Study on the Safety of DS-2325a 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 study is testing a potential new treatment called DS-2325a, which is a type of biological medicine known as a fusion protein. The trial will also use a placebo for comparison. The main goal of the study is to explore the safety and tolerability of DS-2325a in patients with Netherton Syndrome.

Participants in the study will receive either the new treatment or a placebo through injections or infusions. The study will monitor participants for any side effects and gather information on how the body processes the treatment. This includes checking for any reactions at the injection site, changes in vital signs like blood pressure and heart rate, and results from blood and urine tests. The study will also look at how the treatment affects the skin and overall quality of life for those with Netherton Syndrome.

The trial is designed to be double-blind, meaning neither the participants nor the researchers will know who is receiving the actual treatment or the placebo. This helps ensure the results are unbiased. The study will continue for a set period, during which participants will have regular check-ups and assessments to track their progress and any changes in their condition.

1 initial visit

Upon joining the study, you will attend an initial visit. During this visit, you will provide written consent to participate in the study. This is a necessary step to ensure you understand the study and agree to take part.

Your medical history will be reviewed, and a physical examination will be conducted. This includes checking your vital signs such as body temperature, blood pressure, heart rate, and respiratory rate.

Blood and urine samples will be collected for safety laboratory analyses. An electrocardiogram (ECG) will also be performed to check your heart’s activity.

2 treatment phase

You will receive the study medication, DS-2325a, which is a solution for injection or infusion. The medication can be administered either subcutaneously (under the skin) or intravenously (into a vein).

The dosage, frequency, and duration of administration will be determined by the study protocol and communicated to you by the study team.

You will also receive a sodium chloride solution 0.9% as part of the treatment process.

3 monitoring and assessments

Throughout the study, you will be monitored for any adverse events, including serious adverse events and injection site reactions. Regular physical examinations will be conducted to ensure your safety.

Additional blood and urine samples will be collected periodically to monitor your health and the effects of the medication.

You will be asked to complete questionnaires such as the Itch Numerical Rating Scale (NRS) and quality-of-life assessments using the Skindex-29 and the Dermatology-Life-Quality-Index (DLQI) to evaluate the impact of the treatment on your condition.

4 optional skin biopsies

If you agree, optional skin biopsies may be taken to assess the presence of DS-2325a in the skin. This is an optional part of the study and will be discussed with you in detail.

5 end of study

At the end of the study, a final assessment will be conducted. This will include a physical examination, collection of blood and urine samples, and completion of any remaining questionnaires.

The study team will review all the data collected during the study to evaluate the safety and effectiveness of the treatment.

Who Can Join the Study?

Male or female patients aged 18 to 65 years.
Must have a clinical diagnosis of Netherton’s Syndrome (NS), which includes at least 3 out of the following 4 criteria:
- Neonatal erythroderma: Redness of the skin present at birth.
- Bamboo hair and/or alopecia: Fragile hair that breaks easily and/or hair loss.
- Chronic atopy: Long-term allergic conditions such as food allergy, asthma, rhino-conjunctivitis (allergy affecting the nose and eyes), or eczema for at least 2 years.
- Ichthyosis linearis circumflexa or scaling erythroderma: Specific types of skin scaling or redness.
Must have received previous treatment for NS that did not work and need further care.
Must have documentation showing the absence of a specific protein called LEKTI in the skin or confirmed mutations in the SPINK5 gene.
NS must affect at least 20% of the body surface area at both the initial screening and the start of the study.
Must provide written informed consent to participate in the study before the screening process begins.
Must be willing to have skin samples collected from both affected and unaffected skin areas using a tape method.

Who Cannot Join the Study?

Patients who are not diagnosed with Netherton’s Syndrome cannot participate. Netherton’s Syndrome is a rare genetic skin disorder.
Patients who are younger than 3 years old cannot participate.
Patients who are not able to understand or follow the study procedures cannot participate.
Patients who have other serious health conditions that might interfere with the study cannot participate.
Patients who are pregnant or breastfeeding cannot participate.
Patients who have participated in another clinical trial recently cannot participate.
Patients who are allergic to the study medication or its ingredients cannot participate.

Where you can join this trial?

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Other Sites

Site Name	City	Country	Status
Ajjjozurwr Pqsfjjhz Htrferjr Dz Pyqje	Paris	France

Trial status

Country	Status	Recruitment Start
France	Not recruiting	29.06.2023

Trial locations

Investigated drugs:

DS-2325a is a medication being studied for its potential to help people with Netherton Syndrome, a rare skin condition. The main goal of this trial is to see if DS-2325a is safe and can be tolerated by patients. Researchers are also looking to see if it shows any early signs of being effective in treating the symptoms of Netherton Syndrome. This medication is being tested to understand how it behaves in the body and to gather information on its possible benefits for patients with this condition.

Investigated diseases:

Netherton's syndrome

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 hair abnormalities such as brittle hair shafts. The condition often presents in infancy with widespread skin inflammation and scaling. As the child grows, the skin symptoms may persist, and individuals may experience recurrent infections due to compromised skin barrier function. The syndrome is caused by mutations in the SPINK5 gene, which affects the production of a protein important for skin and hair health. Over time, the skin may become thickened and more prone to irritation and infection.

Trial ID:

2022-502853-32-00

Protocol code:

DS2325-119

NCT ID:

NCT05979831

Trial Phase:

Human Pharmacology (Phase I) – Other

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Study on the Safety of DS-2325a for Patients with Netherton Syndrome

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