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    The EU Clinical Trials Register currently displays   42312   clinical trials with a EudraCT protocol, of which   6968   are clinical trials conducted with subjects less than 18 years old.
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    Summary
    EudraCT Number:2013-001881-40
    Sponsor's Protocol Code Number:2797
    National Competent Authority:UK - MHRA
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2013-08-05
    Trial results View 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 ConcernedUK - MHRA
    A.2EudraCT number2013-001881-40
    A.3Full title of the trial
    An Extension Study to Assess the Safety and Efficacy of Intermittent Bilateral Intraputamenal Glial Cell Line-Derived Neurotrophic Factor (GDNF) Infusions Administered via onvection Enhanced Delivery (CED) in Subjects with Parkinson’s Disease
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Extension study to Assess the Benefit and Safety of Administering Intermittent GDNF Infusions in Parkinson's Disease (PD)
    A.3.2Name or abbreviated title of the trial where available
    Intermittent Bilateral GDNF for Parkinson’s Disease
    A.4.1Sponsor's protocol code number2797
    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 SponsorNorth Bristol NHS Trust (NBT)
    B.1.3.4CountryUnited Kingdom
    B.3.1 and B.3.2Status of the sponsorNon-Commercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportParkinson's UK
    B.4.2CountryUnited Kingdom
    B.4.1Name of organisation providing supportThe Cure Parkinson's Trust
    B.4.2CountryUnited Kingdom
    B.4.1Name of organisation providing supportMedGenesis Therapeutix Inc.
    B.4.2CountryCanada
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationNorth Bristol NHS Trust (NBT)
    B.5.2Functional name of contact pointClinical Trials Manager Helen Lewis
    B.5.3 Address:
    B.5.3.1Street AddressResearch & Innovation, Southmead Hospital
    B.5.3.2Town/ cityBristol
    B.5.3.3Post codeBS10 5NB
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number+441173238602
    B.5.5Fax number+441173236192
    B.5.6E-mailresearch@nbt.nhs.uk
    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 nameGlial Cell Line-Derived Neurotrophic Factor (GDNF)
    D.3.4Pharmaceutical form Concentrate for solution for 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 INNLiatermin
    D.3.9.3Other descriptive namer-metHuGDNF
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number9.0 to 11.0
    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) Yes
    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 Yes
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Parkinson's disease
    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 18.1
    E.1.2Level LLT
    E.1.2Classification code 10034008
    E.1.2Term Parkinson's syndrome
    E.1.2System Organ Class 100000004852
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To compare the effects of intermittent bilateral intraputamenal GDNF infusions on OFF-state motor function after 18 months of treatment with the effects after 9 months of treatment in subjects who completed in Study 2553.
    E.2.2Secondary objectives of the trial
    To compare the effects of intermittent bilateral intraputamenal GDNF infusions on ON-state motor function, motor complications, & ON- & OFF-state activities of daily living after 18m treatment with the effects after 9m treatment in subjects who completed Study 2553.

    • To assess the safety of intermittent bilateral intraputamenal GDNF infusions at 18m in subjects who received GDNF or placebo for 9m in Study 2553.

    • To explore the effects of intermittent bilateral intraputamenal GDNF infusions on other motor & non-motor functions, quality of life assessments, & imaging endpoints at 18 months in subjects who completed Study 2553.

    • To compare the results for various motor outcomes between the subjects who started GDNF early (i.e. were randomized to GDNF in Study 2553) & those who started GDNF late (i.e. were randomized to placebo in Study 2553).

    • Pilot Extension: To generate long-term safety data & provide continued access to GDNF until results of Study 2553 are available
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    In order to qualify for entry into the study, subjects MUST meet all of the following criteria:
    1. Enrolled and completed treatment in the Pilot or Primary Study Stages of Study 2553.
    2. Females of childbearing potential must have a negative pregnancy test at study entry and be willing to use an approved (by the PI or designee) form of contraception until the end of the study.
    3. Males with female partners of childbearing potential must be willing to use condoms for contraception until the end of the study.
    4. Provision of informed consent.
    E.4Principal exclusion criteria
    Subjects who meet any of the following criteria will NOT be eligible for inclusion in the study:
    1. Discontinued treatment early in Study 2553.
    2. Had any significant (in the opinion of the PI or designee) protocol deviation in Study 2553; this includes receipt of any disallowed anti-parkinsonian treatment or any investigational treatment.
    3. Presence of clinically significant (in the opinion of the PI) depression.
    4. MoCA score < 24 at the final assessment in Study 2553.
    5. Any new medical condition which might impair outcome measure assessments or safety measures including ability to undergo MRI scanning.
    E.5 End points
    E.5.1Primary end point(s)
    The primary endpoint of the study is the percentage change from baseline to the end of treatment in the practically defined OFF-state UPDRS motor score (part III).
    E.5.1.1Timepoint(s) of evaluation of this end point
    9 months of treatment in extension study (18 months treatment overall including treatment in study 2553)

    E.5.2Secondary end point(s)
    • Percentage change from baseline to the end of treatment in UPDRS motor score (part III) in the ON-state (following a levodopa challenge)
    • Percentage change from baseline to the end of treatment in UPDRS ADL (part II) .
    • Change from baseline to the end of treatment in PD diary ratings; i.e., total OFFtime per day, total good quality ON-time (ON without dyskinesias or ON with non-troublesome dyskinesias) and ON-time with troublesome dyskinesias
    E.5.2.1Timepoint(s) of evaluation of this end point
    9 months of treatment in extension study (18 months treatment overall including treatment in study 2553)

    Pilot subjects only will be eligible for up to an additional 80 weeks of Treatment (Pilot Extension).
    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 No
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    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) 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 No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    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
    All patients previously completed study 2553
    E.8.2.4Number of treatment arms in the trial1
    E.8.3 The trial involves single site in the Member State concerned Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    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 No
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.7Trial has a data monitoring committee No
    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 years3
    E.8.9.1In the Member State concerned months6
    E.8.9.1In the Member State concerned days0
    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: 30
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 12
    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 state42
    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)
    Subjects who complete the 9-month treatment period in study 2553 will be offered the opportunity to enroll in this open-label active treatment extension study
    The continuation of GDNF treatment beyond the planned extension study can be in no way guaranteed and may well stop even if the subjects have received great benefit.
    Pilot subjects only will be eligible for Treatment until December 2016(Pilot Extension).
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2013-09-16
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2013-08-09
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2017-02-15
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