Familial Mediterranean FeverPhase 4NCT02602028
The Comparison of the Efficacy of Once and Twice Daily Colchicine Dosage in Pediatric Patients With FMF
A Phase 4 study testing colchicine in people with Familial Mediterranean Fever, run by Saglik Bilimleri Universitesi Gulhane Tip Fakultesi at 1 site in Turkey (Türkiye).
Trial 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… Read more about this study
- Who is it for?
- Ages 5 to 16 years
- Conditions the study is for
- Familial Mediterranean Fever
- What is being tested?
- colchicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 79 participants
- Where
- 1 siteTurkey (Türkiye)
- Study dates
- Apr 2011to Aug 2013
- Phase
- Phase 4
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 5 to 16 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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
Who cannot join
- Major congenital malformation
- Organ transplantation
- Hepatic disorder
- Chronic kidney disease
- AA amyloidosis
- Thyroid disease
- Rheumatologic disorders other than FMF
Trial locations and contacts
1 site in 1 country.
Turkey (Türkiye)1 site
FMF Arthritis Vasculitis and Orphan Disease Research in Paediatric Rheumatology (FAVOR)
Ankara, 06018
About this study
Official title: The Comparison of the Efficacy of Once and Twice Daily Colchicine Dosage in Pediatric Patients With Familial Mediterranean Fever: A Randomized Trial
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.
Read the 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.
- Conditions
- Familial Mediterranean Fever
- Being tested
- colchicine
- Keywords
- Familial Mediterranean Fever, Colchicine
- Medical subject headings
- Familial Mediterranean Fever
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
colchicineDrug
Also called colchicum
to compare once daily dosage schema of colchicine and twice daily dosage schema of colchicine
| Study group | Role | What they receive |
|---|---|---|
| once daily dosage schema of colchicine | Experimental | The once daily dosage group was prescribed as once daily at 08:00 a.m. (Total 1mg) Receives: Drug: colchicine |
| twice daily dosage schema of colchicine | Experimental | Twice daily dosage group received the treatment twice daily at 08:00 a.m. and 08:00 p.m. (Total 1mg) Receives: Drug: colchicine |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: At baseline and every 12 weeks, up to 24 weeks. Change in disease severity as assessed by the Mor severity scoring system.
Adverse events
Adverse Events That Are Related to Treatment
Measured: up to 24 weeks
Secondary outcomes (1)
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.
Measured: At baseline and every 12 weeks, up to 24 weeks.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Nov 2015 (estimated)
- Study startApr 2011
- Primary completionApr 2013
- Study completionAug 2013
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Familial Mediterranean FeverStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind
- Who is masked
- Investigator
- Enrolment
- 79 participants (actual)
Sponsor and investigators
- Lead sponsor
- Saglik Bilimleri Universitesi Gulhane Tip Fakultesi
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Erkan Demirkaya, Saglik Bilimleri Universitesi Gulhane Tip Fakultesi
- Investigators
- Erkan Demirkaya, MD, MSc, Principal investigator, Saglik Bilimleri Universitesi Gulhane Tip FakultesiCengizhan Acikel, MD, MPH, Study chair, Saglik Bilimleri Universitesi Gulhane Tip Fakultesi
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02602028
- Sponsor's protocol ID
- 1491-1437-11/1539
- Status last verified
- Nov 2015
- First submitted
- 5 Nov 2015
- Last updated
- 11 Nov 2015
- Results posted
- No
- Data monitoring committee
- No
Publications
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.
PubMed 27055417
Study at a glance
From the registry record, last updated by the sponsor on 11 Nov 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Comparison of the Efficacy of Once and Twice Daily Colchicine Dosage in Pediatric Patients With Familial Mediterranean Fever: A Randomized Trial
- ClinicalTrials.gov ID
- NCT02602028
- Acronym / sponsor ID
- 1491-1437-11/1539
- Conditions
- Familial Mediterranean Fever
- Keywords
- Familial Mediterranean Fever, Colchicine
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 79 participants (actual)
- Lead sponsor
- Saglik Bilimleri Universitesi Gulhane Tip FakultesiUniversity, hospital or other
- Study start
- Apr 2011
- Primary completion
- Apr 2013 (actual)
- Study completion
- Aug 2013 (actual)
- First posted
- 11 Nov 2015 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| once daily dosage schema of colchicine | Experimental | Drug: colchicineThe once daily dosage group was prescribed as once daily at 08:00 a.m. (Total 1mg) |
| twice daily dosage schema of colchicine | Experimental | Drug: colchicineTwice daily dosage group received the treatment twice daily at 08:00 a.m. and 08:00 p.m. (Total 1mg) |
Who may qualify
- Ages
- 5 to 16 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (7 listed)
- Major congenital malformation
- Organ transplantation
- Hepatic disorder
- Chronic kidney disease
- AA amyloidosis
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| colchicineAlso: colchicumto compare once daily dosage schema of colchicine and twice daily dosage schema of colchicine | Drug | once daily dosage schema of colchicine, twice daily dosage schema of colchicine |
Primary outcome
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
Adverse events
Time frame: up to 24 weeks
Adverse Events That Are Related to Treatment
1 secondary outcome
Number of Participants With Abnormal Laboratory Values
Time frame: At baseline and every 12 weeks, up to 24 weeks.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Nov 2015.
- Derived from this study: PubMed 27055417Polat 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.
Related trials for Familial Mediterranean Fever
RecruitingObservational study
Familial Mediterranean Fever and Related Disorders: Genetics and Disease Characteristics
Familial Mediterranean Fever (FMF), Autoinflammation
RecruitingPhase 3
A Study of Apremilast in Children With Oral Ulcers Associated With Behçet's Disease or Juvenile Psoriatic Arthritis
Behçet's Disease, Juvenile Psoriatic Arthritis
Not yet recruitingPhase 3
Goflikicept for Resolution and Prevention of Attacks in Pediatric and Adolescent Patients With Colchicine-Resistant or Colchicine-Intolerant Familial Mediterranean Fever
Familial Mediterranean Fever, FMF
Not yet recruitingObservational study
Genotype and Phenotype in Pediatric Familial Mediterranean Fever
Familial Mediterranean Fever
Not yet recruitingObservational study
Serum EBI-3 Protein Levels in Behçet's Disease
Behçet Disease
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Erkan Demirkaya, MD, MSc principal investigator
Saglik Bilimleri Universitesi Gulhane Tip Fakultesi
Cengizhan Acikel, MD, MPH study chair
Saglik Bilimleri Universitesi Gulhane Tip Fakultesi
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing colchicine in people with Familial Mediterranean Fever, run by Saglik Bilimleri Universitesi Gulhane Tip Fakultesi at 1 site in Turkey (Türkiye).
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 11 Nov 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Turkey (Türkiye). Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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 study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02602028, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 11 Nov 2015.