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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT01357239




Registration number
NCT01357239
Ethics application status
Date submitted
18/05/2011
Date registered
20/05/2011
Date last updated
12/05/2015

Titles & IDs
Public title
Safety and Efficacy of AFQ056 in Adolescent Patients With Fragile X Syndrome
Scientific title
A Randomized, Double-blind, Placebo-controlled, Parallel Group Study to Evaluate the Efficacy and Safety of AFQ056 in Adolescent Patients With Fragile X Syndrome
Secondary ID [1] 0 0
2010-022638-96
Secondary ID [2] 0 0
CAFQ056B2214
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
Experimental: 25 mg bid -

Experimental: 50 mg bid -

Experimental: 100 mg bid -

Placebo comparator: Placebo -

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 Edition (ABC-CFX) Total Score in Stratum I Patients Exposed to AFQ056 100 mg Bid
Timepoint [1] 0 0
Baseline to week 12
Secondary outcome [1] 0 0
Change From Baseline in Behavioral Symptoms of Fragile X Syndrome Using the ABC-CFX Total Score in Stratum II Patients Exposed to All 3 Doses of AFQ056
Timepoint [1] 0 0
Baseline to week 12
Secondary outcome [2] 0 0
Change From Baseline in Behavioral Symptoms of Fragile X Syndrome Using the ABC-CFX Total Score in Stratum I Patients Exposed to the Two Lower Doses of AFQ056 (25 mg Bid and 50 mg Bid)
Timepoint [2] 0 0
Baseline to week 12
Secondary outcome [3] 0 0
Global Improvement of Symptoms in Fragile X Using the Clinical Global Impression- Improvement (CGI-I) Scale in Stratum I Patients
Timepoint [3] 0 0
12 weeks
Secondary outcome [4] 0 0
Global Improvement of Symptoms in Fragile X Using the Clinical Global Impression- Improvement (CGI-I) Scale in Stratum II Patients
Timepoint [4] 0 0
12 weeks
Secondary outcome [5] 0 0
Global Improvement of Symptoms in Fragile X Using the Clinical Global Impression-Improvement (CGI-I) Scale in Stratum I
Timepoint [5] 0 0
12 weeks
Secondary outcome [6] 0 0
Global Improvement of Symptoms in Fragile X Using the Clinical Global Impression-Improvement (CGI-I) Scale in Stratum II
Timepoint [6] 0 0
12 weeks
Secondary outcome [7] 0 0
Change From Baseline in Irritability, Lethargy/Withdrawal, Stereotypic Behavior, Hyperactivity, Inappropriate Speech and Social Avoidance Assessed by the Individual Subscales of the ABC-CFX Scale in Stratum I
Timepoint [7] 0 0
Baseline to week 12
Secondary outcome [8] 0 0
Change From Baseline in Irritability, Lethargy/Withdrawal, Stereotypic Behavior, Hyperactivity, Inappropriate Speech, and Social Avoidance Assessed by the Individual Subscales of the ABC-CFX Scale in Stratum II
Timepoint [8] 0 0
Baseline to week 12
Secondary outcome [9] 0 0
Proportion of Patients With Clinical Response, Where Response is Defined as a 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, Stratum I
Timepoint [9] 0 0
12 weeks
Secondary outcome [10] 0 0
Proportion of Patients With Clinical Response, Where Response is Defined as a 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, Stratum II
Timepoint [10] 0 0
12 weeks
Secondary outcome [11] 0 0
Change From Baseline in Repetitive Behaviors Assessed Using the Repetitive Behavior Scale - Revised (RBS-R) Total and Subscale Scores in Stratum I
Timepoint [11] 0 0
Baseline to week 12
Secondary outcome [12] 0 0
Change From Baseline in Repetitive Behaviors Assessed Using the Repetitive Behavior Scale - Revised (RBS-R) Total and Subscale Scores in Stratum II
Timepoint [12] 0 0
Baseline to 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
12 Years
Maximum age
17 Years
Sex
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
* Weigh less than 32 kg
* Females who are sexually active

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
1/05/2011
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
1/01/2014
Sample size
Target
Accrual to date
Final
139
Recruitment in Australia
Recruitment state(s)
NSW,VIC
Recruitment hospital [1] 0 0
Novartis Investigative Site - Westmead
Recruitment hospital [2] 0 0
Novartis Investigative Site - Parkville
Recruitment postcode(s) [1] 0 0
2145 - Westmead
Recruitment postcode(s) [2] 0 0
3052 - Parkville
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Georgia
Country [3] 0 0
United States of America
State/province [3] 0 0
Illinois
Country [4] 0 0
United States of America
State/province [4] 0 0
Massachusetts
Country [5] 0 0
United States of America
State/province [5] 0 0
Nebraska
Country [6] 0 0
United States of America
State/province [6] 0 0
New York
Country [7] 0 0
United States of America
State/province [7] 0 0
Tennessee
Country [8] 0 0
United States of America
State/province [8] 0 0
Texas
Country [9] 0 0
Belgium
State/province [9] 0 0
Bruxelles
Country [10] 0 0
Belgium
State/province [10] 0 0
Leuven
Country [11] 0 0
Canada
State/province [11] 0 0
Alberta
Country [12] 0 0
Canada
State/province [12] 0 0
British Columbia
Country [13] 0 0
Canada
State/province [13] 0 0
Quebec
Country [14] 0 0
Denmark
State/province [14] 0 0
Glostrup
Country [15] 0 0
France
State/province [15] 0 0
Bron
Country [16] 0 0
France
State/province [16] 0 0
Paris
Country [17] 0 0
Germany
State/province [17] 0 0
Dresden
Country [18] 0 0
Germany
State/province [18] 0 0
Mainz
Country [19] 0 0
Germany
State/province [19] 0 0
München
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
Indonesia
State/province [22] 0 0
Jawa Tengah
Country [23] 0 0
Israel
State/province [23] 0 0
Ramat Gan
Country [24] 0 0
Italy
State/province [24] 0 0
GE
Country [25] 0 0
Italy
State/province [25] 0 0
RM
Country [26] 0 0
Italy
State/province [26] 0 0
Padova
Country [27] 0 0
Netherlands
State/province [27] 0 0
Rotterdam
Country [28] 0 0
Spain
State/province [28] 0 0
Andalucia
Country [29] 0 0
Spain
State/province [29] 0 0
Barcelona
Country [30] 0 0
Spain
State/province [30] 0 0
Cataluña
Country [31] 0 0
Sweden
State/province [31] 0 0
Spånga
Country [32] 0 0
Switzerland
State/province [32] 0 0
Lausanne
Country [33] 0 0
Switzerland
State/province [33] 0 0
Zurich
Country [34] 0 0
Turkey
State/province [34] 0 0
Istanbul
Country [35] 0 0
United Kingdom
State/province [35] 0 0
Edinburgh

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

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
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

No information has been provided regarding IPD availability


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.

Results are available at https://clinicaltrials.gov/study/NCT01357239