ong-term, safety and tolerability study of AFQ056 in adolescent patients with Fragile X Syndrome (Open–label)

Phase 1

• Conditions: Fragile X syndrome; MedDRA version: 15.0Level: PTClassification code 10017324Term: Fragile X syndromeSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

• Registration Number: EUCTR2011-002379-40-FR
• Lead Sponsor: ovartis Pharma Services AG

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2012-08-24
• Last Update Posted: 12 September 2016

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 180

Inclusion Criteria

Group 1 patients:
• Must have completed the CAFQ056B2214 core study within one week of enrollment into the open-label study
• Has a caregiver who spends, on average, at least 6 hours per day with the patient , who is willing to and capable of supervising treatment, providing input into efficacy and safety assessments, and accompanying the patient to study visits.

Group 2 patients:
• Must meet one of the following conditions:
o completed Study CAFQ056B2131
o completed Study CAFQ056B2214 but enrollment into the current study was delayed for more than a week
o discontinued prematurely from Study CAFQ056B2214 due to intolerability of the dosage in the patient’s assigned treatment group

• Has a caregiver who spends, on average, at least 6 hours per day with the patient , who is willing to and capable of supervising treatment, providing input into efficacy and safety assessments, and accompanying the patient to study visits

Other protocol-defined inclusion criteria may apply
Are the trial subjects under 18? yes
Number of subjects for this age range: 160
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 20
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

• Discontinuation from CAFQ056B2214 or CAFQ056B2131 studies due to safety reasons
• Female patients who are sexually active at any time during the study
• Any advanced, severe or unstable disease
• History and/or presence of schizophrenia, bipolar disease, psychosis, confusional states and/or repeated hallucinations as per DSM-IV criteria
• History of suicidal behavior or considered a high suicidal risk
• History of severe self-injurious behavior
• History of uncontrolled seizure disorder or resistant to therapy within the past 2 years (Patients who are clinically stable under anti-convulsant therapy for the past 2 years are not excluded)
• History of clinically significant allergies requiring hospitalization or non-inhaled corticosteroid therapy (asthma, anaphylaxis, etc.)
• History of malignancy of any organ system (other than localized basal cell carcinoma of the skin), treated or untreated, within the past 5 years, regardless of whether or not there is evidence of local recurrence or metastases
• patients who are using (or used within 6 weeks before baseline) digoxin or warfarin
• Using (or used within 6 weeks before randomization) concomitant medications that are potent inhibitors or inducers of CYP3A4
• Using glutamatergic agents (riluzole, memantine, etc.) or lithium within 6 weeks of baseline
• patients who weigh less than 32 kg (= 10th percentile of body weight for a 12-year old child)

Other protocol-defined exclusion criteria may apply

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified