Health TrialsbyHealthPanel

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

ConditionNetherton Syndrome
Trial statusRecruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexAll
Age12-65
SponsorBioCryst Pharmaceuticals

About this trial

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.

Eligibility criteria

This trial accepts healthy volunteers

Qualifiers

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/m², inclusive (Parts 1 and 2)

Disqualifiers

None

Trial design

Design model

Sequential

Treatments tested in this trial

  • BCX17725

    Drug

    BCX17725 for injection

  • Placebo

    Drug

    Placebo for injection

Treatment groups

78 Participants
are divided into 6 treatment groups

6

Treatment groups

See each treatment group below.

Group A: Part 1 - BCX17725 single doseExperimental treatment 1 intervention
Group B: Part 1 - placebo single doseExperimental treatment 1 intervention
Group C: Part 2 - BCX17725 multiple dosesExperimental treatment 1 intervention
Group D: Part 2 - placebo multiple dosesExperimental treatment 1 intervention
Group E: Part 3 - BCX17725 multiple dosesExperimental treatment 1 intervention
Group F: Part 4 - BCX17725 multiple dosesExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

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

Time frame
From screening through EOS (ie, through Day 78 in Parts 1 and 3, and Day 106 in Part 2)
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.

Time frame
From baseline to Week 12

Secondary outcomes

1

Maximum observed serum concentration (Cmax)

Cmax of BCX17725

Time frame
Up to Day 64 (Part 1), Day 92 (Part 2), and Day 78 (Part 3)
2

Time to maximum observed serum concentration (Tmax)

Time to Cmax of BCX17725

Time frame
Up to Day 64 (Part 1), Day 92 (Part 2), and Day 78 (Part 3)
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

Time frame
Up to Day 64 (Part 1), Day 92 (Part 2), and Day 78 (Part 3)
4

Terminal elimination half-life (t1/2)

Terminal elimination half-life (t1/2) of BCX17725

Time frame
Up to Day 64 (Part 1), Day 92 (Part 2), and Day 78 (Part 3)

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

BioCryst Pharmaceuticals

Lead sponsor

Source ClinicalTrials.gov
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