Controlled Ceasing of Colchicine Therapy in Familial Mediterranean Fever (FMF) Patients With Single MEFV (Mediterranean Fever) Gene Mutation
|ClinicalTrials.gov Identifier: NCT02175589|
Recruitment Status : Unknown
Verified June 2014 by yonatan butbul MD, Rambam Health Care Campus.
Recruitment status was: Enrolling by invitation
First Posted : June 26, 2014
Last Update Posted : June 26, 2014
|Condition or disease||Intervention/treatment||Phase|
|Familial Mediterranean Fever||Other: Colchicine Cessation||Phase 2|
The diagnosis of FMF is mainly clinical and genetic tests are only used to confirm the diagnosis . Even though the disease is autosomal recessive, not all FMF patients have two recognizable MEFV mutations. The phenotype of FMF patients varies according to the genotype, as shown by a number of studies showing that patients with one MEFV mutation have milder disease or even no symptoms. Some of the previously mentioned studies have shown that ceasing colchicine prophylaxis in these patients caused no recurrence. So far, no prospective controlled study has tested the effect of colchicine cessation in this group of FMF patients. The investigators presume that asymptomatic FMF patients with a single mutation can stop regular colchicine treatment while remaining under close follow-up.
The purpose of the work:
To examine the effect of colchicine cessation in a defined group of asymptomatic FMF patients with a single mutation in MEFV gene.
Methods and study population:
The work will be a controlled prospective comparative study including FMF patients aged 2-18 years. Patients included will be those who were asymptomatic for six months prior to entering the study and were regularly treated with colchicine, and with a normal serum level of Serum Amyloid A (SAA). The study group will include patients with a single MEFV mutation that will stop colchicine therapy, and the control group will include FMF who will continue regular colchicine treatment. Follow-up in both groups will include clinical and laboratory (serum SAA levels) evaluation.
The study end points and renewal of the colchicine:
Any patient that develops acute symptoms of FMF will be immediately invited to the rheumatology clinic for medical examination. In addition, patients will be invited to the clinic after 3 and 6 months from the beginning of the study. At any clinic visit (scheduled or not) the patients will be assessed clinically and laboratory (serum SAA levels). The study will be stopped and colchicine will be renewed if at any of the above mentioned clinic visit the patient will be diagnosed as having a classic FMF attack or the SAA level will be above 10 mg / l.
The importance of the study:
If the investigators conclude that colchicine prophylaxis can be safely discontinued in this group of FMF patients this will save them a treatment currently defined as a treatment for life.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||80 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||None (Open Label)|
|Official Title:||Controlled Ceasing of Colchicine Therapy in Familial Mediterranean Fever (FMF) Patients With Single MEFV (Mediterranean Fever) Gene Mutation|
|Study Start Date :||June 2014|
|Estimated Primary Completion Date :||January 2015|
|Estimated Study Completion Date :||January 2015|
Colchicine Cessation in FMF patients with one MEFV mutation
Other: Colchicine Cessation
No Intervention: Control group
The control group includes FMF patients that will be kept on a daily colchicine treatment
- Acute clinical episode of FMF [ Time Frame: 6 months ]Acute clinical episode of FMF diagnosed by one of the investigators at any clinic visit assigned at 3 or 6 months after the cessation of colchicine treatment or at an unassigned visit if the patient attained the clinic due to an acute symptoms of FMF
- High level of Serum Amyloid A (SAA) in serum [ Time Frame: 6 months ]High level of SAA (above 10 mg/l) at any clinic visit assigned at 3 or 6 months after the cessation of colchicine treatment or at an unassigned visit if the patient attained the clinic due to an acute symptoms of FMF
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02175589
|Pediatric rheumatology clinic, Rambam Medical Center|
|Schneider children's hospital|
|Petach Tikva, Israel|
|Study Chair:||Yonatan Butbul, MD||Rambam Health Care Campus|
|Principal Investigator:||Riva Brik, MD||Rambam Health Care Campus|