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

Novartis

Status and phase: Completed; Phase 2

Conditions: Fragile X Syndrome

Treatments: Drug: AFQ056; Drug: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT01253629

2009-013667-19 (EudraCT Number)

CAFQ056A2212

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: 175 patients

Sex: All

Ages: 18 to 45 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

Other protocol-defined inclusion/exclusion criteria may apply

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

---

175 participants in 4 patient groups, including a placebo group

25 mg bid AFQ056

Experimental group

Description:

1 capsule of 25 mg and 1 capsule of placebo per intake

Treatment:

Drug: AFQ056

50 mg bid AFQ056

Experimental group

Description:

2 capsules of 25 mg per intake

Treatment:

Drug: AFQ056

100 mg bid AFQ056

Experimental group

Description:

1 capsule of 100 mg and 1 capsule of placebo per intake

Treatment:

Drug: AFQ056

Placebo

Placebo Comparator group

Description:

2 capsules of placebo per intake

Treatment:

Drug: Placebo