Safety and efficacy of AFQ056in adolescent patients with Fragile X Syndrome

A randomized, double-blind, placebo-controlled, parallel group study to evaluate the efficacy and safety of AFQ056 in adolescent patients with Fragile X Syndrome

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2010-022638-96-GB

Enrollment

160

Registered

2011-02-21

Start date

2011-06-15

Completion date

Unknown

Last updated

2019-06-30

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Fragile X Syndrome MedDRA version: 14.1 Level: PT Classification code 10017324 Term: Fragile X syndrome System Organ Class: 10010331 - Congenital, familial and genetic disorders

Interventions

Product Name: AFQ056 Product Code: AFQ056 Pharmaceutical Form: Capsule, hard CAS Number: 543906-09-8 Current Sponsor code: AFQ056

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: Patients with Fragile X Syndrome, who are at least moderately ill based on a Clinical Global Impression Severity score of at least 4 and have qualifying scores on the ABC-C and IQ test at visit 1 Other protocol-defined inclusion criteria may apply Are the trial subjects under 18? yes Number of subjects for this age range: 120 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: -Advanced, severe or unstable disease that may interfere with the study outcome evaluations -Cancer within the past 5 years, other than localized skin cancer -Current treatment with more than two psychoactive medications, excluding anti-epileptics -History of severe self-injurous behaviour -Weigh less than 32kg -Females who are sexually active Other protocol-defined exclusion criteria may apply

Design outcomes

Primary

Measure	Time frame
Main Objective: • To assess the efficacy of AFQ056 100mg BID versus placebo in reducing the ABC-C Total score (using the FXS-specific algorithm - ABC-CFX) after 12 weeks of treatment in FXS patients in Stratum I.; Secondary Objective: Key secondary objectives: To assess the efficacy of three doses of AFQ056 versus placebo in reducing the ABC-CFX total score after 12 weeks of treatment in FXS patients with partially methylated (PM) FMR1 gene To assess the efficacy of two lower doses of AFQ056 (25mg BID and 50mg BID) versus placebo in reducing the ABC-CFX total score after 12 weeks of treatment in FXS patients with FMFMR1 gene ;Primary end point(s): Change from baseline in behavioural symptoms of Fragile X Syndrome using the Aberrant Behaviour Checklist - Community (ABC-CFX) Total score in Stratum I; Timepoint(s) of evaluation of this end point: - Timeframe: 12 weeks At multiple visits: Visits 1, 2, 2.2, 3, 4, 5, 6 and 7	—

Measure

Time frame

Main Objective: • To assess the efficacy of AFQ056 100mg BID versus placebo in reducing the ABC-C Total score (using the FXS-specific algorithm - ABC-CFX) after 12 weeks of treatment in FXS patients in Stratum I.; Secondary Objective: Key secondary objectives: To assess the efficacy of three doses of AFQ056 versus placebo in reducing the ABC-CFX total score after 12 weeks of treatment in FXS patients with partially methylated (PM) FMR1 gene To assess the efficacy of two lower doses of AFQ056 (25mg BID and 50mg BID) versus placebo in reducing the ABC-CFX total score after 12 weeks of treatment in FXS patients with FMFMR1 gene ;Primary end point(s): Change from baseline in behavioural symptoms of Fragile X Syndrome using the Aberrant Behaviour Checklist - Community (ABC-CFX) Total score in Stratum I; Timepoint(s) of evaluation of this end point: - Timeframe: 12 weeks At multiple visits: Visits 1, 2, 2.2, 3, 4, 5, 6 and 7

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Secondary

Measure	Time frame
Secondary end point(s): - Abberant Behaviour Checklist - Community Edition (ABC-CFX): Total score and subscales - Clinical Global Impression - Improvement (CGI-I) - Repetitive Behaviour Scale - Revised (RBS-R) - Safety and tolerability as measured by changes in vital signs, ECGs, laboratory values and percentages of adverse events and serious adverse events, Neuropsychiatric Inventory - Questionnaire (NPI-Q) ; Timepoint(s) of evaluation of this end point: - Timeframe: 12 weeks for each secondary endpoint - ABC-CFX: Visits 1, 2, 2.2, 3, 4, 5, 6 and 7 - CGI-I: Visits 4, 5, 6 and 7 -RBS-R: Visits 2, 3, 5 and 7 - Safety and tolerability: Timeframe 12 weeks -Neuropsychiatric Inventory - Questionnaire (NPI-Q) at visits 2.2, 3, 4, 5, 6 and 7 / Early discontinuation	—

Measure

Time frame

Secondary end point(s): - Abberant Behaviour Checklist - Community Edition (ABC-CFX): Total score and subscales - Clinical Global Impression - Improvement (CGI-I) - Repetitive Behaviour Scale - Revised (RBS-R) - Safety and tolerability as measured by changes in vital signs, ECGs, laboratory values and percentages of adverse events and serious adverse events, Neuropsychiatric Inventory - Questionnaire (NPI-Q) ; Timepoint(s) of evaluation of this end point: - Timeframe: 12 weeks for each secondary endpoint - ABC-CFX: Visits 1, 2, 2.2, 3, 4, 5, 6 and 7 - CGI-I: Visits 4, 5, 6 and 7 -RBS-R: Visits 2, 3, 5 and 7 - Safety and tolerability: Timeframe 12 weeks -Neuropsychiatric Inventory - Questionnaire (NPI-Q) at visits 2.2, 3, 4, 5, 6 and 7 / Early discontinuation

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Countries

Australia, Belgium, Canada, Denmark, France, Germany, Israel, Italy, Netherlands, Spain, Sweden, Switzerland, United Kingdom, United States

Contacts

Public ContactMedical Collaboration Centre

Novartis Pharmaceuticals UK Ltd

medinfo.uk@novartis.com+441276698370

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026