xMedica

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)

En recrutementAccepte des participants actuellement, selon la fiche du registre.
Contacter cette étude

En bref

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 participants recherchés66 sites12 pays

Catégories

Neurologie44%Maladies rares et génétiques44%Psychiatrie et santé mentale13%

Enregistré dans 1 registre

Une même étude peut être enregistrée dans plusieurs registres. Nous l'affichons une seule fois et renvoyons vers chaque fiche que nous détenons.

Les informations de l'essai sont affichées telles que publiées par le registre, dans leur langue d'origine.

Cette étude vous intéresse ?

Se connecter ou créez un compte pour manifester votre intérêt et suivre cette étude.

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.

Résumé

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.

Affections

  • Huntington Disease

Éligibilité

SexeTous
Âges23 Years – 65 Years
Volontaires sainsNon

Éligibilité telle qu'elle figure dans le registre

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.

Éligibilité en énoncés simples

Les critères de cette fiche n'ont pas encore été décomposés en énoncés distincts. Le texte du registre ci-dessus est complet et fait foi.

Conception de l'étude

Type d'étudeInterventionnelle
PhasePhase 3
RépartitionRandomisée
Modèle d'interventionPARALLEL
Objectif principalTREATMENT
InsuQUADRUPLE (4)
Recrutement400 participants recherchés

Promoteur et collaborateurs

  • Prilenia Promoteur
  • Ferrer Internacional S.A. Collaborateurs

Groupes et interventions

  • 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.

Interventions

  • Médicament Placebo

    Matched placebo hard gelatin capsule

  • Médicament Pridopidine

    Pridopidine hard gelatin capsule

Critères de jugement

  1. Critère de jugement principal

    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.

    Délai Change from Baseline to Week 52

  2. Critère de jugement secondaire

    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.

    Délai Change from Baseline to Week 52

  3. Critère de jugement secondaire

    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.

    Délai Change from Baseline to Week 52

  4. Critère de jugement secondaire

    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.

    Délai Change from Baseline to Week 52

Dates

Date de début1 juillet 2026 (réelle)
Achèvement principal1 juin 2028 (estimée)
Achèvement1 juin 2030 (estimée)
Première publication27 mai 2026 (réelle)
Dernière mise à jour17 septembre 2026
Résultats publiésNon précisé dans la fiche du registre
Statut vérifié pour la dernière foismai 2026

Réelle signifie que l'événement a eu lieu. Estimée signifie que le promoteur s'y attend. Les deux ont un sens différent.

Lieux

5 sites sont en recrutement

Belgium

ÉtablissementVilleÉtat ou régionStatut
Hopital ErasmeAnderlechtPas encore en recrutement
Cliniques Universitaires Saint-LucWoluwe-Saint-LambertPas encore en recrutement

Canada

ÉtablissementVilleÉtat ou régionStatut
University of CalgaryCalgaryAlbertaPas encore en recrutement
University of AlbertaEdmontonAlbertaPas encore en recrutement
Centre Hospitalier de l'Universite de Montreal (CHUM)MontrealQuebecPas encore en recrutement
North York General HospitalTorontoOntarioPas encore en recrutement
University of British ColumbiaVancouverBritish ColumbiaPas encore en recrutement

Czechia

ÉtablissementVilleÉtat ou régionStatut
Vseobecna Fakultni Nemocnice V PrazePraguePas encore en recrutement

France

ÉtablissementVilleÉtat ou régionStatut
Centre Hospitalier Universitaire D'AngersAngersPas encore en recrutement
CHU Henri Mondor (APHP)CréteilPas encore en recrutement
Centre Hospitalier Universitaire Grenoble AlpesLa TronchePas encore en recrutement
Centre Hospitalier Regional De MarseilleMarseillePas encore en recrutement

Germany

ÉtablissementVilleÉtat ou régionStatut
Universitaetsklinikum Aachen AöRAachenPas encore en recrutement
Katholisches Klinikum Bochum gGmbHBochumPas encore en recrutement
Universitätsklinikum ErlangenErlangenPas encore en recrutement
George-Huntington-Institut GmbHMünsterPas encore en recrutement
Isar Amper KlinikumTaufkirchenPas encore en recrutement
Universitaetsklinikum Ulm AöRUlmPas encore en recrutement

Italy

ÉtablissementVilleÉtat ou régionStatut
Azienda Unita Sanitaria Locale Di BolognaBolognaPas encore en recrutement
IRCCS Istituto Auxologico ItalianoMilanPas encore en recrutement
Azienda Ospedaliera Universitaria Federico II Di NapoliNaplesPas encore en recrutement
Azienda Ospedale Università di PadovaPadovaPas encore en recrutement
Fondazione Policlinico Universitario Agostino Gemelli IRCCSRomaPas encore en recrutement
Casa Sollievo Della SofferenzaSan Giovanni RotondoPas encore en recrutement

Netherlands

ÉtablissementVilleÉtat ou régionStatut
Leids Universitair Medisch Centrum (LUMC)LeidenPas encore en recrutement

Poland

ÉtablissementVilleÉtat ou régionStatut
Krakowska Akademia Neurologii Sp. z o.o.KrakowPas encore en recrutement
Wojskowy Instytut Medycyny LotniczejWarsawPas encore en recrutement

Portugal

ÉtablissementVilleÉtat ou régionStatut
Unidade Local De Saude De Coimbra E.P.ECoimbraPas encore en recrutement
Unidade Local De Saude De Santa Maria E.P.E.LisbonPas encore en recrutement
Unidade Local De Saude De Santo Antonio E.P.E.PortoPas encore en recrutement
CNS Saude Lda.Torres VedrasPas encore en recrutement

Spain

ÉtablissementVilleÉtat ou régionStatut
Hospital Universitario De CrucesBarakaldoPas encore en recrutement
Hospital De La Santa Creu I Sant PauBarcelonaPas encore en recrutement
Hospital Universitario Ramon Y CajalMadridPas encore en recrutement
Hospital Universitario Central De AsturiasOviedoPas encore en recrutement
Hospital Universitario Virgen del RocíoSevillePas encore en recrutement

United Kingdom

ÉtablissementVilleÉtat ou régionStatut
NHS GrampianAberdeenPas encore en recrutement
Birmingham and Solihull Mental Health Foundation NHS TrustBirminghamPas encore en recrutement
Cardiff UniversityCardiffPas encore en recrutement
The Walton Centre NHS Foundation TrustLiverpoolPas encore en recrutement
CNTW NHS Foundation TrustNewcastle upon TynePas encore en recrutement

United States

ÉtablissementVilleÉtat ou régionStatut
Johns Hopkins UniversityBaltimoreMarylandPas encore en recrutement
The University of Alabama at BirminghamBirminghamAlabamaPas encore en recrutement
University of Miami-U Health Boca RatonBoca RatonFloridaPas encore en recrutement
University of Vermont Medical CenterBurlingtonVermontPas encore en recrutement
Massachusetts General HospitalCharlestownMassachusettsPas encore en recrutement
Rush University Medical CenterChicagoIllinoisPas encore en recrutement
Northwestern UniversityChicagoIllinoisPas encore en recrutement
University of CincinnatiCincinnatiOhioPas encore en recrutement
Cleveland ClinicClevelandOhioPas encore en recrutement

16 sites supplémentaires sont indiqués dans la fiche du registre.

Documents de l'étude

Aucun document n'est lié dans cette fiche du registre.

Évolution au fil du temps

  1. 7 août 2026

    Statut modifié

    Status changed from Not yet recruiting to Recruiting

    NOT_YET_RECRUITINGRECRUITING

  2. 7 août 2026

    Recrutement ouvert

    Recruitment opened

    NOT_YET_RECRUITINGRECRUITING

  3. 7 août 2026

    Site ajouté

    66 sites added (66 total)

    066

Registre: ClinicalTrials.gov · NCT########

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