Clinical Trials Register

Summary
EudraCT Number:	2011-002640-27
Sponsor's Protocol Code Number:	GED-301-01-11
National Competent Authority:	Germany - BfArM
Clinical Trial Type:	EEA CTA
Trial Status:	Completed
Date on which this record was first entered in the EudraCT database:	2012-08-23
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

Germany - BfArM

A.2

EudraCT number

2011-002640-27

A.3

Full title of the trial

A phase II multicenter, randomized, double-blind, controlled vs placebo, dose-finding study on the efficacy and safety of GED-0301, in patients with active Crohn‟s disease (Ileo-Colitis)

Multizentrische randomisierte, doppelblinde placebokontrollierte Phase II Studie zur Bewertung der wirksamen Dosis sowie der Wirksamkeit und der Sicherheit von GED-0301 bei Patienten mit Morbus Crohn in akutem Krankheitsstadium (Ileo-Colitis)

A.3.1

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

A phase II multicenter, randomized, double-blind, controlled vs placebo, dose-finding study on the efficacy and safety of GED-0301, in patients with active Crohn’s disease (Ileo-Colitis)

A.3.2

Name or abbreviated title of the trial where available

I.G.O.N. 1 STUDY

I.G.O.N. 1 Studie

A.4.1

Sponsor's protocol code number

GED-301-01-11

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	GIULIANI
B.1.3.4	Country	Italy
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	GIULIANI S.P.A.
B.4.2	Country	Italy
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	GIULIANI S.P.A.
B.5.2	Functional name of contact point	PHARMA DIVISION
B.5.3	Address:
B.5.3.1	Street Address	VIA P. PALAGI 2
B.5.3.2	Town/ city	MILANO
B.5.3.3	Post code	20129
B.5.3.4	Country	Italy
B.5.4	Telephone number	+390220541
B.5.5	Fax number	+39022054222
B.5.6	E-mail	sbellinvia@giulianipharma.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	GED-0301
D.3.2	Product code	40 mg
D.3.4	Pharmaceutical form	Modified-release 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	NA
D.3.9.1	CAS number	NA
D.3.9.2	Current sponsor code	NA
D.3.9.3	Other descriptive name	GED-0301
D.3.9.4	EV Substance Code	NA
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.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	GED-0301
D.3.2	Product code	10 mg
D.3.4	Pharmaceutical form	Modified-release 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	NA
D.3.9.1	CAS number	NA
D.3.9.2	Current sponsor code	NA
D.3.9.3	Other descriptive name	GED-0301
D.3.9.4	EV Substance Code	NA
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	10
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	Modified-release 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

Morbus Crohn in akutem Krankheitsstadium

E.1.1.1

Medical condition in easily understood language

ACTIVE CROHN'S DISEASE

Morbus Crohn in akutem Krankheitsstadium

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	14.1
E.1.2	Level	LLT
E.1.2	Classification code	10021315
E.1.2	Term	Ileitis terminal
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

a. Efficacy: the primary efficacy endpoint will be the percentage of patients in remission defined as CDAI < 150 at day 15 (after 14 days of study drug treatment) wich is maintained at Week 4.
b. Evaluation of safety of GED-0301, 14-day oral administration.

E.2.2

Secondary objectives of the trial

- % of patients in remission at week 2,4 and at week 12
- % of patients attaining 70-point clinical response (defined as a decrease from baseline in CDAI score of 70 points or more) at week 4 and at week 12.
- % of patients attaining 100-point clinical response (defined as a decrease from baseline in CDAI score of 100 points or more) at week 4 and at week 12.
- % of steroid-dependent patients achieving steroid therapy discontinuation at week 12
- Change in the endoscopic index of severity for Crohn’s Disease (SES-CD).This analysis will be done in the patients who accept to perform ileocolonoscopy before treatment and at week 4

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1. Written informed consent, personally signed and dated by the patient prior any study-related procedures is carried out. 2. Male and female outpatients aging 18-75 years old. 3. Female patients not of childbearing potential (women in menopause defined as surgically sterile or one year postmenopausal); female patients of childbearing potential upon negative pregnancy testing at screening and using highly effective contraception (Pearl Index < 1) during the study. 4. Patients with Crohn’s disease in the active phase at screening visit (the activity is defined using the Crohn’s disease Activity Index (CDAI) according to the European guidelines). 5. Crohn’s disease limited to terminal ileum and/or right colon,.that has been documented by instrumental information on localization and extension according to inclusion criteria 9. . 6. Patients with a CDAI score of >220 and ≤400 for at least one week prior to enrollment. 7. No treatment with biologics (e.g.:infliximab, adalimumab, or natalizumab), in the 90 days prior the enrolment. 8. Patients with steroid resistance or steroid dependence, defined according to the ECCO consensus document 9. Absence of Strictures with pre-stenotic dilatation documented by ultrasonography or Rx or NMR, performed within 1 year prior to the enrollment 10. Ability to understand and comply with study procedures and restrictions.

E.4

Principal exclusion criteria

1. Pregnant or breast-feeding women. 2. Patients with Ulcerative Colitis. 3. Patients with Crohn’s disease involving the stomach and/or the proximal small intestine, or patients with lesions confined to the transverse or left colon as assessed by standard clinical criteria. 4. Patients treated with biologics(e.g.:infliximab, adalimumab, or natalizumab), in the 90 days prior the enrolment. 5. Patients in treatment with a standard dose of immunomodulators (e.g.: azathioprine, mercaptopurine, methotrexate) maintained stably for less than 6 months (patients treated with a stable dose for at least 6 months are eligible).The standard doses, according to ECCO consensus document, are 2-2.5 mg/kg/day for azathioprine and 1-1.5 mg/kg/day for mercaptopurine. 6. Oral and/or systemic antibiotic treatment within 3 weeks before screening. 7. Presence of local complications (e.g. abscesses, strictures and fistulae), and/or extra-
intestinal manifestations (arthritis or arthralgia, iritis or uveitis, erythema nodosum,or phyoderma gangrenosum or aphthous stomatitis, fever >37.8°C) dysplasia and malignancies. 8. A history of colon surgery performed within the past 12 months prior to first dose or an ileal resection more than 70 cm in the past. 9. Strictures with pre-stenotic dilatation. 10. Presence of stoma or ileo-recto-anastomosis. 11. Screening laboratory values within the following parameters:  APTT > 1.5 ULN  plateletcount ≤100,000 /mm3  serum creatinine >1.5 ULN  total bilirubin >1.5 ULN (excluding Gilbert Syndrome)  AST and ALT >1.5 ULN. 12. QTc interval >450 msec for males and >470 msec for females. 13. Current or relevant previous history of serious, severe or unstable (acute or progressive) physical or psychiatric illness, including infections, malignancy, medical disorder that may require treatment (e.g. renal or hepatic impairment) or that make the subject unlikely to fully complete the study, or any condition that presents undue risk from the study medication or procedures. 14. History of alcohol or other drug abuse within the last year. 15. Patients potentially presenting poor reliability (e.g. bad mental conditions). 16. Known hypersensitivity to oligonucleotides or any ingredient in the study products. 17. Patients who used another investigational agent or who took part in a clinical trial within the last 6 months prior first dose.

E.5 End points

E.5.1

Primary end point(s)

a. Efficacy: the primary efficacy endpoint will be the percentage of patients in remission defined as CDAI < 150 at day 15 (after 14 days of study drug treatment) which is maintained at week 4.
b. Evaluation of safety of GED-0301, 14-day oral administration.

E.5.1.1

Timepoint(s) of evaluation of this end point

14 days and 28 days

E.5.2

Secondary end point(s)

% of pts in remission at week 2,4 and at week 12 - % of patients attaining 70-point clinical response (defined as a decrease from baseline in CDAI score of 70 points or more) at week 4 and at week 12. - % of patients attaining 100-point clinical response (defined as a decrease from baseline in CDAI score of 100 points or more) at week 4 and at week 12. - % of steroid-dependent patients achieving steroid therapy discontinuation at week 12 - Change in the endoscopic index of severity for Crohn’s Disease (SES-CD). This analysis will be done in the patients who accept to perform ileocolonoscopy before treatment and at week 4.

E.5.2.1

Timepoint(s) of evaluation of this end point

14 days, week 4 and week 12

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

E.6.7

Pharmacodynamic

E.6.8

Bioequivalence

E.6.9

Dose response

Yes

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

Yes

E.8.2.3.1

Comparator description

Dieselbe Prüfsubstanz in anderer Dosierung.

Same IMP used at different dosage.

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

E.8.6.2

Trial being conducted completely outside of the EEA

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

Number of subjects for this age range:

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:

140

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

160

F.4.2.2

In the whole clinical trial

160

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 since patients will be treated following clinical practice.

Nicht zutreffend. Patienten werden nach medizinischem Standard weiterbehandelt.

G. Investigator Networks to be involved in the Trial
G.4 Investigator Network to be involved in the Trial: 1

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

Competent Authority Decision

Authorised

Date of Competent Authority Decision

2012-11-05

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2013-03-08

P. End of Trial
P.	End of Trial Status	Completed
P.	Date of the global end of the trial	2013-09-30

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