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    Summary
    EudraCT Number:2020-002822-10
    Sponsor's Protocol Code Number:PL101-HD301
    National Competent Authority:Netherlands - Competent Authority
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2020-11-24
    Trial results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedNetherlands - Competent Authority
    A.2EudraCT number2020-002822-10
    A.3Full title of the trial
    A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Parallel Arm, Multicenter Study Evaluating the Efficacy and Safety of Pridopidine in Patients with Early Stage of Huntington Disease
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study of Pridopidine in Patients with Early Stage of Huntington Disease
    A.3.2Name or abbreviated title of the trial where available
    PRidopidine Outcome on Function in Huntington Disease (PROOF-HD)
    A.4.1Sponsor's protocol code numberPL101-HD301
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT04556656
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorPrilenia Neurotherapeutics Ltd.
    B.1.3.4CountryIsrael
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportPrilenia Neurotherapeutics Ltd.
    B.4.2CountryIsrael
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationPrilenia Neurotherapeutics Ltd.
    B.5.2Functional name of contact pointHead of Clinical Operations
    B.5.3 Address:
    B.5.3.1Street AddressHamenofim 10
    B.5.3.2Town/ cityHerzliya
    B.5.3.3Post code4672561
    B.5.3.4CountryIsrael
    B.5.4Telephone number+972 77-5558482
    B.5.6E-mailyael.cohen@prilenia.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation number05-2139
    D.3 Description of the IMP
    D.3.1Product namePridopidine
    D.3.2Product code PL101
    D.3.4Pharmaceutical form Capsule, hard
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNPRIDOPIDINE HYDROCHLORIDE
    D.3.9.1CAS number 882737-42-0
    D.3.9.2Current sponsor codePridiopidine
    D.3.9.3Other descriptive namePridiopidine
    D.3.9.4EV Substance CodeSUB78405
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number45
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboCapsule, hard
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Huntington Disease
    E.1.1.1Medical condition in easily understood language
    A hereditary disorder of the central nervous system that affects muscle coordination and leads to cognitive decline and psychiatric problems.
    E.1.1.2Therapeutic area Diseases [C] - Nervous System Diseases [C10]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10070668
    E.1.2Term Huntington's disease
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Main study: To assess the effect of pridopidine on functional capacity in participants with stage 1-2 HD
    OLE study:
    Efficacy: To evaluate the long-term treatment effect of pridopidine in participants with HD who previously completed the Main study
    Safety and Tolerability:
    To evaluate long-term safety and tolerability of pridopidine in participants with HD who previously completed the Main study
    E.2.2Secondary objectives of the trial
    Key Secondary
    • TFC responder analysis following pridopidine treatment
    Secondary
    • To evaluate the effect of pridopidine on motor function
    • To evaluate the effect of pridopidine on other measures of efficacy
    Safety and Tolerability:
    • To evaluate the safety and tolerability of pridopidine
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Main study:
    1. Twenty-five years of age (inclusive) and older, at the time of signing the informed consent.
    2. Diagnosis of HD based on clinical features and the presence of ≥36 CAG repeats in the HTT, confirmed by historical laboratory quantified results or by a diagnostic test at screening.
    3. Diagnostic confidence level (DCL) of 4 (unequivocal motor signs, ≥ 99% confidence) on the standardized motor exam UHDRS-TMS.
    4. Adult-onset HD with onset of signs and symptoms ≥18 years of age.
    5. Stage 1 or Stage 2 HD, defined as a UHDRS-TFC score of ≥7, at screening.
    6. UHDRS-Independence scale (IS) score ≤90% at screening.
    7. UHDRS-TMS ≥20 at the Screening visit.
    8. Must meet all criteria required to move forward with the Randomization Authorization Flow (RAF) and be considered eligible by the RAF Reviewer.
    9. Male or female.
    10. Female participants of childbearing potential must have a negative β-human chorionic gonadotropin (β-HCG) test at screening and baseline, be sterile, or be postmenopausal.
    11. Female participants of childbearing potential whose male partners are potentially fertile (i.e., no vasectomy) must use highly effective birth control methods stable for at least 3 months prior to screening, for the duration of the study and for 30 days after discontinuation of the study drug.
    12. Male participants must be sterile, or if they are potentially fertile/reproductively competent (not surgically [e.g., vasectomy] or congenitally sterile) and their female partners are of childbearing potential, they must use, together with their female partners, effective birth control methods for the duration of the study and for 90 days after study drug discontinuation.
    13. For participants taking allowed antipsychotic, antidepressant, or other psychotropic medication, the dosing of medication as listed in Section 10.6, must be stable for at least 4 weeks before the Baseline visit and throughout the study (unless clinically necessary to change).
    14. For participants taking allowed concomitant medications, dosing of medications must be stable for a t least 4 weeks prior to the Baseline
    visit (note: Amiodarone is not allowed within 6 weeks of Baseline visit)
    15. Capable of providing signed informed consent for the Main study which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.
    Open-label Extension:
    1. Completed the EoS visit of the Main study on treatment without important protocol deviations impacting efficacy and safety assessments.
    2.Capable of providing signed informed consent for the OLE.
    3.Must meet all criteria required to move forward with the OLE assessments.
    E.4Principal exclusion criteria
    Main study:
    1. Prolonged QTcF interval (defined as a QTcF interval of >450 ms for male and >470 ms for female) at screening.
    2. Clinically significant heart disease within 12 weeks before randomization, defined as follows:
    a. Participants with clinically significant heart disease, a clinically significant history of arrhythmia, symptomatic or uncontrolled atrial fibrillation despite treatment, or confirmed ventricular tachycardia, or presence of left bundle branch block.
    b. Participants with a known history of congenital long QT syndrome or a first degree relative with this condition.
    c. Clinically significant bradycardia, sick sinus syndrome, complete atrioventricular block, congestive heart failure, polymorphic ventricular tachycardia, clinically relevant hypocalcemia, hypokalemia or hypomagnesemia.
    3. History of epilepsy or seizures within the last 5 years.
    4. Serious medical illness (includes, but not limited to, uncontrolled hypertension; respiratory disease, including severe forms of asthma; severe hepatic disease (confirmed Hepatitis B virus [HBV], Hepatitis C virus [HCV];, confirmed human immunodeficiency virus [HIV]); renal disease; acquired immune deficiency syndrome; and unstable psychiatric or other neurologic disorders) and metastatic cancer. For serious kidney and and liver liver illnesses see also exclusion criterion 12 (laboratory test abnormalities)
    5. Known intracranial neoplasms, vascular malformations, history of cerebrovascular accident, or intracranial hemorrhage.
    6. Female participants who are pregnant, planning to become pregnant or breastfeeding
    7. Medications that prolong QT interval, taken within 4 weeks of the Baseline visit (note, Amiodarone is not allowed within 6 weeks of the Baseline visit) or at any timepoint during the study, including non-allowed antipsychotic medications, tricyclic antidepressants, and/or Class I antiarrhythmics
    8. Use of pridopidine within 12 months before the Baseline visit.
    9. Treatment with any investigational product within 6 weeks or 5 half-lives (whichever is longer) before the Screening visit or a plan to participate in another clinical study that assesses any investigational product during the study.
    10. Gene therapy at any time
    11. Prior participation in studies with tominersen at any time
    12.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.
    13. Have any of the following laboratory test abnormalities at screening
    a. CrCl <30 mL/min at screening, calculated using the CockcroftGault equation: (140–age) × mass (kg) × [0.85 if female] / 72 × serum creatinine (mg/dL).
    b. Aspartate aminotransferase (AST) ≥2.5 × upper limit of normal (ULN)
    c. Alanine aminotransferase (ALT) ≥2.5 × ULN
    d. Gamma- glutamyl transferase (GGT) ≥3.0 × ULN
    e. Total bilirubin >1,5 mg/dL, except participants with unconjugated hyperbilirubinemia without other liver function derangements or other
    explanations for the elevated bilirubin (consistent with diagnosis of Gilbert's syndrome)
    14. Alcohol and/or substance use disorder within the 6 months prior to screening, as defined by the Diagnostic and Statistical Manual–Fifth Edition (DSM-5) Text Revision criteria for substance use.
    15. Active suicidal ideation as measured by a most severe suicide ideation score of 4 (Active Suicidal Ideation with Some Intent to Act, without Specific Plan) or 5 (Active Suicidal Ideation with Specific Plan and Intent) on the C-SSRS if the ideation occurred within 1 year of Screening, or participants who answered “Yes” on any of the 5 C-SSRS Suicidal Behavior Items (actual attempt, interrupted attempt, aborted attempt, preparatory acts, or behavior), if the attempt or acts were performed within 1 year of screening, or participants who, in the opinion of the investigator, present a serious risk of suicide.
    16. Known allergy to any ingredient of the study drug (pridopidine, silicified microcrystalline cellulose, or magnesium stearate)
    17. Vulnerable participant (e.g., people kept in detention), or participant unfit to participate in a clinical study due to living circumstances, e.g. without sufficient family or social support, stable residence, sustainable financial and general healthcare and resources.
    18. An employee or a family member of an employee of the Sponsor, Investigator or investigator study site, or otherwise dependent on the Sponsor, the Investigator or the investigator study site.
    E.5 End points
    E.5.1Primary end point(s)
    Main study: Change from baseline to Week 65 in the UHDRS-TFC score
    OLE study:
    Efficacy: Change in UHDRS-TFC; UHDRS-TMS
    Safety and Tolerability:
    Incidence (count and rate) of AEs and SAEs overall, by severity, by relationship to study drug, and those that led to withdrawal from the study
    •Incidence and shifts of clinically significant abnormalities in ECG, laboratory tests and vital signs
    •Analysis of C-SSRS throughout the study
    •Tolerability:
    -The number (%) of participants who complete the OLE treatment period
    -The number (%) of participants who fail to complete the OLE treatment period due to AEs
    E.5.1.1Timepoint(s) of evaluation of this end point
    Main study: Baseline and week 65
    OLE study: according to the protocol
    E.5.2Secondary end point(s)
    Key Secondary
    1. Proportion of participants with no worsening (change ≥ 0 point) from baseline to Week 65 in UHDRS-TFC
    Secondary
    2. Change from baseline to Week 65 in the UHDRS-Total Motor Score (TMS)
    3. Change from baseline to Week 65 in Quantitative motor (Q-Motor) finger tapping (Digitomotography)
    4. Change from baseline to Week 65 in Composite UDHRS (cUHDRS) total score
    5. Change from baseline to Week 52 in UHDRS-TFC score
    6. Change from baseline to Week 52 in UHDRS-TMS score
    7. Proportion of participants with no worsening from baseline in Clinical Global Impression of Change (CGI-C) at Week 65
    8. Change from baseline to Week 78 in UHDRS-TFC score
    Safety and Tolerability
    • Incidence (count and rate) of adverse events (AEs) and serious AEs (SAEs) overall, by severity, by relationship to study drug, and those that led to withdrawal from the study
    • Incidence and shifts of clinically significant abnormalities in electrocardiogram (ECG), laboratory tests and vital signs
    • Analysis of Columbia Suicide Severity Rating Scale (C-SSRS) throughout the study
    • Tolerability:
    − The number (%) of participants who complete study the treatment period
    − The number (%) of participants who fail to complete the treatment period
    − The number (%) of participants who fail to complete the treatment period due to the prolongation of the Fridericia corrected QT interval (QTcF) compared to baseline and QT-change stopping rules
    E.5.2.1Timepoint(s) of evaluation of this end point
    According to the protocol
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic Yes
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group Yes
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA30
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    E.8.7Trial has a data monitoring committee Yes
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    The Main study will be considered completed when all participants either complete the study through Week 65 or discontinue earlier (ET visit).
    OLE: The OLE study will be considered completed 6 months after the last participant completes the double-blind Treatment period. OLE
    duration may be extended pending emerging data from the double-blind portion of the study. A substantial protocol amendment for the
    extension of the OLE will be submitted after analysis of the data from
    the Main study.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months3
    E.8.9.1In the Member State concerned days30
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months6
    E.8.9.2In all countries concerned by the trial days6
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 420
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 60
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state16
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 240
    F.4.2.2In the whole clinical trial 480
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    None
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    G.4.1Name of Organisation Huntington Study Group
    G.4.3.4Network Country United States
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2020-11-24
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-02-26
    P. End of Trial
    P.End of Trial StatusOngoing
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