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    Summary
    EudraCT Number:2020-003120-17
    Sponsor's Protocol Code Number:PDY16963
    National Competent Authority:Germany - BfArM
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2021-02-23
    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 number2020-003120-17
    A.3Full title of the trial
    A 3-part study to evaluate the efficacy and safety of venglustat in combination with Cerezyme in adult and pediatric patients with Gaucher disease Type 3 (GD3) with open-label long-term treatment
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Venglustat in Combination with Cerezyme in Adult and Pediatric Patients with Gaucher Disease Type 3
    A.3.2Name or abbreviated title of the trial where available
    LEAP2IT
    A.4.1Sponsor's protocol code numberPDY16963
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT02843035
    A.5.3WHO Universal Trial Reference Number (UTRN)U1111-1156-4278
    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
    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
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationSanofi-Aventis Deutschland GmbH
    B.5.2Functional name of contact point
    B.5.3.4CountryGermany
    B.5.6E-mailmedinfo.de@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 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/3/14/1374
    D.3 Description of the IMP
    D.3.1Product nameVenglustat
    D.3.2Product code GZ402671
    D.3.4Pharmaceutical form Tablet
    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 INNVenglustat malate
    D.3.9.1CAS number 1629063-78-0
    D.3.9.2Current sponsor codeGZ402671
    D.3.9.4EV Substance CodeSUB166602
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    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 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 nameVenglustat
    D.3.2Product code GZ402671
    D.3.4Pharmaceutical form Tablet
    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 INNVenglustat malate
    D.3.9.1CAS number 1629063-78-0
    D.3.9.2Current sponsor codeGZ402671
    D.3.9.3Other descriptive nameGenz-682452-AU
    D.3.9.4EV Substance CodeSUB166602
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number6
    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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboTablet
    D.8.4Route of administration of the placeboOral use
    D.8 Placebo: 2
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboTablet
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Gaucher disease type 3
    E.1.1.1Medical condition in easily understood language
    Gaucher disease type 3
    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 20.0
    E.1.2Level PT
    E.1.2Classification code 10075699
    E.1.2Term Gaucher's disease type III
    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
    Part 1, cohort 3:
    - Evaluate cerebrospinal fluid (CSF) biomarkers in adult Gaucher disease (GD) Type 3 patients that distinguish GD3 from adult Gaucher disease Type 1 (GD1) patients.
    - Screen adult and pediatric GD3 patients who qualify for treatment with venglustat in Parts 2 and 3.

    Part 2 and 3, cohort 3:
    - Evaluate the efficacy of venglustat in combination with Cerezyme in adult and pediatric GD3 patients by assessing:
    -Ataxia using the Scale for the Assessment and Rating of Ataxia (SARA)
    -Cognition using the Repeatable Battery for the Assessment of Neuropsychological Status (RBANS)
    E.2.2Secondary objectives of the trial
    Parts 2 and 3, cohort 3:
    -Evaluate the efficacy of venglustat in combination with Cerezyme in adult and pediatric GD3 patients by assessing:
    -CSF Lyso-GL1 levels
    -Modified Friedreich´s Ataxia Rating Scale - Activities of Daily Living (FARS-ADL)
    -Brain resting-state functional Magnetic Resonance Imaging (rs-fMRI) reflecting connectivity between parieto-occipital areas
    -Bone disease manifestations

    -Evaluate safety and tolerability of venglustat in combination with Cerezyme in adult and pediatric GD3 patients

    -Evaluate PK of venglustat in adult and pediatric GD3 patients.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - GD3 adult participant =18 years of age.

    - GD3 pediatric participant is =12 years of age <18 years and =30 kg of weight. For male patients 12 to <18 years of age: There is a potential risk for venglustat to impact male fertility particularly when used in patients who have not reached a certain stage of sexual maturation. If a patient has not reached Tanner Stage 3 by the screening visit, the Investigator should assess and discuss with the patient the potential benefits and risks when considering eligibility for study participation. In this benefit/risk assessment-discussion, the parent(s)/legal guardian(s), the patient (if considered able by local regulation), and the Sponsor’s medical representative (if considered appropriate) shall be consulted.
    E.4Principal exclusion criteria
    - Substrate reduction therapy or chaperone therapy for GD within 6 months prior to enrollment.

    - Participant has had a partial or total splenectomy within 3 years prior to randomization.

    - Participant is blood transfusion-dependent.

    - Prior esophageal varices or liver infarction or current liver enzymes (alanine aminotransferase [ALT]/ aspartate aminotransferase [AST]) or total bilirubin >2 times the upper limit of normal, unless
    the patient has a diagnosis of Gilbert Syndrome.

    - Participant has any clinically significant disease, other than GD, including cardiovascular (congenital cardiac defect, coronary artery disease, valve disease or left sided heart failure; clinically significant arrhythmias or conduction defect), hepatic, gastrointestinal, pulmonary, neurologic, endocrine, metabolic (eg, hypokalemia, hypomagnesemia) or psychiatric disease, other medical conditions, or serious intercurrent illnesses that may preclude participation.

    - Participant has renal insufficiency, as defined by an estimated glomerular filtration rate <30 mL/min/1.73m2 at the screening visit.

    - Participant has received an investigational product within 30 days prior to enrollment.

    - Participant has a history of cancer, with the exception of basal cell carcinoma.

    - Participant has myoclonic seizures.

    - Participant is pregnant or lactating.

    - Participant has, according to World Health Organization (WHO) Grading, a cortical cataract >onequarter of the lens circumference (Grade cortical cataract-2) or a posterior subcapsular cataract >2
    mm (Grade posterior subcapsular cataract-2). Patients with nuclear cataracts will not be excluded.

    - Participant requires use of invasive ventilatory support.

    - Participant requires use of noninvasive ventilator support while awake for longer than 12 hours daily.

    - Participant is unable to receive treatment with Cerezyme due to a known hypersensitivity or is unwilling to receive Cerezyme treatment to ensure maintenance of Gaucher treatment goals.

    - Participant is currently receiving potentially cataractogenic medications (corticosteroids, psoralens used in dermatology with ultraviolet light therapy [PUVA], typical antipsychotics, and glaucoma
    medications) or any medication that may worsen the vision of a patient with cataract (eg, alphaadrenergic glaucoma medications).

    - Participant has received strong or moderate inducers or inhibitors of CYP3A within 15 days or 5 half-lives from screening, whichever is longer, prior to enrolment in Part 2. This also includes the
    consumption of grapefruit, grapefruit juice, or grapefruit containing products within 72 hours of starting venglustat administration in Parts 2 and 3.

    - Participant is scheduled for in-patient hospitalization including elective surgery, during the study.

    - Participant has had a major organ transplant (e.g., bone marrow or liver).

    - Participant, in the opinion of the investigator, is unable to adhere to the requirements of the study or unable to undergo study assessments (e.g., contraindications for magnetic resonance imaging).
    E.5 End points
    E.5.1Primary end point(s)
    - Change in Scale for Assessment and Rating of Ataxia (SARA) modified total score from baseline to Week 52 (Cohort 3 Part 2)

    - Change in Repeatable Battery for the Assessment of Neuropsychological Status (RBANS) total scale index score from baseline to Week 52 (Cohort 3 Part 2)
    E.5.1.1Timepoint(s) of evaluation of this end point
    Baseline and Week 52
    E.5.2Secondary end point(s)
    1/ Percent Change in CSF Lyso-GL1 levels from baseline to Baseline and Week 52 Week 52 (Cohort 3 Part 2)

    2/ Change in Modified Friedreich´s Ataxia Rating Scale - Activities of Daily Living (FARS-ADL) score from baseline to Week 52 (Cohort 3 Part 2)

    3/ Change in connectivity between vision perception (resting state networks 3) and action execution (resting state network 6) areas by task-free blood oxygenation level-dependent (BOLD) fMRI from baseline to Week 52 (Cohort 3 Part 2)

    4/ Change in dual-energy X-ray absorptiometry (DXA) lumbar spine bone mineral density (BMD) T-score from baseline to Week 52 (Cohort 3 Part 2)

    5/ Change in bone MRI bone marrow burden (BMB) total score from baseline to Week 52 (Cohort 3 Part 2)

    6/ Number of participants with TEAEs (Cohort 3 Part 2 and 3)

    7/ Growth rate (Cohort 3 Part 2 and 3)

    8/ Bone age by hand X-ray (Cohort 3 Part 2 and 3)

    9/ Sexual maturation (Tanner stage) (Cohort 3 Part 2 and 3)

    10/ Immunogenicity testing to assess possible IAR to Cerezyme (Cohort 3 Part 1)

    11/ Beck Depression Inventory-II (BDI-II) (Cohort 3 Part 2 and 3)

    12/ Maximum venglustat plasma concentration (Cmax) (Cohort 3 Part 2)

    13/ Time to maximum venglustat plasma concentration (tmax) (Cohort 3 Part 2)

    14/ Area under the venglustat plasma concentration versus time curve from time 0 to 24 hours (AUC0-24) (Cohort 3 Part 2)

    15/ Trough venglustat plasma concentration (Ctrough) (Cohort 3 Part 2 and 3)

    16/ Venglustat CSF concentration (Cohort 3 Part 2) (only participants ≥18 years of age)
    E.5.2.1Timepoint(s) of evaluation of this end point
    1/ to 5/ : Baseline and Week 52

    6/ to 9/ ; 11/ : Up to Week 182

    10/ : 60 days (Part 1 - screening phase)

    12/ to 14/ : Day 1 pre-dose and 1, 2, 4, 8 and 24 hours post-dose

    15/ : Pre-dose at Day 2 and Week 4, 52, 78, 104, 156

    16/ : Week 52
    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 No
    E.6.5Efficacy No
    E.6.6Pharmacokinetic No
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    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) 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 Yes
    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.8.2.4Number 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 EEA9
    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
    Brazil
    Canada
    China
    Egypt
    Japan
    Taiwan
    Turkey
    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 years2
    E.8.9.1In the Member State concerned months10
    E.8.9.1In the Member State concerned days24
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months8
    E.8.9.2In all countries concerned by the trial days27
    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: 10
    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: 10
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 24
    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 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
    For some adult participants, the reason linked to their medical condition (e.g. cognition impact and in accordance with national requirement)
    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 34
    F.4.2.2In the whole clinical trial 12
    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 Decision2021-03-03
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-04-12
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    P.Date of the global end of the trial2021-06-10
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