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AF056

Phase 2

Fragile X Syndrome | Small molecule | Other |Novartis AG|Last Updated: Feb 11, 2020

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
RandomizedDouble-BlindPLACEBO_CONTROLLEDBiomarker
Total Trials1
Total Enrollment30
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT00718341Efficacy, Safety and Tolerability of AFQ056 in Fragile X PatientsPHASE2 COMPLETED 30Jun 1, 2008 -Feb 11, 20203 France, Italy +1
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Study Endpoints
Primary Endpoints
Aberrant-Behavior Checklist- Community Edition
Secondary Endpoints
28 days treatment with AFQ056 on behavior (communication, socialization, daily living, repetitive behaviors, anxiety/avoidance, clinical global improvement)
28 days treatment with AFQ056 on cognition (receptive language, attention, vigilance…)
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelCROSSOVER
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
1ACTIVE_COMPARATOR -
2PLACEBO_COMPARATOR -
Interventions
NameTypeDescription
AF056DRUG -
PlaceboDRUG -
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Eligibility Criteria
Age Range18 Years — 35 Years
SexMALE
Healthy VolunteersNo
Study Sites3

Inclusion Criteria: * Male, non-smoking patients between 18 and 35 years of age (both inclusive). * Patients with fmr1 full mutation (\> 200 CGG repeats) * Patients with a Clinical Global Impression Severity Score (CGI-S) of \> 4 (moderately ill) * Patients with a score of \>20 in the ABC-C scale (...

Countries:FranceItalySwitzerland
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