Efficacy, Safety and Tolerability of AFQ056 in Fragile X Patients
NCT ID: NCT00718341
Last Updated: 2020-02-11
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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COMPLETED
PHASE2
30 participants
INTERVENTIONAL
2008-06-30
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
CROSSOVER
TREATMENT
QUADRUPLE
Study Groups
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1
AF056
2
Placebo
Interventions
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AF056
Placebo
Eligibility Criteria
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Inclusion Criteria
* 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 (at screening)
* Patients with a mental age of ≥ 48 months as measured by the Stanford-Binet test
Exclusion Criteria
* Patients with a history of seizures in the past 5 years without any therapeutic treatment controlling the disorders.
* Patients under stable anti-convulsant therapies that experienced seizures in the 2 years prior to randomization
* Patients with ECG abnormalities, autonomic dysfunctions, bronchospastic diseases, drug or atopic allergy
* Any surgical or medical condition which might significantly alter the absorption, distribution, metabolism or excretion of drugs
* Patients using (or have used within four weeks before randomization) concomitant medications that are potent inhibitors of CYP3A4 (e.g., ketoconazole, ritonavir, etc.)
18 Years
35 Years
MALE
No
Sponsors
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Novartis
INDUSTRY
Responsible Party
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Novartis
Principal Investigators
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Novartis
Role: PRINCIPAL_INVESTIGATOR
Novartis investigator site
Locations
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Novartis Investigator Site
Bron, , France
Novartis Investigator Site
Rome, , Italy
Novartis Investigator Site
Lausanne, , Switzerland
Countries
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References
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Jacquemont S, Curie A, des Portes V, Torrioli MG, Berry-Kravis E, Hagerman RJ, Ramos FJ, Cornish K, He Y, Paulding C, Neri G, Chen F, Hadjikhani N, Martinet D, Meyer J, Beckmann JS, Delange K, Brun A, Bussy G, Gasparini F, Hilse T, Floesser A, Branson J, Bilbe G, Johns D, Gomez-Mancilla B. Epigenetic modification of the FMR1 gene in fragile X syndrome is associated with differential response to the mGluR5 antagonist AFQ056. Sci Transl Med. 2011 Jan 5;3(64):64ra1. doi: 10.1126/scitranslmed.3001708.
Other Identifiers
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CAFQ056A2204
Identifier Type: -
Identifier Source: org_study_id
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