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    Summary
    EudraCT Number:2014-001367-13
    Sponsor's Protocol Code Number:IASO_MF
    National Competent Authority:Austria - BASG
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2014-07-07
    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 ConcernedAustria - BASG
    A.2EudraCT number2014-001367-13
    A.3Full title of the trial
    An open-label, prospective, single-centre, phase II study to assess dose and dose interval requirements with respect to efficacy and safety of AOP2014, PEG-Proline-Interferon alpha-2b, in patients with primary myelofibrosis (grade MF-0 and MF-1)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    The study to assess the efficacy and safety of new Interferon alpha-2b formulation in patients with primary myelofibrosis
    A.4.1Sponsor's protocol code numberIASO_MF
    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 SponsorMedizinische Universtät Wien, Universitätsklinik für Innere Medizin I
    B.1.3.4CountryAustria
    B.3.1 and B.3.2Status of the sponsorNon-Commercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportAOP ORPHAN PHARMACEUTICALS AG
    B.4.2CountryAustria
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAOP ORPHAN PHARMACEUTICALS AG
    B.5.2Functional name of contact pointMedical Information
    B.5.3 Address:
    B.5.3.1Street AddressWilhelminenstrasse 91/II f
    B.5.3.2Town/ cityVienna
    B.5.3.3Post code1160
    B.5.3.4CountryAustria
    B.5.6E-mailsanda.sturlan@aoporphan.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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product namePegylated proline-interferon alpha- 2b
    D.3.2Product code AOP2014
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNPEGylated Proline - interferon alpha - 2b recombinant
    D.3.9.1CAS number 1335098-50-4
    D.3.9.2Current sponsor codeAOP2014
    D.3.9.3Other descriptive namePEGINTERFERON ALFA-2B
    D.3.9.4EV Substance CodeSUB12549MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number0.5
    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
    Primary Myelofibrosis (grade MF-0 and MF-1) according to the WHO criteria
    E.1.1.1Medical condition in easily understood language
    Primary myelofibrosis, disease in which abnormal blood cells and fibers build up inside the bone marrow.
    E.1.1.2Therapeutic area Diseases [C] - Blood and lymphatic diseases [C15]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 17.0
    E.1.2Level LLT
    E.1.2Classification code 10053134
    E.1.2Term Osteomyelofibrosis
    E.1.2System Organ Class 100000004864
    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 efficacy of novel Pegylated-Pro Interferon α-2b formulation in terms of response rate in both, Interferon naive and pretreated patients with early primary myelofibrosis (MF-0 to MF-1).
    The favourable pharmacokinetic profile of AOP2014 suggests possible dose reduction and/or treatment interval prolongation options, which, at preserved efficacy, may improve the tolerability of IFN α treatment.
    E.2.2Secondary objectives of the trial
    To evaluate quality of life, frequency of disease related events, confirmatory surrogate response parameter, safety observations and dose response correlations.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Written informed consent obtained prior to any study specific screening activities and able to comply with this protocol.
    2. Patients age ≥18 years
    3. Confirmed diagnosis of primary myelofibrosis (grade MF-0 and MF-1) according to the WHO criteria (2008). Including either
    newly diagnosed Interferon naive patients or patients pre-treated with Pegasys® or PegIntron®, who had stable disease for
    the previous 3 months.
    4. If female of childbearing potential – have a negative urine pregnancy test result within 7 days prior to the scheduled first
    application of investigational product and agree to employ adequate birth control measures for the duration of the study.
    5. Eastern Cooperative Oncology Group performance status ≤ 2.

    E.4Principal exclusion criteria
    1. Diagnosis of any other myeloproliferative disorder
    2. Any clinically significant illness or surgery within 4 weeks prior to dosing
    3. Systemic infections, e.g. hepatitis B, hepatitis C, or HIV at screening
    4. Uncontrolled hypertension (systolic > 150 mmHg and diastolic > 100 mmHg, or clinically significant (i.e. active)
    cardiovascular disease: CVA/stroke (≤ 3 months prior to enrolment), myocardial infarction (≤ 3 months prior to enrolment),
    significant coronary artery stenosis, unstable angina, New York Heart Association (NYHA) Class 2 or greater Congestive
    heart failure, or serious cardiac arrhythmia requiring medication.
    5. History of malignant disease, including solid tumours (except basal cell and squamous cell carcinomas of the skin and
    carcinoma in situ of the cervix that have been completely excised and are considered cured) and haematological
    malignancies within the last 3 years
    6. History of severe allergic (like anaphylaxis) or hypersensitivity reactions (like angioedema), any known or suspected
    intolerance to the investigational product
    7. Use of any investigational drug or participation in any investigational drug study within the last 4 weeks
    8. Clinically significant history or known presence of psychiatric disorders, including but not limited to depression, anxiety
    and sleep disorders
    9. HADS score equal to or above 11 on either or both of the subscales (Appendix 3)
    10. Any risk of suicide at screening or previous suicide attempts
    11. Organ transplant, past or planned
    12. Inadequate liver function defined by serum (total) bilirubin > 2,5 x ULN and/or AST and ALT > 2,5 x ULN
    13. Clinically significant ECG findings
    14. History of renal disease requiring haemodialysis or seizure disorder requiring anticonvulsant therapy
    15. Pregnant or lactating females (pregnancy test in fertile women to be assessed within 7 days prior to study treatment start)
    16. Acute or chronic infections or autoimmune diseases (collagen diseases, polyarthritis, immune thrombocythemia, thyroiditis,
    psoriasis, lupus nephritis or any other autoimmune disorder)
    17. Advanced primary Myelofibrosis (> Grade 2)
    18. Clinically relevant pulmonary infiltrates, pneumonia, and pneumonitis at screening
    19. Any significant morbidity or abnormality which may interfere with the study participation
    20. History of active substance or alcohol abuse within the last year
    21. Evidence of severe retinopathy (e.g. cytomegalovirus retinitis, macular degeneration) or clinically relevant ophthalmological
    disorder (due to diabetes mellitus or hypertension)
    22. Thyroid dysfunction not adequately controlled
    23. Patients tested positively with TgAb and / or TPOAb at screening
    24. History of uncontrolled severe seizure disorder
    25. Leukocytopenia at the time of screening
    26. Thrombocytopenia at the time of screening
    E.5 End points
    E.5.1Primary end point(s)
    • Haematological response rate according to IWG-MRT criteria1 (platelets ≥100 x 109/l and < UNL (upper normal limit)
    neutrophils ≥1 x 109/l and < UNL, < 2% immature myeloid cells
    • LDH serum level ≤ 300 IU/L (all lab parameters measured by blinded central lab)
    • Spleen size vs. baseline (preferably volumetric measurement by Sono or MRI)
    E.5.1.1Timepoint(s) of evaluation of this end point
    after 6 months of the treatment
    E.5.2Secondary end point(s)
    • Quality of life Assessment through EORTC QLQ-C30 and MPN-SAF TSS questionnaires
    • Number of Disease related signs and symptoms within vs 6 month pre-treatment period
    • Maintenance or improvement of Molecular Response analysed in peripheral blood granulocytes.
    CR: Eradication of pre-existing abnormality. PR: ≥50% decrease in allele burden (partial response applies only to patients
    with at least 20% mutant allele burden et baseline)
    • Bone marrow histology if routinely available
    • Monitoring of Surrogate Response Parameters such as inflammation parameters
    • Maintenance of prognosis evolution assessed according to DIPSS
    E.5.2.1Timepoint(s) of evaluation of this end point
    after 6 months of the treatment
    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) 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.8.3 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 No
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA No
    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 years1
    E.8.9.1In the Member State concerned months6
    E.8.9.1In the Member State concerned 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: 20
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 4
    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 state24
    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 patients having benefit from AOP2014 treatment at the end of the 6 months core phase of the study will enter the long term extension study to collect additional relevant efficacy and safety data.
    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 Decision2014-08-08
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2014-07-30
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2019-04-24
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