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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)

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT01482143
First Posted: November 30, 2011
Last Update Posted: September 30, 2014
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
  Purpose
The aim of this study is to characterize the pharmacokinetics and safety/tolerability of AFQ056 in children with Fragile X Syndrome(FXS)

Condition Intervention Phase
Fragile X Syndrome Drug: AFQ056 Phase 1

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
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)

Resource links provided by NLM:


Further study details as provided by Novartis ( Novartis Pharmaceuticals ):

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) [ 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 ]
  • 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 ]
  • 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 Measures:
  • Physical examination [ Time Frame: Screening: once anytime between Day -30 and Day -1; once anytime between 24-72 hours after Day 7 ]
  • 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 ]
  • Electrocardiograms [ Time Frame: Screening: once anytime between Day -30 and Day -1; once anytime between 24-72 hours after Day 7 ]
  • hematology [ Time Frame: Screening: once anytime between Day -30 and Day -1; once anytime between 24-72 hours after Day 7 ]
  • blood chemistry [ Time Frame: Screening: once anytime between Day -30 and Day -1; once anytime between 24-72 hours after Day 7 ]
  • neurological examination [ Time Frame: Screening: once anytime between Day -30 and Day -1; once on Day 7 ]
  • Adverse events (AE) monitoring [ Time Frame: During the study (total of approximately 32 days) and 3 days after study completion ]
  • Serious adverse events (SAE) monitoring [ Time Frame: During the study (total of approximately 32 days) and 30 days after study completion ]

Enrollment: 21
Study Start Date: March 2012
Study Completion Date: October 2013
Primary Completion Date: October 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: All Study subjects Drug: AFQ056

  Eligibility

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Ages Eligible for Study:   3 Years to 11 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01482143


Locations
United States, California
Novartis Investigative Site
Sacramento, California, United States, 95817
United States, Illinois
Novartis Investigative Site
Chicago, Illinois, United States, 60612
United States, Tennessee
Novartis Investigative Site
Nashville, Tennessee, United States, 37232-7548
Spain
Novartis Investigative Site
Sant Cugat, Catalunya, Spain, 08190
Sponsors and Collaborators
Novartis Pharmaceuticals
Investigators
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
  More Information

Responsible Party: Novartis Pharmaceuticals
ClinicalTrials.gov Identifier: NCT01482143     History of Changes
Other Study ID Numbers: CAFQ056B2154
2011-004867-65 ( EudraCT Number )
First Submitted: November 21, 2011
First Posted: November 30, 2011
Last Update Posted: September 30, 2014
Last Verified: September 2014

Keywords provided by Novartis ( Novartis Pharmaceuticals ):
Fragile X Syndrome
pharmacokinetics
safety and tolerability

Additional relevant MeSH terms:
Syndrome
Fragile X Syndrome
Disease
Pathologic Processes
Mental Retardation, X-Linked
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
Sex Chromosome Disorders
Chromosome Disorders
Congenital Abnormalities
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Heredodegenerative Disorders, Nervous System