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    Summary
    EudraCT Number:2015-001578-17
    Sponsor's Protocol Code Number:SHP-GCB-402
    National Competent Authority:UK - MHRA
    Clinical Trial Type:EEA CTA
    Trial Status:GB - no longer in EU/EEA
    Date on which this record was first entered in the EudraCT database:2015-12-21
    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 number2015-001578-17
    A.3Full title of the trial
    An Open-label, Multicenter, Single-arm, Phase 4 Study of the Effect of
    Treatment with Velaglucerase alfa on Bone-related Pathology in
    Treatment-naïve Patients with Type 1 Gaucher Disease
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    An Open-Label, Phase IV Study of Velaglucerase alfa on Bone Related Pathology in Adult, Treatment-Naïve Patients with Type 1 Gaucher Disease
    A.4.1Sponsor's protocol code numberSHP-GCB-402
    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 SponsorShire Human Genetic Therapies, 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 supportShire Human Genetic Therapies, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationShire
    B.5.2Functional name of contact pointTosha Doornek
    B.5.3 Address:
    B.5.3.1Street Address300 Shire Way
    B.5.3.2Town/ cityLexington,
    B.5.3.3Post codeMA 02421
    B.5.3.4CountryUnited States
    B.5.4Telephone number+14065426575
    B.5.6E-mailtosha.doornek@takeda.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 VPRIV 400 Units powder for solution of infusion
    D.2.1.1.2Name of the Marketing Authorisation holderShire Pharmaceuticals Ireland Limited
    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/3/10/752
    D.3 Description of the IMP
    D.3.4Pharmaceutical form Powder 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 INNVELAGLUCERASE ALFA
    D.3.9.1CAS number 884604-91-5
    D.3.9.2Current sponsor codeGA-GCB
    D.3.9.3Other descriptive nameGene activated human glucocerebrosidase, velaglucerase alfa
    D.3.9.4EV Substance CodeSUB31010
    D.3.10 Strength
    D.3.10.1Concentration unit IU/ml international unit(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number100
    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
    Gaucher Disease
    E.1.1.1Medical condition in easily understood language
    Gaucher disease is caused by a defective enzyme. Due to this, fatty substances build up in the cells of the body, especially in liver, spleen and bone marrow.
    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 20.0
    E.1.2Level PT
    E.1.2Classification code 10075697
    E.1.2Term Gaucher's disease type I
    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 effect of VPRIV therapy (60 U/kg EOW) in treatment-naïve patients with type 1 Gaucher disease on change from baseline in lumbar spine (LS) bone mineral density (BMD) Z-score as measured by dual energy x-ray absorptiometry (DXA) after 24 months of treatment
    E.2.2Secondary objectives of the trial
    The secondary objectives are to evaluate the effect of VPRIV therapy (60U/kg EOW) over time
    in treatment-naïve patients with type 1 Gaucher disease on:
    · BMD as measured by the change from baseline in LS BMD Z-score after 12 months of treatment
    · BMB as measured by magnetic resonance imaging (MRI) of the LS and femur after 12 and 24 months of treatment
    · Hemoglobin concentration after 12 and 24 months of treatment
    · Platelet count after 12 and 24 months of treatment
    · Liver volume measured by abdominal MRI after 12 and 24 months of treatment
    · Spleen volume measured by MRI after 12 and 24 months of treatment
    · Bone pain as measured by the BPI after 12 and 24 months of treatment
    · Fatigue measured by the Brief Fatigue Inventory (BFI) after 12 and 24 months of treatment
    · WHO BMD classification (normal bone density, osteopenia, osteoporosis) based on LS T-scores after 12 and 24 months of treatment
    · Safety
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Each patient must meet the following criteria to be enrolled in this study.
    1. The patient has a documented diagnosis of type 1 Gaucher disease, as documented by deficient GCB activity in leukocytes (whole blood only) or cultured skin fibroblasts. Diagnosis by only dry blood spot test is insufficient. Diagnosis may be based on results obtained prior to Screening if documented in the patient’s medical history.
    2. Patients must have a LS BMD Z-score < -1 or BMD T-score of < -1 as measured by DXA during the screening phase.
    3. Patient is treatment-naïve, ie, has not received ERT or SRT in the 12 months prior to enrollment.
    4. The patient is ≥18 and ≤70 years of age.
    5. Female patients of childbearing potential must agree to use a medically acceptable method of contraception at all times during the study. (See Section 6.8.3 for acceptable methods of contraception).
    6. The patient, or patient’s legally authorized representative(s), if applicable, understands the nature, scope, and possible consequences of the study and has provided written informed consent that has been approved by the Institutional Review Board/Independent Ethics Committee (IRB/IEC).
    7. The patient must be sufficiently cooperative to participate in this clinical study as judged by the investigator.
    E.4Principal exclusion criteria
    Patients who meet any of the following criteria will be excluded from the study.
    1. Neurological symptoms indicating that the patient may have type 3 Gaucher disease.
    2. A significant comorbidity, which, as determined by the investigator, might affect study data or confound the study results (eg, malignancies, primary biliary cirrhosis, autoimmune liver disease, etc).
    3. Any osteoporosis-specific treatment (eg, bisphosphonates) or treatment with erythropoietin (or erythropoietin-like substances) during the past year.
    4. Structural, joint-associated bone damage of such extent and severity that the investigator deems it could impact participation in the study and assessment of relevant study endpoints (eg, pain).
    5. The patient is pregnant or lactating.
    6. The patient has had a splenectomy.
    7. The patient is enrolled in another clinical study that involves clinical investigations or use of any investigational product (drug or device) within 30 days prior to study enrollment or at any time during the study.
    8. Severe vitamin D deficiency to the level that would be expected to result in osteomalacia (vitamin D < 10 ng/mL [25 nmol/L]). If there is mild vitamin D insufficiency at screening (vitamin D > 10 and < 30 ng/mL) treat with 4000 IU vitamin D per day for 1 month and rescreen.
    9. The patient has previously interrupted ERT for safety reasons.
    10. The patient has had hypersensitivity to the active substance or to any of the excipients.
    E.5 End points
    E.5.1Primary end point(s)
    Change from baseline to 24 months (Week 103 [end of study]) in LS BMD Z-score as measured by DXA.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Assessments will be performed at the screening visit and Weeks 51 and 103 (end of
    study).
    E.5.2Secondary end point(s)
    The secondary efficacy endpoints are:
    ·Change from baseline to 12 months (Week 51) in LS BMD Z-score. Assessments will be performed at the screening visit and Weeks 51 and 103 (end of study).
    ·Change from baseline to 12 months (Week 51) and 24 months (Week 103 [end of study]) in BMB score (MRI of lumbar spine and femur). Assessments will be performed at the baseline visit and Weeks 51 and 103 (end of study).
    ·Change from baseline over time in hemoglobin concentration. Assessments will be performed at baseline and Weeks 13, 25, 37, 51, 65, 77, 89, and 103 (end of study).
    ·Change from baseline over time in platelet count. Assessments will be performed at baseline and Weeks 13, 25, 37, 51, 65, 77, 89, and 103 (end of study).
    ·Change from baseline to 12 months (Week 51) and 24 months (Week 103 [end of study]) in normalized liver volume as measured by abdominal MRI. Assessments will be performed at the baseline visit and Weeks 51 and 103 (end of study).
    ·Change from baseline to 12 months (Week 51) and 24 months (Week 103 [end of study]) in normalized spleen volume as measured by MRI. Assessments will be performed at the baseline visit and Weeks 51 and 103 (end of study).
    ·Change from baseline to 12 months (Week 51) and 24 months (Week 103 [end of study]) in bone pain as measured by the BPI. Assessments will be performed at the baseline visit and Weeks 13, 25, 37, 51, 65, 77, 89, and 103 (end of study).
    ·Change from baseline to 12 months (Week 51) and 24 months (Week 103 [end of study]) in overall fatigue as measured by the BFI. Assessments will be performed at the baseline visit and Weeks 13, 25, 37, 51, 65, 77, 89, and 103 (end of study).
    ·Shifts in WHO BMD classifications (normal bone density, osteopenia, and osteoporosis) based on LS T-scores. Assessments will be performed at baseline (screening visit) and Weeks 51 and 103 (end of study).
    E.5.2.1Timepoint(s) of evaluation of this end point
    . Assessments will be performed at the baseline visit and Weeks 51 and 103 (end of study).

    . Assessments will be performed at the screening visit and Weeks 51 and 103 (end of study).
    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 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) 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.8.3 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
    Germany
    Israel
    Spain
    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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years5
    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 years5
    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: 18
    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 19
    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)
    At the end of the study the patient’s doctor or primary care doctor will discuss the options for continued 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 Decision2016-01-06
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2016-02-17
    P. End of Trial
    P.End of Trial StatusGB - no longer in EU/EEA
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