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Clinical study to assess the pharmacokinetics, safety & tolerability of single and multiple oral doses of AFQ056 in children with Fragile X Syndrome

Phase 1
Conditions
Fragile X Syndrome
MedDRA version: 14.1Level: PTClassification code 10017324Term: Fragile X syndromeSystem Organ Class: 10010331 - Congenital, familial and genetic disorders
Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Registration Number
EUCTR2011-004867-65-ES
Lead Sponsor
ovartis Farmacéutica, S.A.
Brief Summary

Not available

Detailed Description

Not available

Recruitment & Eligibility

Status
ot Recruiting
Sex
All
Target Recruitment
21
Inclusion Criteria

Genetically confirmed diagnosis of FXS

At Screening and first baseline, vital signs, body weight and BMI must be age-specific within normal ranges
Are the trial subjects under 18? yes
Number of subjects for this age range: 24
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

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.

Study & Design

Study Type
Interventional clinical trial of medicinal product
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
Secondary Outcome Measures
NameTimeMethod
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