Clinical Trials Register

Summary
EudraCT Number:	2011-004867-65
Sponsor's Protocol Code Number:	CAFQ056B2154
Clinical Trial Type:	Outside EU/EEA
Date on which this record was first entered in the EudraCT database:	2012-02-29
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
H.4 THIRD COUNTRY IN WHICH THE TRIAL WAS FIRST AUTHORISED

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A. Protocol Information

A.2

EudraCT number

2011-004867-65

A.3

Full title of the trial

Sequential, two-period study to assess the pharmacokinetics, safety & tolerability of single and multiple oral doses of AFQ056 in patients with FXS (Fragile X syndrome) aged 5-11 years (Cohort 1) and 3-4 years (Cohort 2)

A.3.1

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

Clinical study to assess the pharmacokinetics, safety & tolerability of single and multiple oral doses of AFQ056 in children with Fragile X Syndrome

A.4.1

Sponsor's protocol code number

CAFQ056B2154

A.5.2

US NCT (ClinicalTrials.gov registry) number

NCT01482143

A.7

Trial is part of a Paediatric Investigation Plan

Yes

A.8

EMA Decision number of Paediatric Investigation Plan

P/152/2011

B. Sponsor Information
B.Sponsor: 1
B.1.1	Name of Sponsor	Novartis Pharma Services AG
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	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 Pharma AG
B.5.2	Functional name of contact point	Clinical Trial Information Desk
B.5.3	Address:
B.5.3.1	Street Address	Novartis Campus
B.5.3.2	Town/ city	Basel
B.5.3.3	Post code	4056
B.5.3.4	Country	Switzerland
B.5.4	Telephone number	+41613241111
B.5.5	Fax number	+41613248001
B.5.6	E-mail	clinicaltrial.enquiries@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	Powder for oral suspension
D.3.4.1	Specific paediatric formulation	Yes
D.3.7	Routes of administration for this IMP	Oral use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.9.1	CAS number	543906-09-8
D.3.9.2	Current sponsor code	AFQ056
D.3.10	Strength
D.3.10.1	Concentration unit	mg/ml milligram(s)/millilitre
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	10 mg/ml
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

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

Fragile X Syndrome

E.1.1.1

Medical condition in easily understood language

Fragile X Syndrome

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	14.1
E.1.2	Level	PT
E.1.2	Classification code	10017324
E.1.2	Term	Fragile X syndrome
E.1.2	System Organ Class	10010331 - Congenital, familial and genetic disorders

E.1.3

Condition being studied is a rare disease

Yes

E.2 Objective of the trial

E.2.1

Main objective of the trial

To assess the pharmacokinetics of single and multiple oral AFQ056 doses
in patients with FXS aged 5-11 years (cohort 1) and 3-4 years if included
in the study (cohort 2)

E.2.2

Secondary objectives of the trial

To evaluate the safety and tolerability of single and multiple oral AFQ056
in patients with FXS aged 5 11 years (cohort 1) and 3-4 years if included
in the study (cohort 2)

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

Genetically confirmed diagnosis of FXS

At Screening and first baseline, vital signs, body weight and BMI must be
age-specific within normal ranges

E.4

Principal exclusion criteria

Use of any other investigational drug within 30 days or 5 half-lives
(whichever is longer) of the investigational drug prior to screening until
end of study visit.

History of hypersensitivity to AFQ056 or any mGluR antagonist.

Female patients who are confirmed or suspected to be sexually active.

History or presence of any clinically significant disease of any major
system organ class, within the past 2 years prior to screening including
but not limited to psychiatric, neurological, cardiovascular, endocrine,
metabolic, renal, or gastrointestinal disorders (except for typical
features of FXS).

Smokers.

Loss of ≥10% of total blood volume within 8 weeks (or less if required
for this age group and/or by local regulation) prior to dosing or longer if
required for this age group and/or by local regulation.

Significant illness that did not completely resolve at least four weeks
prior to the first baseline visit.

Any abnormal laboratory values at screening or first baseline that are in
the opinion of the investigator clinically significant and may jeopardize
the safety of the study subject.

Use of (or use within at least 5 half lives before dosing) concomitant
medications that are strong/moderate inhibitors or inducers of
CYP1A1/2, CYP2C9/19 or CYP3A4

History or presence of Hepatitis B/C or HIV at screening

E.5 End points

E.5.1

Primary end point(s)

The area under the plasma (or serum or blood) concentration-time curve
from time zero to infinity [mass x time / volume] (AUCinf)

The area under the plasma (or serum or blood) concentration-time curve
from time zero to the time of the last quantifiable concentration [mass x
time/ volume] (AUClast)

Maximum observed plasma concentration (Cmax)

E.5.1.1

Timepoint(s) of evaluation of this end point

The area under the plasma (or serum or blood) concentration-time curve
from time zero to infinity [mass x time / volume] (AUCinf) :Time Frame:
Day 1 (period 1): 0.5, 2, 4, 8, 12, 24 hours post-dose; Day 7 (period 2):
pre-dose; 0.5, 2, 4, 8 hours post dose

The area under the plasma (or serum or blood) concentration-time curve
from time zero to the time of the last quantifiable concentration [mass x
time/ volume] (AUClast):Time Frame: Day 1 (period 1): 0.5, 2, 4, 8, 12, 24
hours post-dose; Day 7 (period 2): pre-dose; 0.5, 2, 4, 8 hours post dose

Maximum observed plasma concentration (Cmax): Day 1 (period 1): 0.5,
2, 4, 8, 12, 24 hours post-dose; Day 7 (period 2): pre-dose; 0.5, 2, 4, 8
hours post dose

E.5.2

Secondary end point(s)

Physical examination
Vital signs and body measurements
ECG
hematology
blood chemistry
neurological examination
Adverse events (AE) and Serious adverse events (SAE) monitoring

E.5.2.1

Timepoint(s) of evaluation of this end point

Measure #1, 2, 3:Time Frame: Screening: once anytime between Day -30
and Day -1, Day -1 (period 1), once anytime between 24-72 hours after
Day 7 (period 2)

Measure #4, 5: Time Frame: Screening: once anytime between Day -30
and Day -1, Day -1 (period 1), Day -1 (period 2); once anytime between
24-72 hours after Day 7 (period 2)

Measure #6:Time Frame: Screening: once anytime between Day -30 and
Day -1, Day -1 (period 1), Day 7 (period 2)

Measure #7:Time Frame: During the study (total of approximately 32
days) and 3 days after study completion

Measure #8:Time Frame: During the study (total of approximately 32
days) and 30 days after study completion

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

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

Yes

E.6.13.1

Other scope of the trial description

Tolerability

E.7

Trial type and phase

E.7.1

Human pharmacology (Phase I)

Yes

E.7.1.1

First administration to humans

E.7.1.2

Bioequivalence study

E.7.1.3

Other

Yes

E.7.1.3.1

Other trial type description

PK, safety, tolerability

E.7.2

Therapeutic exploratory (Phase II)

E.7.3

Therapeutic confirmatory (Phase III)

E.7.4

Therapeutic use (Phase IV)

E.8 Design of the trial

E.8.1

Controlled

E.8.1.1

Randomised

E.8.1.2

Open

Yes

E.8.1.3

Single blind

E.8.1.4

Double blind

E.8.1.5

Parallel group

E.8.1.6

Cross over

E.8.1.7

Other

Yes

E.8.1.7.1

Other trial design description

single group, open, non-randomized

E.8.2

Comparator of controlled trial

E.8.2.1

Other medicinal product(s)

E.8.2.2

Placebo

E.8.2.3

Other

E.8.2.4

Number of treatment arms in the trial

E.8.3

Will this trial be conducted at a single site globally?

E.8.4

Will this trial be conducted at multiple sites globally?

Yes

E.8.6 Trial involving sites outside the EEA

E.8.6.2

Trial being conducted completely outside of the EEA

E.8.6.3

Specify the countries outside of the EEA in which trial sites are planned

Spain

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

E.8.9 Initial estimate of the duration of the trial

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

Yes

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)

Yes

F.1.1.5.1

Number of subjects for this age range:

F.1.1.6

Adolescents (12-17 years)

F.1.2

Adults (18-64 years)

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

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

Yes

F.3.3.6.1

Details of subjects incapable of giving consent

Children from age 5 - 11 years (cohort 1) and age 3 - 4 years (cohort 2)
with Fragile X syndrom. Fragile X syndrome is a form of mental
retardation. The legal guardian or legally acceptable representative
must sign Informed Consent.

F.3.3.7

Others

F.4 Planned number of subjects to be included

F.4.2

For a multinational trial

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)

Patients will be offered the opportunity to enroll into an open-label
long-term extension study with AFQ056.

G. Investigator Networks to be involved in the Trial

H.4 Third Country in which the Trial was first authorised
H.4.1	Third Country in which the trial was first authorised:	United States

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