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Colchicine Resistant/Intolerant Familial Mediterranean FeverPhase 2CONTROL FMFNCT01148797

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

A Phase 2 study testing Canakinumab in people with Colchicine Resistant/Intolerant Familial Mediterranean Fever, run by Novartis Pharmaceuticals at 5 sites in Israel.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

A study designed to evaluate the role of treatment with a biological agent - Canakinumab in pediatric (age 4-20) Familial Mediterranean Fever (FMF) patients that are intolerant or resistant for colchicine treatment. The study hypothesis is that Canakinumab will reduce attack frequency and severity. Read more about this study

Who is it for?
Ages 4 to 20 years
What is being tested?
Canakinumab
Could I receive a placebo?
No, everyone receives an active treatment
How many people
15 participants
Where
5 sitesIsrael
Study dates
Dec 2010to Feb 2012
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 4 to 20 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • 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).
  • 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.
  • 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.
  • 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)
  • 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
  • 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.
  • 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.

Who cannot join

  • Patients with end-organ dysfunction due to amyloidosis (e.g. existing biopsy proven amyloidosis or proteinuria > 0.5 gram per day)
  • 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.
  • Presence or history of any other inflammatory rheumatic disease
  • 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.
  • 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.
  • 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.
Show all 16Show fewer
  • The subject has positive human immunodeficiency virus (HIV) status or current (acute or chronic) hepatitis B or C
  • Subjects who are pregnant or lactating
  • 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
  • Malignancy, except for successfully excised squamous or basal cell carcinoma of the skin
  • 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 received a live virus vaccine within 3 months prior to the baseline visit.
  • 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.
  • History of/or current psychiatric illness that would interfere with ability to comply with protocol requirements or provide 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.
  • Other protocol-defined inclusion/exclusion criteria may apply

Trial locations and contacts

5 sites in 1 country.

5 sites in 1 country

Israel5 sites

  • Rambam Health Care Campus

    Haifa

  • Shaare Zedek Medical Center

    Jerusalem

  • Meir Medical Center Kfar Saba

    Kfar Saba

  • Rabin Medical Center

    Petah Tikva

  • The Chaim Sheba Medical Center

    Ramat Gan

Other trials in Israel

About this study

Official 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

A study designed to evaluate the role of treatment with a biological agent - Canakinumab in pediatric (age 4-20) Familial Mediterranean Fever (FMF) patients that are intolerant or resistant for colchicine treatment.

The study hypothesis is that Canakinumab will reduce attack frequency and severity.

Being tested
Canakinumab
Keywords
FMF, Colchicine resistant, Colchicine intolerant, Flare rate, Canakinumab

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • CanakinumabDrug

Study groups in NCT01148797
Study groupRoleWhat they receive
CanakinumabExperimentalNot described
Receives: Drug: Canakinumab

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. 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

    Measured: 0-3 months

Secondary outcomes (3)

  1. To assess the effect of canakinumab with regard to percentage of subjects with no attacks during the 3 months treatment period

    Measured: 0-3 months

  2. To evaluate the safety and tolerability of canakinumab by monitoring adverse events (AEs) and subject discontinuations due to an AE

    Measured: 3 months

  3. To assess the change in frequency of FMF attacks during the treatment period

    Measured: 3 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted22 Jun 2010 (estimated)
  2. Study startDec 2010
  3. Primary completionFeb 2012

Resources

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
Single group
Masking
None (open label)
Enrolment
15 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01148797
Sponsor's protocol ID
CACZ885D2204
Acronym
CONTROL FMF
Status last verified
Nov 2016
First submitted
21 Jun 2010
Last updated
18 Nov 2016
Results posted
No

Publications

  • Lidar M, Livneh A. Familial Mediterranean fever: clinical, molecular and management advancements. Neth J Med. 2007 Oct;65(9):318-24.

    PubMed 17954950

  • Bhat A, Naguwa SM, Gershwin ME. Genetics and new treatment modalities for familial Mediterranean fever. Ann N Y Acad Sci. 2007 Sep;1110:201-8. doi: 10.1196/annals.1423.022.

    PubMed 17911435

  • Mor A, Shinar Y, Zaks N, Langevitz P, Chetrit A, Shtrasburg S, Rabinovitz E, Livneh A. Evaluation of disease severity in familial Mediterranean fever. Semin Arthritis Rheum. 2005 Aug;35(1):57-64. doi: 10.1016/j.semarthrit.2005.02.002.

    PubMed 16084225

  • Masters SL, Simon A, Aksentijevich I, Kastner DL. Horror autoinflammaticus: the molecular pathophysiology of autoinflammatory disease (*). Annu Rev Immunol. 2009;27:621-68. doi: 10.1146/annurev.immunol.25.022106.141627.

    PubMed 19302049

  • Soylemezoglu O, Arga M, Fidan K, Gonen S, Emeksiz HC, Hasanoglu E, Buyan N. Unresponsiveness to colchicine therapy in patients with familial Mediterranean fever homozygous for the M694V mutation. J Rheumatol. 2010 Jan;37(1):182-9. doi: 10.3899/jrheum.090273. Epub 2009 Dec 15.

    PubMed 20008920

  • Roldan R, Ruiz AM, Miranda MD, Collantes E. Anakinra: new therapeutic approach in children with Familial Mediterranean Fever resistant to colchicine. Joint Bone Spine. 2008 Jul;75(4):504-5. doi: 10.1016/j.jbspin.2008.04.001. Epub 2008 Jun 9.

    PubMed 18541452

  • Kuijk LM, Govers AM, Frenkel J, Hofhuis WJ. Effective treatment of a colchicine-resistant familial Mediterranean fever patient with anakinra. Ann Rheum Dis. 2007 Nov;66(11):1545-6. doi: 10.1136/ard.2007.071498. No abstract available.

    PubMed 17934085

  • Moser C, Pohl G, Haslinger I, Knapp S, Rowczenio D, Russel T, Lachmann HJ, Lang U, Kovarik J. Successful treatment of familial Mediterranean fever with Anakinra and outcome after renal transplantation. Nephrol Dial Transplant. 2009 Feb;24(2):676-8. doi: 10.1093/ndt/gfn646. Epub 2008 Nov 25.

    PubMed 19033248

  • Lachmann HJ, Kone-Paut I, Kuemmerle-Deschner JB, Leslie KS, Hachulla E, Quartier P, Gitton X, Widmer A, Patel N, Hawkins PN; Canakinumab in CAPS Study Group. Use of canakinumab in the cryopyrin-associated periodic syndrome. N Engl J Med. 2009 Jun 4;360(23):2416-25. doi: 10.1056/NEJMoa0810787.

    PubMed 19494217

  • Manuel O, Kumar D. QuantiFERON-TB Gold assay for the diagnosis of latent tuberculosis infection. Expert Rev Mol Diagn. 2008 May;8(3):247-56. doi: 10.1586/14737159.8.3.247.

    PubMed 18598104

  • Brik R, Butbul-Aviel Y, Lubin S, Ben Dayan E, Rachmilewitz-Minei T, Tseng L, Hashkes PJ. Canakinumab for the treatment of children with colchicine-resistant familial Mediterranean fever: a 6-month open-label, single-arm pilot study. Arthritis Rheumatol. 2014 Nov;66(11):3241-3. doi: 10.1002/art.38777. No abstract available.

    PubMed 25049046

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Canakinumab in people with Colchicine Resistant/Intolerant Familial Mediterranean Fever, run by Novartis Pharmaceuticals at 5 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 7 inclusion and 16 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: finished, as of the sponsor's last update on 18 Nov 2016. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 5 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?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

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?

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). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01148797, which also lists 11 linked publications.

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 18 Nov 2016.

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