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AF056

Phase 2

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

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLED
Total Trials1
Total Enrollment30

FDA Designations

No designations recorded

Clinical trial landscape

AF056 · 1 trial · 1 indication

Phase 2 1
NCT00718341Efficacy, Safety and Tolerability of AFQ056 in Fragile X PatientsFragile X Syndrome
COMPLETED30 Analytics
PHASE2COMPLETED
Efficacy, Safety and Tolerability of AFQ056 in Fragile X Patients
Fragile X SyndromeUnlock trial analytics

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 to 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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Frequently asked questions about AF056

What is AF056 used for?

AF056 is an investigational small molecule being developed for the treatment of Fragile X Syndrome, a rare genetic condition. It is currently in Phase 2 clinical development and has not been approved by regulatory authorities.

Who makes AF056?

AF056 is being developed by Novartis AG, a multinational pharmaceutical company. Novartis is listed on the stock exchange under the ticker symbol NVS.

What phase is AF056 in?

AF056 is in Phase 2 clinical development. It is an investigational drug and has not yet received regulatory approval. The drug is being studied for its efficacy, safety, and tolerability in patients with Fragile X Syndrome.

What clinical trials is AF056 in?

AF056 has been studied in one completed Phase 2 clinical trial, identified as NCT00718341. This trial evaluated the efficacy, safety, and tolerability of AF056 in 30 male patients with Fragile X Syndrome, aged 18 years and older, across France, Italy, and Switzerland.

Is AF056 the same as AFQ056?

Yes, AF056 is also known as AFQ056. The clinical trial NCT00718341, which studied the drug in Fragile X Syndrome patients, uses the name AFQ056 in its official title.