Two-period, single-dose pharmacokinetic study in male adolescent patients with Fragile X Syndrome (FXS).

• Register: EUCTR
• Last refreshed on: 19 March 2012
• Main ID: EUCTR2010-019353-18-Outside-EU/EEA
• Date of registration: 31/10/2011
• Prospective Registration: Yes
• Primary sponsor: Novartis Pharma Services AG
• Public title: Two-period, single-dose pharmacokinetic study in male adolescent patients with Fragile X Syndrome (FXS).
• Scientific title: A sequential, open-label, two-period study to assess the pharmacokinetics, safety and tolerability of two dose levels of AFQ056 in male, adolescent patients with Fragile X Syndrome (12 to 18 years inclusive)
• Target sample size: 12
• Recruitment status: NA
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2010-019353-18
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no Randomised: no Open: no Single blind: no Double blind: no Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: no

Countries of recruitment

Switzerland

Contacts

• Name: Clinical Trial Information Desk
• Affiliation: Novartis Pharma AG

• Name: Clinical Trial Information Desk
• Affiliation: Novartis Pharma AG

Key inclusion & exclusion criteria

Inclusion criteria:
1. Male patients with FXS aged 12 - 18 years.

2. Confirmed diagnosis of FXS by sequencing (>200 CGG repeats in the fmr1 gene)

3. Absence of cardiovascular, gastrointestinal, renal, orthopedic, neurological or nervous system co-morbidities (excluding FXS symptoms)

4. Body weight and body mass index must be within 10th to 90th percentile of the age-specific range

Other protocol-defined inclusion criteria may apply
Are the trial subjects under 18? yes
Number of subjects for this age range: 12
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
1. Any subject who does not fulfill each of the inclusion criteria

2. Smokers (reported tobacco use within the last 3 months prior to screening and/or urine cotinine =500 ng/mL

3. Significant illness within 2 weeks prior to the screening visit.

4. Any surgical or medical condition which might significantly alter the absorption, distribution, metabolism or excretion of drugs

5. Any use of CYP3A4 inhibitors or inducers within 4 weeks prior to dosing

6. Use of any herbal supplement or over the counter drugs within 4 weeks prior to dosing.

7. Positive HIV or hepatisis B/C

8. History of drug or alcohol abuse within 12 months prior to dosing

Other protocol-defined exclusion criteria may apply


Age minimum:
Age maximum:
Gender:
Female: no
Male: yes

Health Condition(s) or Problem(s) studied

Fragile X Syndrome
MedDRA version: 14.0 Level: PT Classification code 10017324 Term: Fragile X syndrome System Organ Class: 10010331 - Congenital, familial and genetic disorders

Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

Intervention(s)

Product Name: AFQ056
Product Code: AFQ056
Pharmaceutical Form: Capsule, hard
INN or Proposed INN: mavoglurant
CAS Number: 543906-09-8
Current Sponsor code: AFQ056
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 25-
Pharmaceutical form of the placebo: Capsule, hard
Route of administration of the placebo: Oral use

Product Name: AFQ056
Product Code: AFQ056
Pharmaceutical Form: Capsule, hard
INN or Proposed INN: mavoglurant
CAS Number: 543906-09-8
Current Sponsor code: AFQ056
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 100-
Pharmaceutical form of the placebo: Capsule, hard
Route of administration of the placebo: Oral use

Primary Outcome(s)

Main Objective: - To assess the pharmacokinetics of single oral AFQ056 doses (25 mg, then 50 mg or 100 mg) in adolescent males aged 12-18 years, with Fragile X Syndrome (FXS)

Primary end point(s): Pharmacokinetic parameters:
- AUCInf,
- AUC last
- Cmax

Timepoint(s) of evaluation of this end point: Sampling at 1, 2.5, 4, 8, 12, 16 and 24 hours post dose in each treatment period 1 & 2

Secondary Objective: - To assess the safety and tolerability of single oral AFQ056 doses (25 mg, then 50 mg or 100 mg) in adolescent males aged 12-18 years, with FXS

Secondary Outcome(s)

Secondary end point(s): Safety and tolerability assessment:
- Collection of all Adverse events
- Hematology
- Blood chemistry
- Urinalysis
- Vital signs
- Physical condition
- Body weight
- ECG
- Neurological assessment

Timepoint(s) of evaluation of this end point: Safety and tolerability continuously monitored throughout the course of the trial

Secondary ID(s)

CAFQ056B2131

Source(s) of Monetary Support

Novartis Pharma Services AG