Efficacy, Safety and Tolerability of AFQ056 in Fragile X Patients

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT00718341
• Date of registration: 17/07/2008
• Prospective Registration: No
• Primary sponsor: Novartis
• Public title: Efficacy, Safety and Tolerability of AFQ056 in Fragile X Patients
• Scientific 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
• Date of first enrolment: June 2008
• Target sample size: 30
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT00718341
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Crossover Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 2

Countries of recruitment

France, Italy, Switzerland

Contacts

• Name: Novartis
• Affiliation: Novartis investigator site

Key inclusion & exclusion 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.



Age minimum: 18 Years
Age maximum: 35 Years
Gender: Male

Health Condition(s) or Problem(s) studied

Fragile X Syndrome

Intervention(s)

Drug: AF056

Drug: Placebo

Primary Outcome(s)

Aberrant-Behavior Checklist- Community Edition

Secondary Outcome(s)

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…)

Secondary ID(s)

CAFQ056A2204

Source(s) of Monetary Support

Please refer to primary and secondary sponsors