Clinical Trials Register

Summary
EudraCT Number:	2019-003113-34
Sponsor's Protocol Code Number:	CLYS006X2202
National Competent Authority:	Hungary - National Institute of Pharmacy
Clinical Trial Type:	EEA CTA
Trial Status:	Prematurely Ended
Date on which this record was first entered in the EudraCT database:	2020-01-09
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

2019-003113-34

A.3

Full title of the trial

A randomized, multi-center, subject and investigator blinded, placebo controlled, parallel group study to assess the efficacy, safety and tolerability of LYS006 in patients with mild to moderate ulcerative colitis

Véletlen besorolásos, többközpontú, a beteg és a vizsgálatvezető számára vak elrendezésű, placebokontrollos, párhuzamos csoportos vizsgálat a LYS006 hatásosságának, biztonságosságának és tolerálhatóságának értékelésére enyhe-középsúlyos colitis ulcerosában szenvedő betegeknél

A.3.1

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

Study of efficacy, safety and tolerability of LYS006 in patients with mild to moderate ulcerative colitis

A LYS006 hatásosságának, biztonságosságának és tolerálhatóságának vizsgálata enyhe-középsúlyos colitis ulcerosában szenvedő betegeknél

A.3.2

Name or abbreviated title of the trial where available

A.4.1

Sponsor's protocol code number

CLYS006X2202

A.5.2

US NCT (ClinicalTrials.gov registry) number

NCT04074590

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	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 Hungary Kft.
B.5.2	Functional name of contact point	Public Information Desk
B.5.3	Address:
B.5.3.1	Street Address	Bartók Béla út 43-47.
B.5.3.2	Town/ city	Budapest
B.5.3.3	Post code	1114
B.5.3.4	Country	Hungary
B.5.4	Telephone number	00 36 1 457-6500
B.5.5	Fax number	00 36 1 457-6600
B.5.6	E-mail	infoph.hungary@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	LYS006
D.3.2	Product code	LYS006
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	not yet defined
D.3.9.1	CAS number	unavailable
D.3.9.2	Current sponsor code	LYS006
D.3.9.3	Other descriptive name	LYS006
D.3.9.4	EV Substance Code	SUB182365
D.3.10	Strength
D.3.10.1	Concentration unit	mg milligram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	5
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	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

Mild to moderate ulcerative colitis

E.1.1.1

Medical condition in easily understood language

Ulcerative colitis

E.1.1.2

Therapeutic area

Diseases [C] - Digestive System Diseases [C06]

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	10009900
E.1.2	Term	Colitis ulcerative
E.1.2	System Organ Class	10017947 - Gastrointestinal disorders

E.1.2 Medical condition or disease under investigation

E.1.2	Version	20.1
E.1.2	Level	LLT
E.1.2	Classification code	10045365
E.1.2	Term	Ulcerative colitis
E.1.2	System Organ Class	10017947 - Gastrointestinal disorders

E.1.3

Condition being studied is a rare disease

E.2 Objective of the trial

E.2.1

Main objective of the trial

To assess the induction of clinical remission by LYS006 in patients with mild to moderate ulcerative colitis compared to placebo

E.2.2

Secondary objectives of the trial

To assess safety and tolerability of LYS006 in patients with mild to
moderate ulcerative colitis compared to placebo.

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

The patients eligible for inclusion in this study must fulfill all of the following criteria:
1. Written informed consent must be obtained before any assessment is performed.
2. Male and female subjects aged 18-75 years at Screening.
3. Patients diagnosed with UC established at least 3 months prior to screening and history including at least
one relapse.
4. Patients with mild to moderate UC which is defined by a total Mayo Score between 5 and 10 (inclusive), comprised of the following subscores;
- Endoscopic subscore: 2 or 3 (determined from central reading of screening endoscopy)
- Stool frequency subscore: 1-3
- Rectal bleeding subscore: 1-3
- Physician's global assessment subscore: 1-2.
5. Disease must extend beyond 15 cm from the anal verge (assessed by screening endoscopy).
6. Patients must have responded inadequately or have intolerance to conventional therapy with oral 5-ASA prior to screening, and be willing to continue 5-ASA at a stable dose during the study treatment period.
7. Able to communicate well with the investigator, to understand and comply with the requirements of the study.

E.4

Principal exclusion criteria

The patients fulfilling any of the following criteria are not eligible for inclusion in this study:
- Use of investigational drugs within 4 weeks or 5 half-lives of screening, whichever is longer; or longer if required by local regulations.
- Confirmed laboratory findings at screening showing:
• Total white blood cell count (WBC) outside the range of 3,000 – 12,000 /μL.
o Subjects with mild leukocytosis (WBC not higher than 15,000 /μL) may be eligible, if the elevated WBC, according to the Investigator, is attributable to disease activity and/or to corticosteroid therapy and other causes such as hematological or infectious diseases can be excluded;
• Platelets <100,000/μL;
• Hemoglobin <9 g/dL and/or other signs of severe anemia
- Are taking any of the prohibited medications listed in Table 6 2 and do not fulfill the washout period at the time of randomization.
- Patients receiving concomitant medication(s) that is/are known to inhibit OAT3 or BCRP and that cannot be discontinued or replaced by safe alternative medication within 5 halflives or 1 week (whichever is longer) prior to baseline and for the duration of the study.
- Has severe UC during the screening period.
- Has previous diagnosis with Crohn's disease, indeterminate colitis, microscopic colitis or acute diverticulitis based on medical history.
- Any severe, progressive or uncontrolled medical or psychiatric condition, or other factors at randomization that in the judgment of the investigator prevents the patient from participating in the study.
- Active systemic infections (other than common cold) during the 2 weeks prior to randomization. Evidence of Clostridium difficile infection or other intestinal pathogen infection within 4 weeks prior to the first dose of study drug.
- History of toxic megacolon, abdominal abscess, symptomatic colonic
stricture, or stoma; history or is at risk of colectomy.
- Pregnant or nursing (lactating) women, where pregnancy is defined as the state of a female after conception and until the termination of gestation, confirmed by a positive hCG laboratory test at Screening.
- Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using basic methods of contraception during dosing of study Treatment.
- Patients with clinically relevant primary sclerosing cholangitis

Other protocol-defined exclusion criteria may apply.

E.5 End points

E.5.1

Primary end point(s)

Clinical remission rate

E.5.1.1

Timepoint(s) of evaluation of this end point

Week 8 using the full Mayo score

E.5.2

Secondary end point(s)

Number and severity of adverse events / number of subjects with adverse events
Safety and tolerability based on general safety measurements (safety laboratory parameters, vital signs and ECG parameters)

E.5.2.1

Timepoint(s) of evaluation of this end point

From Baseline visit till end of trial visit (Day 87)

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

Yes

E.6.10

Pharmacogenetic

Yes

E.6.11

Pharmacogenomic

E.6.12

Pharmacoeconomic

E.6.13

Others

Yes

E.6.13.1

Other scope of the trial description

tolerability and impact on patient’s quality of life

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

Yes

E.8.4

The trial involves multiple sites in the 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

Russian Federation

United States

Bulgaria

Czechia

Germany

Hungary

Norway

Poland

Slovakia

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

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:

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)

None

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

2020-02-25

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2020-02-18

P. End of Trial
P.	End of Trial Status	Prematurely Ended
P.	Date of the global end of the trial	2022-11-07

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