NCT01075906

Pharmacokinetics Study of Colchicine in Familial Mediterranean Fever (FMF) Patients

An Open-label, Parallel-group, Multiple-Dose, Pharmacokinetic and Safety Study of Colchicine Pediatric Formulation in Pediatric and Adult Patients With FMF

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 75 (actual)
• Sponsor: Mutual Pharmaceutical Company, Inc. · Industry
• Sex: All
• Age: 2 Years – 65 Years
• Healthy volunteers: Accepted

Summary

Colchicine is widely recognized as safe and effective treatment of Familial Mediterranean Fever (FMF) in children and adults. Colchicine is currently used to treat FMF in younger patients by inexact dosing through breaking or crushing adult-dose tablets. An age-appropriate sprinkle formulation will allow for more accurate dosing in pediatric patients. The primary objective of this study is to evaluate and compare the steady-state pharmacokinetics of multiple oral doses of colchicine sprinkle capsules administered to pediatric and adult FMF patients. Secondary objectives include evaluation of the safety and tolerability of this regimen in pediatric and adult FMF patients and measurement of the levels of acute phase reactants (i.e, serum amyloid A \[SAA\], erythrocyte sedimentation rate \[ESR\], C-reactive protein \[CRP\]) at baseline and after dosing.

Detailed description

FMF patients who have not been taking colchicine (colchicine-naïve patients) will be enrolled into a 1 week dose-titration period (Days -7 to -1). Beginning on Day -7, a pre-dose blood sample will be collected from the colchicine-naïve patient population for determination of pharmacodynamic markers. Patients will then be administered a low starting dose of colchicine (as determined by the principal investigator) titrated up to the study colchicine dose which is 0.6 mg (2 capsules) in children ≥2 to \< 6 years old, 0.9 mg (3 capsules) in children ≥6 to \< 12, 1.2 mg (4 capsules) in children ≥12 to \< 16 and adults ≥16 and \< 65. On Day 2, patients will return to the clinic for collection of a pre-dose blood sample followed by administration of the study dose of colchicine. On Days 3-7, patients (parent/guardian) will self-medicate with the study dose of colchicine recording the time of dosing and any adverse events. On Days 8-14, patients (parent/guardian) will self-medicate with the study dose of colchicine recording the time of dosing and any adverse events. On the morning of Day 15, patients will return to the clinic for collection of a pre-dose blood sample followed by administration of the study dose of colchicine. Blood samples will be collected post-dose at times sufficient to adequately define the pharmacokinetics of colchicine and its metabolites. Safety and tolerability of this dosing regimen will be determined by evaluation of vital signs and adverse events during the study and upon completion of the study. All adverse events will be evaluated by the investigator and reported in the subject's case report form.

Conditions

• Familial Mediterranean Fever

Interventions

Type	Name	Description
DRUG	colchicine sprinkle capsules	0.3 mg
DRUG	colchicine sprinkle capsules	0.3 mg

Timeline

• Start date: 2010-08-01
• Primary completion: 2011-12-01
• Completion: 2011-12-01
• First posted: 2010-02-25
• Last updated: 2012-01-10

Locations

10 sites across 4 countries: United States, Armenia, Israel, Turkey (Türkiye)