A study to check how safe, beneficial and tolerable the drugcanakinumab is for patients with Crypoyrin Associated Periodic Syndromes (CAPS).
- Conditions
- The following cryopyrin-associated periodic syndromes:Familial Cold Autoinflammatory Syndrome,Muckle-Wells Syndrome,or Neonatal Onset Multisystem Inflammatory DiseaseMedDRA version: 14.1Level: PTClassification code 10068850Term: Cryopyrin associated periodic syndromeSystem Organ Class: 10010331 - Congenital, familial and genetic disordersTherapeutic area: Diseases [C] - Immune System Diseases [C20]
- Registration Number
- EUCTR2007-004367-22-Outside-EU/EEA
- Lead Sponsor
- ovartis Pharma Services AG
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- A
- Sex
- All
- Target Recruitment
- 166
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.
Other protocol-defined inclusion criteria may apply.
Are the trial subjects under 18? yes
Number of subjects for this age range: 46
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 115
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 5
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 exclusion criteria may apply
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: This study will provide long-term safety and efficacy data for ACZ885 (a fully human anti-interleukin-1ß [anti-IL-1ß] monoclonal antibody) given as an injection subcutaneously in patients who participated in the CACZ885A2102 (NCT00487708), CACZ885D2201 (NCT00685373) or CACZ885D2304(NCT00465985) studies or newly identified patients with the following cryopyrin-associated periodic syndromes: Familial Cold Autoinflammatory Syndrome, Muckle-Wells Syndrome or Neonatal Onset Multisystem Inflammatory Disease.;Secondary Objective: ;Primary end point(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;Timepoint(s) of evaluation of this end point: 2 years depending on when the participant enters the study
- Secondary Outcome Measures
Name Time Method Secondary end point(s): 1. 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.<br>2. Immunogenicity of Canakinumab (ACZ885)<br>3. Pharmacokinetics;Timepoint(s) of evaluation of this end point: Every 8 weeks during the course of the trial for at least 6 months with a maximum duration of 2 years<br>