Clinical Trials Register

Summary
EudraCT Number:	2019-000968-18
Sponsor's Protocol Code Number:	MG0003
National Competent Authority:	Hungary - National Institute of Pharmacy
Clinical Trial Type:	EEA CTA
Trial Status:	Completed
Date on which this record was first entered in the EudraCT database:	2019-07-23
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

Hungary - National Institute of Pharmacy

A.2

EudraCT number

2019-000968-18

A.3

Full title of the trial

A Phase 3, randomized, double-blind, placebo-controlled study evaluating efficacy and safety of rozanolixizumab in adult patients with generalized myasthenia gravis.

3. fázisú, randomizált, kettős vak, placebo-kontrollált vizsgálat a rozanolixizumab hatásosságának és biztonságosságának vizsgálata céljából generalizált myasthenia gravisszal diagnosztizált felnőttek körében.

A.3.1

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

A study to test efficacy and safety of rozanolixizumab in adult patients with generalized myasthenia gravis

Vizsgálat a rozanolixizumab hatásosságának és biztonságosságának vizsgálatára generalizált myasthenia gravisszal diagnosztizált felnőttek körében

A.3.2

Name or abbreviated title of the trial where available

MycarinGstudy

A.4.1

Sponsor's protocol code number

MG0003

A.5.2

US NCT (ClinicalTrials.gov registry) number

NCT03971422

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 SPRL
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 SPRL
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.5.6	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	No
D.2.5.1	Orphan drug designation number
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 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	SUB166282
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	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.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	Solution for infusion
D.8.4	Route of administration of the placebo	Subcutaneous use

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

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.

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	20.0
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

Demonstrate the clinical efficacy of rozanolixizumab in patients with generalized myasthenia gravis (MG)

E.2.2

Secondary objectives of the trial

Assess safety and tolerability of rozanolixizumab in myasthenia gravis (MG) patients

E.2.3

Trial contains a sub-study

Yes

E.2.3.1

Full title, date and version of each sub-study and their related objectives

Study participants who have consented can participate in the pharmacogenomics sub-study.

E.3

Principal inclusion criteria

- Study participant must be ≥18 years of age, at the time of signing the informed consent
- Study participant has documented diagnosis of generalized myasthenia gravis (gMG) at Visit 1, based on study participant’s history and supported by previous evaluations
- Study participant has a confirmed positive record of autoantibodies against acetylcholine receptor (AChR) or muscle-specific kinase (MuSK) prior to Visit 1
- Study participant has Myasthenia Gravis Foundation of America (MGFA) Class II to IVa at Visit 1
- Study participant with a myasthenia gravis-activities of daily living (MG-ADL) score of at least 3 AND a quantitative myasthenia gravis (QMG) score of at least 11 at Visit 1 and at Baseline

E.4

Principal exclusion criteria

- Study participant has a clinically relevant active infection (eg, sepsis, pneumonia, or abscess) in the opinion of the Investigator, or had a serious infection (resulting in hospitalization or requiring parenteral antibiotic treatment) within 6 weeks prior to the first dose of investigational medicinal product (IMP)
- Study participant has experienced hypersensitivity reaction after exposure to other antineonatal Fc receptor (FcRn) drugs
- Study participant with severe (defined as Grade 3 on the MG-ADL scale) weakness affecting oropharyngeal or respiratory muscles, or who has myasthenic crisis or impending crisis a Visit 1

E.5 End points

E.5.1

Primary end point(s)

Change from Baseline to Visit 10 in Myasthenia Gravis-Activities of Daily Living (MG-ADL) score

E.5.1.1

Timepoint(s) of evaluation of this end point

Baseline and Visit 10 (Day 43)

E.5.2

Secondary end point(s)

1. Percentage of participants achieving Myasthenia Gravis-Activities of Daily Living (MG-ADL) response at Visit 10
2. Change from Baseline to Visit 10 in the Myasthenia Gravis-Composite score
3. Change from Baseline to Visit 10 in Quantitative Myasthenia Gravis (QMG) score to Visit 10
4. Change from Baseline to Visit 10 in the Myasthenia Gravis (MG) Symptoms Patient Reported Outcome (PRO) ‘Fatigability’ score
5. Change from Baseline to Visit 10 in the Myasthenia Gravis (MG) Symptoms Patient Reported Outcome (PRO) ‘Physical Fatigue, Limb and Axial Weakness’ score
6. Change from Baseline to Visit 10 in the Myasthenia Gravis (MG) Symptoms Patient Reported Outcome (PRO) ‘Bulbar’ score
7. Occurrence of treatment-emergent adverse events (TEAEs)
8. Treatment-emergent adverse events (TEAEs) leading to withdrawal of investigational medicinal product (IMP)

E.5.2.1

Timepoint(s) of evaluation of this end point

1. Visit 10 (Day 43)
2.- 6. Baseline and Visit 10 (Day 43)
7. and 8. From Baseline until End of Study Visit (up to Week 14)

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

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

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

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

Canada

Czech Republic

Denmark

France

Germany

Hungary

Italy

Poland

Russian Federation

Spain

United Kingdom

United States

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:

216

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

121

F.4.2.2

In the whole clinical trial

240

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 subjects will be allowed to enroll in an extension study.

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

2019-09-25

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2019-09-13

P. End of Trial
P.	End of Trial Status	Completed
P.	Date of the global end of the trial	2021-10-26

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