Clinical Study to Assess the Pharmacokinetics, Safety and Tolerability of Single and Multiple Oral Doses of AFQ056 in Children With Fragile X Syndrome (FXS)

NCT01482143 | Completed Phase 1

• 2 other identifiers: CAFQ056B21542011-004867-65
• Study Type: interventional
• Target: 21
• Locations: 2 countries
• Sites: 4

Brief Summary

The aim of this study is to characterize the pharmacokinetics and safety/tolerability of AFQ056 in children with Fragile X Syndrome(FXS)

Conditions

Fragile X Syndrome

Keywords

Fragile X Syndrome; pharmacokinetics; safety and tolerability

Outcome Measures

Primary Outcomes (3)

• The area under the plasma (or serum or blood) concentration-time curve from time zero to infinity [mass x time / volume] (AUCinf)

  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: 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: 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 Outcomes (8)

• Physical examination

  Screening: once anytime between Day -30 and Day -1; once anytime between 24-72 hours after Day 7

• Vital signs and body measurements

  Screening: once anytime between Day -30 and Day -1; once anytime between 24-72 hours after Day 7

• Electrocardiograms

  Screening: once anytime between Day -30 and Day -1; once anytime between 24-72 hours after Day 7

• hematology

  Screening: once anytime between Day -30 and Day -1; once anytime between 24-72 hours after Day 7

• blood chemistry

  Screening: once anytime between Day -30 and Day -1; once anytime between 24-72 hours after Day 7

Study Arms (1)

All Study subjects

EXPERIMENTAL

Drug: AFQ056

Interventions

AFQ056 DRUG

All Study subjects

Eligibility Criteria

• Age: 3 Years - 11 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (4)

Novartis Investigative Site

Sacramento, California, 95817, United States

Location

Novartis Investigative Site

Chicago, Illinois, 60612, United States

Location

Novartis Investigative Site

Nashville, Tennessee, 37232-7548, United States

Location

Novartis Investigative Site

Sant Cugat del Vallès, Catalonia, 08190, Spain

Location

Related Links

• Results for CAFQ056B2154 can be found on the Novartis Clinical Trial Results Website

MeSH Terms

Conditions

Fragile X Syndrome

Interventions

mavoglurant

Condition Hierarchy (Ancestors)

X-Linked Intellectual Disability; Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Nervous System Diseases; Sex Chromosome Disorders; Chromosome Disorders; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Heredodegenerative Disorders, Nervous System

Study Officials

• Novartis Pharmaceuticals

  Novartis Pharmaceuticals

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: OTHER
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 21, 2011
• First Posted: November 30, 2011
• Study Start: March 1, 2012
• Primary Completion: October 1, 2013
• Study Completion: October 1, 2013
• Last Updated: December 8, 2020

Record last verified: 2014-09

Locations

ES (1); US (3)