Clinical Trials Register

Summary
EudraCT Number:	2020-002637-15
Sponsor's Protocol Code Number:	APL2-C3G-204
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:	2021-06-04
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

2020-002637-15

A.3

Full title of the trial

An Open-Label, Randomized, Controlled, Phase 2 Study to Evaluate the Safety and Efficacy of Pegcetacoplan in the Treatment of Post-Transplant Recurrenceof C3G or IC-MPGN

Studio randomizzato, controllato, in aperto, di fase 2 per valutare la sicurezza e l'efficacia di pegcetacoplan nel trattamento della recidiva post-trapianto di C3G o IC-MPGN

A.3.1

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

Phase 2, multicenter, open-label, randomized, controlled study is designed to evaluate the safety and efficacy of APL-2 in patients who have post-transplant recurrence of C3G or IC-MPGN

Studio randomizzato, controllato, in aperto, di fase 2, progettato per valutare la sicurezza e l'efficacia di APL-2 in pazienti che hanno recidive post-trapianto di C3G o IC-MPGN

A.3.2

Name or abbreviated title of the trial where available

NOBLE

A.4.1

Sponsor's protocol code number

APL2-C3G-204

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	APELLIS PHARMACEUTCIALS, INC.
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	Apellis 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	Apellis Pharmaceuticals, Inc.
B.5.2	Functional name of contact point	Clin. Development and Med. Affairs
B.5.3	Address:
B.5.3.1	Street Address	100 5th Avenue, 3rd Floor
B.5.3.2	Town/ city	Waltham
B.5.3.3	Post code	MA 02451
B.5.3.4	Country	United States
B.5.6	E-mail	federico@apellis.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	Pegcetacoplan
D.3.2	Product code	[APL-2]
D.3.4	Pharmaceutical form	Solution for injection/infusion
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Subcutaneous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	Pegcetacoplan
D.3.9.1	CAS number	2019171-69-6
D.3.9.2	Current sponsor code	APL-2
D.3.9.4	EV Substance Code	SUB195466
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	1080
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	Yes
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	Prevenar 13 suspension for injection
D.2.1.1.2	Name of the Marketing Authorisation holder	Pfizer Limited
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	Prevenar 13 suspension for injection
D.3.2	Product code	[na]
D.3.4	Pharmaceutical form	Suspension 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	Pneumococcal polysaccharide serotype 1, 3, 4, 5, 6A, 6B, 7F, 9V, 14, 18C, 19A, 19F, 23F
D.3.9.2	Current sponsor code	na
D.3.9.3	Other descriptive name	Pneumococcal polysaccharide serotype 11 2.2 µg Pneumococcal polysaccharide serotype 31 2.2 µg Pneumococcal polysaccharide serotype 41 2.2 µg Pneumococcal polysaccharide serotype 51 2.2 µg Pneumococcal polysaccharide serotype 6A1 2.2 µg Pneumococcal polysaccharide serotype 6B1 4.4 µg Pneumococcal polysaccharide serotype 7F1 2.2 µg Pneumococcal polysaccharide serotype 9V1 2.2 µg Pneumococcal polysaccharide serotype 141 2.2 µg Pneumococcal polysaccharide serotype 18C1 2.2 µg Pneumococcal polysaccha
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	31
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	Pneumococcal polysaccharide vaccine
D.2.1.1.2	Name of the Marketing Authorisation holder	Merck Sharp & Dohme Limited
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	Pneumococcal polysaccharide vaccine solution for injection in a vial
D.3.2	Product code	[na]
D.3.4	Pharmaceutical form	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	Pneumococcal Polysaccharide Vaccine
D.3.9.2	Current sponsor code	na
D.3.9.3	Other descriptive name	The 0.5 mL dose of vaccine contains 25 micrograms of each of the following 23 pneumococcal polysaccharide serotypes: 1, 2, 3, 4, 5, 6B, 7F, 8, 9N, 9V, 10A, 11A, 12F, 14, 15B, 17F, 18C, 19F, 19A, 20, 22F, 23F, 33F.
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.3.11 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	GlaxoSmithKline UK
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 Meningococcal Group B vaccine for injection in pre-filled syringe
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	Recombinant Neisseria meningitidis group B NHBA fusion protein
D.3.9.2	Current sponsor code	na
D.3.9.4	EV Substance Code	AS14
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.3.9.4	EV Substance Code	AS15
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.3.9.4	EV Substance Code	AS16
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	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	Ciprofloxacin 500mg Film-Coated Tablets
D.2.1.1.2	Name of the Marketing Authorisation holder	Accord Healthcare Limited
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	Ciprofloxacin 500mg Film-Coated Tablets
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	ciprofloxacin hydrochloride
D.3.9.1	CAS number	0085721-33-1
D.3.9.2	Current sponsor code	na
D.3.9.4	EV Substance Code	SUB07470MIG
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	Hiberix
D.2.1.1.2	Name of the Marketing Authorisation holder	GlaxoSmithKline (Ireland) Limited
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	Hiberix
D.3.2	Product code	[na]
D.3.4	Pharmaceutical form	Powder and suspension for suspension 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	Haemophilus influenza coupled to tetanus toxoid
D.3.9.2	Current sponsor code	na
D.3.9.3	Other descriptive name	Each 0.5 ml dose of the vaccine contains 10 micrograms of purified capsular polysaccharide of Haemophilus type b covalently bound to approximately 25 micrograms tetanus toxoid
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	35
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	Phenoxymethylpenicillin potassium
D.2.1.1.2	Name of the Marketing Authorisation holder	Medreich Plc
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	Phenoxymethylpenicillin potassium TABLETS BP 250 mg
D.3.2	Product code	[na]
D.3.4	Pharmaceutical form	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	PHENOXYMETHYLPENICILLIN POTASSIUM
D.3.9.1	CAS number	132-98-9
D.3.9.2	Current sponsor code	na
D.3.9.4	EV Substance Code	SUB03766MIG
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	250
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: 8
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 Limited
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 powder and solvent for solution for injection in pre-filled syringe
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 A polysaccharide
D.3.9.2	Current sponsor code	na
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 C polysaccharide
D.3.9.2	Current sponsor code	na
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
D.3.9.2	Current sponsor code	na
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
D.3.9.2	Current sponsor code	na
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)	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)	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

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

complement 3 glomerulopathy (C3G)/immune complex membranoproliferative glomerulonephritis (IC-MPGN)

glomerulopatia da complemento 3 (C3G) / glomerulonefrite membranoproliferativa a depositi di immunocomplessi (IC-MPGN)

E.1.1.1

Medical condition in easily understood language

Rare kidney disease which can reoccur after a kidney transplant.

Malattia renale rara che può ripresentarsi dopo un trapianto renale.

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	20.1
E.1.2	Level	LLT
E.1.2	Classification code	10063210
E.1.2	Term	Transplant glomerulopathy
E.1.2	System Organ Class	100000004870

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 evaluate the efficacy of pegcetacoplan in improving the underlying pathophysiology of complement 3 glomerulopathy (C3G)/immune complex membranoproliferative glomerulonephritis (IC-MPGN) after 12 weeks of treatment.

Valutare l'efficacia di pegcetacoplan sul miglioramento della fisiopatologia sottesa della glomerulopatia da complemento 3 (C3G) o della glomerulonefrite membranoproliferativa a depositi di immunocomplessi (IC-MPGN) dopo 12 settimane di trattamento.

E.2.2

Secondary objectives of the trial

To evaluate the safety of pegcetacoplan for up to 52 weeks in patients with recurrent C3G/IC-MPGN in a renal allograft.
To evaluate the effect of pegcetacoplan on key clinical manifestations of the disease after 52 weeks of treatment.

Valutare le sicurezza di pegcetacoplan per un periodo fino a 52 settimane in pazienti che presentano recidiva di C3G/IC-MPGN in un allotrapianto di rene.
Valutare l'effetto di pegcetacoplan sulle manifestazioni cliniche fondamentali dopo 52 settimane di trattamento.

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

Individuals must meet all of the following criteria at screening visits to be included in the study:
1. Patients of at least 18 years of age at screening
2. Patients must have clinical and pathologic evidence of recurrent C3G or IC-MPGN, as evidenced by all of the following:a. A diagnosis of C3G or IC-MPGN, with at least 2+ staining for C3c in therenal allograft, confirmed by a central pathologist, based on the screening renal allograft biopsyb. Clinical evidence of C3G or IC-MPGN recurrence in the form of at least 1 g/day of proteinuria in a 24-hour urine collection that is attributable todisease recurrence in the opinion of the investigatorc. C3G or IC-MPGN must be primary and not secondary to another condition (eg, infection, malignancy, monoclonal gammopathy, autoimmunity, chronic antibody-mediated rejection, chronic thrombotic microangiopathy, or a medication)
3. Stable (not improving) or worsening disease, in the opinion of the investigator, in the 2 months preceding the first dose of pegcetacoplan
4. eGFR =30 mL/min/1.73 m2, calculated by the Chronic Kidney Disease–Epidemiology Collaboration (CKD-EPI) creatinine equation for adults
5. No more than 50% glomerulosclerosis or interstitial fibrosis on the screening renal biopsy
6. Stable regimen for recurrent C3G/IC-MPGN for at least 4 weeks prior to the screening renal allograft biopsy and from the time of the screening renal allograft biopsy until randomization
7. Willing to receive required vaccinations against N. meningitides, S. pneumoniae, and H. influenzae if applicable vaccination records are not available
8. Women of childbearing potential (WOCBP) must have a negative bloodpregnancy test at screening (and negative urine pregnancy at Visit 4) and must agree to use protocoldefined methods of contraception from screening through 12 weeks after receiving last dose of pegcetacoplan
9. Men must agree to use protocol-defined methods of contraception andagree to refrain from donating semen from screening through 12 weeks after receiving last dose of pegcetacoplan
10. Willing and able to provide written informed consent
11. Able to understand and willing to comply with all scheduled procedures and other requirements of the study in the opinion of the investigator
12. Willing and able to self-administer pegcetacoplan or have an identified caregiver who can perform the administration

Per essere ammessi allo studio, alle visite di screening i soggetti devono soddisfare tutti i criteri elencati di seguito.
1. Pazienti di almeno 18 anni di età allo screening
2. I pazienti devono presentare evidenza clinica e patologica di recidiva di C3G o IC-MPGN, confermata da:
a. diagnosi di C3G o IC-MPGN, con almeno 2+ colorazioni per C3c nell'allotrapianto renale, confermata da un patologo centrale, sulla base della biopsia di screening dell'allotrapianto renale
b. evidenza clinica di recidiva di C3G o IC-MPGN sotto forma di almeno 1 g/giorno di proteinuria nella raccolta delle urine delle 24 ore
c. la C3G o l'IC-MPGN deve essere una patologia primaria e non secondaria a un'altra patologia (per. es., infezione, neoplasia maligna, gammopatia monoclonale, autoimmunità, rigetto cronico anticorpo-mediato, microangiopatia trombotica cronica o da un farmaco)
3. Malattia stabile (che non migliora) o in peggioramento, secondo l'opinione dello sperimentatore, nei 2 mesi precedenti alla somministrazione della prima dose di pegcetacoplan
4. eGFR = 30 mL/min/1,73 m2, calcolata mediante l'equazione CKD-EPI (Chronic Kidney Disease–Epidemiology Collaboration) per la creatinina negli adulti
5. Non oltre il 50% di glomerulosclerosi o fibrosi interstiziale sul campione bioptico di screening dell'allotrapianto renale
6. Regime stabile per recidiva di C3G/IC-MPGN perlomeno nelle 4 settimane precedenti alla biopsia di screening dell'allotrapianto renale e a partire dal momento di tale biopsia di screening fino alla randomizzazione
7. Disponibilità a essere vaccinati contro Neisseria meningitidis, Streptococcus pneumoniae e Haemophilus influenzae se il tesserino personale delle vaccinazioni applicabili non fosse disponibile
8. Le donne in età fertile devono risultare negative al test di gravidanza nel sangue allo screening (e al test di gravidanza nelle urine alla Visita 4); devono inoltre accettare di far uso di metodi contraccettivi definiti dal protocollo dal momento dello screening fino al 12 settimane dopo la somministrazione dell'ultima dose di pegcetacoplan
9. Gli uomini devono accettare di far uso di metodi contraccettivi definiti dal protocollo e di astenersi dalla donazione di sperma a partire dallo screening fino al 12 settimane dopo la somministrazione dell'ultima dose di pegcetacoplan
10. Pazienti disposti e in grado di accordare il consenso informato scritto.
11. Pazienti in grado di comprendere e disposti ad aderire alle indagini programmate dello studio e agli altri requisiti dello studio, secondo l'opinione dello sperimentatore
12. Pazienti disposti e capaci di autosomministrarsi pegcetacoplan o assistiti da un caregiver identificato che possa eseguire la somministrazione

E.4

Principal exclusion criteria

Individuals meeting any of the following criteria at screening or baselineare ineligible to participate in this study:
1. Absolute neutrophil count <1000 cells/mm3 during screening (not including Day 1)
2. Previous treatment with pegcetacoplan
3. Evidence of rejection on the screening renal allograft biopsy that requires treatment
4. Diagnosis or history of HIV, hepatitis B, or hepatitis C infection or positive serology at screening indicative of infection
5. Malignancy, except for the following:a. Cured basal or squamous cell skin cancerb. Curatively treated in situ diseasec. Malignancy free and off treatment for =5 years
6. Significant renal disease in the renal allograft secondary to another condition (eg, infection, malignancy, monoclonal gammopathy, rejection,or a medication) that would confound interpretation of the study results
7. Participation in any other investigational drug trial or exposure to other investigational agent, device, or procedure within 30 days or 5 half-lives from the last dose of the investigational agent (whichever is longer) prior to screening period
8. Women who are currently breastfeeding
9. Inability to cooperate or any condition that, in the opinion of the investigator, could increase the patient's risk by participating in the study or confound the outcome of the study
10. Evidence of drug or alcohol abuse or dependence, in the opinion of the investigator

Non saranno eleggibili a partecipare al questo studio i pazienti che soddisfano allo screening o al basale uno qualsiasi dei criteri elencati di seguito.
1. Conta assoluta dei neutrofili < 1.000 cellule/mm3 durante lo screening (Giorno 1 escluso)
2. Precedente trattamento con pegcetacoplan
3. Evidenza di rigetto sul campione bioptico di screening dell'allotrapianto renale necessitante di trattamento
4. Diagnosi o anamnesi di infezione da HIV, epatite B o C o sieropositività allo screening indicativa d'infezione
5. Neoplasia maligna, fatta eccezione per:
a. carcinoma cutaneo basocellulare o squamocellulare curato
b. patologia in situ trattata con intento curativo
c. paziente libero da tumore maligno e non in terapia da = 5 anni
6. Importante patologia renale nell'allotrapianto renale secondaria a un'altra patologia (per es., infezione, neoplasia maligna, gammopatia monoclonale, rigetto o da farmaco) che confonderebbe l'interpretazione dei risultati dello studio
7. Partecipazione ad altre sperimentazioni con un farmaco sperimentale o esposizione ad altro agente, dispositivo o intervento sperimentale nei 30 giorni o 5 emivite dalla somministrazione dell'ultima dose dell'agente sperimentale (a seconda di quale periodo sia il più lungo) prima dello screening
8. Donne che allattano con latte materno
9. Incapacità di collaborazione o qualsiasi patologia che, a giudizio dello sperimentatore, potrebbe aumentare il rischio per il paziente se partecipasse allo studio oppure confondere l'esito dello studio
10. Evidenza di abuso o dipendenza da sostanze o alcol, a giudizio dello sperimentatore

E.5 End points

E.5.1

Primary end point(s)

The primary efficacy endpoint is the proportion of patients with reduction in C3c staining on renal biopsy after 12 weeks of treatment with pegcetacoplan.

L'endpoint primario di efficacia è la percentuale di pazienti che presenta una riduzione della colorazione per C3c su campione bioptico renale dopo 12 settimane di trattamento con pegcetacoplan.

E.5.1.1

Timepoint(s) of evaluation of this end point

Week 12 Biopsy

Settimana 12 Biopsia

E.5.2

Secondary end point(s)

The secondary endpoints are as follows:
• The number and incidence of treatment-related AEs
• The proportion of patients with reduction in C3c staining on renal biopsy after 52 weeks of treatment
• The proportion of patients achieving at least a 50% reduction in proteinuria over time
• The proportion of patients achieving complete clinical remission of proteinuria, defined as normalization of proteinuria
• The proportion of patients with stabilization or improvement in estimated glomerular filtration rate (eGFR) over time

Gli endpoint secondari sono:
• Numero e incidenza degli eventi avversi (AE) correlati al trattamento
• Percentuale di pazienti con riduzione della colorazione per C3c su campione bioptico renale dopo 52 settimane di trattamento
• Percentuale di pazienti che raggiunge una riduzione pari ad almeno il 50% della proteinuria nel tempo
• Percentuale di pazienti che raggiunge la remissione clinica completa della proteinuria, definita come la normalizzazione della proteinuria
• Percentuale di pazienti che presenta stabilizzazione o miglioramento della velocità di filtrazione glomerulare stimata (eGFR) nel tempo

E.5.2.1

Timepoint(s) of evaluation of this end point

Week 52 Biopsy

Settimana 52 Biopsia

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

E.6.5

Efficacy

E.6.6

Pharmacokinetic

Yes

E.6.7

Pharmacodynamic

Yes

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

Yes

E.8.1.3

Single blind

E.8.1.4

Double blind

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

E.8.2.3

Other

Yes

E.8.2.3.1

Comparator description

Variazione del programma di trattamento: il gruppo 2 non riceverà pegcetacoplan durante la porzione

Variation in treatment schedule: Group2 will not receive pegcetacoplan during the Controlled Portion

E.8.2.4

Number of treatment arms in the trial

E.8.3

The trial involves single site in the Member State concerned

Yes

E.8.4

The trial involves multiple sites in the Member State concerned

E.8.4.1

Number of sites anticipated in Member State concerned

E.8.5

The trial involves multiple Member States

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

Brazil

United States

Austria

France

Italy

Netherlands

Spain

Switzerland

United Kingdom

Argentina

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 end of the study will be the last patient's last visit (LVLS) or the last patient's scheduled visit/assessment as indicated in the Schedule of Events OR as requested by the sponsor.

La fine dello studio avverrà all'ultima visita del paziente (LVLS) o all'ultima visita/valutazione programmata del paziente come indicato nel Calendario degli eventi OPPURE come richiesto dallo sponsor.

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

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

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)

All patients who complete study treatment and don't enter Part B, the long-term extension, will return to the site for follow-up visits for 24 weeks, where assessments will be performed per the SoE in Protocol, and where the visit window allowances are also provided. Patients who discontinue treatment early and don't elect to continue their participation in the study will complete a follow-up visit 6 weeks after discontinuation of treatment and also the exit visit, 6 weeks thereafter.

Tutti i pazienti che completano il trattamento in studio e non partecipano alla Parte B(estensione a lungo termine)torneranno al centro per le visite di follow-up per 24 settimane, per eseguire le valutazioni previste dal protocollo e verranno anche fornite le finestre di visita. I pazienti che interrompono il trattamento in anticipo e non scelgono di continuare lo studio completeranno una visita di follow-up 6 settimane dopo l'interruzione del trattamento e la visita di uscita, 6 settimane dopo

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

2021-04-09

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2020-12-15

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

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