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

Status

Completed

Phases

Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT00718341

Enrollment

Registered

2008-07-18

Start date

2008-06-30

Completion date

Unknown

Last updated

2020-02-11

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Fragile X Syndrome

Keywords

Fragile X Syndrome, adults, efficacy

Brief summary

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.

Interventions

DRUGAF056

DRUGPlacebo

Study design

Allocation

RANDOMIZED

Intervention model

CROSSOVER

Primary purpose

TREATMENT

Masking

QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)

Eligibility

Sex/Gender

MALE

Age

18 Years to 35 Years

Healthy volunteers

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/

Design outcomes

Primary

Measure	Time frame
Aberrant-Behavior Checklist- Community Edition	—

Secondary

Measure	Time frame
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…)	—

Countries

France, Italy, Switzerland

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026