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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:2011-002154-32
    Sponsor's Protocol Code Number:AT2220-010
    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:2011-10-07
    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 number2011-002154-32
    A.3Full title of the trial
    AN OPEN-LABEL MULTI-CENTER, INTERNATIONAL STUDY TO INVESTIGATE DRUG-DRUG INTERACTIONS BETWEEN AT2220 AND ALGLUCOSIDASE ALFA IN PATIENTS WITH POMPE DISEASE
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Drug-drug interaction study between AT2220 and ERT
    A.4.1Sponsor's protocol code numberAT2220-010
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT01380743
    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
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAmicus Therapeutics
    B.5.2Functional name of contact pointPatient Advocacy
    B.5.3 Address:
    B.5.3.1Street Address1 Cedarbrook Drive
    B.5.3.2Town/ cityCranbury
    B.5.3.3Post code08512
    B.5.3.4CountryUnited States
    B.5.4Telephone number1609662-2000
    B.5.5Fax number1609662-2001
    B.5.6E-mailpatientadvocacy@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 Powder for oral solution
    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 INNDuvoglustat hydrochloride
    D.3.9.2Current sponsor codeAT2220
    D.3.9.3Other descriptive name1-deoxynojirimycin hydrochloride
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number50
    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 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 Powder for oral solution
    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 INNDuvoglustat hydrochloride
    D.3.9.2Current sponsor codeAT2220
    D.3.9.3Other descriptive name1-deoxynojirimycin hydrochloride
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number200
    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
    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] - Nutritional and Metabolic Diseases [C18]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 14.1
    E.1.2Level LLT
    E.1.2Classification code 10036143
    E.1.2Term Pompe's disease
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    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 single ascending oral doses of AT2220 administered 1 hour before administration of alglucosidase alfa in patients with Pompe disease
    • To evaluate the effect of single ascending oral doses of AT2220 administered 1 hour before administration of a single intravenous infusion of alglucosidase alfa on the plasma pharmacokinetics of recombinant human acid alpha-glucosidase (rhGAA) in patients with Pompe disease
    E.2.2Secondary objectives of the trial
    • To assess acid alpha glucosidase (GAA) enzyme activity and protein levels in skeletal muscle at Day 3 or Day 7 following a single intravenous infusion with alglucosidase alfa alone and after pre-administration of single ascending oral doses of AT2220
    • To evaluate the concentration of AT2220 in skeletal muscle at Day 3 or Day 7 after pre-administration of single ascending oral doses of AT2220. Subjects will be assigned to either Day 3 or Day 7 for both treatment periods.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Male or female, diagnosed with Pompe disease and between 18 and 65 years of age, inclusive
    2. Subject has been on a stable regimen and dose of alglucosidase alfa for at least 3 months before screening (stable regimen defined as currently receiving alglucosidase alfa every 2 weeks and stable dose defined as not varying by more than ± 10%)
    3. Subject has an estimated glomerular filtration rate (eGFR) ≥ 50 mL/min at Screening; eGFR to be estimated using the 4-parameter Modification of Diet in Renal Disease (MDRD) equation:
    eGFR (mL/min/1.73 m^2) = 175 x (Scr)^(-1.154) x (Age)^(-0.203) x (0.742 if female) x (1.212 if African-American)
    4. Male and female subjects of childbearing potential agree to use medically accepted methods of contraception during the study and for 30 days after study drug administration
    5. Subject is willing and able to provide written informed consent and 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 3 months before Screening
    2. Subject has clinically significant unstable cardiac disease (e.g., cardiac disease requiring active management, such as symptomatic arrhythmia, unstable angina or NYHA class III or IV congestive heart failure)
    3. Subject requiring invasive mechanical ventilation or is confined to a wheelchair
    4. Subject has a history of allergy or sensitivity to study drug (including excipients) or other iminosugars (e.g., miglustat, miglitol)
    5. Subject is pregnant or breastfeeding
    6. Subject tests positive for hepatitis B surface antigen or hepatitis C antibody
    7. Subject has received any investigational/experimental drug or device within 30 days of Screening
    8. Subject has any concurrent illness or condition that may preclude the subject from fulfilling the protocol requirements or suggests to the investigator that the potential subject may have an unacceptable risk by participating in this study
    E.5 End points
    E.5.1Primary end point(s)
    • Safety, including AEs (including infusion-associated reactions), clinical laboratory tests (hematology, urinalysis, serum chemistry including creatine kinase, LDH, alkaline phosphatase, ALT and AST), Urine glucose Hex4, 12-lead ECGs, physical examinations, vital signs, and muscle strength tests
    • Plasma acid alpha glucosidase Cmax and AUC (estimated) for enzyme activity after an alglucosidase alfa intravenous infusion alone and after pre-administration of single ascending oral doses of AT2220; total protein concentration will also be evaluated for each infusion
    E.5.1.1Timepoint(s) of evaluation of this end point
    Tests described in E.5.1 will be performed throughout the study
    E.5.2Secondary end point(s)
    • Acid alpha glucosidase levels in muscle at Day 3 or Day 7after a single intravenous administration of alglucosidase alfa alone and in combination with single ascending oral doses of AT2220 by measuring acid alpha glucosidase enzyme activity and protein levels
    • The concentration of AT2220 in skeletal muscle tissue homogenate at Day 3 or Day 7 after pre-administration of single ascending oral doses of AT2220 in Treatment Period 2.
    • Acid alpha glucosidase levels in muscle at Day 3 or Day 7after a single intravenous administration of alglucosidase alfa alone and in combination with single ascending oral doses of AT2220 by measuring acid alpha glucosidase enzyme activity and protein levels. The concentration of AT2220 in skeletal muscle tissue homogenate at Day 3 or Day 7 after pre-administration of single ascending oral doses of AT2220 in Treatment Period 2.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Please refer to E.5.2
    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 No
    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 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 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.8.3 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 concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA5
    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
    Canada
    France
    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
    LVLS
    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 months0
    E.8.9.1In the Member State concerned days30
    E.8.9.2In all countries concerned by the trial years0
    E.8.9.2In all countries concerned by the trial months0
    E.8.9.2In all countries concerned by the trial days30
    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: 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 No
    F.4 Planned number of subjects to be included
    F.4.1In the member state2
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 6
    F.4.2.2In the whole clinical trial 22
    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)
    Standard of care
    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 Decision2011-11-04
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2011-12-07
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2013-01-04
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