Huntington's DiseasePhase 2NCT02006472

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

A Phase 2 study testing Pridopidine in people with Huntington's Disease, run by Prilenia at 58 sites in 12 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Trial 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). Read more about this study

Who is it for?: Ages 21 years and older
Conditions the study is for: Huntington's Disease
What is being tested?: Pridopidine
Type of treatment: Pill or oral medicine
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 408 participants
Where: 58 sitesin 12 countries
Study dates: 28 Feb 2014to 7 Jul 2016
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 21 years and older
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

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.

Show all 11Show fewer

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.

Who cannot join

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

Trial locations and contacts

58 sites in 12 countries.

Country

City, postcode or hospital

58 sites in 12 countries

United States16 sites

Investigational Site 12202
Baltimore, Maryland
Investigational Site 12206
Baltimore, Maryland
Investigational Site 12207
Chicago, Illinois
Investigational Site 12205
Cincinnati, Ohio
Investigational Site 12201
Englewood, Colorado
Investigational Site 12197
Kirkland, Washington
Investigational Site 12199
La Jolla, California
Investigational Site 12204
Los Angeles, California
Investigational Site 12200
Manhasset, New York
Investigational Site 12203
New York, New York
Investigational Site 12209
Pittsburgh, Pennsylvania
Investigational Site 12210
Richmond, Virginia
Investigational Site 12198
Rochester, New York
Investigational Site 12208
Salt Lake City, Utah
Investigational Site 12196
Washington D.C., District of Columbia
Investigational Site 12211
Winston-Salem, North Carolina

Other trials in United States

United Kingdom8 sites

Investigational Site 34058
Birmingham
Investigational Site 34054
Cambridge
Investigational Site 34059
Cardiff
Investigational Site 34056
Headington
Investigational Site 34060
London
Investigational Site 34055
Manchester
Investigational Site 34061
Newcastle upon Tyne
Investigational Site 34057
Sheffield

Other trials in United Kingdom

France6 sites

Investigational Site 35123
Angers
Investigational Site 35122
Créteil
Investigational Site 35125
Lille
Investigational Site 35124
Marseille
Investigational Site 35121
Salouël
Investigational Site 35165
Toulouse

Other trials in France

Italy5 sites

Investigational Site 30083
Florence
Investigational Site 30080
Milan
Investigational Site 30082
Naples
Investigational Site 30081
Pozzilli
Investigational Site 30084
San Giovanni Rotondo

Other trials in Italy

Australia4 sites

Investigational Site 78055
Caulfield South
Investigational Site 78056
Kew
Investigational Site 78058
Subiaco
Investigational Site 78057
Westmead

Other trials in Australia

Germany4 sites

Investigational Site 32408
Berlin
Investigational Site 32410
Bochum
Investigational Site 32409
Münster
Investigational Site 32407
Ulm

Other trials in Germany

Poland4 sites

Investigational Site 53150
Gdansk
Investigational Site 53149
Krakow
Investigational Site 53148
Poznan
Investigational Site 53151
Warsaw

Other trials in Poland

Canada3 sites

Investigational Site 11037
Ottawa, Ontario
Investigational Site 11036
Toronto, Ontario
Investigational Site 11035
Vancouver, British Columbia

Other trials in Canada

Russia3 sites

Investigational Site 50215
Kazan'
Investigational Site 50213
Moscow
Investigational Site 50214
Nizhny Novgorod

Other trials in Russia

Austria2 sites

Investigational Site 33021
Innsbruck
Investigational Site 33027
Vienna

Other trials in Austria

Denmark2 sites

Investigational Site 39028
Aarhus
Investigational Site 39027
Copenhagen

Other trials in Denmark

Netherlands1 site

Investigational Site 38059
Leiden

Other trials in Netherlands

About this study

Official title: A Phase 2, Dose-Finding, Randomized, Parallel-Group, Double-Blind, Placebo-Controlled Study, Evaluating the Safety and Efficacy of Pridopidine 45, 67.5, 90, and 112.5 mg Twice-Daily vs Placebo for Symptomatic Treatment in Patients With Huntington's Disease

Read the 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
Being tested: Pridopidine, Placebo
Keywords: Huntington's Disease, Pridopidine, Pride-HD
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
Also called TV7820
22.5 mg and 45 mg capsules
PlaceboOther
Capsules matching drug

Study groups in NCT02006472
Study group	Role	What they receive
Pridopidine 45 mg	Experimental	Twice daily Receives: Drug: Pridopidine; Other: Placebo
Pridopidine 67.5 mg	Experimental	Twice daily Receives: Drug: Pridopidine; Other: Placebo
Pridopidine 90 mg	Experimental	Twice daily Receives: Drug: Pridopidine; Other: Placebo
Pridopidine 112.5 mg	Experimental	Twice daily Receives: Drug: Pridopidine; Other: Placebo
Placebo	Placebo comparator	Twice daily Receives: Other: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

Change From Baseline in Unified Huntington's Disease Rating Scale-Total Motor Score (UHDRS-TMS) at Week 26
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.
Measured: 26 weeks

Secondary outcomes (1)

Number of Patients With Adverse Events
Measured: 52 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

28 Feb 20147 Jul 2016

First posted10 Dec 2013 (estimated)
Study start28 Feb 2014
Primary completion16 Dec 2015
Study completion7 Jul 2016

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 (4)Papers the sponsor has linked to this study.
Other trials for Huntington's 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: Randomised
Intervention model: Parallel groups
Masking: Quadruple blind
Who is masked: Participant, Care provider, Investigator, Outcomes assessor
Enrolment: 408 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT02006472
Sponsor's protocol ID: TV7820-CNS-20002
Other IDs: 2013-001888-23
Status last verified: May 2021
First submitted: 5 Dec 2013
Last updated: 19 Jul 2021
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes

Publications

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.
PubMed 33164941
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.
PubMed 36811812
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.
PubMed 30776019
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.
PubMed 30563778

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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All Huntington's Disease trials

Study at a glance

From the registry record, last updated by the sponsor on 19 Jul 2021. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Phase 2, Dose-Finding, Randomized, Parallel-Group, Double-Blind, Placebo-Controlled Study, Evaluating the Safety and Efficacy of Pridopidine 45, 67.5, 90, and 112.5 mg Twice-Daily vs Placebo for Symptomatic Treatment in Patients With Huntington's Disease
ClinicalTrials.gov ID: NCT02006472
Acronym / sponsor ID: TV7820-CNS-20002
Conditions: Huntington's Disease
Keywords: Huntington's Disease, Pridopidine, Pride-HD
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 408 participants (actual)
Lead sponsor: PrileniaIndustry (pharma, biotech, devices)
Collaborators: European Huntington's Disease Network, Huntington Study Group
Study start: 28 Feb 2014 (actual)
Primary completion: 16 Dec 2015 (actual)
Study completion: 7 Jul 2016 (actual)
First posted: 10 Dec 2013 (estimated)
Results: Posted 19 Jul 2021

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Detailed description

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Quadruple blind (participant, care provider, investigator, outcomes assessor)
Number of arms: 5

Arm	Type	Receives
Pridopidine 45 mg	Experimental	Drug: Pridopidine, Other: PlaceboTwice daily
Pridopidine 67.5 mg	Experimental	Drug: Pridopidine, Other: PlaceboTwice daily
Pridopidine 90 mg	Experimental	Drug: Pridopidine, Other: PlaceboTwice daily
Pridopidine 112.5 mg	Experimental	Drug: Pridopidine, Other: PlaceboTwice daily
Placebo	Placebo comparator	Other: PlaceboTwice daily

Who may qualify

Ages: 21 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (11 listed)

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%

Key exclusion criteria (6 listed)

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

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
PridopidineAlso: TV782022.5 mg and 45 mg capsules	Drug	Pridopidine 112.5 mg, Pridopidine 45 mg, Pridopidine 67.5 mg, Pridopidine 90 mg
PlaceboCapsules matching drug	Other	Placebo, Pridopidine 112.5 mg, Pridopidine 45 mg, Pridopidine 67.5 mg, Pridopidine 90 mg

Primary outcome

Change From Baseline in Unified Huntington's Disease Rating Scale-Total Motor Score (UHDRS-TMS) at Week 26
Time frame: 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.

1 secondary outcome

Number of Patients With Adverse Events
Time frame: 52 weeks

Results and safety

Results posted to the registry on 19 Jul 2021, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Placebo	82	70
Pridopidine 45 mg BID	81	59
Pridopidine 67.5 mg BID	82	65
Pridopidine 90 mg BID	81	67
Pridopidine 112.5 mg BID	82	62

Primary outcome: Change From Baseline in Unified Huntington's Disease Rating Scale-Total Motor Score (UHDRS-TMS) at Week 26

Time frame: 26 weeks. Mean (Standard Error). Unit: score on a scale

Measure	Placebo n = 81	Pridopidine 45 mg BID n = 75	Pridopidine 67.5 mg BID n = 79	Pridopidine 90 mg BID n = 81	Pridopidine 112.5 mg BID n = 81
Value	-4.79 (0.99)	-3.37 (1.05)	-3.09 (1.02)	-4.13 (1.00)	-2.74 (1.01)

Placebo vs Pridopidine 45 mg BID: LSM difference: 1.42 (95% CI -1.39 to 4.23), p = 0.3202, Mixed Models Analysis
Placebo vs Pridopidine 67.5 mg BID: LSM difference: 1.7 (95% CI -1.06 to 4.46), p = 0.2266, Mixed Models Analysis
Placebo vs Pridopidine 90 mg BID: LSM difference: 0.66 (95% CI -2.07 to 3.39), p = 0.6348, Mixed Models Analysis

Population analysed: Full analysis set (FAS) i.e. patients who received at least one dose of study drug and had at least one post-baseline efficacy assessment

1 secondary outcome is reported in the full results.

Adverse events

Time frame: 52 weeks. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Placebo	0 of 82 (0%)	45 of 82 (54.9%)	0 of 82
Pridopidine 45 mg BID	8 of 81 (9.9%)	49 of 81 (60.5%)	0 of 81
Pridopidine 67.5 mg BID	9 of 82 (11%)	63 of 82 (76.8%)	0 of 82
Pridopidine 90 mg BID	9 of 81 (11.1%)	57 of 81 (70.4%)	1 of 81
Pridopidine 112.5 mg BID	9 of 82 (11%)	53 of 82 (64.6%)	1 of 82

Limitations and caveats (from the sponsor): Analyses for Early HD population were conducted as additional analyses

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 19 Jul 2021.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Background: PubMed 33164941McGarry 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.
Derived from this study: PubMed 36811812Darpo 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.
Derived from this study: PubMed 30776019Rodrigues 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.
Derived from this study: PubMed 30563778Reilmann 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.

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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.

The record lists no contact. Use the official record to reach the sponsor.

58 sites in 12 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Pridopidine in people with Huntington's Disease, run by Prilenia at 58 sites in 12 countries.

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 11 inclusion and 6 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 19 Jul 2021. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 58 sites in United States, United Kingdom, France, Italy, Australia and 7 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. 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 19 Jul 2021). 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?

Change From Baseline in Unified Huntington's Disease Rating Scale-Total Motor Score (UHDRS-TMS) at Week 26 (time frame: 26 weeks). 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 NCT02006472, which also lists 4 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 19 Jul 2021.