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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT01253629
Registration number
NCT01253629
Ethics application status
Date submitted
2/12/2010
Date registered
3/12/2010
Date last updated
23/12/2020
Titles & IDs
Public title
Safety and Efficacy of AFQ056 in Adult Patients With Fragile X Syndrome
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Scientific title
A Randomized, Double-blind, Placebo-controlled, Parallel Group Study to Evaluate AFQ056 in Adult Patients With Fragile X Syndrome
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Secondary ID [1]
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2009-013667-19
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Secondary ID [2]
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CAFQ056A2212
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Fragile X Syndrome
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Condition category
Condition code
Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Mental Health
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Learning disabilities
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Other
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Research that is not of generic health relevance and not applicable to specific health categories listed above
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - AFQ056
Treatment: Drugs - Placebo
Experimental: 25 mg bid AFQ056 - 1 capsule of 25 mg and 1 capsule of placebo per intake
Experimental: 50 mg bid AFQ056 - 2 capsules of 25 mg per intake
Experimental: 100 mg bid AFQ056 - 1 capsule of 100 mg and 1 capsule of placebo per intake
Placebo Comparator: Placebo - 2 capsules of placebo per intake
Treatment: Drugs: AFQ056
AFQ056, was provided as hard gelatin capsules, 25mg and 100 mg oral dosage strengths, identical in appearance were used
Treatment: Drugs: Placebo
Placebo medication identical in appearance to active medication was provided
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Change from baseline in behavioral symptoms of Fragile X Syndrome using the Aberrant Behavior Checklist - Community (ABC-C) Total score in Stratum I
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Assessment method [1]
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The ABC-C is a 58-item questionnaire that should have been completed as much as possible by the same rater. It is comprised of five subscales (irritability, lethargy/social withdrawal, stereotypic behavior, hyperactivity and inappropriate speech) plus the total score which ranks from 0 to 174 in patients who were fully methylated (FM)
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Timepoint [1]
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12 weeks
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Secondary outcome [1]
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Change from baseline in behavioral symptoms of Fragile X Syndrome (FXS) using the ABC-C Total score in Stratum II
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Assessment method [1]
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The ABC-C is a 58-item questionnaire that should have been completed as much as possible by the same rater. It is comprised of five subscales (irritability, lethargy/social withdrawal, stereotypic behavior, hyperactivity and inappropriate speech). Assessing the reduction in the (ABC-C) total score after 12 weeks of treatment in FXS patients with partially methylated (PM) FMR1 gene.
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Timepoint [1]
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12 weeks
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Secondary outcome [2]
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Global improvement of symptoms in Fragile X using the Clinical Global Impression-Improvement (CGI-I) scale
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Assessment method [2]
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The CGI-I score ranges from 1 to 7 (with 1 being "very much improved", 4 being "no change" to 7 being "very much worse")
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Timepoint [2]
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12 weeks
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Secondary outcome [3]
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Change from baseline in irritability, lethargy/social withdrawal, stereotypic behavior, hyperactivity, and inappropriate speech assessed by the individual subscales of the ABC-C scale
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Assessment method [3]
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comprised of five subscales (irritability, lethargy/social withdrawal, stereotypic behavior, hyperactivity and inappropriate speech) plus the total score which ranks from 0 to 174
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Timepoint [3]
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12 weeks
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Secondary outcome [4]
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The proportion of patients with clinical response in the ABC-C total score
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Assessment method [4]
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response is defined as reduction of at least 25% from baseline in the ABC-CFX total score and a score of 1 (very much improved) or 2 (much improved) on the CGI-I scale at Week 12
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Timepoint [4]
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12 weeks
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Secondary outcome [5]
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improvement of repetitive behavior as measured by changes in the RBS-R
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Assessment method [5]
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The Repetitive Behavior Scale - Revised (RBS-R) includes six domains: ritualistic behavior, sameness behavior, stereotypic behavior, self-injurious behavior, compulsive behavior, and restricted interests. A negative change from baseline indicates improvement
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Timepoint [5]
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Week 12
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Eligibility
Key inclusion criteria
- Patients with Fragile X Syndrome, who are at least moderately ill based on a Clinical
Global Impression Severity score of at least 4 and have qualifying scores on the ABC-C
and IQ test at Visit 1
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Minimum age
18
Years
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Maximum age
45
Years
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
- Advanced, severe or unstable disease that may interfere with the study outcome
evaluations
- Cancer within the past 5 years, other than localized skin cancer
- Current treatment with more than two psychoactive medications, excluding
anti-epileptics
- History of severe self-injurious behavior
Other protocol-defined inclusion/exclusion criteria may apply
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Blinded (masking used)
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Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 2
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Completed
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
1/11/2010
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
1/08/2013
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Sample size
Target
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Accrual to date
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Final
175
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Recruitment in Australia
Recruitment state(s)
NSW,VIC
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Recruitment hospital [1]
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Novartis Investigative Site - Ryde
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Recruitment hospital [2]
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Novartis Investigative Site - Waratah
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Recruitment hospital [3]
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Novartis Investigative Site - Caulfield
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Recruitment postcode(s) [1]
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2112 - Ryde
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Recruitment postcode(s) [2]
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2298 - Waratah
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Recruitment postcode(s) [3]
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3161 - Caulfield
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Recruitment outside Australia
Country [1]
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United States of America
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Arizona
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United States of America
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California
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United States of America
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Georgia
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United States of America
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Illinois
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Indiana
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Massachusetts
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Nebraska
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New York
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Pennsylvania
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South Carolina
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Tennessee
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United States of America
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Texas
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Canada
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Ontario
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Canada
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Quebec
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Denmark
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Glostrup
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France
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Bron Cedex
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France
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Paris
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Germany
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Berlin
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Germany
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Mainz
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Germany
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Tübingen
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Germany
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Würzburg
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Italy
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GE
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Italy
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RM
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Spain
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Andalucia
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Spain
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Cataluña
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Switzerland
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Lausanne
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Switzerland
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Zurich
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United Kingdom
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Edinburgh
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
Novartis Pharmaceuticals
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Address
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Country
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Ethics approval
Ethics application status
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Summary
Brief summary
This Phase IIb study is designed to assess whether 3 doses of AFQ056 are safe and effective
in treating the behavioral symptoms of Fragile X Syndrome.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT01253629
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Novartis Pharmaceuticals
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Address
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Novartis Pharmaceuticals
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Contact person for public queries
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT01253629
Download to PDF