Clinical Trials Register

Summary
EudraCT Number:	2020-005232-30
Sponsor's Protocol Code Number:	AMUC-2023
National Competent Authority:	Bulgarian Drug Agency
Clinical Trial Type:	EEA CTA
Trial Status:	Completed
Date on which this record was first entered in the EudraCT database:	2021-06-02
Trial 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

Expand All Collapse All

A. Protocol Information

A.1

Member State Concerned

Bulgarian Drug Agency

A.2

EudraCT number

2020-005232-30

A.3

Full title of the trial

A Phase 2, Randomized, Double-Blinded, Placebo-Controlled, Parallel Group Study Evaluating the Efficacy and Safety of Amiselimod (MT-1303) in Subjects with Mild to Moderate Ulcerative Colitis (UC)

A.3.1

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

Effects of Amiselimod in patients with mild to moderate Ulcerative Colitis.

A.4.1

Sponsor's protocol code number

AMUC-2023

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	Salix Pharmaceuticals, Inc.
B.1.3.4	Country	United States
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	Salix Pharmaceuticals, Inc.
B.4.2	Country	United States
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Salix Pharmaceuticals, Inc.
B.5.2	Functional name of contact point	Clinical Trial Manager
B.5.3	Address:
B.5.3.1	Street Address	400 Somerset Corporate Boulevard
B.5.3.2	Town/ city	Bridgewater,
B.5.3.3	Post code	NJ 08807
B.5.3.4	Country	United States
B.5.6	E-mail	alison.magnotti-nagel@bauschhealth.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	Amiselimod
D.3.2	Product code	MT-1303
D.3.4	Pharmaceutical form	Capsule
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	amiselimod
D.3.9.3	Other descriptive name	MT-1303
D.3.9.4	EV Substance Code	SUB118859
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	0.2
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.IMP: 2
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	Amiselimod
D.3.2	Product code	MT-1303
D.3.4	Pharmaceutical form	Capsule
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	amiselimod
D.3.9.3	Other descriptive name	MT-1303
D.3.9.4	EV Substance Code	SUB118859
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	0.4
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
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

Ulcerative Colitis (UC)

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	20.1
E.1.2	Level	LLT
E.1.2	Classification code	10045365
E.1.2	Term	Ulcerative colitis
E.1.2	System Organ Class	100000004856

E.1.3

Condition being studied is a rare disease

E.2 Objective of the trial

E.2.1

Main objective of the trial

Assess the efficacy and safety of oral amiselimod (MT-1303) compared to placebo at 12 weeks as the induction treatment in subjects with active mild to moderate ulcerative colitis (UC).

E.2.2

Secondary objectives of the trial

Assess the efficacy and safety of maintenance treatment with open-label amiselimod for up to 36 weeks (OLE Period) following completion of the Double-Blind Period.

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

Subjects will be eligible if they are male or female aged between 18 to 75 years at time of consent (inclusive) with stable vital signs and a diagnosis of active mild UC (modified Mayo Score of 3 or 4) or moderate UC (modified Mayo Score of 5 to 8) confirmed at least 12 weeks prior to randomization by clinical and endoscopic evidence and corroborated by a histopathology report.
Subjects must have an endoscopic subscore of ≥2 from and evidence of active UC extending ≥15 cm from the anal verge confirmed by a screening colonoscopy.
If subjects are receiving oral or rectal 5-aminosalicylates (5-ASAs) or oral corticosteroids (≤20 mg prednisolone equivalent per day) for treatment of their UC, they must be on a stable dose for at least 28 days prior to randomization.
Subjects who complete the Double-Blind Period of the study who, in the opinion of the Investigator, would benefit from continued treatment may participate in the OLE Period.

E.4

Principal exclusion criteria

Subjects will be excluded from the study if they have:
• Any of the following: a diagnosis of Crohn’s disease, indeterminate colitis, colitis (pseudomembranous, microscopic, or ischemic) or coeliac disease, current or recent (within 12 weeks prior to randomization) evidence of fulminant colitis, proctitis (defined as a rectal inflammation within 15 cm from the anal verge), abdominal abscess, toxic megacolon, bowel obstruction, or bowel perforation; a history or evidence of any colonic resection or subtotal or total colectomy, ileostomy, colostomy, known fixed symptomatic stenosis of the intestine,
unresected adenomatous colonic polyps, or colonic mucosal dysplasia.
Clinically significant infections (e.g., pneumonia, pyelonephritis, or septicemia) within 4 weeks prior to randomization or previous clinically significant infections
requiring hospitalization within 6 months prior to randomization, active or latent tuberculosis, infections of hepatitis B, hepatitis C, human immunodeficiency virus
(HIV), or history of disseminated herpes zoster .
• Active SARS-CoV-2 infection or complications related to COVID-19.
• A history of, or currently active, primary or secondary immunodeficiency, presence of progressive multifocal leukoencephalopathy (PML), or presence of demyelinating diseases.
• A history or evidence of two or more failures with biologic treatment for UC.
• Currently taking any medication for treatment of UC other than oral or rectal 5-ASAs or oral corticosteroids (≤20 mg prednisolone equivalent per day)
• Been taking enemas or suppositories (other than stable dose of 5-ASA) for treatment of UC within 2 weeks prior to the Screening Visit.
• Been taking an unstable dose of probiotics or antidiarrheals 2 weeks prior to the Screening Visit.
• Had recent myocardial infarction, unstable angina, stroke, transient ischemic attack, decompensated heart failure with hospitalization, Class III/IV heart failure, Mobitz Type II 2nd degree or 3rd degree atrioventricular (AV) block, sick
sinus syndrome, prolonged QT interval, Wolff Parkinson White or other conduction abnormalities, low heart rate, ongoing treatment with Class I or Class III anti-arrhythmic drugs, heart-rate-lowering calcium-channel blockers, β-blockers or with any other drugs which can reduce the heart rate, have known high risk for QT/QTc prolongation, or have clinically significant abnormal findings in 12-lead ECG that the Investigator considers may jeopardize the subject’s health.
• Forced expiratory volume in one second (FEV1) or forced expiratory vital capacity (FVC) <70% of predicted values at screening. For sites where DLCO will be assessed, the value (mL/min/mmHg) is < 80% of the predicted normal value for age, height, and gender.
• Macular oedema as assessed by OCT.
• History of non-response or treatment failure with amiselimod or other sphingosine 1-phosphate (S1P) receptor modulators.
• Fecal microbiota transplantation (FMT) within 12 months prior to the Screening Visit.
• Any of the following laboratory abnormalities:
o Hemoglobin (Hb) <9.0 g/dL.
o White blood cell (WBC) count <3.50 × 10^9/L (<3,500/μL).
o Absolute neutrophil count <1.50 × 10^9/L (<1,500/μL).
o Absolute lymphocyte count <0.80 × 10^9/L (<800/μL).
o Aspartate aminotransferase (AST) or alaninevaminotransferase (ALT) >2 × the upper limit of normal (ULN).
o Bilirubin >1.5 x the ULN; subjects with Gilbert’s syndrome may be enrolled with total bilirubin up to 5.0 mg/dl.
• Positive stool tests for enteric pathogens, pathogenic ova or parasites, or Clostridium difficile (C. difficile) during the Screening Period. If subject has a history of recent C. difficile infection (within 60 days prior to Screening Visit), they should not be considered for study enrollment until subject has been treated for C. difficile and is symptom free for at least 14 days prior to the Screening Visit.
• Any physical or mental conditions which would interfere with the study participation, collection of data, or study completion as determined by the Investigator.

E.5 End points

E.5.1

Primary end point(s)

Change from Baseline in the modified Mayo Score at Day 85. The modified Mayo Score is the sum of:
• Endoscopy subscore (excludes friability); plus
• Rectal bleeding subscore; plus
• Stool frequency subscore.

E.5.1.1

Timepoint(s) of evaluation of this end point

at Day 85.

E.5.2

Secondary end point(s)

• The proportion of subjects with endoscopic improvement at
Day 85. Endoscopic improvement is a Mayo endoscopic
subscore of ≤1.
• The change from Baseline in the 2-component Mayo Score (rectal bleeding plus endoscopic subscores) at Day 85.
• The proportion of subjects with clinical remission at Day 85 based on the modified Mayo Score defined as follows:
o Endoscopy subscore of ≤1 (excludes friability);
and
o Rectal bleeding subscore of 0; and
o At least one-point decrease in stool frequency subscore from Baseline to achieve a stool frequency subscore of ≤1.

E.5.2.1

Timepoint(s) of evaluation of this end point

at Day 85.

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

E.6.10

Pharmacogenetic

E.6.11

Pharmacogenomic

E.6.12

Pharmacoeconomic

E.6.13

Others

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

Moldova, Republic of

Ukraine

Taiwan

Australia

Belarus

Georgia

Japan

Korea, Republic of

Russian Federation

Serbia

United States

Bulgaria

Czechia

Estonia

Germany

Hungary

Italy

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

Last patient last visit

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:

320

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

134

F.4.2.2

In the whole clinical trial

336

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

2021-06-22

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2021-05-17

P. End of Trial
P.	End of Trial Status	Completed
P.	Date of the global end of the trial	2024-09-03

Select Country:	Select Age Range:
Select Trial Status:	Select Trial Phase:
Select Gender:
Select Date Range: to
Select Rare Disease:
IMP with orphan designation in the indication
Orphan Designation Number:
Results Status:
Clear advanced search filters

Austria
Belgium
Bulgaria
Croatia
Cyprus
Czechia
Denmark
Estonia
Finland
France
Germany
Greece
Hungary
Iceland
Ireland
Italy
Latvia
Liechtenstein
Lithuania
Luxembourg
Malta
Netherlands
Norway
Poland
Portugal
Romania
Slovakia
Slovenia
Spain
Sweden
United Kingdom
Outside EU/EEA

Clinical trials