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

NCT02175589 | Unknown Phase 2

• 1 other identifier: 0080-14-RMB
• Study Type: interventional
• Target: 80
• Locations: 1 country
• Sites: 2

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.

Conditions

Familial Mediterranean Fever

Keywords

Familial Mediterranean Fever; Monozygous; MEFV; Colchicine; Serum Amyloid A

Outcome Measures

Primary Outcomes (1)

• Acute clinical episode of FMF

  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

  6 months

Secondary Outcomes (1)

• High level of Serum Amyloid A (SAA) in serum

  6 months

Study Arms (2)

Study group

OTHER

Colchicine Cessation in FMF patients with one MEFV mutation

Other: Colchicine Cessation

Control group

NO INTERVENTION

The control group includes FMF patients that will be kept on a daily colchicine treatment

Interventions

Colchicine Cessation OTHER

Colchicine Cessation

Study group

Eligibility Criteria

• Age: 2 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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).

You may not qualify if:

• 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

Sponsors & Collaborators

• Rambam Health Care Campus: lead
• Schneider Children's Hospital: collaborator

Study Sites (2)

Pediatric rheumatology clinic, Rambam Medical Center

Haifa, Israel

Location

Schneider children's hospital

Petah Tikva, Israel

Location

MeSH Terms

Conditions

Familial Mediterranean Fever

Condition Hierarchy (Ancestors)

Hereditary Autoinflammatory Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Yonatan Butbul, MD

  Rambam Health Care Campus

  STUDY CHAIR

• Riva Brik, MD

  Rambam Health Care Campus

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Senior pediatrician & pediatric rheumatologist

Study Record Dates

• First Submitted: June 24, 2014
• First Posted: June 26, 2014
• Study Start: June 1, 2014
• Primary Completion: January 1, 2015
• Study Completion: January 1, 2015
• Last Updated: June 26, 2014

Record last verified: 2014-06

Locations

IL (2)