Evaluate the Safety and Efficacy of Canakinumab in Pediatric Patients With Colchicine Intolerant or Colchicine Resistant Familial Mediterranean Fever (FMF)

• Register: ClinicalTrials.gov
• Last refreshed on: 28 November 2016
• Main ID: NCT01148797
• Date of registration: 21/06/2010
• Prospective Registration: Yes
• Primary sponsor: Novartis Pharmaceuticals
• Public title: Evaluate the Safety and Efficacy of Canakinumab in Pediatric Patients With Colchicine Intolerant or Colchicine Resistant Familial Mediterranean Fever (FMF) CONTROL FMF
• Scientific title: A 6 Month Phase 2, Multi-Center, Open-label, Single Arm Study to Evaluate the Safety and Efficacy of Treatment With Canakinumab in Pediatric Patients With Colchicine Intolerant or Colchicine Resistant Familial Mediterranean Fever
• Date of first enrolment: December 2010
• Target sample size: 15
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01148797
• Study type: Interventional
• Study design: Allocation: Non-Randomized, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
• Phase: Phase 2

Countries of recruitment

Israel

Contacts

• Name: Eliad Ben Dayan
• Affiliation: Novartis Pharmaceuticals

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Male and female subjects between 4 and 20 years of age with active type 1 FMF disease
(according to Tel-Hashomer Long criteria for diagnosis of FMF) and genetic
confirmation of diagnosis (for the study - genetic confirmation is defined as either
homozygous or compound heterozygous).

2. Subjects must have type 1 disease characterized by recurrent and short episodes of
inflammation and serositis, with an average of at least 3 well documented acute FMF
attacks during the previous 3 months that are confirmed by the treating physician,
lasting less then a week, and a minimum 14 day- attack free interval between attacks.

3. Subjects must have received an adequate trial of colchicine, defined as treatment of
at least 1-2 mg/d (based on subjects age) for at least 3 months, 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.

4. Subjects treated with anti-IL-1 therapies must complete washout and have experienced
at least 2 attacks since (e.g. Anakinra: 3 day washout; Rilonacept: 4 week washout)

5. Subjects treated with anti-TNF drugs must undergo appropriate washout. Prior to
randomization, use of Etanercept must be discontinued for 4 weeks or use of
Adalimumab or Infliximab must be discontinued for 8 weeks - a full list of washout
periods for current treatments will be supplied

6. 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 each dose of study medication.

7. 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.

Exclusion Criteria:

1. Patients with end-organ dysfunction due to amyloidosis (e.g. existing biopsy proven
amyloidosis or proteinuria > 0.5 gram per day)

2. Subjects taking oral or IV steroids within 1 month prior to baseline. Subjects taking
steroids for reasons other than FMF - may be enrolled into the study based on
discussion with the investigator and sponsor.

3. Presence or history of any other inflammatory rheumatic disease

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

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

6. Positive PPD test (according to local guidance) where a latent or active TB infection
cannot be excluded via QuantiFERON (T-Spot or radiographic imaging if needed) or via
Chest x-ray.

7. The subject has positive human immunodeficiency virus (HIV) status or current (acute
or chronic) hepatitis B or C

8. Subjects who are pregnant or lactating

9. Presence of any active or chronic 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 screening

10. Malignancy, except for successfully excised squamous or basal cell carcinoma of the
skin

11. 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.

12. The subject has received a live virus vaccine within 3 months prior to the baseline
visit.

13. 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 follow with the protocol.

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

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

Other protocol-defined inclusion/exclusion criteria may apply



Age minimum: 4 Years
Age maximum: 20 Years
Gender: Both

Health Condition(s) or Problem(s) studied

Colchicine Resistant/Intolerant Familial Mediterranean Fever

Intervention(s)

Drug: Canakinumab

Primary Outcome(s)

To measure the effect of canakinumab on the frequency of FMF attacks, defined as percentage of subjects with at least 50% reduction in the attack frequency during a 3 month treatment period [Time Frame: 0-3 months]

Secondary Outcome(s)

To evaluate the safety and tolerability of canakinumab by monitoring adverse events (AEs) and subject discontinuations due to an AE [Time Frame: 3 months]

To assess the effect of canakinumab with regard to percentage of subjects with no attacks during the 3 months treatment period [Time Frame: 0-3 months]

To assess the change in frequency of FMF attacks during the treatment period [Time Frame: 3 months]

Secondary ID(s)

CACZ885D2204

Source(s) of Monetary Support

Please refer to primary and secondary sponsors