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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT06539507
Registration number
NCT06539507
Ethics application status
Date submitted
30/07/2024
Date registered
6/08/2024
Date last updated
3/10/2024
Titles & IDs
Public title
A Study of the Safety, Tolerability, Pharmacokinetics, and Immunogenicity of BCX17725
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Scientific 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
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Secondary ID [1]
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U1111-1303-9510
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Secondary ID [2]
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BCX17725-101
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Netherton Syndrome
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Condition category
Condition code
Skin
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Other skin conditions
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Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Other
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Research that is not of generic health relevance and not applicable to specific health categories listed above
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - BCX17725
Treatment: Drugs - Placebo
Experimental: Part 1 - BCX17725 single dose - Participants randomized to BCX17725 will receive BCX17725 as a single dose in sequential ascending dose cohorts
Experimental: Part 1 - placebo single dose - Participants randomized to placebo will receive placebo as a single dose
Experimental: Part 2 - BCX17725 multiple doses - Participants randomized to BCX17725 will receive BCX17725 as multiple doses in sequential ascending dose cohorts
Experimental: Part 2 - placebo multiple doses - Participants randomized to placebo will receive placebo as multiple doses
Experimental: Part 3 - BCX17725 multiple doses - Participants will receive BCX17725 as multiple doses
Treatment: Drugs: BCX17725
BCX17725 for injection
Treatment: Drugs: Placebo
Placebo for injection
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Incidence of treatment-emergent adverse events (TEAEs)
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Assessment method [1]
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Incidence of TEAEs as assessed by Common Terminology Criteria for Adverse Events (CTCAE) from screening through end-of-study (EOS) in each study part
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Timepoint [1]
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From screening through EOS (ie, through Day 78 in Parts 1 and 3, and Day 106 in Part 2)
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Secondary outcome [1]
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Maximum observed serum concentration (Cmax)
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Assessment method [1]
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Cmax of BCX17725
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Timepoint [1]
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Up to Day 64 (Part 1), Day 92 (Part 2), and Day 78 (Part 3)
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Secondary outcome [2]
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Time to maximum observed serum concentration (Tmax)
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Assessment method [2]
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Time to Cmax of BCX17725
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Timepoint [2]
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Up to Day 64 (Part 1), Day 92 (Part 2), and Day 78 (Part 3)
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Secondary outcome [3]
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Area under the serum concentration-time curve (AUC) from time 0 to the time of last measurable concentration (AUC0-t)
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Assessment method [3]
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AUC0-t of BCX17725
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Timepoint [3]
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Up to Day 64 (Part 1), Day 92 (Part 2), and Day 78 (Part 3)
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Secondary outcome [4]
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Terminal elimination half-life (t1/2)
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Assessment method [4]
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Terminal elimination half-life (t1/2) of BCX17725
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Timepoint [4]
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Up to Day 64 (Part 1), Day 92 (Part 2), and Day 78 (Part 3)
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Secondary outcome [5]
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Number of participants who are anti-drug antibody (ADA)-positive (baseline and post-baseline) and number of participants who have treatment-emergent ADAs
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Assessment method [5]
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Incidence of ADAs to BCX17725
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Timepoint [5]
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Day 1 pre-dose and up to Day 64 (Part 1), Day 92 (Part 2), and Day 78 (Part 3)
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Eligibility
Key inclusion criteria
Key
* 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
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Minimum age
18
Years
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Maximum age
65
Years
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Sex
Both males and females
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Can healthy volunteers participate?
Yes
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Key exclusion criteria
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Blinded (masking used)
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Who is / are masked / blinded?
The people receiving the treatment/s
The people analysing the results/data
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Intervention assignment
Other
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Other design features
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Phase
Phase 1
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Recruiting
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
26/09/2024
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
1/07/2025
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Actual
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Sample size
Target
72
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Accrual to date
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Final
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Recruitment in Australia
Recruitment state(s)
QLD
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Recruitment hospital [1]
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Investigative site - Brisbane
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Recruitment postcode(s) [1]
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- Brisbane
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Funding & Sponsors
Primary sponsor type
Commercial sector/industry
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Name
BioCryst Pharmaceuticals
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Address
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Country
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Ethics approval
Ethics application status
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Summary
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).
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Trial website
https://clinicaltrials.gov/study/NCT06539507
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Address
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Country
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Phone
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Fax
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Email
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Contact person for public queries
Name
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BioCryst Pharmaceuticals, Inc.
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Address
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Country
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Phone
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+1 919 859 1302
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Fax
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Email
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clinicaltrials@biocryst.com
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Contact person for scientific queries
Data sharing statement
What supporting documents are/will be available?
No Supporting Document Provided
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results not provided in
https://clinicaltrials.gov/study/NCT06539507
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