Persistent unresponsive wakefulness syndrome (UWS) MedDRA version: 20.1 Level: LLT Classification code 10049615 Term: Late effects of head trauma System Organ Class: 100000004863
Conditions
Interventions
Trade Name: OXERVATE
Product Name: oxervate
Product Code: [rh-NGF]
Pharmaceutical Form: Eye drops, solution
INN or Proposed INN: CENEGERMIN
CAS Number: 1772578-74-1
Current Sponsor code: rh-NGF
Other
Sponsors
FONDAZIONE POLICLINICO UNIVERSITARIO AGOSTINO GEMELLI IRCCS UNIVERSITA' CATTOLICA DEL SACRO CUORE
Eligibility
Sex/Gender
All
Inclusion criteria
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. Are the trial subjects under 18? yes Number of subjects for this age range: F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: - Intraparenchymal hemorrhages - intraventricular hemorrhages - subdural hematomas - epidural hematomas.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: -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);Secondary Objective: -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;Primary 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;Timepoint(s) of evaluation of this end point: 36 month | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary 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);Timepoint(s) of evaluation of this end point: 36 months | — |
Countries
Italy
Contacts
Public ContactDirezione Scientifica Fondazione Po
Direzione scientifica Fondazione Policlinico A.Gemelli IRCCS
Outcome results
None listed