An open-label, long-term safety and efficacy study of ACZ885 (anti-interleukin-1β 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 Muckle-Wells Syndrome with overlapping symptoms of Neonatal Onset Multisystem Inflammatory Disease - ND
- Conditions
- Muckle-Wells Syndrome, Familial Cold autoinflammatory syndromeMedDRA version: 9.1Level: LLTClassification code 10064569Term: Muckle-Wells syndrome
- Registration Number
- EUCTR2007-004367-22-IT
- Lead Sponsor
- OVARTIS FARMA
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 80
Patients eligible for inclusion in this study have to fulfill all of the following criteria: 1. Male and female patients at least 4 years of age at the time of the screening visit. 2. Patient?s informed consent for ≥ 18 years of age before any assessment is performed. 3. Parent or legal guardian?s written informed consent and child?s assent, if appropriate, are required before any assessment is performed for patients < 18 years of age. 4. Patients with a diagnosis of FCAS, MWS, or MWS with overlapping symptoms of NOMID (very severe MWS patients, including central nervous system (CNS) symptoms, can be included only with prior agreement from Novartis). At study entry, new patients should have symptoms requiring pharmacological intervention. At the time of screening, patients can be either untreated or treated (i.e. under ACZ885, anakinra, or any other investigational IL-1 blocking therapy). 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 upon study entry. 5. For patients under anakinra therapy or any other investigational IL-1 blocking therapy, these treatments should be discontinued prior to the baseline visit (see Section 6.5.8 for specific time points for discontinuing treatments prior to the baseline visit). 6. Patients from the A2102 study may enter this study upon signing informed consent irrespective of whether they are in remission or flaring. 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. 7. Patients who completed the D2304 study may enter this study upon signing informed consent. A patient is defined as completing the study if he/she completed the D2304 study up to and including Visit 15 (End of Study Visit). 8. Patients who discontinued from the A2102 or D2304 studies and for whom in the Investigator?s judgment (with prior agreement from Novartis) continued treatment with ACZ885 in this study is considered appropriate. 9. Body weight ≥ 15 kg. 10. Able to communicate with the investigator and comply with the requirements of the study (for children the parent can assist when necessary). PLS SEE PROTOCOL
Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range
1. Pregnant or nursing (lactating) women, where pregnancy is defined as the state of a female after conception and until the termination of gestation, confirmed by a positive hCG laboratory test (> 5 mIU/mL). 2. Women of child-bearing potential (WOCBP), defined as all women physiologically capable of becoming pregnant, including women whose career, lifestyle, or sexual orientation excludes or prevents them from having intercourse with a male partner and women whose partners have been sterilized by vasectomy or other means, unless they meet the following definition of postmenopausal state: 12 months of natural (spontaneous) amenorrhea or 6 months of spontaneous amenorrhea with serum FSH levels >40 mIU/mL or are using one or more of the following acceptable methods of contraception: surgical sterilization (e.g., bilateral tubal ligation, hysterectomy) hormonal contraception (implantable, patch, oral) double-barrier methods (any double combination of: IUD, male or female condom with spermicidal gel, diaphragm, sponge, cervical cap) Acceptable methods of contraception may include total abstinence at the discretion of the investigator in cases where the age, career, lifestyle, or sexual orientation of the patient ensures compliance. Periodic abstinence (e.g., calendar, ovulation, symptothermal, post-ovulation methods) and withdrawal are not acceptable methods of contraception. Reliable contraception should be maintained throughout the study (from the date of screening) and for 3 months following the last dose. 3. 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. 4. History of being immunocompromised, including a positive HIV at screening (ELISA and Western blot) test result. 5. A positive Hepatitis B surface antigen (HBsAg) or Hepatitis C antibody test result. 6. Live vaccinations within 3 months prior to the start of the trial, during the trial, and up to 3 months following the last dose. 7. History of drug or alcohol abuse within the 12 months prior to dosing. 8. Donation or loss of 400 mL or more of blood within 8 weeks prior to dosing for adults (For children the cut-off amount of donation or loss of blood should be based on the Investigator's judgment & according to the local regulations. Please also refer to Appendix 4 for the recommended sample volume per blood draw for children). 9. History of significant medical conditions, which in the Investigator?s opinion would exclude the patient from participating in this trial (this can be discussed with Novartis on a case by case basis in case of uncertainty). 10. History of recurrent and/or evidence of active bacterial, fungal, or viral infections. PLS SEE PROTOCOL
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method