Ignite Creation Date:
2024-05-06 @ 2:58 AM
Last Modification Date:
2024-10-26 @ 11:26 AM
Study NCT ID:
NCT02175589
Status:
UNKNOWN
Last Update Posted:
2014-06-26
First Post:
2014-06-24
Brief Title:
Controlled Ceasing of Colchicine Therapy in Familial Mediterranean Fever FMF Patients With Single MEFV Mediterranean Fever Gene Mutation
Sponsor:
Rambam Health Care Campus
Organization:
Rambam Health Care Campus
Study Overview
Official Title:
Controlled Ceasing of Colchicine Therapy in Familial Mediterranean Fever FMF Patients With Single MEFV Mediterranean Fever Gene Mutation
Status:
UNKNOWN
Status Verified Date:
2014-06
Last Known Status:
ENROLLING_BY_INVITATION
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
The purpose of this study is to evaluate the effect of discontinuation of colchicine treatment in a specific group of asymptomatic FMF patients with a single mutation in MEFV gene both from a clinical and laboratory aspects
Detailed Description:
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
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 Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None