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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   41225   clinical trials with a EudraCT protocol, of which   6755   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) .
     
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    Summary
    EudraCT Number:2015-002571-24
    Sponsor's Protocol Code Number:15/0118
    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:2019-02-12
    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 number2015-002571-24
    A.3Full title of the trial
    A Phase IIA Prospective, Single-Centre, Open Label Clinical Trial to Evaluate the Safety, Tolerability and Pharmacodynamic Effects of Ambroxol in Patients with Parkinson Disease: Ambroxol in Disease Modification in Parkinson Disease
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Pilot Study to Evaluate the Safety, Tolerability (any side effects of the drug) and Pharmacodynamic (biochemical and physiological effects of drugs). The effects of Ambroxol in Patients with Parkinson Disease: Ambroxol in Disease Modification in Parkinson Disease.
    A.3.2Name or abbreviated title of the trial where available
    AiM-PD
    A.4.1Sponsor's protocol code number15/0118
    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 SponsorJoint Research Office
    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 supportThe Cure Parkinson's Trust
    B.4.2CountryUnited Kingdom
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationLeonard Wolfson Experimental Neurology Centre Clinical Research Facility
    B.5.2Functional name of contact pointGayle D'Souza
    B.5.3 Address:
    B.5.3.1Street AddressUCL, Institute of Neurology & The National Hospital for Neurology and Neurosurgery
    B.5.3.2Town/ cityQueen Square, London
    B.5.3.3Post codeWC1N 3BG
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number02034484541
    B.5.5Fax number02034484547
    B.5.6E-mailg.d'souza@ucl.ac.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 Yes
    D.2.1.1.1Trade name AMBROSAN 60mg Tablets
    D.2.1.1.2Name of the Marketing Authorisation holderPRO.MED.CS Praha a.s., TelĨská 1, 140 00 Praha 4, Czech Republic
    D.2.1.2Country which granted the Marketing AuthorisationCzech Republic
    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 nameAmbroxol
    D.3.4Pharmaceutical form Tablet
    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 INNAmbroxol hydrochloride
    D.3.9.4EV Substance CodeAS1
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number60
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Parkinson disease
    E.1.1.1Medical condition in easily understood language
    Parkinson's disease affects the nerve cells in the brain that produce dopamine. Symptoms include muscle rigidity, tremors, and changes in speech and gait and there is no cure.
    E.1.1.2Therapeutic area Diseases [C] - Nervous System Diseases [C10]
    MedDRA Classification
    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 central nervous system (CNS), cerebrospinal fluid (CSF) penetration and binding to GCase of ambroxol by the parameters outline (modulation of GCase activity & ambroxol level) at 5 intra-participant dose escalations from day 1 to day 186 at 60 mg TID (day 1-7), 120 mg TID (day 8-14), 180 mg TID (day 15-21), 300 mg TID (day 22-28) and 420 mg TID (day 29-186).
    E.2.2Secondary objectives of the trial
    • To assess the safety and tolerability of the Glucocerebrosidase (GCase) modulating chaperone ambroxol in Parkinson disease participants with and without Gaucher gene (GBA) mutation at 5 intra-participant dose escalations from day 1 to 186.
    • To measure the pharmacodynamic effects of ambroxol on GCase activity in blood and CSF following ambroxol oral administration at 5 intra-participant dose escalations from day 1 to 186.
    • To quantify the effect of ambroxol on biomarkers of Parkinson and neurodegeneration at 5 intra-participant dose escalations from day 1 to 186.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Male or female;
    2. Age ≥ 40 and ≤ 80 years of age;
    3. Confirmed diagnosis of Parkinson disease at any time; and Hoehn and Yahr criteria, confirmed staged between I – III, inclusive;
    4. Able and willing to provide informed consent prior to any study related assessments and procedures at screening visit 1;
    5. Capable of complying with all study procedures, including fasting lumbar puncture;
    6. Willing to provide a blood sample for screening genomic for Parkinson Disease related DNA analysis and/or consent to Investigators obtaining and using participants previous DNA results if applicable;
    7. Willing and able to self-administer oral ambroxol medication, from day 1 to 186 (at 60 mg TID (day 1-7), 120 mg TID (day 8-14), 180 mg TID (day 15-21), 300 mg TID (day 22-28) and 420 mg TID (day 29-186));
    8. Able to travel to the participating study site;
    9. A female participant is eligible to participate if she is of:
    • Non-childbearing potential defined as pre-menopausal females with a
    documented tubal ligation or hysterectomy; or postmenopausal defined as 12
    consecutive months of spontaneous amenorrhea, at least 6 weeks post-surgical
    bilateral oophorectomy (with or without hysterectomy) or post tubal
    ligation. In questionable cases, menopausal status will be confirmed by
    demonstrating levels of follicle stimulating hormone (FSH) 25.8 – 134.8 IU/L
    and oestradiol < 201 pmol/l at entry.
    • Women of child-bearing potential must use accepted contraceptive methods
    (listed below), and must have a negative serum at screening visit 1 and
    urine pregnancy tests at subsequent visits if applicable. An additional
    pregnancy test will be performed, and results obtained, prior to
    administration of the first dose of ambroxol.

    Accepted contraception methods:
    • True abstinence: When this is in line with the preferred and usual lifestyle of the participant. [Periodic abstinence (e.g., calendar, ovulation, symptothermal, post-ovulation methods) and withdrawal are not acceptable methods of contraception).

    Contraceptive Methods with a Failure Rate of < 1%:
    • Oral contraceptive, either combined or progestogen alone;
    • Injectable progestogen;
    • Implants of levonorgestrel;
    • Estrogenic vaginal ring;
    • Percutaneous contraceptive patches; -
    • Intrauterine device (IUD) or intrauterine system (IUS) that meets the <1% failure rate as stated in the product label;
    Please note:
    - All male and female participants of child bearing potential must agree with their partners to use double-barrier birth control or abstinence while participating in the study and for 2 weeks following the last dose of the study drug.
    - Participants may continue to take PD medications including glutamate antagonists, anticholinergics, dopamine agonists, Levodopa (L-DOPA and decarboxylase (DDC) inhibitor), Monoamine oxidase B (MAO-B) inhibitors catechol-O-methyltransferase (COMT) inhibitors, beta blockers, selective serotonin uptake inhibitors (SSRIS), tricyclic antidepressants (TCAs) and indomethacin.

    E.4Principal exclusion criteria
    Participants are excluded from participating in this study if 1 or more of the following criteria are met:
    1. Current treatment with anticoagulants (e.g. warfarin) that might preclude safe completion of the lumbar puncture and in the opinion of the Investigator;
    2. Current use of investigational medicinal product or participation in another interventional clinical trial or who have done so within 30 days prior to the first dose in the current study;
    3. Exposure to more than three investigational medicinal products within 12 months prior to the first dose in the current study;
    4. Confirmed dysphagia that would preclude self-administration of ambroxol up to 7 tablets TID for the duration of day 1 to day 186);
    5. Significant known lower spinal malformations or other spinal abnormalities that would preclude lumbar puncture;
    6. History of known sensitivity to the study medication,ambroxol or its excipients (lactose monohydrate, granulated microcrystalline cellulose, copovidone and magnesium stearate) in the opinion of the investigator that contraindicates their participation;
    7. History of known rare hereditary disorders of galactose intolerance, Lapp lactase deficiency or glucose-galactose malabsorption;
    8. Evidence or history of hypersensitivity to lidocaine or its derivatives;
    9. History of drug abuse or alcoholism in the opinion of the Investigator that would preclude participation in the study;
    10. Donation of blood (one unit or 350 ml) within three months prior to receiving the first dose of the study drug;
    11. Pregnant or breastfeeding;
    12. All participants of child bearing potential in the opinion of the Investigator that would preclude participation in the study and who do not agree to use double-barrier birth control or abstinence while participating in the study and for two weeks following the last dose of study drug;
    13. Any clinically significant or unstable medical or surgical condition that in the opinion of the PI or PI-delegated clinician may put the participant at risk when participating in the study or may influence the results of the study or affect the participant’s ability to take part in the study, as determined by medical history, physical examinations, electrocardiogram (ECG), or laboratory tests. Such conditions may include:
    a) Impaired renal function
    b) Moderate/Severe hepatic impairment
    c) A major cardiovascular event (e.g. myocardial infarction, acute coronary syndrome, decompensated congestive heart failure, pulmonary embolism, coronary revascularisation that occurred within 6 months prior to the screening visit.
    E.5 End points
    E.5.1Primary end point(s)
    To assess the central nervous system (CNS), cerebrospinal fluid (CSF) penetration by the parameters outlines (GCase activity, ambroxol level) from day 1 to day 186.
    E.5.1.1Timepoint(s) of evaluation of this end point
    The end point is at Month 6
    E.5.2Secondary end point(s)
    CSF biomarkers of Parkinson disease and neurodegeneration
    CSF ambroxol levels
    Safety data, i.e.:
    - Adverse events
    - Laboratory safety data
    - Physical and neurological examinations
    - ECG
    E.5.2.1Timepoint(s) of evaluation of this end point
    The end point is at Month 6
    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 No
    E.6.4Safety Yes
    E.6.5Efficacy No
    E.6.6Pharmacokinetic No
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic Yes
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.6.13.1Other scope of the trial description
    A single-centre, interventional, open label clinical trial to assess the safety, tolerability and ph
    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 No
    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 Yes
    E.8.1.7.1Other trial design description
    A proof of concept clinical trial
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    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.8.4.1Number of sites anticipated in Member State concerned1
    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
    Last participant last visit (LPLV) is visit 7, day 279 but if the participant consents to the optional LP at visit 7, day 279 they will receive a telephone call (telephone visit 12) to record any AEs within 5 days of visit 7.
    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 months0
    E.8.9.1In the Member State concerned days1
    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 days3
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    F.1.1Number of subjects for this age range: 0
    F.1.1.1In Utero No
    F.1.1.1.1Number of subjects for this age range: 0
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.2.1Number of subjects for this age range: 0
    F.1.1.3Newborns (0-27 days) No
    F.1.1.3.1Number of subjects for this age range: 0
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.4.1Number of subjects for this age range: 0
    F.1.1.5Children (2-11years) No
    F.1.1.5.1Number of subjects for this age range: 0
    F.1.1.6Adolescents (12-17 years) No
    F.1.1.6.1Number of subjects for this age range: 0
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 10
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 10
    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.3.3.7.1Details of other specific vulnerable populations
    Male partners using effective double barrier contraception with their partners of child bearing pote
    F.4 Planned number of subjects to be included
    F.4.1In the member state20
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 20
    F.4.2.2In the whole clinical trial 20
    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)
    Participants will have continued and ongoing standard of care appointments with their treating Neurologist and their GP and participants will not have access to the study drug after the study has ended.
    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 Decision2016-07-27
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2016-10-14
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2018-04-26
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