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Pridopidine Phase 3 Study in Huntington's Disease (PRECISE-HD)

14. september 2026 oppdatert av: Prilenia

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

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.

Studieoversikt

Status

Rekruttering

Forhold

Intervensjon / Behandling

Detaljert beskrivelse

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.

Studietype

Intervensjonell

Registrering (Antatt)

400

Fase

  • Fase 3

Kontakter og plasseringer

Denne delen inneholder kontaktinformasjon for de som utfører studien, og informasjon om hvor denne studien blir utført.

Studiekontakt

Studiesteder

  • Belgia
      • Anderlecht, Belgia
        • Har ikke rekruttert ennå
        • Hopital Erasme
      • Woluwe-Saint-Lambert, Belgia
        • Har ikke rekruttert ennå
        • Cliniques universitaires Saint-Luc
  • Canada
    • Alberta
      • Calgary, Alberta, Canada
        • Har ikke rekruttert ennå
        • University Of Calgary
      • Edmonton, Alberta, Canada
        • Har ikke rekruttert ennå
        • University of Alberta
    • British Columbia
      • Vancouver, British Columbia, Canada
        • Har ikke rekruttert ennå
        • University of British Columbia
    • Ontario
      • Toronto, Ontario, Canada
        • Har ikke rekruttert ennå
        • North York General Hospital
    • Quebec
      • Montreal, Quebec, Canada
        • Har ikke rekruttert ennå
        • Centre hospitalier de l'Université de Montréal (CHUM)
  • Forente stater
    • Alabama
      • Birmingham, Alabama, Forente stater, 35294
        • Har ikke rekruttert ennå
        • The University of Alabama at Birmingham
    • California
      • La Jolla, California, Forente stater, 92037
        • Rekruttering
        • University of California, San Diego
        • Ta kontakt med:
      • Los Angeles, California, Forente stater, 90095
        • Har ikke rekruttert ennå
        • UCLA Medical Center
      • Sacramento, California, Forente stater, 95817
        • Har ikke rekruttert ennå
        • UC Davis Medical Center
    • District of Columbia
      • Washington D.C., District of Columbia, Forente stater, 20007
        • Har ikke rekruttert ennå
        • Georgetown University
    • Florida
      • Boca Raton, Florida, Forente stater, 33486
        • Har ikke rekruttert ennå
        • University of Miami-U Health Boca Raton
      • Gainesville, Florida, Forente stater, 32608
        • Har ikke rekruttert ennå
        • University of Florida (Norman Fixel Institute for Neurological Diseases)
    • Illinois
      • Chicago, Illinois, Forente stater, 60611
        • Har ikke rekruttert ennå
        • Northwestern University
      • Chicago, Illinois, Forente stater, 60612
        • Har ikke rekruttert ennå
        • Rush University Medical Center
    • Kansas
      • Kansas City, Kansas, Forente stater, 66103
        • Har ikke rekruttert ennå
        • University of Kansas Medical Center
      • Wichita, Kansas, Forente stater, 67226
        • Rekruttering
        • Hereditary Neurological Disease Centre
        • Ta kontakt med:
    • Maryland
      • Baltimore, Maryland, Forente stater, 60612
        • Har ikke rekruttert ennå
        • Johns Hopkins University
    • Massachusetts
      • Charlestown, Massachusetts, Forente stater, 02129
        • Har ikke rekruttert ennå
        • Massachusetts General Hospital
    • Minnesota
      • Golden Valley, Minnesota, Forente stater, 55427
        • Har ikke rekruttert ennå
        • Struthers Parkinson's Center / Health Partners Institute
    • New York
      • New York, New York, Forente stater, 10032
        • Har ikke rekruttert ennå
        • Columbia University
    • North Carolina
      • Durham, North Carolina, Forente stater, 27705
        • Har ikke rekruttert ennå
        • Duke University
    • Ohio
      • Cincinnati, Ohio, Forente stater, 45219
        • Har ikke rekruttert ennå
        • University of Cincinnati
      • Cleveland, Ohio, Forente stater, 44195
        • Har ikke rekruttert ennå
        • Cleveland Clinic
      • Columbus, Ohio, Forente stater, 43221
        • Rekruttering
        • The Ohio State University
    • Oregon
      • Portland, Oregon, Forente stater, 97239
        • Har ikke rekruttert ennå
        • Oregon Health and Science University (OHSU)
    • Tennessee
      • Nashville, Tennessee, Forente stater, 37212
        • Rekruttering
        • Vanderbilt University Medical Center
    • Texas
      • Houston, Texas, Forente stater, 77030
        • Har ikke rekruttert ennå
        • UT Health, Houston, McGovern Medical School
    • Vermont
      • Burlington, Vermont, Forente stater, 05401
        • Har ikke rekruttert ennå
        • University of Vermont Medical Center
    • Washington
      • Kirkland, Washington, Forente stater, 98034
        • Har ikke rekruttert ennå
        • EvergreenHealth Research Department
      • Seattle, Washington, Forente stater, 98195
        • Rekruttering
        • University of Washington
  • Frankrike
      • Angers, Frankrike
        • Har ikke rekruttert ennå
        • Centre Hospitalier Universitaire d'Angers
      • Créteil, Frankrike
        • Har ikke rekruttert ennå
        • CHU Henri Mondor (APHP)
      • La Tronche, Frankrike
        • Har ikke rekruttert ennå
        • Centre Hospitalier Universitaire Grenoble Alpes
      • Marseille, Frankrike
        • Har ikke rekruttert ennå
        • Centre Hospitalier Regional De Marseille
  • Italia
      • Bologna, Italia
        • Har ikke rekruttert ennå
        • Azienda Unita Sanitaria Locale Di Bologna
      • Milan, Italia
        • Har ikke rekruttert ennå
        • IRCCS Istituto Auxologico Italiano
      • Naples, Italia
        • Har ikke rekruttert ennå
        • Azienda Ospedaliera Universitaria Federico II di Napoli
      • Padova, Italia
        • Har ikke rekruttert ennå
        • Azienda Ospedale Università di Padova
      • Roma, Italia
        • Har ikke rekruttert ennå
        • Fondazione Policlinico Universitario Agostino Gemelli IRCCS
      • San Giovanni Rotondo, Italia
        • Har ikke rekruttert ennå
        • Casa Sollievo Della Sofferenza
  • Nederland
      • Leiden, Nederland
        • Har ikke rekruttert ennå
        • Leids Universitair Medisch Centrum (LUMC)
  • Polen
      • Krakow, Polen
        • Har ikke rekruttert ennå
        • Krakowska Akademia Neurologii Sp. z o.o.
      • Warsaw, Polen
        • Har ikke rekruttert ennå
        • Wojskowy Instytut Medycyny Lotniczej
  • Portugal
      • Coimbra, Portugal
        • Har ikke rekruttert ennå
        • Unidade Local De Saude De Coimbra E.P.E
      • Lisbon, Portugal
        • Har ikke rekruttert ennå
        • Unidade Local De Saude De Santa Maria E.P.E.
      • Porto, Portugal
        • Har ikke rekruttert ennå
        • Unidade Local De Saude De Santo Antonio E.P.E.
      • Torres Vedras, Portugal
        • Har ikke rekruttert ennå
        • CNS Saude Lda.
  • Spania
      • Barakaldo, Spania
        • Har ikke rekruttert ennå
        • Hospital Universitario de Cruces
      • Barcelona, Spania
        • Har ikke rekruttert ennå
        • Hospital de La Santa Creu i Sant Pau
      • Madrid, Spania
        • Har ikke rekruttert ennå
        • Hospital Universitario Ramon y Cajal
      • Oviedo, Spania
        • Har ikke rekruttert ennå
        • Hospital Universitario Central de Asturias
      • Seville, Spania
        • Har ikke rekruttert ennå
        • Hospital Universitario Virgen del Rocío
  • Storbritannia
      • Aberdeen, Storbritannia
        • Har ikke rekruttert ennå
        • NHS Grampian
      • Birmingham, Storbritannia
        • Har ikke rekruttert ennå
        • Birmingham and Solihull Mental Health Foundation NHS Trust
      • Cardiff, Storbritannia
        • Har ikke rekruttert ennå
        • Cardiff University
      • Liverpool, Storbritannia
        • Har ikke rekruttert ennå
        • The Walton Centre NHS Foundation Trust
      • Newcastle upon Tyne, Storbritannia
        • Har ikke rekruttert ennå
        • CNTW NHS Foundation Trust
  • Tsjekkia
      • Prague, Tsjekkia
        • Har ikke rekruttert ennå
        • Vseobecna fakultni nemocnice v Praze
  • Tyskland
      • Aachen, Tyskland
        • Har ikke rekruttert ennå
        • Universitaetsklinikum Aachen AöR
      • Bochum, Tyskland
        • Har ikke rekruttert ennå
        • Katholisches Klinikum Bochum gGmbH
      • Erlangen, Tyskland
        • Har ikke rekruttert ennå
        • Universitätsklinikum Erlangen
      • Münster, Tyskland
        • Har ikke rekruttert ennå
        • George-Huntington-Institut GmbH
      • Taufkirchen, Tyskland
        • Har ikke rekruttert ennå
        • Isar Amper Klinikum
      • Ulm, Tyskland
        • Har ikke rekruttert ennå
        • Universitaetsklinikum Ulm AöR

Deltakelseskriterier

Forskere ser etter personer som passer til en bestemt beskrivelse, kalt kvalifikasjonskriterier. Noen eksempler på disse kriteriene er en persons generelle helsetilstand eller tidligere behandlinger.

Kvalifikasjonskriterier

Alder som er kvalifisert for studier

  • Voksen
  • Eldre voksen

Tar imot friske frivillige

Nei

Beskrivelse

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.

Studieplan

Denne delen gir detaljer om studieplanen, inkludert hvordan studien er utformet og hva studien måler.

Hvordan er studiet utformet?

Designdetaljer

  • Primært formål: Behandling
  • Tildeling: Randomisert
  • Intervensjonsmodell: Parallell tildeling
  • Masking: Firemannsrom

Våpen og intervensjoner

Deltakergruppe / Arm
Intervensjon / Behandling
Eksperimentell: Pridopidine
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.
Legemiddel: Pridopidin
Pridopidin hard gelatinkapsel
Placebo komparator: Placebo
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.
Legemiddel: Placebo
Matched placebo hard gelatin capsule

Hva måler studien?

Primære resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
Change in Composite Unified Huntington's Disease Rating Scale (cUHDRS)
Tidsramme: Change from Baseline to Week 52

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.
Change from Baseline to Week 52

Sekundære resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
Change in Total Functional Capacity (TFC) score
Tidsramme: Change from Baseline to Week 52
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.
Change from Baseline to Week 52
Change in Quantitative Motor (Q-Motor) Finger Tapping Inter-Onset Interval (FT-IOI) mean
Tidsramme: Change from Baseline to Week 52
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.
Change from Baseline to Week 52
Change in Stroop Word Reading (SWR) score
Tidsramme: Change from Baseline to Week 52
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.
Change from Baseline to Week 52

Samarbeidspartnere og etterforskere

Det er her du vil finne personer og organisasjoner som er involvert i denne studien.

Sponsor

Prilenia

Samarbeidspartnere

Ferrer Internacional S.A.

Studierekorddatoer

Disse datoene sporer fremdriften for innsending av studieposter og sammendragsresultater til ClinicalTrials.gov. Studieposter og rapporterte resultater gjennomgås av National Library of Medicine (NLM) for å sikre at de oppfyller spesifikke kvalitetskontrollstandarder før de legges ut på det offentlige nettstedet.

Studer hoveddatoer

Studiestart (Faktiske)

1. juli 2026

Primær fullføring (Antatt)

1. juni 2028

Studiet fullført (Antatt)

1. juni 2030

Datoer for studieregistrering

Først innsendt

20. mai 2026

Først innsendt som oppfylte QC-kriteriene

20. mai 2026

Først lagt ut (Faktiske)

27. mai 2026

Oppdateringer av studieposter

Sist oppdatering lagt ut (Faktiske)

17. september 2026

Siste oppdatering sendt inn som oppfylte QC-kriteriene

14. september 2026

Sist bekreftet

1. mai 2026

Mer informasjon

Begreper knyttet til denne studien

Ytterligere relevante MeSH-vilkår

Andre studie-ID-numre

  • PL101-HD302/ FNP253-CT-2502
  • 2026-526093-16-00 (Ctis)

Legemiddel- og utstyrsinformasjon, studiedokumenter

Studerer et amerikansk FDA-regulert medikamentprodukt

Ja

Studerer et amerikansk FDA-regulert enhetsprodukt

Nei

Denne informasjonen ble hentet direkte fra nettstedet clinicaltrials.gov uten noen endringer. Hvis du har noen forespørsler om å endre, fjerne eller oppdatere studiedetaljene dine, vennligst kontakt register@clinicaltrials.gov. Så snart en endring er implementert på clinicaltrials.gov, vil denne også bli oppdatert automatisk på nettstedet vårt. .

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