Clinical Trials Register

Summary
EudraCT Number:	2018-000768-27
Sponsor's Protocol Code Number:	IFX-1-P2.5
National Competent Authority:	Spain - AEMPS
Clinical Trial Type:	EEA CTA
Trial Status:	Prematurely Ended
Date on which this record was first entered in the EudraCT database:	2019-01-18
Trial results	View 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

Spain - AEMPS

A.2

EudraCT number

2018-000768-27

A.3

Full title of the trial

A RANDOMIZED, DOUBLE-BLIND, DOUBLE-DUMMY, ACTIVE-CONTROLLED, MULTICENTER, 2-PART PHASE II STUDY ON REPLACEMENT OF STEROIDS BY IFX-1 IN ACTIVE GRANULOMATOSIS WITH POLYANGIITIS (GPA) AND MICROSCOPIC POLYANGIITIS (MPA)

Estudio en fase II aleatorizado, doble ciego, con doble simulación, con control activo, multicéntrico y de 2 partes sobre la sustitución de corticoesteroides por IFX-1 en la granulomatosis con poliangitis (GPA) y la poliangitis microscópica (PAM) activas

A.3.1

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

Efficacy and safety study of a monoclonal antibody to replace steroids for treatment of patients with Granulomatosis with Polyangiitis (GPA) and Microscopic Polyangiitis (MPA) diseases.

Estudio de eficacia y seguridad de un anticuerpo monoclonal que reemplaza a los esteroides para el tratamiento de pacientes con granulomatosis con poliangitis (GPA) y la poliangitis microscópica (PAM).

A.4.1

Sponsor's protocol code number

IFX-1-P2.5

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	InflaRx GmbH
B.1.3.4	Country	Germany
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	InflaRx GmbH
B.4.2	Country	Germany
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	InflaRx GmbH
B.5.2	Functional name of contact point	InflaRx GmbH
B.5.3	Address:
B.5.3.1	Street Address	Winzerlaer Str. 2
B.5.3.2	Town/ city	Jena
B.5.3.3	Post code	07745
B.5.3.4	Country	Germany
B.5.4	Telephone number	+349322754003111
B.5.6	E-mail	info@inflarx.de

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	IFX-1
D.3.4	Pharmaceutical form	Concentrate for solution for 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	not yet available
D.3.9.2	Current sponsor code	IFX-1 (former code: CaCP29)
D.3.9.3	Other descriptive name	chimeric monoclonal antibody, IgG4 subtype
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	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
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	Comparator
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	PredniSONE Tablets, USP
D.2.1.1.2	Name of the Marketing Authorisation holder	Vintage Pharms
D.2.1.2	Country which granted the Marketing Authorisation	United States
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	Glucocorticoids (GC)
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	PREDNISONE
D.3.9.1	CAS number	53-03-2
D.3.9.4	EV Substance Code	SUB10020MIG
D.3.10	Strength
D.3.10.1	Concentration unit	mg milligram(s)
D.3.10.2	Concentration type	range
D.3.10.3	Concentration number	5 to 10
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	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
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	Comparator
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	GALEN® tablet
D.2.1.1.2	Name of the Marketing Authorisation holder	GALENpharma GmbH
D.2.1.2	Country which granted the Marketing Authorisation	Germany
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	Glucocorticoids (GC)
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	PREDNISONE
D.3.9.1	CAS number	53-03-2
D.3.9.4	EV Substance Code	SUB10020MIG
D.3.10	Strength
D.3.10.1	Concentration unit	mg milligram(s)
D.3.10.2	Concentration type	range
D.3.10.3	Concentration number	5 to 10
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	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
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	Concentrate for solution for infusion
D.8.4	Route of administration of the placebo	Intravenous use
D.8 Placebo: 2
D.8.1	Is a Placebo used in this Trial?	Yes
D.8.3	Pharmaceutical form of the placebo	Capsule, hard
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

Active Granulomatosis with Polyangiitis (Wegener’s) (GPA) and Microscopic Polyangiitis (MPA)

Granulomatosis con poliangitis (GPA) y la poliangitis microscópica (PAM) activas

E.1.1.1

Medical condition in easily understood language

GPA and MPA are severe rare diseases causing pain and swelling of blood vessels and other tissues.

GPA y PAM son enfermedades raras y severas que causan dolor e hinchazón de los vasos sanguíneos y otros tejidos.

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	20.0
E.1.2	Level	PT
E.1.2	Classification code	10063344
E.1.2	Term	Microscopic polyangiitis
E.1.2	System Organ Class	10047065 - Vascular disorders

E.1.2 Medical condition or disease under investigation

E.1.2	Version	20.0
E.1.2	Level	PT
E.1.2	Classification code	10072579
E.1.2	Term	Granulomatosis with polyangiitis
E.1.2	System Organ Class	10047065 - Vascular 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

The primary objective is to evaluate the efficacy of IFX-1 treatment as a replacement for glucocorticoids [GC] therapy in subjects with GPA and MPA.

El objetivo principal es evaluar la eficacia del tratamiento con IFX-1 como sustitución del tratamiento con glucocorticoides (GC) en pacientes con GPA y PAM.

E.2.2

Secondary objectives of the trial

•To assess safety and tolerability of IFX-1
•To compare GC-induced toxicity of standard-dose GC and reduced dose GC with IFX-1 treatment
•To generate data for PK and PD modelling of IFX-1 treatment.

- Evaluar la seguridad y la tolerabilidad de IFX-1
- Comparar la toxicidad inducida por GC de la dosis estándar de GC y de la dosis reducida de GC con el tratamiento con IFX-1
- Generar datos para los modelos de farmacocinética (FC) y farmacodinámica (FD) del tratamiento con IFX-1.

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1. Male or female, ≥ 18 years of age.
2. Written informed consent obtained from subject.
3. Diagnosis of GPA or MPA according to the definitions of the Chapel Hill Consensus Conference (CHCC).
4.History of positive antigen-specific ANCA testing since the time of diagnosis or at screening, or documented evidence of either anti-proteinase 3 (anti-PR3) or anti-myeloperoxidase (anti-MPO) (for newly diagnosed subjects a recent positive antigen-specific ANCA testing is mandatory for inclusion)
5. Have ≥ 1 “major” item, or ≥ 3 other items, or ≥ 2 renal items on the Birmingham Vasculitis Activity Score Version 3 (BVASv3).
6. Newly diagnosed or relapsed GPA or MPA that requires treatment with CYC or RTX plus GCs.
7. Estimated glomerular filtration rate (eGFR) ≥ 20 mL/min/1.73 m².

1. Hombre o mujer ≥ 18 años de edad.
2. Consentimiento informado por escrito obtenido del sujeto.
3. Diagnóstico de GPA o PAM, de acuerdo con las definiciones de la Conferencia de consenso de Chapel Hill (Chapel Hill Consensus Conference, CHCC).
4. Antecedentes de resultado positivo en la prueba de anticuerpos anticitoplasma de neutrófilos (ANCA) específicos de antígeno desde el momento del diagnóstico o en el momento de la selección, o indicios documentados de anti-proteinasa 3 (anti-PR3) o anti mieloperoxidasa (anti-MPO) (para los sujetos de nuevo diagnóstico es obligatorio un resultado positivo reciente de la prueba de ANCA específicos de antígeno o positividad documentada de anti-PR3 o anti-MPO para la inclusión).
5. Tener ≥ 1 ítems importantes, o ≥ 3 ítems mínimos, o ≥ 2 ítems renales en la puntuación de la actividad de la vasculitis de Birmingham, versión 3 (Birmingham Vasculitis Activity Score Version 3, BVASv3).
6. GPA o PAM recién diagnosticada o recidivante que requiere tratamiento con CYC o RTX más GC.
7. Tasa de filtración glomerular estimada (TFGe) ≥ 20 ml/min/1,73 m².

E.4

Principal exclusion criteria

1. Any other multi-system autoimmune disease as listed in Appendix 18.4.
2. Require mechanical ventilation because of alveolar hemorrhage at screening.
3. Known hypersensitivity to any investigational medicinal product (IMP) (i.e. GC, IFX-1) and/or any excipients.
4. Subject with rare hereditary problems of galactose intolerance, total lactase deficiency or glucose-galactose malabsorption.
5. Have required management of infections, as follows:
a. Chronic infection requiring anti-infective therapy (such as latent tuberculosis, pneumocystis, aspergillosis, cytomegalovirus, herpes simplex virus, herpes zoster and atypical mycobacteria)
b. Use of intravenous antibacterials, antivirals, anti-fungals, or anti parasitic agents within 30 days of screening.
6. Current and/or history (within the previous 5 years) of drug and/or alcohol abuse and/or dependence.
7. Hepatitis B virus, (HBV) hepatitis C virus (HCV), or human immunodeficiency virus (HIV) viral test showing evidence of active or chronic viral infection at screening or a documented history of HIV, or an active or chronic hepatitis B or hepatitis C infection.
8. Any of the following abnormal laboratory findings at screening:
a. White blood cells < 3,500/mm3
b. Platelet count < 100,000/mm3
c. Transaminase values (AST and/or ALT) ≥ 2.5 times the upper limit of normal range (ULN)
d. Total bilirubin ≥ 1.5 times ULN
e. Alkaline Phosphatase (ALP) > 3 times ULN
9. Current or history of malignancy, lymphoproliferative, or myeloproliferative disorder except squamous cell or basal cell carcinomas of the skin and cervical carcinoma in situ with curative surgical treatment.
10. Received CYC or RTX within 12 weeks before screening; if on AZA, MMF or MPS or MTX at the time of screening, these drugs must be withdrawn prior to receiving the first dose of CYC or RTX.
11. Received > 3 g cumulative intravenous GCs within 4 weeks before screening.
12. Received an oral daily dose of a GC of > 10 mg prednisone-equivalent for more than 6 weeks continuously prior to screening.
13. Received a CD20 inhibitor, anti-tumor necrosis factor treatment, abatacept, alemtuzumab, any other experimental or biological therapy, intravenous immunoglobulin or plasma exchange, antithymocyte globulin, or required renal dialysis within 12 weeks before screening.
14. Received a live vaccination within 4 weeks before screening or planned between screening and Week 24.
15. Either active or latent tuberculosis treatment is ongoing.
16. Pregnant or lactating.
17. Clinically significant abnormal electrocardiogram (ECG) during screening.
18. Female subjects of childbearing potential unwilling or unable to use a highly effective method of contraception (pearl index < 1%) during treatment and for at least 3 months after last administration of IFX- 1/Placebo-IFX-1 (or up to 12 months, the timeframes for Standard of Care agents have to be considered as described in the respective Prescribing Information/SPCstimeframes). Contraception methods regarded as highly effective methods and the duration of contraception are further described in Section 7.7.
19. Evidence or suspicion that the subject might not comply with the requirements of the study protocol.
20. The subject is an employee or direct relative of an employee of the sponsor (InflaRx GmbH).
21. The subject is imprisoned or lawfully kept in an institution.
22. The subject has participated in an investigational clinical study during the 12 weeks (or 5 times the half-life of the previous IMP, whichever is longer) before screening, or plans to participate in another investigational clinical study during their participation in this study.
23.Male subjects with female partners of childbearing potential unwilling
to use contraception (condoms) during treatment and for at least 3
months after last administration of IFX-1/Placebo-IFX-1.

1. Cualquier otra enfermedad autoinmunitaria multisistémica de las que se indican en el Apéndice.
2. Requerir ventilación mecánica a causa de una hemorragia alveolar en la selección.
3. Hipersensibilidad conocida a cualquier producto en investigación (PEI) (es decir, GC, IFX-1) y/o cualquiera de sus excipientes.
4. Sujetos con problemas hereditarios raros de intolerancia a la galactosa, deficiencia total de lactasa o absorción inadecuada de la glucosa-galactosa.
5. Haber requerido tratamiento para infecciones, como sigue:
a. Infección crónica que requiera tratamiento antiinfeccioso (como tuberculosis latente, neumocistosis, aspergilosis, citomegalovirus, virus del herpes simple, herpes zóster y micobacterias atípicas).
b. Uso de antibacterianos, antivirales, antimicóticos o antiparasitarios intravenosos en los 30 días previos a la selección.
6. Uso actual y/o antecedentes (en los 5 años anteriores) de abuso y/o dependencia de drogas y/o alcohol.
7. Prueba del virus de la hepatitis B (VHB), virus de la hepatitis C (VHC) o virus de la inmunodeficiencia humana (VIH) que muestre indicios de una infección vírica activa o crónica en la selección o antecedentes documentados de VIH o de una infección activa o crónica por el virus de la hepatitis B o la hepatitis C.
8 Cualquiera de los siguientes resultados analíticos anómalos en la selección:
a. Leucocitos < 3500/mm3
b. Recuento de plaquetas <100 000/mm3
c. Valores de transaminasas (AST y/o ALT) ≥ 2,5 veces el límite superior de la normalidad (LSN)
d. Bilirrubina total ≥ 1,5 veces el LSN
e. Fosfatasa alcalina (FA) > 3 veces el LSN
9. Tener antecedentes de, o tener en la actualidad, neoplasia maligna, trastorno infoproliferativo o mieloproliferativo excepto carcinoma espinocelular o basocelular de la piel y carcinoma cervicouterino in situ con tratamiento quirúrgico curativo.
10. Haber recibido CYC o RTX en las 12 semanas anteriores a la selección; si se está tratando con AZA, MMF o MPS o MTX en el momento de la selección, estos fármacos deben ser retirados antes de recibir la primera dosis de CYC o RTX.
11. Haber recibido una dosis acumulada > 3 g de GC intravenosos en las 4 semanas anteriores a la selección.
12. Haber recibido una dosis oral diaria > 10 mg de un GC equivalente a la prednisona durante más de 6 semanas de forma continua antes de la selección.
13. Haber recibido un inhibidor de CD20, tratamiento con antagonistas del factor de necrosis tumoral, abatacept, alemtuzumab, cualquier otro tratamiento experimental o biológico, inmunoglobulina intravenosa o plasmaféresis, globulina antitimocítica, o necesitó diálisis renal en las 12 semanas anteriores a la selección.
14. Haber recibido una vacuna viva en las 4 semanas anteriores a la selección o tener previsto recibirla entre la selección y la semana 24.
15. El tratamiento para la tuberculosis activa o latente está en curso
16. Paciente embarazada o en periodo de lactancia.
17. Anomalía clínicamente significativa en el electrocardiograma (ECG) durante la selección.
18. Pacientes en edad fértil que no puedan o no estén dispuestas a utilizar un método anticonceptivo altamente eficaz (índice de Pearl < 1 %) durante el tratamiento y durante al menos 3 meses después de la última administración de IFX-1/placebo-IFX-1 (o hasta 12 meses, se deben considerar los plazos de los agentes de la práctica clínica habitual como se describe en la respectiva ficha técnica/RCP). Los métodos anticonceptivos considerados altamente eficaces y la duración de la anticoncepción se describen con más detalle en la Sección 7.7.
19. Indicios o sospecha de que el sujeto podría no cumplir con los requisitos del protocolo del estudio.
20. El sujeto es un empleado o familiar directo de un empleado del promotor.
21. El sujeto está preso o recluido en una institución por ley.
22. El sujeto ha participado en un estudio clínico de investigación clínica en las 12 semanas (o 5 veces la semivida del PEI anterior, lo que sea más prolongado) anteriores a la selección, o tiene previsto participar en otro estudio de investigación clínica durante su participación en este estudio.
23. Sujetos varones con parejas de sexo femenino en edad fértil que no estén dispuestas a utilizar un método anticonceptivo (preservativo) durante el tratamiento y durante al menos 3 meses después de la última administración de IFX-1/placebo-IFX-1.

E.5 End points

E.5.1

Primary end point(s)

The primary efficacy endpoint is the proportion of subjects achieving clinical response defined as reduction in BVASv3 ≥ 50% at Week 16 compared to baseline and no worsening in any body system. Subjects who receive rescue therapy until Week 16 will be considered as not having achieved clinical response.

El criterio de valoración principal es la proporción de pacientes que consiguen una respuesta clínica se define como una reducción en la BVASv3 ≥ 50 % en la semana 16 en comparación con el inicio y ningún empeoramiento en ningún sistema orgánico. Se considerará que no han alcanzado una respuesta clínica los sujetos que reciban tratamiento de rescate hasta la semana 16.

E.5.1.1

Timepoint(s) of evaluation of this end point

Week 16

Semana 16

E.5.2

Secondary end point(s)

1.Proportion of subjects with clinical response, defined as reduction in BVASv3 ≥ 50% and no worsening in any body system at each measurement time point except Week 16. Subjects who receive rescue therapy will be considered as not having achieved clinical response at each time point later than the first administration of rescue therapy
2.Proportion of subjects with a clinical remission defined as having a BVASv3 = 0 at Week 16
3.Absolute values and absolute and relative change from Day 1 in the VDI at Week 16
4.Absolute values and absolute and relative change from Day 1 in the PGA at Week 16
5.Absolute values and absolute and relative change from Day 1 in eGFR in mL/min/1.73 m² at Week 16.

1. Proporción de pacientes con una respuesta clínica, que se define como una reducción en la BVASv3 ≥ 50 % y ningún empeoramiento en ningún sistema orgánico en cada punto de medición, excepto el de la semana 16. Los sujetos que reciben terapia de rescate serán considerados como que no han logrado una respuesta clínica en cada momento posterior a la primera administración de la terapia de rescate.
2. Proporción de sujetos con una remisión clínica, definida como tener una puntuación en la BVASv3 = 0 en la semana 16
3. Valores absolutos y cambio absoluto y relativo desde el día 1 en el Índice de daño de vasculitis (Vasculitis Damage Index, VDI) en la semana 16
4. Valores absolutos y cambio absoluto y relativo desde el día 1 en la Evaluación global por parte del médico (Physician Global Assessment, PGA) en la semana 16
5. Valores absolutos y cambio absoluto y relativo desde el día 1 en la TFGe en ml/min/1,73 m² en la semana 16

E.5.2.1

Timepoint(s) of evaluation of this end point

1. At weeks 0 (Day 1), 1, 2, 4, 8, 12, 20, 24
2. Week 16
3. 4. and 5. From Day 1 to week 16

E.6 and E.7 Scope of the trial

E.6

Scope of the trial

E.6.1

Diagnosis

E.6.2

Prophylaxis

E.6.3

Therapy

E.6.4

Safety

Yes

E.6.5

Efficacy

Yes

E.6.6

Pharmacokinetic

Yes

E.6.7

Pharmacodynamic

Yes

E.6.8

Bioequivalence

E.6.9

Dose response

E.6.10

Pharmacogenetic

E.6.11

Pharmacogenomic

E.6.12

Pharmacoeconomic

E.6.13

Others

E.7

Trial type and phase

E.7.1

Human pharmacology (Phase I)

E.7.1.1

First administration to humans

E.7.1.2

Bioequivalence study

E.7.1.3

Other

E.7.1.3.1

Other trial type description

E.7.2

Therapeutic exploratory (Phase II)

Yes

E.7.3

Therapeutic confirmatory (Phase III)

E.7.4

Therapeutic use (Phase IV)

E.8 Design of the trial

E.8.1

Controlled

Yes

E.8.1.1

Randomised

Yes

E.8.1.2

Open

E.8.1.3

Single blind

E.8.1.4

Double blind

Yes

E.8.1.5

Parallel group

Yes

E.8.1.6

Cross over

E.8.1.7

Other

Yes

E.8.1.7.1

Other trial design description

Double dummy

E.8.2

Comparator of controlled trial

E.8.2.1

Other medicinal product(s)

Yes

E.8.2.2

Placebo

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

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

Belgium

Czech Republic

France

Germany

Ireland

Italy

Netherlands

Russian Federation

Spain

Sweden

United Kingdom

E.8.7

Trial has a data monitoring committee

Yes

E.8.8

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

LVLS

LVLP

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)

Patients will return to standard of care

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

2019-01-17

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2018-12-05

P. End of Trial
P.	End of Trial Status	Prematurely Ended

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Select Date Range: to
Select Rare Disease:
IMP with orphan designation in the indication
Orphan Designation Number:
Results Status:
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