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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
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/study/NCT01253629
Trial related presentations / publications
Berry-Kravis E, Des Portes V, Hagerman R, Jacquemont S, Charles P, Visootsak J, Brinkman M, Rerat K, Koumaras B, Zhu L, Barth GM, Jaecklin T, Apostol G, von Raison F. Mavoglurant in fragile X syndrome: Results of two randomized, double-blind, placebo-controlled trials. Sci Transl Med. 2016 Jan 13;8(321):321ra5. doi: 10.1126/scitranslmed.aab4109.
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

For IPD and results data, please see https://clinicaltrials.gov/study/NCT01253629