- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT01433354
Long-term, Safety and Tolerability Study of AFQ056 in Adolescent Patients With Fragile X Syndrome (Open-label)
An Open-label Study to Evaluate the Long-term Safety and Tolerability of AFQ056 in Adolescent Patients With Fragile X Syndrome
Study Overview
Study Type
Enrollment (Actual)
Phase
- Phase 2
- Phase 3
Contacts and Locations
Study Locations
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New South Wales
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Westmead, New South Wales, Australia, 2145
- Novartis Investigative Site
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Victoria
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Parkville, Victoria, Australia, 3052
- Novartis Investigative Site
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Bruxelles, Belgium, 1200
- Novartis Investigative Site
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Leuven, Belgium, 3000
- Novartis Investigative Site
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Glostrup, Denmark, 2600
- Novartis Investigative Site
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Bron Cedex, France, 69677
- Novartis Investigative Site
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Mainz, Germany, 55131
- Novartis Investigative Site
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München, Germany, 80639
- Novartis Investigative Site
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Tübingen, Germany, 72076
- Novartis Investigative Site
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Würzburg, Germany, 97070
- Novartis Investigative Site
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Ramat Gan, Israel, 52621
- Novartis Investigative Site
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Padova, Italy, 35128
- Novartis Investigative Site
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GE
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Genova, GE, Italy, 16147
- Novartis Investigative Site
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Rotterdam, Netherlands, 3015 CE
- Novartis Investigative Site
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Andalucia
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Málaga, Andalucia, Spain, 29009
- Novartis Investigative Site
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Barcelona
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Sabadell, Barcelona, Spain, 08208
- Novartis Investigative Site
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Cataluña
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Sant Cugat, Cataluña, Spain, 08190
- Novartis Investigative Site
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Spånga, Sweden, 16374
- Novartis Investigative Site
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Lausanne, Switzerland
- Novartis Investigative Site
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Zurich, Switzerland, 8091
- Novartis Investigative Site
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Edinburgh, United Kingdom, EH10 5HF
- Novartis Investigative Site
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California
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Sacramento, California, United States, 95817
- Novartis Investigative Site
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Georgia
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Decatur, Georgia, United States, 30033
- Novartis Investigative Site
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Illinois
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Chicago, Illinois, United States, 60612
- Novartis Investigative Site
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Massachusetts
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Boston, Massachusetts, United States, 02115
- Novartis Investigative Site
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Nebraska
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Omaha, Nebraska, United States, 68198-5575
- Novartis Investigative Site
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New York
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Staten Island, New York, United States, 10314
- Novartis Investigative Site
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Tennessee
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Nashville, Tennessee, United States, 37212
- Novartis Investigative Site
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
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 Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
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Experimental: 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.
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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.
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
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Incidence and Severity of Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time Frame: Prior to first dose in extension study, Baseline (start of study treatment in extension study) to End of trial
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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
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Collaborators and Investigators
Sponsor
Publications and helpful links
Study record dates
Study Major Dates
Study Start
Primary Completion (Actual)
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimate)
Study Record Updates
Last Update Posted (Estimate)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Pathologic Processes
- Nervous System Diseases
- Neurologic Manifestations
- Neurobehavioral Manifestations
- Disease
- Congenital Abnormalities
- Genetic Diseases, Inborn
- Genetic Diseases, X-Linked
- Mental Retardation, X-Linked
- Intellectual Disability
- Heredodegenerative Disorders, Nervous System
- Chromosome Disorders
- Sex Chromosome Disorders
- Syndrome
- Fragile X Syndrome
Other Study ID Numbers
- CAFQ056B2278
- 2011-002379-40 (EudraCT Number)
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Fragile X Syndrome
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University of California, DavisNational Institute of Mental Health (NIMH)CompletedFragile X PremutationUnited States
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University of California, DavisNational Institute on Aging (NIA); Forest LaboratoriesCompletedFragile X-Associated Tremor/Ataxia Syndrome | Fragile X Premutation CarriersUnited States
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Ovid Therapeutics Inc.CompletedFragile X Syndrome (FXS)United States
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Guido A. Davidzon, MD, SMWithdrawn
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Marinus PharmaceuticalsUniversity of California, Davis; U.S. Army Medical Research and Development...Completed
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RTI InternationalEunice Kennedy Shriver National Institute of Child Health and Human Development...CompletedFragile X Syndrome (FXS)United States
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Novartis PharmaceuticalsTerminated
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Sheba Medical CenterElMindA LtdRecruitingFragile X Associated Tremor-ataxia Syndrome | FXTASIsrael
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University of California, DavisUniversity of Alberta; St. Justine's HospitalRecruitingNeurobehavioral Manifestations | Genetic Diseases, X-Linked | Intellectual Disability | Fragile X Syndrome | Sex Chromosome Disorders | Fragile X Mental Retardation Syndrome | Trinucleotide Repeat Expansion | Fra(X) Syndrome | FXS | Mental Retardation, X LinkedUnited States, Canada
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University of AlbertaSt. Justine's HospitalRecruitingNeurobehavioral Manifestations | Genetic Diseases, X-Linked | Mental Retardation, X-Linked | Intellectual Disability | Fragile X Syndrome | Sex Chromosome Disorders | Fragile X Mental Retardation Syndrome | Trinucleotide Repeat Expansion | Fra(X) Syndrome | FXSCanada
Clinical Trials on AFQ056
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Novartis PharmaceuticalsTerminatedFragile X SyndromeGermany, United States, Switzerland, Australia, Canada, Denmark, France, Italy, United Kingdom, Spain
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Novartis PharmaceuticalsCompletedFragile X SyndromeUnited States, Switzerland, Germany, Australia, Canada, Denmark, France, Italy, Spain, United Kingdom
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Novartis PharmaceuticalsCompletedFragile X SyndromeUnited States, Spain
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Novartis PharmaceuticalsCompletedMild Moderate | or Severe Renal ImpairmentGermany
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Novartis PharmaceuticalsCompletedHepatic ImpairmentGermany, United States, Hungary
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Novartis PharmaceuticalsTerminatedEfficacy, Safety and Tolerability of AFQ056 in Patients With Huntington's Disease in Reducing ChoreaChorea | Huntington's DiseaseGermany, United Kingdom
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Novartis PharmaceuticalsCompletedFragile X SyndromeUnited States, Belgium, Australia, Israel, Switzerland, Germany, France, Canada, Denmark, Indonesia, Italy, Netherlands, Spain, Sweden, Turkey, United Kingdom
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Elizabeth Berry-KravisNational Institute of Neurological Disorders and Stroke (NINDS); Novartis PharmaceuticalsCompleted
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Yale UniversityCompletedFamilial Alcoholism VulnerabilityUnited States
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Novartis PharmaceuticalsTerminatedPatient Diagnosed With OCD and Resistant to SSRI Treatment | Failed SSRI Over 12 Weeks at Appropriate DosesGermany, Switzerland, Bulgaria, United States, Czech Republic