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.
연구 개요
상세 설명
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.
연구 유형
등록 (추정된)
단계
- 3단계
연락처 및 위치
연구 연락처
- 이름: Prilenia Medical Information
- 전화번호: 018575745755
- 이메일: MedInfo@prilenia.com
연구 장소
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Leiden, 네덜란드
- 아직 모집하지 않음
- Leids Universitair Medisch Centrum (LUMC)
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Aachen, 독일
- 아직 모집하지 않음
- Universitaetsklinikum Aachen AöR
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Bochum, 독일
- 아직 모집하지 않음
- Katholisches Klinikum Bochum gGmbH
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Erlangen, 독일
- 아직 모집하지 않음
- Universitätsklinikum Erlangen
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Münster, 독일
- 아직 모집하지 않음
- George-Huntington-Institut GmbH
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Taufkirchen, 독일
- 아직 모집하지 않음
- Isar Amper Klinikum
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Ulm, 독일
- 아직 모집하지 않음
- Universitaetsklinikum Ulm AöR
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Alabama
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Birmingham, Alabama, 미국, 35294
- 아직 모집하지 않음
- The University of Alabama at Birmingham
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California
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La Jolla, California, 미국, 92037
- 모병
- University of California, San Diego
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연락하다:
- Alex Scott
- 전화번호: 858-249-0569
- 이메일: als074@health.ucsd.edu
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Los Angeles, California, 미국, 90095
- 아직 모집하지 않음
- UCLA Medical Center
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Sacramento, California, 미국, 95817
- 아직 모집하지 않음
- UC Davis Medical Center
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District of Columbia
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Washington D.C., District of Columbia, 미국, 20007
- 아직 모집하지 않음
- Georgetown University
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Florida
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Boca Raton, Florida, 미국, 33486
- 아직 모집하지 않음
- University of Miami-U Health Boca Raton
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Gainesville, Florida, 미국, 32608
- 아직 모집하지 않음
- University of Florida (Norman Fixel Institute for Neurological Diseases)
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Illinois
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Chicago, Illinois, 미국, 60611
- 아직 모집하지 않음
- Northwestern University
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Chicago, Illinois, 미국, 60612
- 아직 모집하지 않음
- Rush University Medical Center
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Kansas
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Kansas City, Kansas, 미국, 66103
- 아직 모집하지 않음
- University of Kansas Medical Center
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Wichita, Kansas, 미국, 67226
- 모병
- Hereditary Neurological Disease Centre
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연락하다:
- Gregory Suter
- 전화번호: 316-609-3020
- 이메일: gregory@hndcentre.com
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Maryland
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Baltimore, Maryland, 미국, 60612
- 아직 모집하지 않음
- Johns Hopkins University
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Massachusetts
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Charlestown, Massachusetts, 미국, 02129
- 아직 모집하지 않음
- Massachusetts General Hospital
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Minnesota
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Golden Valley, Minnesota, 미국, 55427
- 아직 모집하지 않음
- Struthers Parkinson's Center / Health Partners Institute
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New York
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New York, New York, 미국, 10032
- 아직 모집하지 않음
- Columbia University
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North Carolina
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Durham, North Carolina, 미국, 27705
- 아직 모집하지 않음
- Duke University
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Ohio
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Cincinnati, Ohio, 미국, 45219
- 아직 모집하지 않음
- University of Cincinnati
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Cleveland, Ohio, 미국, 44195
- 아직 모집하지 않음
- Cleveland Clinic
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Columbus, Ohio, 미국, 43221
- 모병
- The Ohio State University
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Oregon
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Portland, Oregon, 미국, 97239
- 아직 모집하지 않음
- Oregon Health and Science University (OHSU)
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Tennessee
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Nashville, Tennessee, 미국, 37212
- 모병
- Vanderbilt University Medical Center
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Texas
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Houston, Texas, 미국, 77030
- 아직 모집하지 않음
- UT Health, Houston, McGovern Medical School
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Vermont
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Burlington, Vermont, 미국, 05401
- 아직 모집하지 않음
- University of Vermont Medical Center
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Washington
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Kirkland, Washington, 미국, 98034
- 아직 모집하지 않음
- EvergreenHealth Research Department
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Seattle, Washington, 미국, 98195
- 모병
- University of Washington
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Anderlecht, 벨기에
- 아직 모집하지 않음
- Hopital Erasme
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Woluwe-Saint-Lambert, 벨기에
- 아직 모집하지 않음
- Cliniques universitaires Saint-Luc
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Barakaldo, 스페인
- 아직 모집하지 않음
- Hospital Universitario de Cruces
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Barcelona, 스페인
- 아직 모집하지 않음
- Hospital de La Santa Creu i Sant Pau
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Madrid, 스페인
- 아직 모집하지 않음
- Hospital Universitario Ramon y Cajal
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Oviedo, 스페인
- 아직 모집하지 않음
- Hospital Universitario Central de Asturias
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Seville, 스페인
- 아직 모집하지 않음
- Hospital Universitario Virgen del Rocío
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Aberdeen, 영국
- 아직 모집하지 않음
- NHS Grampian
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Birmingham, 영국
- 아직 모집하지 않음
- Birmingham and Solihull Mental Health Foundation NHS Trust
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Cardiff, 영국
- 아직 모집하지 않음
- Cardiff University
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Liverpool, 영국
- 아직 모집하지 않음
- The Walton Centre NHS Foundation Trust
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Newcastle upon Tyne, 영국
- 아직 모집하지 않음
- CNTW NHS Foundation Trust
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Bologna, 이탈리아
- 아직 모집하지 않음
- Azienda Unita Sanitaria Locale Di Bologna
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Milan, 이탈리아
- 아직 모집하지 않음
- IRCCS Istituto Auxologico Italiano
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Naples, 이탈리아
- 아직 모집하지 않음
- Azienda Ospedaliera Universitaria Federico II di Napoli
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Padova, 이탈리아
- 아직 모집하지 않음
- Azienda Ospedale Università di Padova
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Roma, 이탈리아
- 아직 모집하지 않음
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
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San Giovanni Rotondo, 이탈리아
- 아직 모집하지 않음
- Casa Sollievo Della Sofferenza
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Prague, 체코
- 아직 모집하지 않음
- Vseobecna fakultni nemocnice v Praze
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Alberta
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Calgary, Alberta, 캐나다
- 아직 모집하지 않음
- University Of Calgary
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Edmonton, Alberta, 캐나다
- 아직 모집하지 않음
- University of Alberta
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British Columbia
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Vancouver, British Columbia, 캐나다
- 아직 모집하지 않음
- University of British Columbia
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Ontario
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Toronto, Ontario, 캐나다
- 아직 모집하지 않음
- North York General Hospital
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Quebec
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Montreal, Quebec, 캐나다
- 아직 모집하지 않음
- Centre hospitalier de l'Université de Montréal (CHUM)
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Coimbra, 포르투갈
- 아직 모집하지 않음
- Unidade Local De Saude De Coimbra E.P.E
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Lisbon, 포르투갈
- 아직 모집하지 않음
- Unidade Local De Saude De Santa Maria E.P.E.
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Porto, 포르투갈
- 아직 모집하지 않음
- Unidade Local De Saude De Santo Antonio E.P.E.
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Torres Vedras, 포르투갈
- 아직 모집하지 않음
- CNS Saude Lda.
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Krakow, 폴란드
- 아직 모집하지 않음
- Krakowska Akademia Neurologii Sp. z o.o.
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Warsaw, 폴란드
- 아직 모집하지 않음
- Wojskowy Instytut Medycyny Lotniczej
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Angers, 프랑스
- 아직 모집하지 않음
- Centre Hospitalier Universitaire d'Angers
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Créteil, 프랑스
- 아직 모집하지 않음
- CHU Henri Mondor (APHP)
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La Tronche, 프랑스
- 아직 모집하지 않음
- Centre Hospitalier Universitaire Grenoble Alpes
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Marseille, 프랑스
- 아직 모집하지 않음
- Centre Hospitalier Regional De Marseille
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참여기준
자격 기준
공부할 수 있는 나이
- 성인
- 고령자
건강한 자원 봉사자를 받아들입니다
설명
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.
공부 계획
연구는 어떻게 설계됩니까?
디자인 세부사항
- 주 목적: 치료
- 할당: 무작위
- 중재 모델: 병렬 할당
- 마스킹: 네 배로
무기와 개입
참가자 그룹 / 팔 |
개입 / 치료 |
|---|---|
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실험적: 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.
|
프리도피딘 경질 젤라틴 캡슐
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위약 비교기: 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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연구는 무엇을 측정합니까?
주요 결과 측정
결과 측정 |
측정값 설명 |
기간 |
|---|---|---|
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Change in Composite Unified Huntington's Disease Rating Scale (cUHDRS)
기간: 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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2차 결과 측정
결과 측정 |
측정값 설명 |
기간 |
|---|---|---|
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Change in Total Functional Capacity (TFC) score
기간: 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
기간: 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
기간: 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
|
공동 작업자 및 조사자
스폰서
연구 기록 날짜
연구 주요 날짜
연구 시작 (실제)
기본 완료 (추정된)
연구 완료 (추정된)
연구 등록 날짜
최초 제출
QC 기준을 충족하는 최초 제출
처음 게시됨 (실제)
연구 기록 업데이트
마지막 업데이트 게시됨 (실제)
QC 기준을 충족하는 마지막 업데이트 제출
마지막으로 확인됨
추가 정보
이 정보는 변경 없이 clinicaltrials.gov 웹사이트에서 직접 가져온 것입니다. 귀하의 연구 세부 정보를 변경, 제거 또는 업데이트하도록 요청하는 경우 register@clinicaltrials.gov. 문의하십시오. 변경 사항이 clinicaltrials.gov에 구현되는 즉시 저희 웹사이트에도 자동으로 업데이트됩니다. .