Clinical trial • Phase I/II • Ophthalmology

Nerve growth factor, recombinant (R100E) for Optic pathway glioma

Phase I/II trial of Nerve growth factor, recombinant (R100E) for Optic pathway glioma.

Overview

Trial Therapeutic Area
Ophthalmology
Trial Disease
Optic pathway glioma
Trial Stage
Phase I/II
Drug Modality
Peptide/protein/enzyme
Paediatric Trial
Yes

Key dates

Initial CTIS Submission Date
10-09-2024
First CTIS Authorization Date
05-11-2024

Trial design

Randomised, the placebo composition is the same of the imp except the active ingredient (placebo arm); no dose or schedule for comparator specified-controlled Phase I/II trial across 1 site in Italy.

Randomised
Yes
Comparator
The Placebo composition is the same of the IMP except the active ingredient (placebo arm); no dose or schedule for comparator specified
Target Sample Size
36

Eligibility

Recruits 36 paediatric patients.

Vulnerable Population
Paediatric participants are included (age range starts at 3). Subject information and informed consent forms are available for adults, parents, children and teenagers (documents: CHF6467 ISF ICF adult; CHF6467 ISF ICF parents; CHF6467 ISF ICF children; CHF6467 ISF ICF teenagers). 'isVulnerablePopulationSelected' is false in the record. Specific consent/assent procedures or languages are not specified in the available data.

Inclusion criteria

  • {"criterion_text":"- 1. Paediatric or adult subjects between the ages of 3 and 40 (including extremes).\n- 2.Diagnosis of OPG-induced visual damage, with or without type 1 neurofibromatosis (NF-1)\n- 3.\tStable disease with two brain MRI checks, performed at least 6 months before screening."}

Exclusion criteria

  • {"criterion_text":"- 1.\tNo concomitant ophthalmological disorder that may affect electrophysiological evaluation.\n- 2.\tNo radiotherapy or chemotherapy or any other specific antineoplastic treatment within 9 months before entry."}

Endpoints

Primary endpoints

  • {"endpoint_text":"- The largest radius of the field of viewmeasured in degrees visual angle by kinetic perimetry according to Goldmann with the aim V/4e","definition_or_measurement_approach":"Measured by kinetic perimetry according to Goldmann with the aim V/4e (largest radius of the field of view measured in degrees visual angle)."}

Recruitment

Planned Sample Size
36
Recruitment Window Months
36
Consent Approach
Subject information and informed consent forms are available for adults, parents, children and teenagers (documents listed in the dossier). Parental/guardian consent and age-appropriate child/teenager information/assent documents are provided (files: CHF6467 ISF ICF parents; CHF6467 ISF ICF children; CHF6467 ISF ICF teenagers). No specific languages or additional consent process details are provided in the available data.

Geography

Total Number Of Sites
1
Total Number Of Participants
36

Italy

Latest Decision Or Authorization Date
23-03-2026
Number Of Sites
1
Number Of Participants
36

Sites

Site Name
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Department Name
UOC Oculistica
Principal Investigator Name
Antonio Ruggiero
Principal Investigator Email
Antonio.Ruggiero@unicatt.it
Contact Person Name
Antonio Ruggiero
Contact Person Email
Antonio.Ruggiero@unicatt.it
Number Of Participants
36

Sponsor

Primary sponsor

Full Name
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Organisation Type
Hospital/Clinic/Other health care facility
Country Of Registered Address
Italy

Investigational products

Investigational Product Name
PAINLESS Nerve Growth Factor CHF6467
Active Substance
Nerve growth factor, recombinant (R100E)
Modality
Peptide/protein/enzyme
Routes Of Administration
Ocular (eye drops)
Route
Ocular (eye drops)
Authorisation Status
Investigational
Investigational Product Name
The Placebo composition is the same of the IMP except the active ingredient
Modality
Other

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