Clinical Trial: The Comparison of the Efficacy of Once and Twice Daily Colchicine Dosage in Pediatric Patients With FMF

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: The Comparison of the Efficacy of Once and Twice Daily Colchicine Dosage in Pediatric Patients With Familial Mediterranean Fever: A Randomized Trial

Brief Summary:

It was aimed to examine the efficacy and safety of once daily dosage schema of colchicine in pediatric patients with FMF compared to twice daily dosage schema.

In this 24-week, multicentric, randomized, controlled, noninferiority trial, pediatric patients newly diagnosed with FMF, carrying homozygote or compound heterozygote mutation and did not receive any treatment, were included. Patients were randomly assigned using block randomization method to receive treatment with once or twice daily doses. Clinical and laboratory characteristics and medication side effects were recorded and compared between groups. The study complied with Good Clinical Practice and the Consolidated Standards for Reporting of Trials (CONSORT) statement.

Detailed Summary:

Study design This study was conducted by members of the FMF Arthritis Vasculitis and Orphan disease Research in Pediatric Rheumatology (FAVOR, www.favor.org.tr) at 10 centers in Turkey. This is a multicentric randomized controlled, noninferiority trial of two parallel groups being followed up in pediatric rheumatology outpatient clinics. The randomization was done at baseline visit and patients were assessed in two more visits, three months apart. The study complied with Good Clinical Practice (GCP) and the Consolidated Standards for Reporting of Trials (CONSORT) statement.

Participants Inclusion criteria Pediatric patients who were newly diagnosed with Familial Mediterranean Fever according to Yalcinkaya or Tel-Hashomer criteria and who were confirmed with the genetic analysis as having compound heterozygote or homozygote mutation were enrolled. Eligible patients between ages of 5-16 and weighted 15-30 kg who and had not received any treatment were included. All patients included were asked the physicians to be proved and recorded as having at least one FMF attack before enrollment for the study.

Exclusion criteria Patients with a major congenital malformation, with a risk of pregnancy, and with other chronic diseases such as organ transplantation, hepatic disorder chronic kidney disease and AA amyloidosis, thyroid disease or rheumatologic disorders other than FMF, were excluded.

Baseline assessment and Outcome Measures At the baseline visit, medical history and complaints about the disease were questioned and physical examination and laboratory tests were performed. In following visits, any attack or findings due to colchicine since the last visit were investigated in addition to baseline visit.

Interventions The once dail
Sponsor: Saglik Bilimleri Universitesi Gulhane Tip Fakultesi

Current Primary Outcome:

• Mor severity scoring system [ Time Frame: At baseline and every 12 weeks, up to 24 weeks. Change in disease severity as assessed by the Mor severity scoring system. ]
  The primary objectives of this study were to compare the effectiveness of once and twice daily colchicine dosage regimens regarding taking control of disease symptoms which can be reducing disease severity which was assessed by modified Mor scoring system and
• Adverse events [ Time Frame: up to 24 weeks ]
  Adverse Events That Are Related to Treatment

Original Primary Outcome: Same as current

Current Secondary Outcome: Number of Participants With Abnormal Laboratory Values [ Time Frame: At baseline and every 12 weeks, up to 24 weeks. ]

Original Secondary Outcome: Same as current

Information By: Saglik Bilimleri Universitesi Gulhane Tip Fakultesi

Dates:
Date Received: November 5, 2015
Date Started: April 2011
Date Completion:
Last Updated: November 9, 2015
Last Verified: November 2015