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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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ClinicalTrials.gov Identifier: NCT02602028
Recruitment Status : Completed
First Posted : November 11, 2015
Last Update Posted : November 11, 2015
Sponsor:
Information provided by (Responsible Party):
Erkan Demirkaya, Saglik Bilimleri Universitesi Gulhane Tip Fakultesi

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.

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Study Type : Interventional  (Clinical Trial)
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 : April 2011
Actual Primary Completion Date : April 2013
Actual Study Completion Date : August 2013


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 Name: 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 Name: colchicum




Primary Outcome Measures :
  1. Mor severity scoring system [ Time Frame: 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

  2. Adverse events [ Time Frame: up to 24 weeks ]
    Adverse Events That Are Related to Treatment


Secondary Outcome Measures :
  1. Number of Participants With Abnormal Laboratory Values [ Time Frame: 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.



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Ages Eligible for Study:   5 Years to 16 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02602028


Locations
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Turkey
FMF Arthritis Vasculitis and Orphan Disease Research in Paediatric Rheumatology (FAVOR)
Ankara, Turkey, 06018
Sponsors and Collaborators
Saglik Bilimleri Universitesi Gulhane Tip Fakultesi
Investigators
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Principal Investigator: Erkan Demirkaya, MD, MSc Saglik Bilimleri Universitesi Gulhane Tip Fakultesi
Study Chair: Cengizhan Acikel, MD, MPH Saglik Bilimleri Universitesi Gulhane Tip Fakultesi
Additional Information:
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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Responsible Party: Erkan Demirkaya, MD, MSc, Assoc. Prof., Saglik Bilimleri Universitesi Gulhane Tip Fakultesi
ClinicalTrials.gov Identifier: NCT02602028    
Other Study ID Numbers: 1491-1437-11/1539
First Posted: November 11, 2015    Key Record Dates
Last Update Posted: November 11, 2015
Last Verified: November 2015
Keywords provided by Erkan Demirkaya, Saglik Bilimleri Universitesi Gulhane Tip Fakultesi:
Familial Mediterranean Fever
Colchicine
Additional relevant MeSH terms:
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Brucellosis
Familial Mediterranean Fever
Hereditary Autoinflammatory Diseases
Fever
Body Temperature Changes
Gram-Negative Bacterial Infections
Bacterial Infections
Genetic Diseases, Inborn
Skin Diseases, Genetic
Skin Diseases
Colchicine
Gout Suppressants
Antirheumatic Agents
Tubulin Modulators
Antimitotic Agents
Mitosis Modulators
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents