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    The EU Clinical Trials Register currently displays   43839   clinical trials with a EudraCT protocol, of which   7280   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

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    EudraCT Number:2014-000533-22
    Sponsor's Protocol Code Number:LAL-CL08
    National Competent Authority:Finland - Fimea
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2015-06-05
    Trial results View results
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    A. Protocol Information
    A.1Member State ConcernedFinland - Fimea
    A.2EudraCT number2014-000533-22
    A.3Full title of the trial
    A Phase 2, Open Label, Multicenter Study to Evaluate the Safety, Tolerability, Efficacy, and Pharmacokinetics of Sebelipase Alfa in Infants with Rapidly Progressive Lysosomal Acid Lipase Deficiency
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study of sebelipase alfa in young children with Lysosomal Acid Lipase Deficiency (LALD).
    A.4.1Sponsor's protocol code numberLAL-CL08
    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 SponsorAlexion Pharmaceuticals 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 supportAlexion Pharmaceuticals Inc
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAlexion Pharmaceuticals Inc
    B.5.2Functional name of contact pointHarold Pestana,Assoc Dir Clin Ops
    B.5.3 Address:
    B.5.3.1Street Address33 Hayden Avenue
    B.5.3.2Town/ cityLexington, MA
    B.5.3.3Post code02421
    B.5.3.4CountryUnited States
    B.5.4Telephone number17813579900
    B.5.5Fax number17813579901
    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/3/10/827
    D.3 Description of the IMP
    D.3.1Product nameSebelipase Alfa
    D.3.2Product code SBC-102
    D.3.4Pharmaceutical form Concentrate for solution for injection/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 INNsebelipase alfa
    D.3.9.1CAS number 1276027-63-4
    D.3.9.2Current sponsor codeSBC-102
    D.3.9.3Other descriptive namelysosomal acid lipase, Esterase, cholesterol (human gene LIPA), Lysosomal acid lipase (human gene LIPA); USAN: sebelipase alpha
    D.3.9.4EV Substance CodeSUB121647
    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. cell therapy medicinal product No
    D. therapy medical product No
    D. Engineered Product No
    D. ATIMP (i.e. one involving a medical device) No
    D. 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
    Lysosomal Acid Lipase Deficiency (LALD)
    E.1.1.1Medical condition in easily understood language
    Insufficient lysosomal acid lipase activity leading to accumulation of fats in the human body.
    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 19.1
    E.1.2Level HLT
    E.1.2Classification code 10024579
    E.1.2Term Lysosomal storage disorders
    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
    The primary objective of the study is to evaluate the safety and tolerability of sebelipase alfa in infants with rapidly progressive LAL Deficiency.
    E.2.2Secondary objectives of the trial
    The secondary objectives are to evaluate the effect of sebelipase alfa
    therapy on: (1) Survival at 12 months of age (2) Survival past 12 months of age; (3) Growth parameters; (4) Hepatomegaly, splenomegaly, and liver function; (5) Hematological parameters; and (6) to characterize the PK of sebelipase alfa delivered by intravenous (IV) infusion.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    A subject must meet all of the following criteria to be eligible to participate in
    this study:
    1. Subject’s parent or legal guardian (if applicable) consents to participation in the study.
    2.Confirmation of documented decreased LAL activity relative to the normal range of the lab performing the assay or a documented result of molecular genetic testing confirming a diagnosis of LAL Deficiency
    3. Substantial clinical concerns, in the opinion of Investigator and Sponsor,
    of rapid disease progression requiring urgent medical intervention
    including, but not restricted to, the following:
    a) Marked abdominal distension and hepatomegaly
    b) Failure to thrive as evidenced by:
    i) Weight for height is 2 or more SD below the mean for gender and age
    ii) Weight curve has crossed downward by more than 2 major percentile lines on the WHO growth curves (1st, 3rd, 5th, 10th,
    25th, 50th, 75th, 90th, 95th, 97th, 99th) after having previously achieved a stable pattern of growth
    c) Disturbance of coagulation (e.g., requirement for FFP; two values of PT >15 sec or PTT > 40 sec)
    d) Severe anemia (e.g., requirement for blood transfusion or hemoglobin < 8 g/dL)
    e) Sibling with rapidly progressive course of LAL Deficiency
    E.4Principal exclusion criteria
    A subject who meets any of the following criteria will be ineligible to
    participate in this study:
    1. Clinically important concurrent disease or co-morbidities which, in the opinion of the Investigator and Sponsor, would interfere with study participation, including, but not restricted to:
    a) Additional severe congenital abnormality
    b) Presence of severe infection that requires treatment with parenteral anti-infective treatment within the past 14 days
    c) Previous history of circulatory collapse requiring inotropic support for more than 48 hours
    d) Congestive heart failure
    e) Acute or chronic renal failure
    f) Other extenuating circumstances such as life-threatening under nutrition or rapidly progressive liver disease
    2. Subject will be > 8 months of age at the time of first dosing.
    3. Subject has received an investigational medicinal product other than
    sebelipase alfa within 14 days prior to the first dose of sebelipase alfa in
    this study.
    4. Myeloablative preparation, or other systemic pre-transplant conditioning, for hematopoietic stem cell or liver transplantation.
    5. Previous hematopoietic stem cell or liver transplant.
    6. Known hypersensitivity to eggs.
    E.5 End points
    E.5.1Primary end point(s)
    Safety endpoints will include the incidence of adverse events (AEs), serious adverse events (SAEs), and IRRs; changes from baseline clinical laboratory tests; changes in vital signs (blood pressure, heart rate, respiratory rate, and temperature) during and post-infusion relative to pre-infusion values; physical examination findings; use of concomitant medications/therapies; and characterization of ADAs, including seroconversion rate, time to eroconversion, median and peak immunoglobulin G (IgG) ADA titer, time to peak IgG ADA titer, and tolerization. The impact of ADAs on safety endpoints will also be explored. Further characterization of ADAs, including inhibitory and/or neutralizing ADAs and measurement of specific ADA subtypes (e.g., IgE), may be performed, if appropriate.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Screening will occur within 21 days.Eligible patients will receive intravenous infusions of sebelipase alfa for up to 3 years. All patients will receive a starting dose of 1 mg·kg-1 qow. Dose increases (up to 3 mg·kg-1 qw) will be permitted during the treatment period. The follow-up period will be a minimum of 30 days following the last administration of sebelipase alfa.
    E.5.2Secondary end point(s)
    Survival endpoints will include proportion of subjects surviving to 12, 18 and 24 months of age, and other timepoints, as data permit, and estimated median age at death. Survival rates and median age at death derived from this study will be compared to rates/times reported in the literature and previous studies. Other efficacy endpoints will include changes from baseline in percentiles and/or z-scores for WFA, WFL/WFH, and LFA/HFA and the corresponding growth status indicators of underweight, wasting, and stunting, as well as changes from baseline in z-scores for head circumference-for-age (HCFA) and mid-upper arm circumference-for-age (MUACFA); changes from baseline in aspartate aminotransferase (AST) and ALT; normalization of hemoglobin levels without requirement for blood transfusion; and change from baseline in serum ferritin.
    Exploratory efficacy endpoints will include changes and/or percent changes from baseline (or first available measurement if baseline data are unavailable) in alkaline phosphatase, gamma glutamyltransferase (GGT), albumin, and bilirubin (direct, indirect, and total); liver and spleen size/volume, as measured by ultrasound or magnetic resonance imaging (MRI); hepatomegaly and/or splenomegaly (abdominal girth, liver size, and spleen size) by physical examination; platelet levels; and serum lipid levels (total cholesterol, triglycerides, high density lipoprotein [HDL], low density lipoprotein [LDL]). Development will also be assessed using Denver II developmental screening test total score and four functional area scores (fine motor-adaptive, gross motor, personal-social, and language skills).
    The impact of anti-sebelipase alfa antibodies (ADAs) on efficacy endpoints will also be explored.
    The PK endpoint will include maximum observed serum concentration (Cmax), as data permit. The impact of ADAs on SBC-sebelipase alfa PK will also be explored.
    Exploratory disease-related biomarkers, which may be identified based on emerging information from the sebelipase alfa development program and scientific literature, will be analyzed by changes or percent changes from baseline.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Screening will occur within 21 days.Eligible patients will receive intravenous infusions of sebelipase alfa for up to 3 years. All patients will receive a starting dose of 1 mg·kg-1 qow. Dose increases (up to 3 mg·kg-1 qw) will be permitted during the treatment period. The follow-up period will be a minimum of 30 days following the last administration of sebelipase alfa.
    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 Yes
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    E.6.10Pharmacogenetic Yes
    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. 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 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 trial1
    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 EEA1
    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
    United Kingdom
    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
    The LVLS will be a follow-up at least 30 days after the last dose of study drug
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    E.8.9.1In the Member State concerned months7
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months7
    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) Yes
    F. of subjects for this age range: 2
    F.1.1.4Infants and toddlers (28 days-23 months) Yes
    F. of subjects for this age range: 8
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) No
    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 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 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 Yes
    F. of subjects incapable of giving consent
    Eligible patients under 8 months of age will be enrolled into the study. Patient’s parent or legal guardian consents to participation in the study.
    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 5
    F.4.2.2In the whole clinical trial 10
    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)
    There is no approved treatment for the condition. The subjects will continue having their disease managed as agreed with their treating physicians or may be eligible for access to sebelipase alfa through extension of a clinical trial, marketed product, or expanded access/compassionate access to IMP.
    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 Decision2015-06-09
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-06-23
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2018-10-30
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