Long-term, Safety and Tolerability Study of AFQ056 in Adolescent Patients With Fragile X Syndrome (Open-label)

NCT ID: NCT01433354

Last Updated: 2016-03-24

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE2/PHASE3
• Total Enrollment: 119 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2011-11-30
• Study Completion Date: 2014-09-30

Brief Summary

The purpose of this study is to generate long-term safety, tolerability and efficacy data for AFQ056 in eligible adolescent patients with FXS who have participated in the CAFQ056B2214 study, the PK study CAFQ056B2131, or another study of AFQ056 which included FXS patients below 18 years of age provided the patient is at least 12 years of age at the time of entry into the current study.

Conditions

Fragile X Syndrome

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

AFQ056 Treatment

All patients will initiate treatment with AFQ056 at a starting dose of 25 mg b.i.d. The dose will be titrated from 25 mg b.i.d to 50 mg b.i.d., 75 mg b.i.d. and 100 mg b.i.d. at weekly intervals. Dose adjustments (up- and down-titrations) will be permitted as needed to manage any tolerability issues and to ensure that patients reach their highest tolerated dose, not to exceed 100 mg b.i.d.

Group Type: EXPERIMENTAL

AFQ056

Intervention Type: DRUG

The investigational drug, AFQ056, will be provided as hard gelatin capsules. Two different oral dosage strengths,25mg and 100 mg, identical in appearance, will be used.

Interventions

AFQ056

The investigational drug, AFQ056, will be provided as hard gelatin capsules. Two different oral dosage strengths,25mg and 100 mg, identical in appearance, will be used.

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

• Group 1 patients:
• Must have completed study CAFQ056B2214 or another study of AFQ056 which included FXS patients below 18 years of age within one week of enrollment into the open-label study.
• Has a caregiver or caregivers who spend, 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:
• Completed Study CAFQ056B2131
• Completed Study CAFQ056B2214 or another study of AFQ056 which included FXS patients below 18 years of age but enrollment into the current study was delayed for more than a week.
• Discontinued prematurely from Study CAFQ056B2214 or another study of AFQ056 which included FXS patients below 18 years of age due to intolerability of the dosage in the patient's assigned treatment group.
• Has a caregiver or caregivers who spend, 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.

Exclusion Criteria

• Discontinuation from Study CAFQ056B2214 or CAFQ056B2131 or another study of AFQ056 which included FXS patients below 18 years of age 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 baseline) concomitant medications that are potent inhibitors or inducers of CYP3A4
• Using glutamatergic agents (riluzole, memantine, etc.) or lithium within 6 weeks of baseline

• Minimum Eligible Age: 12 Years
• Maximum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Novartis Pharmaceuticals

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Novartis Pharmaceuticals

Role: STUDY_DIRECTOR

Novartis Pharmaceuticals

Locations

Novartis Investigative Site

Sacramento, California, United States

Novartis Investigative Site

Decatur, Georgia, United States

Novartis Investigative Site

Chicago, Illinois, United States

Novartis Investigative Site

Boston, Massachusetts, United States

Novartis Investigative Site

Omaha, Nebraska, United States

Novartis Investigative Site

Staten Island, New York, United States

Novartis Investigative Site

Nashville, Tennessee, United States

Novartis Investigative Site

Westmead, New South Wales, Australia

Novartis Investigative Site

Parkville, Victoria, Australia

Novartis Investigative Site

Brussels, , Belgium

Novartis Investigative Site

Leuven, , Belgium

Novartis Investigative Site

Glostrup Municipality, , Denmark

Novartis Investigative Site

Bron, , France

Novartis Investigative Site

Mainz, Germany, Germany

Novartis Investigative Site

München, , Germany

Novartis Investigative Site

Tübingen, , Germany

Novartis Investigative Site

Würzburg, , Germany

Novartis Investigative Site

Ramat Gan, , Israel

Novartis Investigative Site

Genova, GE, Italy

Novartis Investigative Site

Padua, , Italy

Novartis Investigative Site

Rotterdam, , Netherlands

Novartis Investigative Site

Málaga, Andalusia, Spain

Novartis Investigative Site

Sabadell, Barcelona, Spain

Novartis Investigative Site

Sant Cugat del Vallès, Catalonia, Spain

Novartis Investigative Site

Spånga, , Sweden

Novartis Investigative Site

Lausanne, , Switzerland

Novartis Investigative Site

Zurich, , Switzerland

Novartis Investigative Site

Edinburgh, , United Kingdom

Countries

United States; Australia; Belgium; Denmark; France; Germany; Israel; Italy; Netherlands; Spain; Sweden; Switzerland; United Kingdom

References

Bailey DB Jr, Berry-Kravis E, Wheeler A, Raspa M, Merrien F, Ricart J, Koumaras B, Rosenkranz G, Tomlinson M, von Raison F, Apostol G. Mavoglurant in adolescents with fragile X syndrome: analysis of Clinical Global Impression-Improvement source data from a double-blind therapeutic study followed by an open-label, long-term extension study. J Neurodev Disord. 2016;8:1. doi: 10.1186/s11689-015-9134-5. Epub 2015 Dec 15.

Reference Type: DERIVED

PMID: 26855682 (View on PubMed)

Other Identifiers

2011-002379-40

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

CAFQ056B2278

Identifier Type: -

Identifier Source: org_study_id