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    The EU Clinical Trials Register currently displays   42869   clinical trials with a EudraCT protocol, of which   7063   are clinical trials conducted with subjects less than 18 years old.
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    Summary
    EudraCT Number:2013-002257-30
    Sponsor's Protocol Code Number:AT2220-017
    National Competent Authority:UK - MHRA
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2013-10-21
    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 ConcernedUK - MHRA
    A.2EudraCT number2013-002257-30
    A.3Full title of the trial
    AN OPEN-LABEL SAFETY AND DOSE-FINDING STUDY OF INTRAVENOUS DUVOGLUSTAT CO-ADMINISTERED WITH RECOMBINANT HUMAN ACID α-GLUCOSIDASE IN SUBJECTS WITH POMPE DISEASE
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A dose finding study with intravenous administration of duvoglustat hydrochloride (AT2220) and Myozyme in Pompe patients
    A.4.1Sponsor's protocol code numberAT2220-017
    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 SponsorAmicus Therapeutics, 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 supportAmicus Therapeutics, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAmicus Therapeutics, Inc.
    B.5.2Functional name of contact pointPatient Advocacy
    B.5.3 Address:
    B.5.3.1Street Address30 A Upper High Street
    B.5.3.2Town/ cityThame Oxon
    B.5.3.3Post codeOX9 3EX
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number16096622000
    B.5.5Fax number16096625010
    B.5.6E-mailclinicaltrials@amicusrx.com
    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 nameduvoglustat hydrochloride
    D.3.2Product code AT2220
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous bolus use (Noncurrent)
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNduvoglustat HCl
    D.3.9.1CAS number 73285-50-4
    D.3.9.2Current sponsor codeAT2220 HCl
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number30.6
    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 Myozyme
    D.2.1.1.2Name of the Marketing Authorisation holderGenzyme Europe B.V., Gooimeer 10, 1411 DD, Naarden, The Netherlands
    D.2.1.2Country which granted the Marketing AuthorisationUnited Kingdom
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/01/018
    D.3 Description of the IMP
    D.3.1Product nameMyozyme
    D.3.4Pharmaceutical form Powder for concentrate for solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous drip use (Noncurrent)
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNALGLUCOSIDASE ALFA
    D.3.9.1CAS number 420784-05-0
    D.3.9.4EV Substance CodeSUB21275
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number0.5 to 4
    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
    Pompe disease
    E.1.1.1Medical condition in easily understood language
    Pompe disease is an enzyme deficiency which results in buildup of glycogen, primarily in muscle cells, that leads to a progressive loss of muscle function.
    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 20.0
    E.1.2Level LLT
    E.1.2Classification code 10036143
    E.1.2Term Pompe's disease
    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
    To evaluate the safety of intravenous (IV) duvoglustat administered immediately before and at point(s) during and after IV infusion of recombinant human acid α glucosidase (rhGAA);
    To determine the optimal dose and administration regimen of IV duvoglustat based on plasma, urine, and muscle pharmacokinetics (PK) of IV duvoglustat
    E.2.2Secondary objectives of the trial
    To evaluate the effects of IV duvoglustat on plasma rhGAA activity
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1.Male or female, diagnosed with Pompe disease and between 16 and 65 years of age, inclusive (where required by local authorities, the minimum age will be 18 years of age)
    2.Subject has never been treated with rhGAA (rhGAA-naïve)
    OR Subject has been treated with Lumizyme (rhGAA-experienced) for at least 12 months, AND for at least the 12 weeks before the Screening Visit, has been consistently treated with Lumizyme as per its local commercial labeling (or as per local regulations, with prior approval of the sponsor), including being on a stable dose, with a stable infusion duration (between 3 and 5 hours) and biweekly schedule (stable defined as not varying by more than ± 10%)
    For subjects who fall outside of these criteria, entry into the clinical trial is at the sponsor’s discretion
    3.Subject, whether male or female, agrees to use medically accepted methods of contraception during the study and for 30 days after their last administration of duvoglustat (unless subject is not of childbearing potential)
    4.Subject is willing and able to provide written informed consent and, as applicable, assent. Subjects under 18 years of age will provide written informed assent, and written informed consent will be provided by their parent or legal guardian
    5.Subject is able to comply with all study procedures
    E.4Principal exclusion criteria
    1.Subject has had a documented transient ischemic attack, stroke, unstable angina, or myocardial infarction within the 12 weeks before the Screening Visit
    2.Subject has clinically significant unstable cardiac disease (ie, any cardiac disease requiring active management, such as symptomatic arrhythmia, unstable angina, or NYHA class III or IV congestive heart failure)
    3.Subject requires any invasive mechanical ventilation
    4.Subject is restricted to a wheelchair or is otherwise not fully ambulatory; subjects who require wheelchair assistance or walkers under special circumstances may be eligible at the discretion of the sponsor
    5.Subject has a history of allergy or sensitivity to study drug (including excipients) or other iminosugars (eg, miglitol, miglustat)
    6.Subject is pregnant or breastfeeding
    7.Subject has a positive medical history for hepatitis A, B, or C, or HIV (human immunodeficiency virus)
    8.Subject has received any investigational/experimental device within the 30 days before the Screening Visit
    9.Subject is taking, or has taken at any time in the 12 weeks before the Screening Visit, a prohibited medication [miglitol (eg, Glyset); acarbose (eg, Precose, Glucobay); voglibose (eg, Volix, Vocarb, Volibo); oseltamivir (eg, Tamiflu); dietary supplements containing mulberry extract; chloroquine; any investigational/experimental drug]
    10.Subject is taking, or has taken at any time in the 26 weeks before the Screening Visit, the prohibited medication miglustat (eg, Zavesca)
    11.Subject is taking, or has taken at any time in the 12 months before the Screening Visit, any immunosuppressant drug (eg, cyclosporins, azathioprines, methotrexate, monoclonal antibodies that block the immune system, and long-term corticosteroids). Subjects who require corticosteroids for acute management of infusion reactions may be allowed with sponsor approval
    12.Subject has any concurrent illness or condition that may preclude the subject from fulfilling the protocol requirements or suggests to the investigator that the subject may have an unacceptable risk by participating in this study
    E.5 End points
    E.5.1Primary end point(s)
    For the primary study objective of evaluating the safety of IV duvoglustat the parameters are as follows: adverse events (AEs), clinical laboratory evaluations (serum chemistry, hematology, and urinalysis), concomitant medications, ECGs, physical examination, weight, vital signs, 6MWT, and FVC

    For the primary study objective of determining the optimal dose and administration regimen of IV duvoglustat, it will be based on plasma, urine, and muscle PK of IV duvoglustat.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Throughout the study.
    E.5.2Secondary end point(s)
    For the secondary study objective of evaluating the effects of IV duvoglustat on plasma rhGAA activity, the parameters will be as specified in the SAP.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Throughout the study.
    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 No
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    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 No
    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 No
    E.8.2.4Number of treatment arms in the trial2
    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 Yes
    E.8.5.1Number of sites anticipated in the EEA2
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Netherlands
    United Kingdom
    United States
    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
    Database lock. Amicus relies on the use of bioanalytical endpoints that are assessed at multiple specialty labs. These data are not captured on the CRF. Analyzing and summarizing all these lab data takes considerably longer than the standard 12 months from LSLV due to the complexity of sample analyses. As such, the appropriate definition of EoT needs to be marked by database lock, which allows time needed to merge the bioanalyical data with the CRF data.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years0
    E.8.9.1In the Member State concerned months10
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years0
    E.8.9.2In all countries concerned by the trial months10
    E.8.9.2In all countries concerned by the trial days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 2
    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) Yes
    F.1.1.6.1Number of subjects for this age range: 2
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 21
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 1
    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 Yes
    F.3.3.7.1Details of other specific vulnerable populations
    Patients 16 - 17 years of age.
    F.4 Planned number of subjects to be included
    F.4.1In the member state8
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 12
    F.4.2.2In the whole clinical trial 24
    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)
    None
    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 Decision2013-11-14
    N.Ethics Committee Opinion of the trial application
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    P.Date of the global end of the trial2013-12-06
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