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    Summary
    EudraCT Number:2013-000107-17
    Sponsor's Protocol Code Number:EAT2TREAT
    National Competent Authority:Germany - BfArM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2013-04-05
    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 ConcernedGermany - BfArM
    A.2EudraCT number2013-000107-17
    A.3Full title of the trial
    Pharmacological therapy of episodic ataxia type 2: a placebo-controlled comparison of the efficacy of 4-aminopyridine sustained-release (Fampyra TM) and acetazolamide (Acemit TM)
    Pharmakologische Therapie der Episodischen Ataxie Typ 2: Placebokontrollierter Vergleich der Wirksamkeit von retardiertem 4-Aminopyridin (Fampyra®) und Acetazolamid (Acemit®)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Comparsion of the therapy with Fampyra and Acemit in patients with episodic ataxia type 2
    A.3.2Name or abbreviated title of the trial where available
    4-aminopyridine sustained-release (Fampyra TM) and acetazolamide (Acemit TM) in patients with EA2
    4-Aminopyridin-R (Fampyra®) und Acetazolamid (Acemit®) bei Episodischer Ataxie Typ 2
    A.4.1Sponsor's protocol code numberEAT2TREAT
    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 SponsorHospital of the University of Munich, Grosshadern
    B.1.3.4CountryGermany
    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 supportBundesministerium für Bildung und Forschung (BMBF)
    B.4.2CountryGermany
    B.4.1Name of organisation providing supportBiogen Idec International GmbH
    B.4.2CountrySwitzerland
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationHospital of the University of Munich
    B.5.2Functional name of contact pointDSGZ
    B.5.3 Address:
    B.5.3.1Street AddressMarchioninistr. 15
    B.5.3.2Town/ cityMunich
    B.5.3.3Post code81377
    B.5.3.4CountryGermany
    B.5.4Telephone number00498970956987
    B.5.5Fax number00498970958795
    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 Fampyra TM
    D.2.1.1.2Name of the Marketing Authorisation holderBiogen Idec Ltd.
    D.2.1.2Country which granted the Marketing AuthorisationGermany
    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.3.4Pharmaceutical form Prolonged-release 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 INNFAMPRIDINE
    D.3.9.1CAS number 504-24-5
    D.3.9.4EV Substance CodeSUB07505MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number10
    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.IMP: 2
    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 Acemit TM
    D.2.1.1.2Name of the Marketing Authorisation holdermedphano Arzneimittel GmbH
    D.2.1.2Country which granted the Marketing AuthorisationGermany
    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.3.4Pharmaceutical 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 INNACETAZOLAMIDE
    D.3.9.1CAS number 59-66-5
    D.3.9.4EV Substance CodeSUB05219MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number250
    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 placeboFilm-coated tablet
    D.8.4Route of administration of the placeboOral use
    D.8 Placebo: 2
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboTablet
    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
    Familial episodic ataxia (EA) represents a genetically and phenotypically diverse group of rare autosomal dominant hereditary disorders characterized by episodes of imbalance and incoordination that are variably associated with interictal neurological signs and typically triggered by physical exertion and emotional stress. Episodic ataxia type 2 (EA2) with associated interictal nystagmus and other ocular motor abnormalities is the most common and the best characterized of all EA syndromes.
    E.1.1.1Medical condition in easily understood language
    Episodic ataxia type 2 is a hereditary disorder characterized by attacks with imbalance, vertigo and abnormal eye movements lasting up to days.
    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 10067447
    E.1.2Term Episodic ataxia
    E.1.2System Organ Class 100000004850
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Primary objective:
    1) Analysis of the efficacy of the two active IMPs (4-aminopyridine sustained-released, acetazolamide) regarding reduction of the frequency of attacks compared to placebo
    2) Quantifying the differences between the two IMPs (4-aminopyridine sustained-released, acetazolamide) regarding reduction of the frequency of attacks
    E.2.2Secondary objectives of the trial
    Secondary objective:
    1) Quantitative description and comparison of the median duration and intensity of attacks under 4-aminopyridine sustained-released vs. acetazolamide vs. placebo measured within the last 30 days of a 12-week treatment phase
    2) Quantitative description and comparison of the gait variability at maximum walking speed at the end of a 12-week treatment phase under 4-aminopyridine sustained-released vs. acetazolamide vs. placebo
    3) Analysis of the absolute change of the SARA scores and quality of life scores in the 3 treatment groups compared to the baseline score of each phase
    4) Comparison of the frequency of (S)AEs under 4-aminopyridine sustained-released vs. acetazolamide vs. placebo
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Subjects will only be randomized in the study if they meet all of the following criteria:

    1. Written informed consent of the subject
    2. Patients (male or female) aged 18 years or older
    3. Genetically confirmed episodic ataxia type 2 or a familial history of ataxia with clinical picture of episodic ataxia type 2 starting in childhood
    4. Subjects, with the ability to follow study instructions and likely to attend and complete all required visits (Compliance)
    E.4Principal exclusion criteria
    Subjects will not be randomized in the study if any of the following criteria applies:

    1. Weight of 40 kg or less
    2. Pregnancy or breast-feeding
    3. Concurrend treatment with inhibitors of organic cation transporter 2 (OCT2), e.g. cimetidine
    4. Known hypersensitivity to aminopyridine and/or acetazolamide/sulfonamide
    5. Cardiovascular diseases e.g. recent heart attack (within the last 3 months), cardiac arrhythmia (QTc interval > 500 ms, atrial fibrillation, AV block grade ≥ II), unstable angina pectoris, severe heart failure (NYHA class IV), severe arterial hypertension (grade III according to the guidelines of the German society of cardiology, 2008)
    6. Recently occurred stroke (within the last 3 months)
    7. Epileptic seizure currently or in the past
    8. Asthma (severity ≥ grade III)
    9. Obstructive lung disease (e.g. pulmonary emphysema)
    10. Liver insufficiency defined as GOT/GPT/total bilirubin > 3 x upper range
    (e.g. cirrhosis of the liver)
    11. Milde or severe renal failure (Creatinine Clearance ≤ 80ml/min)
    12. Addison's disease
    13. Unadjusted thyroid dysfunction
    14. Acute gastric and intestinal ulcer
    15. Known hyperchloremic acidosis
    16. Depressed sodium and/or potassium blood serum levels
    17. Chronic noncongestive glaucoma
    18. Hypercalcaemia
    19. Articular gout
    20. Known African anaemia
    21. Diabetes mellitus type I/II
    22. Other acute, serious illness of the subject
    23. Subject is unable to understand the scope, significance and consequences of this clinical trial and is unable to comply with the study design
    24. Previous participation in this clinical trial or participation in any clinical trial taking an investigational medicinal product within 30 days prior to written informed consent for this clinical trial
    E.5 End points
    E.5.1Primary end point(s)
    Primary endpoint:
    number of attacks within the last 30 days of each 12-week treatment phase
    E.5.1.1Timepoint(s) of evaluation of this end point
    12 weeks after treatment with IMP (for each treatment phase)
    E.5.2Secondary end point(s)
    Secondary endpoint:
    1) Median duration and intensity of the attacks within the last 30 days of each 12-week treatment
    phase
    2) Change (relative change, log scale) of the gait variability (CV) at maximum walking speed measured at the end of a 12-week treatment phase (Visit 3, 6 and 9) as well as 4 weeks after taking the last IMP (follow-up visit) compared to the beginning of a 12-week tratment phase (Visit 1, 4 and 7)
    3) Absolute change of VDADL and EQ-5D-5L (quality of life scores) measured at the end of a 12-week treatment phase (Visit 3, 6 and 9) as well as 4 weeks after taking the last IMP (follow-up visit) compared to the beginning of a12-week treatment phase (Visit 1, 4 and 7)
    4) Absolute change of SARA scores measured at the end of a 12-week treatment phase (Visit 3, 6 and 9) as well as 4 weeks after taking the last IMP (follow-up visit) compared to the beginning of the respective 12-week treatment phase (Visit 1, 4 and 7)
    E.5.2.1Timepoint(s) of evaluation of this end point
    Regrading end point 1:
    At the beginning and 12 weeks after treatment with IMP (for each treatment phase)
    Regarding end point 2, 3 and 4:
    At the beginning and 12 weeks after treatment with IMP (for each treatment phase) as well as 4 weeks after taking the last IMP (follow-up)
    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) 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 No
    E.8.1.6Cross over Yes
    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 trial6
    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 months0
    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.1.3Elderly (>=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 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 state30
    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)
    4 weeks after last administration of IMP, there will be a follow-up visit.
    After the clinical trial, patients will have the opportunity to make regular appointments and to get medical advice in the german center of vertigo and balance disorders.
    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 Decision2013-05-22
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2013-05-15
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2016-10-06
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