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    Summary
    EudraCT Number:2019-002282-35
    Sponsor's Protocol Code Number:rh-NGF
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2021-05-24
    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 ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2019-002282-35
    A.3Full title of the trial
    Nerve growth factor in pediatric severe traumatic brain injury : translational and clinical studies on a candidate biomarker and therapeutic drug
    Fattore di crescita neuronale nel trauma cranico grave pediatrico: studi traslazionali e clinici su un candidato biomarker e farmacologico
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Nerve growth factor in pediatric severe traumatic brain injury : translational and clinical studies on a candidate biomarker and therapeutic drug
    Fattore di crescita neuronale nel trauma cranico grave pediatrico: studi traslazionali e clinici su un candidato biomarker e farmacologico
    A.3.2Name or abbreviated title of the trial where available
    Nerve growh factor in children with traumatic brain injury
    Fattori di crescita nervoso nei bambini con trauma cranico
    A.4.1Sponsor's protocol code numberrh-NGF
    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 SponsorFONDAZIONE POLICLINICO UNIVERSITARIO AGOSTINO GEMELLI IRCCS UNIVERSITA' CATTOLICA DEL SACRO CUORE
    B.1.3.4CountryItaly
    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 supportMinistero della Salute Ricerca Finalizzata
    B.4.2CountryItaly
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationDirezione scientifica Fondazione Policlinico A.Gemelli IRCCS
    B.5.2Functional name of contact pointDirezione Scientifica Fondazione Po
    B.5.3 Address:
    B.5.3.1Street AddressLargo Agostino Gemelli 8
    B.5.3.2Town/ cityRoma
    B.5.3.3Post code00168
    B.5.3.4CountryItaly
    B.5.4Telephone number0630155701
    B.5.6E-maildirezione.scientifica@policlinicogemelli.it
    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.2.1.1.1Trade name OXERVATE
    D.2.1.1.2Name of the Marketing Authorisation holderDompé farmaceutici S.p.A. (Dompé)
    D.2.1.2Country which granted the Marketing AuthorisationItaly
    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 nameoxervate
    D.3.2Product code [rh-NGF]
    D.3.4Pharmaceutical form Eye drops, solution
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntranasal use (Noncurrent)
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNCENEGERMIN
    D.3.9.1CAS number 1772578-74-1
    D.3.9.2Current sponsor coderh-NGF
    D.3.9.3Other descriptive namerh-NGF
    D.3.10 Strength
    D.3.10.1Concentration unit ng nanogram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number50
    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 Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    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
    Persistent unresponsive wakefulness syndrome (UWS)
    Danni neurologici gravi secondari a trauma cranico (UWS)
    E.1.1.1Medical condition in easily understood language
    Persistent unresponsive wakefulness syndrome (UWS)
    Danni neurologici gravi secondari a trauma cranico (UWS)
    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 20.1
    E.1.2Level LLT
    E.1.2Classification code 10049615
    E.1.2Term Late effects of head trauma
    E.1.2System Organ Class 100000004863
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    - Primary aim: evidence of changes in clinical and neurological conditions in children with severe neurological impairment caused by traumatic brain injury after treatment with intranasal NGF by mucosal atomized device (MAD)
    Evidenza di modifiche delle condizioni cliniche e neuroradiologiche in bambini con danno neurologico grave secondario al trauma cranico, in seguito al trattamento con NGF intranasale somministrato tramite MAD
    E.2.2Secondary objectives of the trial
    - Secondary aim: safety and tolerability of the treatment with intranasal NGF in children; changes in the quality of life measured by the pediatric quality of life inventory
    Sicurezza e tollerabilità del trattamento con NGF intranasale nei bambini; cambiamenti nella qualità della vita misurati tramite il test della qualità della vita pediatrica
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Children (aged between 6 months and 5 years) who satisfy all the following criteria:
    - specific brain lesions, such as Diffuse Axonal injury (DAI) or other brain lesions secondary to traumatic brain injuries, as documented by brain MRI or brain CT scan
    - severe cognitive, motor and neurosensory deficits resulting from traumatic brain lesions, as measured by the sensorimotor score “ASIA-IMSOP Classification”
    - all neurological deficits have to be stabilized, unresponsive to any treatment in place and have a clinical, neuroradiological and instrumental documentation that supports the diagnosis of UWS. In any case, the neurological deficit must be characterized by severe neurosensory, cognitive and motor deficits. We will use the sensorimotor scale ASIA-IMSOP; EEG recordings; Functional rating performed with SCIM (Spinal Cord Independence Measure), Version III
    - written informed consent signed by the parents or by legally appointed guardians.
    - Bambini di età compresa tra 6 mesi e 5 anni
    - gravi deficit cognitivi, motori e neurosensoriali derivanti da lesioni cerebrali traumatiche
    - i deficit neurologici devono essere stabilizzati, non rispondenti a qualsiasi trattamento in atto e avere una documentazione clinica, neuroradiologica e strumentale che supporti la diagnosi di UWS
    - aver ottenuto il consenso informato scritto da parte dei genitori, del Comitato Etico e dell’AIFA
    E.4Principal exclusion criteria
    - Intraparenchymal hemorrhages
    - intraventricular hemorrhages
    - subdural hematomas
    - epidural hematomas.
    - Emorragie intraparenchimali
    - Emorragie intraventricolari
    - Ematomi subdurali
    - Ematomi epidurali
    E.5 End points
    E.5.1Primary end point(s)
    - Improvement of clinical and neurological conditions of treated children. Neuro-radiological ameliorations, documented by MRI, of brain lesions. Changes in brain metabolism, testified by cerebral PET, and in cerebral perfusion, documented by brain SPECT.
    - Improvement of neuro-sensorial potentials, such as visual evoked potentials and sensory-motor potentials.
    - Significant differences of these results in terms of primary outcomes that may be highlighted with the timing of the execution of the therapy in children with severe TBI.
    - Proteomics and gene expression tools will also be used to analyze selected biomarkers on serum samples from enrolled patients, to be correlated with these clinical endpoints
    Miglioramento delle condizioni cliniche e neurologiche dei bambini trattati. Miglioramenti neuroradiologici, documentati dalla RM, delle lesioni cerebrali. Cambiamenti nel metabolismo cerebrale, testimoniato da PET cerebrale, e nella perfusione cerebrale, documentato da SPECT cerebrale.
    - Miglioramento dei potenziali neurosensoriali, quali potenziali visivi evocati e potenziali sensoriali-motori.
    - Differenze significative di questi risultati in termini di esiti primari che possono essere evidenziate con i tempi di esecuzione della terapia nei bambini con grave TBI.
    - Proteomica e strumenti di espressione genica saranno utilizzati anche per analizzare i biomarcatori selezionati su campioni di siero di pazienti arruolati, da correlare con questi endpoint clinici
    E.5.1.1Timepoint(s) of evaluation of this end point
    36 month
    36 mesi
    E.5.2Secondary end point(s)
    safety and tolerability of the treatment with intranasal NGF in children
    -modifications of the amplitude and phase of neurosensorial potentials, such as VEPs and ERG, after treatment with intranasal NGF
    - changes in the quality of life measured by the pediatric quality of life inventory (PedsQL)
    Sicurezza e tollerabilità del trattamento con NGF intranasale
    Modifiche dell'ampiezza e della fase di potenziali neurosensoriali, quali PEV e ERG, dopo il trattamento con NGF intranasale
    Modifiche della qualità di vita misurate tramite la scala PedsQL
    E.5.2.1Timepoint(s) of evaluation of this end point
    36 months
    36 mesi
    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 No
    E.6.4Safety No
    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 No
    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.8.4.1Number of sites anticipated in Member State concerned1
    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 Information not present in EudraCT
    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
    - Improvement of clinical and neurological conditions of treated children. Neuro-radiological ameliorations, documented by MRI, of brain lesions. Changes in brain metabolism, testified by cerebral PET, and in cerebral perfusion, documented by brain SPECT.
    - Improvement of neuro-sensorial potentials, such as visual evoked potentials and sensory-motor potentials.
    Miglioramento delle condizioni cliniche e neurologiche dei bambini trattati.
    Miglioramento neuroradiologico documentato mediante RMN delle lesioni cerebrali.
    Modifiche del metabolismo cerebrale e della perfusine cerebrale testimoniate dalla PET e dal SPET rispettivamente.
    Miglioramento deipotenzialineurosensoriali quali potenziali evocati visivi e potenziali neurosensoriali
    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 months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months0
    E.8.9.2In all countries concerned by the trial days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    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) Yes
    F.1.1.4.1Number of subjects for this age range: 3
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 2
    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
    Children aged between 6 month to 5 years
    Bambini di età compresa tra 6 mesi e 5 anni
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state5
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 5
    F.4.2.2In the whole clinical trial 5
    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)
    Follow up at 6 months and at 12 months after the study conclusion
    Follow up a 6 mesi e a 12 mesi dal termine dello studio
    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 Decision2020-05-13
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-02-06
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2024-07-27
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