Trial record 12 of 47 for:
Open Studies | "Eosinophilia"
Efficacy, Safety and Tolerability of ACZ885 in Pediatric Patients With the Following Cryopyrin-associated Periodic Syndromes: Familial Cold Autoinflammatory Syndrome, Muckle-Wells Syndrome, or Neonatal Onset Multisystem Inflammatory Disease
This study is currently recruiting participants.
Verified May 2012 by Novartis
Sponsor:
Novartis Pharmaceuticals
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier:
NCT01302860
First received: February 22, 2011
Last updated: May 7, 2012
Last verified: May 2012
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Purpose
This trial will assess the safety, efficacy and tolerability of ACZ885 in patients aged 4 years and younger with cryopyrin associated periodic syndromes (CAPS)
| Condition | Intervention | Phase |
|---|---|---|
|
Cryopyrin-associated Periodic Syndromes Familial Cold Autoinflammatory Syndrome Muckle-Wells Syndrome Neonatal Onset Multisystem Inflammatory Disease |
Biological: ACZ885 |
Phase 3 |
| Study Type: | Interventional |
| Study Design: | Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment |
| Official Title: | A One-year Open-label, Multicenter Trial to Assess Efficacy, Safety and Tolerability of Canakinumab (ACZ885) and the Efficacy and Safety of Childhood Vaccinations in Patients Aged 4 Years or Younger With Cryopyrin Associated Periodic Syndromes (CAPS) |
Resource links provided by NLM:
Genetics Home Reference related topics:
familial cold autoinflammatory syndrome
familial Mediterranean fever
Muckle-Wells syndrome
neonatal onset multisystem inflammatory disease
Drug Information available for:
Canakinumab
U.S. FDA Resources
Further study details as provided by Novartis:
Primary Outcome Measures:
- To assess the efficacy of canakinumab with respect to the treatment response in CAPS patients 4 years and younger [ Time Frame: A maximum of 56 weeks ] [ Designated as safety issue: No ]
Secondary Outcome Measures:
- To assess the efficacy of canakinumab with respect to the treatment response in CAPS patients 2 years and younger [ Time Frame: A maximum of 56 weeks ] [ Designated as safety issue: No ]
- Safety and tolerability as assessed by overall frequency of adverse events and number of patients completing the study in patients 2 years and younger and the overall population [ Time Frame: A maximum of 56 weeks ] [ Designated as safety issue: Yes ]
- To assess the presence of protective antibody levels following immunization with inactivated (killed) vaccines [ Time Frame: A maximum of 56 weeks ] [ Designated as safety issue: Yes ]
- To evaluate the safety of canakinumab treatment in pediatric patients receiving a concomitant vaccination [ Time Frame: A maximum of 56 weeks ] [ Designated as safety issue: Yes ]
- To evaluate the proportion of patients with vaccinated-associated reactions [ Time Frame: A maximum of 56 weeks ] [ Designated as safety issue: No ]
- To assess the reduction of inflammation marker (C-reactive protein (CRP) or serum amyloid A (SAA)) after treatment initiation [ Time Frame: A maximum of 56 weeks ] [ Designated as safety issue: No ]
| Estimated Enrollment: | 15 |
| Study Start Date: | November 2010 |
| Estimated Study Completion Date: | January 2015 |
| Estimated Primary Completion Date: | January 2015 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
| Experimental: Canakinumab | Biological: ACZ885 |
Eligibility| Ages Eligible for Study: | up to 4 Years |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion Criteria:
- Male and female patients that are 28 days up to 60 months of age at the time of the screening visit.
- Body weight > or = 2.5 kg.
- Parent or legal guardian's written informed consent is required before any assessment is performed for patients.
- At study entry, patients should have a clinical diagnosis of FCAS, MWS, or NOMID and symptoms requiring pharmacological intervention. 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.
- For patients treated with an IL-1 blocking agent (i.e. anakinra, rilonacept), these treatments should be discontinued prior to the baseline visit and patients must demonstrate active disease prior to treatment.
- Patients who are scheduled to receive an immunization, according to their local vaccination guidelines, with an inactivated vaccine must be willing to participate in the assessment schedule for vaccinated patients.
Exclusion Criteria:
- Preterm neonates for whom, in the Investigator's judgment, participation in the study is not deemed appropriate.
- History of recurrent and/or evidence of active bacterial, fungal, or viral infections (including HIV).
- Patients with immunodeficiency or treatment with immunosuppressive drugs.
- Live vaccinations within < or = 3 months prior to screening. No live vaccinations will be allowed throughout the course of this study and up to 3 months following the last dose.
Patients with an increased risk of tuberculosis (TB) infection according to following risk factors:
- Patients with recent close contact with persons known to have active pulmonary TB disease
- Foreign-born patients from countries with a high prevalence of tuberculosis
- Patients with recent tuberculosis infection (including children > 6 months with a positive PPD test [defined as an induration of at least 10mm])
- Patients with end-stage renal disease
- Patients with diabetes mellitus
- Patients receiving immunosuppressive therapy
- Patients with hematologic cancers.
- Participation in another trial within the last 30 days or 5 half-lives of the investigational compound (whichever is longer).
- Familial and social conditions rendering regular medical assessment not possible.
- Pediatric patients with neutropenia (absolute neutrophil count [ANC] < 1.5 x 10 to the 9th/l)
Other protocol defined inclusion/exclusion criteria may apply
Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01302860
Contacts
| Contact: Novartis Pharmaceuticals | 41 61 324 1111 |
Locations
| Belgium | |
| Novartis Investigative site | Recruiting |
| Bruxelles, Belgium | |
| Novartis Investigative site | Recruiting |
| Laeken, Belgium | |
| Canada | |
| Novartis Investigative site | Not yet recruiting |
| Toronto, Canada | |
| France | |
| Novartis Investigative site | Not yet recruiting |
| Le Kremlin-Bicetre, France | |
| Novartis Investigative site | Not yet recruiting |
| Paris, France | |
| Germany | |
| Novartis Investigative site | Recruiting |
| Dresden, Germany | |
| Novartis Investigative site | Recruiting |
| Sankt Augustin, Germany | |
| Novartis Investigative site | Recruiting |
| Tubingen, Germany | |
| Spain | |
| Novartis Investigative site | Recruiting |
| Campanar, Spain | |
| Novartis Investigative site | Recruiting |
| Oloriz, Spain | |
| United Kingdom | |
| Novartis Investigative site | Not yet recruiting |
| Liverpool, United Kingdom | |
| Novartis Investigative site | Not yet recruiting |
| London, United Kingdom | |
Sponsors and Collaborators
Novartis Pharmaceuticals
Investigators
| Study Director: | Novartis Pharmaceuticals | Novartis Pharmaceuticals |
More Information
No publications provided
| Responsible Party: | Novartis ( Novartis Pharmaceuticals ) |
| ClinicalTrials.gov Identifier: | NCT01302860 History of Changes |
| Other Study ID Numbers: | CACZ885D2307 |
| Study First Received: | February 22, 2011 |
| Last Updated: | May 7, 2012 |
| Health Authority: | United States: Food and Drug Administration Germany: Paul-Ehrlich-Institut Spain: Spanish Agency of Medicines France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis) United Kingdom: Medicines and Healthcare Products Regulatory Agency Belgium: Belgian Health Care Knowledge Center Canada: Health Canada |
Keywords provided by Novartis:
|
Cryopyrin-associated periodic syndromes (CAPS) Familial Cold Autoinflammatory Syndrome (FCAS) Muckle-Wells Syndrome (MWS) or Neonatal Onset Multisystem Inflammatory Disease (NOMID) children systemic autoinflammatory disease CIAS-1 gene NALP-3 |
NLRP3 ACZ885 Ilaris human monoclonal anti-human interleukin-1 beta (IL-beta) antibody autosomal dominant familial autoinflammatory syndrome |
Additional relevant MeSH terms:
|
Eosinophilia Cryopyrin-Associated Periodic Syndromes Cellulitis Hereditary Autoinflammatory Diseases Genetic Diseases, Inborn Skin Diseases, Genetic Skin Diseases Skin Diseases, Infectious |
Infection Suppuration Connective Tissue Diseases Inflammation Pathologic Processes Leukocyte Disorders Hematologic Diseases |
ClinicalTrials.gov processed this record on May 23, 2013