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    Summary
    EudraCT Number:2010-024068-16
    Sponsor's Protocol Code Number:LAL-CL01
    National Competent Authority:UK - MHRA
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2011-01-10
    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 number2010-024068-16
    A.3Full title of the trial
    An open label multicenter study to evaluate the safety, tolerability and pharmacokinetics of SBC-102 in adult patients with liver dysfunction due to lysosomal acid lipase deficiency.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    SBC-102 in patients with liver dysfunction due to LAL deficiency
    A.3.2Name or abbreviated title of the trial where available
    SBC-102 in patients with liver dysfunction due to LAL deficiency
    A.4.1Sponsor's protocol code numberLAL-CL01
    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 SponsorSynageva Biopharma Corp.
    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 support
    B.4.2Country
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationPremier Research Group Ltd.
    B.5.2Functional name of contact pointSophie Bark-Jones
    B.5.3 Address:
    B.5.3.1Street Address1st Floor, Rubra 2, Mulberry Business Park
    B.5.3.2Town/ cityFishponds Road, Wokingham
    B.5.3.3Post codeRG41 2GY
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number01189364040
    B.5.5Fax number01189364001
    B.5.6E-mailsophie.bark-jones@premier-research.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 numberEMA/OD/104/10
    D.3 Description of the IMP
    D.3.1Product namerecombinant human lysosomal acid lipase (rhLAL)
    D.3.2Product code SBC-102
    D.3.4Pharmaceutical form 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 INNnot available
    D.3.9.2Current sponsor codeSBC-102
    D.3.9.3Other descriptive namerecombinant human lysosomal acid lipase (rhLAL)
    D.3.9.4EV Substance CodeAS3
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number2
    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 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 Yes
    D.3.11.13.1Other medicinal product typerecombinant human lysosomal acid lipase (rhLAL)
    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
    Lysosomal Acid Lipase (LAL) Deficiency is a rare autosomal recessive lipid storage disorder that is caused by deficient activity or absence, of the lysosomal enzyme, LAL. It is an extremely rare disorder, with an estimated prevalence of less than 0.2 lives per 100,000. Although a single disease, LAL Deficiency has two phenotypes, Cholesteryl Ester Storage Disease (CESD) and Wolman Disease (WD). Both forms of the disease lead to the accumulation of fats, in various tissues and cell types.
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 14.0
    E.1.2Level SOC
    E.1.2Classification code 10027433
    E.1.2Term Metabolism and nutrition disorders
    E.1.2System Organ Class 10027433 - Metabolism and nutrition disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 14.0
    E.1.2Level HLGT
    E.1.2Classification code 10021605
    E.1.2Term Inborn errors of metabolism
    E.1.2System Organ Class 10027433 - Metabolism and nutrition disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 14.0
    E.1.2Level HLT
    E.1.2Classification code 10024579
    E.1.2Term Lysosomal storage disorders
    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
    The primary objective of the study is to evaluate the safety and tolerability of SBC-102 in patients with liver dysfunction due to LAL deficiency (vital signs, physical examination, clinical laboratory tests, immunogenicity tests, adverse event assessment, concomitant medications).
    E.2.2Secondary objectives of the trial
    The secondary objective is to characterise the pharmacokinetics of SBC-102 delivered by intravenous infusion after single and multiple doses [pre and post infusion Day 0 and 21].
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Patient understands the full nature and purpose of the study, including possible risks and side effects, and is willing and able to comply with all study procedures and provide informed consent 2. Male or female patients ≥ 18 years ≤ 65 years of age 3. Documented decreased LAL activity relative to the normal range of the lab performing the assay or documented result of molecular genetic testing confirming diagnosis of LAL deficiency 4. Evidence of liver involvement based on clinical presentation and/or laboratory test results (ALT or AST ≥ 1.5xULN) 5. If on a statin or ezetimibe, must be on a stable dose for at least 4 weeks prior to screening 6. All women must have negative serum pregnancy test at screening and cannot be breast feeding 7. Female subjects of childbearing potential must agree to use a highly effective and approved contraceptive method(s) for the duration of the study and continue to use for 30 days after last dose of study drug. A highly effective method of contraception is defined as: a. strict abstinence; b. bilateral tubal ligation; c. combined oral contraceptives (estrogens and progesterone), implanted or injectable contraceptives on a stable dose for at least 1 month prior to the Screening visit; d. hormonal intra uterine device (IUD) inserted at least 1 month prior to the Screening visit; e. vasectomized partner for at least 3 months prior to the Screening visit. Male subjects, and their partners must be using, and continue to use for 30 days after last dose of study drug, an acceptable method of birth control. Women considered not of childbearing potential must be surgically sterile (total hysterectomy, bilateral salpingo-oophorectomy) or post-menopausal, which is defined as a complete cessation of menstruation for at least one year after the age of 45 years.
    E.4Principal exclusion criteria
    1. Clinically significant concurrent disease, serious inter-current illness, concomitant medications or other extenuating circumstances that, in the opinion of the Investigator, would either interfere with study participation or the interpretation of the effects of SBC-102. 2. Clinically significant abnormal values on laboratory screening tests, other than liver function or lipid panel tests. Subjects with an abnormal laboratory value that is of borderline significance may be allowed to undergo repeat testing once within a 30 day period. 3. Patient has participated in a study employing an investigational drug within 30 days of the screening 4. Child-Pugh Class C or AST and/or ALT persistently elevated > 3xULN at screening (2 or more occasions) 5. Previous hemopoietic bone marrow or liver transplant 6. Patient has received prior treatment with enzyme replacement therapy 7. Subject has a total score of 8 or more on a screening Alcohol Use Disorders Identification Test (AUDIT) 8. Patients with known hypersensitivity to eggs
    E.5 End points
    E.5.1Primary end point(s)
    The primary objective is to evaluate the safety and tolerability of SBC-102 in patients with liver dysfunction due to LAL deficiency.
    E.5.1.1Timepoint(s) of evaluation of this end point
    At study visits.
    E.5.2Secondary end point(s)
    The secondary objective is to characterise the pharmacokinetics of SBC-102 delivered by IV infusion after single and multiple doses [pre and post infusion Day 0 and 21].
    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 No
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    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) Yes
    E.7.1.1First administration to humans Yes
    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) No
    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 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 concerned1
    E.8.5The trial involves multiple Member States No
    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
    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
    Last visit of last subject.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years0
    E.8.9.1In the Member State concerned months4
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years0
    E.8.9.2In all countries concerned by the trial months4
    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.1.1Number of subjects for this age range: 0
    F.1.1.1In Utero No
    F.1.1.1.1Number of subjects for this age range: 0
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.2.1Number of subjects for this age range: 0
    F.1.1.3Newborns (0-27 days) No
    F.1.1.3.1Number of subjects for this age range: 0
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.4.1Number of subjects for this age range: 0
    F.1.1.5Children (2-11years) No
    F.1.1.5.1Number of subjects for this age range: 0
    F.1.1.6Adolescents (12-17 years) No
    F.1.1.6.1Number of subjects for this age range: 0
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 0
    F.1.3Elderly (>=65 years) No
    F.1.3.1Number of subjects for this age range: 0
    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 No
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception No
    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 9
    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)
    Given the lack of clinical experience with SBC-102, the investigational drug can only be administered as part of a clinical trial at this stage. Subjects may be eligible to resume SBC-102 under a separate protocol to assess the long-term safety and efficacy of SBC-102 in patients with LAL Deficiency/CESD phenotype. Initiation of the long-term extension study is contingent upon regulatory approval of the protocol after review of additional data including safety data from the current study.
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2011-03-08
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2011-02-21
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2012-01-06
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