Canakinumab to Treat Neonatal-Onset Multisystem Inflammatory Disease

Conditions

• CINCA Syndrome
• NOMID

Eligibility Criteria

Sex

ALL

Age

2 Years - 25 Years

Healthy Volunteers

Not accepted

Interventions

• Canakinumab — DRUG

Study Details

This study will examine whether a medicine called canakinumab is safe and effective for treating patients with neonatal-onset multisystem inflammatory disease (NOMID), also known as chronic infantile neurologic, cutaneous, articular (CINCA) syndrome. This disease can cause rash, joint deformities, brain inflammation, problems with the eyes and learning difficulties. Canakinumab is an experimental drug that inhibits the action of a protein produced by the body called human IL-1beta, which is responsible for the symptoms in NOMID and also contributes to many other kinds of inflammatory diseases. Patients 2 years of age and older with NOMID / CINCA may be eligible for this study. Participants undergo the following procedures: Screening Phase * Medical history and review of medical records * Blood tests * Daily diary of symptoms and medicines take Washout/Lead-in Phase * Discontinuation of anakinra or other medications, a 6 to 48-hour run-in period (only for patients who discontinued anakinra or other IL-1 blocking therapy). Treatment Phase * Injection of canakinumab under the skin every 8 weeks for 6 months * Monitoring and evaluations during treatment, including: * Quality-of-life questionnaires and daily diary * Vital signs measurements (heartbeat, blood pressure, temperature) * Blood tests * Electrocardiogram * Tuberculosis skin test * Neurological, eye and skin examinations at beginning and end of study * Cognitive evaluation at beginning and end of study * Lumbar puncture (spinal tap) at the beginning of the study, 2 weeks after the second dosing of canakinumab and at the end of the study * X-rays and bone density scan at beginning and end of study * Magnetic resonance imaging (MRI) of the head at beginning and end of study Follow-up Phase * Monthly clinic visits after the last dose of canakinumab for a minimum of 60 days End-of-Study Evaluation * Series of tests 8 weeks after last dose of canakinumab to evaluate treatment response and side effects

Key Dates

Start date

Jan 26, 2009

Status verified

Jun 2021

Primary completion

Feb 17, 2011

Completion

Feb 17, 2011

Study Design

Enrollment

7 participants (actual)

Allocation

NA

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Arms

• Experimental: Canakinumab
  Participants received body-weight stratified dosage of canakinumab treatment at 300 milligrams (mg) (for participants weighing more than 40 kilograms (kg)) and at 2 mg/kg (for participants weight less than or equal to 40 kg) subcutaneously every 4-8 weeks as per investigator discretion for a treatment period of 6 months. The first 3 Neonatal-Onset Multisystem Inflammatory Disease (NOMID) participants enrolled received a dose of 150 mg (\>40 kg) and 2 mg/kg for children \<40 kg. Since this dose was insufficient to fully control the symptoms of the disease the 300 mg / 4mg/kg dose was introduced by Protocol Amendment 2.

Primary Outcome Measure

Percentage of Participants With Complete Remission and Relapse After 6 Months of Canakinumab Treatment. [ Time Frame: 6 months ]

Locations (1)

Find similar trials in Bethesda, MD

Related Studies

• Natural History, Pathogenesis, and Outcome of Autoinflammatory Diseases (NOMID/CAPS, DIRA, CANDLE, SAVI, NLRC4-MAS, Still'S-like Diseases, and Other Undifferentiated Autoinflammatory Diseases)
  Recruiting · National Institute of Allergy and Infectious Diseases (NIAID) · Bethesda, Maryland