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
Conditions
Interventions
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)
C
Sponsors
Novartis Pharma Services AG
Eligibility
Sex/Gender
Male
Inclusion 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
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 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);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;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
| Measure | Time frame |
|---|---|
| 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 | — |
Countries
Switzerland
Contacts
Public ContactClinical Trial Information Desk
Novartis Pharma AG
Outcome results
None listed