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    Summary
    EudraCT Number:2019-000954-67
    Sponsor's Protocol Code Number:ATB200-07
    National Competent Authority:Austria - BASG
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2020-03-13
    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 ConcernedAustria - BASG
    A.2EudraCT number2019-000954-67
    A.3Full title of the trial
    A Phase 3 Open-label Extension Study to Assess the Long-term Safety and Efficacy of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late-onset Pompe Disease
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study investigating the long-term safety and efficacy of intravenous (IV) ATB200 when Co-administrated with oral AT2221 in adult subjects with Pompe disease
    A.4.1Sponsor's protocol code numberATB200-07
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT04138277
    A.5.4Other Identifiers
    Name:US INDNumber:127,387
    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 Address3675 Market Street
    B.5.3.2Town/ cityPhiladelphia
    B.5.3.3Post codePA 19104
    B.5.3.4CountryUnited States
    B.5.4Telephone number001215921 7600
    B.5.5Fax number001215921 7900
    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 Yes
    D.2.5.1Orphan drug designation numberEU/3/18/2000
    D.3 Description of the IMP
    D.3.1Product nameATB200
    D.3.2Product code ATB200
    D.3.4Pharmaceutical form Powder for concentrate for solution for injection/infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNcipaglucosidase alfa
    D.3.9.1CAS number 420784-05-0
    D.3.9.2Current sponsor codeATB200
    D.3.9.4EV Substance CodeSUB21275
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number15
    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.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 Yes
    D.2.5.1Orphan drug designation numberEU/3/18/2129
    D.3 Description of the IMP
    D.3.1Product nameAT2221
    D.3.2Product code AT2221
    D.3.4Pharmaceutical form Capsule, hard
    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 INNMIGLUSTAT
    D.3.9.1CAS number 72599-27-0
    D.3.9.2Current sponsor codeAT2221
    D.3.9.3Other descriptive nameAT2221 65 mg Formulated Capsules
    D.3.9.4EV Substance CodeSUB20049
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number65
    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
    Adult Subjects With Late Onset Pompe Disease (LOPD)
    E.1.1.1Medical condition in easily understood language
    A change in the genetic material resulting in lower levels of an enzyme called acid alpha-glucosidase (GAA). The GAA enzyme helps the body to break down glycogen, a type of stored sugar.
    E.1.1.2Therapeutic area Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 24.0
    E.1.2Level LLT
    E.1.2Classification code 10075702
    E.1.2Term Pompe's disease late onset
    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
    The primary objective of this study is to assess the long-term safety and tolerability of ATB200/AT2221 co-administration
    E.2.2Secondary objectives of the trial
    - to assess the long-term efficacy of ATB200/AT2221 co-administration on:
    • ambulatory function, as measured by the 6-minute walk test (6MWT)
    • pulmonary function, as measured by sitting forced vital capacity (FVC) (% predicted)
    • on muscle strength
    • on health-related patient-reported outcomes
    • motor function
    • overall clinical impression, as assessed by both physician and subject
    • measures of pulmonary function other than FVC (% predicted)
    • biomarkers of muscle injury and disease substrate
    - to assess the immunogenicity of ATB200/AT2221 co-administration
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Subjects Who Participated in Study ATB200-03:
    1. Subject must provide signed informed consent prior to any study-related procedures being performed. If the subject is under 20 years of age, the subjects must provide written informed consent.
    2. Subject must have completed Study ATB200-03.
    Note: Subjects who were forced to withdraw from Study ATB200-03 for a logistical reason not related to the efficacy or safety of ATB200/AT2221 (eg, hospitalization for a car accident, COVID-19 pandemic, or emergency surgery) and which resulted in several consecutive missed doses may be eligible to participate in this study upon approval by the Amicus medical monitor.
    3. Female subjects of childbearing potential and male subjects must agree to use medically accepted methods of contraception during the study and for 90 days after the last dose of study drug.
    E.4Principal exclusion criteria
    Subjects Who Participated in Study ATB200-03:
    1. Subject plans to receive gene therapy or participate in another interventional study for Pompe disease.
    2. Subject has a hypersensitivity to any of the excipients in ATB200 or AT2221, or has a medical condition or any other extenuating circumstance that may, in the opinion
    of the investigator or medical monitor, pose an undue safety risk to the subject or may compromise his/her ability to comply with or adversely impact protocol requirements. This includes clinical depression (as diagnosed by a psychiatrist or other mental health professional) with uncontrolled or poorly controlled symptoms.
    3. Subject, if female, is pregnant or breastfeeding.
    4. Subject, whether male or female, is planning to conceive a child during the study.
    E.5 End points
    E.5.1Primary end point(s)
    Long-term safety: incidence of treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), and AEs leading to discontinuation of study drug, frequency and severity of immediate and late IARs, and any abnormalities noted in other safety assessments (eg, clinical laboratory tests, ECGs, vital signs). Immunogenicity to ATB200 will also be described.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Every study visit until regulatory approval or marketing authorization and/or commercialization in the participating subject’s country, or study termination by the sponsor.
    E.5.2Secondary end point(s)
    - Efficacy:
    • change from baseline in 6-minute walk distance (6MWD)
    • change from baseline in 6MWD (% predicted)
    • change from baseline in sitting FVC (% predicted)
    • change from baseline in the manual muscle test score for the lower extremities
    • change from baseline in the total score for the PROMIS – physical function
    • change from baseline in the total score for the PROMIS – fatigue
    • change from baseline in the following variables related to motor function:
    − GSGC total score
    − time to complete the 10-meter walk (ie, assessment of gait) of the GSGC test
    − time to complete the 4-stair climb of the GSGC test
    − time to complete the Gower’s maneuver of the GSGC test
    − time to arise from a chair as part of the GSGC test
    − change from baseline in the time to complete the TUG test
    • change from baseline in the following variables related to muscle strength:
    − manual muscle test score for the upper extremities
    − manual muscle test total score (upper and lower extremities combined)
    − quantitative muscle test value (kg) for the upper extremities
    − quantitative muscle test value (kg) for the lower extremities
    − quantitative muscle test total value (kg) (upper and lower extremities combined)
    • change from baseline in the following variables from patient-reported outcome measures:
    − total score for the PROMIS – dyspnea
    − total score for the PROMIS – upper extremity
    − R-PAct Scale total score
    − EQ-5D-5L health status
    • actual value of the subject’s functional status (improving, stable, or declining) pertaining to the effects of study drug in the following areas of life, as measured by the SGIC:
    − overall physical well-being
    − effort of breathing
    − muscle strength
    − muscle function
    − ability to move around
    − activities of daily living
    − energy level
    − level of muscular pain
    • actual value of the subject’s functional status (improving, stable, or declining), as measured by the PGIC
    • change from baseline in the following measures of pulmonary function, as follows:
    − sitting SVC (% predicted)
    − MIP (cmH2O)
    − MIP (% predicted)
    − MEP (cmH2O)
    − MEP (% predicted)
    − SNIP (cmH2O)

    Pharmacodynamic endpoints are as follows:
    • change from baseline in serum CK level
    • change from baseline in urinary Hex4 level
    E.5.2.1Timepoint(s) of evaluation of this end point
    - Efficacy: Week 12, 26; after Week 26, every 26 weeks until regulatory approval or marketing authorization and/or commercialization in the participating subject’s country, or study termination by the sponsor.
    - Pharmacodynamic: Week 2, 4, 6, 12, 26; after Week 26, every 26 weeks until regulatory approval or marketing authorization and/or commercialization in the participating subject’s country, or study termination by the sponsor.
    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 Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others Yes
    E.6.13.1Other scope of the trial description
    - Effect of ATB200/AT2221 co-administration on biomarkers of muscle injury and disease substrate
    - Immunogenecity of ATB200/AT2221 co-administration
    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 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 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 EEA26
    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
    Argentina
    Australia
    Austria
    Belgium
    Bosnia and Herzegovina
    Bulgaria
    Canada
    Denmark
    France
    Germany
    Greece
    Hungary
    Italy
    Japan
    Korea, Republic of
    Netherlands
    Poland
    Romania
    Slovakia
    Slovenia
    Sweden
    Taiwan
    United Kingdom
    United States
    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
    LPLV
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years4
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years4
    E.8.9.2In all countries concerned by the trial months0
    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 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: 92
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 18
    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 state1
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 54
    F.4.2.2In the whole clinical trial 110
    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)
    Treatment with open-Label ATB200/AT2221 is planned to continue until regulatory approval or marketing authorization and/or commercialization in the participating study subject’s respective country, or study termination by the sponsor.
    After participation in the trial and in case of no marketing authorization and/or commercialization, patients will have the option to be treated with the current standard of care therapy.
    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 Decision2020-03-20
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-06-19
    P. End of Trial
    P.End of Trial StatusOngoing
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