Clinical Trials Register

Summary
EudraCT Number:	2015-001925-18
Sponsor's Protocol Code Number:	GED-0301-CD-002
National Competent Authority:	Czechia - SUKL
Clinical Trial Type:	EEA CTA
Trial Status:	Prematurely Ended
Date on which this record was first entered in the EudraCT database:	2015-12-22
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

2015-001925-18

A.3

Full title of the trial

A Phase 3, randomized, double-blind, placebo-controlled, multicenter study to investigate the efficacy and safety of mongersen (GED-0301) for the treatment of subjects with active Crohn’s disease.

Randomizované, dvojitě zaslepené, placebem kontrolované multicentrické klinické hodnocení fáze 3 ke zjištění účinnosti a bezpečnosti přípravku Mongersen (GED-0301) pro léčbu pacientů s aktivní Crohnovou nemocí

A.3.1

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

A study to evaluate the drug Mongersen (GED-0301) for the treatment of Crohn’s disease

A.3.2

Name or abbreviated title of the trial where available

A randomized, double-blind, study to explore the efficacy and safety of GED-0301 in Crohn's disease

A.4.1

Sponsor's protocol code number

GED-0301-CD-002

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	Celgene Corporation
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	Celgene Corporation
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	Celgene Corporation
B.5.2	Functional name of contact point	ClinicalTrialDisclosure
B.5.3	Address:
B.5.3.1	Street Address	9225 Indian Creek Parkway, Suite 900
B.5.3.2	Town/ city	Overland Park, Kansas
B.5.3.3	Post code	66210
B.5.3.4	Country	United States
B.5.4	Telephone number	+18882601599
B.5.5	Fax number	+19132660394
B.5.6	E-mail	ClinicalTrialDisclosure@celgene.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	Mongersen
D.3.2	Product code	GED-0301
D.3.4	Pharmaceutical form	Film-coated tablet
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	mongersen
D.3.9.1	CAS number	1443994-98-6
D.3.9.2	Current sponsor code	GED-0301
D.3.9.4	EV Substance Code	SUB30963
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	40
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	Yes
D.3.11.13.1	Other medicinal product type	antisense-oligonucleotide

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	Film-coated tablet
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

Active Crohn's disease

E.1.1.1

Medical condition in easily understood language

Crohn’s disease (an Inflammatory Bowel Disease)

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	LLT
E.1.2	Classification code	10021315
E.1.2	Term	Ileitis terminal
E.1.2	System Organ Class	100000016693

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 evaluate the efficacy of GED-0301 compared with placebo on clinical
activity at Week 12 in subjects with active Crohn's disease (CD).

E.2.2

Secondary objectives of the trial

To evaluate the efficacy of GED-0301 compared with placebo on
endoscopic outcomes in subjects with active CD
To evaluate the long-term efficacy of GED-0301 compared with placebo
on clinical activity in subjects with active CD
To evaluate the efficacy of GED-0301 compared with placebo on
corticosteroid-free clinical remission in subjects with active CD
To evaluate the safety and tolerability of GED-0301 in subjects with
active CD.

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

A PK substudy (sparse PK study) was incorporated into the study at selected study sites to monitor for
systemic exposure of GED-0301 in subjects with active CD. Intensive collection of PK samples will be performed in a subset of subjects that have a CDAI score of ≥ 300 and ≤ 450, with a minimum of 16 participants. An intensive PK sub-study is not taking place in the Member State concerned by this application

E.3

Principal inclusion criteria

Diagnosis of CD with a duration of at least 3 months prior to the
Screening Visit
Presence of ileitis, ileocolitis or colitis, as determined by ileocolonoscopy
at screening.
Active disease, defined as a CDAI score ≥ 220 and ≤ 450 at screening
Must have a 7-day average stool frequency ≥ 3.5 or abdominal pain ≥
1.5 at screening.
Must have a total SES-CD ≥ 6 at screening, or the ileum segmental SESCD
≥ 4 at screening
Must have failed or experienced intolerance to at least one of the
following: budesonide; systemic corticosteroids; immunosuppressants
(ie, azathioprine [AZA], 6-mercaptopurine
[6-MP], or methotrexate [MTX]); or biologics for the treatment of CD (ie,
infliximab, adalimimumab, certolizumab or vedolizumab)

E.4

Principal exclusion criteria

Diagnosis of ulcerative colitis (UC), indeterminate colitis, ischemic
colitis, microscopic colitis, radiation colitis or diverticular diseaseassociated
colitis
Local manifestations of CD such as abscesses, short bowel syndrome; or
other disease complications for which surgery might be indicated or
could confound the evaluation of efficacy
Strictures with prestenotic dilatation, requiring procedural intervention,
or with obstructive symptoms. In addition, colonic strictures that are not
passable with an adult colonoscope, or strictures in the ileum or
ileocecal valve that are fibrotic in nature, will be excluded.
Any intestinal resection within 6 months or any intra-abdominal surgery
within 3 months prior to the Screening Visit
Prior treatment with mycophenolic acid, tacrolimus, sirolimus,
cyclosporine, thalidomide or apheresis (eg, Adacolumn®) within 8 weeks
prior to the Screening Visit
Use of intravenous (IV) corticosteroids within 2 weeks prior to the
Screening Visit
Use of topical GI treatments such as 5-aminosalicylic acid (5-ASA) or
corticosteroid enemas or suppositories within 2 weeks prior to the
Screening Visit
Use of bile acid sequestrants, (eg, cholestyramine) within 3 weeks prior
to the Screening Visit
Prior treatment with biologics for the treatment of CD (approved or
investigational), other than infliximab, adalimimumab, certolizumab or
vedolizumab
Prior treatment with more than 3 biologics for the treatment of CD (ie,
infliximab, adalimimumab, certolizumab or vedolizumab). Treatment
with a biologic within 8 weeks prior to the Screening Visit, or 5
elimination half lives, whichever is longer.

E.5 End points

E.5.1

Primary end point(s)

Efficacy as clinical remision: the proportion of subjects achieving clinical
remission defined as a CDAI score < 150, at Week 12

E.5.1.1

Timepoint(s) of evaluation of this end point

Week 12

E.5.2

Secondary end point(s)

Proportion of subjects achieving:
- clinical remission, defined as a CDAI score < 150, at Week 52.
- endoscopic response-50 (ER-50), defined as a reduction of at least
50% in SES-CD compared with baseline, at Week 52.
Proportion of subjects with clinical response:
- defined as a decrease from baseline in CDAI ≥ 100 points, at Week 12.
- defined as a decrease from baseline in CDAI ≥ 100 points, at Week 4.
Proportion of subjects:
- achieving clinical remission, defined as a CDAI score < 150, at Week 4.
- who achieve corticosteroid-free clinical remission (CDAI <150) at
Week 52 among subjects receiving oral corticosteroids for CD at
baseline.
- achieving sustained clinical remission, defined as a CDAI score < 150,
at both Week 12 and Week 52.
- with endoscopic response-25 (ER-25), defined as a reduction of at least
25% in the SES-CD compared with baseline, at Week 12.
- endoscopic remission, defined as SES-CD ≤ 2, at Week 52.
Type, frequency, severity, seriousness, and relationship of AEs to IP.
Number of subjects who discontinue investigational product (IP) due to
any AE.
Clinically significant changes in vital signs, ECG and/or laboratory
findings.

E.5.2.1

Timepoint(s) of evaluation of this end point

as given above

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

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)

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

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

311

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

Australia

Austria

Belgium

Bulgaria

Canada

Croatia

Czech Republic

Denmark

Estonia

France

Germany

Greece

Hungary

Israel

Italy

Korea, Republic of

Latvia

Netherlands

New Zealand

Norway

Poland

Portugal

Romania

Russian Federation

Serbia

Slovakia

South Africa

Spain

Sweden

Switzerland

Turkey

Ukraine

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

End of Study is defined as either the date of the LVLS to complete the post-treatment follow-up, or the date of receipt of the last data point from the LS that is required for primary, secondary and/or exploratory analysis, as prespecified in the protocol, whichever is the later date.

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:

1010

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

452

F.4.2.2

In the whole clinical trial

1064

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

2016-04-01

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2015-01-13

P. End of Trial
P.	End of Trial Status	Prematurely Ended
P.	Date of the global end of the trial	2017-10-19

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