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Study on Canakinumab for Patients with PAPA Syndrome

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying a rare condition known as PAPA syndrome, which stands for pyogenic sterile arthritis, pyoderma gangrenosum, and acne. This syndrome is a potentially serious inflammatory condition that can cause joint pain, skin ulcers, and severe acne. The study will investigate the effects of a medication called Canakinumab, which is a solution for injection. Canakinumab is designed to block a specific protein in the body that plays a role in inflammation, potentially helping to reduce the symptoms of PAPA syndrome.

The purpose of this study is to provide evidence on how effective Canakinumab is in treating PAPA syndrome. Participants in the study will receive either Canakinumab or a placebo, which looks like the medication but does not contain the active ingredient. The study is designed to compare the effects of Canakinumab with the placebo to see if the medication can prevent flare-ups of the disease. The study will be conducted in a way that neither the participants nor the researchers know who is receiving the actual medication or the placebo, which is known as a double-blind study.

Participants will receive injections every four weeks and will be monitored to see how their symptoms change over time. The study aims to determine if Canakinumab can effectively prevent disease flares in those who respond to the treatment. This research is important as it seeks to find a reliable treatment option for individuals affected by PAPA syndrome, potentially improving their quality of life.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Enrollment

    Upon joining the study, a clinical diagnosis of PAPA syndrome with an active flare is required.

    A mutation of the PSTPIP1 gene must be present.

    Written informed consent is necessary for participation. For those under 18, consent from a parent or legal guardian and assent from the child, if appropriate, are required.

  2. Step 2

    Initial treatment phase

    Participants receive canakinumab, a medication administered as a solution for injection.

    The injection is given subcutaneously (under the skin).

    The dosage is 150 mg/ml.

  3. Step 3

    Randomized withdrawal phase

    Participants who respond to canakinumab enter a randomized phase where they may receive either canakinumab or a placebo.

    The placebo is a solution for injection that appears identical to canakinumab.

    The purpose is to determine if canakinumab can prevent disease flares compared to the placebo.

  4. Step 4

    Ongoing treatment

    Canakinumab or placebo is administered every 4 weeks.

    The study aims to provide evidence of the efficacy of IL-1 blockade in managing PAPA syndrome.

  5. Step 5

    Study completion

    The estimated end date for the study is July 31, 2027.

    Participants will be monitored throughout the study to assess the prevention of disease flares.

Who can join the trial?

5 criteria

  • Must have a clinical diagnosis of PAPA syndrome with an active flare at the time of joining the study. PAPA syndrome is a rare genetic condition that causes inflammation.
  • Must have a mutation in the PSTPIP1 gene. This gene is related to the condition and can be identified through genetic testing.
  • For patients 18 years or older, written informed consent is required before any study assessments. This means you agree to participate after understanding the study details.
  • For patients under 18 years, written informed consent from a parent or legal guardian is needed, along with the child's agreement if suitable.
  • Both male and female patients can participate.

Who cannot join the trial?

10 criteria

  • Patients with any other serious health condition that might interfere with the study.
  • Patients who are currently participating in another clinical trial.
  • Patients who have had a recent infection or illness that could affect the study results.
  • Patients who are pregnant or breastfeeding.
  • Patients who have a history of allergic reactions to similar medications.
  • Patients who are unable to follow the study procedures or attend the required visits.
  • Patients who have a history of drug or alcohol abuse.
  • Patients who have received certain medications that might interfere with the study.
  • Patients with a compromised immune system, which means their body's defense against infections is weakened.
  • Patients who have had a recent surgery or are planning to have surgery during the study period.
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Investigated drugs

Canakinumab is a medication used in this clinical trial to study its effectiveness in treating a condition called PAPA syndrome, which involves inflammation. It works by blocking a specific protein in the body that can cause inflammation, helping to reduce symptoms like joint pain and skin problems.

What is already known about the treatment

Canakinumab – Canakinumab is administered as an injection under the skin. It is currently being studied in clinical trials for its effectiveness in treating PAPA syndrome, a rare inflammatory condition. The main therapeutic indication for canakinumab is to reduce inflammation by blocking a protein called interleukin-1 (IL-1), which plays a key role in the body's inflammatory response. It belongs to the pharmacological class of monoclonal antibodies, specifically targeting IL-1 to help manage symptoms associated with inflammatory diseases.

Investigated diseases

PAPA syndrome – PAPA syndrome is a rare genetic disorder characterized by a combination of symptoms including arthritis, skin lesions, and inflammation. The name stands for Pyogenic Arthritis, Pyoderma gangrenosum, and Acne, which are the primary manifestations of the condition. It typically begins in childhood with episodes of joint inflammation that can lead to joint damage over time. Skin symptoms may include painful sores and severe acne, which can be persistent and difficult to manage. The condition is caused by mutations in a specific gene, leading to an abnormal inflammatory response in the body. The progression of symptoms can vary widely among individuals, with some experiencing more severe manifestations than others.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2024-517155-13-00Protocol codePAPA-CanEstimated enrolment24 patientsSponsorIRCCS Istituto Giannina Gaslini

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