Clinical Trials Register

Summary
EudraCT Number:	2016-002698-36
Sponsor's Protocol Code Number:	MG0002
National Competent Authority:	Czechia - SUKL
Clinical Trial Type:	EEA CTA
Trial Status:	Completed
Date on which this record was first entered in the EudraCT database:	2017-04-03
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

Czechia - SUKL

A.2

EudraCT number

2016-002698-36

A.3

Full title of the trial

A Multicenter, Randomized, Investigator- and Subject-Blind, Placebo-Controlled, Treatment Sequence Study Evaluating the Safety, Tolerability, and Efficacy of UCB7665 in Subjects with Moderate to Severe Myasthenia Gravis.

MULTICENTRICKÁ, RANDOMIZOVANÁ, PLACEBEM KONTROLOVANÁ STUDIE SEKVENČNÍ LÉČBY, ZASLEPENÁ VŮČI ZKOUŠEJÍCÍMU A SUBJEKTU, KE ZHODNOCENÍ BEZPEČNOSTI, SNÁŠENLIVOSTI A ÚČINNOSTI PŘÍPRAVKU UCB7665 U SUBJEKTŮ SE STŘEDNĚ VÁŽNOU AŽ ZÁVAŽNOU MYASTENIÍ GRAVIS

A.3.1

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

Study to test the safety, tolerability and efficacy of UCB7665 in subjects with moderate to severe myasthenia gravis.

STUDIE KE ZHODNOCENÍ BEZPEČNOSTI, SNÁŠENLIVOSTI A ÚČINNOSTI PŘÍPRAVKU UCB7665 U SUBJEKTŮ SE STŘEDNĚ VÁŽNOU AŽ ZÁVAŽNOU MYASTENIÍ GRAVIS

A.4.1

Sponsor's protocol code number

MG0002

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-Str. 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	UCB7665
D.3.2	Product code	UCB7665
D.3.4	Pharmaceutical form	Powder for 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.3.9.3	Other descriptive name	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	100
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	No
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	Yes
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	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

Moderate to severe 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] - 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	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

Evaluate the clinical efficacy of UCB7665 as an chronic-intermittent treatment in subjects with generalized myasthenia gravis (MG) who are classified as moderate to severe

E.2.2

Secondary objectives of the trial

- Gather data for future study planning, whether for chronic-intermittent treatment or a longer therapy option by evaluating the general concept that UCB7665 has a clinical effect in patients with generalized myasthenia gravis (MG)
- Evaluate the safety and tolerability of UCB7665 administered by subcutaneous (sc) infusion in subjects with MG
- Assess the effect of UCB7665 as measured by total immunoglobulin G (IgG) concentrations in serum

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

- Subject has a well-documented diagnosis of myasthenia gravis (MG) at Visit 1 (Screening), based on subject history and supported by previous evaluations
- Subject would currently be considered for treatment with immunological therapy (immunoglobulin/plasma exchange (IVIG/PLEX)) by the investigator
- Subject has well-documented record of autoantibodies against antiacetylcholine
receptor (Anti-AChR) or anti-muscle specific kinase (Anti-MuSK) prior to Screening
- Female subjects must either be: postmenopausal, permanently sterilized or if childbearing potential applicable will use a highly effective method of birth control
- Male subjects must be willing to use a method of contraception

E.4

Principal exclusion criteria

- Subject has previously received treatment in this study or subject has previously been exposed to UCB7665
- Subject has participated in another study of an investigational medicinal product (IMP; or a medical device) within the previous 30 days of Screening or is currently participating in another study of an
investigational medicinal product (IMP; or a medical device)
- Subject has a known hypersensitivity to any components of the IMP
- Subject has a history of hyperprolinemia, since L-proline is a constituent of the UCB7665 IMP
- Subjects with Myasthenia Gravis (MG) only affecting the ocular muscles
- Subjects with severe weakness affecting oropharyngeal or respiratory muscles, or who have myasthenic crisis at Screening or impending crisis
- Subject has quantitative myasthenia gravis (QMG) score of <11 at Baseline
- Subject has a serum total immunoglobulin G (IgG) level <= 6g/L at Screening
- Absolute neutrophil count <1500 cells/mm^3
- Subject has any medical condition (acute or chronic illness) or psychiatric condition that, in the opinion of the investigator, could jeopardize or would compromise the subject’s ability to participate in this study
- Subject has any laboratory abnormality that, in the opinion of the investigator, is clinically significant, has not resolved at randomization, and could jeopardize or would compromise the subject’s ability to participate in this study
- Subject has received a live vaccination within 8 weeks prior to the Baseline Visit; or intends to have a live vaccination during the course of the study or within 7 weeks following the final dose of IMP
- Subject has received any experimental biological agent within or outside of a clinical study in the past 3 months or within 5 half-lives prior to Baseline (whichever is longer)

E.5 End points

E.5.1

Primary end point(s)

Change from Baseline in Quantitative Myasthenia Gravis (QMG)
score to Visit 9

E.5.1.1

Timepoint(s) of evaluation of this end point

Baseline and Visit 9 (Day 29)

E.5.2

Secondary end point(s)

Change from Baseline in Myasthenia Gravis-Composite score to
Visit 9
Change from Baseline in Myasthenia Gravis-Activities of Daily
Living (MGADL) score to Visit 9

E.5.2.1

Timepoint(s) of evaluation of this end point

Baseline and Visit 9 (Day 29)

E.6 and E.7 Scope of the trial

E.6

Scope of the trial

E.6.1

Diagnosis

E.6.2

Prophylaxis

E.6.3

Therapy

Yes

E.6.4

Safety

Yes

E.6.5

Efficacy

Yes

E.6.6

Pharmacokinetic

Yes

E.6.7

Pharmacodynamic

Yes

E.6.8

Bioequivalence

E.6.9

Dose response

Yes

E.6.10

Pharmacogenetic

E.6.11

Pharmacogenomic

Yes

E.6.12

Pharmacoeconomic

E.6.13

Others

Yes

E.6.13.1

Other scope of the trial description

Tolerability

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

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

Germany

Spain

United States

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

Last subject last visit (LSLV)

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)

No post-trial medication will be provided

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

2017-05-26

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2017-04-05

P. End of Trial
P.	End of Trial Status	Completed
P.	Date of the global end of the trial	2018-08-06

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