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

NCT01075906 | Completed Phase 1 DMC

• 1 other identifier: MPC-006-09-1001
• Study Type: interventional
• Target: 75
• Locations: 4 countries
• Sites: 10

Brief 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.

Conditions

Familial Mediterranean Fever

Keywords

pharmacokinetics

Outcome Measures

Primary Outcomes (3)

• Maximum Plasma Concentration

  pharmacokinetic samples collected pre-dose on Days 1, 2 and 15 and at 0.25 - 0.5 hours, 1.5-2.5 hours and at 5-8 hours post-dose on Days 1 and 15

  15 days

• Area Under the Concentration Time Curve from Time Zero to the Time of Last Measured Concentration (AUC 0-t)

  Pharmacokinetic samples collected pre-dose on Days 1,2 and 15 and at 0.25-0.5 hours, 1.5-2.5 hours and 5-8 hours post-dose on Days 1 and 15.

  15 days

• Area Under the Concentration Time Curve from Zero through Infinity

  Pharmacokinetic samples collected pre-dose on Days 1, 2 and 15 and at 0.25-0.5 hours, 1.5-2.5 hours and at 5-8 hours post-dose on Days 1 and 15

  15 days

Secondary Outcomes (1)

• Acute Phase Reactant (ESR, CRP, SAA) Levels

  15 days

Study Arms (2)

Colchicine

EXPERIMENTAL

Colchicine Sprinkle Capsules, 0.3 mg - dose administered according to age range on Day 1

Drug: colchicine sprinkle capsules

colchicine at steady state

EXPERIMENTAL

colchicine sprinkle capsules 0.3 mg - dose administered according to age range on Day 15 following once daily dosing of colchicine on Days 2 - 14

Drug: colchicine sprinkle capsules

Interventions

colchicine sprinkle capsules DRUG

0.3 mg

Colchicine

Eligibility Criteria

• Age: 2 Years - 65 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Patients age 2-65 years with a confirmed clinical diagnosis of FMF,
• Non-pregnant, and
• If of child-bearing potential, using effective contraceptive measures.

You may not qualify if:

• Recent participation (within 30 days) in other research studies,
• Pregnant or lactating,
• History or current infection of human immunodeficiency virus (HIV), hepatitis A, B or C,
• Current or recent use of any drugs/drug classes or combinations thereof that may affect the absorption or metabolism of colchicine,
• Clinically relevant abnormal clinical laboratories at screening,
• Current or recent (\<6 months) history of severe, unstable or uncontrolled neurological, cardiovascular, gastrointestinal, hematological, moderate or severe hepatic and/or renal disease, or evidence of other diseases at the physical examination conducted at the screening.

Sponsors & Collaborators

• Mutual Pharmaceutical Company, Inc.: lead

Study Sites (10)

Childrens Hospital Los Angeles

Los Angeles, California, 90027, United States

Location

Center of Medical Genetics and Primary Health Care

Yerevan, Yerevan, 0010, Armenia

Location

Soroka Medical Center

Beersheba, Beer Sheba, 84141, Israel

Location

Rambam Medical Center

Haifa, Haifa District, 24035, Israel

Location

Hadassah Medical Center

Jerusalem, Israel, 91120, Israel

Location

Pediatric Rheumatology Unit - Shaare Zedek Medical Center

Jerusalem, Jerusalem, 91031, Israel

Location

Safra Children's Hospital

Tel Litwinsky, Tel Hashomer, 52621, Israel

Location

Sheba Medical Center

Tel Litwinsky, Tel Hashomer, 52621, Israel

Location

Hacettepe University

Ankara, Ankara, 06100, Turkey (Türkiye)

Location

Cerrahpasa Medical Facility

Istanbul, Istanbul, 34303, Turkey (Türkiye)

Location

MeSH Terms

Conditions

Familial Mediterranean Fever

Condition Hierarchy (Ancestors)

Hereditary Autoinflammatory Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Matthew Davis, MD

  Mutual Pharmaceutical Company, Inc.

  STUDY CHAIR

Study Design

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

Study Record Dates

• First Submitted: February 24, 2010
• First Posted: February 25, 2010
• Study Start: August 1, 2010
• Primary Completion: December 1, 2011
• Study Completion: December 1, 2011
• Last Updated: January 10, 2012

Record last verified: 2012-01

Locations

TU (2); US (1); IL (6); AR (1)