Ongoing, recruitingPhase I and Phase II (Integrated)- OtherInterventional

A Phase 1b/2, Double-Blind, Placebo-Controlled, Randomized, Parallel-Arm Study to Explore Safety, Pharmacokinetics, and Early Clinical Signal of Efficacy of DS-2325a in Patients with Netherton Syndrome

This research study is for people with Netherton Syndrome and is designed to test a new medicine called DS-2325a. It's a 'Phase 1b/2' study, which means we're looking closely at how safe the medicine is for people, how it moves through the body, and if there are any early hints that it could help improve the condition. Some participants will receive the actual medicine, while others will receive a placebo (a substance that looks like the medicine but has no active ingredients). This helps us understand the medicine's true effects. We'll be carefully checking for any side effects and how people feel during the study, as well as measuring changes in their skin and other symptoms.

At a glance

Status

Ongoing, recruiting

Phase

Phase I and Phase II (Integrated)- Other

Sponsor

Daiichi Sankyo Inc.

Enrolment target

Start

17 Jul 2023

Netherton's Syndrome

What is this study about?

You've found information about a research study looking into a potential new treatment for Netherton Syndrome. Netherton Syndrome is a rare skin condition that can cause red, scaly skin and other symptoms. This particular study is testing a new medicine called DS-2325a.

The main goals of this study are to understand how safe DS-2325a is for people with Netherton Syndrome and how their bodies handle the medicine. Researchers will also be looking for any early signs that the medicine might improve the symptoms of Netherton Syndrome. This study uses a method where some people will get the active medicine, DS-2325a, and others will get a placebo. A placebo looks exactly like the medicine but contains no active ingredients. This is a common and important way to fairly compare the new medicine's effects.

Throughout the study, the research team will keep a close eye on participants. They'll check for any side effects, including serious ones, and monitor vital signs like temperature and blood pressure. They'll also review blood and urine tests and heart readings. To see if the medicine is working, they'll measure things like skin redness and scaling, and ask participants about their itching and how the condition affects their daily life. All of these checks help the researchers gather vital information about DS-2325a.

Key takeaways

This study is testing a new medicine (DS-2325a) for Netherton Syndrome.
It aims to check the medicine's safety and early signs of effectiveness.
Participants will receive either the medicine or a placebo (dummy treatment).
Close monitoring for side effects and symptom changes will happen throughout.
It's for adults aged 18 and over, both men and women.
You can withdraw from the study at any time without affecting your normal care.

Who may be eligible?

To join this study, you need to be at least 18 years old. Both men and women are welcome to participate. Researchers are looking for people who have Netherton Syndrome.

There might be other specific health requirements or conditions that would qualify or disqualify someone from joining. For example, some existing health problems or medications might mean someone can't take part, as they could affect the safety of the new medicine or the accuracy of the study results.

If you're interested, it's really important to talk to the study doctors or your own doctor. They can give you all the details about what's needed to join and help you decide if it's right for you.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you 18 years old or older?
Do you have Netherton Syndrome?
Are you able to attend regular clinic visits?
Are you comfortable with injections?
Are you willing to potentially receive a placebo?
Are you able to understand and agree to the study requirements?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you will be given either the new medicine, DS-2325a, or a placebo (a dummy medicine with no active ingredients). You won't know which one you're getting, and neither will the study team – this is called 'double-blind'. Both medicines will be given as injections.

Throughout the study, you'll have regular visits to the clinic. During these visits, the study team will carry out several assessments. This includes physical check-ups, taking your temperature, blood pressure, heart rate, and breathing rate. They will also take blood and urine samples for tests and perform an ECG (a heart tracing). In addition, they will carefully check your skin for any changes in redness or scaling, and you'll be asked about your symptoms, like itching, and how your condition affects your daily life by filling out questionnaires. Some participants might also be asked if they are willing to have small skin biopsies taken. The total length of your participation in the study, including the number of visits and follow-up, will be fully explained to you by the study team.

Potential risks and benefits

Taking part in any study has potential benefits and risks. You might not directly benefit from this study, but the information gained could help others with Netherton Syndrome in the future. The medicine, DS-2325a, is new, so there's a chance it could cause side effects or unexpected reactions. The study team will closely monitor you and tell you about any known risks. You can choose to stop participating in the study at any time, for any reason, without it affecting your usual medical care.

Locations (1)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

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Unverified
France

Common questions

What is Netherton Syndrome?

Netherton Syndrome is a rare genetic skin condition that causes red, scaly skin, especially from birth, and can also affect hair and cause other health issues.

What does 'double-blind' mean?

Double-blind means neither you nor the doctors treating you will know if you are receiving the active medicine or the placebo. This helps ensure the study results are fair and unbiased.

What is a placebo?

A placebo is a substance that looks exactly like the study medicine but contains no active ingredients. It's used to compare against the real medicine.

Will I have to pay to join the study?

No, you will not have to pay to participate in the study. All study-related care and medicines are provided free of charge.

Can I leave the study if I change my mind?

Yes, you can choose to leave the study at any time, for any reason. Your decision will not affect your medical care.