Evaluating a new treatment for optic pathway glioma using a painless nerve growth factor

Safety and Efficacy of Multiple Doses of the PAINLESS Nerve Growth Factor CHF6467 in Paediatric Subjects With Optic Pathway Glioma (OPG). A Randomized Clinical Trial (RCT)

Phase 2 Interventional Catholic University of the Sacred Heart · NCT05733572

This study is testing a new painless treatment to see if it can help protect the vision of people with optic pathway glioma from getting worse.

Quick facts

PhasePhase 2
Study typeInterventional
Enrollment36 (estimated)
Ages3 Years to 40 Years
SexAll
SponsorCatholic University of the Sacred Heart Academic / other
Drugs / interventionschemotherapy
Locations1 site (Rome)
Trial IDNCT05733572 on ClinicalTrials.gov

What this trial studies

This clinical trial investigates the safety and efficacy of CHF6467, a recombinant and painless form of nerve growth factor, in patients with optic pathway glioma (OPG) who experience visual damage. The study aims to assess whether CHF6467 can prevent further visual deterioration by targeting neuroprotection rather than tumor reduction. Participants will receive either CHF6467 or a placebo, and their visual function will be monitored over time. The trial includes both pediatric and adult subjects aged 3 to 40 with a confirmed diagnosis of OPG.

Who should consider this trial

Good fit: Ideal candidates for this study are pediatric or adult patients aged 3 to 40 with OPG-induced visual damage.

Not a fit: Patients with concurrent ophthalmological disorders that could interfere with the evaluation or those who have received specific antineoplastic treatments within the last 9 months may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly improve visual outcomes for patients suffering from optic pathway glioma.

How similar studies have performed: Previous exploratory studies have shown promising results with similar approaches using nerve growth factor in preventing visual damage in OPG patients.

Eligibility criteria

Show full inclusion / exclusion 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.

Exclusion Criteria:

- 1. concomitant ophthalmological disorder that may affect electrophysiological evaluation.

2. radiotherapy or chemotherapy or any other specific antineoplastic treatment within 9 months before entry.

Where this trial is running

Rome

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions Optic Pathway GliomaOptic pathway glioma, nerve growth factoroptic atrophy
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.