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The European Union Clinical Trials Register allows you to search for protocol and results information on:
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    The EU Clinical Trials Register currently displays   41018   clinical trials with a EudraCT protocol, of which   6709   are clinical trials conducted with subjects less than 18 years old.
    The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

    Phase 1 trials conducted solely in adults and that are not part of an agreed PIP are not public in the EU CTR (refer to European Guidance 2008/C 168/02   Art. 3 par. 2 and   Commission Guideline 2012/C 302/03,   Art. 5) .
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
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    EudraCT Number:2007-006721-27
    Sponsor's Protocol Code Number:CERE-120-06
    National Competent Authority:Austria - BASG
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2008-09-15
    Trial results
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    A. Protocol Information
    A.1Member State ConcernedAustria - BASG
    A.2EudraCT number2007-006721-27
    A.3Full title of the trial
    A Phase II, Multicenter, Randomized and Controlled Open-Label Trial Comparing the Safety and Efficacy of Bilateral Intraputaminal (IPu) Administration of CERE-120 (Adeno-Associated Virus Serotype 2 [AAV2]-Neurturin [NTN]) Combined with Best Medical Therapy (BMT) versus BMT-alone in Subjects With Idiopathic Parkinson’s Disease.
    A.4.1Sponsor's protocol code numberCERE-120-06
    A.7Trial is part of a Paediatric Investigation Plan Information not present in EudraCT
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorCeregene 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 support
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisation
    B.5.2Functional name of contact point
    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 nameCERE-120
    D.3.2Product code CERE-120
    D.3.4Pharmaceutical form Suspension for injection
    D.3.4.1Specific paediatric formulation Information not present in EudraCT
    D.3.7Routes of administration for this IMPIntracerebral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.9.2Current sponsor codeCERE-120
    D.3.10 Strength
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number6.7E+12 vg/ml
    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) Information not present in EudraCT
    D. cell therapy medicinal product No
    D. therapy medical product Yes
    D. Engineered Product Information not present in EudraCT
    D. ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D. on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy Information not present in EudraCT
    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 Information not present in EudraCT
    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
    idiopathic Parkinson's Disease
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 9.1
    E.1.2Level LLT
    E.1.2Classification code 10061536
    E.1.2Term Parkinson's disease
    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 safety and efficacy of bilateral IPu CERE-120 (AAV2-NTN) administration with an optimized and stable regimen of antiparkinsonian medication, “Best Medical Therapy” (BMT), in subjects with idiopathic Parkinson's Disease.
    E.2.2Secondary objectives of the trial
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Males and females ages 35 – 75 years old. Females must be either surgically sterile or 2-years post-menopausal and have a negative serum HCG pregnancy test at Screening and Baseline. All patients must agree to practice adequate barrier method contraception for at least six months after CERE-120 administration;
    2. Diagnosis of bilateral, idiopathic PD based on UK Brain Bank criteria with motor complications despite adequate antiparkinsonian therapy. This will be based on medical history and neurologic examination (the presence of at least 2 of the following: resting tremor, rigidity, or bradykinesia);
    3. At least 5 years disease duration since diagnosis of PD;
    4. A robust response to dopaminergic therapy, i.e., approximately a 50% reduction of UPDRS Part III Motor score in the 12-hrs practically defined “off” condition when the subject receives an L-Dopa equivalent of their standard morning dose of antiparkinsonian therapy;
    5. A Hoehn and Yahr “on” score of 3 or less and a score of 1 or 0 on item #30 of the UPDRS Part III: Motor Examination in the “on” condition at Screening;
    6. Each subject must also have:
    • A score of ≥30 on the UPDRS Part III: Motor Examination in the practically defined “off” condition at the Screening Visit;
    • A score of ≤ 25 on the UPDRS Part III: Motor Examination in the “on” condition at the Screening visit;
    7. Stable, optimized (BMT) regimen of antiparkinsonian medications and stable parkinsonian features, for the 3-week period preceding the Baseline Visit;
    8. Clinically significant motor fluctuations with an average of at least 2 hours of “off” time per day (based on reported history at Screening), despite adequate antiparkinsonian therapy;
    9. Subject is willing to not undergo DBS during the course of the study and the investigator believes this is medically acceptable;
    10. Subject is willing to not receive treatment with apomorphine IV infusions, routine administration of apomorphine injections or Duodopa® during the course of the study and the investigator believes this is medically acceptable. (Apomorphine injection may given as rescue therapy if the investigator feels this is required);
    11. The subject has a score of 130 or greater on the Mattis Dementia Scale at Screening;
    12. The subject has no evidence of Major Depression as evidenced by a score of 0-19 on the MADRS at Baseline;
    13. Normal coagulation tests and normal platelet levels;
    14. The subject is expected to be able to understand and comply with the required visit schedule and all required tests and procedures;
    15. The subject is physically and mentally capable of performing the necessary protocol-specified assessments;
    16. The subject is medically able to undergo the surgery required to complete the surgical procedure as determined by medical and surgical history, clinical and laboratory evaluations and any other evaluations that are part of the standard practice at the institution in which the subject is to undergo surgery;
    17. Signed informed consent is obtained before any study-specific procedure, including assessments required during the Eligibility Evaluation Period.
    E.4Principal exclusion criteria
    1. Subjects with atypical or secondary parkinsonism;
    2. Any subject for whom, in the judgment of the principal investigator, participation in the study would pose a safety risk, including but not limited to:
    • history of significant drug–induced hallucinations (within the last twelve months from proposed surgical procedure), psychosis, schizophrenia, or any recent major affective disorder;
    • treatment with neuroleptics for psychosis, schizophrenia, or any recent major affective disorder within 1 year before the anticipated surgical procedure;
    • history, within two years before the anticipated surgical procedure, of drug or alcohol abuse;
    • significant cardiovascular risks or history of cardiovascular disorder, unless the subject is cleared after careful medical evaluation;
    • any disorder that precludes a surgical procedure or alters wound healing (e.g., signs of sepsis or inadequately treated infection);
    • any clinically significant physical or laboratory finding;
    • any concomitant serious medical illness that might pose a safety risk;
    3. Evidence of significant brain atrophy per Screening MRI;
    4. Presence of any known brain abnormality that may interfere with the assessments of safety or efficacy or would, in the judgment of the investigator, represent a surgical risk to the subject;
    5. Any medical disability (e.g., severe degenerative arthritis, compromised nutritional state, peripheral neuropathy) or any clinical evidence of cognitive impairment that would interfere with the assessment of safety and efficacy in this trial or would compromise the ability of the subject to undergo study procedures (e.g., MRI), or to give informed consent;
    6. History of treatment of PD by any procedure involving intracranial surgery or implantation of a device;
    7. Receipt of antiplatelet agents or anticoagulation therapy for at least 10 days prior to surgical procedure;
    8. Chemotherapy, cytotoxic therapy, or immunotherapy (e.g., IL-2, IL-12, interferon) within 6 weeks prior to the surgical procedure;
    9. Vaccinations within 30 days prior to the surgical procedure. (Also no vaccinations within 30 days after their surgical procedure, unless deemed necessary by the investigator for the subject’s safety);
    10. History of prior gene transfer therapy;
    11. Treatment with an investigational agent within 60 days before the anticipated surgical procedure;
    12. Subjects who are not able to travel alone according to their own judgment or to the investigator’s opinion, should be accompanied by a partner or caregiver during traveling to the study visits;
    13. Subjects who will be unavailable for the duration of the trial, likely to be noncompliant with the protocol, or who are felt to be unsuitable by the Investigator for any other reason.
    E.5 End points
    E.5.1Primary end point(s)
    To compare the ability of CERE-120 plus BMT versus BMT-only to reduce the severity of motor symptoms as assessed by the change from baseline in the Unified Parkinson’s Disease Rating Scale (UPDRS) part III (Motor Score) in the practically defined “off” condition, after 12 months of observation.
    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. 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 Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    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. description
    best medical treatment for parkinson's disease only
    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.8.4.1Number of sites anticipated in Member State concerned1
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA12
    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 Information not present in EudraCT
    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
    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 months2
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months2
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    F.1.1.1In Utero Information not present in EudraCT
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) Information not present in EudraCT
    F.1.1.3Newborns (0-27 days) Information not present in EudraCT
    F.1.1.4Infants and toddlers (28 days-23 months) Information not present in EudraCT
    F.1.1.5Children (2-11years) Information not present in EudraCT
    F.1.1.6Adolescents (12-17 years) Information not present in EudraCT
    F.1.2Adults (18-64 years) Yes
    F.1.3Elderly (>=65 years) Yes
    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 No
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception No
    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 state7
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 50
    F.4.2.2In the whole clinical trial 50
    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)
    Patients treated with CERE-120 will be asked to enter a long-term follow-up trial. Those who do not want to participate in the LTFU trial, will be followed up to obtain annual safety data via a Sponsor designee. Patients in the BMT group have the possibility to receive CERE-120 after completing the CERE-120-06 trial, provided that CERE-120 benefit/risk profile remains positive. Both the long term follow-up as well as the CERE-120 treatment for the BMT group will be done under separate protocols.
    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 Decision2008-10-20
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2008-10-20
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    P.Date of the global end of the trial2008-12-03
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