Canakinumab in Patients With Active Hyper-IgD Syndrome

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier:
NCT01303380
First received: February 23, 2011
Last updated: March 4, 2014
Last verified: March 2014
  Purpose

This pilot study is designed to evaluate the efficacy, the safety, and the pharmacokinetics (PK) / pharmacodynamics (PD) of canakinumab treatment in patients with HIDS.


Condition Intervention Phase
Mevalonate Kinase Deficiency
Drug: Canakinumab
Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: An Open-label, Multicenter, Efficacy and Safety Pilot Study of 6-month Canakinumab Treatment With up to 6-month Follow-up in Patients With Active Hyper-IgD Syndrome (HIDS)

Resource links provided by NLM:


Further study details as provided by Novartis:

Primary Outcome Measures:
  • The primary efficacy variable is the number of flares observed during the 6-month treatment period. [ Time Frame: 1 year ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Number of patients who flare during treatment period [ Time Frame: 1 year ] [ Designated as safety issue: No ]
  • Flare severity during the treatment period for patients who have at least 1 flare during the treatment period as assessed by the physician's global assessment of HIDS flare severity score. [ Time Frame: 1 year ] [ Designated as safety issue: No ]
  • Flare severity during the treatment period for patients who have at least 1 flare during the treatment period as assessed by patient's global assessment of HIDS flare severity score. [ Time Frame: 1 year ] [ Designated as safety issue: No ]
  • HIDS signs and symptoms severity during the treatment period for patients who have at least 1 flare during the treatment period as assessed by physician assessment of 4 key signs and symptoms severity score. [ Time Frame: 1 year ] [ Designated as safety issue: No ]
  • HIDS signs and symptoms severity during the treatment period for patients who have at least 1 flare during the treatment period as assessed by patient assessment of 4 key signs and symptoms severity score. [ Time Frame: 1 year ] [ Designated as safety issue: No ]

Estimated Enrollment: 15
Study Start Date: May 2011
Estimated Study Completion Date: October 2014
Estimated Primary Completion Date: October 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Canakinumab Drug: Canakinumab

  Eligibility

Ages Eligible for Study:   2 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Patients with a diagnosis of HIDS proven by DNA analysis and/or enzymatic studies.
  2. At time of start of drug treatment: active HIDS as evidenced by a physician global assessment of HIDS flare severity ≥ 2 and CRP values >10 mg/L (normal CRP < or = 10 mg/L).
  3. Patients who have a history of > or = 3 febrile acute HIDS flares in a 6-month period when not receiving prophylaxis treatment (e.g. anakinra daily treatment) with a duration of each flare lasting > or = 4 days and limiting the normal daily activities.

Exclusion Criteria:

  1. Pregnant or nursing (lactating) women.
  2. History of being immunocompromised, including a positive HIV at screening (ELISA and Western blot) test result.
  3. Positive Hepatitis B or Hepatitis C.
  4. Live vaccinations within 3 months prior to the start of the trial
  5. Positive tuberculosis screening test.

Other protocol-defined inclusion/exclusion criteria may apply

  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01303380

Locations
Spain
Novartis Investigative site
Barcelona, Spain
Novartis Investigative site
Valencia, Spain
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: NCT01303380     History of Changes
Other Study ID Numbers: CACZ885D2402, 2010-020904-31
Study First Received: February 23, 2011
Last Updated: March 4, 2014
Health Authority: Spain: Agencia Española de Medicamentos y Productos Sanitarios

Keywords provided by Novartis:
Hyper IgD syndrome
canakinumab
HIDS
mevalonate kinase deficiency
MKD

Additional relevant MeSH terms:
Mevalonate Kinase Deficiency
Peroxisomal Disorders
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Hypergammaglobulinemia
Blood Protein Disorders
Hematologic Diseases
Hereditary Autoinflammatory Diseases
Genetic Diseases, Inborn
Metabolism, Inborn Errors
Metabolic Diseases
Immunoproliferative Disorders
Immune System Diseases
Antibodies, Monoclonal
Immunologic Factors
Physiological Effects of Drugs
Pharmacologic Actions

ClinicalTrials.gov processed this record on August 01, 2014