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)

Start 18 May 2023 · Status Ongoing, recruitment ended · 1 EU/EEA countries · 1 sites · Protocol CHF6467-OPG

Overview

Sponsor-declared trial summary

Phase Phase I and Phase II (Integrated) - Other

Status Ongoing, recruitment ended

Participants planned 36

Countries 1

Sites 1

Optic Pathway Glioma (OPG)

The purpose 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

Key facts

Sponsor: Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Participant type: Pediatric, Patients
Age range: 0-17 years, 18-64 years
Gender: Male and Female
Therapeutic area: Diseases [C] - Eye Diseases [C11]
Trial duration: 18 May 2023 → ongoing
Decision date (initial): 2024-11-05
Transition trial: Yes
Low-intervention: No
Rare-disease indication: Yes
Vulnerable population: No

External identifiers

EU CT number: 2024-515753-16-00
EudraCT number: 2020-005264-60

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Efficacy, Therapy

Conditions and MedDRA coding

Optic Pathway Glioma (OPG)

Version	Level	Code	Term	System organ class
20.0	PT	10073338	Optic glioma	100000004864

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 1

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

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.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

The largest radius of the field of viewmeasured in degrees visual angle by kinetic perimetry according to Goldmann with the aim V/4e

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Nerve Growth Factor, Recombinant (R100E)

PRD11499894 · Product

Active substance: Nerve Growth Factor, Recombinant (R100E)
Substance synonyms: CHF6467
Pharmaceutical form: EYE DROPS
Route of administration: OCULAR USE
Authorisation status: Not Authorised
MA holder: FONDAZIONE POLICLINICO UNIVERSITARIO AGOSTINO GEMELLI IRCCS
Paediatric formulation: No
Orphan designation: No

Placebo 1

The Placebo composition is the same of the IMP except the active ingredient

N/A · Product

Other product name: N/A
Pharmaceutical form: N/A
ATC code: N/A — N/A
Marketing authorisation: N/A
MA holder: N/A
MA country: Iceland
Paediatric formulation: No

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Fondazione Policlinico Universitario Agostino Gemelli IRCCS

Sponsor organisation: Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Address: Largo Francesco Vito 1
City: Rome
Postcode: 00168
Country: Italy

Scientific contact point

Organisation: Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Contact name: Antonio Ruggiero

Public contact point

Organisation: Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Contact name: Antonio Ruggiero

Locations

1 EU/EEA country · 1 investigational sites

By country

Country	MS status	Planned subjects	Sites
Italy	Ongoing, recruitment ended	36	1
Rest of world	—	0	—

Investigational sites

Italy

1 site · Ongoing, recruitment ended

Fondazione Policlinico Universitario Agostino Gemelli IRCCS

UOC Oculistica, Largo Francesco Vito 1, 00168, Rome

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Country	Trial start	Trial end	Recruitment start	Recruitment end	Early termination
Italy	2023-05-18		2023-06-12	2026-03-18

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 12 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Type	Title	Version
Protocol (for publication)	CHF6467 General Protocol v 8 20Jan2023	8
Protocol (for publication)	CHF6467 Protocol v 8 20Jan2023	8
Recruitment arrangements (for publication)	Recruitment arrangments	1
Subject information and informed consent form (for publication)	CHF6467 GDPR patients v 1 29Oct2020	1
Subject information and informed consent form (for publication)	CHF6467 GP letter 2 20Jan2023	2
Subject information and informed consent form (for publication)	CHF6467 ISF ICF adult v 4 28Feb2023	4
Subject information and informed consent form (for publication)	CHF6467 ISF ICF children v 2 10Dec2020	2
Subject information and informed consent form (for publication)	CHF6467 ISF ICF parents v 4 28Feb2023	4
Subject information and informed consent form (for publication)	CHF6467 ISF ICF teenagers v 4 28Feb2023	4
Subject information and informed consent form (for publication)	CHF6467_01_emergency_card_patient	1
Summary of Product Characteristics (SmPC) (for publication)	This document is not applicable	1
Synopsis of the protocol (for publication)	CHF6467 Synopsis v 3 20Jan2023	3

Application history

4 submissions — initial application plus substantial / non-substantial modifications

#	Type	Code	Submitted	Reference MS	Conclusion	Decision date
1	INITIAL	IN	2024-09-10	Italy	Acceptable 2024-10-07	2024-11-05
2	SUBSTANTIAL MODIFICATION	SM-1	2024-11-19	Italy	Acceptable 2025-01-14	2025-01-16
3	NON SUBSTANTIAL MODIFICATION	NSM-2	2025-02-21	Italy	Acceptable 2025-01-14	2025-02-21
4	NON SUBSTANTIAL MODIFICATION	NSM-3	2026-03-23	Italy	Acceptable 2025-01-14	2026-03-23