Efficacy and Safety of ACZ885 in Patients With the Following Cryopyrin-associated Periodic Syndromes: Familial Cold Autoinflammatory Syndrome, Muckle-Wells Syndrome, or Neonatal Onset Multisystem Inflammatory Disease

• Register: ClinicalTrials.gov
• Last refreshed on: 19 October 2017
• Main ID: NCT00685373
• Date of registration: 27/05/2008
• Prospective Registration: No
• Primary sponsor: Novartis
• Public title: Efficacy and Safety of ACZ885 in Patients With the Following Cryopyrin-associated Periodic Syndromes: Familial Cold Autoinflammatory Syndrome, Muckle-Wells Syndrome, or Neonatal Onset Multisystem Inflammatory Disease
• Scientific title: An Open-label, Long-term Safety and Efficacy Study of ACZ885 (Anti-interleukin-1ß Monoclonal Antibody) Administered for at Least 6 Months in Patients With the Following Cryopyrin-associated Periodic Syndromes: Familial Cold Autoinflammatory Syndrome, Muckle-Wells Syndrome, or Neonatal Onset Multisystem Inflammatory Disease
• Date of first enrolment: May 2008
• Target sample size: 166
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT00685373
• Study type: Interventional
• Phase: Phase 3

Countries of recruitment

Belgium, France, Germany, India, Italy, Spain, Turkey, United Kingdom, United States

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Male and female patients at least 3 years of age

2. Diagnosis of Familial Cold Autoinflammatory Syndrome, Muckle-Wells Syndrome or
Neonatal Onset Multisystem Inflammatory Disease. Prior agreement between the
Investigator and Novartis for study eligibility is required for patients who do not
have a molecular diagnosis of NALP3 mutations available (either testing not performed,
or testing performed but negative)upon study entry. For those patients who have not
been molecularly tested for NALP3 mutations, molecular testing should be performed
during the course of the study

3. For patients under anakinra therapy or any other investigational IL-1 blocking
therapy, these treatments should be discontinued prior to the baseline visit.

4. Patients from the CACZ885A2102 study may enter this study. However, dosing at Visit 2
(Baseline Visit) can only occur if either 1) the patient is experiencing disease flare
or 2) at least two months have elapsed from their last injection even in the absence
of flare, whichever is earlier.

5. Patients who completed the CACZ885D2304 study may enter this study

6. Patients who completed the CACZ885D2201 study may enter this study

7. Patients who discontinued from the CACZ885A2102, CACZ885D2201 or CACZ885D2304 studies
and for whom in the Investigator's judgment (with prior agreement from Novartis)
continued treatment with ACZ885 in this study is considered appropriate.

Exclusion Criteria:

1. Participation in any clinical investigation within 4 weeks prior to dosing or longer
if required by local regulation with the exception of trials with anakinra, other
investigational IL-1 blocking therapies, and/or ACZ885.

2. History of being immunocompromised, including a positive HIV at screening (ELISA and
Western blot) test result.

3. A positive Hepatitis B surface antigen (HBsAg) or Hepatitis C antibody test result is
not allowed.

4. No live vaccinations within 3 months prior to the start of the trial, during the
trial, and up to 3 months following the last dose.

5. History of recurrent and/or evidence of active bacterial, fungal, or viral infections.

6. Positive tuberculin skin test reaction (PPD 5 tuberculin units or as according to
local standard practice) (>= 5 mm induration) at 48 to 72 hours after administration
at the screening visit or within 2 months prior to the screening visit. Patients who
have a positive PPD skin test with a documentation of BCG vaccination, who are at low
environmental risk for tuberculosis (TB) infection or reactivation, and have a
negative chest X-ray can be included.

Other protocol-defined inclusion/exclusion criteria may apply



Age minimum: 3 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Cryopyrin-Associated Periodic Syndromes

Neonatal Onset Multisystem Inflammatory Disease

Familial Cold Autoinflammatory Syndrome

Muckle Wells Syndrome

Intervention(s)

Drug: Canakinumab (ACZ885)

Primary Outcome(s)

The Number of Participants With Adverse Events (AEs), Death, Serious Adverse Events (SAEs), Discontinuation of Study Drug Due to an AE, Infections and Infestations and Injection Site Reactions [Time Frame: 2 years depending on when the participant enters the study]

Secondary Outcome(s)

The Percentage of Participants Without Disease Relapse as Determined by the Physician's Global Assessment of Autoinflammatory Disease Activity, Assessment of Skin Disease and Inflammation Markers. [Time Frame: Every 8 weeks during the course of the trial for at least 6 months with a maximum duration of 2 years]

Immunogenicity of Canakinumab (ACZ885) [Time Frame: Every 8 weeks during the course of the trial for at least 6 months with a maximum duration of 2 years]

Pharmacokinetics [Time Frame: Every 8 weeks during the course of the trial for at least 6 months with a maximum duration of 2 years]

Secondary ID(s)

CACZ885D2306

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 27/05/2011
• URL: https://clinicaltrials.gov/ct2/show/results/NCT00685373