NCT00991146

Brief 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. 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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
19

participants targeted

Target at below P25 for phase_3

Timeline
Completed

Started Oct 2009

Geographic Reach
1 country

3 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

October 1, 2009

Completed
5 days until next milestone

First Submitted

Initial submission to the registry

October 6, 2009

Completed
1 day until next milestone

First Posted

Study publicly available on registry

October 7, 2009

Completed
2.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

February 1, 2012

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

February 1, 2012

Completed
Last Updated

September 25, 2020

Status Verified

September 1, 2020

Enrollment Period

2.3 years

First QC Date

October 6, 2009

Last Update Submit

September 23, 2020

Conditions

Cryopyrin-associated Periodic SyndromesFamilial Cold Autoinflammatory SyndromeMuckle-Wells SyndromeNeonatal Onset Multisystem Inflammatory Disease

Keywords

CAPSFCASMWSNOMIDcryopyrin-associated periodic syndromesFamilial Cold Autoinflammatory SyndromeMuckle-Wells SyndromeNeonatal Onset Multisystem Inflammatory DiseasecanakinumabACZ885childrensystemic autoinflammatory diseaseNALP-3human monoclonal anti-human interleukin-1beta (IL-1beta)antibodyautosomal dominantfamilial autoinflammatory syndrome

Outcome Measures

Primary Outcomes (1)

  • Number of participants without a relapse

    24 weeks

Secondary Outcomes (5)

  • Number of complete responder patients

    29 days

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

    24 months

  • Number of patients experiencing a relapse during the entire study

    24 months

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

    24 months

  • Presence of antibody against canakinumab

    24 months

Study Arms (1)

canakinumab

EXPERIMENTAL
Drug: canakinumab

Interventions

canakinumabDRUG

canakinumab

canakinumab

Eligibility Criteria

Age2 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • 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
  • For MWS patients:
  • periodic fever
  • headache/migraine
  • arthralgia
  • urticarial skin rash
  • conjunctivitis
  • myalgia
  • sensorineural hearing impairment
  • For FCAS patients:
  • +6 more criteria

You may not qualify if:

  • 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.
  • 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
  • +14 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (3)

Novartis Investigative Site

Fukuoka, Fukuoka, 812-8582, Japan

Location

Novartis Investigative Site

Yokohama, Kanagawa, 236-0004, Japan

Location

Novartis Investigative Site

Kyoto, Kyoto, 606-8507, Japan

Location

Related Publications (1)

  • 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.

    PMID: 23380020RESULT

MeSH Terms

Conditions

Cryopyrin-Associated Periodic SyndromesMultiple Pterygium Syndrome, Autosomal Dominant

Interventions

canakinumab

Condition Hierarchy (Ancestors)

Hereditary Autoinflammatory DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesSkin Diseases, GeneticSkin DiseasesSkin and Connective Tissue DiseasesChronic Inducible UrticariaChronic UrticariaUrticariaSkin Diseases, VascularCold UrticariaHypersensitivity, ImmediateHypersensitivityImmune System DiseasesChronic DiseaseDisease AttributesPathologic ProcessesPathological Conditions, Signs and Symptoms

Study Officials

  • Novartis Pharmaceuticals

    Novartis Pharmaceuticals

    STUDY DIRECTOR

Study Design

Study Type
interventional
Phase
phase 3
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

October 6, 2009

First Posted

October 7, 2009

Study Start

October 1, 2009

Primary Completion

February 1, 2012

Study Completion

February 1, 2012

Last Updated

September 25, 2020

Record last verified: 2020-09

Locations

JP(3)