AzTrials
AzTrials

Cryopyrin-associated Periodic SyndromesPhase 3NCT00991146

Efficacy and Safety Study of Canakinumab Administered for 6 Months (24 Weeks) in Japanese Patients With Cryopyrin-associated Periodic Syndromes Followed by an Extension Phase

A Phase 3 study testing canakinumab in people with Cryopyrin-associated Periodic Syndromes and Familial Cold Autoinflammatory Syndrome, run by Novartis Pharmaceuticals at 3 sites in Japan.

Completed

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

Official record
Search all trials

Trial summary

To date there are no approved effective therapies for the treatment of cryopyrin-associated periodic syndromes (CAPS) including Familial Cold Autoinflammatory Syndrome (FCAS), Muckle-Wells Syndrome (MWS), or Neonatal Onset Multisystem Inflammatory Disease (NOMID) in Japan. Read more about this study

Who is it for?
Ages 2 years and older
What is being tested?
canakinumab
Could I receive a placebo?
No, everyone receives an active treatment
How many people
19 participants
Where
3 sitesJapan
Study dates
Oct 2009to Feb 2012
Phase
Phase 3
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 years and older
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • At study entry, patients should have a clinical diagnosis of FCAS, MWS or NOMID and require medication. At the time of screening, patients can be either untreated or treated with other medication.
  • Presence, or history of at least 2 of the following symptoms:
  • For NOMID patients:
  • Typical NOMID urticarial rash
  • Signs of central nervous system (CNS) involvement such as increased intracranial pressure and/or papilledema and/or cerebral spinal fluid pleiocytosis and/or stroke and/or seizures, and/or sensorineural hearing loss
  • Typical arthropatic changes on X-rays: epiphysal and/or patellar overgrowth With start of NOMID symptoms before or at 6 months of age
Show all 21Show fewer
  • For MWS patients:
  • periodic fever
  • headache/migraine
  • arthralgia
  • urticarial skin rash
  • conjunctivitis
  • myalgia
  • sensorineural hearing impairment
  • For FCAS patients:
  • urticarial skin rash
  • fever/chills
  • conjunctivitis
  • joint pain
  • Patients requiring oral steroids, NSAIDs and/or disease-modifying antirheumatic drugs (DMARDs) can be enrolled if they are on a stable dose (oral steroids: < 20 mg/day or < or = 0.4 mg/kg prednisone or prednisone equivalent, whichever applies) for at least 4 weeks prior to the screening visit.
  • Able to communicate with the investigator and comply with the requirements of the study (for children the parent can assist when necessary).

Who cannot join

  • Pregnant or nursing (lactating) women.
  • All women capable of becoming pregnant unless they are postmenopausal or are using one or more methods of contraception.
  • Participation in any other study within 30 days
  • Infection with HIV, Hepatitis B or C.
  • Live vaccinations within 3 months prior to the start of the trial, during the trial, and up to 3 months following the last dose.
  • History of drug or alcohol abuse within the 12 months prior to dosing.
Show all 31Show fewer
  • Donation or loss of 400 mL or more of blood within 8 weeks prior to dosing for adults.
  • History of significant medical conditions, which in the doctor's opinion would exclude the patient from participating in this trial.
  • History of renal transplantation.
  • Presence of any additional rheumatic diseases or significant systemic diseases. For example, major chronic infectious/ inflammatory/ immunologic disease (such as inflammatory bowel disease, psoriatic arthritis, spondyloarthropathy, systemic lupus erythematosus in addition to the autoinflammatory disease).
  • Presence of any of the following laboratory abnormalities: ALT or AST greater than 2 times the upper limit of normal (ULN), platelet count less than 100x109/L.
  • History of recurrent and/or evidence of clinically significant active bacterial, fungal, or viral infections.
  • History of contact with patients with suspected tuberculosis symptoms; or history or complication of tuberculosis infection.
  • Use of the following therapies:
  • Etanercept in the 4 weeks prior to the baseline visit (Day 1) and thereafter
  • Adalimumab in the 8 weeks prior to the baseline visit (Day 1) and thereafter
  • Infliximab in the 12 weeks prior to the baseline visit (Day 1) and thereafter
  • Rituximab in the 26 weeks prior to the baseline visit (Day 1) and thereafter
  • Tocilizumab in the 3 weeks prior to the baseline visit (Day 1) and thereafter
  • Any other investigational biologics in the 8 weeks prior to the baseline visit (Day 1) and thereafter (with the exception of anakinra therapy-see below)
  • Anakinra therapy after the baseline visit (Day 1). Last anakinra injection should occur not less than 6 hours prior to the canakinumab injection at Day 1
  • Leflunomide in the 4 weeks prior to the baseline visit (Day 1) and thereafter. After the completion of leflunomide treatment a cholestiramine in dose 8 g 3 times per day for 14 days is recommended.
  • Thalidomide in the 4 weeks prior to the baseline visit (Day 1) and thereafter
  • Cyclosporine in the 4 weeks prior to the baseline visit (Day 1) and thereafter
  • i.v. immunoglobulin (i.v. Ig) in the 8 weeks prior to the baseline visit (Day 1) and thereafter
  • 6-Mercaptopurine, azathioprine, cyclophosphamide, or chlorambucil in the 12 weeks prior to the baseline visit (Day 1) and thereafter
  • Dapsone, mycophenolate mofetil in the 3 weeks prior to the baseline visit (Day 1) and thereafter
  • > or = 20 mg/day or >0.4 mg/kg, whichever applies, of prednisone or prednisone equivalent in the 4 week prior to the baseline visit (Day 1) and thereafter
  • Methyl prednisone pulse therapy in the 4 weeks prior to baseline visit and thereafter
  • History of allergic reaction to similar drugs. No additional exclusions may be applied by the doctor, in order to ensure that the study population will be representative of all eligible patients.
  • Other protocol-defined inclusion/exclusion criteria may apply

Trial locations and contacts

3 sites in 1 country.

Japan3 sites

  • Novartis Investigative Site

    Fukuoka, Fukuoka, 812-8582

  • Novartis Investigative Site

    Kyoto, Kyoto, 606-8507

  • Novartis Investigative Site

    Yokohama, Kanagawa, 236-0004

Other trials in Japan

About this study

Official title: An Open-label, Efficacy and Safety Study of Canakinumab (Anti-interleukin-1β Monoclonal Antibody) Administered for 6 Months (24 Weeks) in Japanese Patients With the Following Cryopyrin-associated Periodic Syndromes: Familial Cold Autoinflammatory Syndrome, Muckle-Wells Syndrome, or Neonatal Onset Multisystem Inflammatory Disease, Followed by an Extension Phase to Provide Canakinumab to Study Patients Until it is Approved and Marketed in Japan

To date there are no approved effective therapies for the treatment of cryopyrin-associated periodic syndromes (CAPS) including Familial Cold Autoinflammatory Syndrome (FCAS), Muckle-Wells Syndrome (MWS), or Neonatal Onset Multisystem Inflammatory Disease (NOMID) in Japan.

The study will assess the efficacy and safety of canakinumab in Japanese patients with cryopyrin-associated periodic syndromes (CAPS). In previous and currently ongoing CAPS studies (CACZ885A2102, CACZ885D2201, CACZ885D2304, CACZ885D2306), it has been observed that treatment with canakinumab in patients with CAPS contributed to ensure absence of relapse, to improve signs and symptoms and to prevent secondary disease complications. However, no Japanese patients have been included in those studies. This study will allow access for Japanese patients to a new potentially efficacious treatment for CAPS patients with a convenient dosing regimen.

Being tested
canakinumab
Keywords
CAPS, FCAS, MWS, NOMID, cryopyrin-associated periodic syndromes, Familial Cold Autoinflammatory Syndrome, Muckle-Wells Syndrome, Neonatal Onset Multisystem Inflammatory Disease, canakinumab, ACZ885, children, systemic autoinflammatory disease, NALP-3, human monoclonal anti-human interleukin-1beta (IL-1beta)antibody, autosomal dominant, familial autoinflammatory syndrome
Medical subject headings
Cryopyrin-Associated Periodic Syndromes, Multiple Pterygium Syndrome, Autosomal Dominant

What participants receive

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

  • canakinumabDrug

    canakinumab

Study groups in NCT00991146
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. Number of participants without a relapse

    Measured: 24 weeks

Secondary outcomes (5)

  1. Number of complete responder patients

    Measured: 29 days

  2. Clinical improvement (or resolution) with regards to: central nervous system (CNS) involvement, eye disease, hearing impairment, skin disease, joint disease, fever, and kidney function

    Measured: 24 months

  3. Number of patients experiencing a relapse during the entire study

    Measured: 24 months

  4. How much canakinumab is contained in the patients' blood (pharmacokinetics) and what are the effect of canakinumab on the patients' body (pharmacodynamic)

    Measured: 24 months

  5. Presence of antibody against canakinumab

    Measured: 24 months

Timeline

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

  1. First posted7 Oct 2009 (estimated)
  2. Study startOct 2009
  3. Primary completionFeb 2012
  4. Study 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 3
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
19 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT00991146
Sponsor's protocol ID
CACZ885D2308
Status last verified
Sep 2020
First submitted
6 Oct 2009
Last updated
25 Sep 2020
Results posted
No
FDA-regulated drug
No
FDA-regulated device
No

Publications

  • Imagawa T, Nishikomori R, Takada H, Takeshita S, Patel N, Kim D, Lheritier K, Heike T, Hara T, Yokota S. Safety and efficacy of canakinumab in Japanese patients with phenotypes of cryopyrin-associated periodic syndrome as established in the first open-label, phase-3 pivotal study (24-week results). Clin Exp Rheumatol. 2013 Mar-Apr;31(2):302-9. Epub 2013 Feb 1.

    PubMed 23380020

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing canakinumab in people with Cryopyrin-associated Periodic Syndromes and Familial Cold Autoinflammatory Syndrome, run by Novartis Pharmaceuticals at 3 sites in Japan.

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 21 inclusion and 31 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 25 Sep 2020. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 3 sites in Japan. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

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?

Number of participants without a relapse (time frame: 24 weeks). 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 NCT00991146, which also lists 1 linked publication.

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 25 Sep 2020.

AzTrials uses cookies for analytics and advertising. You can accept or reject them; the site works either way. Privacy Policy.

Free account

Log in to keep searching

You’ve used your 3 free searches. AzTrials is free: log in or create a free account to keep searching clinical trials and save the ones you want to follow.

Create a free accountLog in