NCT01576367

Brief Summary

This trial will provide long-term safety, efficacy and tolerability of ACZ885 in CAPS patients that completed the CACZ885D2307 study

Trial Health

93
On Track

Trial Health Score

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

Enrollment
17

participants targeted

Target at below P25 for phase_3

Timeline
Completed

Started Jan 2012

Typical duration for phase_3

Geographic Reach
7 countries

12 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

January 16, 2012

Completed
1 month until next milestone

First Submitted

Initial submission to the registry

February 17, 2012

Completed
2 months until next milestone

First Posted

Study publicly available on registry

April 12, 2012

Completed
3.5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 13, 2015

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

October 13, 2015

Completed
10 months until next milestone

Results Posted

Study results publicly available

August 3, 2016

Completed
Last Updated

September 11, 2018

Status Verified

August 1, 2018

Enrollment Period

3.7 years

First QC Date

February 17, 2012

Results QC Date

June 22, 2016

Last Update Submit

August 13, 2018

Conditions

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

Keywords

Cryopyrin-associated periodic syndromes (CAPS)Familial Cold Autoinflammatory Syndrome (FCAS)Muckle-Wells Syndrome (MWS)Neonatal Onset Multisystem Inflammatory Disease (NOMID)children, systemic autoinflammatory diseaseCIAS-1 geneNALP-3NLRP3ACZ885canakinumabhuman monoclonal anti-human interleukin-1 antibodyautosomal dominantfamilial autoinflammatory syndromechildhood immunizations vaccinations

Outcome Measures

Primary Outcomes (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 Serological Inflammation Markers.

    Disease relapse following complete response is defined as inflammation markers: C-Reactive Protein (CRP) and/or Serum Amyloid A (SAA) result \> 30 mg/L AND Physician's Global Assessment of Autoinflammatory Disease Activity \> minimal or Physician's Global Assessment \>= minimal AND Skin Disease Assessment \> minimal. Physician's Global Assessment of Autoinflammatory Disease Activity and Skin Disease Assessment (urticarial skin rash) are completed by the investigator using a 5 point rating scale: absent, minimal, mild, moderate and severe.

    Week /80, 104, 128, and 152 (A minimum of 6 months and maximum of 24 months)

Secondary Outcomes (4)

  • Immunogenicity of Canakinumab (ACZ885). Number of Participants With Anti-canakinumab Antibodies

    minimum of 6 months and maximum of 24 months

  • Change From Baseline (Core Study Baseline) in C--Reactive Protein (CRP) and Serum Amyloid A (SAA) Concentrations

    Week 0, 80, 104, 128 and 152, last assessment

  • Frequency Counts of Physician's Global Assessment of Autoinflammatory Disease and Skin Disease

    minimum of 6 months and maximum of 24 months

  • Number of Vaccination Cases With Protective Antibody Levels Following Immunization With Inactivated Vaccines

    pre-vaccine dose, Day 28 post-vaccine

Study Arms (1)

canakinumab

EXPERIMENTAL

Patients will receive a standard dose at an equivalent of 2 mg/kg s.c. of canakinumab (ACZ885) every 8 weeks. Possible dose and/or dosing regimen adjustments that can be administered include: 4 mg/kg s.c. (every 4 to 8 weeks) 6 mg/kg s.c. (every 4 to 8 weeks) 8 mg/kg s.c. (every 4 to 8 weeks)

Biological: ACZ885

Interventions

ACZ885BIOLOGICAL
Also known as: Canakinumab
canakinumab

Eligibility Criteria

Age1 Year - 4 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • Patients who completed the core CACZ885D2307 study (a patient is defined as having completed the core study if they completed the study up to and including the EOS visit with no major protocol deviations in the core).
  • Male and female patients that are ≥ 1 year of age at the time of the roll-over visit.
  • Parent or legal guardian written informed consent must be obtained before any assessment in the extension CACZ885D2307E1 study is performed.

You may not qualify if:

  • Patients for who continued treatment in the CACZ885D2307E1 extension study is not considered appropriate by the treating physician.
  • Patients who discontinued from the core CACZ885D2307 study

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (12)

Novartis Investigative Site

Brussels, 1200, Belgium

Location

Novartis Investigative Site

Laken, 1020, Belgium

Location

Novartis Investigative Site

Toronto, Ontario, M5G 1X8, Canada

Location

Novartis Investigative Site

Le Kremlin-Bicêtre, 94275, France

Location

Novartis Investigative Site

Berlin, 13353, Germany

Location

Novartis Investigative Site

Dresden, 01307, Germany

Location

Novartis Investigative Site

Saint Augustin, 53757, Germany

Location

Novartis Investigative Site

Tübingen, 72076, Germany

Location

Novartis Investigative Site

Granada, Andalusia, 18012, Spain

Location

Novartis Investigative Site

Valencia, Valencia, 46026, Spain

Location

Novartis Investigative Site

Lausanne, 1011, Switzerland

Location

Novartis Investigative Site

London, WC1N 1EH, United Kingdom

Location

Related Publications (1)

  • Brogan PA, Hofer M, Kuemmerle-Deschner JB, Kone-Paut I, Roesler J, Kallinich T, Horneff G, Calvo Penades I, Sevilla-Perez B, Goffin L, Lauwerys BR, Lachmann HJ, Uziel Y, Wei X, Laxer RM. Rapid and Sustained Long-Term Efficacy and Safety of Canakinumab in Patients With Cryopyrin-Associated Periodic Syndrome Ages Five Years and Younger. Arthritis Rheumatol. 2019 Nov;71(11):1955-1963. doi: 10.1002/art.41004. Epub 2019 Sep 9.

    PMID: 31161734DERIVED

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

Results Point of Contact

Title
Study Director
Organization
Novartis Pharmaceuticals
862-778-8300

Study Officials

  • Novartis Pharmaceuticals

    Novartis Pharmaceuticals

    STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 3
Allocation
NON RANDOMIZED
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

February 17, 2012

First Posted

April 12, 2012

Study Start

January 16, 2012

Primary Completion

October 13, 2015

Study Completion

October 13, 2015

Last Updated

September 11, 2018

Results First Posted

August 3, 2016

Record last verified: 2018-08

Locations

BE(2)ES(2)GB(1)CH(1)DE(4)FR(1)CA(1)