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)

In reclutamentoAccetta partecipanti ora, secondo la scheda del registro.

Contatta questo studio

In breve

Fase 3400 partecipanti ricercati66 centri12 paesi

Categorie

Neurologia44%Malattie rare e genetiche44%Psichiatria e salute mentale13%

Registrato in 1 registro

ClinicalTrials.govNCT07609108Apri questa scheda su ClinicalTrials.govSincronizzato 3 giorni fa

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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.

Present: Registered before enrolment beganFirst posted 2026-05-27; recorded start 2026-07-01
Present: Has a defined primary outcomeA primary outcome measure is listed in the record
Present: The primary outcome states a time frameThe primary outcome measure records a time frame
Present: Participants are randomly assignedAllocation is recorded as randomised
Present: Has a comparison groupThe record lists a placebo or comparator arm, or more than one study arm
Present: Uses blinding (masking)A masking level is recorded in the record
Not stated: An ethics committee is not stated in the registry recordThis registry's ingested record has no ethics-committee field
Not stated: Regulatory oversight (such as an IND or IDE) is not stated in the registry recordThis registry's ingested record has no regulatory-authorisation field
Not stated: 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.
Present: No cost to participants is mentioned in the recordChecked the summary, description and eligibility text; no cost-to-participant phrase found
Present: Sponsor has 2 other studies in this databaseCounted from the lead sponsor named in the record (Prilenia)
Not stated: 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.

Sintesi

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.

Patologie

Huntington Disease

Idoneità

Idoneità
Sesso	Tutti
Età	23 Years – 65 Years
Volontari sani	No

Idoneità come riportata nel registro

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.

Idoneità in frasi semplici

I criteri di questa scheda non sono ancora stati suddivisi in affermazioni distinte. Il testo del registro qui sopra è completo ed è la versione autorevole.

Disegno dello studio

Disegno dello studio
Tipo di studio	Interventistico
Fase	Fase 3
Assegnazione	Randomizzata
Modello di intervento	PARALLEL
Finalità principale	TREATMENT
Mascheramento	QUADRUPLE (4)
Arruolamento	400 partecipanti ricercati

Sponsor e collaboratori

Prilenia Sponsor
Ferrer Internacional S.A. Collaboratori

Bracci e interventi

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.

Interventi

Farmaco Placebo
Matched placebo hard gelatin capsule
Farmaco Pridopidine
Pridopidine hard gelatin capsule

Esiti misurati

Esito primario
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.
Orizzonte temporale Change from Baseline to Week 52
Esito secondario
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.
Orizzonte temporale Change from Baseline to Week 52
Esito secondario
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.
Orizzonte temporale Change from Baseline to Week 52
Esito secondario
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.
Orizzonte temporale Change from Baseline to Week 52

Date

Date
Data di inizio	1 luglio 2026 (effettiva)
Conclusione primaria	1 giugno 2028 (stimata)
Conclusione	1 giugno 2030 (stimata)
Prima pubblicazione	27 maggio 2026 (effettiva)
Ultimo aggiornamento	17 settembre 2026
Risultati pubblicati	Non indicato nella scheda del registro
Stato verificato l'ultima volta	maggio 2026

«Effettiva» significa che l'evento è avvenuto. «Stimata» significa che lo sponsor lo prevede. Le due cose non sono equivalenti.

Centri

5 centri stanno reclutando

Belgium

Belgium
Struttura	Città	Stato o regione	Stato
Hopital Erasme	Anderlecht		Non ancora in reclutamento
Cliniques Universitaires Saint-Luc	Woluwe-Saint-Lambert		Non ancora in reclutamento

Canada

Canada
Struttura	Città	Stato o regione	Stato
University of Calgary	Calgary	Alberta	Non ancora in reclutamento
University of Alberta	Edmonton	Alberta	Non ancora in reclutamento
Centre Hospitalier de l'Universite de Montreal (CHUM)	Montreal	Quebec	Non ancora in reclutamento
North York General Hospital	Toronto	Ontario	Non ancora in reclutamento
University of British Columbia	Vancouver	British Columbia	Non ancora in reclutamento

Czechia

Czechia
Struttura	Città	Stato o regione	Stato
Vseobecna Fakultni Nemocnice V Praze	Prague		Non ancora in reclutamento

France

France
Struttura	Città	Stato
Centre Hospitalier Universitaire D'Angers	Angers	Non ancora in reclutamento
CHU Henri Mondor (APHP)	Créteil	Non ancora in reclutamento
Centre Hospitalier Universitaire Grenoble Alpes	La Tronche	Non ancora in reclutamento
Centre Hospitalier Regional De Marseille	Marseille	Non ancora in reclutamento

Germany

Germany
Struttura	Città	Stato
Universitaetsklinikum Aachen AöR	Aachen	Non ancora in reclutamento
Katholisches Klinikum Bochum gGmbH	Bochum	Non ancora in reclutamento
Universitätsklinikum Erlangen	Erlangen	Non ancora in reclutamento
George-Huntington-Institut GmbH	Münster	Non ancora in reclutamento
Isar Amper Klinikum	Taufkirchen	Non ancora in reclutamento
Universitaetsklinikum Ulm AöR	Ulm	Non ancora in reclutamento

Italy

Italy
Struttura	Città	Stato
Azienda Unita Sanitaria Locale Di Bologna	Bologna	Non ancora in reclutamento
IRCCS Istituto Auxologico Italiano	Milan	Non ancora in reclutamento
Azienda Ospedaliera Universitaria Federico II Di Napoli	Naples	Non ancora in reclutamento
Azienda Ospedale Università di Padova	Padova	Non ancora in reclutamento
Fondazione Policlinico Universitario Agostino Gemelli IRCCS	Roma	Non ancora in reclutamento
Casa Sollievo Della Sofferenza	San Giovanni Rotondo	Non ancora in reclutamento

Netherlands

Netherlands
Struttura	Città	Stato o regione	Stato
Leids Universitair Medisch Centrum (LUMC)	Leiden		Non ancora in reclutamento

Poland

Poland
Struttura	Città	Stato o regione	Stato
Krakowska Akademia Neurologii Sp. z o.o.	Krakow		Non ancora in reclutamento
Wojskowy Instytut Medycyny Lotniczej	Warsaw		Non ancora in reclutamento

Portugal

Portugal
Struttura	Città	Stato
Unidade Local De Saude De Coimbra E.P.E	Coimbra	Non ancora in reclutamento
Unidade Local De Saude De Santa Maria E.P.E.	Lisbon	Non ancora in reclutamento
Unidade Local De Saude De Santo Antonio E.P.E.	Porto	Non ancora in reclutamento
CNS Saude Lda.	Torres Vedras	Non ancora in reclutamento

Spain

Spain
Struttura	Città	Stato
Hospital Universitario De Cruces	Barakaldo	Non ancora in reclutamento
Hospital De La Santa Creu I Sant Pau	Barcelona	Non ancora in reclutamento
Hospital Universitario Ramon Y Cajal	Madrid	Non ancora in reclutamento
Hospital Universitario Central De Asturias	Oviedo	Non ancora in reclutamento
Hospital Universitario Virgen del Rocío	Seville	Non ancora in reclutamento

United Kingdom

United Kingdom
Struttura	Città	Stato
NHS Grampian	Aberdeen	Non ancora in reclutamento
Birmingham and Solihull Mental Health Foundation NHS Trust	Birmingham	Non ancora in reclutamento
Cardiff University	Cardiff	Non ancora in reclutamento
The Walton Centre NHS Foundation Trust	Liverpool	Non ancora in reclutamento
CNTW NHS Foundation Trust	Newcastle upon Tyne	Non ancora in reclutamento

United States

United States
Struttura	Città	Stato o regione	Stato
Johns Hopkins University	Baltimore	Maryland	Non ancora in reclutamento
The University of Alabama at Birmingham	Birmingham	Alabama	Non ancora in reclutamento
University of Miami-U Health Boca Raton	Boca Raton	Florida	Non ancora in reclutamento
University of Vermont Medical Center	Burlington	Vermont	Non ancora in reclutamento
Massachusetts General Hospital	Charlestown	Massachusetts	Non ancora in reclutamento
Rush University Medical Center	Chicago	Illinois	Non ancora in reclutamento
Northwestern University	Chicago	Illinois	Non ancora in reclutamento
University of Cincinnati	Cincinnati	Ohio	Non ancora in reclutamento
Cleveland Clinic	Cleveland	Ohio	Non ancora in reclutamento

Nella scheda del registro sono indicati altri 16 centri.

Documenti dello studio

In questa scheda del registro non sono collegati documenti.

Modifiche nel tempo

7 agosto 2026
Stato modificato
Status changed from Not yet recruiting to Recruiting
NOT_YET_RECRUITINGRECRUITING
7 agosto 2026
Reclutamento aperto
Recruitment opened
NOT_YET_RECRUITINGRECRUITING
7 agosto 2026
Centro aggiunto
66 sites added (66 total)
066

Registro: ClinicalTrials.gov · NCT########

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