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Trial record 17 of 47 for:    "Fragile X syndrome"

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:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier:
NCT01482143
First received: November 21, 2011
Last updated: September 29, 2014
Last verified: September 2014
  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: 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:

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 ] [ Designated as safety issue: No ]
  • 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 ] [ Designated as safety issue: No ]
  • 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 ] [ Designated as safety issue: No ]

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

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

Ages Eligible for Study:   3 Years to 11 Years
Genders Eligible for Study:   Both
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
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, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: 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

No publications provided

Responsible Party: Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier: NCT01482143     History of Changes
Other Study ID Numbers: CAFQ056B2154, 2011-004867-65
Study First Received: November 21, 2011
Last Updated: September 29, 2014
Health Authority: United States: Food and Drug Administration
Switzerland: Swissmedic

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

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

ClinicalTrials.gov processed this record on November 20, 2014