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

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Brief Title

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

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 Description

      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 daily dosage group was prescribed as once daily at 08:00 a.m. and the
      twice daily dosage group received the treatment twice daily at 08:00 a.m. and 08:00 p.m.
      Disease severity was assessed via Mor scoring system, modified for pediatric patients in all
      visits. A physician from each center was chosen to be responsible for data collection. After
      each visit the data was registered to a web-based registry system in "www.favor.org.tr" web
      site. To ensure accurate, complete, and reliable data, the following procedures were
      followed: data collection, encoding, and storage had been provided for the centers; a
      training session had been held to provide instruction on the protocol; periodic meetings had
      been held with study coordinators the principal investigator stayed in contact with the study
      coordinators by mail, telephone, and/or fax; and finally a data manager reviewed and
      evaluated the data.

      Colchicine dosage The required colchicine dosage was calculated as total 1 mg daily according
      to internationally accepted advisory. All patients were prescribed 0.5 mg Colchicine tablets.
      Patients in once daily dosage group were given 2 tablets of Colchicine at 08.00 a.m. Twice
      daily dosage group received 1 tablet of 0,5 mg Colchicine at 08:00 a.m. and 1 tablet of 0,5
      mg Colchicine at 08:00 p.m.
    

Study Phase

Phase 4

Study Type

Interventional


Primary Outcome

Mor severity scoring system

Secondary Outcome

 Number of Participants With Abnormal Laboratory Values

Condition

Familial Mediterranean Fever

Intervention

colchicine

Study Arms / Comparison Groups

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

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

79

Start Date

April 2011

Completion Date

August 2013

Primary Completion Date

April 2013

Eligibility Criteria

        Inclusion Criteria:

          -  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

        Exclusion criteria:

          -  Major congenital malformation

          -  Organ transplantation

          -  Hepatic disorder

          -  Chronic kidney disease

          -  AA amyloidosis

          -  Thyroid disease

          -  Rheumatologic disorders other than FMF
      

Gender

All

Ages

5 Years - 16 Years

Accepts Healthy Volunteers

No

Contacts

Erkan Demirkaya, MD, MSc, , 

Location Countries

Turkey

Location Countries

Turkey

Administrative Informations


NCT ID

NCT02602028

Organization ID

1491-1437-11/1539


Responsible Party

Principal Investigator

Study Sponsor

Saglik Bilimleri Universitesi Gulhane Tip Fakultesi


Study Sponsor

Erkan Demirkaya, MD, MSc, Principal Investigator, Saglik Bilimleri Universitesi Gulhane Tip Fakultesi


Verification Date

November 2015