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New drug trial hopes to tame rare skin disease

NCT ID NCT06539507

Disease control Sponsor: BioCryst Pharmaceuticals Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 25, 2026 · Last updated Aug 28, 2026 · Updated 3 times

Summary

This early-stage trial tests a new drug called BCX17725 in healthy volunteers and people with Netherton syndrome, a rare genetic skin condition. The study aims to check safety, how the drug moves through the body, and whether it can reduce skin redness and scaling. About 78 participants will take part across four parts, with the final part also looking at effectiveness over 12 weeks.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
BCX17725
What this could lead to
If successful, this could point toward a treatment that controls the severe skin symptoms of Netherton syndrome.
What could go wrong
This is a very early Phase 1 trial, so safety and effectiveness are not yet known. The drug may not work or could have side effects.
Why investors are watching

BioCryst is testing BCX17725, a first-in-human drug, in healthy people and in patients with Netherton syndrome, a rare skin disease. For a small company, this early-stage readout matters because it is the first look at whether the drug is safe and shows any sign of working. A clear result could shape the company's pipeline value.

If it works: If the drug proves safe and shows benefit in Netherton syndrome, BioCryst could advance it to later-stage trials. That would give the company a new product candidate in a rare disease with few treatment options.

If it fails: Phase 1 trials often fail on safety or tolerability, and this one is no exception. A poor result or delay could set the program back and leave BioCryst without a clear next step for BCX17725.

AI-written from the trial record. Speculative, and not investment advice.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1

The first testing in people. Mainly checks safety and dose, usually in a small group.

Participants

About 78 people

The number the study aims to enrol. It can still change while the study runs.

Started

Sep 2024

Expected to finish

Dec 2026

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

12 to 65 years

Sex

Anyone

Healthy volunteers

Accepted

You do not need to have the condition being studied to take part.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Key Inclusion Criteria: * Male or non-pregnant, non-lactating female aged 18 to 55 years, inclusive (Parts 1 and 2), 18 to 65 years, inclusive (Part 3), or 12 to 65 years, inclusive (Part 4) * Confirmed diagnosis of Netherton syndrome (Parts 3 and 4) * IGA score of ≥ 3 (Parts 3 and 4) and IASI score of ≥ 16 (Part 4) * BMI between 18 and 30 kg/m\^2, inclusive (Parts 1 and 2) * Estimated glomerular filtration rate (eGFR) of ≥ 90 mL/min/1.73 m\^2 (Parts 1 and 2) or ≥ 60 mL/min/1.73 m\^2 (Part 3) * Agree to follow the protocol contraception requirements from screening until 90 days after the last dose of study drug * In the opinion of the investigator, expected to adequately comply with all required study procedures and restrictions for the duration of the study

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Conditions

The condition(s) this trial relates to.

Netherton syndrome

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Netherton syndrome

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    11 sites in 5 countries. The list below names each one and where it is.

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  4. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Study contacts

  • Contact

    Email: •••••@•••••

Locations

  • Dawes Fretzin Clinical Research Group, LLC

    RECRUITING

    Indianapolis, Indiana, 46250, United States

  • Erasmus Universitair Medisch Centrum (EMC)

    RECRUITING

    Rotterdam, 3015 GD, Netherlands

  • Hôpital Saint-Louis

    RECRUITING

    Paris, 75010, France

  • Maastricht Universitair Medisch Centrum (MUMC+)

    RECRUITING

    Maastricht, 6229 HX, Netherlands

  • Northwestern Dermatology CTU

    RECRUITING

    Chicago, Illinois, 60611, United States

  • Nucleus Network

    COMPLETED

    Brisbane, Queensland, 4006, Australia

  • Stanford University School of Medicine

    RECRUITING

    Palo Alto, California, 94304, United States

  • Therapeutics Clinical Research

    RECRUITING

    San Diego, California, 92123, United States

  • Universitätsklinikum Heidelberg

    NOT_YET_RECRUITING

    Heidelberg, 69120, Germany

  • Veracity Clinical Research

    RECRUITING

    Brisbane, Queensland, 4102, Australia

    Contact Email: •••••@•••••

  • Westmead Hospital - Department of Dermatology

    RECRUITING

    Sydney, New South Wales, 2145, Australia

  • Yale Center for Clinical Investigation

    RECRUITING

    New Haven, Connecticut, 06519, United States

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