Canakinumab in Patients With Active Hyper-IgD Syndrome

NCT01303380 | Completed Phase 2 Results

• 2 other identifiers: CACZ885D24022010-020904-31
• Study Type: interventional
• Target: 9
• Locations: 1 country
• Sites: 3

Brief Summary

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

Conditions

Mevalonate Kinase Deficiency

Keywords

Hyper IgD syndrome; canakinumab; HIDS; mevalonate kinase deficiency; MKD

Outcome Measures

Primary Outcomes (1)

• Number of Flares Per Participant During Historical Period and Treatment Period

  A flare was defined as Physician Global Assessment of HIDS flare severity score of ≥ 2 and a C-reactive protein (CRP) value \> 10 mg/L. Flares during a historical period were defined as most recent 6-months in which the participant has not received treatment for their HIDS other than symptomatic treatment with NSAIDs and/or corticosteroids.

  Historical period, Month 6 (End of treatment period)

Secondary Outcomes (22)

• Number of Flares Per Participant at During Treatment Period and 24 Month Extension Period

  Month 6 (End of treatment period), Month 36 (End of Long term treatment Period 2)

• Number of Participants Who Flared at Month 6, Month 24 and Month 36

  Baseline, Month 6 (End of treatment period), Month 24 (End of Long term treatment period 1) and Month 36 (End of Long term treatment period 2)

• Number of Participants With Flare Events Based on Physician Assessed HIDS Flare Severity Score

  Any flare event [Baseline up to Month 36 (End of long term treatment period 2)]

• Number of Participants With Flare Events Based on Participant Assessed HIDS Flare Severity Score

  Baseline, Month 6 (End of treatment period), Month 12 (End of follow up period), Month 24 (End of Long term treatment period 1) and Month 36 (End of Long term treatment period 2)

• Percentage of Participants With Defined Grades of Participants Assessed Symptom Control

  Baseline, Month 6 (End of treatment period), Month 12 (End of follow up period), Month 24 (End of Long term treatment period 1) and Month 36 (End of Long term treatment period 2)

Study Arms (1)

Canakinumab

EXPERIMENTAL

Drug: Canakinumab

Interventions

Canakinumab DRUG

Canakinumab

Eligibility Criteria

• Age: 24 Months+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Patients with a diagnosis of HIDS proven by DNA analysis and/or enzymatic studies.
• 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).
• 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.

You may not qualify if:

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

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (3)

Novartis Investigative Site

Esplugues de Llobregat, Barcelona, 08950, Spain

Location

Novartis Investigative Site

Madrid, Madrid, 28046, Spain

Location

Novartis Investigative Site

Valencia, Valencia, 46026, Spain

Location

Related Publications (1)

• Arostegui JI, Anton J, Calvo I, Robles A, Iglesias E, Lopez-Montesinos B, Banchereau R, Hong S, Joubert Y, Junge G, Pascual V, Yague J. Open-Label, Phase II Study to Assess the Efficacy and Safety of Canakinumab Treatment in Active Hyperimmunoglobulinemia D With Periodic Fever Syndrome. Arthritis Rheumatol. 2017 Aug;69(8):1679-1688. doi: 10.1002/art.40146. Epub 2017 Jul 5.

  PMID: 28482144 DERIVED

MeSH Terms

Conditions

Mevalonate Kinase Deficiency

Interventions

canakinumab

Condition Hierarchy (Ancestors)

Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Hypergammaglobulinemia; Blood Protein Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Hereditary Autoinflammatory Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolism, Inborn Errors; Peroxisomal Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases; Immunoproliferative Disorders; Immune System Diseases

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 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 23, 2011
• First Posted: February 24, 2011
• Study Start: March 1, 2011
• Primary Completion: July 1, 2014
• Study Completion: July 1, 2014
• Last Updated: November 5, 2015
• Results First Posted: August 19, 2015

Record last verified: 2015-10

Locations

ES (3)