ICH GCP - EU Clinical trials Registry

Summary
EudraCT Number:	2017-004207-52
Sponsor's Protocol Code Number:	CQGE031C2202
National Competent Authority:	Germany - PEI
Clinical Trial Type:	EEA CTA
Trial Status:	Completed
Date on which this record was first entered in the EudraCT database:	2018-03-08
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

A. Protocol Information

A.1

Member State Concerned

Germany - PEI

A.2

EudraCT number

2017-004207-52

A.3

Full title of the trial

A multicenter, randomized, double-blind, placebo-controlled phase 2b dose-finding study to investigate the efficacy and safety of ligelizumab (QGE031) in adolescent patients with Chronic Spontaneous Urticaria (CSU)

A.3.1

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

QGE031 Phase 2b dose-finding study to investigate efficacy and safety in adolescents with CSU

A.4.1

Sponsor's protocol code number

CQGE031C2202

A.7

Trial is part of a Paediatric Investigation Plan

Yes

A.8

EMA Decision number of Paediatric Investigation Plan

P/329/2017

B. Sponsor Information
B.Sponsor: 1
B.1.1	Name of Sponsor	Novartis Pharma AG
B.1.3.4	Country	Switzerland
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	Novartis Pharma AG
B.4.2	Country	Switzerland
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Novartis Pharma GmbH
B.5.2	Functional name of contact point	Medizinischer Infoservice (MCC)
B.5.3	Address:
B.5.3.1	Street Address	Roonstrasse 25
B.5.3.2	Town/ city	Nürnberg
B.5.3.3	Post code	90429
B.5.3.4	Country	Germany
B.5.4	Telephone number	+49 1802 232300
B.5.5	Fax number	+49 911 27312160
B.5.6	E-mail	infoservice.novartis@novartis.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	Ligelizumab
D.3.2	Product code	QGE031
D.3.4	Pharmaceutical form	Concentrate for 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	Ligelizumab
D.3.9.2	Current sponsor code	QGE031
D.3.9.3	Other descriptive name	Anti-IgE antibody
D.3.9.4	EV Substance Code	SUB32387
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	120
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	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 injection/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

Chronic Spontaneous Urticaria

E.1.1.1

Medical condition in easily understood language

Chronic Hives

E.1.1.2

Therapeutic area

Diseases [C] - Skin and Connective Tissue Diseases [C17]

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	10072757
E.1.2	Term	Chronic spontaneous urticaria
E.1.2	System Organ Class	10040785 - Skin and subcutaneous tissue disorders

E.1.3

Condition being studied is a rare disease

No

E.2 Objective of the trial

E.2.1

Main objective of the trial

The primary objective of this study is to assess the change in the Urticaria Activity Score (UAS7) between baseline and Week 24

E.2.2

Secondary objectives of the trial

- To evaluate the efficacy of ligelizumab doses with respect to UAS7 change from baseline
- To evaluate the efficacy of ligelizumab doses on complete response in Urticaria Activity Score (UAS7=0), Hives Severity Score (HSS=0), Itch Severity score (ISS=0)
- To investigate the effects on ISS7 and HSS7 when compared to baseline
- To investigate the pharmacokinetics and Pharmacodynamics of ligelizumab
- Change from baseline in the Children Dermatology Life Quality Index
- To evaluate the safety (including immunogenicity) and tolerability of ligelizumab versus placebo in patients with CSU

E.2.3

Trial contains a sub-study

No

E.3

Principal inclusion criteria

- Parent or legal guardian’s written informed consent and child’s assent
- Male and female adolescent patients aged ≥ 12 to <18 years at the time of screening
- CSU diagnosis ≥ 6 months and presence of itch and hives for at least 6 consecutive weeks at any time prior to enrollment
- Diagnosis of CSU refractory to approved doses of H1-antihistamines at the time of randomization
- Other protocol-defined criteria may apply

E.4

Principal exclusion criteria

- Clearly defined underlying etiology for chronic urticarias other than CSU
- Any other skin disease associated with chronic itching that might confound the study evaluations and results
- Previous exposure to omalizumab
- History of anaphylaxis
- Other protocol-defined criteria may apply

E.5 End points

E.5.1

Primary end point(s)

Change in the Urticaria Activity Score (UAS7) between baseline and Week 24

E.5.1.1

Timepoint(s) of evaluation of this end point

24 weeks

E.5.2

Secondary end point(s)

- UAS7 change from baseline over time
- Rate of complete responders (UAS7=0, HSS7=0, ISS7 =0) over time
- Itch severity score and Hives severity score change from baseline over time
- Model-based estimate of clearance and volume of distribution using at least 7 samples
- Summary statistics of change in Total IgE over time
- Children Dermatology Life Quality Index change from baseline over time
- Adverse events, ECG-intervals and interpretation, vital signs (blood pressure, pulse rate) and clinical laboratory evaluation

E.5.2.1

Timepoint(s) of evaluation of this end point

- At each protocol defined study visit

E.6 and E.7 Scope of the trial

E.6

Scope of the trial

E.6.1

Diagnosis

No

E.6.2

Prophylaxis

No

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

No

E.6.9

Dose response

Yes

E.6.10

Pharmacogenetic

No

E.6.11

Pharmacogenomic

No

E.6.12

Pharmacoeconomic

No

E.6.13

Others

Yes

E.6.13.1

Other scope of the trial description

Quality of Life

E.7

Trial type and phase

E.7.1

Human pharmacology (Phase I)

No

E.7.1.1

First administration to humans

No

E.7.1.2

Bioequivalence study

No

E.7.1.3

Other

No

E.7.1.3.1

Other trial type description

E.7.2

Therapeutic exploratory (Phase II)

Yes

E.7.3

Therapeutic confirmatory (Phase III)

No

E.7.4

Therapeutic use (Phase IV)

No

E.8 Design of the trial

E.8.1

Controlled

Yes

E.8.1.1

Randomised

Yes

E.8.1.2

Open

No

E.8.1.3

Single blind

No

E.8.1.4

Double blind

Yes

E.8.1.5

Parallel group

Yes

E.8.1.6

Cross over

No

E.8.1.7

Other

No

E.8.2

Comparator of controlled trial

E.8.2.1

Other medicinal product(s)

No

E.8.2.2

Placebo

Yes

E.8.2.3

Other

No

E.8.2.4

Number of treatment arms in the trial

3

E.8.3

The trial involves single site in the Member State concerned

No

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

2

E.8.5

The trial involves multiple Member States

Yes

E.8.5.1

Number of sites anticipated in the EEA

8

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

No

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

Argentina

Belgium

Canada

Germany

Spain

Switzerland

Taiwan

Turkey

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

Completion of the study will be considered when all patients will have
completed 24 weeks of treatment epoch and the full follow up epoch or
have prematurely withdrawn from the study

E.8.9 Initial estimate of the duration of the trial

E.8.9.1

In the Member State concerned years

2

E.8.9.1

In the Member State concerned months

E.8.9.1

In the Member State concerned days

8

E.8.9.2

In all countries concerned by the trial years

2

E.8.9.2

In all countries concerned by the trial months

2

F. Population of Trial Subjects

F.1 Age Range

F.1.1

Trial has subjects under 18

Yes

F.1.1

Number of subjects for this age range:

48

F.1.1.1

In Utero

No

F.1.1.2

Preterm newborn infants (up to gestational age < 37 weeks)

No

F.1.1.3

Newborns (0-27 days)

No

F.1.1.4

Infants and toddlers (28 days-23 months)

No

F.1.1.5

Children (2-11years)

No

F.1.1.6

Adolescents (12-17 years)

Yes

F.1.1.6.1

Number of subjects for this age range:

48

F.1.2

Adults (18-64 years)

No

F.1.3

Elderly (>=65 years)

No

F.2 Gender

F.2.1

Female

Yes

F.2.2

Male

Yes

F.3 Group of trial subjects

F.3.1

Healthy volunteers

No

F.3.2

Patients

Yes

F.3.3

Specific vulnerable populations

Yes

F.3.3.1

Women of childbearing potential not using contraception

No

F.3.3.2

Women of child-bearing potential using contraception

Yes

F.3.3.3

Pregnant women

No

F.3.3.4

Nursing women

No

F.3.3.5

Emergency situation

No

F.3.3.6

Subjects incapable of giving consent personally

No

F.3.3.7

Others

No

F.4 Planned number of subjects to be included

F.4.1

In the member state

9

F.4.2

For a multinational trial

F.4.2.1

In the EEA

21

F.4.2.2

In the whole clinical trial

48

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)

Not applicable

G. Investigator Networks to be involved in the Trial

N. Review by the Competent Authority or Ethics Committee in the country concerned

N.

Competent Authority Decision

Authorised

N.

Date of Competent Authority Decision

2018-05-15

N.

Ethics Committee Opinion of the trial application

N.

Ethics Committee Opinion: Reason(s) for unfavourable opinion

N.

Date of Ethics Committee Opinion

P. End of Trial
P.	End of Trial Status	Completed
P.	Date of the global end of the trial	2021-01-05

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