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Controlled Ceasing of Colchicine Therapy in Familial Mediterranean Fever (FMF) Patients With Single MEFV (Mediterranean Fever) Gene Mutation

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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
Sponsor:
Collaborator:
Schneider Children's Hospital
Information provided by (Responsible Party):
yonatan butbul MD, Rambam Health Care Campus

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.

Condition or disease Intervention/treatment Phase
Familial Mediterranean Fever Other: Colchicine Cessation Phase 2

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 80 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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


Arm Intervention/treatment
Study group
Colchicine Cessation in FMF patients with one MEFV mutation
Other: Colchicine Cessation
Colchicine Cessation

No Intervention: Control group
The control group includes FMF patients that will be kept on a daily colchicine treatment



Primary Outcome Measures :
  1. 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


Secondary Outcome Measures :
  1. 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



Information from the National Library of Medicine

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Ages Eligible for Study:   2 Years to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Patients diagnosed with FMF based on clinical criteria
  • FMF patients diagnosed of having at least one common MEFV mutation will be assigned to the study group. FMF patients who staid on colchicine treatment will be assigned to the control group, regardless of their genotype.
  • Patients who were on a continuous colchicine prophylactic treatment for six months prior to entering the study.
  • FMF patients who were free of acute FMF symptoms for six months prior to entering th study
  • Patients were included in the study only if they had normal serum level of SAA (up to 10 mg / l).

Exclusion Criteria:

  • Patients that in the six months prior to entering the study continued to have classic FMF episodes despite being on a continuous prophylactic colchicine
  • Patients that had high level of SAA (above 10 mg/l) despite being on prophylactic colchicine treatment

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): NCT02175589


Locations
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Israel
Pediatric rheumatology clinic, Rambam Medical Center
Haifa, Israel
Schneider children's hospital
Petach Tikva, Israel
Sponsors and Collaborators
Rambam Health Care Campus
Schneider Children's Hospital
Investigators
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Study Chair: Yonatan Butbul, MD Rambam Health Care Campus
Principal Investigator: Riva Brik, MD Rambam Health Care Campus
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Responsible Party: yonatan butbul MD, Senior pediatrician & pediatric rheumatologist, Rambam Health Care Campus
ClinicalTrials.gov Identifier: NCT02175589    
Other Study ID Numbers: 0080-14-RMB
First Posted: June 26, 2014    Key Record Dates
Last Update Posted: June 26, 2014
Last Verified: June 2014
Keywords provided by yonatan butbul MD, Rambam Health Care Campus:
Familial Mediterranean Fever
Monozygous
MEFV
Colchicine
Serum Amyloid A
Additional relevant MeSH terms:
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Brucellosis
Familial Mediterranean Fever
Hereditary Autoinflammatory Diseases
Fever
Body Temperature Changes
Signs and Symptoms
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