Efficacy, Safety and Tolerability of AFQ056 in Fragile X Patients - Full Text View

Purpose

This study will evaluate the safety, tolerability and efficacy of multiple doses of AFQ056 in patients with Fragile X Syndrome. The dose range will be 50 to 150 mg b.i.d. The primary read-out of efficacy is reduction in Aberrant-Behavior Checklist score.

Condition	Intervention	Phase
Fragile X Syndrome	Drug: AF056 Drug: Placebo	Phase 2


Study Type:	Interventional
Study Design:	Allocation: Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Crossover Assignment Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor) Primary Purpose: Treatment
Official Title:	A Multi-centre, Randomized, Double-blind, Placebo Controlled, Two-period, Crossover Proof-of-concept Study in Male Patients With Fragile X Syndrome to Assess the Efficacy, Safety and Tolerability of Multiple Oral Doses of AFQ056

Resource links provided by NLM:

Genetics Home Reference related topics: CASK-related intellectual disability fragile X syndrome MECP2 duplication syndrome PPM-X syndrome Renpenning syndrome tetrasomy 18p

MedlinePlus related topics: Fragile X Syndrome

Genetic and Rare Diseases Information Center resources: Fragile X Syndrome X-linked Mental Retardation and Macro-orchidism

U.S. FDA Resources

Further study details as provided by Novartis:

Primary Outcome Measures:

Aberrant-Behavior Checklist- Community Edition

Secondary Outcome Measures:

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…)


Enrollment:	30
Study Start Date:	June 2008
Primary Completion Date:	February 2009 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
Active Comparator: 1	Drug: AF056
Placebo Comparator: 2	Drug: Placebo

Eligibility


Ages Eligible for Study:	18 Years to 35 Years
Genders Eligible for Study:	Male
Accepts Healthy Volunteers:	No

Criteria

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 (at screening)
Patients with a mental age of ≥ 48 months as measured by the Stanford-Binet test

Exclusion Criteria:

Patients with DSM-IV diagnosis of schizophrenia, history and/or presence of psychosis, confusional states and/or repeated hallucinations.
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.)

Other protocol-defined inclusion/exclusion criteria may apply.

Contacts and Locations

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00718341

Locations


France
Novartis Investigator Site
Bron cedex, France, 69677
Italy
Novartis Investigator Site
Rome, Italy, 00168
Switzerland
Novartis Investigator Site
Lausanne, Switzerland, 1011

Sponsors and Collaborators

Novartis

Investigators


Principal Investigator:	Novartis	Novartis investigator site

More Information

No publications provided by Novartis

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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.


Responsible Party:	External Affairs, Novartis
ClinicalTrials.gov Identifier:	NCT00718341 History of Changes
Other Study ID Numbers:	CAFQ056A2204
Study First Received:	July 17, 2008
Last Updated:	May 5, 2010
Health Authority:	France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)

Keywords provided by Novartis:


Fragile X Syndrome, adults, efficacy

Additional relevant MeSH terms:


Fragile X Syndrome Syndrome Chromosome Disorders Congenital Abnormalities Disease Genetic Diseases, Inborn Genetic Diseases, X-Linked Heredodegenerative Disorders, Nervous System	Intellectual Disability Mental Retardation, X-Linked Nervous System Diseases Neurobehavioral Manifestations Neurologic Manifestations Pathologic Processes Sex Chromosome Disorders

ClinicalTrials.gov processed this record on March 01, 2015