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

NCT02602028 | Completed Phase 4

• 1 other identifier: 1491-1437-11/1539
• Study Type: interventional
• Target: 79
• Locations: 1 country
• Sites: 1

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.

Conditions

Familial Mediterranean Fever

Keywords

Familial Mediterranean Fever; Colchicine

Outcome Measures

Primary Outcomes (2)

• 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

  At baseline and every 12 weeks, up to 24 weeks. Change in disease severity as assessed by the Mor severity scoring system.

• Adverse events

  Adverse Events That Are Related to Treatment

  up to 24 weeks

Secondary Outcomes (1)

• Number of Participants With Abnormal Laboratory Values

  At baseline and every 12 weeks, up to 24 weeks.

Study Arms (2)

once daily dosage schema of colchicine

EXPERIMENTAL

The once daily dosage group was prescribed as once daily at 08:00 a.m. (Total 1mg)

Drug: colchicine

twice daily dosage schema of colchicine

EXPERIMENTAL

Twice daily dosage group received the treatment twice daily at 08:00 a.m. and 08:00 p.m. (Total 1mg)

Drug: colchicine

Interventions

colchicine DRUG

to compare once daily dosage schema of colchicine and twice daily dosage schema of colchicine

Also known as: colchicum

once daily dosage schema of colchicine; twice daily dosage schema of colchicine

Eligibility Criteria

• Age: 5 Years - 16 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Diagnosed with Familial Mediterranean Fever according to Yalcinkaya or
• Tel-Hashomer criteria
• Diagnosis confirmed with the genetic analysis as compound heterozygote or
• homozygote mutation
• Patients between ages of 5-16 and weighted 15-30 kg
• Not received any treatment for FMF

You may not qualify if:

• Major congenital malformation
• Organ transplantation
• Hepatic disorder
• Chronic kidney disease
• AA amyloidosis
• Thyroid disease
• Rheumatologic disorders other than FMF

Sponsors & Collaborators

• Saglik Bilimleri Universitesi Gulhane Tip Fakultesi: lead

Study Sites (1)

FMF Arthritis Vasculitis and Orphan Disease Research in Paediatric Rheumatology (FAVOR)

Ankara, 06018, Turkey (Türkiye)

Location

Related Publications (1)

• Polat A, Acikel C, Sozeri B, Dursun I, Kasapcopur O, Gulez N, Simsek D, Saldir M, Dokurel I, Poyrazoglu H, Bakkaloglu S, Delibas A, Ekinci Z, Ayaz NA, Kandur Y, Peru H, Kurt YG, Polat SR, Unsal E, Makay B, Gok F, Ozen S, Demirkaya E; FMF Arthritis Vasculitis and Orphan Disease Research in Pediatric Rheumatology (FAVOR). Comparison of the efficacy of once- and twice-daily colchicine dosage in pediatric patients with familial Mediterranean fever--a randomized controlled noninferiority trial. Arthritis Res Ther. 2016 Apr 7;18:85. doi: 10.1186/s13075-016-0980-7.

  PMID: 27055417 DERIVED

MeSH Terms

Conditions

Familial Mediterranean Fever

Interventions

Colchicine

Condition Hierarchy (Ancestors)

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

Intervention Hierarchy (Ancestors)

Alkaloids; Heterocyclic Compounds

Study Officials

• Erkan Demirkaya, MD, MSc

  Saglik Bilimleri Universitesi Gulhane Tip Fakultesi

  PRINCIPAL INVESTIGATOR

• Cengizhan Acikel, MD, MPH

  Saglik Bilimleri Universitesi Gulhane Tip Fakultesi

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: RANDOMIZED
• Masking: SINGLE
• Who Masked: INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: MD, MSc, Assoc. Prof.

Study Record Dates

• First Submitted: November 5, 2015
• First Posted: November 11, 2015
• Study Start: April 1, 2011
• Primary Completion: April 1, 2013
• Study Completion: August 1, 2013
• Last Updated: November 11, 2015

Record last verified: 2015-11

Locations

TU (1)