Safety and Efficacy of AFQ056 in Adolescent Patients With Fragile X Syndrome - Full Text View

Sponsor:	Novartis
Information provided by (Responsible Party):	Novartis
ClinicalTrials.gov Identifier:	NCT01357239

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 Phase 3

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 the Efficacy and Safety of AFQ056 in Adolescent Patients With Fragile X Syndrome

Resource links provided by NLM:

Genetics Home Reference related topics: 17q21.31 microdeletion syndrome MECP2 duplication syndrome PPM-X 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:	160
Study Start Date:	May 2011
Estimated Study Completion Date:	August 2012
Estimated Primary Completion Date:	August 2012 (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:	12 Years to 17 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
Weigh less than 32 kg
Females who are sexually active

Other protocol-defined inclusion/exclusion criteria may apply

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT01357239

Contacts

Contact: Novartis Pharmaceuticals

862-778-8300

Show 28 Study Locations

Sponsors and Collaborators

Novartis

Investigators

Study Director:

Novartis Pharmaceuticals

More Information

No publications provided

Responsible Party:	Novartis
ClinicalTrials.gov Identifier:	NCT01357239 History of Changes
Other Study ID Numbers:	CAFQ056B2214, 2010-022638-96
Study First Received:	May 18, 2011
Last Updated:	May 14, 2012
Health Authority:	United States: Food and Drug Administration Denmark: Danish Medicines Agency France: Afssaps - French Health Products Safety Agency Germany: BfArM Italy: Ethics Committee United Kingdom: Medicines and Healthcare Products Regulatory Agency Switzerland: Swissmedic Australia: Department of Health and Ageing Therapeutic Goods Administration Sweden: Medical Products Agency Spain: Agencia Española de Medicamentos y Productos Sanitarios Canada: Health Canada

Keywords provided by Novartis:

Fragile X Syndrome
Martin-Bell Syndrome
Genetic Diseases
X-Linked
Escalante's syndrome

Additional relevant MeSH terms:

Fragile X Syndrome
Mental Retardation, X-Linked
Mental Retardation
Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases

Sex Chromosome Disorders
Chromosome Disorders
Congenital Abnormalities
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Heredodegenerative Disorders, Nervous System

ClinicalTrials.gov processed this record on May 23, 2012