Efficacy, Safety and Tolerability of AFQ056 in Fragile X Patients

This study has been completed.
Sponsor:
Information provided by:
Novartis
ClinicalTrials.gov Identifier:
NCT00718341
First received: July 17, 2008
Last updated: May 5, 2010
Last verified: May 2010

July 17, 2008
May 5, 2010
June 2008
February 2009   (final data collection date for primary outcome measure)
Aberrant-Behavior Checklist- Community Edition
Same as current
Complete list of historical versions of study NCT00718341 on ClinicalTrials.gov Archive Site
  • 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…)
Same as current
Not Provided
Not Provided
 
Efficacy, Safety and Tolerability of AFQ056 in Fragile X Patients
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

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.

Not Provided
Interventional
Phase 2
Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Crossover Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Fragile X Syndrome
  • Drug: AF056
  • Drug: Placebo
  • Active Comparator: 1
    Intervention: Drug: AF056
  • Placebo Comparator: 2
    Intervention: Drug: Placebo
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.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
30
Not Provided
February 2009   (final data collection date for primary outcome measure)

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.

Male
18 Years to 35 Years
No
Contact information is only displayed when the study is recruiting subjects
France,   Italy,   Switzerland
 
NCT00718341
CAFQ056A2204
No
External Affairs, Novartis
Novartis
Not Provided
Principal Investigator: Novartis Novartis investigator site
Novartis
May 2010

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP