Clinical Trials Register

Summary
EudraCT Number:	2021-001198-22
Sponsor's Protocol Code Number:	ALXN2040-GA-201
National Competent Authority:	Italy - Italian Medicines Agency
Clinical Trial Type:	EEA CTA
Trial Status:	Ongoing
Date on which this record was first entered in the EudraCT database:	2022-01-26
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

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A. Protocol Information

A.1

Member State Concerned

Italy - Italian Medicines Agency

A.2

EudraCT number

2021-001198-22

A.3

Full title of the trial

A Phase 2, Double-Masked, Placebo-Controlled, Dose Range Finding Study of Danicopan (ALXN2040) in Patients with Geographic Atrophy (GA) Secondary to Age-Related Macular Degeneration (AMD)

Studio di fase 2, in doppio cieco, controllato con placebo, per la determinazione dell'intervallo di dosaggio di Danicopan (ALXN2040) in pazienti con atrofia geografica (AG) secondaria a degenerazione maculare legata all'età (DMLE)

A.3.1

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

A Study of Danicopan in Participants With Geographic Atrophy Secondary to Age-Related Macular Degeneration

Uno studio su Danicopan in partecipanti con atrofia geografica secondaria a degenerazione maculare legata all'età

A.3.2

Name or abbreviated title of the trial where available

A.4.1

Sponsor's protocol code number

ALXN2040-GA-201

A.5.2

US NCT (ClinicalTrials.gov registry) number

NCT05019521

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	ALEXION PHARMACEUTICALS INCORPORATED
B.1.3.4	Country	United States
B.3.1 and B.3.2	Status of the sponsor	Commercial
B.4 Source(s) of Monetary or Material Support for the clinical trial:
B.4.1	Name of organisation providing support	Alexion Pharmaceuticals, Inc.
B.4.2	Country	United States
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Alexion Europe SAS
B.5.2	Functional name of contact point	European Clinical Trial Information
B.5.3	Address:
B.5.3.1	Street Address	103-105 Rue Anatole France
B.5.3.2	Town/ city	Levallois-Perret
B.5.3.3	Post code	92300
B.5.3.4	Country	France
B.5.4	Telephone number	0033147100615
B.5.5	Fax number	0033147100611
B.5.6	E-mail	clinicaltrials.eu@alexion.com

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	Danicopan (ALXN2040)
D.3.2	Product code	[ACH-0144471]
D.3.4	Pharmaceutical form	Film-coated tablet
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Oral use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	danicopan
D.3.9.1	CAS number	1903768-17-1
D.3.9.2	Current sponsor code	ACH-0144471
D.3.9.4	EV Substance Code	SUB189885
D.3.10	Strength
D.3.10.1	Concentration unit	mg milligram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	100
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)	No
	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	No
D.3.11.9	Recombinant medicinal product	No
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.IMP: 2
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	Yes
D.2.1.1.1	Trade name	Menveo
D.2.1.1.2	Name of the Marketing Authorisation holder	GSK Vaccines S.r.l. Italy EU/1/10/614-002-003
D.2.1.2	Country which granted the Marketing Authorisation	Italy
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	Menveo
D.3.2	Product code	[na]
D.3.4	Pharmaceutical form	Powder and solvent for solution for injection
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Intramuscular use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	MENINGOCOCCAL GROUP A OLIGOSACCHARIDES CONJUGATED TO CORYNEBACTERIUM DIPHTHERIAE C7 (ß197) M8 (CRM197) PROTEIN
D.3.9.2	Current sponsor code	na
D.3.9.3	Other descriptive name	MENINGOCOCCAL GROUP A OLIGOSACCHARIDES CONJUGATED TO CORYNEBACTERIUM DIPHTHERIAE C7 (ß197) M8 (CRM197) PROTEIN
D.3.9.4	EV Substance Code	SUB77061
D.3.10	Strength
D.3.10.1	Concentration unit	µg microgram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	10
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	N. MENINGITIDIS GROUP C (STRAIN C11) OLIGOSACCHARIDE CONJUGATED TO CORYNEBACTERIUM DIPHTHERIAE CRM197 ADSORBED ON ALUMINUM HYDROXIDE
D.3.9.2	Current sponsor code	na
D.3.9.3	Other descriptive name	N. MENINGITIDIS GROUP C (STRAIN C11) OLIGOSACCHARIDE CONJUGATED TO CORYNEBACTERIUM DIPHTHERIAE CRM197 ADSORBED ON ALUMINUM HYDROXIDE
D.3.9.4	EV Substance Code	SUB194220
D.3.10	Strength
D.3.10.1	Concentration unit	µg microgram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	5
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	MENINGOCOCCAL GROUP W OLIGOSACCHARIDES CONJUGATED TO CORYNEBACTERIUM DIPHTHERIAE C7 (ß197) M8 (CRM197) PROTEIN
D.3.9.2	Current sponsor code	na
D.3.9.3	Other descriptive name	MENINGOCOCCAL GROUP W OLIGOSACCHARIDES CONJUGATED TO CORYNEBACTERIUM DIPHTHERIAE C7 (ß197) M8 (CRM197) PROTEIN
D.3.9.4	EV Substance Code	SUB77063
D.3.10	Strength
D.3.10.1	Concentration unit	µg microgram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	5
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	MENINGOCOCCAL GROUP Y OLIGOSACCHARIDES CONJUGATED TO CORYNEBACTERIUM DIPHTHERIAE C7 (Â197) M8 (CRM197) PROTEIN
D.3.9.2	Current sponsor code	na
D.3.9.3	Other descriptive name	MENINGOCOCCAL GROUP Y OLIGOSACCHARIDES CONJUGATED TO CORYNEBACTERIUM DIPHTHERIAE C7 (Â197) M8 (CRM197) PROTEIN
D.3.9.4	EV Substance Code	SUB77060
D.3.10	Strength
D.3.10.1	Concentration unit	µg microgram(s)
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	No
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)	Yes
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	No
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.IMP: 3
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	Yes
D.2.1.1.1	Trade name	Bexsero
D.2.1.1.2	Name of the Marketing Authorisation holder	GSK Vaccines S.r.l., Via Fiorentina 1, 53100 Siena, Italy EU/1/12/812/001-004
D.2.1.2	Country which granted the Marketing Authorisation	Italy
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	Bexsero
D.3.2	Product code	[/]
D.3.4	Pharmaceutical form	Suspension for injection in pre-filled syringe
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Intramuscular use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	Outer membrane vesicles (OMV) from Neisseria meningitidis group B strain NZ98/254 measured as amount of total protein containing the PorA P1.4
D.3.9.2	Current sponsor code	na
D.3.9.3	Other descriptive name	RECOMBINANT NEISSERIA MENINGITIDIS GROUP B FHBP FUSION PROTEIN PRODUCED IN E.COLI CELLS BY RECOMBINANT DNA TECHNOLOGY ADSORBED ON ALUMINIUM HYDROXIDE
D.3.9.4	EV Substance Code	SUB96090
D.3.10	Strength
D.3.10.1	Concentration unit	µg microgram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	25
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	RECOMBINANT NEISSERIA MENINGITIDIS GROUP B NHBA FUSION PROTEIN
D.3.9.2	Current sponsor code	na
D.3.9.3	Other descriptive name	RECOMBINANT NEISSERIA MENINGITIDIS GROUP B NHBA FUSION PROTEIN
D.3.9.4	EV Substance Code	SUB191635
D.3.10	Strength
D.3.10.1	Concentration unit	µg microgram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	50
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	RECOMBINANT NEISSERIA MENINGITIDIS GROUP B NADA PROTEIN
D.3.9.2	Current sponsor code	na
D.3.9.3	Other descriptive name	RECOMBINANT NEISSERIA MENINGITIDIS GROUP B NADA PROTEIN
D.3.9.4	EV Substance Code	SUB191633
D.3.10	Strength
D.3.10.1	Concentration unit	µg microgram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	50
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	RECOMBINANT NEISSERIA MENINGITIDIS GROUP B FHBP FUSION PROTEIN
D.3.9.2	Current sponsor code	na
D.3.9.3	Other descriptive name	RECOMBINANT NEISSERIA MENINGITIDIS GROUP B FHBP FUSION PROTEIN
D.3.9.4	EV Substance Code	SUB191634
D.3.10	Strength
D.3.10.1	Concentration unit	µg microgram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	50
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	No
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)	Yes
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
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.IMP: 4
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	Yes
D.2.1.1.1	Trade name	Ciprofloxacina Mylan Generics
D.2.1.1.2	Name of the Marketing Authorisation holder	Mylan S.p.A. - AIC 037219247
D.2.1.2	Country which granted the Marketing Authorisation	Italy
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	CIPROFLOXACINA
D.3.2	Product code	[na]
D.3.4	Pharmaceutical form	Film-coated tablet
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Oral use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	CIPROFLOXACINA
D.3.9.2	Current sponsor code	na
D.3.9.3	Other descriptive name	CIPROFLOXACINA
D.3.10	Strength
D.3.10.1	Concentration unit	mg milligram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	500
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)	No
	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	No
D.3.11.9	Recombinant medicinal product	No
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.IMP: 5
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	Yes
D.2.1.1.1	Trade name	Trumenba
D.2.1.1.2	Name of the Marketing Authorisation holder	Pfizer Europe MA EEIG, Belgium EU/1/17/1187/001-006
D.2.1.2	Country which granted the Marketing Authorisation	European Union
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	Trumenba
D.3.2	Product code	[na]
D.3.4	Pharmaceutical form	Suspension for injection in pre-filled syringe
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Intramuscular use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	NEISSERIA MENINGITIDIS GROUP B FHBP PROTEIN SUBFAMILY A
D.3.9.2	Current sponsor code	na
D.3.9.3	Other descriptive name	NEISSERIA MENINGITIDIS GROUP B FHBP PROTEIN SUBFAMILY A
D.3.9.4	EV Substance Code	SUB182443
D.3.10	Strength
D.3.10.1	Concentration unit	µg microgram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	60
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	NEISSERIA MENINGITIDIS GROUP B FHBP PROTEIN SUBFAMILY B
D.3.9.2	Current sponsor code	na
D.3.9.3	Other descriptive name	NEISSERIA MENINGITIDIS GROUP B FHBP PROTEIN SUBFAMILY B
D.3.9.4	EV Substance Code	SUB182444
D.3.10	Strength
D.3.10.1	Concentration unit	µg microgram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	60
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	No
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)	Yes
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	No
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.IMP: 6
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	Yes
D.2.1.1.1	Trade name	Velamox
D.2.1.1.2	Name of the Marketing Authorisation holder	Neopharmed Gentili S.p.A., Italy 023097013
D.2.1.2	Country which granted the Marketing Authorisation	Italy
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	Velamox
D.3.2	Product code	[na]
D.3.4	Pharmaceutical form	Capsule, hard
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Oral use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	Amoxicilline
D.3.9.2	Current sponsor code	na
D.3.9.3	Other descriptive name	AMOXICILLIN TRIHYDRATE
D.3.9.4	EV Substance Code	SUB00504MIG
D.3.10	Strength
D.3.10.1	Concentration unit	mg milligram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	500
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)	No
	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	No
D.3.11.9	Recombinant medicinal product	No
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	Film-coated tablet
D.8.4	Route of administration of the placebo	Oral use

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

Geographic Atrophy (AD) secondary to Age-related Macular Degeneration (AMD).

Atrofia geografica (AG) secondaria a degenerazione maculare legata all'età (DMLE)

E.1.1.1

Medical condition in easily understood language

Geographic Atrophy (GA) is an advanced form of age-related macular degeneration. It is a progressive, irreversible condition of the eye that causes severe loss of visual function

L'Atrofia geografica (GA) è una forma avanzata di degenerazione maculare legata all'età. È una condizione progressiva e irreversibile dell'occhio che causa una grave perdita della funzione visiva

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	20.1
E.1.2	Level	LLT
E.1.2	Classification code	10063947
E.1.2	Term	Geographic atrophy
E.1.2	System Organ Class	100000004853

E.1.3

Condition being studied is a rare disease

E.2 Objective of the trial

E.2.1

Main objective of the trial

This is a dose finding study designed to evaluate the efficacy, safety, and pharmacokinetics of danicopan in participants with GA secondary to AMD.

Questo è uno studio sulla determinazione del dosaggio progettato per valutare l’efficacia, la sicurezza e la farmacocinetica (PK) di danicopan in pazienti affetti da AG secondaria a DMLE

E.2.2

Secondary objectives of the trial

To evaluate the effect of danicopan on disease progression utilizing anatomical measures compared to placebo
To evaluate the effect of danicopan on disease progression utilizing functional measures compared to placebo
To evaluate the PK and PD of danicopan in patients with GA secondary to AMD
To evaluate the safety and tolerability of danicopan in patients with GA secondary to AMD

- Valutare l’effetto di danicopan sulla progressione della malattia, rispetto al placebo, utilizzando misurazioni anatomiche
- Valutare l’effetto di danicopan sulla progressione della malattia, rispetto al placebo, utilizzando misurazioni funzionali
- Valutare la PK e la PD di danicopan in pazienti affetti da AG secondaria a DMLE
- Valutare la sicurezza e la tollerabilità di danicopan in pazienti con AG secondaria a DMLE

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

Key Inclusion Criteria:
• Age >= 70 years, male or female
• Presentation of GA secondary to AMD in at least 1 eye.
• Study eye must have the specified VA (range of 84 to 4 letters; 20/20 to 20/800) using Early Treatment Diabetic Retinopathy Study charts at starting distance of 4 meters.
• GA area of 0.5 to 17.76 mm2 (~0.25 to 7 disc area [DA]) per eye measured by FAF

Criteri di inclusione principali:
- Maschio o femmina di >= 70 anni di età
- Presentazione di AG secondaria a DMLE in almeno un occhio
- L’occhio in studio deve presentare la VA specificata (intervallo da 84 a 4 lettere; da 20/20 a 20/800) utilizzando le tabelle dello Studio sulla retinopatia diabetica in trattamento precoce (ETDRS) a una distanza iniziale di 4 metri
- Area di AG da 0,5 a 17,76 mm2 (area del disco [Disc Area, DA] da ~0,25 a 7) per occhio misurata mediante AFF

E.4

Principal exclusion criteria

Key Exclusion Criteria:
• GA in either eye due to cause other than AMD.
• Have previously received intravitreal anti-vascular endothelial growth factor injections in study eye.
• Have previously received any complement/stem cell/gene therapy for any ophthalmological condition.
• Previous participation in interventional clinical studies for treatment of drusen, nascent GA or GA (except vitamins or minerals) irrespective of route of administration (ocular or systemic) in either eye.
• Presence of active ocular diseases in either eye that in the opinion of the Investigator compromises or confounds visual function or interferes with study assessments.
• Known or suspected complement deficiency.
• History or presence of any clinically relevant co-morbidities or any uncontrolled conditions.
• Hypersensitivity to fluorescein sodium for injection, the investigational drug (danicopan) or any of its excipients.

Note: Additional inclusion/exclusion criteria may apply, per protocol.

Criteri di esclusione principali:
- AG in almeno uno dei due occhi per cause diverse dalla DMLE
- Aver precedentemente ricevuto iniezioni intravitreali di fattore di crescita endoteliale anti-vascolare (VEGF) nell’occhio in studio
- Aver ricevuto in precedenza qualsiasi terapia con complemento/cellule staminali/geni per qualsiasi condizione oftalmica
- Precedente partecipazione a studi clinici interventistici per il trattamento di drusen, AG nascente o AG (eccetto vitamine o minerali) indipendentemente dalla via di somministrazione (oculare o sistemica) in almeno uno dei due occhi
- Presenza di una malattia oculare attiva in almeno uno dei due occhi che, nell’opinione dello sperimentatore, comprometta o confonda la funzione visiva o interferisca con le valutazioni dello studio
- Deficit del complemento noto o sospetto
- Anamnesi o presenza di qualsiasi comorbilità clinicamente rilevanti o condizioni non controllate
- Ipersensibilità alla fluoresceina sodica per iniezione, al farmaco sperimentale (danicopan) o a uno qualsiasi dei suoi eccipienti

Nota: possono essere applicati ulteriori criteri di inclusione/esclusione, per protocollo.

E.5 End points

E.5.1

Primary end point(s)

Mean rate of change from Baseline at Week 52 in the square root (sqrt) of total GA lesion area (mm/year) in the study eye as measured by fundus autofluorescence (FAF)

Tasso medio di variazione rispetto al basale alla Settimana 52 nella radice quadrata (sqrt) dell’area totale della lesione AG (mm/anno) nell’occhio in studio come misurato mediante autofluorescenza del fondo (AFF)

E.5.1.1

Timepoint(s) of evaluation of this end point

week 52

settimana 52

E.5.2

Secondary end point(s)

- Mean Rate Of Change From Baseline At Week 104 In The Sqrt Of The Total GA Lesion Area In The Study Eye And From Baseline At Week 52 And Week 104 In The Fellow Eye And Both Eyes Combined (Regardless Of Baseline GA Status) As Measured By FAF [Time Frame: Baseline, Week 52 and Week 104]
- Mean Rate Of Change From Baseline At Week 52 And Week 104 In The Total GA Lesion Area In The Study Eye, The Fellow Eye, And Both Eyes Combined (Regardless Of Baseline GA Status) As Measured By FAF [Time Frame: Baseline, Week 52 and Week 104]
- Mean Change From Baseline At Week 52 And Week 104 In The Total GA Lesion Area In The Study Eye, The Fellow Eye, And Both Eyes Combined (Regardless Of Baseline GA Status) As Measured By FAF [Time Frame: Baseline, Week 52 and Week 104]
- Percent Change From Baseline At Week 52 And Week 104 In The Total GA Lesion Area In The Study Eye, The Fellow Eye, And Both Eyes Combined (Regardless Of Baseline GA Status) As Measured By FAF [Time Frame: Baseline, Week 52 and Week 104]
- Mean Change From Baseline At Week 52 And Week 104 In The Sqrt Of The Total GA Lesion Area In The Study Eye, The Fellow Eye, And Both Eyes Combined (Regardless Of Baseline GA Status) As Measured By FAF [Time Frame: Baseline, Week 52 and Week 104]
- Percent Change From Baseline At Week 52 And Week 104 In The Sqrt Of The Total GA Lesion Area In The Study Eye, The Fellow Eye, And Both Eyes Combined (Regardless Of Baseline GA Status) As Measured By FAF [Time Frame: Baseline, Week 52 and Week 104]
- Mean Change From Baseline At Week 52 And Week 104 In Monocular Best-corrected Visual Acuity (BCVA) Scores In The Study Eye And Fellow Eye As Assessed By The Early Treatment Diabetic Retinopathy Study (ETDRS) Chart At Four Meters
[Time Frame: Baseline, Week 52 and Week 104]
- Mean Change From Baseline At Week 52 And Week 104 In Monocular Low Luminance Visual Acuity (LLVA) Scores In The Study Eye And Fellow Eye As Assessed By The ETDRS Chart At Four Meters
[Time Frame: Baseline, Week 52 and Week 104]
- Mean Change From Baseline At Week 52 And Week 104 In Low Luminance Deficit In The Study Eye And Fellow Eye
[Time Frame: Baseline, Week 52 and Week 104]
- Mean Change In National Eye Institute Visual Function Questionnaire, 25-item Version (NEI VFQ 25) Scores From Baseline At Week 52 And Week 104
[Time Frame: Baseline, Week 52 and Week 104]
- Plasma Concentration Of Danicopan Over Time
[Time Frame: Up to 4 hours postdose]
- Ex Vivo Serum Alternative Pathway Activity
[Time Frame: Up to 4 hours postdose]
- Plasma Concentration Of Bb Fragment Of Complement Factor B
[Time Frame: Up to 4 hours postdose]
- Incidence Of Treatment-emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), And Ocular TEAEs, SAEs, And Clinical Laboratory Abnormalities, And Events Leading To Discontinuation Of Study Drug
[Time Frame: Day 1 through Week 104]

- Tasso medio di variazione rispetto al basale alla Settimana 104 nella sqrt dell’area totale della lesione AG nell’occhio in studio e rispetto al basale alla Settimana 52 e alla Settimana 104 nell’altro occhio, e nella combinazione di entrambi gli occhi (indipendentemente dallo stato AG al basale) come misurato mediante AFF
[Time Frame: Baseline, Week 52 and Week 104]
- Tasso medio di variazione rispetto al basale alla Settimana 52 e alla Settimana 104 nell’area totale della lesione AG nell’occhio in studio, nell’altro occhio e nella combinazione di entrambi gli occhi (indipendentemente dallo stato AG al basale) come misurato mediante AFF
[Time Frame: Baseline, Week 52 and Week 104]
- Variazione media rispetto al basale alla Settimana 52 e alla Settimana 104 nell’area totale della lesione AG nell’occhio in studio, nell’altro occhio e nella combinazione di entrambi gli occhi (indipendentemente dallo stato AG al basale) come misurato mediante AFF
[Time Frame: Baseline, Week 52 and Week 104]
- Variazione percentuale rispetto al basale alla Settimana 52 e alla Settimana 104 nell’area totale della lesione AG nell’occhio in studio, nell’altro occhio e nella combinazione di entrambi gli occhi (indipendentemente dallo stato AG al basale) come misurato mediante AFF
[Time Frame: Baseline, Week 52 and Week 104]
- Variazione media rispetto al basale alla Settimana 52 e alla Settimana 104 nella Sqrt dell’area totale della lesione AG nell’occhio in studio, nell’altro occhio e nella combinazione di entrambi gli occhi (indipendentemente dallo stato AG al basale) come misurato mediante AFF
[Time Frame: Baseline, Week 52 and Week 104]
- Variazione percentuale rispetto al basale alla Settimana 52 e alla Settimana 104 nella Sqrt dell’area totale della lesione AG nell’occhio in studio, nell’altro occhio e nella combinazione di entrambi gli occhi (indipendentemente dallo stato AG al basale) come misurato mediante AFF
[Time Frame: Baseline, Week 52 and Week 104]
- Variazione media rispetto al basale alla Settimana 52 e alla Settimana 104 nei punteggi della migliore acuità visiva corretta (BCVA) monoculare nell’occhio in studio e nell’altro occhio, come valutato mediante la tabella dello Studio sulla retinopatia diabetica in trattamento precoce (ETDRS) a quattro metri
[Time Frame: Baseline, Week 52 and Week 104]
- Variazione media rispetto al basale alla Settimana 52 e alla Settimana 104 nei punteggi di acuità visiva a bassa luminanza (LLVA) monoculare nell’occhio in studio e nell’altro occhio, come valutato mediante la tabella ETDRS a quattro metri
[Time Frame: Baseline, Week 52 and Week 104]
- Variazione media rispetto al basale alla Settimana 52 e alla Settimana 104 nel deficit di bassa luminanza nell’occhio in studio e nell’altro occhio
[Time Frame: Baseline, Week 52 and Week 104]
- Variazione media nei punteggi NEI VFQ-25 (questionario sulla funzione visiva del National Eye Institute, versione a 25 voci) rispetto al basale alla Settimana 52 e alla Settimana 104
[Time Frame: Baseline, Week 52 and Week 104]
- Concentrazioni plasmatiche di danicopan nel tempo
[Time Frame: Up to 4 hours postdose]
- Ex vivo attività AP (pathway alternativo) sierica
[Time Frame: Up to 4 hours postdose]
- Concentrazione plasmatica del frammento Bb del fattore B del complemento
[Time Frame: Up to 4 hours postdose]
- Incidenza di eventi avversi emergenti dal trattamento (TEAEs), eventi avversi seri (SAEs) e TEAEs e SAEs oculari, valori clinici di laboratorio anomali, nonché eventi che portano all’interruzione del farmaco dello studio
[Time Frame: Day 1 through Week 104]

E.5.2.1

Timepoint(s) of evaluation of this end point

week 52

settimana 52

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

E.6.4

Safety

Yes

E.6.5

Efficacy

Yes

E.6.6

Pharmacokinetic

Yes

E.6.7

Pharmacodynamic

Yes

E.6.8

Bioequivalence

E.6.9

Dose response

Yes

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

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

E.8.4

The trial involves multiple sites in the Member State concerned

Yes

E.8.4.1

Number of sites anticipated in Member State concerned

E.8.5

The trial involves multiple Member States

Yes

E.8.5.1

Number of sites anticipated in the EEA

E.8.6 Trial involving sites outside the EEA

E.8.6.1

Trial being conducted both within and outside the EEA

Yes

E.8.6.2

Trial being conducted completely outside of the EEA

Information not present in EudraCT

E.8.6.3

If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned

Australia

Canada

Czechia

France

Germany

Hungary

Italy

Japan

Korea, Republic of

Latvia

New Zealand

Slovakia

Spain

United Kingdom

United States

E.8.7

Trial has a data monitoring committee

Yes

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

The end of the study is defined as the date the last patient completes the last visit (including the OLE, Taper, and Follow-up).

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

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)

F.1.1.6

Adolescents (12-17 years)

F.1.2

Adults (18-64 years)

F.1.3

Elderly (>=65 years)

Yes

F.1.3.1

Number of subjects for this age range:

330

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

F.3.3.1

Women of childbearing potential not using contraception

F.3.3.2

Women of child-bearing potential using contraception

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

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

160

F.4.2.2

In the whole clinical trial

330

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)

The Protocol does not define any post-study treatment plan. Patients completing the study may receive commercial Danicopan once available in their country.

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

2022-02-08

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2021-11-10

P. End of Trial
P.	End of Trial Status	Ongoing

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