Efficacy, Safety and Tolerability of AFQ056 in Fragile X Patients

• Sponsor: Novartis (Industry)
• Overall Status: Completed
• CT.gov ID: NCT00718341
• Collaborator: (none)
• Enrollment: 30
• Locations: 3
• Arms: 2
• Patients Per Site: 10

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Fragile X Syndrome	Drug: AF056; Drug: Placebo	Phase 2

Study Design

• Study Type: Interventional
• 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: Jun 1, 2008
• Actual Primary Completion Date: Feb 1, 2009

Arms and Interventions

Arm	Intervention/Treatment
Active Comparator: 1	Drug: AF056
Placebo Comparator: 2	Drug: Placebo

Outcome Measures

Primary Outcome Measures

1. Aberrant-Behavior Checklist- Community Edition []

Secondary Outcome Measures

1. 28 days treatment with AFQ056 on behavior (communication, socialization, daily living, repetitive behaviors, anxiety/avoidance, clinical global improvement) []

2. 28 days treatment with AFQ056 on cognition (receptive language, attention, vigilance…) []

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years to 35 Years
• 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.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Novartis Investigator Site	Bron cedex		France	69677
2	Novartis Investigator Site	Rome		Italy	00168
3	Novartis Investigator Site	Lausanne		Switzerland	1011

Sponsors and Collaborators

• Novartis

Investigators

• Principal Investigator: Novartis, Novartis investigator site

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• ClinicalTrials.gov Identifier: NCT00718341
• Other Study ID Numbers: CAFQ056A2204
• First Posted: Jul 18, 2008
• Last Update Posted: Feb 11, 2020
• Last Verified: May 1, 2010

• Keywords provided by , ,: Fragile X Syndrome, adults, efficacy
• Additional relevant MeSH terms: Fragile X Syndrome; Syndrome

Study Results

No Results Posted as of Feb 11, 2020