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    Summary
    EudraCT Number:2014-004265-25
    Sponsor's Protocol Code Number:2-STEP
    National Competent Authority:Netherlands - Competent Authority
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2015-01-19
    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 ConcernedNetherlands - Competent Authority
    A.2EudraCT number2014-004265-25
    A.3Full title of the trial
    2-STEP: A single-centre, phase II study to evaluate the safety, tolerability and pharmacokinetics of 2-Iminobiotin (2-IB) in neonates with gestational age of ≥36 weeks with moderate to severe perinatal asphyxia treated with therapeutic hypothermia
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study looking at 2-Iminobiotin as treatment for newborn children suffering from insufficient oxygen supply to the brain in addition to standard treatment with cooling.
    A.4.1Sponsor's protocol code number2-STEP
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/035/2012
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorUniversity Medical Centre Utrecht
    B.1.3.4CountryNetherlands
    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 supportNeurophyxia B.V.
    B.4.2CountryNetherlands
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationUniversity Medical Centre Utrecht
    B.5.2Functional name of contact pointDepartment of Neonatoloy
    B.5.3 Address:
    B.5.3.1Street AddressLundlaan 6
    B.5.3.2Town/ cityUtrecht
    B.5.3.3Post code3584 EA
    B.5.3.4CountryNetherlands
    B.5.4Telephone number31887554544
    B.5.6E-mailf.groenendaal@umcutrecht.nl
    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/3/098/701
    D.3 Description of the IMP
    D.3.1Product name2-Iminobiotin
    D.3.2Product code NEU-01
    D.3.4Pharmaceutical form Solution for infusion
    D.3.4.1Specific paediatric formulation Yes
    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 INN2-Iminobiotin
    D.3.9.1CAS number 13395-35-2
    D.3.9.2Current sponsor codeNEU-01
    D.3.9.3Other descriptive name2-Iminobiotin
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number0.75
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    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
    Perinatal asphyxia
    E.1.1.1Medical condition in easily understood language
    Oxygen shortage during birth resulting in damage to the brain
    E.1.1.2Therapeutic area Diseases [C] - Nervous System Diseases [C10]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 19.0
    E.1.2Level LLT
    E.1.2Classification code 10003500
    E.1.2Term Asphyxia neonatal
    E.1.2System Organ Class 100000004855
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 19.0
    E.1.2Level LLT
    E.1.2Classification code 10004943
    E.1.2Term Birth asphyxia
    E.1.2System Organ Class 100000004855
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To explore the short term safety and tolerability of 2-IB and the pharmacokinetic profile of 2-IB when given on top of therapeutic hypothermia
    E.2.2Secondary objectives of the trial
    •To gather preliminary signs of short term efficacy as defined by the Lac/NAA ratios using MRS at 3-7 days after birth and the percentage of surviving patients with a normal aEEG at 60h after birth.

    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Neonates with ≥ 36 and <44 weeks gestation who are eligible to receive therapeutic hypothermia.
    2. Ability to start treatment within 12 hours after birth.
    E.4Principal exclusion criteria
    1. Inability to insert an indwelling catheter (umbilical venous catheter or percutaneously inserted central catheter, preferably multiple lumen) for administration of the drug or an arterial line for recurrent blood sampling.
    2. Major congenital malformations, specifically malformations that may affect the renal function.
    E.5 End points
    E.5.1Primary end point(s)
    Safety assessments include: vital signs, clinical laboratory parameters, clinical evaluation and (severe) adverse events and local tolerance.

    Pharmacokinetic assessment: a maximum of five PK samples will be taken. The exact time points to determined before start of the study based on all data available at that moment.
    The following pharmacokinetic parameters will be calculated for each patient, using the actual sampling times:
    • C-max (observed maximum plasma concentration)
    • AUC-0-6h (area under the plasma concentration-time curve from time 0 to 6h after administration)
    • AUC-0-∞ (area under the plasma concentration-time curve from time 0 to infinity)
    • T-end of infusion (time at maximum plasma concentration).
    •t1/2 (terminal elimination half-life)
    •CL (clearance)
    •Vd (volume of distribution)
    E.5.1.1Timepoint(s) of evaluation of this end point
    Safety assessments will be recorded during treatment period of 2-iminobiotin and will continue for at least 96 hours or until discharge from the hospital

    Blood sampling for pharmacokinetic assessment will occur during the treatment period of 2-iminobiotin and shortly thereafter
    E.5.2Secondary end point(s)
    •To gather preliminary signs of short term efficacy as defined by the Lac/NAA ratios using MRS at 3-7 days after birth and the percentage of surviving patients with a normal aEEG at 60h after birth.
    E.5.2.1Timepoint(s) of evaluation of this end point
    MRI(incl DWI):3-7 days after birth.
    aEEG: will be recorded before start treatment till at least 72 hours after birth.
    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 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 Information not present in EudraCT
    E.8.1.2Open Information not present in EudraCT
    E.8.1.3Single blind Information not present in EudraCT
    E.8.1.4Double blind Information not present in EudraCT
    E.8.1.5Parallel group Information not present in EudraCT
    E.8.1.6Cross over Information not present in EudraCT
    E.8.1.7Other Information not present in EudraCT
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) Information not present in EudraCT
    E.8.2.2Placebo Information not present in EudraCT
    E.8.2.3Other Information not present in EudraCT
    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 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
    LVLS, or earlier if subject has dropped out or died.
    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 months0
    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 Yes
    F.1.1Number of subjects for this age range: 12
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) Yes
    F.1.1.2.1Number of subjects for this age range: 2
    F.1.1.3Newborns (0-27 days) Yes
    F.1.1.3.1Number of subjects for this age range: 10
    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) 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.3.3.6.1Details of subjects incapable of giving consent
    Neonates of ≥36 and<44 weeks gestation
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state12
    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)
    no
    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 Decision2015-01-19
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-04-15
    P. End of Trial
    P.End of Trial StatusCompleted
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