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    Summary
    EudraCT Number:2016-002851-92
    Sponsor's Protocol Code Number:AN-EPI3334
    National Competent Authority:Hungary - National Institute of Pharmacy
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2016-07-25
    Trial 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 ConcernedHungary - National Institute of Pharmacy
    A.2EudraCT number2016-002851-92
    A.3Full title of the trial
    An Open-Label Study Evaluating the Efficacy and Safety of Liprotamase in Subjects with Exocrine Pancreatic Insufficiency due to Cystic Fibrosis
    A liprotamáz hatásosságát és biztonságosságát értékelő, nyílt vizsgálat, cisztás fibrózis okozta exokrin hasnyálmirigy elégtelenségű alanyoknál
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    NA
    NA
    A.3.2Name or abbreviated title of the trial where available
    EASY: Extended Access to Sollpura over Years
    NA
    A.4.1Sponsor's protocol code numberAN-EPI3334
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT02823964
    A.5.4Other Identifiers
    Name:BB-IND No.Number:062,037
    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 SponsorANTHERA 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 supportAnthera Pharmaceuticals, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAnthera Pharmaceuticals, Inc.
    B.5.2Functional name of contact pointNicole Ramza
    B.5.3 Address:
    B.5.3.1Street Address25801 Industrial Boulevard, Suite B
    B.5.3.2Town/ cityHayward, CA
    B.5.3.3Post code94545
    B.5.3.4CountryUnited States
    B.5.4Telephone number1510856-5607
    B.5.6E-mailnramza@anthera.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/3/04/222
    D.3 Description of the IMP
    D.3.1Product nameLiprotamase
    D.3.4Pharmaceutical form Capsule
    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 INNNOT ASSIGNED
    D.3.9.1CAS number 9001-62-1
    D.3.9.3Other descriptive nameLIPASE
    D.3.9.4EV Substance CodeSUB12545MIG
    D.3.10 Strength
    D.3.10.1Concentration unit IU international unit(s)
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number10000 to 40000
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNNOT ASSIGNED
    D.3.9.1CAS number 9000-99-1
    D.3.9.3Other descriptive namePROTEASE
    D.3.9.4EV Substance CodeSUB15036MIG
    D.3.10 Strength
    D.3.10.1Concentration unit IU international unit(s)
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number10000 to 40000
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNNOT ASSIGNED
    D.3.9.1CAS number 9000-90-2
    D.3.9.3Other descriptive nameAMYLASE
    D.3.9.4EV Substance CodeSUB12892MIG
    D.3.10 Strength
    D.3.10.1Concentration unit IU international unit(s)
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number1500 to 6000
    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 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
    Cystic Fibrosis-Related Exocrine Pancreatic Insufficiency
    Cisztás fibrózis okozta exokrin hasnyálmirigy elégtelenség
    E.1.1.1Medical condition in easily understood language
    Maldigestion of dietary macronutients (pancreas not producing enough enzymes for digestion of fat, sugars and proteins) in Cystic Fibrosis
    Tápanyag lebontási zavar (a hasnyálmirigy nem termel elegendő enzimet a zsírok, cukrok és fehérjék lebontásához) cisztás fibrózisban
    E.1.1.2Therapeutic area Diseases [C] - Immune System Diseases [C20]
    MedDRA Classification
    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 this study is to evaluate the safety of long-term use of liprotamase in the management of Cystic Fibrosis-Related Exocrine Pancreatic Insufficiency (EPI). Safety will be evaluated based on the occurrence of adverse events (AEs) and serious AEs (SAEs); including but not limited to abnormal laboratory results, abnormal findings in physical examinations and abnormal vital signs deemed clinically significant by the investigator.
    A vizsgálat elsődleges célja a liprotamáz hosszútávú használatának értékelése a cisztás fibrózis okozta exokrin hasnyálmirigy elégtelenség (EPI) kezelésében. A biztonságosság értékelése a nemkívánatos események (AE-k) és a súlyos nemkívánatos események (SAE-k) előfordulásán alapul; beleértve, de nem korlátozva az alábbiakra: rendellenes laboratóriumi eredmények, rendellenes fizikális vizsgálati eredmények, valamint a vizsgáló által klinikailag jelentősnek ítélt rendellenes vitális jelek.
    E.2.2Secondary objectives of the trial
    The secondary endpoints will include:
    • Change in weight, height, and BMI from baseline.
    • Changes in measures of growth including weight, height, and BMI relative to population anthropomorphic measures.
    • Signs and symptoms of malabsorption including stool frequency, stool consistency, bloating, steatorrhea, abdominal pain, and flatulence compared with baseline.
    A másodlagos végpontok az alábbiak:
    • A testsúly, testmagasság és BMI változása a kiinduló állapothoz képest.
    • A növekedés mértékének változása, beleértve a testsúlyt, testmagasságot, és BMI-t a népesség antropomorfikus értékeihez viszonyítva.
    • A felszívódási zavar jelei és tünetei, beleértve a széklet gyakoriságát, a széklet állagát, a puffadást, a zsírszékletet, a hasi fájdalmat és a felfúvódást, a kiinduló állapothoz viszonyítva.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Subjects are eligible for enrollment if they meet the following inclusion criteria:
    1. Male or female subjects who received liprotamase and completed Study AN-EPI3331.
    2. Signed informed consent by subject and/or subject’s legally authorized representative.
    Az alanyok akkor vonhatóak be a vizsgálatba, ha megfelelnek az alábbi bevonási feltételeknek:
    1. Férfi vagy nő beteg, aki liprotamázt kapott és befejezte az AN-EPI3331 vizsgálatot.
    2. Az alany vagy a nyilatkozattételre jogosult aláírta a beleegyező nyilatkozatot.
    E.4Principal exclusion criteria
    1. Any medical, psychological, or social condition that, at the discretion of the investigator, may put the subject at increased risk by participating in this study or confound interpretation of study results.
    2. Treatment with any PERT other than study drug, investigational drugs, or devices
    3. Females who are nursing, pregnant, intending to become pregnant, or intending to nurse during the time of the study, or who have a positive pregnancy test at Visit Day 1. All sexually-active subjects of reproductive potential are required to remain on a reliable method of birth control. Females and males are required to continue using a reliable method of birth control throughout the study, and for at least 3 months following completion of study therapy. A reliable method of birth control is defined as one of the following: oral or injectable contraceptives, intrauterine device, contraceptive implants, tubal ligation, hysterectomy, or a double-barrier method (diaphragm with spermicidal foam or jelly, or a condom), abstinence or vasectomy.
    1. Bármilyen egészségügyi, pszichológiai vagy szociális körülmény fennállása, amely a vizsgáló megítélése szerint komoly kockázatot jelenthet a beteg számára a vizsgálatban való részvétel esetén, vagy amely megzavarhatja a vizsgálati eredmények értelmezését.
    2. A vizsgálati készítményen kívül bármilyen egyéb enzimpótló készítmény, illetve más vizsgálati készítmény vagy vizsgálati eszköz használata.
    3. Azok a nők, akik szoptatnak, várandósak, vagy szoptatni szeretnének, illetve teherbe kívánnak esni a vizsgálat idején, vagy akiknek pozitív a terhességi tesztje az 1. napi viziten. Az összes szexuálisan aktív, fogamzóképes alanynak megbízható születésszabályozási módszert kell alkalmaznia. A nőknek és férfiaknak a megbízható születésszabályozási módszer alkalmazását folytatniuk kell végig a vizsgálat során, majd a vizsgálati terápia befejezését követően még legalább 3 hónapig. A megbízható születésszabályozási módszer az alábbiak egyikével határozható meg: szájon át szedett vagy injekcióban beadott fogamzásgátló, méhen belüli eszköz, beültetett fogamzásgátló, petevezeték elkötés, méheltávolítás vagy kettős-gát módszer (pesszárium spermicid habbal vagy zselével vagy óvszerrel), önmegtartóztatás vagy ondóvezeték elkötés.
    E.5 End points
    E.5.1Primary end point(s)
    The primary objective of this study is to evaluate the safety of long-term use of liprotamase in the management of EPI due to CF amongst subjects who received liprotamase and completed the prior study with liprotamase (AN-EPI3331). Safety will be evaluated based on adverse events (AEs) and serious AEs (SAEs); including but not limited to abnormal laboratory results, abnormal findings in physical examinations, and abnormal vital signs deemed clinically significant by the investigator.
    By-subject listings of all AEs, including abnormal and clinically significant vital sign parameters and physical examination results will be presented.
    Az elsődleges végpont elemzés a liprotamáz biztonságosságát értékeli olyan felnőtt és gyermek alanyok esetében, akik befejezték az AN-EPI3331 vizsgálatot. A biztonságosság értékelése a nemkívánatos eseményeken (AE-k) és a súlyos nemkívánatos eseményeken (SAE-k) alapul; beleértve, de nem korlátozva az alábbiakra: rendellenes laboratóriumi eredmények, rendellenes fizikális vizsgálati eredmények, valamint a vizsgáló által klinikailag jelentősnek ítélt rendellenes vitális jelek.
    Témánként kerülnek felsorolásra az AE-k, ide értve a a rendellenes és klinikailag jelentősnek ítélt vitális jeleket és a fizikális vizgálatok eredményeit.
    E.5.1.1Timepoint(s) of evaluation of this end point
    At each study Visit during the treatment period.
    Minden vizsgálati Viziten a kezelési periódus során.
    E.5.2Secondary end point(s)
    In this study efficacy will be evaluated from measures of body weight and BMI (for adults) and growth (for pediatric subjects) relative to baseline and/or relative to published population growth charts. In addition, efficacy (i.e. control of EPI) will be evaluated from the signs and symptoms of malabsorption. The secondary endpoints will include:
    • Change in weight, height, and BMI from baseline.
    • Changes in measures of growth including weight, height, and BMI relative to population anthropomorphic measures.
    • Signs and symptoms of malabsorption including stool frequency, stool consistency, bloating, steatorrhea, abdominal pain, and flatulence compared with baseline.
    For efficacy analyses of change from baseline, baseline will be defined as the last observation prior to initiation of liprotamase dosing.
    A vizsgálatban a hatékonyságot a testsúly-mérésekből, (felnőtteknél) BMI-ből, (gyermekeknél) a növekedés alapján értékelik, összehasonlítva a kiinduló állapottal és/vagy a publikált növekedési táblázatokkal. Ezen kívül a hatékonyságot (értsd: az EPI kontrollálása) a felszívódási zavar jelei és tünetei alapján értékelik. A másodlagos végpontok az alábbiak:
    • A testsúly, testmagasság és BMI változása a kiinduló állapothoz képest.
    • A növekedés mértékének változása, beleértve a testsúlyt, testmagasságot, és BMI-t a népesség antropomorfikus értékeihez viszonyítva.
    • A felszívódási zavar jelei és tünetei, beleértve a széklet gyakoriságát, a széklet állagát, a puffadást, a zsírszékletet, a hasi fájdalmat és a felfúvódást, a kiinduló állapothoz viszonyítva.
    A kiinduló állapottól mért hatékonysági analíziseknél a kiinduló állapot definíciója a liprotamáz adagolás előtti legutolsó megfigyelés.
    E.5.2.1Timepoint(s) of evaluation of this end point
    At each study Visit during the treatment period.
    Minden vizsgálati Viziten a kezelési periódus során.
    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) Yes
    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.8.3 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 concerned4
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA18
    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
    Israel
    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
    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 months
    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.1.1Number of subjects for this age range: 35
    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.1.1.5.1Number of subjects for this age range: 15
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 20
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 35
    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 minors, the parents will have to give their consent. The minors will also have to give their assent.
    Kiskorú esetében a szülő adja a belegyezést. A kiskorú hozzájárulását adja.
    F.3.3.7Others Information not present in EudraCT
    F.4 Planned number of subjects to be included
    F.4.1In the member state10
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 36
    F.4.2.2In the whole clinical trial 70
    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)
    The patient will return to original PERT therapy.
    A betegek visszatérnek a korábbi PERT kezelésre.
    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 Decision2016-09-08
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2016-08-31
    P. End of Trial
    P.End of Trial StatusOngoing
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