Phase 2
N=139
Safety and Efficacy of AFQ056 in Adolescent Patients With Fragile X Syndrome
Fragile X Syndrome
Bottom Line
View on ClinicalTrials.gov: NCT01357239 ↗Enrolled (actual)
139
Serious AEs
1.4%
Results posted
May 2015
Primary outcome: Primary: Change From Baseline in Behavioral Symptoms of Fragile X Syndrome Using the Aberrant Behavior Checklist-Community Edition (ABC-CFX) Total Score in Stratum I Patients Exposed to AFQ056 100 mg Bid — 8.6; -9.4 Score
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- AFQ056 (Drug); Placebo (Drug)
- Age
- Pediatric · 12+ yrs
- Sex
- All
- Sponsor
- Novartis Pharmaceuticals
- Primary completion
- Jan 2014
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change From Baseline in Behavioral Symptoms of Fragile X Syndrome Using the Aberrant Behavior Checklist-Community Edition (ABC-CFX) Total Score in Stratum I Patients Exposed to AFQ056 100 mg Bid |
8.6; -9.4 | — |
| SECONDARY Change From Baseline in Behavioral Symptoms of Fragile X Syndrome Using the ABC-CFX Total Score in Stratum II Patients Exposed to All 3 Doses of AFQ056 |
-6.8; -2.8; -5.7; -3.5 | — |
| SECONDARY Change From Baseline in Behavioral Symptoms of Fragile X Syndrome Using the ABC-CFX Total Score in Stratum I Patients Exposed to the Two Lower Doses of AFQ056 (25 mg Bid and 50 mg Bid) |
-11.8; -3.4; -9.4 | — |
| SECONDARY Global Improvement of Symptoms in Fragile X Using the Clinical Global Impression- Improvement (CGI-I) Scale in Stratum I Patients |
3.3; 3.8; 3.5; 3.1 | — |
| SECONDARY Global Improvement of Symptoms in Fragile X Using the Clinical Global Impression- Improvement (CGI-I) Scale in Stratum II Patients |
3.5; 3.2; 3.5; 3.4 | — |
| SECONDARY Global Improvement of Symptoms in Fragile X Using the Clinical Global Impression-Improvement (CGI-I) Scale in Stratum I |
1; 0; 0; 0; 0; 0 | — |
| SECONDARY Global Improvement of Symptoms in Fragile X Using the Clinical Global Impression-Improvement (CGI-I) Scale in Stratum II |
0; 0; 0; 0; 3; 5 | — |
| SECONDARY Change From Baseline in Irritability, Lethargy/Withdrawal, Stereotypic Behavior, Hyperactivity, Inappropriate Speech and Social Avoidance Assessed by the Individual Subscales of the ABC-CFX Scale in Stratum I |
-3.5; 2.2; 4.2; -2.5; -1.4; -3.5 | — |
| SECONDARY Change From Baseline in Irritability, Lethargy/Withdrawal, Stereotypic Behavior, Hyperactivity, Inappropriate Speech, and Social Avoidance Assessed by the Individual Subscales of the ABC-CFX Scale in Stratum II |
-1.5; -1.3; -0.8; 0.0; -0.8; -0.4 | — |
| SECONDARY 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, Stratum I |
1; 0; 1; 2 | — |
| SECONDARY 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, Stratum II |
1; 4; 1; 2 | — |
| SECONDARY Change From Baseline in Repetitive Behaviors Assessed Using the Repetitive Behavior Scale - Revised (RBS-R) Total and Subscale Scores in Stratum I |
-2.3; -8.5; 1.5; -6.2; -1.0; -1.5 | — |
| SECONDARY Change From Baseline in Repetitive Behaviors Assessed Using the Repetitive Behavior Scale - Revised (RBS-R) Total and Subscale Scores in Stratum II |
-4.3; -5.9; -2.4; -5.0; 0.1; 0.1 | — |
Summary
This Phase IIb study is designed to assess whether 3 doses of AFQ056 are safe and effective in treating the behavioral symptoms of Fragile X Syndrome.
Eligibility 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
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
- Weigh less than 32 kg
- Females who are sexually active
Other protocol-defined inclusion/exclusion criteria may apply
Data sourced from ClinicalTrials.gov (NCT01357239). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.