The Comparison of the Efficacy of Once and Twice Daily Colchicine Dosage in Pediatric Patients With FMF
Primary Purpose
Familial Mediterranean Fever
Status
Completed
Phase
Phase 4
Locations
Turkey
Study Type
Interventional
Intervention
colchicine
Sponsored by
About this trial
This is an interventional treatment trial for Familial Mediterranean Fever focused on measuring Familial Mediterranean Fever, Colchicine
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
Sites / Locations
- FMF Arthritis Vasculitis and Orphan Disease Research in Paediatric Rheumatology (FAVOR)
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Experimental
Arm Label
once daily dosage schema of colchicine
twice daily dosage schema of colchicine
Arm Description
The once daily dosage group was prescribed as once daily at 08:00 a.m. (Total 1mg)
Twice daily dosage group received the treatment twice daily at 08:00 a.m. and 08:00 p.m. (Total 1mg)
Outcomes
Primary Outcome Measures
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
Adverse Events That Are Related to Treatment
Secondary Outcome Measures
Number of Participants With Abnormal Laboratory Values
Laboratory findings such as erythrocyte sedimentation rate (ESR), C-reactive protein (CRP), serum amyloid A (SAA) levels were used for the indication of inflammation.
Full Information
NCT ID
NCT02602028
First Posted
November 5, 2015
Last Updated
November 9, 2015
Sponsor
Saglik Bilimleri Universitesi Gulhane Tip Fakultesi
1. Study Identification
Unique Protocol Identification Number
NCT02602028
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
Study Type
Interventional
2. Study Status
Record Verification Date
November 2015
Overall Recruitment Status
Completed
Study Start Date
April 2011 (undefined)
Primary Completion Date
April 2013 (Actual)
Study Completion Date
August 2013 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Saglik Bilimleri Universitesi Gulhane Tip Fakultesi
4. Oversight
Data Monitoring Committee
No
5. 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.
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Familial Mediterranean Fever
Keywords
Familial Mediterranean Fever, Colchicine
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Parallel Assignment
Masking
Investigator
Allocation
Randomized
Enrollment
79 (Actual)
8. Arms, Groups, and Interventions
Arm Title
once daily dosage schema of colchicine
Arm Type
Experimental
Arm Description
The once daily dosage group was prescribed as once daily at 08:00 a.m. (Total 1mg)
Arm Title
twice daily dosage schema of colchicine
Arm Type
Experimental
Arm Description
Twice daily dosage group received the treatment twice daily at 08:00 a.m. and 08:00 p.m. (Total 1mg)
Intervention Type
Drug
Intervention Name(s)
colchicine
Other Intervention Name(s)
colchicum
Intervention Description
to compare once daily dosage schema of colchicine and twice daily dosage schema of colchicine
Primary Outcome Measure Information:
Title
Mor severity scoring system
Description
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
Time Frame
At baseline and every 12 weeks, up to 24 weeks. Change in disease severity as assessed by the Mor severity scoring system.
Title
Adverse events
Description
Adverse Events That Are Related to Treatment
Time Frame
up to 24 weeks
Secondary Outcome Measure Information:
Title
Number of Participants With Abnormal Laboratory Values
Description
Laboratory findings such as erythrocyte sedimentation rate (ESR), C-reactive protein (CRP), serum amyloid A (SAA) levels were used for the indication of inflammation.
Time Frame
At baseline and every 12 weeks, up to 24 weeks.
10. Eligibility
Sex
All
Minimum Age & Unit of Time
5 Years
Maximum Age & Unit of Time
16 Years
Accepts Healthy Volunteers
No
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Erkan Demirkaya, MD, MSc
Organizational Affiliation
Saglik Bilimleri Universitesi Gulhane Tip Fakultesi
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Cengizhan Acikel, MD, MPH
Organizational Affiliation
Saglik Bilimleri Universitesi Gulhane Tip Fakultesi
Official's Role
Study Chair
Facility Information:
Facility Name
FMF Arthritis Vasculitis and Orphan Disease Research in Paediatric Rheumatology (FAVOR)
City
Ankara
ZIP/Postal Code
06018
Country
Turkey
12. IPD Sharing Statement
Citations:
PubMed Identifier
27055417
Citation
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.
Results Reference
derived
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The Comparison of the Efficacy of Once and Twice Daily Colchicine Dosage in Pediatric Patients With FMF
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