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

NCT ID: NCT01302860

Last Updated: 2017-03-29

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 17 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2010-11-30
• Study Completion Date: 2014-11-30

Brief Summary

This trial will assess the safety, efficacy and tolerability of ACZ885 in patients aged 4 years and younger with cryopyrin associated periodic syndromes (CAPS)

Conditions

Muckle-Wells Syndrome; Cryopyrin-associated Periodic Syndromes; Familial Cold Autoinflammatory Syndrome; Neonatal Onset Multisystem Inflammatory Disease

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Canakinumab

Canakinumab s.c. injection (2 mg/kg) was administered every 8 weeks.

Group Type: EXPERIMENTAL

ACZ885

Intervention Type: DRUG

Interventions

ACZ885

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

1. Male and female patients that are 28 days up to 60 months of age at the time of the screening visit.

2. Body weight > or = 2.5 kg.

3. Parent or legal guardian's written informed consent is required before any assessment is performed for patients.

4. 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.

5. 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.

6. 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

1. Preterm neonates for whom, in the Investigator's judgment, participation in the study is not deemed appropriate.

2. History of recurrent and/or evidence of active bacterial, fungal, or viral infections (including HIV).

3. Patients with immunodeficiency or treatment with immunosuppressive drugs.

4. 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.

5. 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.

6. Participation in another trial within the last 30 days or 5 half-lives of the investigational compound (whichever is longer).

7. Familial and social conditions rendering regular medical assessment not possible.

8. Pediatric patients with neutropenia (absolute neutrophil count [ANC] < 1.5 x 10 to the 9th/l)

• Minimum Eligible Age: 1 Month
• Maximum Eligible Age: 60 Months
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Novartis Pharmaceuticals

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Novartis Pharmaceuticals

Role: STUDY_DIRECTOR

Novartis Pharmaceuticals

Locations

Novartis Investigative Site

Brussels, , Belgium

Novartis Investigative Site

Laken, , Belgium

Novartis Investigative Site

Toronto, Ontario, Canada

Novartis Investigative Site

Le Kremlin-Bicêtre, , France

Novartis Investigative Site

Berlin, , Germany

Novartis Investigative Site

Dresden, , Germany

Novartis Investigative Site

Sankt Augustin, , Germany

Novartis Investigative Site

Tübingen, , Germany

Novartis Investigative Site

Granada, Andalusia, Spain

Novartis Investigative Site

Valencia, Valencia, Spain

Novartis Investigative Site

Lausanne, , Switzerland

Novartis Investigative Site

London, , United Kingdom

Countries

Belgium; Canada; France; Germany; Spain; Switzerland; United Kingdom

References

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.

Reference Type: DERIVED

PMID: 31161734 (View on PubMed)

Other Identifiers

2009-016859-22

Identifier Type: -

Identifier Source: secondary_id

CACZ885D2307

Identifier Type: -

Identifier Source: org_study_id