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

Fragile X Syndrome Clinical Trial

Official title:

An Open-label Study to Evaluate the Long-term Safety and Tolerability of AFQ056 in Adolescent Patients With Fragile X Syndrome

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT01433354
• Other study ID #: CAFQ056B2278
• Secondary ID: 2011-002379-40
• Status: Terminated
• Phase: Phase 2/Phase 3
• First received: August 31, 2011
• Last updated: September 14, 2015
• Start date: November 2011
• Est. completion date: September 2014

Study information

• Verified date: September 2015
• Source: Novartis
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug AdministrationDenmark: Danish Medicines AgencyFrance: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)Germany: Federal Institute for Drugs and Medical DevicesItaly: Ethics CommitteeUnited Kingdom: Medicines and Healthcare Products Regulatory AgencyAustralia: National Health and Medical Research CouncilSwitzerland: SwissmedicSweden: Medical Products AgencyCanada: Health CanadaIsrael: Ministry of HealthNetherlands: Medicines Evaluation Board (MEB)Spain: Agencia Española de Medicamentos y Productos SanitariosTurkey: Ministry of HealthBelgium: Federal Agency for Medicinal Products and Health Products
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 119
• Est. completion date: September 2014
• Est. primary completion date: September 2014
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 12 Years to 18 Years

Eligibility

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

Other protocol-defined inclusion/exclusion criteria may apply.

Study Design

Allocation: Non-Randomized, Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Fragile X Syndrome
• Syndrome

Intervention

Drug:
• 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.

Locations

Country	Name	City	State
Australia	Novartis Investigative Site	Parkville	Victoria
Australia	Novartis Investigative Site	Westmead	New South Wales
Belgium	Novartis Investigative Site	Bruxelles
Belgium	Novartis Investigative Site	Leuven
Denmark	Novartis Investigative Site	Glostrup
France	Novartis Investigative Site	Bron Cedex
Germany	Novartis Investigative Site	Mainz
Germany	Novartis Investigative Site	München
Germany	Novartis Investigative Site	Tübingen
Germany	Novartis Investigative Site	Würzburg
Israel	Novartis Investigative Site	Ramat Gan
Italy	Novartis Investigative Site	Genova	GE
Italy	Novartis Investigative Site	Padova
Netherlands	Novartis Investigative Site	Rotterdam
Spain	Novartis Investigative Site	Málaga	Andalucia
Spain	Novartis Investigative Site	Sabadell	Barcelona
Spain	Novartis Investigative Site	Sant Cugat	Cataluña
Sweden	Novartis Investigative Site	Spånga
Switzerland	Novartis Investigative Site	Lausanne
Switzerland	Novartis Investigative Site	Zurich
United Kingdom	Novartis Investigative Site	Edinburgh
United States	Novartis Investigative Site	Boston	Massachusetts
United States	Novartis Investigative Site	Chicago	Illinois
United States	Novartis Investigative Site	Decatur	Georgia
United States	Novartis Investigative Site	Nashville	Tennessee
United States	Novartis Investigative Site	Omaha	Nebraska
United States	Novartis Investigative Site	Sacramento	California
United States	Novartis Investigative Site	Staten Island	New York

Sponsors (1)

Lead Sponsor	Collaborator
Novartis Pharmaceuticals

Countries where clinical trial is conducted

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

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Incidence and Severity of Adverse Events (AEs) and Serious Adverse Events (SAEs)	Adverse events were summarized for the open-label treatment period, where the open-label treatment period is defined based on how AEs were collected and reported according to the manner in which participants entered the current study and which treatment (AFQ056 or placebo) they were receiving in the previous study.; AEs which were continuing from the core study or that started after the end of core study but prior to first dose of open-label study medication in the extension study for Category 1 participants are shown under 'Prior to Ext. first dose'.; AEs which started during the open-label treatment period are presented based on the last AFQ056 dose taken on or before the onset date of the AE (25 mg bid; 50 mg bid; 75 mg bid; or 100 mg bid). No efficacy data presented as study was terminated.	Prior to first dose in extension study, Baseline (start of study treatment in extension study) to End of trial	Yes