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

Fragile X Syndrome Clinical Trial

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)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01482143
• Other study ID #: CAFQ056B2154
• Secondary ID: 2011-004867-65
• Status: Completed
• Phase: Phase 1
• Start date: March 2012
• Est. completion date: October 2013

Study information

• Verified date: September 2014
• Source: Novartis
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

Recruitment information / eligibility

• Status: Completed
• Enrollment: 21
• Est. completion date: October 2013
• Est. primary completion date: October 2013
• Accepts healthy volunteers: No
• Gender: All
• Age group: 3 Years to 11 Years

Eligibility

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

Related Conditions & MeSH terms

• Fragile X Syndrome
• Syndrome

Intervention

Drug:
• AFQ056

Locations

Country	Name	City	State
Spain	Novartis Investigative Site	Sant Cugat	Catalunya
United States	Novartis Investigative Site	Chicago	Illinois
United States	Novartis Investigative Site	Nashville	Tennessee
United States	Novartis Investigative Site	Sacramento	California

Sponsors (1)

Lead Sponsor	Collaborator
Novartis Pharmaceuticals

Countries where clinical trial is conducted

United States,  Spain, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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
Primary	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
Primary	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	Physical examination		Screening: once anytime between Day -30 and Day -1; once anytime between 24-72 hours after Day 7
Secondary	Vital signs and body measurements		Screening: once anytime between Day -30 and Day -1; once anytime between 24-72 hours after Day 7
Secondary	Electrocardiograms		Screening: once anytime between Day -30 and Day -1; once anytime between 24-72 hours after Day 7
Secondary	hematology		Screening: once anytime between Day -30 and Day -1; once anytime between 24-72 hours after Day 7
Secondary	blood chemistry		Screening: once anytime between Day -30 and Day -1; once anytime between 24-72 hours after Day 7
Secondary	neurological examination		Screening: once anytime between Day -30 and Day -1; once on Day 7
Secondary	Adverse events (AE) monitoring		During the study (total of approximately 32 days) and 3 days after study completion
Secondary	Serious adverse events (SAE) monitoring		During the study (total of approximately 32 days) and 30 days after study completion

External Links

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