Clinical Trials Register

Summary
EudraCT Number:	2021-001229-26
Sponsor's Protocol Code Number:	ALXN2050-MG-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:	2021-10-19
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

2021-001229-26

A.3

Full title of the trial

A Phase 2, Randomized, Double Blind, Placebo Controlled Multicenter Study to Evaluate the Efficacy and Safety of ALXN2050 in Adult Participants with
Generalized Myasthenia Gravis

Studio di fase 2, multicentrico, randomizzato, in doppio cieco, controllato con placebo, per valutare l’efficacia e la sicurezza di ALXN2050 in partecipanti adulti con miastenia gravis generalizzata

A.3.1

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

Phase 2 Study of ALXN2050 in Adult Participants with Generalized Myasthenia Gravis

Studio di fase 2 su ALXN2050 in partecipanti adulti con miastenia gravis generalizzata

A.3.2

Name or abbreviated title of the trial where available

A.4.1

Sponsor's protocol code number

ALXN2050-MG-201

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 Pharmaceuticals Inc.
B.5.2	Functional name of contact point	Clinical Development Sciences
B.5.3	Address:
B.5.3.1	Street Address	121 Seaport Boulevard
B.5.3.2	Town/ city	Boston
B.5.3.3	Post code	MA 02210
B.5.3.4	Country	United States
B.5.4	Telephone number	000000
B.5.5	Fax number	000000
B.5.6	E-mail	marcus.yountz@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	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	[N/A]
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	N/A
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	N/A
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	N/A
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	N/A
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: 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	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	[N/A]
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	N/A
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.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	Solution for injection/infusion 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	N/A
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	N/A
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	N/A
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	N/A
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	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	[N/A]
D.3.4	Pharmaceutical form	Powder and solvent for solution 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	N/A
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	N/A
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: 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	Nimenrix
D.2.1.1.2	Name of the Marketing Authorisation holder	Pfizer Europe MA EEIG EU/1/12/767/001-004
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	Nimenrix
D.3.2	Product code	[N/A]
D.3.4	Pharmaceutical form	Powder and solvent for solution 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	tetanus protein
D.3.9.2	Current sponsor code	N/A
D.3.9.3	Other descriptive name	tetanus protein
D.3.9.4	EV Substance Code	SUB35034
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	44
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	NEISSERIA MENINGITIDIS GROUP A POLYSACCHARIDE CONJUGATED WITH TETANUS TOXOID CARRIER PROTEIN
D.3.9.2	Current sponsor code	N/A
D.3.9.3	Other descriptive name	NEISSERIA MENINGITIDIS GROUP A POLYSACCHARIDE CONJUGATED WITH TETANUS TOXOID CARRIER PROTEIN
D.3.9.4	EV Substance Code	SUB36479
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	NEISSERIA MENINGITIDIS GROUP Y POLYSACCHARIDE CONJUGATED TO TETANUS TOXOID CARRIER PROTEIN
D.3.9.2	Current sponsor code	N/A
D.3.9.3	Other descriptive name	NEISSERIA MENINGITIDIS GROUP Y POLYSACCHARIDE CONJUGATED TO TETANUS TOXOID CARRIER PROTEIN
D.3.9.4	EV Substance Code	SUB36482
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	NEISSERIA MENINGITIDIS SEROGROUP C POLYSACCHARIDE (PSC) CONJUGATED TO TETANUS TOXOID (TT)
D.3.9.2	Current sponsor code	N/A
D.3.9.3	Other descriptive name	NEISSERIA MENINGITIDIS SEROGROUP C POLYSACCHARIDE (PSC) CONJUGATED TO TETANUS TOXOID (TT)
D.3.9.4	EV Substance Code	SUB31471
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: 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	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	ALXN2050
D.3.2	Product code	[-]
D.3.4	Pharmaceutical form	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.3.9.1	CAS number	208617-00-7
D.3.9.2	Current sponsor code	ALXN2050
D.3.9.3	Other descriptive name	(1R,3S,5R)-2-(2-(3-acetyl-5-(2-methylpyrimidin-5-yl)-1H-indazol-1-yl)acetyl)-N-(6- bromo-3-methylpyridin-2-yl)-5-methyl-2-azabicyclo[3.1.0]hexane-3-carboxamide
D.3.9.4	EV Substance Code	SUB204167
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	60
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

Myasthenia Gravis

Miastenia gravis

E.1.1.1

Medical condition in easily understood language

Generalized Myasthenia Gravis

Miastenia gravis generalizzata

E.1.1.2

Therapeutic area

Diseases [C] - Immune System Diseases [C20]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	21.1
E.1.2	Level	PT
E.1.2	Classification code	10028417
E.1.2	Term	Myasthenia gravis
E.1.2	System Organ Class	10029205 - Nervous system disorders

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

To assess the efficacy of ALXN2050 compared with placebo in the treatment of generalized MG (gMG) based on improvement in the Myasthenia Gravis Activities of Daily Living (MG ADL) total score.

Valutare l’efficacia di ALXN2050 rispetto al placebo nel trattamento della MG generalizzata (gMG) in base al miglioramento del punteggio totale di attività della vita quotidiana nella miastenia gravis (Myasthenia Gravis Activities of Daily Living, [MG-ADL])

E.2.2

Secondary objectives of the trial

- To assess the efficacy of ALXN2050 compared with placebo in the treatment of gMG based on improvement in the Quantitative Myasthenia
Gravis (QMG) total score
- To assess the efficacy of ALXN2050 compared with placebo in the treatment of gMG based on improvement in quality of life measures
- To assess the efficacy of ALXN2050 compared with placebo in the treatment of gMG based on additional endpoints involving the MG ADL
total score
PK/PD
-To characterize the PK/PD of ALXN2050 and to establish the PK/PD relationship in participants with gMG
Biomarker:
-To assess the effect of factor D inhibition on complement biomarkers
Safety:
-To characterize the overall safety of ALXN2050 compared with placebo in participants with gMG

- Valutare l’efficacia di ALXN2050 rispetto al placebo nel trattamento della gMG in base al miglioramento del punteggio totale della miastenia gravis quantitativa (Quantitative Myasthenia Gravis, [QMG])
- Valutare l’efficacia di ALXN2050 rispetto al placebo nel trattamento della gMG in base al miglioramento delle misure di qualità della vita
- Valutare l’efficacia di ALXN2050 rispetto al placebo nel trattamento della gMG sulla base di endpoint aggiuntivi che coinvolgono il punteggio totale di MG-ADL
PK/PD
- Caratterizzare la farmacocinetica/farmacodinamica (pharmacokinetcs/pharmacodynamics, [PK/PD]) di ALXN2050 e stabilire il rapporto PK/PD nei partecipanti affetti da gMG
Biomarcatori
- Valutare l’effetto dell’inibizione del fattore D sui biomarcatori del complemento
Sicurezza
- Caratterizzare la sicurezza complessiva di ALXN2050 rispetto al placebo nei partecipanti affetti da gMG

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

• Diagnosed with MG at least 3 months (90 days) prior to the date of the Screening Visit.
• Myasthenia Gravis Foundation of America Clinical Classification Class II to IV at the Screening Visit.
• MG-ADL total score must be = 5 (with at least 50% of the score attributed to non-ocular elements) at the Screening Visit and at randomization (Day 1).
• Participants receiving treatment with protocol-specified immunosuppressive therapies, corticosteroids, or acetylcholinesterase inhibitors must have been receiving treatment and on a stable dose prior to the date of the Screening Visit, with no changes to the regimen expected during screening, the PEP, and/or the ETP.

• Diagnosi di MG formulata almeno 3 mesi (90 giorni) prima della data della visita di screening.
• Classificazione clinica di classe da II a IV alla visita di screening secondo la Fondazione americana per la miastenia gravis.
• Il punteggio totale di MG-ADL deve essere =5 (almeno il 50% del punteggio deve essere attribuito agli elementi non oculari) alla visita di screening e alla randomizzazione (Giorno 1).
• I partecipanti che ricevono il trattamento con terapie immunosoppressive specificate dal protocollo, corticosteroidi o inibitori della acetilcolinesterasi, devono averlo ricevuto e in una dose stabile prima della data della visita di screening, senza alcuna modifica al regime previsto durante lo screening, la
profilassi post-esposizione (Post-Exposure Prophylaxis, [PEP]) e/o il periodo di estensione del trattamento (Extended Treatment Period, [ETP]).

E.4

Principal exclusion criteria

• Estimated glomerular filtration rate = 30 milliliters/minute/1.73 squared meters during Screening calculated by Chronic Kidney Disease Epidemiology Collaboration.
• History of thymectomy, thymomectomy, or any other thymic surgery within 12 months prior to the Screening Visit.
• Any untreated thymic malignancy, carcinoma, or thymoma. Participants with a history of treated thymic malignancy or carcinoma are eligible for enrollment if they meet pre-specified conditions outlined in the protocol.
• Clinical features consistent with Clinical Deterioration at the time of the Screening Visit or at any time during the Screening Period prior to randomization (Day 1).
• Use of the following within the time periods specified below:
a. Intravenous immunoglobulin G or subcutaneous immunoglobulin within the 4 weeks (28 days) prior to the Screening Visit.
b. Use of tacrolimus or cyclosporine within the 4 weeks (28 days) prior to the Screening Visit.

• Velocità di filtrazione glomerulare stimata = 30 millilitri/minuto/1,73 metri quadrati durante lo screening calcolata mediante collaborazione sull’epidemiologia della malattia renale cronica.
• Anamnesi di timectomia, timomectomia o qualsiasi altro intervento chirurgico timico nei 12 mesi precedenti la visita di screening.
• Qualsiasi tumore maligno timico, carcinoma o timoma non trattato. I partecipanti con anamnesi di tumore maligno timico o carcinoma trattati sono idonei per l’arruolamento se soddisfano le condizioni pre-specificate evidenziate nel protocollo.
• Caratteristiche cliniche che sono coerenti con il peggioramento clinico al momento della visita di screening o in qualsiasi momento durante il periodo di screening prima della randomizzazione (Giorno 1).
• Uso di quanto segue nei periodi di tempo specificati di seguito:
a. Immunoglobulina G per via endovenosa o immunoglobulina sottocutanea nelle 4 settimane (28 giorni) precedenti la visita di screening.
b. Uso di tacrolimus o ciclosporina nelle 4 settimane (28 giorni) precedenti la visita di screening.

E.5 End points

E.5.1

Primary end point(s)

1. Proportion of participants with a Myasthenia Gravis Activities of Daily Living (MG-ADL) total score reduction of = 2 points in any 4 consecutive weeks during the first 8 weeks and who did not receive rescue therapy

1. Percentuale di partecipanti con una riduzione del punteggio totale di MG-ADL =2 punti in un periodo qualsiasi di 4 settimane consecutive durante le prime 8 settimane e che non ha ricevuto la terapia di salvataggio

E.5.1.1

Timepoint(s) of evaluation of this end point

Any 4 consecutive weeks during first 8 weeks

Un periodo qualsiasi di 4 settimane consecutive durante le prime 8 settimane

E.5.2

Secondary end point(s)

2. Change from baseline in QMG total score at Week 8
3. Proportion Of Participants Meeting Prespecified Threshold In The QMG Total Score at Week 8
4. Proportion Of Participants Meeting Prespecified Threshold In The QMG Total Score In Any 4 Consecutive Weeks During The First 8 Weeks And
Who Did Not Receive Rescue Therapy
5. Change From Baseline In MG-ADL Total Score At Week 8
6. Proportion Of Participants Meeting Prespecified Threshold In The MGADL Total Score In Any 4 Consecutive Weeks During The First 8 Weeks
And Who Did Not Receive Rescue Therapy
7. Proportion Of Participants Meeting Prespecified Threshold In The MGADL Total Score At Week 8
8. Change From Baseline In Quality Of Life In Neurological Disorders Fatigue Questionnaire (Neuro-QoL) Fatigue Score At Week 8
9. Maximum Peak Plasma Concentration (Cmax) Of ALXN2050 Over Time
10. Pre-dose Concentration (Ctrough) Of ALXN2050 Over Time
11. Absolute Values And Change From Baseline In Plasma Concentration Of Bb Fragment Of Complement Factor B At Week 8
12. Absolute Values And Change From Baseline In Serum Alternative Pathway Activity At Week 8
13. Plasma Factor D Concentration Over Time
14. Serum Complement Component 3 Concentration Over Time
15. Serum Classical Pathway Activity Over Time

2. Variazione rispetto al basale del punteggio totale della QMG alla Settimana 8
3. Percentuale di partecipanti che soddisfano una soglia prespecificata nel punteggio totale della Scala QMG alla Settimana 8
4. Percentuale di partecipanti che soddisfano una soglia prespecificata nel punteggio totale della QMG in un periodo qualsiasi di 4 settimane consecutive durante le prime 8 settimane e che non ha ricevuto la terapia di salvataggio
5. Variazione rispetto al basale del punteggio totale di MG-ADL alla Settimana 8
6. Percentuale di partecipanti che soddisfano una soglia prespecificata nel punteggio totale delle MG-ADL in un periodo qualsiasi di 4 settimane consecutive durante le prime 8 settimane e che non ha ricevuto la terapia di salvataggio
7. Percentuale di partecipanti che soddisfano una soglia prespecificata nel punteggio totale delle MGADL alla Settimana 8
8. Variazione rispetto al basale del punteggio del questionario sulla qualità della vita nei disturbi neurologici in relazione all’affaticamento (Neuro-QoL) alla Settimana 8
9. Concentrazione plasmatica massima di picco (Cmax) di ALXN2050 nel tempo
10. Concentrazione pre-dose (Ctrough) di ALXN2050 nel tempo
11. Valori assoluti e variazione rispetto al basale nella concentrazione plasmatica del frammento Bb del fattore B del complemento alla Settimana 8
12. Valori assoluti e variazione rispetto al basale nell’attività della via alternativa sierica alla Settimana 8
13. Concentrazione plasmatica del fattore D nel tempo
14. Concentrazione sierica del componente 3 del complemento nel tempo
15. Attività della via classica sierica nel tempo

E.5.2.1

Timepoint(s) of evaluation of this end point

- week 8
- baseline through week 8

- settimana 8
- dal basale alla settimana 8

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

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

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

Canada

Korea, Republic of

Taiwan

United States

Germany

Italy

Spain

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

The date of the last visit of the last randomized participant in the study

La data dell’ultima visita dell’ultimo partecipante randomizzato allo studio

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)

Yes

F.1.2.1

Number of subjects for this age range:

F.1.3

Elderly (>=65 years)

Yes

F.1.3.1

Number of subjects for this age range:

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

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)

None

Nessuno

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

2021-12-21

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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Orphan Designation Number:
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