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

Sponsor

Saglik Bilimleri Universitesi Gulhane Tip Fakultesi (Other)

Overall Status

Completed

CT.gov ID

NCT02602028

Collaborator

(none)

Enrollment

Location

Arms

Duration (Months)

2.8

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Familial Mediterranean Fever	Drug: colchicine	Phase 4

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 Design

Study Type:

Interventional

Actual Enrollment :

79 participants

Allocation:

Randomized

Intervention Model:

Parallel Assignment

Masking:

Single (Investigator)

Primary Purpose:

Treatment

Official Title:

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

Study Start Date :

Apr 1, 2011

Actual Primary Completion Date :

Apr 1, 2013

Actual Study Completion Date :

Aug 1, 2013

Arms and Interventions

Arm	Intervention/Treatment
Experimental: once daily dosage schema of colchicine The once daily dosage group was prescribed as once daily at 08:00 a.m. (Total 1mg)	Drug: colchicine to compare once daily dosage schema of colchicine and twice daily dosage schema of colchicine Other Names: colchicum
Experimental: twice daily dosage schema of colchicine Twice daily dosage group received the treatment twice daily at 08:00 a.m. and 08:00 p.m. (Total 1mg)	Drug: colchicine to compare once daily dosage schema of colchicine and twice daily dosage schema of colchicine Other Names: colchicum

Arm

Intervention/Treatment

Experimental: once daily dosage schema of colchicine

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

Drug: colchicine

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

Other Names:

colchicum

Experimental: twice daily dosage schema of colchicine

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

Drug: colchicine

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

Other Names:

colchicum

Outcome Measures

Primary Outcome Measures

Mor severity scoring system [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 [up to 24 weeks]
Adverse Events That Are Related to Treatment

Secondary Outcome Measures

Number of Participants With Abnormal Laboratory Values [At baseline and every 12 weeks, up to 24 weeks.]
Laboratory findings such as erythrocyte sedimentation rate (ESR), C-reactive protein (CRP), serum amyloid A (SAA) levels were used for the indication of inflammation.

Eligibility Criteria

Criteria

Ages Eligible for Study:

5 Years to 16 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	FMF Arthritis Vasculitis and Orphan Disease Research in Paediatric Rheumatology (FAVOR)	Ankara		Turkey	06018

Sponsors and Collaborators

Saglik Bilimleri Universitesi Gulhane Tip Fakultesi

Investigators

Principal Investigator: Erkan Demirkaya, MD, MSc, Saglik Bilimleri Universitesi Gulhane Tip Fakultesi
Study Chair: Cengizhan Acikel, MD, MPH, Saglik Bilimleri Universitesi Gulhane Tip Fakultesi