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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT01253629




Registration number
NCT01253629
Ethics application status
Date submitted
2/12/2010
Date registered
3/12/2010
Date last updated
12/02/2020

Titles & IDs
Public title
Safety and Efficacy of AFQ056 in Adult Patients With Fragile X Syndrome
Scientific title
A Randomized, Double-blind, Placebo-controlled, Parallel Group Study to Evaluate AFQ056 in Adult Patients With Fragile X Syndrome
Secondary ID [1] 0 0
2009-013667-19
Secondary ID [2] 0 0
CAFQ056A2212
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Fragile X Syndrome 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Mental Health 0 0 0 0
Learning disabilities
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above

Intervention/exposure
Study type
Interventional
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

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Change from baseline in behavioral symptoms of Fragile X Syndrome using the Aberrant Behavior Checklist - Community (ABC-C) Total score in Stratum I - 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)
Timepoint [1] 0 0
12 weeks
Secondary outcome [1] 0 0
Change from baseline in behavioral symptoms of Fragile X Syndrome (FXS) using the ABC-C Total score in Stratum II - 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.
Timepoint [1] 0 0
12 weeks
Secondary outcome [2] 0 0
Global improvement of symptoms in Fragile X using the Clinical Global Impression-Improvement (CGI-I) scale - 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")
Timepoint [2] 0 0
12 weeks
Secondary outcome [3] 0 0
Change from baseline in irritability, lethargy/social withdrawal, stereotypic behavior, hyperactivity, and inappropriate speech assessed by the individual subscales of the ABC-C scale - comprised of five subscales (irritability, lethargy/social withdrawal, stereotypic behavior, hyperactivity and inappropriate speech) plus the total score which ranks from 0 to 174
Timepoint [3] 0 0
12 weeks
Secondary outcome [4] 0 0
The proportion of patients with clinical response in the ABC-C total score - 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
Timepoint [4] 0 0
12 weeks
Secondary outcome [5] 0 0
improvement of repetitive behavior as measured by changes in the RBS-R - 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
Timepoint [5] 0 0
Week 12

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
Minimum age
18 Years
Maximum age
45 Years
Gender
Both males and females
Can healthy volunteers participate?
No
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

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
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
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,VIC
Recruitment hospital [1] 0 0
Novartis Investigative Site - Ryde
Recruitment hospital [2] 0 0
Novartis Investigative Site - Waratah
Recruitment hospital [3] 0 0
Novartis Investigative Site - Caulfield
Recruitment postcode(s) [1] 0 0
2112 - Ryde
Recruitment postcode(s) [2] 0 0
2298 - Waratah
Recruitment postcode(s) [3] 0 0
3161 - Caulfield
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Georgia
Country [4] 0 0
United States of America
State/province [4] 0 0
Illinois
Country [5] 0 0
United States of America
State/province [5] 0 0
Indiana
Country [6] 0 0
United States of America
State/province [6] 0 0
Massachusetts
Country [7] 0 0
United States of America
State/province [7] 0 0
Nebraska
Country [8] 0 0
United States of America
State/province [8] 0 0
New York
Country [9] 0 0
United States of America
State/province [9] 0 0
Pennsylvania
Country [10] 0 0
United States of America
State/province [10] 0 0
South Carolina
Country [11] 0 0
United States of America
State/province [11] 0 0
Tennessee
Country [12] 0 0
United States of America
State/province [12] 0 0
Texas
Country [13] 0 0
Canada
State/province [13] 0 0
Ontario
Country [14] 0 0
Canada
State/province [14] 0 0
Quebec
Country [15] 0 0
Denmark
State/province [15] 0 0
Glostrup
Country [16] 0 0
France
State/province [16] 0 0
Bron Cedex
Country [17] 0 0
France
State/province [17] 0 0
Paris
Country [18] 0 0
Germany
State/province [18] 0 0
Berlin
Country [19] 0 0
Germany
State/province [19] 0 0
Mainz
Country [20] 0 0
Germany
State/province [20] 0 0
Tübingen
Country [21] 0 0
Germany
State/province [21] 0 0
Würzburg
Country [22] 0 0
Italy
State/province [22] 0 0
GE
Country [23] 0 0
Italy
State/province [23] 0 0
RM
Country [24] 0 0
Spain
State/province [24] 0 0
Andalucia
Country [25] 0 0
Spain
State/province [25] 0 0
Cataluña
Country [26] 0 0
Switzerland
State/province [26] 0 0
Lausanne
Country [27] 0 0
Switzerland
State/province [27] 0 0
Zurich
Country [28] 0 0
United Kingdom
State/province [28] 0 0
Edinburgh

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Novartis Pharmaceuticals
Address
Country

Ethics approval
Ethics application status

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.
Trial website
https://clinicaltrials.gov/show/NCT01253629
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Novartis Pharmaceuticals
Address 0 0
Novartis Pharmaceuticals
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Summary results
Other publications