Pridopidine Phase 3 Study in Huntington's Disease (PRECISE-HD)
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.
Descripción general del estudio
Estado
Condiciones
Intervención / Tratamiento
Descripción detallada
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.
Tipo de estudio
Inscripción (Estimado)
Fase
- Fase 3
Contactos y Ubicaciones
Estudio Contacto
- Nombre: Prilenia Medical Information
- Número de teléfono: 018575745755
- Correo electrónico: MedInfo@prilenia.com
Ubicaciones de estudio
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Aachen, Alemania
- Aún no reclutando
- Universitaetsklinikum Aachen AöR
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Bochum, Alemania
- Aún no reclutando
- Katholisches Klinikum Bochum gGmbH
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Erlangen, Alemania
- Aún no reclutando
- Universitätsklinikum Erlangen
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Münster, Alemania
- Aún no reclutando
- George-Huntington-Institut GmbH
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Taufkirchen, Alemania
- Aún no reclutando
- Isar Amper Klinikum
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Ulm, Alemania
- Aún no reclutando
- Universitaetsklinikum Ulm AöR
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Anderlecht, Bélgica
- Aún no reclutando
- Hopital Erasme
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Woluwe-Saint-Lambert, Bélgica
- Aún no reclutando
- Cliniques universitaires Saint-Luc
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Alberta
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Calgary, Alberta, Canadá
- Aún no reclutando
- University Of Calgary
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Edmonton, Alberta, Canadá
- Aún no reclutando
- University of Alberta
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British Columbia
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Vancouver, British Columbia, Canadá
- Aún no reclutando
- University of British Columbia
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Ontario
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Toronto, Ontario, Canadá
- Aún no reclutando
- North York General Hospital
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Quebec
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Montreal, Quebec, Canadá
- Aún no reclutando
- Centre hospitalier de l'Université de Montréal (CHUM)
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Prague, Chequia
- Aún no reclutando
- Vseobecna fakultni nemocnice v Praze
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Barakaldo, España
- Aún no reclutando
- Hospital Universitario de Cruces
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Barcelona, España
- Aún no reclutando
- Hospital de La Santa Creu i Sant Pau
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Madrid, España
- Aún no reclutando
- Hospital Universitario Ramon y Cajal
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Oviedo, España
- Aún no reclutando
- Hospital Universitario Central de Asturias
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Seville, España
- Aún no reclutando
- Hospital Universitario Virgen del Rocío
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Alabama
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Birmingham, Alabama, Estados Unidos, 35294
- Aún no reclutando
- The University of Alabama at Birmingham
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California
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La Jolla, California, Estados Unidos, 92037
- Reclutamiento
- University of California, San Diego
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Contacto:
- Alex Scott
- Número de teléfono: 858-249-0569
- Correo electrónico: als074@health.ucsd.edu
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Los Angeles, California, Estados Unidos, 90095
- Aún no reclutando
- UCLA Medical Center
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Sacramento, California, Estados Unidos, 95817
- Aún no reclutando
- UC Davis Medical Center
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District of Columbia
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Washington D.C., District of Columbia, Estados Unidos, 20007
- Aún no reclutando
- Georgetown University
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Florida
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Boca Raton, Florida, Estados Unidos, 33486
- Aún no reclutando
- University of Miami-U Health Boca Raton
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Gainesville, Florida, Estados Unidos, 32608
- Aún no reclutando
- University of Florida (Norman Fixel Institute for Neurological Diseases)
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Illinois
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Chicago, Illinois, Estados Unidos, 60611
- Aún no reclutando
- Northwestern University
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Chicago, Illinois, Estados Unidos, 60612
- Aún no reclutando
- Rush University Medical Center
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Kansas
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Kansas City, Kansas, Estados Unidos, 66103
- Aún no reclutando
- University of Kansas Medical Center
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Wichita, Kansas, Estados Unidos, 67226
- Reclutamiento
- Hereditary Neurological Disease Centre
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Contacto:
- Gregory Suter
- Número de teléfono: 316-609-3020
- Correo electrónico: gregory@hndcentre.com
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Maryland
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Baltimore, Maryland, Estados Unidos, 60612
- Aún no reclutando
- Johns Hopkins University
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Massachusetts
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Charlestown, Massachusetts, Estados Unidos, 02129
- Aún no reclutando
- Massachusetts General Hospital
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Minnesota
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Golden Valley, Minnesota, Estados Unidos, 55427
- Aún no reclutando
- Struthers Parkinson's Center / Health Partners Institute
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New York
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New York, New York, Estados Unidos, 10032
- Aún no reclutando
- Columbia University
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North Carolina
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Durham, North Carolina, Estados Unidos, 27705
- Aún no reclutando
- Duke University
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Ohio
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Cincinnati, Ohio, Estados Unidos, 45219
- Aún no reclutando
- University of Cincinnati
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Cleveland, Ohio, Estados Unidos, 44195
- Aún no reclutando
- Cleveland Clinic
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Columbus, Ohio, Estados Unidos, 43221
- Reclutamiento
- The Ohio State University
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Oregon
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Portland, Oregon, Estados Unidos, 97239
- Aún no reclutando
- Oregon Health and Science University (OHSU)
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Tennessee
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Nashville, Tennessee, Estados Unidos, 37212
- Reclutamiento
- Vanderbilt University Medical Center
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Texas
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Houston, Texas, Estados Unidos, 77030
- Aún no reclutando
- UT Health, Houston, McGovern Medical School
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Vermont
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Burlington, Vermont, Estados Unidos, 05401
- Aún no reclutando
- University of Vermont Medical Center
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Washington
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Kirkland, Washington, Estados Unidos, 98034
- Aún no reclutando
- EvergreenHealth Research Department
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Seattle, Washington, Estados Unidos, 98195
- Reclutamiento
- University of Washington
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Angers, Francia
- Aún no reclutando
- Centre Hospitalier Universitaire d'Angers
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Créteil, Francia
- Aún no reclutando
- CHU Henri Mondor (APHP)
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La Tronche, Francia
- Aún no reclutando
- Centre Hospitalier Universitaire Grenoble Alpes
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Marseille, Francia
- Aún no reclutando
- Centre Hospitalier Regional De Marseille
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Bologna, Italia
- Aún no reclutando
- Azienda Unita Sanitaria Locale Di Bologna
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Milan, Italia
- Aún no reclutando
- IRCCS Istituto Auxologico Italiano
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Naples, Italia
- Aún no reclutando
- Azienda Ospedaliera Universitaria Federico II di Napoli
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Padova, Italia
- Aún no reclutando
- Azienda Ospedale Università di Padova
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Roma, Italia
- Aún no reclutando
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
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San Giovanni Rotondo, Italia
- Aún no reclutando
- Casa Sollievo Della Sofferenza
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Leiden, Países Bajos
- Aún no reclutando
- Leids Universitair Medisch Centrum (LUMC)
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Krakow, Polonia
- Aún no reclutando
- Krakowska Akademia Neurologii Sp. z o.o.
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Warsaw, Polonia
- Aún no reclutando
- Wojskowy Instytut Medycyny Lotniczej
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Coimbra, Portugal
- Aún no reclutando
- Unidade Local De Saude De Coimbra E.P.E
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Lisbon, Portugal
- Aún no reclutando
- Unidade Local De Saude De Santa Maria E.P.E.
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Porto, Portugal
- Aún no reclutando
- Unidade Local De Saude De Santo Antonio E.P.E.
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Torres Vedras, Portugal
- Aún no reclutando
- CNS Saude Lda.
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Aberdeen, Reino Unido
- Aún no reclutando
- NHS Grampian
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Birmingham, Reino Unido
- Aún no reclutando
- Birmingham and Solihull Mental Health Foundation NHS Trust
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Cardiff, Reino Unido
- Aún no reclutando
- Cardiff University
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Liverpool, Reino Unido
- Aún no reclutando
- The Walton Centre NHS Foundation Trust
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Newcastle upon Tyne, Reino Unido
- Aún no reclutando
- CNTW NHS Foundation Trust
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Criterios de participación
Criterio de elegibilidad
Edades elegibles para estudiar
- Adulto
- Adulto Mayor
Acepta Voluntarios Saludables
Descripción
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.
Plan de estudios
¿Cómo está diseñado el estudio?
Detalles de diseño
- Propósito principal: Tratamiento
- Asignación: Aleatorizado
- Modelo Intervencionista: Asignación paralela
- Enmascaramiento: Cuadruplicar
Armas e Intervenciones
Grupo de participantes/brazo |
Intervención / Tratamiento |
|---|---|
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Experimental: 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.
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Cápsula de gelatina dura de pridopidina
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Comparador de placebos: 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.
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Matched placebo hard gelatin capsule
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¿Qué mide el estudio?
Medidas de resultado primarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
|---|---|---|
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Change in Composite Unified Huntington's Disease Rating Scale (cUHDRS)
Periodo de tiempo: Change from Baseline to Week 52
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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
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Medidas de resultado secundarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
|---|---|---|
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Change in Total Functional Capacity (TFC) score
Periodo de tiempo: Change from Baseline to Week 52
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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.
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Change from Baseline to Week 52
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Change in Quantitative Motor (Q-Motor) Finger Tapping Inter-Onset Interval (FT-IOI) mean
Periodo de tiempo: Change from Baseline to Week 52
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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.
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Change from Baseline to Week 52
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Change in Stroop Word Reading (SWR) score
Periodo de tiempo: Change from Baseline to Week 52
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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.
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Change from Baseline to Week 52
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Colaboradores e Investigadores
Patrocinador
Colaboradores
Fechas de registro del estudio
Fechas importantes del estudio
Inicio del estudio (Actual)
Finalización primaria (Estimado)
Finalización del estudio (Estimado)
Fechas de registro del estudio
Enviado por primera vez
Primero enviado que cumplió con los criterios de control de calidad
Publicado por primera vez (Actual)
Actualizaciones de registros de estudio
Última actualización publicada (Actual)
Última actualización enviada que cumplió con los criterios de control de calidad
Última verificación
Más información
Términos relacionados con este estudio
Términos MeSH relevantes adicionales
- Enfermedades Cerebrales
- Enfermedades del Sistema Nervioso Central
- Enfermedades del Sistema Nervioso
- Desordenes mentales
- Enfermedades Genéticas Congénitas
- Trastornos neurocognitivos
- Trastornos cognitivos
- Demencia
- Enfermedades neurodegenerativas
- Trastornos del movimiento
- Trastornos Heredodegenerativos, Sistema Nervioso
- Enfermedades de los ganglios basales
- Discinesias
- Corea
- Enfermedades y anomalías congénitas, hereditarias y neonatales
- Enfermedad de Huntington
- pridopidina
Otros números de identificación del estudio
- PL101-HD302/ FNP253-CT-2502
- 2026-526093-16-00 (Ctis)
Información sobre medicamentos y dispositivos, documentos del estudio
Estudia un producto farmacéutico regulado por la FDA de EE. UU.
Estudia un producto de dispositivo regulado por la FDA de EE. UU.
Esta información se obtuvo directamente del sitio web clinicaltrials.gov sin cambios. Si tiene alguna solicitud para cambiar, eliminar o actualizar los detalles de su estudio, comuníquese con register@clinicaltrials.gov. Tan pronto como se implemente un cambio en clinicaltrials.gov, también se actualizará automáticamente en nuestro sitio web. .