rating Scale (UHDRS) Total Motor Score (TMS) [ Time Frame: Baseline and at Month 12, 24, 36, 48, 60, and 72 ]
   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.

Eligibility Criteria

• Ages Eligible for Study: Child, Adult, Older Adult
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Criteria

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

Contacts and Locations

Sponsors and Collaborators

Prilenia

Investigators

• Principal Investigator: See Central Contact section for questions about; study officials

  Study Documents (Full-Text)

Documents provided by Prilenia:

Study Protocol  [PDF] June 29, 2017

Statistical Analysis Plan  [PDF] January 11, 2018

More Information

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.

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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.

• Responsible Party: Prilenia
• ClinicalTrials.gov Identifier: NCT01306929
• Other Study ID Numbers: ACR16C015
• First Posted: March 2, 2011
• Results First Posted: February 9, 2022
• Last Update Posted: February 9, 2022
• Last Verified: January 2022

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: No

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Prilenia:

Huntington Disease.

Additional relevant MeSH terms:

Huntington Disease; Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Dementia; Chorea; Dyskinesias; Movement Disorders; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Genetic Diseases, Inborn; Cognition Disorders; Neurocognitive Disorders; Mental Disorders