Safety and Efficacy of AFQ056 in Adolescent Patients With Fragile X Syndrome

Novartis

Status and phase: Completed; Phase 2

Conditions: Fragile X Syndrome

Treatments: Drug: Placebo; Drug: AFQ056

Study type: Interventional

Funder types: Industry

Identifiers

NCT01357239

2010-022638-96 (EudraCT Number)

CAFQ056B2214

Details and patient eligibility

About

This Phase IIb study is designed to assess whether 3 doses of AFQ056 are safe and effective in treating the behavioral symptoms of Fragile X Syndrome.

Enrollment: 139 patients

Sex: All

Ages: 12 to 17 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Patients with Fragile X Syndrome, who are at least moderately ill based on a Clinical Global Impression Severity score of at least 4 and have qualifying scores on the ABC-C and IQ test at Visit 1

Exclusion criteria

• Advanced, severe or unstable disease that may interfere with the study outcome evaluations
• Cancer within the past 5 years, other than localized skin cancer
• Current treatment with more than two psychoactive medications, excluding anti-epileptics
• History of severe self-injurious behavior
• Weigh less than 32 kg
• Females who are sexually active

Other protocol-defined inclusion/exclusion criteria may apply

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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139 participants in 4 patient groups, including a placebo group

25 mg bid

Experimental group

Treatment:

Drug: AFQ056

50 mg bid

Experimental group

Treatment:

Drug: AFQ056

100 mg bid

Experimental group

Treatment:

Drug: AFQ056

Placebo

Placebo Comparator group

Treatment:

Drug: Placebo