Clinical Trials Register

Summary
EudraCT Number:	2021-001200-15
Sponsor's Protocol Code Number:	ALXN1210-DM-310
National Competent Authority:	Italy - Italian Medicines Agency
Clinical Trial Type:	EEA CTA
Trial Status:	Prematurely Ended
Date on which this record was first entered in the EudraCT database:	2021-10-18
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

2021-001200-15

A.3

Full title of the trial

A Phase 2/3, Double-Blind, Randomized, Placebo-Controlled, Parallel Group, Multicenter Study to Evaluate the Efficacy and Safety of Ravulizumab in Adult Participants with Dermatomyositis.

Studio multicentrico di fase 2/3, in doppio cieco, randomizzato, controllato con placebo, a gruppi paralleli per valutare l’efficacia e la sicurezza di ravulizumab in partecipanti adulti affetti da dermatomiosite

A.3.1

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

Ravulizumab versus Placebo in Adult Participants with Dermatomyositis.

Ravulizumab versus placebo in partecipanti adulti affetti da dermatomiosite

A.3.2

Name or abbreviated title of the trial where available

Ravulizumab versus Placebo in Adult Participants with Dermatomyositis.

Ravulizumab versus placebo in partecipanti adulti affetti da dermatomiosite

A.4.1

Sponsor's protocol code number

ALXN1210-DM-310

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	+33147100615
B.5.5	Fax number	+33147100611
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	Yes
D.2.1.1.1	Trade name	Ultomiris
D.2.1.1.2	Name of the Marketing Authorisation holder	Alexion Europe SAS
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	Ravulizumab
D.3.2	Product code	[ALXN1210]
D.3.4	Pharmaceutical form	Concentrate for solution for injection/infusion
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Intravenous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	RAVULIZUMAB
D.3.9.1	CAS number	1803171-55-2
D.3.9.2	Current sponsor code	RAVULIZUMAB
D.3.9.3	Other descriptive name	ALXN1210
D.3.9.4	EV Substance Code	SUB172162
D.3.10	Strength
D.3.10.1	Concentration unit	mg/ml milligram(s)/millilitre
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	10
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)	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	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: 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	Ultomiris
D.2.1.1.2	Name of the Marketing Authorisation holder	Alexion Europe SAS
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	Ravulizumab
D.3.2	Product code	[ALXN1210]
D.3.4	Pharmaceutical form	Concentrate for solution for injection/infusion
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Intravenous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	RAVULIZUMAB
D.3.9.1	CAS number	1803171-55-2
D.3.9.2	Current sponsor code	RAVULIZUMAB
D.3.9.3	Other descriptive name	ALXN1210
D.3.9.4	EV Substance Code	SUB172162
D.3.10	Strength
D.3.10.1	Concentration unit	mg/ml milligram(s)/millilitre
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	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)	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	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: 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	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	[NA]
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	NA
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	na
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 W-135 POLYSACCHARIDE CONJUGATED TO TETANUS TOXOID CARRIER PROTEIN
D.3.9.2	Current sponsor code	NA
D.3.9.3	Other descriptive name	NEISSERIA MENINGITIDIS GROUP W-135 POLYSACCHARIDE CONJUGATED TO TETANUS TOXOID CARRIER PROTEIN
D.3.9.4	EV Substance Code	SUB36481
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	NA
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	NA
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: 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	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	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: 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	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.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	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: 7
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	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	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.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	Concentrate for solution for injection/infusion
D.8.4	Route of administration of the placebo	Intravenous use

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

Dermatomyositis

Dermatomiosite

E.1.1.1

Medical condition in easily understood language

Dermatomyositis is a rare disease that causes muscle inflammation and skin problems (rash and swelling).

La dermatomiosite è una malattia rara che provoca infiammazione muscolare e problemi della pelle (eruzione cutanea e gonfiore).

E.1.1.2

Therapeutic area

Diseases [C] - Immune System Diseases [C20]

MedDRA Classification

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

PART A
To determine the effect of ravulizumab compared with placebo in the treatment of DM based on improvement in Total Improvement Score (TIS) as per 2016 ACR/EULAR Myositis Response Criteria for adult DM (also known as IMACS).

PART B
To determine the effect of ravulizumab compared with placebo in the treatment of DM based on improvement in Total Improvement Score (TIS) as per 2016 ACR/EULAR Myositis Response Criteria for adult DM (also known as IMACS)

PARTE A
Determinare l’effetto di ravulizumab rispetto al placebo nel trattamento della DM in base al miglioramento del Punteggio di miglioramento totale (Total Improvement Score, [TIS]) secondo i Criteri di risposta nella miosite ACR/EULAR 2016 per la DM negli adulti (noti anche come IMACS).

PARTE B
Determinare l’effetto di ravulizumab rispetto al placebo nel trattamento della DM in base al miglioramento del Punteggio di miglioramento totale (TIS) secondo i Criteri di risposta nella miosite ACR/EULAR 2016 per la DM negli adulti (noti anche come IMACS).

E.2.2

Secondary objectives of the trial

PART A
To assess the efficacy of ravulizumab compared with placebo in the treatment of DM based on improvement in efficacy endpoints.
To characterize the PK/PD and immunogenicity of ravulizumab in adult participants with DM.
To characterize the overall safety of ravulizumab in participants with DM.

PART B
To assess the efficacy of ravulizumab compared with placebo in the treatment of DM based on improvement in components of IMACS and other myositis activity measures
To assess the efficacy of ravulizumab compared with placebo in the treatment of DM based on other efficacy endpoints
To characterize the PK/PD and immunogenicity of ravulizumab in adult participants with DM
To characterize the overall safety of ravulizumab in participants with DM

PARTE A
Valutare l’efficacia di ravulizumab rispetto al placebo nel trattamento della DM in base al miglioramento degli endpoint di efficacia
Caratterizzare la PK/PD e l’immunogenicità di ravulizumab nei partecipanti adulti con DM
Caratterizzare la sicurezza complessiva di ravulizumab nei partecipanti con DM

PARTE B
Valutare l’efficacia di ravulizumab rispetto al placebo nel trattamento della DM in base al miglioramento delle componenti dell’IMACS e di altre misure di attività della miosite
Valutare l’efficacia di ravulizumab rispetto al placebo nel trattamento della DM in base ad altri endpoint di efficacia
Caratterizzare la PK/PD e l’immunogenicità di ravulizumab nei partecipanti adulti con DM

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

Participants are eligible to be included in the study only if all of the following criteria apply:
1. 18 years of age or older at the time of signing the informed consent.
2.Body weight = 30 kg at the time of Screening.
3. Male or female.
4. Diagnosis: Meet American College of Rheumatology/European League Against Rheumatism (ACR/EULAR) classification criteria for definite or probable DM (Lundberg, 2017).
5. Participants who have an inadequate response (ie, continued impairment by medical doctor report) or are intolerant to 2 or more DM treatments, including systemic corticosteroids or ISTs (eg, azathioprine, methotrexate, rituximab, IVIg), either in combination or as monotherapy.
6. Vaccinated against Neisseria meningitidis within 3 years prior to, or at the time of, initiating ravulizumab. Participants who initiate study drug treatment less than 2 weeks after receiving a meningococcal vaccine must receive appropriate prophylactic antibiotics until 2 weeks after the vaccination.
7. Female participants of childbearing potential and male participants must follow specified contraception guidance as described in the protocol.

I partecipanti sono idonei all’inclusione nello studio solo se soddisfano tutti i seguenti criteri:
1. Età =18 anni al momento della firma del consenso informato.
2. Peso corporeo =30 kg al momento dello screening.
3. Di ambo i sessi.
4. Diagnosi: soddisfacimento dei criteri di classificazione ACR/EULAR 2017 per DM certa o probabile (Lundberg, 2017).
5. Partecipanti che presentano una risposta inadeguata o sono intolleranti a 2 o più trattamenti per la DM, compresi glucocorticoidi sistemici o terapie immunosoppressive (TIS; ad es., azatioprina, metotrexato, rituximab, immunoglobuline per via endovenosa [Intravenous Immunoglobulin, IVIg]), in combinazione o in monoterapia.
6. Partecipanti in trattamento con glucocorticoidi e/o TIS che sono in terapia stabile come descritto nella Tabella 11 del protocollo e che saranno in grado continuare ad assumere la terapia stabile per tutto il periodo randomizzato controllato. Partecipanti in trattamento con glucocorticoidi e/o TIS che completano un periodo di washout appropriato per questi trattamenti prima della visita del Giorno 1, come descritto nella Tabella 12 (ad es., periodo di washout di 6 mesi per rituximab; periodo di washout di 3 mesi per IVIg o immunoglobuline per via sottocutanea [SCIg]).
7. Le partecipanti di sesso femminile in età fertile e i partecipanti di sesso maschile devono seguire le linee guida per la contraccezione specificate come descritto nel protocollo.

E.4

Principal exclusion criteria

1. Cancer-associated myositis, defined as the diagnosis of myositis within 3 years of the diagnosis of cancer (except basal or squamous cell skin cancer or carcinoma in situ of the cervix that has been excised and cured and at least 3 months before Screening).
2. Evidence of active malignant disease or malignancies diagnosed within the previous 5 years (including hematological malignancies and solid tumors).
3. Participants with other forms of myositis
4. Participants with significant muscle damage (eg, severe muscle atrophy, end stage muscle disease) as per investigator opinion.
5. History of Neisseria meningitidis infection.
6. Human immunodeficiency virus (HIV) infection (evidenced by HIV Type 1 or Type 2 antibody titer).
7. Active systemic bacterial, viral, or fungal infection within 14 days prior to ravulizumab administration.
8. Presence of fever = 38°C (100.4°F) within 7 days prior to study drug administration on Day 1.
9. History of hypersensitivity to murine proteins or to 1 of the excipients of ravulizumab.
10. Pregnant, breastfeeding, or intending to conceive during the course of the study.
11. Inability or unwillingness to adhere to the protocol requirements

1. Miosite associata a tumore, definita come la diagnosi di miosite entro 3 anni dalla diagnosi di tumore (fatta eccezione per carcinoma cutaneo basocellulare o squamocellulare o carcinoma in situ della cervice che è stato asportato e curato e almeno 3 mesi prima dello screening).
2. Evidenza di malattia maligna attiva o neoplasie diagnosticate nei 5 anni precedenti (compresi neoplasie ematologiche e tumori solidi e fatta eccezione per carcinoma cutaneo basocellulare o squamocellulare o carcinoma in situ della cervice che è stato asportato e curato almeno 3 mesi prima dello screening).
3. Partecipanti con altre forme di miosite: diagnosi clinica di miosite da corpi inclusi, polimiosite, miopatia necrotizzante, miosite/miopatia farmaco-indotta, sindrome anti-sintetasi senza DM, miosite associata a tumore (miosite diagnosticata entro 3 anni prima o dopo una diagnosi di neoplasia maligna, fatta eccezione per carcinoma a cellule squamose della pelle, carcinoma a cellule basali, carcinoma in situ con lesioni in qualsiasi area o carcinoma cervicale in situ), miosite con malattia sovrapposta del tessuto connettivo come lupus eritematoso sistemico (LES), artrite reumatoide o sclerosi sistemica. Sono ammessi i partecipanti con sindrome di Sjogren secondaria.
4. Partecipanti con danno muscolare significativo (ad es., atrofia muscolare grave, malattia muscolare allo stadio terminale, RMI con atrofia grave o sostituzione di tessuto fibro-adiposo) secondo il parere dello sperimentatore.
5. Anamnesi di infezione da N meningitidis.
6. Infezione da virus dell’immunodeficienza umana (Human Immunodeficiency Virus, [HIV]) (evidenziata da titolo anticorpale anti-HIV di tipo 1 o tipo 2).
7. Infezione sistemica batterica, virale o micotica attiva nei 14 giorni precedenti la somministrazione di ravulizumab.
8. Presenza di febbre =38 °C nei 7 giorni precedenti la somministrazione del trattamento dello studio il Giorno 1.
9. Anamnesi di ipersensibilità a proteine murine o a 1 degli eccipienti di ravulizumab.
10. Gravidanza, allattamento o intenzione di concepire durante il corso dello studio.
11. Incapacità o riluttanza ad attenersi ai requisiti del protocollo

E.5 End points

E.5.1

Primary end point(s)

PART A
Proportion of participants with a = 20-point improvement response on TIS (TIS20) at Week 26 of the Randomized-Controlled Period

PART B
Proportion of participants with a = 20-point improvement response on TIS (TIS20) at Week 50 of the Randomized-Controlled Period

PARTE A
Percentuale di partecipanti con una risposta di miglioramento =20 punti nel punteggio IMACS-TIS (TIS20) alla Settimana 26 del periodo randomizzato controllato

PARTE B
Percentuale di partecipanti con una risposta di miglioramento =20 punti nel punteggio IMACS-TIS (TIS20) alla Settimana 50 secondo lo stimatore composito definito del periodo randomizzato controllato

E.5.1.1

Timepoint(s) of evaluation of this end point

Part A: Week 26
Part B: Week 50

Part A: Settimana 26
Part B: Settimana 50

E.5.2

Secondary end point(s)

PART A
• Mean TIS at Week 26
• Mean change from baseline in CDASI Activity Score at Week 26
• Change from baseline of IMACS core set measures at Week 26
• Time to first CDASI Activity Score improvement
• Change in Investigator Global Assessment (IGA) at Week 26

PART B
• Mean TIS at Week 50
• Mean change from baseline in MMT-8 at Week 50
• Mean change from baseline in extra-muscular disease activity based on MDAAT at Week 50
• Mean change from baseline in CDASI Activity Score at Week 50
• Mean change from baseline in individual 6 CSM used for TIS calculation
• Time to first CDASI Activity Score improvement

PARTE A
- TIS medio alla Settimana 26
• Variazione media rispetto al basale nel Punteggio di attività CDASI alla Settimana 26
• Variazione rispetto al basale di ciascuna delle misure della serie principale IMACS alla Settimana 26
• Tempo al primo miglioramento (differenze minime clinicamente importanti [Minimally Clinically Important Difference, MCID] = miglioramento di 7 punti) del Punteggio di attività CDASI
• Percentuale di partecipanti con miglioramento del punteggio MCID CDASI alla Settimana 26

PARTE B
Variazione media rispetto al basale nella valutazione dell’attività globale da parte del paziente alla Settimana 50
• Variazione media rispetto al basale nella valutazione dell’attività globale da parte del medico alla Settimana 50
• Variazione media rispetto al basale nell’HAQ alla Settimana 50
• Variazione media rispetto al basale dei valori degli enzimi muscolari alla Settimana 50
• TIS medio ad ogni visita dalla Settimana 2 alla Settimana 50
• Percentuale di partecipanti con TIS20 ad ogni visita

E.5.2.1

Timepoint(s) of evaluation of this end point

week 26

settimana 26

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

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)

Yes

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

Japan

Korea, Republic of

United States

France

Germany

Italy

Spain

United Kingdom

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

A participant is considered to have completed the study if the participant has completed all periods (Randomized-Controlled Period and OLE Period) of the study including the last scheduled procedure shown in the SoA (Section 1.3) of the protcol.
The end of the study is defined as the date the last participant completes the last visit shown in the SoA.

Si considera che un partecipante abbia completato lo studio se ha completato tutti i periodi (periodo controllato randomizzato e periodo OLE) dello studio, inclusa l'ultima procedura programmata indicata nella SoA (Sezione 1.3) del protocollo.
La fine dello studio è definita come la data in cui l'ultimo partecipante completa l'ultima visita indicata nella SoA.

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:

180

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

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

180

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)

This study will be conducted in 2 parts: Part A (Phase 2) and Part B (Phase 3). An OLE period was chosen to ensure that all participants in this study will have the opportunity to receive active treatment after the completion of the Randomized-Controlled Period of the study. This OLE period also allows for further evaluation of longer-term safety and efficacy of the study intervention (74 weeks).

Questo studio sarà condotto in 2 parti: Parte A (Fase 2) e Parte B (Fase 3). È stato scelto un periodo OLE per garantire che tutti i partecipanti a questo studio abbiano l'opportunità di ricevere un trattamento attivo dopo il completamento del Randomized-Controlled Period dello studio. Questo periodo OLE consente anche un'ulteriore valutazione della sicurezza e dell'efficacia a lungo termine dell'intervento dello studio (74 settimane).

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

2021-08-16

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2021-07-28

P. End of Trial
P.	End of Trial Status	Prematurely Ended
P.	Date of the global end of the trial	2023-12-18

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