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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

Titles & IDs
Public title
A Study of the Safety, Tolerability, Pharmacokinetics, and Immunogenicity of BCX17725
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
Secondary ID [1] 0 0
U1111-1303-9510
Secondary ID [2] 0 0
BCX17725-101
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Netherton Syndrome 0 0
Condition category
Condition code
Skin 0 0 0 0
Other skin conditions
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above

Intervention/exposure
Study type
Interventional
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
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Incidence of treatment-emergent adverse events (TEAEs)
Timepoint [1] 0 0
From screening through EOS (ie, through Day 78 in Parts 1 and 3, and Day 106 in Part 2)
Secondary outcome [1] 0 0
Maximum observed serum concentration (Cmax)
Timepoint [1] 0 0
Up to Day 64 (Part 1), Day 92 (Part 2), and Day 78 (Part 3)
Secondary outcome [2] 0 0
Time to maximum observed serum concentration (Tmax)
Timepoint [2] 0 0
Up to Day 64 (Part 1), Day 92 (Part 2), and Day 78 (Part 3)
Secondary outcome [3] 0 0
Area under the serum concentration-time curve (AUC) from time 0 to the time of last measurable concentration (AUC0-t)
Timepoint [3] 0 0
Up to Day 64 (Part 1), Day 92 (Part 2), and Day 78 (Part 3)
Secondary outcome [4] 0 0
Terminal elimination half-life (t1/2)
Timepoint [4] 0 0
Up to Day 64 (Part 1), Day 92 (Part 2), and Day 78 (Part 3)
Secondary outcome [5] 0 0
Number of participants who are anti-drug antibody (ADA)-positive (baseline and post-baseline) and number of participants who have treatment-emergent ADAs
Timepoint [5] 0 0
Day 1 pre-dose and up to Day 64 (Part 1), Day 92 (Part 2), and Day 78 (Part 3)

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
Minimum age
18 Years
Maximum age
65 Years
Sex
Both males and females
Can healthy volunteers participate?
Yes
Key exclusion criteria

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s


The people analysing the results/data
Intervention assignment
Other
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
26/09/2024
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/07/2025
Actual
Sample size
Target
72
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
QLD
Recruitment hospital [1] 0 0
Investigative site - Brisbane
Recruitment postcode(s) [1] 0 0
- Brisbane

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
BioCryst Pharmaceuticals
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
BioCryst Pharmaceuticals, Inc.
Address 0 0
Country 0 0
Phone 0 0
+1 919 859 1302
Fax 0 0
Email 0 0
clinicaltrials@biocryst.com
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


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