A Phase 2 interventional study of AFQ056 and Placebo in Fragile X Syndrome, sponsored by Novartis Pharmaceuticals. Completed at 31 sites in 10 countries. Open to participants aged 18 Years to 45 Years. Per ClinicalTrials.gov, last updated 2020-12-23.
Sponsored by Novartis Pharmaceuticals (part of Novartis) · Phase 2, Interventional, and Treatment
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.
110 studies on the registry are indexed under Fragile X Syndrome; 23 are open to participants now.
This study's enrollment of 175 is above the median of 58 across 88 interventional studies indexed under Fragile X Syndrome.
Browse Fragile X Syndrome studies →Novartis Pharmaceuticals is the lead sponsor of 2,673 studies on the registry; 228 are open to participants now.
Of its 576 completed or terminated interventional studies of FDA-regulated products, 431 (75%) have results posted.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
Other protocol-defined inclusion/exclusion criteria may apply
1 capsule of 25 mg and 1 capsule of placebo per intake
Drug: AFQ056
2 capsules of 25 mg per intake
Drug: AFQ056
1 capsule of 100 mg and 1 capsule of placebo per intake
Drug: AFQ056
2 capsules of placebo per intake
Drug: Placebo
AFQ056, was provided as hard gelatin capsules, 25mg and 100 mg oral dosage strengths, identical in appearance were used
Placebo medication identical in appearance to active medication was provided
Change from baseline in behavioral symptoms of Fragile X Syndrome using the Aberrant Behavior Checklist - Community (ABC-C) Total score in Stratum I
The ABC-C is a 58-item questionnaire that should have been completed as much as possible by the same rater. It is comprised of five subscales (irritability, lethargy/social withdrawal, stereotypic behavior, hyperactivity and inappropriate speech) plus the total score which ranks from 0 to 174 in patients who were fully methylated (FM)
Time frame: 12 weeks
Change from baseline in behavioral symptoms of Fragile X Syndrome (FXS) using the ABC-C Total score in Stratum II
The ABC-C is a 58-item questionnaire that should have been completed as much as possible by the same rater. It is comprised of five subscales (irritability, lethargy/social withdrawal, stereotypic behavior, hyperactivity and inappropriate speech). Assessing the reduction in the (ABC-C) total score after 12 weeks of treatment in FXS patients with partially methylated (PM) FMR1 gene.
Time frame: 12 weeks
Global improvement of symptoms in Fragile X using the Clinical Global Impression-Improvement (CGI-I) scale
The CGI-I score ranges from 1 to 7 (with 1 being "very much improved", 4 being "no change" to 7 being "very much worse")
Time frame: 12 weeks
Change from baseline in irritability, lethargy/social withdrawal, stereotypic behavior, hyperactivity, and inappropriate speech assessed by the individual subscales of the ABC-C scale
comprised of five subscales (irritability, lethargy/social withdrawal, stereotypic behavior, hyperactivity and inappropriate speech) plus the total score which ranks from 0 to 174
Time frame: 12 weeks
The proportion of patients with clinical response in the ABC-C total score
response is defined as 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 at Week 12
Time frame: 12 weeks
improvement of repetitive behavior as measured by changes in the RBS-R
The Repetitive Behavior Scale - Revised (RBS-R) includes six domains: ritualistic behavior, sameness behavior, stereotypic behavior, self-injurious behavior, compulsive behavior, and restricted interests. A negative change from baseline indicates improvement
Time frame: Week 12
This study is completed, as verified in Mar 2016. You cannot join it, but the record below documents what was studied.
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