Huntington DiseasePhase 2OPEN-HARTNCT01306929
Open-label Extension Study of Pridopidine (ACR16) in the Symptomatic Treatment of Huntington Disease
A Phase 2 study testing pridopidine in people with Huntington Disease, run by Prilenia.
Trial 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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Huntington Disease
- What is being tested?
- pridopidine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 134 participants
- Where
- No sites listed yet
- Study dates
- 24 Mar 2011to 5 Jan 2018
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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.
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- 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
About this study
Official title: 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.
- Conditions
- Huntington Disease
- Being tested
- pridopidine
- Keywords
- Huntington Disease.
- Medical subject headings
- Huntington Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
pridopidineDrug
45mg bid
| Study group | Role | What they receive |
|---|---|---|
| pridopidine | Experimental | 45mg bid Receives: Drug: pridopidine |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Patients With at Least One Adverse Event
Measured: 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 outcomes (1)
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.
Measured: Baseline and at Month 12, 24, 36, 48, 60, and 72
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Mar 2011 (estimated)
- Study start24 Mar 2011
- Primary completion5 Jan 2018
- Study completion5 Jan 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Huntington DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 134 participants (actual)
Sponsor and investigators
- Lead sponsor
- Prilenia
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- See Central Contact section for questions about, Principal investigator, study officials
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01306929
- Sponsor's protocol ID
- ACR16C015
- Acronym
- OPEN-HART
- Status last verified
- Jan 2022
- First submitted
- 28 Feb 2011
- Last updated
- 9 Feb 2022
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
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.
PubMed 32508327McGarry 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.
PubMed 28826192
Study at a glance
From the registry record, last updated by the sponsor on 9 Feb 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multi-Center, North American, Open-Label Extension Study of Pridopidine (ACR16) in the Symptomatic Treatment of Huntington's Disease (Open-HART).
- ClinicalTrials.gov ID
- NCT01306929
- Acronym / sponsor ID
- OPEN-HART / ACR16C015
- Conditions
- Huntington Disease
- Keywords
- Huntington Disease.
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 134 participants (actual)
- Lead sponsor
- PrileniaIndustry (pharma, biotech, devices)
- Study start
- 24 Mar 2011 (actual)
- Primary completion
- 5 Jan 2018 (actual)
- Study completion
- 5 Jan 2018 (actual)
- First posted
- 2 Mar 2011 (estimated)
- Results
- Posted 9 Feb 2022
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| pridopidine | Experimental | Drug: pridopidine45mg bid |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- 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.
Key exclusion criteria (10 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| pridopidine45mg bid | Drug | pridopidine |
Primary outcome
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
1 secondary outcome
Unified Huntington's Disease Rating Scale (UHDRS) Total Motor Score (TMS)
Time frame: Baseline and at Month 12, 24, 36, 48, 60, and 72
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Feb 2022.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 32508327McGarry 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.
- Derived from this study: PubMed 28826192McGarry 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.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
See Central Contact section for questions about principal investigator
study officials
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing pridopidine in people with Huntington Disease, run by Prilenia.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 9 Feb 2022. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 9 Feb 2022). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01306929, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Feb 2022.