BCX17725 for Netherton Syndrome

Recruiting at 3 trial locations

Overseen By BioCryst Pharmaceuticals, Inc.

Age: Any Age

Sex: Any

Trial Phase: Phase 1

Sponsor: BioCryst Pharmaceuticals

Disqualifiers: Pregnancy, Lactation, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

78 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment, BCX17725, to determine its safety and effectiveness for people with Netherton syndrome, a rare skin condition causing dry, scaly skin and hair issues. The trial will assess responses to different doses of BCX17725, administered either by IV or under the skin, over 12 weeks. Researchers seek adults and teenagers diagnosed with Netherton syndrome who regularly experience these skin problems. As a Phase 1 trial, this research aims to understand how the treatment works in people, offering participants the opportunity to be among the first to receive it.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial team or your doctor.

Is there any evidence suggesting that BCX17725 is likely to be safe for humans?

A previous study tested BCX17725 to assess its safety and tolerability. This marks the first time BCX17725 is administered to humans, so limited safety information is available. As a Phase 1 trial, researchers focus primarily on its safety and potential side effects.

No major safety issues have emerged so far, but the testing process is still in its early stages. Participants receive both single and multiple doses of BCX17725, and researchers closely monitor for any adverse reactions.

For those considering joining this trial, safety remains the top priority. Researchers take careful steps to monitor all aspects closely. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

BCX17725 is unique because it offers a new approach to treating Netherton Syndrome by targeting specific pathways differently than current options. Most treatments for this condition focus on symptomatic relief, like managing skin inflammation with topical steroids or moisturizing creams. BCX17725 could potentially alter the disease process itself, thanks to its novel mechanism that aims to address the underlying cause of the syndrome. Researchers are particularly excited about BCX17725 because it is administered in flexible dosing regimens, which might optimize its effectiveness and reduce side effects compared to standard treatments. This could be a game-changer for patients who have limited options and are looking for more effective solutions.

What evidence suggests that BCX17725 might be an effective treatment for Netherton syndrome?

Research has shown that BCX17725 addresses a protein shortage linked to Netherton syndrome by blocking KLK5, an enzyme that causes skin problems. By reducing KLK5 activity, the symptoms of Netherton syndrome may be managed. Although detailed information from human studies remains limited, the mechanism of BCX17725 appears promising. This new drug is currently being tested in this trial, with participants receiving either single or multiple doses of BCX17725 or a placebo. Initial results are expected soon, which will help determine its effectiveness for people with Netherton syndrome. ^{1 2 4 6 7}

Are You a Good Fit for This Trial?

This trial is for healthy adults and those with Netherton Syndrome. Healthy participants need a BMI of 18-30 and good kidney function (eGFR ≥ 90). Those with Netherton Syndrome must have an eGFR ≥ 60. All must be able to follow the study plan and agree to use contraception during the study.

Inclusion Criteria

I have been diagnosed with Netherton syndrome.

My BMI is between 18 and 30.

I agree to follow the study's birth control rules until 90 days after my last dose.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Single Ascending Dose (SAD)

Participants receive a single ascending dose of BCX17725 or placebo

Up to 9 weeks

Multiple Ascending Dose (MAD)

Participants receive multiple ascending doses of BCX17725 or placebo

Up to 13 weeks

Open-label Treatment

Participants with Netherton syndrome receive multiple doses of BCX17725

Up to 11 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• BCX17725

Trial Overview The trial tests BCX17725, a new medication given in single or multiple doses, against a placebo. It's designed to check how safe it is, how well tolerated it is by the body, its pharmacokinetics (how it moves through the body), and if it causes immune reactions.

How Is the Trial Designed?

Group I: Part 4 - BCX17725 multiple doses Experimental Treatment 1 Intervention

Participants will receive BCX17725 as multiple doses

Group II: Part 3 - BCX17725 multiple doses Experimental Treatment 1 Intervention

Participants will receive BCX17725 as multiple doses

Group III: Part 2 - placebo multiple doses Experimental Treatment 1 Intervention

Participants randomized to placebo will receive placebo as multiple doses

Group IV: Part 2 - BCX17725 multiple doses Experimental Treatment 1 Intervention

Participants randomized to BCX17725 will receive BCX17725 as multiple doses in sequential ascending dose cohorts

Group V: Part 1 - placebo single dose Experimental Treatment 1 Intervention

Participants randomized to placebo will receive placebo as a single dose

Group VI: Part 1 - BCX17725 single dose Experimental Treatment 1 Intervention

Participants randomized to BCX17725 will receive BCX17725 as a single dose in sequential ascending dose cohorts

Who Is Running the Clinical Trial?

BioCryst Pharmaceuticals

Lead Sponsor

Trials

55

Recruited

4,400+

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT06539507?cond=%22Netherton%20Syndrome%22&viewType=Table&rank=3

A Study of the Safety, Tolerability, Pharmacokinetics, and ... This is a first-in-human, Phase 1/1b, 4-part study that includes the evaluation of safety, tolerability, pharmacokinetics (PK), and immunogenicity of BCX17725 ...

2. ir.biocryst.com ir.biocryst.com/news-releases/news-release-details/biocryst-begins-enrollment-phase-1-trial-evaluating-bcx17725

BioCryst Begins Enrollment in Phase 1 Trial Evaluating ... BCX17725 is a protein therapeutic that is designed to treat the underlying protein deficiency that causes Netherton syndrome by inhibiting KLK5.

3. synapse.patsnap.com synapse.patsnap.com/article/biocryst-starts-phase-1-trial-of-bcx17725-for-netherton-syndrome

BioCryst Starts Phase 1 Trial of BCX17725 for Netherton ... BioCryst aims to release initial data from this trial by the end of 2025. BCX17725 represents a pioneering approach to treating Netherton ...

4. biocrystmedical.com biocrystmedical.com/netherton-syndrome/

Netherton Syndrome (NS): Clinical Studies & Publications Initial results from a Phase 1 clinical trial evaluating BCX17725 are expected in 2025. Educational resources and support. Publications on Netherton Syndrome ...

5. ichthyosis.org.uk ichthyosis.org.uk/new-clinical-trial-looking-at-a-potential-new-drug-for-treating-netherton-syndrome

New clinical trial looking at a potential new drug for treating ... A Phase 1 clinical trial looking at a potential new drug for treating Netherton syndrome has just begun in Australia. The drug is called BCX17725.

6. ctv.veeva.com ctv.veeva.com/study/a-study-of-the-safety-tolerability-pharmacokinetics-and-immunogenicity-of-bcx17725

A Study of the Safety, Tolerability, Pharmacokinetics, and ... In Part 4, the effectiveness, safety, and tolerability of BCX17725 when administered via multiple IV and/or SC doses through 12 weeks will be ...

7. biocrystmedical.com biocrystmedical.com/clinical-trials/

Hereditary Angioedema (HAE) & Netherton Syndrome (NS) ... Phase 1 study evaluating BCX17725 (Active, Recruiting). NCT06539507: This is a first-in-human, Phase 1, three-part study that includes the evaluation of safety, ...