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

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What is this study about?

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.

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 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 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 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 Study?

  • 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 Study?

  • 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.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name City Country Status
Centro Hospitalar Universitario Lisboa Central E.P.E. Lisbon Portugal
Universitaetsklinikum Heidelberg AöR Heidelberg Germany
Katholieke Universiteit te Leuven Leuven Belgium

Other Sites

Site Name City Country Status
Fakultni Nemocnice Brno Brno Czechia
Azienda Ospedaliera Universitaria Meyer IRCCS Florence Italy
Fondazione Luigi Maria Monti Rome Italy
Hospital Sant Joan De Deu Barcelona Esplugues De Llobregat Spain
Universitaetsklinikum Schleswig-Holstein AöR Kiel Germany
Ospedale Pediatrico Bambino Gesu’ Rome Italy
ASMC – IPSMC – Skin And Venereal Diseases Sofia Bulgaria
Uniklinikum Salzburg Salzburg Austria
Suomen Terveystalo Oy Tampere Finland
Auibhkpijj Pskrjqkf Htezgsms Dl Pefbw Paris France
Evtsaoo Uydhsvbseuim Mwksefu Cowerav Rwxghzijd (xjeezob Myk Rotterdam The Netherlands
Lvsgej Mjprucydyk Unhluagxfn Ot Msgwda Munich Germany
Amkkffa Oiaoawmrufx Uhxtzuethptsc Cnqjlzolpbgd Dhwrz Svfpsd E Drxdt Syvogwt Di Tdmeqe Turin Italy
Fnzlyuefk Pfhc Lp Ikobfvpxfyyed Buwohjdwk Dhm Htzacwku Ustzoprhypqjx Ls Ppj Madrid Spain

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
Austria Austria
Not recruiting
15.05.2023
Belgium Belgium
Not recruiting
15.05.2023
Bulgaria Bulgaria
Not recruiting
15.05.2023
Czechia Czechia
Not recruiting
15.05.2023
Finland Finland
Not recruiting
15.05.2023
France France
Not recruiting
15.05.2023
Germany Germany
Not recruiting
15.05.2023
Italy Italy
Not recruiting
15.05.2023
Portugal Portugal
Not recruiting
15.05.2023
Spain Spain
Not recruiting
15.05.2023
The Netherlands The Netherlands
Not recruiting
15.05.2023

Trial locations

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.

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 ID:
2022-501104-10-00
Protocol code:
1368-0104
Trial Phase:
Therapeutic use (Phase IV)

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