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A Study of the Safety, Tolerability, Pharmacokinetics, and Immunogenicity of BCX17725

PHASE1RECRUITING

This is a first-in-human, Phase 1/1b, 3-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 participants with Netherton Syndrome (Part 3).

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Study details:

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.

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Eligibility criteria

Researchers look for people who fit a certain description, called eligibility criteria. See if you qualify.

Inclusion criteria

Male or non-pregnant, non-lactating female aged 18 to 55 years, inclusive (Parts 1 and 2) or aged 18 to 65 years, inclusive (Part 3)

Confirmed diagnosis of Netherton syndrome (Part 3 only)

BMI between 18 and 30 kg/m^2, inclusive (Parts 1 and 2 only)

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

Eligibility

Age eligible for study : 18 and older

Healthy volunteers accepted : Yes

Gender eligible for study: All

Things to know

Study dates

Study start: 2024-09-26

Primary completion: 2025-07-01

Study completion finish: 2025-07-01

Study type

TREATMENT

Phase

PHASE1

Trial ID

NCT06539507

Intervention or treatment

DRUG: BCX17725

DRUG: Placebo

Conditions

• Netherton Syndrome

Condition: Netherton Syndrome
DRUG: BCX17725 and other drugs
Brisbane, Queensland, Australia
Sponsor: BioCryst Pharmaceuticals

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Find a site

Closest Location:

Investigative site

Research sites nearby

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Investigative site
Brisbane, Queensland, Australia

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Participant Group/Arm	Intervention/Treatment
EXPERIMENTAL: Part 1 - BCX17725 single dose Participants randomized to BCX17725 will receive BCX17725 as a single dose in sequential ascending dose cohorts	DRUG: BCX17725 BCX17725 for injection
EXPERIMENTAL: Part 1 - placebo single dose Participants randomized to placebo will receive placebo as a single dose	DRUG: Placebo Placebo for injection
EXPERIMENTAL: Part 2 - BCX17725 multiple doses Participants randomized to BCX17725 will receive BCX17725 as multiple doses in sequential ascending dose cohorts	DRUG: BCX17725 BCX17725 for injection
EXPERIMENTAL: Part 2 - placebo multiple doses Participants randomized to placebo will receive placebo as multiple doses	DRUG: Placebo Placebo for injection
EXPERIMENTAL: Part 3 - BCX17725 multiple doses Participants will receive BCX17725 as multiple doses	DRUG: BCX17725 BCX17725 for injection

What is the study measuring?

Primary outcome

Primary Outcome Measure	Primary Outcome Description	Primary Outcome Time Frame
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	From screening through EOS (ie, through Day 78 in Parts 1 and 3, and Day 106 in Part 2)

Secondary outcome

Secondary Outcome Measure	Secondary Outcome Description	Secondary Outcome Time Frame
Maximum observed serum concentration (Cmax)	Cmax of BCX17725	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	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	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	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	Day 1 pre-dose and up to Day 64 (Part 1), Day 92 (Part 2), and Day 78 (Part 3)

Frequently Asked Questions

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References

Clinical Trials Gov: A Study of the Safety, Tolerability, Pharmacokinetics, and Immunogenicity of BCX17725