Clinical Trials Register

Summary
EudraCT Number:	2012-005000-17
Sponsor's Protocol Code Number:	CAFQ056A2225
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:	2013-07-25
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

2012-005000-17

A.3

Full title of the trial

A randomized, double-blind, placebo-controlled, parallel-group proof of concept study to evaluate the effect of AFQ056 in obsessive compulsive disorder (OCD) patients resistant to Selective Serotonin Reuptake Inhibitor (SSRI) therapy

A.3.1

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

Proof of concept study to evaluate the effect of AFQ056 in obsessive compulsive disorder (OCD) patients resistant to Selective Serotonin Reuptake Inhibitor (SSRI) therapy

A.4.1

Sponsor's protocol code number

CAFQ056A2225

A.5.2

US NCT (ClinicalTrials.gov registry) number

NCT01813019

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 Services 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 Services 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 s.r.o.
B.5.2	Functional name of contact point	Informační služba – klin. hodnocení
B.5.3	Address:
B.5.3.1	Street Address	Na Pankráci 1724/129
B.5.3.2	Town/ city	Praha 4
B.5.3.3	Post code	140 00
B.5.3.4	Country	Czech Republic
B.5.4	Telephone number	+420 2 25775 202
B.5.5	Fax number	+420225775 205
B.5.6	E-mail	dotazy.klinickehodnoceni@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	AFQ056
D.3.2	Product code	AFQ056
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	Mavoglurant
D.3.9.1	CAS number	543906-09-8
D.3.9.2	Current sponsor code	AFQ056
D.3.9.3	Other descriptive name	AFQ056
D.3.9.4	EV Substance Code	SUB29503
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	25
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	AFQ056
D.3.2	Product code	AFQ056
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	Mavoglurant
D.3.9.1	CAS number	543906-09-8
D.3.9.2	Current sponsor code	AFQ056
D.3.9.3	Other descriptive name	AFQ056
D.3.9.4	EV Substance Code	SUB29503
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	50
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: 3
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	AFQ056
D.3.2	Product code	AFQ056
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	Mavoglurant
D.3.9.1	CAS number	543906-09-8
D.3.9.2	Current sponsor code	AFQ056
D.3.9.3	Other descriptive name	AFQ056
D.3.9.4	EV Substance Code	SUB29503
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	100
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	Modified-release tablet
D.8.4	Route of administration of the placebo	Oral use
D.8 Placebo: 2
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
D.8 Placebo: 3
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

obsessive compulsive disorder (OCD)

E.1.1.1

Medical condition in easily understood language

obsessive compulsive disorder

E.1.1.2

Therapeutic area

Diseases [C] - Nervous System Diseases [C10]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	12.0
E.1.2	Level	HLT
E.1.2	Classification code	10002862
E.1.2	Term	Anxiety disorders NEC (incl obsessive compulsive disorder)
E.1.2	System Organ Class	100000004873

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 effects of AFQ056 (16 weeks treatment), using Y-BOCS change from baseline compared with placebo

E.2.2

Secondary objectives of the trial

• To assess the effect of AFQ056, after 16 weeks treatment, on OCD as
measured by Y-BOCS response rates, Clinical Global Impression of
Improvement (CGI-I) and Clinical Global Impression of Severity (CGI-S)
• To assess the effect of AFQ056, after 16 weeks treatment, on global
social functioning using Sheehan Disability Scale (SDS)
• To assess the effect of AFQ056, after 16 weeks treatment, on
depression as measured by the Hamilton Depression Scale (HAMD-17)
• To assess the effect of AFQ056, after 16 weeks treatment, on anxiety
as measured by the Hamilton Anxiety Scale (HAM-A)
• To assess the effect of AFQ056, after 16 weeks treatment, on cognition
in OCD patients as measured by Cogstate
• To assess the safety and tolerability of multiple titrated oral doses of
AFQ056
• To evaluate the pharmacokinetics of AFQ056 in Patients with OCD

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

Patients eligible for inclusion in this study have to fulfill all of the
following criteria:
1. Male and female, non-smokers patients aged between 18 to 65 years
(inclusive),
2. A primary diagnosis of obsessive-compulsive disorder (OCD) as per
Diagnostic and
Statistical Manual of Mental Disorders (DSM-IV-TR 4th ed, year 2000), as
confirmed by
an Independent Rater.
3. Be on a stable appropriate dose of selective serotonin reuptake
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inhibitor (SSRI) treatment for at least 12 weeks prior to Baseline.
4. Have an insufficient response to current SSRI treatment (as per
Inclusion Criterion 4) and confirmed by an Independent Rater.
Note: Treatment with SSRI and related time period must be documented
in the medical records of the patient (e.g. former prescription, former
medical records). Note that
patient self report is not acceptable.
5. Have a Yale-Brown Obsessive Compulsive Scale (Y-BOCS) of score ≥
20 at Screening (and confirmed by an Independent Rater).
6. Patient must have their eligibility confirmed following the remote (or
face to face)
interview conducted by the Independent Rater.
7. Able to communicate well with the investigator, to understand and
comply with the requirements of the study.

E.4

Principal exclusion criteria

Exclusion criteria in this study:
1. Diagnosis of primary OCD symptom of hoarding.
2. History of more than two unsatisfactory trials with different serotonin reuptake inhibitors within a period of 2 years prior to screening (not including the current treatment with SSRI's given in an dequate dose for at least 12 weeks).
3. Diagnosed with any primary DSM-IV-TR Axis I disorder other than OCD (as confirmed by an Independent Rater); with the exception of depression (refer to criterion 4).
4. Moderate - severe depression, as assessed with a HAM-D score >16 and response to the ATRQ (as confirmed by an Independent rater) at Screening.
5. History eating disorder according to DSM-IV within the last 6 months prior to Screening
6. Diagnosed with antisocial personality disorder (DSM-IV-TR Axis II), as confirmed by an ndependent Rater.
7. Has current or past medical history of bipolar disorder, schizophrenia or other psychotic disorders, schizoaffective disorder, autism (with exception of high-functioning or autistic spectrum disorders e.g. Asperger’s Syndrome), borderline personality disorder, or Gilles de la Tourette syndrome within the last 6 months prior to Screening.
8. Smokers (use of tobacco products in the previous 3 months)
9. History (lifetime) of hallucinations/psychosis that would require
antipsychotic treatment or DSM-IV criteria being met
10. Score "yes" on item 4 or item 5 of the Suicidal Ideation section of the
C-SSRS, if this ideation occurred in the past 6 months, or "yes" on any
item of the Suicidal Behavior section, except for the "Non-Suicidal Self-
Injurious Behavior" (item also included in the Suicidal Behavior section),
if this behavior occurred in the past 2 years.
11. Women of child-bearing potential, defined as all women
physiologically capable of becoming pregnant, unless they are using
highly effective methods* of contraception during dosing and for 30 days
after last dosing of study medication.

E.5 End points

E.5.1

Primary end point(s)

Absolute changes from baseline for Y-Bocs

E.5.1.1

Timepoint(s) of evaluation of this end point

Absolute changes from baseline for Y-Bocs at week 17 (end of 16 week dosing)

E.5.2

Secondary end point(s)

effect on OCD as measured by Y-BOCS response rates, if a subject demonstrates at least 25%
reduction in total Y-BOCS from Baseline then they will be classed as a responder whereas if a
subject has a reduction in Y-BOCS of less than 25% then they will be categorized as a non-responder, Clinical Global Impression of Improvement (CGI-I), Clinical Global Impression of Severity (CGI-S)
effect on global social functioning using Sheehan Disability Scale (SDS)
effect on depression as measured by the Hamilton Depression Scale
(HAMD-17)
effect on anxiety as measured by the Hamilton Anxiety Scale (HAM-A)
effect of AFQ056 on cognition in OCD patients as measured by Cogstate safety and tolerability of multiple titrated oral doses of AFQ056 pharmacokinetics of AFQ056 in Patients with OCD

E.5.2.1

Timepoint(s) of evaluation of this end point

after 16 weeks treatment:
effect on OCD as measured by Y-BOCS response rates, Clinical Global Expression of Improvement (CGI-I), Clinical Global Expression of Severity (CGI-S) and Obsessive-Compulsive Inventory-Revised (OCI-R)
effect on global social functioning using Sheehan Disability Scale (SDS)
effect on depression as measured by the Hamilton Depression Scale (HAMD-17)
effect on anxiety as measured by the Hamilton Anxiety Scale (HAM-A)
effect of AFQ056 on cognition in OCD patients as measured by Cogstate during overall trial: safety and tolerability of multiple titrated oral doses of AFQ056 pharmacokinetics of AFQ056 in Patients with OCD

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

Yes

E.6.11

Pharmacogenomic

E.6.12

Pharmacoeconomic

E.6.13

Others

Yes

E.6.13.1

Other scope of the trial description

This study is designed as a Proof of Concept of AFQ056 in Obsessive Compulsive Disorder (OCD). The purpose of this study is to determine whether AFQ056 as an add on therapy to SSRIs can have eneficial effects by reducing the total score of Y-BOCS (Yale and Brown Obsessive Compulsive Scale) in OCD patients resistant to SSRI treatment (failed SSRI over 12 weeks at appropriate doses).

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

Belgium

Bulgaria

Canada

Czech Republic

France

Germany

Italy

Poland

Switzerland

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

Last visit of the last subject

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

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:

120

F.1.3

Elderly (>=65 years)

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

100

F.4.2.2

In the whole clinical trial

120

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)

No plans for treatment or care after the subjects have ended trial participation; expected normal treatment of that condition

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

2014-01-08

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2013-07-24

P. End of Trial
P.	End of Trial Status	Prematurely Ended
P.	Date of the global end of the trial	2015-03-20

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