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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/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
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
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
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
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
Timepoint [3] 0 0
12 weeks
Secondary outcome [4] 0 0
The proportion of patients with clinical response in the ABC-C total score
Timepoint [4] 0 0
12 weeks
Secondary outcome [5] 0 0
improvement of repetitive behavior as measured by changes in the RBS-R
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
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

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
1/11/2010
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
1/08/2013
Sample size
Target
Accrual to date
Final
175
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
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 not provided in https://clinicaltrials.gov/study/NCT01253629