Netherton SyndromePhase 1NCT06539507
A Study of the Safety, Tolerability, Pharmacokinetics, and Immunogenicity of BCX17725
A Phase 1 study testing BCX17725 in people with Netherton Syndrome, run by BioCryst Pharmaceuticals at 12 sites in 5 countries.
Trial summary
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 when administered via single and multiple doses in healthy adult participants (Parts 1 and 2), and multiple doses in adult participants with Netherton syndrome… Read more about this study
- Who is it for?
- Ages 12 to 65 yearsHealthy volunteers can take part
- Conditions the study is for
- Netherton Syndrome
- What is being tested?
- BCX17725
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 76 participants
- Where
- 12 sitesin 5 countries
- Study dates
- 26 Sep 2024to Feb 2027
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 to 65 years
- All sexes
- Healthy volunteers accepted
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
None listed.
Trial locations and contacts
12 sites in 5 countries.
12 sites in 5 countries
United States5 sites
Northwestern Dermatology CTU
Chicago, Illinois, 60611
Dawes Fretzin Clinical Research Group, LLC
Indianapolis, Indiana, 46250
Yale Center for Clinical Investigation
New Haven, Connecticut, 06519
Stanford University School of Medicine
Palo Alto, California, 94304
Therapeutics Clinical Research
San Diego, California, 92123
Australia3 sites
Nucleus Network
Brisbane, Queensland, 4006
Veracity Clinical Research
Brisbane, Queensland, 4120
Westmead Hospital - Department of Dermatology
Sydney, New South Wales, 2145
Netherlands2 sites
Maastricht Universitair Medisch Centrum (MUMC+)
Maastricht, 6229 HX
Erasmus Universitair Medisch Centrum (EMC)
Rotterdam, 3015 GD
About this study
Official title: A Phase 1/1b Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Immunogenicity of Single and Multiple Ascending Doses of BCX17725 in Healthy Participants and Multiple Doses of BCX17725 in Participants With Netherton Syndrome
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 when administered via single and multiple doses in healthy adult participants (Parts 1 and 2), and multiple doses in adult participants with Netherton syndrome (Part 3). In Part 4, the effectiveness, safety, and tolerability of BCX17725 when administered via multiple IV and/or SC doses through 12 weeks will be evaluated in adult and adolescent participants with Netherton syndrome.
Read the detailed description
Parts 1 and 2 are randomized, placebo-controlled, single-ascending-dose (SAD) and multiple-ascending-dose (MAD) study parts, respectively, in healthy participants. Part 3 will evaluate multiple dose administrations in participants with Netherton syndrome in an open-label design. Part 4 will evaluate multiple administrations of BCX17725 in participants with Netherton syndrome in an open-label study design over 12 weeks, with an 8-week post-treatment follow-up period.
- Conditions
- Netherton Syndrome
- Being tested
- BCX17725, Placebo
- Keywords
- First-in-human, Single ascending dose, Multiple ascending dose, Pharmacokinetics, Placebo control, Netherton syndrome
- Medical subject headings
- Netherton Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BCX17725Drug
BCX17725 for injection
PlaceboDrug
Placebo for injection
| Study group | Role | What they receive |
|---|---|---|
| Part 1 - BCX17725 single dose | Experimental | Participants randomized to BCX17725 will receive BCX17725 as a single dose in sequential ascending dose cohorts Receives: Drug: BCX17725 |
| Part 1 - placebo single dose | Experimental | Participants randomized to placebo will receive placebo as a single dose Receives: Drug: Placebo |
| Part 2 - BCX17725 multiple doses | Experimental | Participants randomized to BCX17725 will receive BCX17725 as multiple doses in sequential ascending dose cohorts Receives: Drug: BCX17725 |
| Part 2 - placebo multiple doses | Experimental | Participants randomized to placebo will receive placebo as multiple doses Receives: Drug: Placebo |
| Part 3 - BCX17725 multiple doses | Experimental | Participants will receive BCX17725 as multiple doses Receives: Drug: BCX17725 |
| Part 4 - BCX17725 multiple doses | Experimental | Participants will receive BCX17725 as multiple doses Receives: Drug: BCX17725 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of treatment-emergent adverse events (TEAEs)
Incidence of TEAEs as assessed by Common Terminology Criteria for Adverse Events (CTCAE) from screening through end-of-study (EOS) in each study part
Measured: From screening through EOS (ie, through Day 78 in Parts 1 and 3, and Day 106 in Part 2)
Change from baseline in Ichthyosis Area and Severity Index (IASI) score at Week 12 (Part 4)
IASI measures the severity of erythema (IASI-E) and scaling (IASI-S); the maximum sub-scores for the IASI-E and IASI-S being 24, and the maximum total IASI score being 48. Higher scores indicate worse clinical outcome.
Measured: From baseline to Week 12
Secondary outcomes (9)
Maximum observed serum concentration (Cmax)
Cmax of BCX17725
Measured: Up to Day 64 (Part 1), Day 92 (Part 2), and Day 78 (Part 3)
Time to maximum observed serum concentration (Tmax)
Time to Cmax of BCX17725
Measured: Up to Day 64 (Part 1), Day 92 (Part 2), and Day 78 (Part 3)
Area under the serum concentration-time curve (AUC) from time 0 to the time of last measurable concentration (AUC0-t)
AUC0-t of BCX17725
Measured: Up to Day 64 (Part 1), Day 92 (Part 2), and Day 78 (Part 3)
Terminal elimination half-life (t1/2)
Terminal elimination half-life (t1/2) of BCX17725
Measured: Up to Day 64 (Part 1), Day 92 (Part 2), and Day 78 (Part 3)
Number of participants who are anti-drug antibody (ADA)-positive (baseline and post-baseline) and number of participants who have treatment-emergent ADAs
Incidence of ADAs to BCX17725
Measured: Day 1 pre-dose and up to Day 64 (Part 1), Day 92 (Part 2), and Day 78 (Part 3)
Change from baseline in Investigator Global Assessment (IGA) score at Week 12 (Part 4)
IGA assesses the overall severity of a participant's NS skin disease based on a 5-point scale (0, clear; 1, almost clear; 2, mild; 3, moderate; and 4, severe). Higher scores indicate worse clinical outcome.
Measured: From baseline to Week 12
Change from baseline in Worst Itch Numerical Rating Score (NRS) at Week 12 (Part 4)
Worst Itch Numerical Rating Scale (NRS) is a self-rated, single-item scale designed for assessing the worst itch in the past 7 days. The scale uses an 11-point NRS, scored from 0 (no itch) to 10 (worst possible itch). Higher scores indicate worse clinical outcome.
Measured: From baseline to Week 12
Incidence of TEAEs (Part 4)
Incidence of TEAEs as assessed by CTCAE
Measured: From baseline through Week 20
Serum concentrations of BCX17725 (Part 4)
Serum concentrations of BCX17725
Measured: From baseline through Week 20
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Aug 2024
- Study start26 Sep 2024
- Primary completionDec 2026 (estimated)
- Study completionFeb 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Netherton SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Model description
- In Parts 1 and 2, participants will be randomized to BCX17725 or placebo under double-blind conditions. In Parts 3 and 4, participants will receive open-label BCX17725.
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 76 participants (actual)
Sponsor and investigators
- Lead sponsor
- BioCryst Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06539507
- Sponsor's protocol ID
- BCX17725-101
- Other IDs
- U1111-1303-9510, 2025-521973-16, 2025
- Status last verified
- Nov 2026
- First submitted
- 30 Jul 2024
- Last updated
- 6 Nov 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 Nov 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1/1b Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Immunogenicity of Single and Multiple Ascending Doses of BCX17725 in Healthy Participants and Multiple Doses of BCX17725 in Participants With Netherton Syndrome
- ClinicalTrials.gov ID
- NCT06539507
- Acronym / sponsor ID
- BCX17725-101
- Conditions
- Netherton Syndrome
- Keywords
- First-in-human, Single ascending dose, Multiple ascending dose, Pharmacokinetics, Placebo control, Netherton syndrome
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 76 participants (actual)
- Lead sponsor
- BioCryst PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 26 Sep 2024 (actual)
- Primary completion
- Dec 2026 (estimated)
- Study completion
- Feb 2027 (estimated)
- First posted
- 6 Aug 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 when administered via single and multiple doses in healthy adult participants (Parts 1 and 2), and multiple doses in adult participants with Netherton syndrome (Part 3). In Part 4, the effectiveness, safety, and tolerability of BCX17725 when administered via multiple IV and/or SC doses through 12 weeks will be evaluated in adult and adolescent participants with Netherton syndrome.
Detailed description
Parts 1 and 2 are randomized, placebo-controlled, single-ascending-dose (SAD) and multiple-ascending-dose (MAD) study parts, respectively, in healthy participants. Part 3 will evaluate multiple dose administrations in participants with Netherton syndrome in an open-label design. Part 4 will evaluate multiple administrations of BCX17725 in participants with Netherton syndrome in an open-label study design over 12 weeks, with an 8-week post-treatment follow-up period.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Double blind (participant, investigator)
- Number of arms
- 6
- Model description
- In Parts 1 and 2, participants will be randomized to BCX17725 or placebo under double-blind conditions. In Parts 3 and 4, participants will receive open-label BCX17725.
| Arm | Type | Receives |
|---|---|---|
| Part 1 - BCX17725 single dose | Experimental | Drug: BCX17725Participants randomized to BCX17725 will receive BCX17725 as a single dose in sequential ascending dose cohorts |
| Part 1 - placebo single dose | Experimental | Drug: PlaceboParticipants randomized to placebo will receive placebo as a single dose |
| Part 2 - BCX17725 multiple doses | Experimental | Drug: BCX17725Participants randomized to BCX17725 will receive BCX17725 as multiple doses in sequential ascending dose cohorts |
| Part 2 - placebo multiple doses | Experimental | Drug: PlaceboParticipants randomized to placebo will receive placebo as multiple doses |
| Part 3 - BCX17725 multiple doses | Experimental | Drug: BCX17725Participants will receive BCX17725 as multiple doses |
| Part 4 - BCX17725 multiple doses | Experimental | Drug: BCX17725Participants will receive BCX17725 as multiple doses |
Who may qualify
- Ages
- 12 to 65 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (7 listed)
- 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)
Key exclusion criteria (0 listed)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| BCX17725BCX17725 for injection | Drug | Part 1 - BCX17725 single dose, Part 2 - BCX17725 multiple doses, Part 3 - BCX17725 multiple doses, Part 4 - BCX17725 multiple doses |
| PlaceboPlacebo for injection | Drug | Part 1 - placebo single dose, Part 2 - placebo multiple doses |
Primary outcome
Incidence of treatment-emergent adverse events (TEAEs)
Time frame: From screening through EOS (ie, through Day 78 in Parts 1 and 3, and Day 106 in Part 2)
Incidence of TEAEs as assessed by Common Terminology Criteria for Adverse Events (CTCAE) from screening through end-of-study (EOS) in each study part
Change from baseline in Ichthyosis Area and Severity Index (IASI) score at Week 12 (Part 4)
Time frame: From baseline to Week 12
IASI measures the severity of erythema (IASI-E) and scaling (IASI-S); the maximum sub-scores for the IASI-E and IASI-S being 24, and the maximum total IASI score being 48. Higher scores indicate worse clinical outcome.
9 secondary outcomes
Maximum observed serum concentration (Cmax)
Time frame: Up to Day 64 (Part 1), Day 92 (Part 2), and Day 78 (Part 3)
Time to maximum observed serum concentration (Tmax)
Time frame: Up to Day 64 (Part 1), Day 92 (Part 2), and Day 78 (Part 3)
Area under the serum concentration-time curve (AUC) from time 0 to the time of last measurable concentration (AUC0-t)
Time frame: Up to Day 64 (Part 1), Day 92 (Part 2), and Day 78 (Part 3)
Terminal elimination half-life (t1/2)
Time frame: Up to Day 64 (Part 1), Day 92 (Part 2), and Day 78 (Part 3)
Number of participants who are anti-drug antibody (ADA)-positive (baseline and post-baseline) and number of participants who have treatment-emergent ADAs
Time frame: Day 1 pre-dose and up to Day 64 (Part 1), Day 92 (Part 2), and Day 78 (Part 3)
Change from baseline in Investigator Global Assessment (IGA) score at Week 12 (Part 4)
Time frame: From baseline to Week 12
Change from baseline in Worst Itch Numerical Rating Score (NRS) at Week 12 (Part 4)
Time frame: From baseline to Week 12
Incidence of TEAEs (Part 4)
Time frame: From baseline through Week 20
Serum concentrations of BCX17725 (Part 4)
Time frame: From baseline through Week 20
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Nov 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
12 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing BCX17725 in people with Netherton Syndrome, run by BioCryst Pharmaceuticals at 12 sites in 5 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 0 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 6 Nov 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 12 sites in United States, Australia, Netherlands, France, Germany. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of treatment-emergent adverse events (TEAEs) (time frame: From screening through EOS (ie, through Day 78 in Parts 1 and 3, and Day 106 in Part 2)). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Nov 2026.