Efficacy, Safety and Tolerability of AFQ056 in Fragile X Patients
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ClinicalTrials.gov Identifier: NCT00718341 |
Recruitment Status :
Completed
First Posted : July 18, 2008
Last Update Posted : February 11, 2020
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Fragile X Syndrome | Drug: AF056 Drug: Placebo | Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 30 participants |
Allocation: | Randomized |
Intervention Model: | Crossover Assignment |
Masking: | Quadruple (Participant, Care Provider, 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 |
Study Start Date : | June 2008 |
Actual Primary Completion Date : | February 2009 |

Arm | Intervention/treatment |
---|---|
Active Comparator: 1 |
Drug: AF056 |
Placebo Comparator: 2 |
Drug: Placebo |
- Aberrant-Behavior Checklist- Community Edition
- 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…)

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Ages Eligible for Study: | 18 Years to 35 Years (Adult) |
Sexes Eligible for Study: | Male |
Accepts Healthy Volunteers: | No |
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.

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00718341
France | |
Novartis Investigator Site | |
Bron cedex, France, 69677 | |
Italy | |
Novartis Investigator Site | |
Rome, Italy, 00168 | |
Switzerland | |
Novartis Investigator Site | |
Lausanne, Switzerland, 1011 |
Principal Investigator: | Novartis | Novartis investigator site |
Responsible Party: | External Affairs, Novartis |
ClinicalTrials.gov Identifier: | NCT00718341 |
Other Study ID Numbers: |
CAFQ056A2204 |
First Posted: | July 18, 2008 Key Record Dates |
Last Update Posted: | February 11, 2020 |
Last Verified: | May 2010 |
Fragile X Syndrome, adults, efficacy |
Fragile X Syndrome Syndrome Disease Pathologic Processes Mental Retardation, X-Linked Intellectual Disability 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 |