Pharmacokinetics Study of Colchicine in Familial Mediterranean Fever (FMF) Patients
Condition: Familial Mediterranean Fever
MeSH terms: Brucellosis; Familial Mediterranean Fever; Hereditary Autoinflammatory Diseases; Fever
Study type: Interventional
Phase: Phase 1

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.
    

Intervention name(s): colchicine sprinkle capsules; colchicine sprinkle capsules
Intervention type(s): Drug; Drug
Intervention description(s): 0.3 mg; 0.3 mg
Intervention MeSH terms: Colchicine
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Primary purpose: Basic Science
Keywords: pharmacokinetics