Safety and Efficacy of AFQ056 in Adult Patients With Fragile X Syndrome
This study is currently recruiting participants.
Verified January 2013 by Novartis
Sponsor:
Novartis Pharmaceuticals
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier:
NCT01253629
First received: December 2, 2010
Last updated: January 9, 2013
Last verified: January 2013
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Purpose
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.
| Condition | Intervention | Phase |
|---|---|---|
|
Fragile X Syndrome |
Drug: AFQ056 Drug: Placebo |
Phase 2 |
| Study Type: | Interventional |
| Study Design: | Allocation: Randomized Intervention Model: Parallel Assignment Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor) Primary Purpose: Treatment |
| Official Title: | A Randomized, Double-blind, Placebo-controlled, Parallel Group Study to Evaluate AFQ056 in Adult Patients With Fragile X Syndrome |
Resource links provided by NLM:
Genetics Home Reference related topics:
fragile X syndrome
MECP2 duplication syndrome
PPM-X syndrome
Renpenning syndrome
tetrasomy 18p
MedlinePlus related topics:
Fragile X Syndrome
U.S. FDA Resources
Further study details as provided by Novartis:
Primary Outcome Measures:
- Change from baseline in behavioral symptoms of Fragile X Syndrome using the Aberrant Behavior Checklist - Community (ABC-C) Total score in Stratum I [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]
Secondary Outcome Measures:
- Change from baseline in behavioral symptoms of Fragile X Syndrome using the ABC-C Total score in Stratum II [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]
- Safety and tolerability as measured by changes in vital signs, ECGs, laboratory values and percentages of adverse events and serious adverse events [ Time Frame: 12 weeks ] [ Designated as safety issue: Yes ]
- Global improvement of symptoms in Fragile X using the Clinical Global Impression-Improvement (CGI-I) scale [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]
- Change from baseline in irritability, lethargy/social withdrawal, stereotypic behavior, hyperactivity, and inappropriate speech assessed by the individual subscales of the ABC-C scale [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]
- The proportion of patients with clinical response, where response is defined as a reduction of at least 25% from baseline in the ABC-C total score and a score of 1 (very much improved) or 2 (much improved) on the CGI-I scale [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]
| Estimated Enrollment: | 175 |
| Study Start Date: | November 2010 |
| Estimated Study Completion Date: | December 2013 |
| Estimated Primary Completion Date: | December 2013 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
| Experimental: 25 mg bid | Drug: AFQ056 |
| Experimental: 50 mg bid | Drug: AFQ056 |
| Experimental: 100 mg bid | Drug: AFQ056 |
| Placebo Comparator: Placebo | Drug: Placebo |
Eligibility| Ages Eligible for Study: | 18 Years to 45 Years |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion Criteria:
- Patients with Fragile X Syndrome, who are at least moderately ill based on a Clinical Global Impression Severity score of at least 4 and have qualifying scores on the ABC-C and IQ test at Visit 1
Exclusion Criteria:
- Advanced, severe or unstable disease that may interfere with the study outcome evaluations
- Cancer within the past 5 years, other than localized skin cancer
- Current treatment with more than two psychoactive medications, excluding anti-epileptics
- History of severe self-injurious behavior
Other protocol-defined inclusion/exclusion criteria may apply
Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01253629
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Contacts
| Contact: Novartis Pharmaceuticals | +1(862)778-8300 |
Hide Study LocationsLocations
| United States, Arizona | |
| Novartis Investigative Site | Recruiting |
| Phoenix, Arizona, United States, 85006 | |
| Contact: Sajitha Puthalathi 602-218-8195 | |
| United States, California | |
| Novartis Investigative Site | Recruiting |
| Sacramento, California, United States, 95817 | |
| Contact: Emma Hare 916-703-0472 emma.hare@ucdmc.ucdavis.edu | |
| United States, Georgia | |
| Novartis Investigative Site | Recruiting |
| Decatur, Georgia, United States, 30033 | |
| Contact: Heather Clark 404-778-8484 | |
| United States, Illinois | |
| Novartis Investigative Site | Recruiting |
| Chicago, Illinois, United States, 60612 | |
| Contact: Crystal Hervey 312-942-7250 | |
| United States, Indiana | |
| Novartis Investigative Site | Recruiting |
| Indianapolis, Indiana, United States, 46202 | |
| Contact 855-928-8476 | |
| United States, Massachusetts | |
| Novartis Investigative Site | Not yet recruiting |
| Boston, Massachusetts, United States, 02115 | |
| Novartis Investigative Site | Not yet recruiting |
| Springfield, Massachusetts, United States, 01199 | |
| United States, Nebraska | |
| Novartis Investigative Site | Recruiting |
| Omaha, Nebraska, United States, 68198-5575 | |
| Contact: Brigette Vaughan 402-552-6005 | |
| United States, New York | |
| Novartis Investigative Site | Recruiting |
| Staten Island, New York, United States, 10314 | |
| Contact: Angelo Porto 718-494-8028 | |
| United States, Pennsylvania | |
| Novartis Investigative Site | Recruiting |
| Media, Pennsylvania, United States, 19063 | |
| Contact: Maureen ODonnell 610-891-9024 ext 111 | |
| United States, South Carolina | |
| Novartis Investigative Site | Recruiting |
| Greenwood, South Carolina, United States, 29646 | |
| Contact: Fran Annese 864-941-8100 | |
| United States, Tennessee | |
| Novartis Investigative Site | Recruiting |
| Nashville, Tennessee, United States, 37212 | |
| Contact: Tynya Patterson 615-343-2018 | |
| United States, Texas | |
| Novartis Investigative Site | Terminated |
| Houston, Texas, United States, 77090 | |
| United States, Washington | |
| Novartis Investigative Site | Recruiting |
| Bellevue, Washington, United States, 98004 | |
| Novartis Investigative Site | Withdrawn |
| Seattle, Washington, United States, 98105 | |
| Australia, New South Wales | |
| Novartis Investigative Site | Recruiting |
| Ryde, New South Wales, Australia, 2112 | |
| Novartis Investigative Site | Recruiting |
| Waratah, New South Wales, Australia, 2298 | |
| Australia, Victoria | |
| Novartis Investigative Site | Recruiting |
| Caulfield, Victoria, Australia, 3161 | |
| Canada, Ontario | |
| Novartis Investigative Site | Recruiting |
| Brampton, Ontario, Canada, L6Y 1M5 | |
| Canada, Quebec | |
| Novartis Investigative Site | Recruiting |
| Montreal, Quebec, Canada, H3T1C5 | |
| Novartis Investigative Site | Recruiting |
| Sherbrooke, Quebec, Canada, J1H 5N4 | |
| Denmark | |
| Novartis Investigative Site | Recruiting |
| Glostrup, Denmark, 2600 | |
| France | |
| Novartis Investigative Site | Recruiting |
| Bron Cedex, France, 69677 | |
| Novartis Investigative Site | Recruiting |
| Paris, France, 75013 | |
| Germany | |
| Novartis Investigative Site | Recruiting |
| Berlin, Germany, 12200 | |
| Novartis Investigative Site | Recruiting |
| Mainz, Germany, D-55101 | |
| Novartis Investigative Site | Recruiting |
| Tübingen, Germany, 72076 | |
| Novartis Investigative Site | Recruiting |
| Würzburg, Germany, 97070 | |
| Italy | |
| Novartis Investigative Site | Recruiting |
| Genova, GE, Italy, 16147 | |
| Novartis Investigative Site | Recruiting |
| Roma, RM, Italy, 00168 | |
| Novartis Investigative Site | Withdrawn |
| Padova, Italy, 35128 | |
| Spain | |
| Novartis Investigative Site | Recruiting |
| Málaga, Andalucía, Spain, 29009 | |
| Novartis Investigative Site | Recruiting |
| Sant Cugat, Barcelona, Spain, 08190 | |
| Switzerland | |
| Novartis Investigative Site | Recruiting |
| Lausanne, Switzerland | |
| Novartis Investigative Site | Recruiting |
| Zurich, Switzerland, 8091 | |
| United Kingdom | |
| Novartis Investigative Site | Recruiting |
| Edinburgh, United Kingdom, EH10 5HF | |
Sponsors and Collaborators
Novartis Pharmaceuticals
Investigators
| Study Director: | Novartis Pharmaceuticals | Novartis Pharmaceuticals |
More Information
No publications provided
| Responsible Party: | Novartis ( Novartis Pharmaceuticals ) |
| ClinicalTrials.gov Identifier: | NCT01253629 History of Changes |
| Other Study ID Numbers: | CAFQ056A2212, 2009-013667-19 |
| Study First Received: | December 2, 2010 |
| Last Updated: | January 9, 2013 |
| Health Authority: | United States: Food and Drug Administration Canada: Health Canada Denmark: Danish Medicines Agency France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis) Germany: BfArM Italy: Ethics Committee United Kingdom: Medicines and Healthcare Products Regulatory Agency Australia: Department of Health and Ageing Therapeutic Goods Administration Switzerland: Swissmedic Spain: Agencia Española de Medicamentos y Productos Sanitarios |
Keywords provided by Novartis:
|
Fragile X Syndrome Martin-Bell Syndrome Genetic Diseases X-Linked |
Additional relevant MeSH terms:
|
Fragile X Syndrome Mental Retardation, X-Linked Genetic Diseases, X-Linked Mental Retardation Neurobehavioral Manifestations Neurologic Manifestations |
Nervous System Diseases Sex Chromosome Disorders Chromosome Disorders Congenital Abnormalities Genetic Diseases, Inborn Heredodegenerative Disorders, Nervous System |
ClinicalTrials.gov processed this record on May 21, 2013