Clinical Trials Register

Summary
EudraCT Number:	2020-003230-20
Sponsor's Protocol Code Number:	MG0007
National Competent Authority:	Italy - Italian Medicines Agency
Clinical Trial Type:	EEA CTA
Trial Status:	Completed
Date on which this record was first entered in the EudraCT database:	2021-08-30
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

Italy - Italian Medicines Agency

A.2

EudraCT number

2020-003230-20

A.3

Full title of the trial

An Open-Label Extension Study to Evaluate Rozanolixizumab in Study Participants With Generalized Myasthenia Gravis

Studio di estensione in aperto per valutare Rozanolixizumab in soggetti in studio con miastenia gravis generalizzata

A.3.1

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

A study to evaluate rozanolixizumab in study participants with generalized myasthenia gravis

Studio per valutare Rozanolixizumab in soggetti in studio con miastenia gravis generalizzata

A.3.2

Name or abbreviated title of the trial where available

A.4.1

Sponsor's protocol code number

MG0007

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	UCB Biopharma SRL
B.1.3.4	Country	Belgium
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	UCB Biopharma SRL
B.4.2	Country	Belgium
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	UCB BIOSCIENCES GmbH
B.5.2	Functional name of contact point	Clin Trial Reg & Results Disclosure
B.5.3	Address:
B.5.3.1	Street Address	Alfred-Nobel-Strasse 10
B.5.3.2	Town/ city	Monheim
B.5.3.3	Post code	40789
B.5.3.4	Country	Germany
B.5.4	Telephone number	000000
B.5.5	Fax number	000000
B.5.6	E-mail	clinicaltrials@ucb.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	Yes
D.2.5.1	Orphan drug designation number	EU/3/20/2272
D.3 Description of the IMP
D.3.1	Product name	Rozanolixizumab
D.3.2	Product code	[UCB7665]
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	ROZANOLIXIZUMAB
D.3.9.1	CAS number	1584645-37-3
D.3.9.2	Current sponsor code	UCB7665
D.3.9.4	EV Substance Code	SUB187374
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	140
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.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

Generalized myasthenia gravis

Miastenia gravis generalizzata

E.1.1.1

Medical condition in easily understood language

Myasthenia gravis is an autoimmune disease that causes weakness in your muscles; it is caused by a communication problem between nerves and muscles.

La miastenia gravis è una malattia autoimmune che causa debolezza muscolare; è causata da un problema di comunicazione tra i nervi e i muscoli.

E.1.1.2

Therapeutic area

Diseases [C] - Nervous System Diseases [C10]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

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

E.1.3

Condition being studied is a rare disease

Yes

E.2 Objective of the trial

E.2.1

Main objective of the trial

Assess the safety and tolerability of additional 6-week treatment cycles with rozanolixizumab in study participants with generalized myasthenia gravis (gMG).

Valutare la sicurezza e la tollerabilità di ulteriori cicli di trattamento di 6 settimane con rozanolixizumab in partecipanti allo studio affetti da miastenia gravis generalizzata (gMG)

E.2.2

Secondary objectives of the trial

Assess the efficacy of 6-week treatment cycles with rozanolixizumab in study participants with generalized myasthenia gravis (gMG)

Valutare l’efficacia di cicli di trattamento di 6 settimane con rozanolixizumab in partecipanti allo studio affetti da miastenia gravis generalizzata (gMG)

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

- Study participant must meet one of the following:
a) completed MG0003 [NCT03971422],
b) required rescue therapy during the Observation Period in MG0003, or
c) completed at least 6 visits in MG0004 [NCT04124965]
- Body weight > or = 35 kg at Baseline (Day 1)
- Study participants may be male or female

- Il partecipante allo studio deve soddisfare uno dei seguenti criteri:
a) aver completato MG0003 [NCT03971422],
b) aver avuto bisogno di una "rescue therapy" durante il periodo di osservazione in MG0003, o
c) aver completato almeno 6 visite in MG0004 [NCT04124965]
- Peso corporeo > o = 35 Kg al basale (Giorno 1)
- I partecipanti allo studio possono essere maschi o femmine

E.4

Principal exclusion criteria

- Study participant has a known hypersensitivity to any components of the study medication or other anti-neonatal FC receptor (FcRn) medications
- Study participant with a known tuberculosis (TB) infection, at high risk of acquiring TB infection, or latent tuberculosis infection (LTBI), or current/history of nontuberculous mycobacterial infection (NTMBI)
- Study participant met any mandatory withdrawal or mandatory study drug discontinuation criteria in MG0003, or MG0004, or permanently discontinued study drug in either study
- Study participant intends to have a live vaccination during the course of the study or within 8 weeks following the final dose of rozanolixizumab
- Study participant with severe (defined as Grade 3 on the Myasthenia Gravis-Activities of Daily Living (MG-ADL) scale) weakness affecting oropharyngeal or respiratory muscles, or who has myasthenic crisis or impending crisis

- Il partecipante allo studio ha una nota ipersensibilità a qualsiasi dei componenti del farmaco in studio o ad altri farmaci anti-FcRn (neonatal FC receptor)
- Il partecipante allo studio ha una nota infezione da tubercolosi (TB), un alto rischio di contrarre una infezione TB, o una infezione latente da rubercolosi (LTBI), o una attuale/pregressa infezione micobatterica non tubercolosi (NTMBI)
- Il partecipante allo studio ha riscontrato una qualsiasi interruzione obbligatoria o è rientrato nei criteri di interruzione del farmaco in studio in MG0003, o MG0004 o ha definitivamente sospeso il trattamento con il farmaco in qualsiasi degli studi citati
- Il partecipante allo studio intende sottoporsi a vaccinazione durante lo svolgimento dello studio o nelle 8 settimane seguenti la dose finale di rozanolixizumab
- Il partecipante allo studio con grave debolezza (definita come grado 3 della scala delle attività della miastenia gravis, MG-ADL) che colpisce i muscoli oro-faringei o respiratori, o chi ha una crisi miastenica o una crisi imminente

E.5 End points

E.5.1

Primary end point(s)

1. Percentage of participants with treatment-emergent adverse events (TEAEs)
2. Percentage of participants with TEAEs leading to permanent withdrawal of investigational medicinal product (IMP)

1. Percentuale di partecipanti che manifestano eventi avversi emergenti dal trattamento (TEAE)
2. Percentuale di partecipanti che manifestano TEAE con conseguente ritiro del prodotto medicinale sperimentale (IMP)

E.5.1.1

Timepoint(s) of evaluation of this end point

1.; 2. From Baseline (Day 1) to End of Study (average of 20 months)

1.; 2. dal basale (Giorno 1) fino alla fine dello studio (circa 20 mesi)

E.5.2

Secondary end point(s)

1. Change from Baseline (Day 1) to Day 43 in Myasthenia Gravis-Activities of Daily Living (MG-ADL) score within one treatment cycle
2. Change from Baseline (Day 1) to Day 43 in Quantitative Myasthenia Gravis (QMG) score within one treatment cycle
3. Change from Baseline (Day 1) to Day 43 in Myasthenia Gravis-Composite (MG-C) score within one treatment cycle
4. Change from Baseline (Day 1) to Day 43 in Myasthenia Gravis (MG) Symptoms Patient Reported Outcome (PRO) 'Muscle Weakness Fatigability' score within one treatment cycle
5. Change from Baseline (Day 1) to Day 43 in MG Symptoms PRO 'Physical Fatigue' score within one treatment cycle
6. Change from Baseline (Day 1) to Day 43 in MG Symptoms PRO 'Bulbar symptoms' score within one treatment cycle
7. MG-ADL responder (> or = 2.0-point improvement from Baseline [Day 1] to end of Day 43) within one treatment cycle
8. Time to MG-ADL response (> or = 2.0-point improvement from Baseline [Day 1]) within one treatment cycle
9. Time between consecutive treatment cycles

1. Variazione dal basale (Giorno 1) al Giorno 43 in termini di Punteggio relativo alle attività giornaliere (ADL) entro un ciclo di trattamento
2. Variazione dal basale (Giorno 1) al Giorno 43 in termini di Punteggio quantitativo della miastenia gravis (QMG) entro un ciclo di trattamento
3. Variazione dal basale (Giorno 1) al Giorno 43 in termini di Punteggio composito della miastenia gravis (MG-C) entro un ciclo di trattamento
4. Variazione dal basale (Giorno 1) al Giorno 43 in termini di Punteggio di “affaticamento da debolezza muscolare” degli esiti riferiti dal paziente (PRO) sui sintomi della miastenia gravis (MG) entro un ciclo di trattamento
5. Variazione dal basale (Giorno 1) al Giorno 43 in termini di Punteggio di “affaticamento fisico” dei PRO sui sintomi della MG entro un ciclo di trattamento
6. Variazione dal basale (Giorno 1) al Giorno 43 in termini di Punteggio dei “sintomi bulbari” dei PRO sui sintomi della MG entro un ciclo di trattamento
7. Soggetti responsivi alla MG-ADL (> o = 2,0 punti dal basale [Giorno 1] al Giorno 43) entro un ciclo di trattamento
8. Tempo alla risposta MG-ADL (> o = 2,0 punti dal basale [Giorno 1] al Giorno 43) entro un ciclo di trattamento
9. Tempo tra cicli di trattamento consecutivi

E.5.2.1

Timepoint(s) of evaluation of this end point

1. - 8. From Baseline (Day 1) to end of treatment cycle (up to 6 weeks)
9. From end of the previous treatment cycle (Day 43) to the next treatment cycle (Day 1)

1. - 8. Dal basale (Giorno 1) fino alla fine del ciclo di trattamento (fino a 6 settimane)
9. Dalla fine del precedente ciclo di trattamento (Giorno 43) al ciclo di trattamento successivo (Giorno 1)

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

E.6.10

Pharmacogenetic

E.6.11

Pharmacogenomic

E.6.12

Pharmacoeconomic

E.6.13

Others

Yes

E.6.13.1

Other scope of the trial description

Tolerability, Immunogenicity

Tollerabilità e Immunogenicità

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)

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

Yes

E.8.1.3

Single blind

E.8.1.4

Double blind

E.8.1.5

Parallel group

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

E.8.2.4

Number of treatment arms in the trial

E.8.3

The trial involves single site in the Member State concerned

E.8.4

The trial involves multiple sites in the Member State concerned

Yes

E.8.4.1

Number of sites anticipated in Member State concerned

E.8.5

The trial involves multiple Member States

Yes

E.8.5.1

Number of sites anticipated in the EEA

E.8.6 Trial involving sites outside the EEA

E.8.6.1

Trial being conducted both within and outside the EEA

Yes

E.8.6.2

Trial being conducted completely outside of the EEA

Information not present in EudraCT

E.8.6.3

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

Canada

China

Georgia

Japan

Russian Federation

Serbia

United States

Belgium

Denmark

France

Germany

Hungary

Italy

Poland

Spain

United Kingdom

Czechia

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

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)

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

Yes

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

122

F.4.2.2

In the whole clinical trial

200

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)

Eligible participants will be allowed to receive expanded access.

I partecipanti considerati idonei potranno ricevere un accesso più ampio.

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

2020-12-15

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2020-11-18

P. End of Trial
P.	End of Trial Status	Completed

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Select Rare Disease:
IMP with orphan designation in the indication
Orphan Designation Number:
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