Clinical Study to Assess the Pharmacokinetics, Safety and Tolerability of Single and Multiple Oral Doses of AFQ056 in Children With Fragile X Syndrome (FXS)
Primary Purpose
Fragile X Syndrome
Status
Completed
Phase
Phase 1
Locations
International
Study Type
Interventional
Intervention
AFQ056
Sponsored by
About this trial
This is an interventional other trial for Fragile X Syndrome focused on measuring Fragile X Syndrome, pharmacokinetics, safety and tolerability
Eligibility Criteria
Inclusion Criteria:
- Genetically confirmed diagnosis of FXS
- At Screening and first baseline, vital signs, body weight and body mass index (BMI) must be age-specific within normal ranges.
Exclusion Criteria:
- Use of any other investigational drug within 30 days or 5 half-lives (whichever is longer) of the investigational drug prior to screening until end of study visit.
- History of hypersensitivity to AFQ056 or any mGluR antagonist.
- Female patients who are confirmed or suspected to be sexually active.
- History or presence of any clinically significant disease of any major system organ class, within the past 2 years prior to screening including but not limited to psychiatric, neurological, cardiovascular, endocrine, metabolic, renal, or gastrointestinal disorders (except for typical features of FXS).
- Smokers.
- Loss of ≥10% of total blood volume within 8 weeks (or less if required for this age group and/or by local regulation) prior to dosing or longer if required for this age group and/or by local regulation.
- Significant illness that did not completely resolve at least four weeks prior to the first baseline visit.
- Any abnormal laboratory values at screening or first baseline that are in the opinion of the investigator clinically significant and may jeopardize the safety of the study subject.
- Use of (or use within at least 5 half lives before dosing) concomitant medications that are strong/moderate inhibitors or inducers of CYP1A1/2, CYP2C9/19 or CYP3A4
- History or presence of Hepatitis B/C or HIV at screening
Sites / Locations
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
- Novartis Investigative Site
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
All Study subjects
Arm Description
Outcomes
Primary Outcome Measures
The area under the plasma (or serum or blood) concentration-time curve from time zero to infinity [mass x time / volume] (AUCinf)
The area under the plasma (or serum or blood) concentration-time curve from time zero to the time of the last quantifiable concentration [mass x time / volume] (AUClast)
Maximum observed plasma concentration (Cmax)
Secondary Outcome Measures
Physical examination
Vital signs and body measurements
Electrocardiograms
hematology
blood chemistry
neurological examination
Adverse events (AE) monitoring
Serious adverse events (SAE) monitoring
Full Information
NCT ID
NCT01482143
First Posted
November 21, 2011
Last Updated
December 6, 2020
Sponsor
Novartis Pharmaceuticals
1. Study Identification
Unique Protocol Identification Number
NCT01482143
Brief Title
Clinical Study to Assess the Pharmacokinetics, Safety and Tolerability of Single and Multiple Oral Doses of AFQ056 in Children With Fragile X Syndrome (FXS)
Official Title
Sequential, Two-period Study to Assess the Pharmacokinetics, Safety & Tolerability of Single and Multiple Oral Doses of AFQ056 in Patients With FXS (Fragile X Syndrome) Aged 5-11 Years (Cohort 1) and 3-4 Years (Cohort 2)
Study Type
Interventional
2. Study Status
Record Verification Date
September 2014
Overall Recruitment Status
Completed
Study Start Date
March 2012 (undefined)
Primary Completion Date
October 2013 (Actual)
Study Completion Date
October 2013 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Novartis Pharmaceuticals
4. Oversight
5. Study Description
Brief Summary
The aim of this study is to characterize the pharmacokinetics and safety/tolerability of AFQ056 in children with Fragile X Syndrome(FXS)
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, pharmacokinetics, safety and tolerability
7. Study Design
Primary Purpose
Other
Study Phase
Phase 1
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
21 (Actual)
8. Arms, Groups, and Interventions
Arm Title
All Study subjects
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
AFQ056
Primary Outcome Measure Information:
Title
The area under the plasma (or serum or blood) concentration-time curve from time zero to infinity [mass x time / volume] (AUCinf)
Time Frame
Time Frame: Day 1 (period 1): 0.5, 2, 4, 8, 12, 24 hours post-dose; Day 7 (period 2): pre-dose; 0.5, 2, 4, 8 hours post dose
Title
The area under the plasma (or serum or blood) concentration-time curve from time zero to the time of the last quantifiable concentration [mass x time / volume] (AUClast)
Time Frame
Time Frame: Day 1 (period 1): 0.5, 2, 4, 8, 12, 24 hours post-dose; Day 7 (period 2): pre-dose; 0.5, 2, 4, 8 hours post dose
Title
Maximum observed plasma concentration (Cmax)
Time Frame
Time Frame: Day 1 (period 1): 0.5, 2, 4, 8, 12, 24 hours post-dose; Day 7 (period 2): pre-dose; 0.5, 2, 4, 8 hours post dose
Secondary Outcome Measure Information:
Title
Physical examination
Time Frame
Screening: once anytime between Day -30 and Day -1; once anytime between 24-72 hours after Day 7
Title
Vital signs and body measurements
Time Frame
Screening: once anytime between Day -30 and Day -1; once anytime between 24-72 hours after Day 7
Title
Electrocardiograms
Time Frame
Screening: once anytime between Day -30 and Day -1; once anytime between 24-72 hours after Day 7
Title
hematology
Time Frame
Screening: once anytime between Day -30 and Day -1; once anytime between 24-72 hours after Day 7
Title
blood chemistry
Time Frame
Screening: once anytime between Day -30 and Day -1; once anytime between 24-72 hours after Day 7
Title
neurological examination
Time Frame
Screening: once anytime between Day -30 and Day -1; once on Day 7
Title
Adverse events (AE) monitoring
Time Frame
During the study (total of approximately 32 days) and 3 days after study completion
Title
Serious adverse events (SAE) monitoring
Time Frame
During the study (total of approximately 32 days) and 30 days after study completion
10. Eligibility
Sex
All
Minimum Age & Unit of Time
3 Years
Maximum Age & Unit of Time
11 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Genetically confirmed diagnosis of FXS
At Screening and first baseline, vital signs, body weight and body mass index (BMI) must be age-specific within normal ranges.
Exclusion Criteria:
Use of any other investigational drug within 30 days or 5 half-lives (whichever is longer) of the investigational drug prior to screening until end of study visit.
History of hypersensitivity to AFQ056 or any mGluR antagonist.
Female patients who are confirmed or suspected to be sexually active.
History or presence of any clinically significant disease of any major system organ class, within the past 2 years prior to screening including but not limited to psychiatric, neurological, cardiovascular, endocrine, metabolic, renal, or gastrointestinal disorders (except for typical features of FXS).
Smokers.
Loss of ≥10% of total blood volume within 8 weeks (or less if required for this age group and/or by local regulation) prior to dosing or longer if required for this age group and/or by local regulation.
Significant illness that did not completely resolve at least four weeks prior to the first baseline visit.
Any abnormal laboratory values at screening or first baseline that are in the opinion of the investigator clinically significant and may jeopardize the safety of the study subject.
Use of (or use within at least 5 half lives before dosing) concomitant medications that are strong/moderate inhibitors or inducers of CYP1A1/2, CYP2C9/19 or CYP3A4
History or presence of Hepatitis B/C or HIV at screening
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
Sacramento
State/Province
California
ZIP/Postal Code
95817
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
Nashville
State/Province
Tennessee
ZIP/Postal Code
37232-7548
Country
United States
Facility Name
Novartis Investigative Site
City
Sant Cugat
State/Province
Catalunya
ZIP/Postal Code
08190
Country
Spain
12. IPD Sharing Statement
Links:
URL
https://www.novctrd.com/ctrdweb/trialresult/trialresults/pdf?trialResultId=11925
Description
Results for CAFQ056B2154 can be found on the Novartis Clinical Trial Results Website
Learn more about this trial
Clinical Study to Assess the Pharmacokinetics, Safety and Tolerability of Single and Multiple Oral Doses of AFQ056 in Children With Fragile X Syndrome (FXS)
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