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| ID | Type | Description | Link |
|---|---|---|---|
| 2009-013667-19 | EudraCT Number |
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 25 mg bid AFQ056 | Experimental | 1 capsule of 25 mg and 1 capsule of placebo per intake |
|
| 50 mg bid AFQ056 | Experimental | 2 capsules of 25 mg per intake |
|
| 100 mg bid AFQ056 | Experimental | 1 capsule of 100 mg and 1 capsule of placebo per intake |
|
| Placebo | Placebo Comparator | 2 capsules of placebo per intake |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| AFQ056 | Drug | AFQ056, was provided as hard gelatin capsules, 25mg and 100 mg oral dosage strengths, identical in appearance were used |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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) | 12 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| 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. |
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Inclusion Criteria:
Exclusion Criteria:
Other protocol-defined inclusion/exclusion criteria may apply
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| Name | Affiliation | Role |
|---|---|---|
| Novartis Pharmaceuticals | Novartis Pharmaceuticals | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Novartis Investigative Site | Phoenix | Arizona | 85006 | United States | ||
| Novartis Investigative Site |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 26764156 | Derived | Berry-Kravis E, Des Portes V, Hagerman R, Jacquemont S, Charles P, Visootsak J, Brinkman M, Rerat K, Koumaras B, Zhu L, Barth GM, Jaecklin T, Apostol G, von Raison F. Mavoglurant in fragile X syndrome: Results of two randomized, double-blind, placebo-controlled trials. Sci Transl Med. 2016 Jan 13;8(321):321ra5. doi: 10.1126/scitranslmed.aab4109. |
| Label | URL |
|---|---|
| Results for CAFQ056A2212 from the Novartis Clinical Trial website | View source |
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| Placebo | Drug | Placebo medication identical in appearance to active medication was provided |
|
| 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") | 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 | 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 | 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 | Week 12 |
| Sacramento |
| California |
| 95817 |
| United States |
| Novartis Investigative Site | Decatur | Georgia | 30033 | United States |
| Novartis Investigative Site | Chicago | Illinois | 60612 | United States |
| Novartis Investigative Site | Indianapolis | Indiana | 46202 | United States |
| Novartis Investigative Site | Boston | Massachusetts | 02115 | United States |
| Novartis Investigative Site | Omaha | Nebraska | 68198-5575 | United States |
| Novartis Investigative Site | Staten Island | New York | 10314 | United States |
| Novartis Investigative Site | Media | Pennsylvania | 19063 | United States |
| Novartis Investigative Site | Greenwood | South Carolina | 29646 | United States |
| Novartis Investigative Site | Nashville | Tennessee | 37212 | United States |
| Novartis Investigative Site | Houston | Texas | 77090 | United States |
| Novartis Investigative Site | Ryde | New South Wales | 2112 | Australia |
| Novartis Investigative Site | Waratah | New South Wales | 2298 | Australia |
| Novartis Investigative Site | Caulfield | Victoria | 3161 | Australia |
| Novartis Investigative Site | Brampton | Ontario | L6Y 1M5 | Canada |
| Novartis Investigative Site | Sherbrooke | Quebec | J1H 5N4 | Canada |
| Novartis Investigative Site | Glostrup Municipality | 2600 | Denmark |
| Novartis Investigative Site | Bron | 69677 | France |
| Novartis Investigative Site | Paris | 75013 | France |
| Novartis Investigative Site | Berlin | 12200 | Germany |
| Novartis Investigative Site | Mainz | 55131 | Germany |
| Novartis Investigative Site | Tübingen | 72076 | Germany |
| Novartis Investigative Site | Würzburg | 97070 | Germany |
| Novartis Investigative Site | Genova | GE | 16147 | Italy |
| Novartis Investigative Site | Roma | RM | 00168 | Italy |
| Novartis Investigative Site | Málaga | Andalusia | 29009 | Spain |
| Novartis Investigative Site | Sant Cugat del Vallès | Catalonia | 08190 | Spain |
| Novartis Investigative Site | Lausanne | Switzerland |
| Novartis Investigative Site | Zurich | 8091 | Switzerland |
| Novartis Investigative Site | Edinburgh | EH10 5HF | United Kingdom |
| ID | Term |
|---|---|
| D005600 | Fragile X Syndrome |
| D030342 | Genetic Diseases, Inborn |
| ID | Term |
|---|---|
| D038901 | X-Linked Intellectual Disability |
| D008607 | Intellectual Disability |
| D019954 | Neurobehavioral Manifestations |
| D009461 | Neurologic Manifestations |
| D009422 | Nervous System Diseases |
| D025064 | Sex Chromosome Disorders |
| D025063 | Chromosome Disorders |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D040181 | Genetic Diseases, X-Linked |
| D020271 | Heredodegenerative Disorders, Nervous System |
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| ID | Term |
|---|---|
| C581397 | mavoglurant |
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