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    EudraCT Number:2015-000460-34
    Sponsor's Protocol Code Number:ALCAT
    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:2015-10-12
    Trial results View results
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    A. Protocol Information
    A.1Member State ConcernedGermany - BfArM
    A.2EudraCT number2015-000460-34
    A.3Full title of the trial
    Effects of Acetyl-DL-Leucine on cerebellar ataxia - a multinational, multicenter, randomized, double-blind, placebo-controlled, 2-way crossover phase III trial (ALCAT)
    Wirksamkeit von Acetyl-DL-Leucin bei Patienten mit cerebellärer Ataxie –eine multinationale, multizentrische, randomisierte, doppelblinde, placebo-kontrollierte, Phase III Studie im crossover Design
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Effects of Acetyl-DL-Leucine on cerebellar ataxia
    Wirksamkeit von Acetyl-DL-Leucin bei Patienten mit Kleinhirnerkrankungen
    A.3.2Name or abbreviated title of the trial where available
    A.4.1Sponsor's protocol code numberALCAT
    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
    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 fuer Forschung und Bildung (BMBF)
    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 Studienzentrale
    B.5.3 Address:
    B.5.3.1Street AddressMarchioninistrasse 15
    B.5.3.2Town/ cityMunich
    B.5.3.3Post code81377
    B.5.4Telephone number0049089440076987
    B.5.5Fax number0049089440078795
    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. name Tanganil
    D. of the Marketing Authorisation holderPierre Fabre Medicament
    D.2.1.2Country which granted the Marketing AuthorisationFrance
    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.3.9.3Other descriptive nameTanganil
    D.3.9.4EV Substance CodeSUB05231MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number500
    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. cell therapy medicinal product No
    D. therapy medical product No
    D. Engineered Product No
    D. ATIMP (i.e. one involving a medical device) No
    D. 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 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
    Cerebellar ataxia is a form of ataxia originating in the cerebellum and is most often caused by neurodegenerative disorders of the cerebellum, either hereditary or sporadic. The leading clinical symptoms of cerebellar ataxia are disturbances of stance/gait (> 85%) with recurrent falls, limb ataxia with severe functional impairment of arm and hand movements, dysarthrophonia with impaired oral communication abilities and ocular motor disturbances with impaired vision.
    E.1.1.1Medical condition in easily understood language
    Cerbellar ataxia is a neurodegenerative neurological disorder characterized by disturbances of gait, imbalance, speech and motor skills
    E.1.1.2Therapeutic area Diseases [C] - Nervous System Diseases [C10]
    MedDRA Classification
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary aim of the ALCAT trial is to determine the effect of Acetyl-DL-Leucine compared to placebo intervention on improving motor function measured by the total score of the Scale for the Assessment and Rating of Ataxia (SARA).
    E.2.2Secondary objectives of the trial
    To demonstrate that Acetyl-DL-Leucine is efficacious in
    1. improving motor function measured by the Spinocerebellar Ataxia Functional Index (SCAFI) and SARA subscore items
    2. improving quality of life (EQ-5D-5L) as well as depression (Beck Depression Inventory, BDI-II) and fatigue (Fatigue Severity Score, FSS)
    3. to check for the occurrence of adverse effects of the trial drug.
    4. improving gait patterns measured by gait analysis (examination will be perfomed in the sub group of patients being enrolled in the trial center DSGZ)
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Subjects will only be included in the study if they meet all of the following criteria:
    1) Clinically confirmed cerebellar ataxia (CA) with a total SARA-Score ≥ 3 (range 0-40) of hereditary or non-hereditary degenerative type
    2) Patient did not receive any of the following prohibited medication within 4 weeks prior to randomization: aminopyridines, Acetyl-DL-Leucine, Riluzole, Gabapentin, Varenicline, Chlorzoxazone
    3) The ability to follow study instructions and likely to attend and complete all required visits
    4) Written informed consent of the subject prior to any study specific intervention
    5) Age ≥ 18 years
    E.4Principal exclusion criteria
    Subjects will not be included in the study if any of the following criteria applies:
    1) Subject is not able to give consent
    2) Onset of ataxia in association with stroke, encephalitis, sepsis, hyperthermia or heat stroke
    3) Toxic causes for ataxia of cerebellar type
    4) Rapid progression of ataxia (development of severe ataxia in less than 12 weeks)
    5) Subject suffers from any of the following:
    o chronic diarrhea
    o unexplained visual loss
    o malignancies
    o insulin-dependent diabetes mellitus
    6) Ataxia due to multiple sclerosis, ischemia, hemorrhage or tumor of the posterior fossa as confirmed by imaging
    7) Ataxia due to clinical likely multisystem atrophy type C (MSA-C)
    8) Diagnosis of clinical likely Friedreich ataxia
    9) Known history of hypersensitivity to the investigational drug or derivates
    10) Liver failure defined as AST/ALT > 300 U/l
    11) Simultaneous participation in another clinical trial or participation in any clinical trial involving administration of an investigational medical product within 30 days prior to the beginning of the clinical trial
    12) Subjects with a physical or psychiatric condition which at the investigator’s discretion may put the subject at risk, may confound the trial results, or may interfere with the subject’s participation in this clinical trial
    13) Known or persistent abuse of medication, drugs or alcohol
    14) Females of childbearing potential, who are not using and not willing to use medically reliable methods of contraception for the entire study duration as listed in the patient informed consent form
    15) Current or planned pregnancy or nursing women
    16) Patient has received any of the following prohibited medication within 4 weeks prior to randomization
    o Aminopyridines (including substained-release form)
    o Acetyl-DL-Leucine
    o Riluzole
    o Gabapentin
    o Varenicline
    o Chlorzoxazone

    E.5 End points
    E.5.1Primary end point(s)
    Primary efficacy endpoint:
    The primary efficacy endpoint is defined as the absolute changes in SARA total score from period-level baseline to week 2 after start of treatment start and to the end of the 6-weeks treatment period, i.e. the difference between post-treatment values and the corresponding period-level baseline values:

    Delta (SARAtotal) = SARAtotal (post-treatment) – SARAtotal (period baseline)

    E.5.1.1Timepoint(s) of evaluation of this end point
    after 2 and 6 weeks of treatment with IMP (for each treatment period)
    E.5.2Secondary end point(s)
    Key secondary endpoint(s):
    SCAFI and subscores of SARA, QoL (EQ-5D-5L), depression (BDI-II), fatigue (FSS)

    Assessment of safety:
    Documentation and reporting of side effects (AEs, SAEs, SUSARs)
    E.5.2.1Timepoint(s) of evaluation of this end point
    6 weeks of treatment with IMP (for each treatment period) as well as in follow-up 4 weeks after taking the last IMP.
    Absolute changes at the end of the 6-week treatment period compared to the period-level baseline defined as score at visit 2 (treatment period 1) and at visit 5 (treatment period 2) as well as 4 weeks after taking the last IMP (follow-up).

    Absolute changes in the coefficient of variation gait parameter of gait analysis (examination will be perfomed in the sub group of patients being enrolled in the trial center DSGZ).
    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) 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 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 concerned6
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA7
    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 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 months8
    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 months8
    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: 80
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 28
    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 state90
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 108
    F.4.2.2In the whole clinical trial 108
    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)
    All patients are already known in the clinicals and study centers. Thus, the follow-up and medical care after the end of the clinical trial is possible for the patients, because they are regularly seen in clinical follow-up examinations. The medical file will contain details about the participation in the clinical trial with all collected clinical findings. If a patient improved under the drug tested in the trial, further treatment with this IMP could be offered by the study centers.
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    G.4.1Name of Organisation Deutsche Heredo-Ataxie Gesellschaft Bundesverband eV
    G.4.3.4Network Country Germany
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2015-11-18
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-11-10
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2017-07-07
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