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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
Novartis Pharmaceuticals
About
Eligibility
Locations
Arms
Outcomes
Full info

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

3 Years - 11 Years (Child)All SexesDoes not accept healthy volunteers

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

First Posted
November 21, 2011
Last Updated
December 6, 2020
Sponsor
Novartis Pharmaceuticals
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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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