Back to resultsSafety and Efficacy of AFQ056 in Adult Patients With Fragile X Syndrome
Primary Purpose
Fragile X Syndrome
Status
Completed
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
AFQ056
Placebo
Sponsored by
About this trial
This is an interventional treatment trial for Fragile X Syndrome focused on measuring Fragile X Syndrome, Martin-Bell Syndrome, Genetic Diseases, X-Linked
Eligibility Criteria
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
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
Sites / Locations
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm 4
Arm Type
Experimental
Experimental
Experimental
Placebo Comparator
Arm Label
25 mg bid AFQ056
50 mg bid AFQ056
100 mg bid AFQ056
Placebo
Arm Description
1 capsule of 25 mg and 1 capsule of placebo per intake
2 capsules of 25 mg per intake
1 capsule of 100 mg and 1 capsule of placebo per intake
2 capsules of placebo per intake
Outcomes
Primary Outcome Measures
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)
Secondary Outcome Measures
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.
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")
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
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
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
Full Information
NCT ID
NCT01253629
First Posted
December 2, 2010
Last Updated
December 15, 2020
Sponsor
Novartis Pharmaceuticals
1. Study Identification
Unique Protocol Identification Number
NCT01253629
Brief Title
Safety and Efficacy of AFQ056 in Adult Patients With Fragile X Syndrome
Official Title
A Randomized, Double-blind, Placebo-controlled, Parallel Group Study to Evaluate AFQ056 in Adult Patients With Fragile X Syndrome
Study Type
Interventional
2. Study Status
Record Verification Date
March 2016
Overall Recruitment Status
Completed
Study Start Date
November 2010 (undefined)
Primary Completion Date
August 2013 (Actual)
Study Completion Date
August 2013 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Novartis Pharmaceuticals
4. Oversight
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Fragile X Syndrome
Keywords
Fragile X Syndrome, Martin-Bell Syndrome, Genetic Diseases, X-Linked
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
175 (Actual)
8. Arms, Groups, and Interventions
Arm Title
25 mg bid AFQ056
Arm Type
Experimental
Arm Description
1 capsule of 25 mg and 1 capsule of placebo per intake
Arm Title
50 mg bid AFQ056
Arm Type
Experimental
Arm Description
2 capsules of 25 mg per intake
Arm Title
100 mg bid AFQ056
Arm Type
Experimental
Arm Description
1 capsule of 100 mg and 1 capsule of placebo per intake
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
2 capsules of placebo per intake
Intervention Type
Drug
Intervention Name(s)
AFQ056
Intervention Description
AFQ056, was provided as hard gelatin capsules, 25mg and 100 mg oral dosage strengths, identical in appearance were used
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Placebo medication identical in appearance to active medication was provided
Primary Outcome Measure Information:
Title
Change from baseline in behavioral symptoms of Fragile X Syndrome using the Aberrant Behavior Checklist - Community (ABC-C) Total score in Stratum I
Description
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)
Time Frame
12 weeks
Secondary Outcome Measure Information:
Title
Change from baseline in behavioral symptoms of Fragile X Syndrome (FXS) using the ABC-C Total score in Stratum II
Description
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.
Time Frame
12 weeks
Title
Global improvement of symptoms in Fragile X using the Clinical Global Impression-Improvement (CGI-I) scale
Description
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")
Time Frame
12 weeks
Title
Change from baseline in irritability, lethargy/social withdrawal, stereotypic behavior, hyperactivity, and inappropriate speech assessed by the individual subscales of the ABC-C scale
Description
comprised of five subscales (irritability, lethargy/social withdrawal, stereotypic behavior, hyperactivity and inappropriate speech) plus the total score which ranks from 0 to 174
Time Frame
12 weeks
Title
The proportion of patients with clinical response in the ABC-C total score
Description
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
Time Frame
12 weeks
Title
improvement of repetitive behavior as measured by changes in the RBS-R
Description
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
Time Frame
Week 12
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
45 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Novartis Pharmaceuticals
Organizational Affiliation
Novartis Pharmaceuticals
Official's Role
Study Director
Facility Information:
Facility Name
Novartis Investigative Site
City
Phoenix
State/Province
Arizona
ZIP/Postal Code
85006
Country
United States
Facility Name
Novartis Investigative Site
City
Sacramento
State/Province
California
ZIP/Postal Code
95817
Country
United States
Facility Name
Novartis Investigative Site
City
Decatur
State/Province
Georgia
ZIP/Postal Code
30033
Country
United States
Facility Name
Novartis Investigative Site
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60612
Country
United States
Facility Name
Novartis Investigative Site
City
Indianapolis
State/Province
Indiana
ZIP/Postal Code
46202
Country
United States
Facility Name
Novartis Investigative Site
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02115
Country
United States
Facility Name
Novartis Investigative Site
City
Omaha
State/Province
Nebraska
ZIP/Postal Code
68198-5575
Country
United States
Facility Name
Novartis Investigative Site
City
Staten Island
State/Province
New York
ZIP/Postal Code
10314
Country
United States
Facility Name
Novartis Investigative Site
City
Media
State/Province
Pennsylvania
ZIP/Postal Code
19063
Country
United States
Facility Name
Novartis Investigative Site
City
Greenwood
State/Province
South Carolina
ZIP/Postal Code
29646
Country
United States
Facility Name
Novartis Investigative Site
City
Nashville
State/Province
Tennessee
ZIP/Postal Code
37212
Country
United States
Facility Name
Novartis Investigative Site
City
Houston
State/Province
Texas
ZIP/Postal Code
77090
Country
United States
Facility Name
Novartis Investigative Site
City
Ryde
State/Province
New South Wales
ZIP/Postal Code
2112
Country
Australia
Facility Name
Novartis Investigative Site
City
Waratah
State/Province
New South Wales
ZIP/Postal Code
2298
Country
Australia
Facility Name
Novartis Investigative Site
City
Caulfield
State/Province
Victoria
ZIP/Postal Code
3161
Country
Australia
Facility Name
Novartis Investigative Site
City
Brampton
State/Province
Ontario
ZIP/Postal Code
L6Y 1M5
Country
Canada
Facility Name
Novartis Investigative Site
City
Sherbrooke
State/Province
Quebec
ZIP/Postal Code
J1H 5N4
Country
Canada
Facility Name
Novartis Investigative Site
City
Glostrup
ZIP/Postal Code
2600
Country
Denmark
Facility Name
Novartis Investigative Site
City
Bron Cedex
ZIP/Postal Code
69677
Country
France
Facility Name
Novartis Investigative Site
City
Paris
ZIP/Postal Code
75013
Country
France
Facility Name
Novartis Investigative Site
City
Berlin
ZIP/Postal Code
12200
Country
Germany
Facility Name
Novartis Investigative Site
City
Mainz
ZIP/Postal Code
55131
Country
Germany
Facility Name
Novartis Investigative Site
City
Tübingen
ZIP/Postal Code
72076
Country
Germany
Facility Name
Novartis Investigative Site
City
Würzburg
ZIP/Postal Code
97070
Country
Germany
Facility Name
Novartis Investigative Site
City
Genova
State/Province
GE
ZIP/Postal Code
16147
Country
Italy
Facility Name
Novartis Investigative Site
City
Roma
State/Province
RM
ZIP/Postal Code
00168
Country
Italy
Facility Name
Novartis Investigative Site
City
Málaga
State/Province
Andalucia
ZIP/Postal Code
29009
Country
Spain
Facility Name
Novartis Investigative Site
City
Sant Cugat
State/Province
Cataluña
ZIP/Postal Code
08190
Country
Spain
Facility Name
Novartis Investigative Site
City
Lausanne
Country
Switzerland
Facility Name
Novartis Investigative Site
City
Zurich
ZIP/Postal Code
8091
Country
Switzerland
Facility Name
Novartis Investigative Site
City
Edinburgh
ZIP/Postal Code
EH10 5HF
Country
United Kingdom
12. IPD Sharing Statement
Citations:
PubMed Identifier
26764156
Citation
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.
Results Reference
derived
Links:
URL
https://www.novctrd.com/ctrdweb/trialresult/trialresults/pdf?trialResultId=12423
Description
Results for CAFQ056A2212 from the Novartis Clinical Trial website
Learn more about this trial
Safety and Efficacy of AFQ056 in Adult Patients With Fragile X Syndrome
We'll reach out to this number within 24 hrs