Safety and Efficacy of multiple doses of the PAINLESS Nerve Growth Factor CHF6467 in subjects with Optic Pathway Glioma (OPG). A randomized clinical trial (RCT)

Safety and Efficacy of multiple doses of the PAINLESS Nerve Growth Factor CHF6467 in subjects with Optic Pathway Glioma (OPG). A randomized clinical trial (RCT) - CHF6467-OPG

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2020-005264-60-IT

Enrollment

Registered

2021-06-01

Start date

2021-11-26

Completion date

Unknown

Last updated

2024-12-16

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Optic pathway glioma - optic neuropathy MedDRA version: 20.0 Level: PT Classification code 10073338 Term: Optic glioma System Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)

Interventions

Product Name: Nerve Growth Factor (NGF) Product Code: [CHF6467] Pharmaceutical Form: Eye drops, solution INN or Proposed INN: 00792400 Current Sponsor code: CHF6467 Concentration unit: 1X 100 milligra

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: 1. Paediatric or adult subjects between the ages of 3 and 40 (including extremes). 2.Diagnosis of OPG-induced visual damage, with or without type 1 neurofibromatosis (NF-1) 3. Stable disease with two brain MRI checks, performed at least 6 months before screening. Are the trial subjects under 18? yes Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 6 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range

Exclusion criteria

Exclusion criteria: 1. No concomitant ophthalmological disorder that may affect electrophysiological evaluation. 2. No radiotherapy or chemotherapy or any other specific antineoplastic treatment within 9 months before entry.

Design outcomes

Primary

Measure	Time frame
Main Objective: Main objecive of this randomised study is to assess the safety and efficacy of multiple doses of painless NGF CHF6467 eye drops on the visual function of children or young adults with optic pathway gliomas, whether or not associated with type 1 neurofibromatosis;Secondary Objective: Secondary endpoints: visual acuity, electroretinograms (ERG) Ganzfeld and evoked visual potentials (VEPs), and the study of nerve fibers with optical coherence tomography (OCT);Primary end point(s): The largest radius (the largest) of the field of view measured in degrees of visual angle by kinetic perimetry according to Goldmann with the aim V/4e.;Timepoint(s) of evaluation of this end point: 3 months	—

Measure

Time frame

Main Objective: Main objecive of this randomised study is to assess the safety and efficacy of multiple doses of painless NGF CHF6467 eye drops on the visual function of children or young adults with optic pathway gliomas, whether or not associated with type 1 neurofibromatosis;Secondary Objective: Secondary endpoints: visual acuity, electroretinograms (ERG) Ganzfeld and evoked visual potentials (VEPs), and the study of nerve fibers with optical coherence tomography (OCT);Primary end point(s): The largest radius (the largest) of the field of view measured in degrees of visual angle by kinetic perimetry according to Goldmann with the aim V/4e.;Timepoint(s) of evaluation of this end point: 3 months

—

Secondary

Measure	Time frame
Secondary end point(s): visual acuity, electroretinograms (ERG) Ganzfeld and evoked visual potentials (VEPs), and the study of nerve fibers with optical coherence tomography (OCT);Timepoint(s) of evaluation of this end point: 3 months	—

Countries

Italy

Contacts

Public ContactDIREZIONE SCIENTIFICA

FONDAZIONE POLICLINICO UNIVERSITARIO A. GEMELLI IRCCS

DIREZIONE.SCIENTIFICA@POLICLINICOGEMELLI.IT0630155701

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026