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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT06592131




Registration number
NCT06592131
Ethics application status
Date submitted
29/08/2024
Date registered
19/09/2024

Titles & IDs
Public title
BF844 Safety and Pharmacokinetic Study in Healthy Volunteers
Scientific title
First-in-Human Phase-1, Randomised, Double-Blinded, Placebo Controlled Study to Assess Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Single and Multiple Ascending Doses, As Well As a Food Effect Study, of BF844 When Administered Orally to Healthy Adult Participants
Secondary ID [1] 0 0
BF-844-01
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Usher Syndrome Type 3 0 0
Condition category
Condition code
Ear 0 0 0 0
Other ear disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - BF844

Experimental: Cohort-1 First dose of SAD cohort (6 treatment + 2 placebo) - Drug: BF844

Experimental: Cohort-2 Second dose of SAD cohort (6 treatment + 2 placebo) - Drug: BF844

Experimental: Cohort-3 Third dose of SAD cohort (6 treatment + 2 placebo) - Drug: BF844

Experimental: Cohort-4 Fourth dose of SAD cohort (6 treatment + 2 placebo) - Drug: BF844

Experimental: Cohort-5 Fifth dose of SAD cohort (6 treatment + 2 placebo) - Drug: BF844

Experimental: Cohort-6 Using one of the BF844 doses(with/without food) that were tested in SAD - Drug: BF844

Experimental: Cohort-7 First dose of MAD cohort (6 treatment + 2 placebo) - Drug: BF844

Experimental: Cohort 8 Second dose of MAD cohort (6 treatment + 2 placebo) - Drug: BF844

Experimental: Cohort 9 Third dose of MAD cohort (6 treatment + 2 placebo) - Drug: BF844


Treatment: Drugs: BF844
BF844, a small molecule developed by Usheriii Initiative to prevent or delay the progressive hearing and vision loss in patients with Usher syndrome type 3.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
To determine the safety and tolerability of BF844 when administered orally at ascending single and multiple doses to healthy adult participants.
Timepoint [1] 0 0
Baseline and 14 Days
Primary outcome [2] 0 0
To determine the safety and tolerability of BF844 when administered orally at ascending single and multiple doses to healthy adult participants.
Timepoint [2] 0 0
Baseline and 14 Days
Primary outcome [3] 0 0
To determine the safety and tolerability of BF844 when administered orally at ascending single and multiple doses to healthy adult participants.
Timepoint [3] 0 0
Baseline and 14 Days
Primary outcome [4] 0 0
To determine the safety and tolerability of BF844 when administered orally at ascending single and multiple doses to healthy adult participants.
Timepoint [4] 0 0
Baseline and 14 Days
Primary outcome [5] 0 0
To determine the safety and tolerability of BF844 when administered orally at ascending single and multiple doses to healthy adult participants.
Timepoint [5] 0 0
Baseline and 14 Days
Primary outcome [6] 0 0
To determine the safety and tolerability of BF844 when administered orally at ascending single and multiple doses to healthy adult participants.
Timepoint [6] 0 0
Baseline and 14 Days
Secondary outcome [1] 0 0
To determine the effect of food on the absorption of BF844 when administered as a single oral dose in healthy participants.
Timepoint [1] 0 0
Baseline and 24 hours
Secondary outcome [2] 0 0
To determine the PK of BF844 when administered as multiple oral doses at escalating dose levels in healthy participants.
Timepoint [2] 0 0
Baseline and 7 days
Secondary outcome [3] 0 0
To determine the PK of BF844 when administered as multiple oral doses at escalating dose levels in healthy participants.
Timepoint [3] 0 0
Baseline and 7 days
Secondary outcome [4] 0 0
To determine the PK of BF844 when administered as multiple oral doses at escalating dose levels in healthy participants.
Timepoint [4] 0 0
Baseline and 7 days
Secondary outcome [5] 0 0
To determine the PK of BF844 when administered as multiple oral doses at escalating dose levels in healthy participants.
Timepoint [5] 0 0
Baseline and 7 days
Secondary outcome [6] 0 0
To determine the PK of BF844 when administered as multiple oral doses at escalating dose levels in healthy participants.
Timepoint [6] 0 0
Baseline and 7 days
Secondary outcome [7] 0 0
To determine the effect of food on the absorption of BF844 when administered as a single oral dose in healthy participants.
Timepoint [7] 0 0
Baseline and 24 hours
Secondary outcome [8] 0 0
To determine the effect of food on the absorption of BF844 when administered as a single oral dose in healthy participants.
Timepoint [8] 0 0
Baseline and 24 hours
Secondary outcome [9] 0 0
To determine the effect of food on the absorption of BF844 when administered as a single oral dose in healthy participants.
Timepoint [9] 0 0
Baseline and 24 hours
Secondary outcome [10] 0 0
To determine the effect of food on the absorption of BF844 when administered as a single oral dose in healthy participants.
Timepoint [10] 0 0
Baseline and 24 hours
Secondary outcome [11] 0 0
To determine the PK of BF844 when administered as multiple oral doses at escalating dose levels in healthy participants.
Timepoint [11] 0 0
Baseline and 7 days
Secondary outcome [12] 0 0
To determine the PK of BF844 when administered as multiple oral doses at escalating dose levels in healthy participants.
Timepoint [12] 0 0
Baseline and 7 days
Secondary outcome [13] 0 0
To determine the PK of BF844 when administered as multiple oral doses at escalating dose levels in healthy participants.
Timepoint [13] 0 0
Baseline and 7 days

Eligibility
Key inclusion criteria
* Healthy volunteers (Participants) will be eligible for enrollment in the study only if they meet ALL the following criteria at time of Screening and pre-dosing:

1. Healthy male or female between the ages of 18 and 45 years (inclusive) at the time of Screening. Healthy status will be determined by the Investigator based on medical history, clinical laboratory results, vital sign and electrocardiogram measurements, and physical examination at screening.
2. Willingness and ability to give personal signed informed consent and comply with all scheduled visits, treatment plan, laboratory tests, lifestyle considerations, and other study procedures.
3. Weight of at least 45 kg and a body mass index (BMI) = 19.0 and = 32.0 kg/m2 at Screening.
4. Male participants who are non-sterilized and sexually active with a female partner of childbearing potential must agree to use adequate contraception from the signing of informed consent, throughout the duration of the study, and for 90 days after completion of the study. Male participants must use barrier contraception (e.g., condom). In addition to the male participant using a condom, the female partner of childbearing potential must also be using a highly effective form of contraception (hormonal or non-hormonal). The male participant must be advised and agree not to donate sperm during this period.
5. Female participants of childbearing potential who are sexually active with a non-sterilized male partner must agree to routinely use highly effective contraception from signing of informed consent, throughout the duration of the study, and for 30 days after completion of the study. A highly effective method of contraception for women of childbearing potential is defined as one that has no higher than a 1% failure rate per year. The acceptable methods of contraception are:

Intrauterine device (IUD), Bilateral tubal occlusion, Vasectomised partner (provided that the partner(s) absence of sperm in the ejaculate has been confirmed and documented), and Sexual abstinence (if it is the preferred and usual lifestyle of the participant).
Minimum age
18 Years
Maximum age
45 Years
Sex
Both males and females
Can healthy volunteers participate?
Yes
Key exclusion criteria
Participants meeting ANY of the following criteria at time of Screening and/or pre-dosing will be excluded from enrollment:

1. Any concomitant disease, condition, or treatment that could interfere with the conduct of the study, or that would, in the opinion of the Investigator or Sponsor, pose an unacceptable risk to the participant in the study or interfere with the interpretation of study data.
2. Has received any investigational compound within 30 days or 5 half-lives, whichever is longer, prior to the first dose of study drug.
3. Participant who is a study site employee or who is an immediate family member of a study site employee, (e.g., spouse, parent, child, sibling). Any participant under duress during the consent process.
4. Participant has any clinically significant illness, such as cardiovascular, neurologic, pulmonary, hepatic, renal (estimated or actual GFR less than 90 mL/minute), metabolic, gastrointestinal, urologic, immunologic, endocrine, or psychiatric disease or disorder, or other abnormality, which may affect safety, or potentially confound the study results. It is the responsibility of the Investigator to assess the clinical significance; however, consultation with the Sponsor and/or CRO MM may be warranted.
5. Participant has a known hypersensitivity to any component of the formulation of BF844.
6. Participant has a positive urine result for drugs of abuse (defined as any illicit drug use) at Screening or Check-in (Day -1).
7. Participant who, in the opinion of the Investigator, has a history of excessive alcohol or drug abuse within one year of Screening.
8. Use of or plans to use medications (e.g., S-warfarin, NSAIDs, phenytoin, omeprazole, and clopidogrel) that are significantly metabolized by CYP2C19/ CYP2C9. Additionally, the use of any medications that could have a significant impact on organ function (e.g., barbiturates, omeprazole, and cimetidine). These drugs are prohibited during the study and within 5 half-lives of the individual agent or within 28 days (whichever is longer) prior to enrollment.
9. Use or intended use of prescription or non-prescription medications (including vitamins or supplements) from 7 days prior to dosing or 5 half-lives (whichever is greater), unless the Investigator or medical delegate considers that such use would not significantly impact participant safety or study data.
10. Participant is pregnant or lactating or intending to become pregnant before, during, or within 30 days since the last dose of the study drug; or intending to donate ova during such time.
11. If male, the participant intends to donate sperm during the course of this study or within 90 days after the last dose of the study drug.
12. Participant has current or recent (within 6 months) gastrointestinal disease that would be expected to influence the absorption of drugs (i.e., a history of malabsorption, any surgical intervention known to impact absorption [e.g., bariatric surgery or bowel resection], esophageal reflux, peptic ulcer disease, erosive esophagitis, or frequent [more than once per week] occurrence of heartburn).
13. Participant has a positive test result for: (i) hepatitis B surface antigen (HBsAg), (ii) hepatitis C virus (HCV) antibody, or (iii) human immunodeficiency virus (HIV) infection at Screening.
14. Participant has used nicotine-containing products (including but not limited to cigarettes, pipes, cigars, chewing tobacco, nicotine patches, or nicotine gum) within 28 days prior to Check-in (Day -1). The cotinine test is positive at Screening or Check-in (Day -1) or if the participant is unwilling to abstain from nicotine-containing products throughout the study.
15. Participant has poor peripheral venous access.
16. Participant has donated or lost 450 mL or more of his or her blood volume (including plasmapheresis) or had a transfusion of any blood product within 90 days prior to the first dose of the study drug.
17. Participant has a clinically significant ECG (QTcF =; 450 for males and 470 for females) at Screening or Check-in (Day -1). Entry of any participant with an abnormal, but not clinically significant, ECG must be approved, and documented by signature of the Investigator or medical delegate.
18. Participant has a supine blood pressure outside the ranges of = 90 to = 140 mmHg for systolic and = 40 to = 90 mmHg for diastolic. If out of range, an assessment may be repeated once for eligibility determination at Screening and/or Check-in (Day -1).
19. Participant has a resting heart rate outside the range of 40 to 100 bpm (not on ECGs). If out of range, the assessment may be repeated once for eligibility determination at Screening and/or Check-in (Day -1).
20. Participant has abnormal laboratory values at Screening or Check-in (Day -1) that suggest a clinically significant underlying disease or participant has the following lab abnormalities: ALT and/or AST = 1.5x ULN.
21. Participant is unwilling to avoid strenuous exercise up to 48 hours prior to any safety laboratory blood draws.
22. Participant is unwilling to abstain from consuming caffeine and/or xanthene products (e.g., coffee, tea, chocolate, and caffeine-containing sodas) while confined to the CRU.

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 assessing the outcomes
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
Not yet recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
9/09/2024
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
6/09/2025
Actual
Sample size
Target
76
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Linear Clinical Research - Perth
Recruitment postcode(s) [1] 0 0
- Perth

Funding & Sponsors
Primary sponsor type
Other
Name
EyeXCel Pty. Ltd.
Address
Country
Other collaborator category [1] 0 0
Other
Name [1] 0 0
Usher iii Initiative
Address [1] 0 0
Country [1] 0 0

Ethics approval
Ethics application status

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

Contacts
Principal investigator
Name 0 0
Benedict Tan, MBBS, FRACP
Address 0 0
Linear Clinical Research
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Rachelle Kirk-Burnnand
Address 0 0
Country 0 0
Phone 0 0
+61 439-615-368
Fax 0 0
Email 0 0
rachelle@basebio.com.au
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
The results of the study will be published without referring to individual participant data or compromising confidentiality.


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/NCT06592131