Rilonacept for Treatment of Familial Mediterranean Fever (FMF)

• Register: ClinicalTrials.gov
• Last refreshed on: 19 October 2017
• Main ID: NCT00582907
• Date of registration: 19/12/2007
• Prospective Registration: Yes
• Primary sponsor: The Cleveland Clinic
• Public title: Rilonacept for Treatment of Familial Mediterranean Fever (FMF)
• Scientific title: Phase 2 Study of IL-1 Trap (Rilonacept) for Treatment of Familial Mediterranean Fever (FMF)
• Date of first enrolment: August 2008
• Target sample size: 14
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT00582907
• Study type: Interventional
• Phase: Phase 2

Countries of recruitment

United States

Contacts

• Name: Philip J Hashkes, MD, MSc
• Affiliation: Shaare Zedek Medical Center/The Cleveland Clinic Foundation

Key inclusion & exclusion criteria

Inclusion Criteria:

- Subject has a definitive diagnosis of FMF as by the Tel-Hashomer clinical criteria
(long version of criteria) with at least one mutation on one of the MEFV gene alleles.
However, subjects with an isolated heterozygous mutation of exon 2 of the MEFV gene
(including E148Q) will not be eligible.

- Subject must have an estimated mean of at least one acute FMF attack per month before
and during the month of screening.

- Subject is at least four years of age (with no upper limit of age).

- Subjects must have received an adequate trial of colchicine defined as treatment of at
least 1.5 mg/d for at least 3 months if =6 years old or 1.2 mg/d if less than 6 years,
or an inability to tolerate colchicine due to adverse effects in a dose that controls
acute attacks in the frequency of less than one attack per month.

- If subject is being treated with anakinra at the time of consent, washout must be done
(about 3 days). Subject must experience 2 attacks before randomization visit can
occur.

- If subject has been treated previously with anti-TNF drugs, appropriate washout must
be done. Etanercept must be discontinued for 4 weeks prior to randomization;
Adalimumab and Infliximab must be discontinued for 8 weeks prior to randomization.

- If subject is a female of childbearing potential, she must agree to use adequate
contraception (adequate contraception can include abstinence) for the duration of the
trial and 3 months after and must have a negative serum or urine pregnancy test prior
to administration of study medication.

- If subject is a male and has reached puberty, he must agree to use adequate
contraception or abstinence during the study and for 3 months after discontinuation
from study.

- Subject's parent or legal guardian has provided written informed consent prior to
screening for this study or if subject is older than 18 years has provided informed
consent him/herself.

- Subject, if applicable, has assented to participate prior to screening for this study.

- Subject and, if applicable, parent/legal guardian, agree to comply with study
requirements and are able to come to the clinic for all required study visits.

Exclusion Criteria:

- The subject has existing biopsy proven amyloidosis or proteinuria >0.5 gram per day.

- The subject has another active inflammatory rheumatic disease.

- The subject has an active malignancy of any type, or history of a malignancy.

- The subject has active GI disease (e.g., inflammatory bowel disease), a chronic or
acute renal or hepatic disorder, or a significant coagulation defect.

- The subject has an AST (SGOT), ALT (SGPT) or BUN >2 x ULN or creatinine >1.5 mg/dL or
any other laboratory abnormality considered by the examining physician to be
clinically significant within 28 days before the Baseline visit.

- Current use of an anti-tumor necrosis factor drug.

- The subject has, in the investigator's opinion, a chronic condition (e.g., diabetes,
epilepsy) that is either not stable or well-controlled and may interfere with the
conduct of the study.

- The subject has received any investigational medication within 30 days before the
first dose of study medication or is scheduled to receive an investigational drug,
other than study medications described in this protocol, during the course of the
study.

- The subject has chronic or active infection or any major episode of infection
requiring hospitalization or treatment with i.v. antibiotics within 30 days or oral
antibiotics within 14 days prior to the screening evaluation.

- The subject has known positive human immunodeficiency virus (HIV) status.

- The subject has known past or current hepatitis.

- The subject has received a live virus vaccine within 1 month prior to the baseline
visit.

- The subject has a positive PPD test.

- The subject is sexually active and not practicing effective birth control.

- The subject is pregnant or breast feeding a child.

- Any concurrent medical condition which would, in the investigator's opinion,
compromise the subject's ability to tolerate the study drug or would make the subject
unable to cooperate with the protocol.

- History of/or current psychiatric illness that would interfere with ability to comply
with protocol requirements or give informed consent.

- Subject has a history of alcohol or drug abuse within the past 6 months that would
interfere with ability to comply with protocol requirements.

- Inability to comply with the study requirements for any reason.



Age minimum: 4 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Familial Mediterranean Fever

Intervention(s)

Drug: Rilonacept

Drug: Placebo

Primary Outcome(s)

To Assess the Efficacy of Rilonacept in Decreasing the Number of Acute FMF Attacks. [Time Frame: attacks were assessed at the end of each 3 month treatment course (overall up to 6 month of rilonacept and 6 months of placebo, each)]

To Determine if There is a Medically Important Difference Between the Safety Profiles of Rilonacept vs. Placebo. [Time Frame: 12 months of entire study length]

Secondary Outcome(s)

To Determine the Difference in the Length of Attacks During Treatment With Rilonacept vs. Placebo. [Time Frame: 12 months]

To Determine the Differences in the Erythrocyte Sedimentation Rate Between the Treatment Arms (Rilonacept vs. Placebo). [Time Frame: 3 months (each treatment course, overall 12 months)]

To Determine the Differences in Serum Amyloid A Levels Between the Treatment Arms (Rilonacept vs. Placebo) [Time Frame: 3 months (each treatment course, overall 12 months)]

To Determine the Differences in the Fibrinogen Levels Between the Treatment Arms (Rilonacept vs. Placebo) [Time Frame: 3 months (each treatment course, overall 12 months)]

To Determine the Differences in the Quality of Life Between the Treatment Arms (Rilonacept vs. Placebo). [Time Frame: 12 months]

To Determine the Differences in the FMF Severity Score of the Subjects Between the Treatment Arms (Rilonacept vs. Placebo). [Time Frame: overall 12 months]

To Determine Differences in the Time to the Development of Attacks Between the Treatment Arms (Rilonacept vs. Placebo). [Time Frame: 3 months]

To Determine the Differences in C-Reactive Protein Between the Treatment Arms (Rilonacept vs. Placebo) [Time Frame: 3 months (each treatment course, overall 12 months)]

To Determine the Proportion of Courses in Which Subjects Attained at Least a 50% Decrease in Acute FMF Attacks During Rilonacept Courses as Compared to Placebo Courses. [Time Frame: Up to 3 months for each treatment course]

To Determine the Differences in the Proportion of Time Subjects Received Rilonacept vs Placebo [Time Frame: 12 months]

Percentage of Treatment Courses Without FMF Attacks in Rilonacept Courses as Compared to Placebo Courses. [Time Frame: Each treatment course of up to 3 months]

To Determine the Differences in the Platelet Count Between the Treatment Arms (Rilonacept vs. Placebo) [Time Frame: 3 months (each treatment course, overall 12 months)]

Secondary ID(s)

1R01FD003435-01

FDA 1RO1FD003435-01

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 31/10/2012
• URL: https://clinicaltrials.gov/ct2/show/results/NCT00582907