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