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    Summary
    EudraCT Number:2015-003063-12
    Sponsor's Protocol Code Number:NEU-01-02-1A
    Clinical Trial Type:Outside EU/EEA
    Date on which this record was first entered in the EudraCT database:2016-09-14
    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
    H.4 THIRD COUNTRY IN WHICH THE TRIAL WAS FIRST AUTHORISED
    Expand All   Collapse All
    A. Protocol Information
    A.2EudraCT number2015-003063-12
    A.3Full title of the trial
    TIBC study:
    A multiple-centre Phase 2 trial comparing the efficacy of 2-Iminobiotin (2-IB) treatment to standard care in neonates with gestational age of ≥36 weeks with moderate to severe perinatal asphyxia in DR Congo.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    2-Iminobiotin vas a treatment for neonates born with severe oxygen shortage at birth
    A.3.2Name or abbreviated title of the trial where available
    TIBC
    A.4.1Sponsor's protocol code numberNEU-01-02-1A
    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 SponsorNeurophyxia B.V.
    B.1.3.4CountryNetherlands
    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 supportNeurophyxia B.V.
    B.4.2CountryNetherlands
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationNeurophyxia B.V.
    B.5.2Functional name of contact pointVP Regulatory Affairs
    B.5.3 Address:
    B.5.3.1Street AddressOnderwijsboulevard 225
    B.5.3.2Town/ city's Hertogenbosch
    B.5.3.3Post code5223 DE
    B.5.3.4CountryNetherlands
    B.5.4Telephone number+31629056631
    B.5.6E-mailinfo@neurophyxia.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/3/09/701
    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 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 INN2-Iminobiotin
    D.3.9.1CAS number 13395-35-2
    D.3.9.2Current sponsor codeNEU 01
    D.3.9.4EV Substance CodeSUB51281
    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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboSolution for infusion
    D.8.4Route of administration of the placeboIntravenous use
    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 at 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 10038738 - Respiratory, thoracic and mediastinal disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 19.0
    E.1.2Level PT
    E.1.2Classification code 10028923
    E.1.2Term Neonatal asphyxia
    E.1.2System Organ Class 10038738 - Respiratory, thoracic and mediastinal disorders
    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 10038738 - Respiratory, thoracic and mediastinal disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 19.0
    E.1.2Level HLT
    E.1.2Classification code 10028946
    E.1.2Term Neonatal hypoxia and asphyxia
    E.1.2System Organ Class 100000004868
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 19.0
    E.1.2Level LLT
    E.1.2Classification code 10021147
    E.1.2Term Hypoxic encephalopathy
    E.1.2System Organ Class 100000004852
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 19.0
    E.1.2Level LLT
    E.1.2Classification code 10022820
    E.1.2Term Intrauterine hypoxia and birth asphyxia
    E.1.2System Organ Class 10038738 - Respiratory, thoracic and mediastinal disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 19.0
    E.1.2Level LLT
    E.1.2Classification code 10040441
    E.1.2Term Severe birth asphyxia
    E.1.2System Organ Class 10038738 - Respiratory, thoracic and mediastinal disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 19.0
    E.1.2Level LLT
    E.1.2Classification code 10070512
    E.1.2Term Hypoxic-ischemic encephalopathy
    E.1.2System Organ Class 100000004852
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    This study will consist of two parts, an initial open label pilot part with as main objective to study the pharmacokinetic profile of 2-Iminobiotin (2-IB), followed by a randomised part with as main objective to study the efficacy of 2-IB in a population of new-borns with perinatal asphyxia.
    E.2.2Secondary objectives of the trial
    In both the pilot part and the randomised part the safety profile will be evaluated.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Neonates with ≥ 36 weeks gestation with moderate to severe perinatal asphyxia with a Thompson score ≥ 7 within 6h after birth
    Ability to start treatment within 6 hours after birth
    2.Ability to start treatment within 6 hours after birth
    E.4Principal exclusion criteria
    1. Inability to insert an umbilical venous catheter for administration of the study drug.
    2. Major congenital or chromosomal abnormalities, such as hernia diaphragmatica, omphalocele, trisomy 13 or trisomy 18
    3. Birth weight < 1800 grams
    4. Clear signs of infection
    5. Moribund patients
    E.5 End points
    E.5.1Primary end point(s)
    Pilot part:
    In an open label pilot part the pharmacokinetic and safety profile will be studied. Results will be used to decide on adjustment of the dose in the randomised part of the study.

    Randomised part:
    The short term efficacy will be evaluated as determined by the number of surviving patients with a normal aEEG (continuous normal voltage (CNV)) at 36h after birth.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Pilot Phase: Pharmacokinetic profile will be determined at 5 time points:directly after completion of the first infusion, just before the second and last infusion, and 1h and 4h after the last infusion.
    Randomised phase: aEEG and mortalitry will be evaluated at 36h after birth.
    E.5.2Secondary end point(s)
    In addition to the main efficacy end point described above: several other parameters will be evaluated, including Thompson score, aEEG at other time points, presence of seizures, neuronal markers and neonatal mortality.
    For evaluation of safety vital signs, need for intervention, basic biochemistry, renal function and (serious) adverse events will be monitored.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Efficacy end-points: aEEG, Thompson score, neuronal biomarkers at selected time points during first 72 hours after birth; seizures and mortality during first 7 days after birth.
    Safety end-points: vital signs, blood biochemistry, renal function and need for intervention will be monitored at selected time points within 48 hours after birth; (Serious) Adverse Events will monitored during stay at the NICU for up to a maximum of 7 days.
    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 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 Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other Yes
    E.8.1.7.1Other trial design description
    Randomised phase will be preceded by open lable pilot phase with 8 patients.
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    E.8.3 Will this trial be conducted at a single site globally? No
    E.8.4 Will this trial be conducted at multiple sites globally? Yes
    E.8.6 Trial involving sites outside the EEA
    E.8.6.2Trial being conducted completely outside of the EEA Yes
    E.8.6.3Specify the countries outside of the EEA in which trial sites are planned
    Congo, The Democratic Republic of the
    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
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months6
    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: 94
    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: 10
    F.1.1.3Newborns (0-27 days) Yes
    F.1.1.3.1Number of subjects for this age range: 84
    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 No
    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
    The trial population consists neonates with gestational age of ≥ 36 weeks with moderate to severe perinatal asphyxia. Treatment should start within 6 hours after birth.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.2 For a multinational trial
    F.4.2.2In the whole clinical trial 94
    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)
    It is planned that the the patients in the randomised phase will be asked to enter a follow-up study to evaluate the neurological development in the first 2 years after birth. This will be a separate study.
    G. Investigator Networks to be involved in the Trial
    H.4 Third Country in which the Trial was first authorised
    H.4.1Third Country in which the trial was first authorised: Congo, The Democratic Republic of the
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