Safety and Efficacy of AFQ056 in Adult Patients With Fragile X Syndrome

A randomized, double-blind, placebo-controlled, parallel group study to evaluate AFQ056 in adult patients with Fragile X Syndrome

Status

Active, not recruiting

Phases

Unknown

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2009-013667-19-DK

Enrollment

160

Registered

2010-09-22

Start date

2010-11-16

Completion date

Unknown

Last updated

2014-05-12

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: 15.1 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 Concentration unit: mg milligram(s) Concentration type: equal Concentr

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? no Number of subjects for this age range: 0 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 160 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range 0

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-injurious behavior Other protocol defined exclusion criteria may apply

Design outcomes

Primary

Measure	Time frame
Main Objective: To assess the efficacy of three doses of AFQ056 versus placebo in reducing the ABC-C Total score (using the FXS specific algorithm - ABC-CFX) after 12 weeks of treatment in FXS patients with fully-methylated FMR1 gene.;Secondary Objective: Key secondary objective: To assess the efficacy of AFQ056 versus placebo in reducing irritability, lethargy/social withdrawal, stereotypic behavior, hyperactivity, and inappropriate speech and social avoidance assessed by the corresponding individual subscales of the ABC-CFX after 12 weeks of treatment. Other protocol defined secondary objectives may apply;Primary end point(s): Change from baseline in behavioral symptoms of Fragile X Syndrome using the Aberrant Behavior Checklist - Community (using the FXS specific algorithm - ABC-CFX) Total score in Stratum I ;Timepoint(s) of evaluation of this end point: Timeframe: 12 weeks	—

Measure

Time frame

Main Objective: To assess the efficacy of three doses of AFQ056 versus placebo in reducing the ABC-C Total score (using the FXS specific algorithm - ABC-CFX) after 12 weeks of treatment in FXS patients with fully-methylated FMR1 gene.;Secondary Objective: Key secondary objective: To assess the efficacy of AFQ056 versus placebo in reducing irritability, lethargy/social withdrawal, stereotypic behavior, hyperactivity, and inappropriate speech and social avoidance assessed by the corresponding individual subscales of the ABC-CFX after 12 weeks of treatment. Other protocol defined secondary objectives may apply;Primary end point(s): Change from baseline in behavioral symptoms of Fragile X Syndrome using the Aberrant Behavior Checklist - Community (using the FXS specific algorithm - ABC-CFX) Total score in Stratum I ;Timepoint(s) of evaluation of this end point: Timeframe: 12 weeks

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Secondary

Measure	Time frame
Secondary end point(s): - Change from baseline in behavioral symptoms of Fragile X Syndrome using the FXS specific algorithm - ABC-CFX Total score in Stratum II - Safety and tolerability as measured by changes in vital signs, ECGs, laboratory values and percentages of adverse events and serious adverse events - Global improvement of symptoms in Fragile X using the Clinical Global Impression-Improvement (CGI-I) scale - Change from baseline in irritability, lethargy/social withdrawal, stereotypic behavior, hyperactivity, and inappropriate speech assessed by the individual subscales of the ABC-CFX scale - The proportion of patients with clinical response, where response is defined as a reduction of at least 25% from baseline in the ABC-CFX total score and a score of 1 (very much improved) or 2 (much improved) on the CGI-I scale Other protocol defined endpoints may apply;Timepoint(s) of evaluation of this end point: For each secondary endpoint, Timeframe is 12 weeks	—

Measure

Time frame

Secondary end point(s): - Change from baseline in behavioral symptoms of Fragile X Syndrome using the FXS specific algorithm - ABC-CFX Total score in Stratum II - Safety and tolerability as measured by changes in vital signs, ECGs, laboratory values and percentages of adverse events and serious adverse events - Global improvement of symptoms in Fragile X using the Clinical Global Impression-Improvement (CGI-I) scale - Change from baseline in irritability, lethargy/social withdrawal, stereotypic behavior, hyperactivity, and inappropriate speech assessed by the individual subscales of the ABC-CFX scale - The proportion of patients with clinical response, where response is defined as a reduction of at least 25% from baseline in the ABC-CFX total score and a score of 1 (very much improved) or 2 (much improved) on the CGI-I scale Other protocol defined endpoints may apply;Timepoint(s) of evaluation of this end point: For each secondary endpoint, Timeframe is 12 weeks

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Countries

Australia, Canada, Denmark, France, Germany, Italy, Spain, Switzerland, United Kingdom, United States

Contacts

Public ContactKlinisk forskningsafdeling

Novartis Healthcare A/S

skriv.til@novartis.com+4539168400

Outcome results

None listed

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