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NCT07609108ClinicalTrials.gov

Pridopidine Phase 3 Study in Huntington's Disease

A Phase 3, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of Pridopidine in Participants With Huntington's Disease (HD)

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The goal of this clinical trial is to learn if pridopidine can slow the clinical decline of Huntington's Disease (HD) in adult participants. It will also inform about the safety of pridopidine. The main questions the study aims to answer…

Phase 3400 Teilnehmende gesucht66 Studienzentren12 Länder

Kategorien

Neurologie44%Seltene und genetische Erkrankungen44%Psychiatrie und psychische Gesundheit13%

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How this study is set up

The points below are drawn from the public registry record for this study. Each one cites the field or sentence it came from. A dash (—) means the record does not state something — not that it is missing or wrong. Many well-run studies, especially small ones, leave some of these blank.

  • Registered before enrolment beganFirst posted 2026-05-27; recorded start 2026-07-01
  • Has a defined primary outcomeA primary outcome measure is listed in the record
  • The primary outcome states a time frameThe primary outcome measure records a time frame
  • Participants are randomly assignedAllocation is recorded as randomised
  • Has a comparison groupThe record lists a placebo or comparator arm, or more than one study arm
  • Uses blinding (masking)A masking level is recorded in the record
  • An ethics committee is not stated in the registry recordThis registry's ingested record has no ethics-committee field
  • Regulatory oversight (such as an IND or IDE) is not stated in the registry recordThis registry's ingested record has no regulatory-authorisation field
  • A data monitoring committee is not stated in the registry recordThis registry's ingested record has no data-monitoring-committee fieldA data monitoring committee is not required for many smaller studies, and its absence here is not unusual.
  • No cost to participants is mentioned in the recordChecked the summary, description and eligibility text; no cost-to-participant phrase found
  • Sponsor has 2 other studies in this databaseCounted from the lead sponsor named in the record (Prilenia)
  • No posted results from this sponsor are in this database yetBased on the sponsor’s studies in this database
  • The record lists 1 condition.
  • Lead sponsor type recorded as: industry.
  • Intervention regulatory context: investigational, within a phased regulatory pathway.

These are facts from the public registry record, not a judgement about the study. Discuss any trial with your own doctor before taking part.

What these facts mean, and what to ask · ruleset v1

Trial stature

Three independent measures of this study, described from its registry record — not a recommendation about it. A rigorous study by investigators nobody has heard of is better evidence than a weak study led by a famous one. How these are scored.

Methodological rigourSTRONG

A strong methodological design for a phase 3 study, judged from its ClinicalTrials.gov record.

How this score is built
  • Randomised allocation20/20

    Participants are randomly allocated between arms

  • Blinding20/20

    Quadruple-blind

  • Control arm13/15

    Placebo / sham control arm

  • Primary-outcome specificity10/10

    Named primary outcome with a defined time frame

  • Endpoint type3/10

    Surrogate or intermediate endpoint (conservative default)

  • Multi-centre0/8

    Single site, or site count not stated

  • Data monitoring committee7/7

    A data monitoring committee is in place

  • Prospective registration5/5

    Registered before the study start date

  • Protocol / SAP posted0/5

    No protocol or SAP posted to the registry

ScaleMEDIUM

A medium-sized study, international in scope: 400 participants (target), run at 66 sites, across 12 countries.

How this score is built
  • Enrolment25/40

    400 participants (target)

  • Site count0/25

    Site count not stated in the record

  • Country count0/15

    Country count not stated in the record

  • Planned duration9/10

    Planned over about 49 months

  • Sponsor scale1/10

    Prilenia has led 3 trials in our corpus

Investigator standingUNKNOWN

We have no verifiable track record for the investigator named on this trial. That is common for early-career investigators and for records held outside ClinicalTrials.gov — it is not a negative signal.

How this score is built
  • Investigator standing0/100

    No investigator recorded in the registry for this trial

These describe the registry record only, and today we hold ClinicalTrials.gov data. Absent fields lower a score, and absence often reflects registration practice rather than study quality. A high-stature trial is not necessarily safer or a better choice for you — enrolling in a large definitive trial can mean a higher chance of receiving placebo, while a small early-phase study may be the only route to a new therapy.

Zusammenfassung

The goal of this clinical trial is to learn if pridopidine can slow the clinical decline of Huntington's Disease (HD) in adult participants. It will also inform about the safety of pridopidine. The main questions the study aims to answer are: Does pridopidine slow the overall worsening of HD over 1 year? Does pridopidine slow the worsening of specific aspects of HD over 1 year, namely the clinical progression, the ability to perform daily life activities (functional capacity), the mind's ability to process information (cognition), working of the muscles (motor function), and quality of life? Researchers will compare the drug pridopidine to a placebo (a look-alike substance that contains no drug) to see if pridopidine works better than placebo to treat HD. During the first year of the study, participants will have the same chance to receive either pridopidine or placebo. Participants will: Take 1 pridopidine or placebo capsule twice daily for 12 months. Visit the clinic 6 times within 1 year for checkups and tests. All participants who complete this 1-year placebo-controlled study period will roll over into an additional 2-year study period during which all participants will receive pridopidine treatment, including participants who had received placebo during the first year. During this additional 2-year treatment period participants will visit the clinic a total of 6 times for checkups and tests.

This Phase 3 study consists of two study periods, a randomized, double-blind, placebo-controlled 1-year study period and a 2-year open-label extension (OLE) period. The study assesses the effect of pridopidine 45 mg twice daily on HD in participants who are not using antidopaminergic medications (ADMs) at study start. Main objectives of the study will be to assess the effect of pridopidine on clinical progression of HD within 1 year and to evaluate the effect of pridopidine on functional capacity, cognition, motor function, quality of life (QoL), and speech in participants with HD within 1 year. The target population in this study are adult participants (age 23-65, inclusive) with adult-onset HD (onset of signs and symptoms and a diagnosis ≥21 years of age). Eligible participants have not been using ADMs for at least 6 months prior to the screening visit. In case ADM treatment is deemed necessary by the treating physician during the study, specific ADMs and doses may be initiated. Female participants who are pregnant, planning to become pregnant or breastfeeding are not allowed to enter the study. The treatment period will start with a 2-week titration period of once-daily treatment (1 capsule of pridopidine or placebo taken orally in the morning). From Day 15 onwards, all participants will take the study drug twice daily: in the morning and in the afternoon. Treatment will continue for an additional 50 weeks. The placebo-controlled study period includes 6 in-clinic visits and 5 telephone visits (safety calls) during the 1-year treatment period. The placebo-controlled study period will be followed by a 2-year OLE period. During the OLE period, all eligible participants will receive pridopidine. The OLE will consist of a 2-year treatment period, including an initial 2-week (re)titration period for all participants at pridopidine once daily and a maintenance period of 102 weeks with pridopidine twice daily. The blinding of the original treatment assignment will be maintained for all participants and study personnel during the OLE period. A total of 6 in-clinic visits and 6 telephone visits (safety calls) are planned during the 2-year OLE period.

Erkrankungen

  • Huntington Disease

Eignung

GeschlechtAlle
Alter23 Years – 65 Years
Gesunde FreiwilligeNein

Eignung im Wortlaut des Registers

Inclusion Criteria: * Adult-onset HD (onset of signs and symptoms and a clinical diagnosis at ≥21 years of age). * A diagnosis based on clinical features and the presence of ≥40 CAG repeats in the huntingtin (HTT) gene confirmed by historical laboratory quanitified results or by a diagnostic test at Screening. * Diagnostic confidence level (DCL) of 4 (DCL=4 unequivocal motor signs, ≥99% confidence) on the standardized motor exam Total Motor Score (TMS). * Total Functional Capacity (TFC) score of ≥7 at Screening and Baseline. * Cytosine-Adenine-Guanine (CAG)-Age Product (CAP)100 score ≥95 at Screening. * Independence Scale (IS) score ≤90% at Screening. * Total Motor Score (TMS) of ≥20 at Screening and Baseline. * Not using ADMs (VMAT2i and neuroleptics/antipsychotics) for at least 6 months prior to Screening visit. Importantly, it is not encouraged to discontinue the participants' ADM treatment solely for enrollment in the current trial. Exclusion Criteria: * Clinically significant cardiovascular disease (e.g. QTcF \>450 msec \[males\] or \>470 msec \[females\], arrhythmias, uncontrolled atrial fibrillation, or congenital long QT syndrome), seizure history ≤5 years, significant neurological disorders (e.g. intracranial pathology or cerebrovascular events), active/recent malignancy (unless localized and resolved), or any serious or uncontrolled systemic disease (e.g. hepatic, renal, respiratory, endocrine, infectious \[HBV, HCV, HIV\], or psychiatric) that may pose safety risk or interfere with participation. * Severe hepatic or renal impairment. * Any mutant huntingtin (mHTT) lowering therapy in the past year. * Medications that prolong QT interval, taken within 4 weeks of the baseline visit. * Use of pridopidine within 6 weeks or 5 half-lives before the screening visit. * Previous participation in intracranial gene therapy study. * Laboratory values that fall outside of the central laboratory's reference range at Screening and are considered clinically significantly abnormal by the Investigator and affect the participant's suitability to participate in the study or put the participant at risk if he/she enters the study in the Investigator's opinion. * Female participants who are pregnant, planning to become pregnant or breastfeeding.

Eignung in einfachen Aussagen

Die Kriterien dieses Eintrags wurden noch nicht in einzelne Aussagen aufgeschlüsselt. Der Registertext oben ist vollständig und ist die maßgebliche Fassung.

Studiendesign

StudientypInterventionell
PhasePhase 3
ZuteilungRandomisiert
InterventionsmodellPARALLEL
Primäres ZielTREATMENT
VerblindungQUADRUPLE (4)
Teilnahme400 Teilnehmende gesucht

Sponsor und Mitwirkende

  • Prilenia Sponsor
  • Ferrer Internacional S.A. Mitwirkende

Studienarme und Interventionen

  • PridopidineEXPERIMENTAL

    During the titration period (2 weeks), pridopidine 45 mg capsules will be taken orally once daily (in the morning) for 2 weeks. Afterwards, pridopidine 45 mg capsules will be taken orally twice daily (in the morning and in the afternoon; 7-10 hours apart) for 50 weeks.

  • PlaceboPLACEBO_COMPARATOR

    During the titration period (2 weeks), matched placebo hard gelatin capsules will be taken once daily (in the morning) for 2 weeks. Afterwards, placebo capsules will be taken twice daily (in the morning and in the afternoon; 7-10 hours apart) for 50 weeks.

Interventionen

  • Arzneimittel Placebo

    Matched placebo hard gelatin capsule

  • Arzneimittel Pridopidine

    Pridopidine hard gelatin capsule

Endpunkte

  1. Primärer Endpunkt

    Change in Composite Unified Huntington's Disease Rating Scale (cUHDRS)

    cUHDRS is a combined score to assess disease progression in HD. It includes TFC, TMS, SDMT, and SWR: Total Functional Capacity (TFC) tests the capacity to maintain daily living, finances, care level, occupation. Higher score = better outcome. Best score=13. Worst score=0. Total Motor Score (TMS) assesses motor features (oculomotor, dysarthria, chorea, dystonia, gait, postural stability). Higher score = worse outcome. Best score=0. Worst score= 124. Stroop Word Reading (SWR) measures attention and mental flexibility. Participant reads names of colors printed in black ink. Scores reflect correct responses in 45 sec. Higher score = better outcome. Best score=100. Worst score=0. Symbol Digit Modalities Test (SDMT) tests psychomotor speed and working memory. Participant has 90 sec to match numbers with symbols. Scores = correct answers in 90 sec. Higher score = better outcome. Best score=120. Worst score=0. Total integrated cUHDRS scale range: -7.6 to 24.8. The higher, the better.

    Zeitrahmen Change from Baseline to Week 52

  2. Sekundärer Endpunkt

    Change in Total Functional Capacity (TFC) score

    Total Functional Capacity (TFC) tests the capacity to maintain domestic chores, activities of daily living, finances, care level, and occupation. Scores from 0 - 13. Higher score = better outcome.

    Zeitrahmen Change from Baseline to Week 52

  3. Sekundärer Endpunkt

    Change in Quantitative Motor (Q-Motor) Finger Tapping Inter-Onset Interval (FT-IOI) mean

    Quantitative (Q)-motor is a clinical assessment of fine motor skills that are crucial for daily activities. It is based on the application of pre-calibrated and temperature-controlled force transducers and 3-dimensional position sensors. Higher score = worse outcome.

    Zeitrahmen Change from Baseline to Week 52

  4. Sekundärer Endpunkt

    Change in Stroop Word Reading (SWR) score

    Stroop Word Reading (SWR) measures attention and mental flexibility. Participant reads names of colors printed in black ink. Scores reflect correct responses in 45 sec. Higher score = better outcome.

    Zeitrahmen Change from Baseline to Week 52

Daten

Startdatum1. Juli 2026 (tatsächlich)
Primärer Abschluss1. Juni 2028 (geschätzt)
Abschluss1. Juni 2030 (geschätzt)
Erstveröffentlichung27. Mai 2026 (tatsächlich)
Zuletzt aktualisiert17. September 2026
Ergebnisse veröffentlichtIm Registereintrag nicht angegeben
Status zuletzt bestätigtMai 2026

Tatsächlich bedeutet, dass das Ereignis eingetreten ist. Geschätzt bedeutet, dass der Sponsor es erwartet. Die beiden bedeuten Unterschiedliches.

Standorte

5 Studienzentren rekrutieren

Belgium

EinrichtungStadtBundesland oder RegionStatus
Hopital ErasmeAnderlechtRekrutierung noch nicht begonnen
Cliniques Universitaires Saint-LucWoluwe-Saint-LambertRekrutierung noch nicht begonnen

Canada

EinrichtungStadtBundesland oder RegionStatus
University of CalgaryCalgaryAlbertaRekrutierung noch nicht begonnen
University of AlbertaEdmontonAlbertaRekrutierung noch nicht begonnen
Centre Hospitalier de l'Universite de Montreal (CHUM)MontrealQuebecRekrutierung noch nicht begonnen
North York General HospitalTorontoOntarioRekrutierung noch nicht begonnen
University of British ColumbiaVancouverBritish ColumbiaRekrutierung noch nicht begonnen

Czechia

EinrichtungStadtBundesland oder RegionStatus
Vseobecna Fakultni Nemocnice V PrazePragueRekrutierung noch nicht begonnen

France

EinrichtungStadtBundesland oder RegionStatus
Centre Hospitalier Universitaire D'AngersAngersRekrutierung noch nicht begonnen
CHU Henri Mondor (APHP)CréteilRekrutierung noch nicht begonnen
Centre Hospitalier Universitaire Grenoble AlpesLa TroncheRekrutierung noch nicht begonnen
Centre Hospitalier Regional De MarseilleMarseilleRekrutierung noch nicht begonnen

Germany

EinrichtungStadtBundesland oder RegionStatus
Universitaetsklinikum Aachen AöRAachenRekrutierung noch nicht begonnen
Katholisches Klinikum Bochum gGmbHBochumRekrutierung noch nicht begonnen
Universitätsklinikum ErlangenErlangenRekrutierung noch nicht begonnen
George-Huntington-Institut GmbHMünsterRekrutierung noch nicht begonnen
Isar Amper KlinikumTaufkirchenRekrutierung noch nicht begonnen
Universitaetsklinikum Ulm AöRUlmRekrutierung noch nicht begonnen

Italy

EinrichtungStadtBundesland oder RegionStatus
Azienda Unita Sanitaria Locale Di BolognaBolognaRekrutierung noch nicht begonnen
IRCCS Istituto Auxologico ItalianoMilanRekrutierung noch nicht begonnen
Azienda Ospedaliera Universitaria Federico II Di NapoliNaplesRekrutierung noch nicht begonnen
Azienda Ospedale Università di PadovaPadovaRekrutierung noch nicht begonnen
Fondazione Policlinico Universitario Agostino Gemelli IRCCSRomaRekrutierung noch nicht begonnen
Casa Sollievo Della SofferenzaSan Giovanni RotondoRekrutierung noch nicht begonnen

Netherlands

EinrichtungStadtBundesland oder RegionStatus
Leids Universitair Medisch Centrum (LUMC)LeidenRekrutierung noch nicht begonnen

Poland

EinrichtungStadtBundesland oder RegionStatus
Krakowska Akademia Neurologii Sp. z o.o.KrakowRekrutierung noch nicht begonnen
Wojskowy Instytut Medycyny LotniczejWarsawRekrutierung noch nicht begonnen

Portugal

EinrichtungStadtBundesland oder RegionStatus
Unidade Local De Saude De Coimbra E.P.ECoimbraRekrutierung noch nicht begonnen
Unidade Local De Saude De Santa Maria E.P.E.LisbonRekrutierung noch nicht begonnen
Unidade Local De Saude De Santo Antonio E.P.E.PortoRekrutierung noch nicht begonnen
CNS Saude Lda.Torres VedrasRekrutierung noch nicht begonnen

Spain

EinrichtungStadtBundesland oder RegionStatus
Hospital Universitario De CrucesBarakaldoRekrutierung noch nicht begonnen
Hospital De La Santa Creu I Sant PauBarcelonaRekrutierung noch nicht begonnen
Hospital Universitario Ramon Y CajalMadridRekrutierung noch nicht begonnen
Hospital Universitario Central De AsturiasOviedoRekrutierung noch nicht begonnen
Hospital Universitario Virgen del RocíoSevilleRekrutierung noch nicht begonnen

United Kingdom

EinrichtungStadtBundesland oder RegionStatus
NHS GrampianAberdeenRekrutierung noch nicht begonnen
Birmingham and Solihull Mental Health Foundation NHS TrustBirminghamRekrutierung noch nicht begonnen
Cardiff UniversityCardiffRekrutierung noch nicht begonnen
The Walton Centre NHS Foundation TrustLiverpoolRekrutierung noch nicht begonnen
CNTW NHS Foundation TrustNewcastle upon TyneRekrutierung noch nicht begonnen

United States

EinrichtungStadtBundesland oder RegionStatus
Johns Hopkins UniversityBaltimoreMarylandRekrutierung noch nicht begonnen
The University of Alabama at BirminghamBirminghamAlabamaRekrutierung noch nicht begonnen
University of Miami-U Health Boca RatonBoca RatonFloridaRekrutierung noch nicht begonnen
University of Vermont Medical CenterBurlingtonVermontRekrutierung noch nicht begonnen
Massachusetts General HospitalCharlestownMassachusettsRekrutierung noch nicht begonnen
Rush University Medical CenterChicagoIllinoisRekrutierung noch nicht begonnen
Northwestern UniversityChicagoIllinoisRekrutierung noch nicht begonnen
University of CincinnatiCincinnatiOhioRekrutierung noch nicht begonnen
Cleveland ClinicClevelandOhioRekrutierung noch nicht begonnen

16 weitere Studienzentren sind im Registereintrag aufgeführt.

Studiendokumente

In diesem Registereintrag sind keine Dokumente verlinkt.

Änderungen im Zeitverlauf

  1. 7. August 2026

    Status geändert

    Status changed from Not yet recruiting to Recruiting

    NOT_YET_RECRUITINGRECRUITING

  2. 7. August 2026

    Rekrutierung eröffnet

    Recruitment opened

    NOT_YET_RECRUITINGRECRUITING

  3. 7. August 2026

    Studienzentrum hinzugefügt

    66 sites added (66 total)

    066

Register: ClinicalTrials.gov · NCT########

https://xmedica.io/trials/ctgov/NCT07609108