Familial Mediterranean FeverPhase 2NCT02175589
Controlled Ceasing of Colchicine Therapy in Familial Mediterranean Fever (FMF) Patients With Single MEFV (Mediterranean Fever) Gene Mutation
A Phase 2 study testing Colchicine Cessation in people with Familial Mediterranean Fever, run by Rambam Health Care Campus at 2 sites in Israel.
Trial 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. Read more about this study
- Who is it for?
- Ages 2 to 18 years
- Conditions the study is for
- Familial Mediterranean Fever
- What is being tested?
- Colchicine Cessation
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 80 participants
- Where
- 2 sitesIsrael
- Study dates
- Jun 2014to Jan 2015
- Phase
- Phase 2
- 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 2 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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).
Who cannot join
- 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
Trial locations and contacts
2 sites in 1 country.
Israel2 sites
Pediatric rheumatology clinic, Rambam Medical Center
Haifa
Schneider children's hospital
Petah Tikva
About this study
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.
Read the 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.
- Conditions
- Familial Mediterranean Fever
- Being tested
- Colchicine Cessation
- Keywords
- Familial Mediterranean Fever, Monozygous, MEFV, Colchicine, Serum Amyloid A
- 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.
Colchicine CessationOther
Colchicine Cessation
| Study group | Role | What they receive |
|---|---|---|
| Study group | Other | Colchicine Cessation in FMF patients with one MEFV mutation Receives: Other: Colchicine Cessation |
| Control group | No intervention | The control group includes FMF patients that will be kept on a daily colchicine treatment |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 6 months
Secondary outcomes (1)
High level of Serum Amyloid A (SAA) in serum
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
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Jun 2014 (estimated)
- Study startJun 2014
- Primary completionJan 2015 (estimated)
- Study completionJan 2015 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 80 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Rambam Health Care Campus
- Sponsor type
- University, hospital or other
- Collaborators
- Schneider Children's Hospital
- Responsible party
- Principal investigator, yonatan butbul MD, Rambam Health Care Campus
- Investigators
- Yonatan Butbul, MD, Study chair, Rambam Health Care CampusRiva Brik, MD, Principal investigator, Rambam Health Care Campus
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02175589
- Sponsor's protocol ID
- 0080-14-RMB
- Status last verified
- Jun 2014
- First submitted
- 24 Jun 2014
- Last updated
- 26 Jun 2014
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 26 Jun 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Controlled Ceasing of Colchicine Therapy in Familial Mediterranean Fever (FMF) Patients With Single MEFV (Mediterranean Fever) Gene Mutation
- ClinicalTrials.gov ID
- NCT02175589
- Acronym / sponsor ID
- 0080-14-RMB
- Conditions
- Familial Mediterranean Fever
- Keywords
- Familial Mediterranean Fever, Monozygous, MEFV, Colchicine, Serum Amyloid A
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 80 participants (estimated)
- Lead sponsor
- Rambam Health Care CampusUniversity, hospital or other
- Collaborators
- Schneider Children's Hospital
- Study start
- Jun 2014
- Primary completion
- Jan 2015 (estimated)
- Study completion
- Jan 2015 (estimated)
- First posted
- 26 Jun 2014 (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.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Study group | Other | Other: Colchicine CessationColchicine Cessation in FMF patients with one MEFV mutation |
| Control group | No intervention | –The control group includes FMF patients that will be kept on a daily colchicine treatment |
Who may qualify
- Ages
- 2 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (5 listed)
- 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).
Key exclusion criteria (2 listed)
- 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
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 |
|---|---|---|
| Colchicine CessationColchicine Cessation | Other | Study group |
Primary outcome
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
1 secondary outcome
High level of Serum Amyloid A (SAA) in serum
Time frame: 6 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Jun 2014.
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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.
Yonatan Butbul, MD study chair
Rambam Health Care Campus
Riva Brik, MD principal investigator
Rambam Health Care Campus
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Colchicine Cessation in people with Familial Mediterranean Fever, run by Rambam Health Care Campus at 2 sites in Israel.
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 5 inclusion and 2 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: status not confirmed, as of the sponsor's last update on 26 Jun 2014. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Israel. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
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?
Acute clinical episode of FMF (time frame: 6 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 26 Jun 2014.