Canakinumab in Patients With Active Hyper-IgD Syndrome

Novartis Pharmaceuticals9 enrolled

Overview

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

Study Type

INTERVENTIONAL

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Mevalonate Kinase Deficiency

Interventions

CanakinumabDRUG

Eligibility

Sex: ALLMin age: 24 Months

Medical Language ↔ Plain English

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

Locations (3)

Novartis Investigative Site

Esplugues de Llobregat, Barcelona, Spain

Novartis Investigative Site

Madrid, Madrid, Spain

Novartis Investigative Site

Valencia, Valencia, Spain

Outcomes

Primary Outcomes

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.

Time frame: Historical period, Month 6 (End of treatment period)

Secondary Outcomes

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

A flare was defined as Physician Global Assessment of HIDS flare severity score of ≥ 2 and a CRP value \> 10 mg/L.

Time frame: 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

A flare was defined as Physician Global Assessment of HIDS flare severity score of ≥ 2 and a CRP value \> 10 mg/L.

Time frame: 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

Physician global assessment of severity of HIDS after each flare was based on HIDS flare severity score, a 5- point scale: 0 = Absent signs/symptoms; 1 = Minimal signs/symptoms; 2 = Mild; 3= Moderate; 4 = Severe.

Time frame: 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

Participant's global assessment of severity of HIDS after each flare was based on HIDS flare severity score, a 5-point scale: 0 = Absent signs/symptoms; 1 = Minimal signs/symptoms; 2 = Mild; 3= Moderate; 4 = Severe. Same investigator assessed the same participant throughout the study to ensure consistency between assessments. Investigators reviewed every participant's diary at each visit after their own clinical assessment.

Data from ClinicalTrials.gov

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