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    Summary
    EudraCT Number:2005-002759-42
    Sponsor's Protocol Code Number:AGLU02704
    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:2009-07-24
    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 ConcernedGermany - BfArM
    A.2EudraCT number2005-002759-42
    A.3Full title of the trial
    A Randomized, Double-Blind, Multicenter, Multinational, Placebo-Controlled Study of the Safety, Efficacy, and Pharmacokinetics of Myozyme, Recombinant Human Acid alpha-Glucosidase (rhGAA), Treatment in Patients with Late-Onset Pompe Disease
    A.3.2Name or abbreviated title of the trial where available
    LOTS
    A.4.1Sponsor's protocol code numberAGLU02704
    A.5.1ISRCTN (International Standard Randomised Controlled Trial) NumberN/A
    A.7Trial is part of a Paediatric Investigation Plan Information not present in EudraCT
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorGenzyme Europe BV
    B.1.3.4CountryNetherlands
    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 support
    B.4.2Country
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisation
    B.5.2Functional name of contact point
    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
    D.2.1.1.2Name of the Marketing Authorisation holderGenzyme Europe BV
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    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 solution for infusion
    D.3.4.1Specific paediatric formulation Information not present in EudraCT
    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 n.a.
    D.3.9.2Current sponsor coderhGAA
    D.3.9.3Other descriptive namealglucosidase alpha
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number5
    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) Information not present in EudraCT
    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 Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy Information not present in EudraCT
    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 Information not present in EudraCT
    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 placeboPowder for solution for infusion
    D.8.4Route of administration of the placeboIntravenous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Glycogen Storage Disease type II (Pompe's disease)
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 9.1
    E.1.2Level LLT
    E.1.2Classification code 10036143
    E.1.2Term Pompe's disease
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The overall objective is to evaluate the safety, efficacy, and pharmacokinetics (PK) of Myozyme treatment in patients with late-onset Pompe disease as compared to placebo.

    The primary objectives of the study are: 1) to evaluate the safety profile of Myozyme; 2) to determine the effect of Myozyme treatment on functional endurance as measured by the Six Minute Walk Test (6MWT) 3) to determine the effect of Myozyme treatment on respiratory muscle weakness as measured by Forced Vital Capacity (FVC) in the upright position ; and 4) to determine in a subset of patients the PK profile of Myozyme in patients with late-onset Pompe disease. The study wil be considered to have met its primary efficacy objective if a statistically significant treatment effect of Myozyme over placebo is demonstrated in the 6 MWT distance walked.
    E.2.2Secondary objectives of the trial
    Secondary objectives are: 1) to determine the effect of Myozyme treatment on proximal muscle weakness in the lower limbs as measured by Quantitative Muscle Testing (QMT) in bilateral knee flexors (hamstrings) and knee extensors (quadriceps); 2) to determine the effect of Myozyme treatment on health-related quality of life as measured by the Physical Component Summary (PCS) score of the Medical Outcomes Study (MOS) SF-36.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. The patient must provide signed, informed consent prior to performing any study-related procedures. Consent of a legally authorized guardian(s) is (are) required for patients under 18 years of age. If the patient is under 18 years of age and can understand the written informed consent, signature will be required from both the patient and the authorized guardian(s); 2. The patient must have a diagnosis of Pompe disease based on deficient endogenous GAA activity in cultured skin fibroblasts of ≤ 40% of the normal mean of the testing laboratory and 2 confirmed GAA gene mutations; 3. The patient must be ≥ 8 years of age at the time of enrollment; 4. The patient must have muscle weakness in the lower limbs based on unilateral QMT of the knee extensors defined as < 80% of the predicted value based on age, gender and body size (The National Isometric Muscle Strength [NIMS] Database Consortium, 1996, Arch Phys Med Rehabil); 5. The patient must be able to tolerate pulmonary function testing (PFT) and muscle testing in the supine position. (See exclusion criteria [1] and [2].); 6. The patient must be able to provide reproducible muscle and pulmonary function test results (bilateral
    QMT measurements [% predicted] in knee extensors within 10% of the highest test value obtained from the same side of the body on 2 consecutive days and FVC measurements [in liters] within 10% of the highest test value obtained in the upright position on 2 consecutive days); 7. The patient must have an FVC of ≥ 30% and < 80% predicted in the upright position (American Thoracic Society [ATS], 1991, Am Rev Respir Disease); 8. The patient must have an FEV1/FVC value of ≥ 70% predicted in the upright position (ATS, 1991, Am Rev Respir Disease); 9. The patient must have a postural drop in FVC (liters) of at least 10% from the upright to the supine position [(FVC supine (L) – FVC upright (L))/FVC upright (L)] * 100%; 10. The patient must have testable muscle in bilateral knee flexors and knee extensors, and testable muscle in bilateral elbow flexors and elbow extensors. (Using QMT, a muscle will be defined as “not testable” if the patient: 1) has a contracture > 90 degrees that would keep them from being able to assume the standard testing position, 2) is unable to follow directions, 3) has significant pain with resistance to the motion, or 4) is so weak that force cannot be generated against the testing strap); 11. The patient must be able to ambulate 40 meters (approximately 130 feet) in 6 minutes on each of 2 consecutive tests performed on the same day (use of assistive devices such as a walker, cane, or crutches, is permitted); 12. The patient (and patient’s legal guardian if patient <18 years of age) must have the ability to comply with the clinical protocol; 13. A female patient of childbearing potential must have a negative pregnancy test (urine beta-human chorionic gonadotropin [β-hCG]) at Baseline. Note: All female patients of childbearing potential and sexually mature males must use a medically accepted method of contraception throughout the study.
    E.4Principal exclusion criteria
    1. The patient requires the use of invasive ventilatory support. (Invasive ventilation is defined as any form of ventilatory support applied with the use of an endotracheal tube.); 2. The patient requires the use of noninvasive ventilatory support while awake and in an upright position. (Noninvasive ventilation is defined as any form of ventilatory support applied without the use of an endotracheal tube. For example, patients receiving positive-pressure ventilation support through a facemask or nose piece are considered as ventilated through noninvasive methods.); 3. The patient has received enzyme replacement therapy with GAA from any source; 4. The patient has used an investigational product within 30 days prior to study enrollment, or is currently enrolled in another study which involves clinical evaluations, unless prior approval is given by Genzyme; 5. The patient has a medical condition, serious intercurrent illness, or other extenuating circumstance that, in the opinion of the Investigator, may significantly interfere with study compliance, including all prescribed evaluations and follow-up activities; 6. The patient has a major congenital anomaly.
    E.5 End points
    E.5.1Primary end point(s)
    See objectives section (E2).
    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 No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic Information not present in EudraCT
    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) 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 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 Yes
    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.8.4.1Number of sites anticipated in Member State concerned1
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA6
    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 Information not present in EudraCT
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    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 years
    E.8.9.1In the Member State concerned months4
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years2
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    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) Yes
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.2Adults (18-64 years) Yes
    F.1.3Elderly (>=65 years) Yes
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    The German study site will enroll one patient < 18 years of age.
    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 30
    F.4.2.2In the whole clinical trial 90
    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)
    Patients who complete this protocol may be eligible for participation in future open-label studies provided there are no safety concerns.
    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 Decision2007-07-18
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2007-06-01
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2007-08-29
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