Clinical Trials Register

Summary
EudraCT Number:	2020-005264-60
Sponsor's Protocol Code Number:	CHF6467-OPG
National Competent Authority:	Italy - Italian Medicines Agency
Clinical Trial Type:	EEA CTA
Trial Status:	Trial now transitioned
Date on which this record was first entered in the EudraCT database:	2021-06-01
Trial results

Index
A. PROTOCOL INFORMATION
B. SPONSOR INFORMATION
C. APPLICANT IDENTIFICATION
D. IMP IDENTIFICATION
D.8 INFORMATION ON PLACEBO
E. GENERAL INFORMATION ON THE TRIAL
F. POPULATION OF TRIAL SUBJECTS
G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
P. END OF TRIAL

Expand All Collapse All

A. Protocol Information

A.1

Member State Concerned

Italy - Italian Medicines Agency

A.2

EudraCT number

2020-005264-60

A.3

Full title of the trial

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)

Efficacia e sicurezza di multiple dosi di Painless Nerve Growth factor in pazienti con glioma delle vie ottiche (OPG). Un trial clinico randomizzato

A.3.1

Title of the trial for lay people, in easily understood, i.e. non-technical, language

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)

Efficacia e sicurezza di multiple dosi di Painless Nerve Growth factor in pazienti con glioma delle vie ottiche (OPG). Un trial clinico randomizzato

A.3.2

Name or abbreviated title of the trial where available

CHF6467-OPG

A.4.1

Sponsor's protocol code number

CHF6467-OPG

A.7

Trial is part of a Paediatric Investigation Plan

A.8

EMA Decision number of Paediatric Investigation Plan

B. Sponsor Information
B.Sponsor: 1
B.1.1	Name of Sponsor	FONDAZIONE POLICLINICO UNIVERSITARIO AGOSTINO GEMELLI IRCCS UNIVERSITA' CATTOLICA DEL SACRO CUORE
B.1.3.4	Country	Italy
B.3.1 and B.3.2	Status of the sponsor	Non-Commercial
B.4 Source(s) of Monetary or Material Support for the clinical trial:
B.4.1	Name of organisation providing support	MINISTERO DELLA SALUTE RF 2019
B.4.2	Country	Italy
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	FONDAZIONE POLICLINICO UNIVERSITARIO A. GEMELLI IRCCS
B.5.2	Functional name of contact point	DIREZIONE SCIENTIFICA
B.5.3	Address:
B.5.3.1	Street Address	LARGO A. GEMELLI 8
B.5.3.2	Town/ city	ROMA
B.5.3.3	Post code	00135
B.5.3.4	Country	Italy
B.5.4	Telephone number	0630155701
B.5.5	Fax number	0630155701
B.5.6	E-mail	DIREZIONE.SCIENTIFICA@POLICLINICOGEMELLI.IT

D. IMP Identification
D.IMP: 1
D.1.2 and D.1.3	IMP Role	Test
D.2	Status of the IMP to be used in the clinical trial
D.2.1	IMP to be used in the trial has a marketing authorisation	No
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.1	Product name	Nerve Growth Factor (NGF)
D.3.2	Product code	[CHF6467]
D.3.4	Pharmaceutical form	Eye drops, solution
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Ocular use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	00792400
D.3.9.2	Current sponsor code	CHF6467
D.3.10	Strength
D.3.10.1	Concentration unit	1X 100 milligrams/millilitre
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	5
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	Yes
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	Yes
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	Information not present in EudraCT
D.3.11.3.2	Gene therapy medical product	Information not present in EudraCT
D.3.11.3.3	Tissue Engineered Product	Information not present in EudraCT
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	Information not present in EudraCT
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	Information not present in EudraCT
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	No
D.3.11.5	Radiopharmaceutical medicinal product	No
D.3.11.6	Immunological medicinal product (such as vaccine, allergen, immune serum)	No
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	Yes
D.3.11.9	Recombinant medicinal product	Yes
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another type of medicinal product	No

D.8 Information on Placebo
D.8 Placebo: 1
D.8.1	Is a Placebo used in this Trial?	Yes
D.8.3	Pharmaceutical form of the placebo	Eye drops, solution
D.8.4	Route of administration of the placebo	Ocular use

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

Optic pathway glioma - optic neuropathy

Pazienti affetti da Glioma delle vie ottiche (OPG) con deficit dell’acuità visiva e del campo visivo secondari ad atrofia del nervo ottico determinata da OPG.

E.1.1.1

Medical condition in easily understood language

Optic pathway glioma - optic neuropathy

Pazienti affetti da Glioma delle vie ottiche (OPG) con deficit dell’acuità visiva e del campo visivo secondari ad atrofia del nervo ottico determinata da OPG.

E.1.1.2

Therapeutic area

Diseases [C] - Eye Diseases [C11]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	20.0
E.1.2	Level	PT
E.1.2	Classification code	10073338
E.1.2	Term	Optic glioma
E.1.2	System Organ Class	10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)

E.1.3

Condition being studied is a rare disease

Yes

E.2 Objective of the trial

E.2.1

Main objective of the trial

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

Obiettivo primario del presente studio randomizzato e’ quello di valutare l’efficacia di multiple dosi di painless NGF CHF6467 collirio sulla funzione visiva di bambini o giovani adulti affetti da gliomi delle vie ottiche, associati o meno a neurofibromatosi di tipo 1

E.2.2

Secondary objectives of the trial

Secondary endpoints: visual acuity, electroretinograms (ERG) Ganzfeld and evoked visual potentials (VEPs), and the study of nerve fibers with optical coherence tomography (OCT)

a. confermare la sicurezza del collirio CHF6467;
b. valutare l’effetto dello stesso collirio sui parametri acuita’ visiva, elettroretinogramma e potenziali visivi evocati

E.2.3

Trial contains a sub-study

E.3

Principal 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.

1. Soggetti pediatrici ed adulti con età compresa tra 3 e 40 anni (estremi inclusi).
2. Diagnosi di danno visivo indotto da OPG, con o senza neurofibromatosi di tipo 1 (NF-1)
3. Malattia stabile a due controlli RM cerebrali, eseguita almeno 6 mesi prima dello screening.

E.4

Principal 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.

1. Nessun disturbo oftalmologico concomitante che possa influenzare la valutazione elettrofisiologica.
2. Nessuna radioterapia o chemioterapia o qualsiasi altro trattamento antineoplastico specifico entro 9 mesi prima dell'ingresso.

E.5 End points

E.5.1

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.

Il raggio maggiore (quello piu’ esteso) del campo visivo misurato in gradi di angolo visivo mediante perimetria cinetica secondo Goldmann con la mira V/4e.

E.5.1.1

Timepoint(s) of evaluation of this end point

3 months

3 mesi

E.5.2

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)

l'acuità visiva, elettroretinogrammi (ERG) Ganzfeld e i potenziali visivi evocati (VEPs), ed lo studio delle fibre nervose con tomografia a coerenza ottica (OCT)

E.5.2.1

Timepoint(s) of evaluation of this end point

3 months

3 mesi

E.6 and E.7 Scope of the trial

E.6

Scope of the trial

E.6.1

Diagnosis

E.6.2

Prophylaxis

E.6.3

Therapy

Yes

E.6.4

Safety

Yes

E.6.5

Efficacy

Yes

E.6.6

Pharmacokinetic

E.6.7

Pharmacodynamic

E.6.8

Bioequivalence

E.6.9

Dose response

E.6.10

Pharmacogenetic

E.6.11

Pharmacogenomic

E.6.12

Pharmacoeconomic

E.6.13

Others

E.7

Trial type and phase

E.7.1

Human pharmacology (Phase I)

E.7.1.1

First administration to humans

E.7.1.2

Bioequivalence study

E.7.1.3

Other

E.7.1.3.1

Other trial type description

E.7.2

Therapeutic exploratory (Phase II)

Yes

E.7.3

Therapeutic confirmatory (Phase III)

E.7.4

Therapeutic use (Phase IV)

E.8 Design of the trial

E.8.1

Controlled

Yes

E.8.1.1

Randomised

Yes

E.8.1.2

Open

E.8.1.3

Single blind

E.8.1.4

Double blind

Yes

E.8.1.5

Parallel group

Yes

E.8.1.6

Cross over

E.8.1.7

Other

Yes

E.8.1.7.1

Other trial design description

OPEN LABEL EXTENSION PHASE

E.8.2

Comparator of controlled trial

E.8.2.1

Other medicinal product(s)

E.8.2.2

Placebo

Yes

E.8.2.3

Other

E.8.2.4

Number of treatment arms in the trial

E.8.3

The trial involves single site in the Member State concerned

Yes

E.8.4

The trial involves multiple sites in the Member State concerned

E.8.4.1

Number of sites anticipated in Member State concerned

E.8.5

The trial involves multiple Member States

E.8.6 Trial involving sites outside the EEA

E.8.6.1

Trial being conducted both within and outside the EEA

E.8.6.2

Trial being conducted completely outside of the EEA

Information not present in EudraCT

E.8.7

Trial has a data monitoring committee

E.8.8

Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial

LVLS

E.8.9 Initial estimate of the duration of the trial

E.8.9.1

In the Member State concerned years

E.8.9.1

In the Member State concerned months

E.8.9.1

In the Member State concerned days

E.8.9.2

In all countries concerned by the trial years

E.8.9.2

In all countries concerned by the trial months

E.8.9.2

In all countries concerned by the trial days

F. Population of Trial Subjects

F.1 Age Range

F.1.1

Trial has subjects under 18

Yes

F.1.1.1

In Utero

F.1.1.2

Preterm newborn infants (up to gestational age < 37 weeks)

F.1.1.3

Newborns (0-27 days)

F.1.1.4

Infants and toddlers (28 days-23 months)

F.1.1.5

Children (2-11years)

Yes

F.1.1.5.1

Number of subjects for this age range:

F.1.1.6

Adolescents (12-17 years)

Yes

F.1.1.6.1

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:

F.1.3

Elderly (>=65 years)

F.2 Gender

F.2.1

Female

Yes

F.2.2

Male

Yes

F.3 Group of trial subjects

F.3.1

Healthy volunteers

F.3.2

Patients

Yes

F.3.3

Specific vulnerable populations

Yes

F.3.3.1

Women of childbearing potential not using contraception

F.3.3.2

Women of child-bearing potential using contraception

Yes

F.3.3.3

Pregnant women

F.3.3.4

Nursing women

F.3.3.5

Emergency situation

F.3.3.6

Subjects incapable of giving consent personally

Yes

F.3.3.6.1

Details of subjects incapable of giving consent

SOME SUBJECTS INCLUDED IN THE STUDY WILL NOT BE ABLE TO PROVIDE AN INFORMED CONSENT, THAT IT WILL BE PROVIDED BY THE CHILD'S PARENTS

ALCUNI SOGGETTI TRATTATI INCLUSI NELLO STUDIO, SARANNO BAMBINI INCAPACI DI DARE IL PROPRIO CONSENSO CHE VERRA' FORNITO DAI GENITORI

F.3.3.7

Others

F.4 Planned number of subjects to be included

F.4.1

In the member state

F.4.2

For a multinational trial

F.4.2.1

In the EEA

F.4.2.2

In the whole clinical trial

F.5

Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)

BEST CLINICAL PRACTICE, CLINICAL FOLLOW UP ACCORDING TO STANDARD GUIDELINES

MIGLIORE PRATICA CLINICA, FOLLOW UP CLINICO SECONDO LE LINEE GUIDA

G. Investigator Networks to be involved in the Trial

N. Review by the Competent Authority or Ethics Committee in the country concerned

Competent Authority Decision

Authorised

Date of Competent Authority Decision

2021-11-26

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2021-06-17

P. End of Trial
P.	End of Trial Status	Trial now transitioned

Select Country:	Select Age Range:
Select Trial Status:	Select Trial Phase:
Select Gender:
Select Date Range: to
Select Rare Disease:
IMP with orphan designation in the indication
Orphan Designation Number:
Results Status:
Clear advanced search filters

Austria
Belgium
Bulgaria
Croatia
Cyprus
Czechia
Denmark
Estonia
Finland
France
Germany
Greece
Hungary
Iceland
Ireland
Italy
Latvia
Liechtenstein
Lithuania
Luxembourg
Malta
Netherlands
Norway
Poland
Portugal
Romania
Slovakia
Slovenia
Spain
Sweden
United Kingdom
Outside EU/EEA

Clinical trials