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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


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
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