Skip to content
Clinical Trials – home

Rare diseases

Investigational molecules

Locations

Our offerNewsroom

Not recruitingRare disease

Study to Evaluate the Effectiveness of Spesolimab in Treating Patients with Netherton Syndrome

Verified siteInvestigational
Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

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 skin condition known as Netherton syndrome. This condition is a genetic disorder that affects the skin, hair, and immune system, often leading to red, scaly skin and other complications. The trial is testing a treatment called spesolimab, which is a type of medication known as a monoclonal antibody. Monoclonal antibodies are proteins made in a lab that can bind to specific targets in the body, potentially helping to treat certain diseases.

The purpose of this study is to evaluate how well spesolimab works in treating patients with Netherton syndrome. Participants in the study will receive either spesolimab or a placebo. The medication will be administered in two forms: as a solution for infusion, which is given through a vein, and as a solution for injection, which is given under the skin. The study will last for a period of time, during which participants will receive the treatment and be monitored for any changes in their condition.

Throughout the study, researchers will observe the participants to see if there is an improvement in their skin condition, such as a reduction in redness and scaling. They will also monitor for any side effects or adverse reactions to the treatment. The goal is to determine if spesolimab is a safe and effective treatment option for people with Netherton syndrome.

The research process

The trial runs in 4 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Initial visit

    Upon joining the study, you will attend an initial visit. During this visit, your eligibility will be confirmed based on specific criteria, such as age, weight, and a confirmed diagnosis of Netherton syndrome.

    You will be asked to provide written informed consent, which means you agree to participate in the study after understanding all the details.

  2. Step 2

    Treatment phase

    During the treatment phase, you will receive the study medication, spesolimab, or a placebo. A placebo is a substance with no active medication, used to compare the effects of the actual drug.

    The medication will be administered in two forms: as a solution for infusion (intravenous use) and as a solution for injection (subcutaneous use). The specific dosage and frequency will be explained to you by the study team.

  3. Step 3

    Monitoring and follow-up

    Throughout the study, your health and response to the treatment will be closely monitored. This includes regular check-ups and assessments to evaluate the effectiveness and safety of the treatment.

    You will be asked to report any changes in your condition or any side effects you experience. This information is crucial for understanding the treatment's impact.

  4. Step 4

    End of study

    At the end of the study, a final evaluation will be conducted to assess your overall response to the treatment.

    The study team will discuss the results with you and provide any necessary follow-up care or recommendations.

Who can join the trial?

5 criteria

  • Patients must be male or female and at least 12 years old, weighing at least 35 kg (about 77 pounds).
  • Patients must have a confirmed diagnosis of Netherton syndrome, which is linked to specific genetic changes called SPINK5 mutations.
  • Patients must have at least moderate severity of skin redness, with specific scores on medical assessments (IASI score of 16 or higher, IASI-E score of 8 or higher, and IGA score of 3 or higher).
  • Patients must provide a signed and dated written informed consent and, if applicable, assent, which means they agree to participate in the trial after understanding the details.
  • Women of childbearing potential must be willing and able to use highly effective birth control methods that have a very low chance of failure (less than 1% per year) when used correctly. Details about these methods are available in the trial information.

Who cannot join the trial?

10 criteria

  • Patients with any other skin condition that might interfere with the study.
  • Patients who have had a serious infection in the past 4 weeks.
  • Patients who have received any other investigational drug within the last 30 days.
  • Patients who are pregnant or breastfeeding.
  • Patients with a history of severe allergic reactions to any medication.
  • Patients with any significant medical condition that the study doctor thinks might make it unsafe for them to participate.
  • Patients who have been vaccinated with a live vaccine in the past 4 weeks.
  • Patients who have a history of drug or alcohol abuse in the past year.
  • Patients who are currently participating in another clinical trial.
  • Patients who have a known history of HIV, hepatitis B, or hepatitis C infection.
Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

Tell us about your condition – we search every trial in Europe and connect you with the right site.

We usually reply within a few days

Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Trial locations

Where you can join this trial

Countries are shaded by recruitment status. Click a recruiting country to ask about joining there.

Not recruiting
Not finding your country?

Not sure what to do next?

Joining a clinical trial can seem overwhelming. We guide you step by step, so you know exactly what to expect and how we support you through the process.

See the full process and FAQ

Investigated drugs

Spesolimab is a medication being studied for its potential to help people with Netherton syndrome, a rare skin condition. This medication works by targeting specific parts of the immune system that may be overactive in people with this condition. By doing so, it aims to reduce inflammation and improve the symptoms associated with Netherton syndrome, such as skin redness, scaling, and itching. The goal of the trial is to see how well spesolimab can help manage these symptoms and to ensure it is safe for patients to use.

What is already known about the treatment

Spesolimab – Spesolimab is administered as an injection, typically under the skin, and is currently being studied in clinical trials for its effectiveness and safety in treating Netherton syndrome, a rare genetic skin disorder. In the medical literature, spesolimab is being explored for its potential benefits in managing this condition, although it is not yet widely used in standard medical practice. The main therapeutic indication for spesolimab is to help alleviate symptoms associated with Netherton syndrome. At the molecular level, spesolimab works by blocking certain proteins in the immune system that contribute to inflammation and skin issues. It is classified pharmacologically as a monoclonal antibody, which is a type of medication designed to target specific molecules in the body.

Investigated diseases

Netherton syndrome – Netherton syndrome is a rare genetic disorder that affects the skin, hair, and immune system. It is characterized by red, scaly skin, known as ichthyosis, and a condition called trichorrhexis invaginata, where the hair shafts are brittle and prone to breakage. The skin condition often leads to chronic inflammation and can be accompanied by severe itching. Individuals with Netherton syndrome may also experience recurrent infections due to an impaired immune response. The disease is present from birth and continues throughout life, with symptoms that can vary in severity. It is caused by mutations in the SPINK5 gene, which affects the skin's protective barrier.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IVTrial ID2022-501104-10-00Protocol code1368-0104Estimated enrolment36 patientsSponsorBoehringer Ingelheim Espana S.A.

sourced from the EU Clinical Trials Register and site verification

Want to learn more about this trial or check if you can participate?

Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

Tell us about your condition – we search every trial in Europe and connect you with the right site.