Canakinumab in Patients With Active Hyper-IgD Syndrome

Novartis

Status and phase: Completed; Phase 2

Conditions: Mevalonate Kinase Deficiency

Treatments: Drug: Canakinumab

Study type: Interventional

Funder types: Industry

Identifiers

NCT01303380

CACZ885D2402

2010-020904-31 (EudraCT Number)

Details and patient eligibility

About

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

Enrollment: 9 patients

Sex: All

Ages: 24+ months old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Single Group Assignment

Masking: None (Open label)

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9 participants in 1 patient group

Canakinumab

Experimental group

Treatment:

Drug: Canakinumab