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    Summary
    EudraCT Number:2015-000582-31
    Sponsor's Protocol Code Number:AGLU3306
    Clinical Trial Type:Outside EU/EEA
    Date on which this record was first entered in the EudraCT database:2015-04-15
    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
    H.4 THIRD COUNTRY IN WHICH THE TRIAL WAS FIRST AUTHORISED
    Expand All   Collapse All
    A. Protocol Information
    A.2EudraCT number2015-000582-31
    A.3Full title of the trial
    An Exploratory, Open-Label Study of the Safety and Efficacy of High Dose or High Dosing Frequency Myozyme® (alglucosidase alfa) Treatment in Patients with Pompe Disease Who Do Not Have an Optimal Response to the Standard Dose Regimen
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    An Exploratory, Open-Label Study of the Safety and Efficacy of High Dose or High Dosing Frequency Myozyme® (alglucosidase alfa) Treatment in Patients with Pompe Disease Who Do Not Have an Optimal Response to the Standard Dose Regimen
    A.4.1Sponsor's protocol code numberAGLU3306
    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 SponsorGenzyme Corporation 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 supportGenzyme Corporation Inc
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationGenzyme, a Sanofi Company
    B.5.2Functional name of contact pointTrial Transparency Team
    B.5.3 Address:
    B.5.3.1Street Address-
    B.5.3.2Town/ city-
    B.5.3.4CountryUnited States
    B.5.6E-mailContact-US@sanofi.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 Yes
    D.2.1.1.1Trade name Myozyme 50 mg powder for concentrate for solution for infusion
    D.2.1.1.2Name of the Marketing Authorisation holderGenzyme
    D.2.1.2Country which granted the Marketing AuthorisationUnited States
    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.3.4Pharmaceutical form Powder for concentrate for solution for 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 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/g milligram(s)/gram
    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 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 (acid alpha-glucosidase deficiency)
    E.1.1.1Medical condition in easily understood language
    People with Pompe disease have low levels of an enzyme called acid
    alpha-glucosidase. This enzyme helps the body control levels of glycogen
    (a type of carbohydrate)
    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 17.1
    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
    1. Evaluate the safety and efficacy of alternate alglucosidase alfa dosing regimens
    2. Evaluate differences in efficacy in the 2 dosing arms
    E.2.2Secondary objectives of the trial
    1. Effect of increases in alglucosidase alfa dose or dose frequency on cardiac pathophysiology as measured by Left Ventricular Mass and Left Ventricular Mass Index
    2. Effect of increases in alglucosidase alfa dose or dose frequency on respiratory function as measured by ventilator use diary
    3. Effect of increases in alglucosidase alfa dose or dose frequency on proximal and distal muscle strength as measured by manual muscle testing in patients ≥ 8 years of age
    4. Effect of increases in alglucosidase alfa dose or dose frequency on gross motor function as measured by Gross Motor Function Measure
    5. Effect of increases in alglucosidase alfa dose or dose frequency on functional status as measured by the Pompe Pediatric Evaluation of Disability Index
    6. Effect of increases in alglucosidase alfa dose or dose frequency on health-related quality of life as measured by the Physical Component Summary (PCS) score of the Medical Outcomes Study (MOS) SF-36 for patients ≥14 years of age.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    (1) the patient or the patient’s legal guardian(s) must provide written informed
    consent prior to any study-related procedures being performed; (2) the patient must have a clinical diagnosis of Pompe disease as defined by documented GAA deficiency (deficient endogenous GAA activity) in skin fibroblasts or blood; (3) the patient must have been compliant with the standard dosing regimen of Myozyme (20 mg/kg qow) for a minimum of 6 months prior to study entry; and (4) the patient must have clinical decline or sub-optimal improvement in at least one of the following parameters as compared to their condition prior to beginning Myozyme treatment:

    Cardiac
    Left Ventricular Mass (LVM) Z-score ≥ 6 or LVM Index ≥ 150 g/m2 after a minimum of 6 months of treatment with Myozyme, OR

    Respiratory
    New development of respiratory failure requiring the use of ventilatory assistance (invasive or noninvasive) after a minimum of 6 months of treatment with Myozyme. Ventilatory assistance must have been required for at least 4 weeks prior to study enrollment, OR

    Motor Skills
    For patients < or equal to 2 years of age at study entry, failure to acquire at least 2 new gross motor milestones after a minimum of 6 months of treatment with Myozyme, e.g.:
    1. Turning head side to side (supine)
    2. Grasping small objects with hands
    3. Transferring objects from hand to hand
    4. Holding head upright with body supported
    5. Rolling (supine to prone or prone to supine)
    6. Sitting (supported or unsupported)
    7. Walking (with support, i.e., cruising, or independently)
    8. Walking up stairs (with assistance or independently)

    OR
    For patients > 2 years of age at study entry, worsening of proximal upper extremity muscle weakness as determined by the Investigator through longitudinal assessments of manual muscle testing after a minimum of 6 months of treatment with Myozyme, OR

    For patients > 2 years of age at study entry, worsening of proximal upper extremity muscle weakness as determined by the Investigator through loss of functional use of the upper extremities after a minimum of 6 months of treatment with Myozyme, OR

    Progression to use of an assistive device for ambulation due to worsening of proximal lower extremity muscle weakness after a minimum of 6 months of treatment with Myozyme.





    E.4Principal exclusion criteria
    Patients will be excluded from this study if they meet any of the following exclusion criteria (1) any medical condition which, in the opinion of the Investigator, could interfere with treatment or evaluation of safety and/or efficacy of Myozyme; (2) the patient has major congenital abnormality; (3) the patient has used any investigational product (other than alglucosidase alfa in those regions where the product is not commercially available) within 30 days prior to study enrollment; or (4) the patient is pregnant or lactating.
    E.5 End points
    E.5.1Primary end point(s)
    1. Participants' Efficacy Response During the Treatment Period as Compared to Baseline for Participants With Respiratory Decline on Standard Treatment
    2. Participants' Efficacy Response During the Treatment Period as Compared to Baseline for Participants With Motor Function Decline on Standard Treatment
    3. Summary of Participants Reporting Treatment-Emergent Adverse Events During the Treatment Period
    E.5.1.1Timepoint(s) of evaluation of this end point
    1 and 2 : Baseline, Week 52
    3 : Day 1 up to Week 52
    E.5.2Secondary end point(s)
    1. Baseline Values for Left Ventricular Mass (LVM) Z-Scores
    2. Change From Baseline in Left Ventricular Mass (LVM) Z-Score at Week 52
    3. Baseline Values for Left Ventricular Mass Index (LVMI)
    4. Change From Baseline in Left Ventricular Mass Index (LVMI) at Week 52
    5. Change From Baseline in Ventilator Use at Last Assessment (Approximately Week 52)
    6. Change From Baseline in Body Strength Measured by the Manual Muscle Testing (MMT) Total Score at Week 52
    7. Baseline Values of Raw Scores for Gross Motor Function Measure 66 (GMFM-66) Results
    8. Change From Baseline in Raw Scores for Gross Motor Function Measure 66 (GMFM-66) Results at Week 52
    9. Baseline Values in Mobility as Measured by the Pompe Pediatric Evaluation of Disability Inventory (Pompe PEDI)
    10. Change From Baseline in Mobility as Measured by the Pompe Pediatric Evaluation of Disability Inventory (Pompe PEDI) at Week 52
    11. Baseline Values for Normative Physical Component Summary of Medical Outcomes Study Short Form Health Survey (SF-36)
    12. Change From Baseline in Normative Physical Component Summary of Medical Outcomes Study Short Form Health Survey (SF-36) at Week 52
    E.5.2.1Timepoint(s) of evaluation of this end point
    Day 0
    1-3-7-9-11

    Baseline, Week 52
    2-4-5-6-8-10-12
    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 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) No
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) Yes
    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 Will this trial be conducted at a single site globally? No
    E.8.4 Will this trial be conducted at multiple sites globally? Yes
    E.8.6 Trial involving sites outside the EEA
    E.8.6.2Trial being conducted completely outside of the EEA Yes
    E.8.6.3Specify the countries outside of the EEA in which trial sites are planned
    Australia
    Canada
    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.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months3
    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: 13
    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) Yes
    F.1.1.4.1Number of subjects for this age range: 1
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 5
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 7
    F.1.2Adults (18-64 years) No
    F.1.3Elderly (>=65 years) No
    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 No
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Children
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.2 For a multinational trial
    F.4.2.2In the whole clinical trial 14
    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
    H.4 Third Country in which the Trial was first authorised
    H.4.1Third Country in which the trial was first authorised: United States
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