Clinical trial NCT06539507: A Study of the Safety, Tolerability, Pharmacokinetics, and Immunogenicity of BCX17725
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
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE1
Conditions: Netherton Syndrome
Keywords: First-in-human
Single ascending dose
Multiple ascending dose
Pharmacokinetics
Placebo control
Netherton syndrome
Lead sponsor: BioCryst Pharmaceuticals
Sponsor class: INDUSTRY
First posted: 2024-08-06
Enrollment: 72
Sex: ALL
Minimum age (years): 18
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: yes
Allocation: RANDOMIZED
Intervention model: SEQUENTIAL
Intervention model description: In Parts 1 and 2, participants will be randomized to BCX17725 or placebo under double-blind conditions. In Part 3, participants will receive open-label BCX17725.
Primary purpose: TREATMENT

Brief summary: 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).

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.

Eligibility criteria: Key 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

Primary outcomes:
measure: Incidence of treatment-emergent adverse events (TEAEs)
timeFrame: From screening through EOS (ie, through Day 78 in Parts 1 and 3, and Day 106 in Part 2)
Secondary outcomes:
measure: Maximum observed serum concentration (Cmax)
timeFrame: Up to Day 64 (Part 1), Day 92 (Part 2), and Day 78 (Part 3)
measure: Time to maximum observed serum concentration (Tmax)
timeFrame: Up to Day 64 (Part 1), Day 92 (Part 2), and Day 78 (Part 3)
measure: Area under the serum concentration-time curve (AUC) from time 0 to the time of last measurable concentration (AUC0-t)
timeFrame: Up to Day 64 (Part 1), Day 92 (Part 2), and Day 78 (Part 3)
measure: Terminal elimination half-life (t1/2)
timeFrame: Up to Day 64 (Part 1), Day 92 (Part 2), and Day 78 (Part 3)
measure: Number of participants who are anti-drug antibody (ADA)-positive (baseline and post-baseline) and number of participants who have treatment-emergent ADAs
timeFrame: Day 1 pre-dose and up to Day 64 (Part 1), Day 92 (Part 2), and Day 78 (Part 3)
Other outcomes:
Locations:
city: Brisbane
country: Australia
facility: Investigative site
state: Queensland
Study officials:
References: