Study on the Safety and Effectiveness of SXR1096 Cream for Patients with Netherton Syndrome

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What is this study about?

This clinical trial is focused on studying Netherton syndrome, a rare genetic skin disorder. The treatment being tested is a cream called SXR1096, which is applied directly to the skin. This cream is designed to target specific proteins in the skin that are involved in the symptoms of Netherton syndrome. The purpose of the study is to evaluate the safety and effectiveness of the SXR1096 cream compared to a placebo in patients with this condition.

The study will involve participants using the cream for a period of time, with regular check-ups to monitor their health and the condition of their skin. Participants will apply the cream to affected areas of their skin, and researchers will assess any changes in their symptoms. The study aims to determine if the cream can improve the skin condition and reduce symptoms such as redness, scaling, and itching.

Throughout the study, participants will be closely monitored for any side effects or changes in their health. The study will also measure various aspects of skin health, such as the severity of skin symptoms and the skin’s moisture levels. This research is important for understanding how SXR1096 cream can help people with Netherton syndrome and potentially improve their quality of life.

1 initial treatment phase

The study involves the application of a cream containing the active substance sxr1096. This cream is applied topically, meaning it is used directly on the skin.

The treatment is designed for individuals diagnosed with Netherton syndrome, a condition affecting the skin.

2 application of cream

The cream is applied to specific areas of the skin. Each target area is approximately 9% of the body surface area, similar to the size of one arm.

The application is done as instructed by the study guidelines, ensuring consistent use throughout the trial period.

3 monitoring and assessment

Regular assessments are conducted to monitor safety and effectiveness. This includes checking for any adverse effects and evaluating the condition of the skin.

The primary focus is on changes in the skin’s appearance and condition, using a scoring system to measure improvements or changes.

4 completion of treatment

The study aims to conclude by April 30, 2024. Participants will continue to apply the cream and attend scheduled assessments until the end of the trial.

Final evaluations will be conducted to determine the overall effectiveness and safety of the treatment.

Who Can Join the Study?

Patients must be male or female, aged 18 to 65 years. Adolescents aged 12-17 years can join only after an initial group of 5 adult patients have been treated for at least 7 days.
Patients must agree to provide written informed consent. For adolescents, a legal guardian must explain the consent information to ensure understanding.
Patients must have a clinical diagnosis of Netherton syndrome (NS), meeting at least 3 of the following criteria:
- Neonatal erythroderma: A skin condition present at birth.
- Bamboo hair and/or alopecia: Hair that looks like bamboo and/or hair loss.
- Chronic atopy: Long-term conditions like food allergy, asthma, rhino conjunctivitis, and/or eczema for at least 2 years.
- Ichthyosis linearis circumflexa: A specific skin condition.
Patients must be willing and able to understand and follow study requirements, apply the medication as instructed, and complete the study.
Patients must have absent LEKTI on a skin biopsy test and/or a confirmed mutation in the SPINK5 gene.
NS must affect at least 20% of the patient’s body surface area at both the screening and baseline visits.
The Investigator Global Assessment (IGA) score for two areas to be treated must be 3 or higher, indicating moderate or severe condition. Each target area should be about 9% of the body surface area, similar to the size of one arm.
Females who can have children must either commit to true abstinence (not having heterosexual intercourse) during the trial or use a highly effective method of contraception throughout the study and for 4 weeks after the last study drug application. This also applies to prepubertal females who start menstruating during the study. Acceptable methods include:
- Progestogen-only hormonal contraception (oral, transdermal, injectable, or implantable) that stops ovulation.
- Combined hormonal contraception (containing estrogen and progestogen) taken orally or transdermally.
- Injectable or implanted hormonal contraception.
- Intrauterine devices or hormone-releasing systems.
- Bilateral tubal ligation or tube insert (like the Essure system) done at least 3 months before the study.
- Partner’s vasectomy, confirmed to be the only sexual partner, done at least 3 months before the study with confirmed success.
Females who cannot have children must meet one of the following criteria:
- No menstrual bleeding for 1 year before screening without any other medical reason.
- Documented hysterectomy, bilateral salpingectomy, or bilateral oophorectomy done at least 3 months before screening.
- For adolescents not yet post-pubertal, eligibility requires evaluation by a qualified medical practitioner using an acceptable sexual maturity rating scale (e.g., Tanner).

Who Cannot Join the Study?

Patients who are not diagnosed with Netherton syndrome (NS) cannot participate. Netherton syndrome is a rare genetic skin disorder.
Patients who are younger than 3 years old cannot participate.
Patients who are part of a vulnerable population cannot participate. A vulnerable population includes groups who may have limited ability to protect their own interests.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country	Status
Medizinische Universitaet Innsbruck	Innsbruck	Austria

Other Sites

Site Name	City	Country
Assistance Publique Hopitaux De Paris	Paris	France
Medical Center – University Of Freiburg	Freiburg Im Breisgau	Germany
Karolinska Institutet	Stockholm	Sweden
Aessqdirrf Pqtotfml Hwyetwia Dj Plvyo	Paris	France

Trial status

Country	Status	Recruitment Start
Austria	Not recruiting	10.08.2021
France	Not recruiting	10.08.2021
Germany	Not recruiting	10.08.2021
Sweden	Not recruiting	10.08.2021

Trial locations

Investigated drugs:

Sxr1096

SXR1096 Cream is a topical medication being tested for its safety and effectiveness in treating Netherton syndrome, a rare genetic skin disorder. This cream is applied directly to the skin and is being studied to see if it can help improve the symptoms associated with this condition, such as skin redness, scaling, and irritation. The trial aims to determine if this new treatment can provide relief for patients suffering from Netherton syndrome.

Investigated diseases:

Netherton's syndrome

Netherton Syndrome – Netherton Syndrome is a rare genetic disorder that primarily affects the skin, hair, and immune system. It is characterized by red, scaly skin, often present from birth, and a condition known as ichthyosis linearis circumflexa, which involves circular, scaly patches. Individuals with this syndrome may also have fragile hair that breaks easily, known as trichorrhexis invaginata or “bamboo hair.” The skin issues can lead to increased susceptibility to infections and may cause discomfort due to itching. The condition is caused by mutations in the SPINK5 gene, which affects the skin’s protective barrier. Symptoms can vary in severity and may change over time.

Trial ID:

2023-507743-11-00

Protocol code:

SXR001

Trial Phase:

Phase I and Phase II (Integrated) – First administration to humans

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Study on the Safety and Effectiveness of SXR1096 Cream for Patients with Netherton Syndrome

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