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    Summary
    EudraCT Number:2017-004685-10
    Sponsor's Protocol Code Number:PD-1105
    National Competent Authority:Poland - Office for Medicinal Products
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2018-09-26
    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 ConcernedPoland - Office for Medicinal Products
    A.2EudraCT number2017-004685-10
    A.3Full title of the trial
    A Randomized, Placebo Surgery Controlled, Double-blinded, Multi-center, Phase 2 Clinical Trial, Evaluating the Efficacy and Safety of VY-AADC02 in Advanced Parkinson's Disease With Motor Fluctuations
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    VY-AADC02 for Parkinson's Disease With Motor Fluctuations
    A.4.1Sponsor's protocol code numberPD-1105
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT03562494
    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 SponsorVoyager Therapeutics, Inc.
    B.1.3.4CountryUnited States
    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 supportVoyager Therapeutics, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationVoyager Therapeutics, Inc.
    B.5.2Functional name of contact pointclinicaltrials@vygr.com
    B.5.3 Address:
    B.5.3.1Street Address75 Sidney Street
    B.5.3.2Town/ cityCambridge
    B.5.3.3Post code02139
    B.5.3.4CountryUnited States
    B.5.6E-mailclinicaltrials@vygr.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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameVY-AADC02
    D.3.2Product code VY-AADC02
    D.3.4Pharmaceutical form Solution for injection/infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntracerebral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNPending
    D.3.9.1CAS number 2226647-27-2
    D.3.9.2Current sponsor codeVY-AADC02
    D.3.9.3Other descriptive nameVY-AADC02
    D.3.10 Strength
    D.3.10.1Concentration unit vector genomes (vg)/mL
    D.3.10.2Concentration typeup to
    D.3.10.3Concentration number1.0 x 10e12
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    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) Yes
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product Yes
    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 Yes
    D.3.11.3.5.1CAT classification and reference numberGene Therapy EMA/679228/2016
    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 Yes
    D.3.11.10Medicinal product containing genetically modified organisms Yes
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Patients with Parkinson's Disease with motor fluctuations.
    E.1.1.1Medical condition in easily understood language
    Parkinson's disease
    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 10061536
    E.1.2Term Parkinson's disease
    E.1.2System Organ Class 10029205 - Nervous system disorders
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To assess the distribution, efficacy, and safety of VY-AADC02 in Patients with Parkinson's Disease with Motor Fluctuations.
    E.2.2Secondary objectives of the trial
    Not applicable.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1.- Males and females, 40 to 75 years of age (inclusive).
    2.- Diagnosis of PD, consistent with United Kingdom Brain Bank Criteria.
    3.- Unequivocal responsiveness to dopaminergic therapy, as judged by the Investigator.
    4.- Disease duration from diagnosis of ≥4 years.
    5.- In the judgment of the Investigator, a stable, optimal regimen of Parkinson's medications for at least 4 weeks prior to screening evaluation.
    6.- Stable cognitive and psychological function based on screening evaluations.
    7.- In the judgment of the Investigator, stable Parkinson's features and symptoms for at least 4 weeks prior to screening evaluation.
    8.- Agrees to defer any neurological surgery, including deep brain stimulation, other invasive treatments for PD including duodopa, or the addition of new dopaminergic formulations until after completing the 12-month study visit.
    9.- Ability to travel to study visits.
    E.4Principal exclusion criteria
    1.- Atypical or secondary parkinsonism, including but not limited to symptoms believed to be due to trauma, brain tumor, infection, cerebrovascular disease, other neurological disease, or to drugs, chemicals, or toxins, as determined by the Investigator.
    2.- MoCA score <26.
    3.- Use of tetrahydrocannabinol within 6 months of screening evaluation.
    4.- Brain imaging abnormalities in the striatum or other regions that would substantially increase risk of surgery.
    5.- Contraindication to MRI and/or gadolinium-based contrast agents.
    6.- Prior brain surgery or infusion therapies that could complicate the study procedure or negatively impact study evaluations as determined from participant interview, screening MRI, or medical records.
    7.- History of malignancy other than treated carcinoma in situ within 3 years of screening evaluation.
    8.- Prior gene transfer, current treatment with any investigational agent (drug or device) within 2 months of screening evaluation, or participation or plans to participate in another research study.
    9.- Ongoing treatments or planned treatments that might interfere with interpretation of the study outcome including deep brain stimulation.
    E.5 End points
    E.5.1Primary end point(s)
    1.Change in Patient Rated Motor Fluctuations in the VY-AADC02 group compared to placebo surgery group.

    2.Percent coverage within the putamen at time of administration of VY-AADC02.

    3.Change in AADC enzyme activity (Distribution).

    4.Safety of VY-AADC02 as measured by number of treatment emergent adverse events (TEAEs) and Serious Adverse Events (SAEs).

    5.Safety of VY-AADC02 as measured by changes in vital signs.

    6.Safety of VY-AADC02 as measured by physical examinations and routine clinical laboratory analysis (hematology and clinical chemistry).

    7.Safety of VY-AADC02 as measured by changes in findings on brain images.

    8.Safety of VY-AADC02 as measured by the Columbia-Suicide Severity Rating Scale (C-SSRS).

    9.Safety of VY-AADC02 based on change in impulse control disorders.
    E.5.1.1Timepoint(s) of evaluation of this end point
    1.- Change in Patient Rated Motor Fluctuations in the VY-AADC02 group compared to placebo surgery group will be assessed from Baseline to 12 months post op.

    2.- Percent coverage within the putamen at time of administration of VY-AADC02 will be determined on the Day of Surgery.

    3.- Change in AADC enzyme activity (Distribution) will be evaluated on study 45 and at 12 months post op.

    4.Safety assessments will be measured from time of Informed Consent to 12 months post op + 30 day Follow Up.
    E.5.2Secondary end point(s)
    1.- Change in activities of daily living in the VY-AADC02 group compared to placebo surgery group.
    2.- Change in PD related quality of life in the VY-AADC02 group compared to placebo surgery group.
    3.- Change from baseline in time course response to levodopa in the VY-AADC02 group compared to placebo surgery group measured by the area under the curve (AUC) of repeated UPDRS III scores following a single dose of oral levodopa.
    4.- Change in global function in the VY-AADC02 group compared to placebo surgery group.
    5.- Change in overall non-motor symptoms in the VY-AADC02 group compared to placebo surgery group.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Baseline to 12 months post-op.
    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 No
    E.6.12Pharmacoeconomic Yes
    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) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    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 No
    E.8.2.3Other Yes
    E.8.2.3.1Comparator description
    Placebo (Sham) Partial burr/twist hole without dura penetration
    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 concerned3
    E.8.5The trial involves multiple Member States No
    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
    United States
    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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months5
    E.8.9.1In the Member State concerned days10
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months11
    E.8.9.2In all countries concerned by the trial days12
    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: 38
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 4
    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 state8
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 8
    F.4.2.2In the whole clinical trial 42
    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)
    After study completion patients will be invited to enroll in a long-term follow-up study, pending they meet the inclusion criteria, where they will be followed for up to an additional 8 years. Placebo-(sham) participants may be offered VY-AADC02 as part of a separate protocol if it is determined to be well tolerated, offers potential benefit, and participants meet the new protocol eligibility criteria.
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2019-01-11
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-09-12
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
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