Usher Syndrome Type 3Phase 1NCT06592131
BF844 Safety and Pharmacokinetic Study in Healthy Volunteers
A Phase 1 study testing BF844 in people with Usher Syndrome Type 3, run by EyeXCel Pty. Ltd. at 1 site in Australia.
Trial summary
First-in-Human Phase-1 Clinical Trial to Assess Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Food Effect of Single and Multiple Ascending Doses of BF844 when Administered Orally to Healthy Adult Participants. Read more about this study
- Who is it for?
- Ages 18 to 45 yearsHealthy volunteers can take part
- Conditions the study is for
- Usher Syndrome Type 3
- What is being tested?
- BF844
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 76 participants
- Where
- 1 siteAustralia
- Study dates
- 9 Sep 2024to 6 Sep 2025
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 45 years
- All sexes
- Healthy volunteers accepted
- Adult
Who may be able to join
- 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:
- 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.
- 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.
- Weight of at least 45 kg and a body mass index (BMI) ≥ 19.0 and ≤ 32.0 kg/m2 at Screening.
- 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.
- 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).
Who cannot join
- Participants meeting ANY of the following criteria at time of Screening and/or pre-dosing will be excluded from enrollment:
- 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.
- Has received any investigational compound within 30 days or 5 half-lives, whichever is longer, prior to the first dose of study drug.
- 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.
- 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.
- Participant has a known hypersensitivity to any component of the formulation of BF844.
Show all 23Show fewer
- Participant has a positive urine result for drugs of abuse (defined as any illicit drug use) at Screening or Check-in (Day -1).
- Participant who, in the opinion of the Investigator, has a history of excessive alcohol or drug abuse within one year of Screening.
- 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.
- 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.
- 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.
- 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.
- 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).
- 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.
- 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.
- Participant has poor peripheral venous access.
- 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.
- 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.
- 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).
- 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).
- 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.
- Participant is unwilling to avoid strenuous exercise up to 48 hours prior to any safety laboratory blood draws.
- 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.
Trial locations and contacts
1 site in 1 country.
About this study
Official 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
First-in-Human Phase-1 Clinical Trial to Assess Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Food Effect of Single and Multiple Ascending Doses of BF844 when Administered Orally to Healthy Adult Participants.
Read the detailed description
This is a first-in-human, single center, randomized, double-blinded, single and multiple ascending doses (SAD and MAD) and food effect Phase I study in healthy adult volunteers (HV).
The SAD cohorts will consist of five cohorts of eight participants (6 randomized to treatment + 2 randomized to placebo) in each cohort (Total 40 HV). Additional cohorts may be added.
The food effect (FE) cohort will consist of 12 participants who receive a single dose of BF844 in a cross-over manner. Each participants will receive the same single dose of BF844 under two separate conditions: one after an overnight fast, and the second following a high-fat breakfast 30 minutes prior to dosing.
The MAD cohorts will consist of 3 cohorts of eight participants (6 randomized to treatment + 2 randomized to placebo) in each cohort (Total 24 HV). The subjects in MAD cohorts will be dosed once daily for 7 consecutive days. Additional cohorts may be added.
For each cohort, all HV subjects will be enrolled at the same site To optimise participants safety, BF844 will be administered in a staggered manner as per protocol .
- Conditions
- Usher Syndrome Type 3
- Being tested
- BF844
- Keywords
- treatment, intervention, healthy volunteers, Usher syndrome type 3
- Medical subject headings
- Usher Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BF844Drug
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.
| Study group | Role | What they receive |
|---|---|---|
| Cohort-1 First dose of SAD cohort (6 treatment + 2 placebo) | Experimental | Drug: BF844 Receives: Drug: BF844 |
| Cohort-2 Second dose of SAD cohort (6 treatment + 2 placebo) | Experimental | Drug: BF844 Receives: Drug: BF844 |
| Cohort-3 Third dose of SAD cohort (6 treatment + 2 placebo) | Experimental | Drug: BF844 Receives: Drug: BF844 |
| Cohort-4 Fourth dose of SAD cohort (6 treatment + 2 placebo) | Experimental | Drug: BF844 Receives: Drug: BF844 |
| Cohort-5 Fifth dose of SAD cohort (6 treatment + 2 placebo) | Experimental | Drug: BF844 Receives: Drug: BF844 |
| Cohort-6 Using one of the BF844 doses(with/without food) that were tested in SAD | Experimental | Drug: BF844 Receives: Drug: BF844 |
| Cohort-7 First dose of MAD cohort (6 treatment + 2 placebo) | Experimental | Drug: BF844 Receives: Drug: BF844 |
| Cohort 8 Second dose of MAD cohort (6 treatment + 2 placebo) | Experimental | Drug: BF844 Receives: Drug: BF844 |
| Cohort 9 Third dose of MAD cohort (6 treatment + 2 placebo) | Experimental | Drug: BF844 Receives: Drug: BF844 |
What is being measured
The primary outcome is the main question the study is designed to answer.
To determine the safety and tolerability of BF844 when administered orally at ascending single and multiple doses to healthy adult participants.
Percentage of participants who experience at least 1 treatment-emergent adverse event (TEAE).
Measured: Baseline and 14 Days
To determine the safety and tolerability of BF844 when administered orally at ascending single and multiple doses to healthy adult participants.
Percentage of participants who discontinue due to an adverse event (AE).
Measured: Baseline and 14 Days
To determine the safety and tolerability of BF844 when administered orally at ascending single and multiple doses to healthy adult participants.
Percentage of participants who meet the clinically significant criteria for safety laboratory tests at least once post dose.
Measured: Baseline and 14 Days
To determine the safety and tolerability of BF844 when administered orally at ascending single and multiple doses to healthy adult participants.
Percentage of participants who meet the clinically significant criteria for vital sign measurements at least once post dose.
Measured: Baseline and 14 Days
To determine the safety and tolerability of BF844 when administered orally at ascending single and multiple doses to healthy adult participants.
Percentage of participants who meet the clinically significant criteria for safety electrocardiogram (ECG) parameters at least once post dose.
Measured: Baseline and 14 Days
To determine the safety and tolerability of BF844 when administered orally at ascending single and multiple doses to healthy adult participants.
Treatment-emergent hearing, vision, or balance abnormalities, as measured by audiometry, visual acuity, and clinical examination.
Measured: Baseline and 14 Days
Secondary outcomes (13)
To determine the effect of food on the absorption of BF844 when administered as a single oral dose in healthy participants.
Plasma PK parameters of BF844 at the dose level selected for the food effect study:
t1/2
Measured: Baseline and 24 hours
To determine the PK of BF844 when administered as multiple oral doses at escalating dose levels in healthy participants.
Plasma PK parameters of BF844 at each dose level:
Cmax
Measured: Baseline and 7 days
To determine the PK of BF844 when administered as multiple oral doses at escalating dose levels in healthy participants.
Plasma PK parameters of BF844 at each dose level:
AUClast
Measured: Baseline and 7 days
To determine the PK of BF844 when administered as multiple oral doses at escalating dose levels in healthy participants.
Plasma PK parameters of BF844 at each dose level:
AUC∞
Measured: Baseline and 7 days
To determine the PK of BF844 when administered as multiple oral doses at escalating dose levels in healthy participants.
Plasma PK parameters of BF844 at each dose level:
t1/2
Measured: Baseline and 7 days
To determine the PK of BF844 when administered as multiple oral doses at escalating dose levels in healthy participants.
Urine PK parameters of BF844 at each dose level:
AUC0-24hrs
Measured: Baseline and 7 days
To determine the effect of food on the absorption of BF844 when administered as a single oral dose in healthy participants.
Plasma PK parameters of BF844 at the dose level selected for the food effect study:
Cmax
Measured: Baseline and 24 hours
To determine the effect of food on the absorption of BF844 when administered as a single oral dose in healthy participants.
Plasma PK parameters of BF844 at the dose level selected for the food effect study:
tmax
Measured: Baseline and 24 hours
To determine the effect of food on the absorption of BF844 when administered as a single oral dose in healthy participants.
Plasma PK parameters of BF844 at the dose level selected for the food effect study:
AUClast
Measured: Baseline and 24 hours
To determine the effect of food on the absorption of BF844 when administered as a single oral dose in healthy participants.
Plasma PK parameters of BF844 at the dose level selected for the food effect study:
AUC∞
Measured: Baseline and 24 hours
To determine the PK of BF844 when administered as multiple oral doses at escalating dose levels in healthy participants.
Plasma PK parameters of BF844 at each dose level:
tmax
Measured: Baseline and 7 days
To determine the PK of BF844 when administered as multiple oral doses at escalating dose levels in healthy participants.
Plasma PK parameters of BF844 at each dose level:
AUCτ
Measured: Baseline and 7 days
To determine the PK of BF844 when administered as multiple oral doses at escalating dose levels in healthy participants.
Urine PK parameters of BF844 at each dose level:
Accumulation factor
Measured: Baseline and 7 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Sep 2024
- Study start9 Sep 2024 (estimated)
- Primary completion6 Mar 2025 (estimated)
- Study completion6 Sep 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Usher Syndrome Type 3Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Model description
- This is a first-in-human, single center, randomized, double-blinded, single and multiple ascending doses (SAD and MAD) and food effect Phase I study in healthy adult volunteers (HV). The SAD cohorts will consist of five cohorts of eight participants (6 randomized to treatment + 2 randomized to placebo) in each cohort (Total 40 HV). Additional cohorts may be added. The food effect (FE) cohort will consist of 12 participants who receive a single dose of BF844 in a cross-over manner. Each participants will receive the same single dose of BF844 under two separate conditions: one after an overnight fast, and the second following a high-fat breakfast 30 minutes prior to dosing. The MAD cohorts will consist of 3 cohorts of eight participants (6 randomized to treatment + 2 randomized to placebo) in each cohort (Total 24 HV). The subjects in MAD cohorts will be dosed once daily for 7 consecutive days. Additional cohorts may be added.
- Masking
- Triple blind
- Who is masked
- Participant, Investigator, Outcomes assessor
- Enrolment
- 76 participants (estimated)
Sponsor and investigators
- Lead sponsor
- EyeXCel Pty. Ltd.
- Sponsor type
- University, hospital or other
- Collaborators
- Usher iii Initiative
- Responsible party
- Sponsor
- Investigators
- Benedict Tan, MBBS, FRACP, Principal investigator, Linear Clinical Research
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06592131
- Sponsor's protocol ID
- BF-844-01
- Status last verified
- Sep 2024
- First submitted
- 29 Aug 2024
- Last updated
- 19 Sep 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: The results of the study will be published without referring to individual participant data or compromising confidentiality.
Publications
Alagramam KN, Gopal SR, Geng R, Chen DH, Nemet I, Lee R, Tian G, Miyagi M, Malagu KF, Lock CJ, Esmieu WR, Owens AP, Lindsay NA, Ouwehand K, Albertus F, Fischer DF, Burli RW, MacLeod AM, Harte WE, Palczewski K, Imanishi Y. A small molecule mitigates hearing loss in a mouse model of Usher syndrome III. Nat Chem Biol. 2016 Jun;12(6):444-51. doi: 10.1038/nchembio.2069. Epub 2016 Apr 25.
PubMed 27110679
Study at a glance
From the registry record, last updated by the sponsor on 19 Sep 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official 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
- ClinicalTrials.gov ID
- NCT06592131
- Acronym / sponsor ID
- BF-844-01
- Conditions
- Usher Syndrome Type 3
- Keywords
- treatment, intervention, healthy volunteers, Usher syndrome type 3
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 76 participants (estimated)
- Lead sponsor
- EyeXCel Pty. Ltd.University, hospital or other
- Collaborators
- Usher iii Initiative
- Study start
- 9 Sep 2024 (estimated)
- Primary completion
- 6 Mar 2025 (estimated)
- Study completion
- 6 Sep 2025 (estimated)
- First posted
- 19 Sep 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
First-in-Human Phase-1 Clinical Trial to Assess Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Food Effect of Single and Multiple Ascending Doses of BF844 when Administered Orally to Healthy Adult Participants.
Detailed description
This is a first-in-human, single center, randomized, double-blinded, single and multiple ascending doses (SAD and MAD) and food effect Phase I study in healthy adult volunteers (HV).
The SAD cohorts will consist of five cohorts of eight participants (6 randomized to treatment + 2 randomized to placebo) in each cohort (Total 40 HV). Additional cohorts may be added.
The food effect (FE) cohort will consist of 12 participants who receive a single dose of BF844 in a cross-over manner. Each participants will receive the same single dose of BF844 under two separate conditions: one after an overnight fast, and the second following a high-fat breakfast 30 minutes prior to dosing.
The MAD cohorts will consist of 3 cohorts of eight participants (6 randomized to treatment + 2 randomized to placebo) in each cohort (Total 24 HV). The subjects in MAD cohorts will be dosed once daily for 7 consecutive days. Additional cohorts may be added.
For each cohort, all HV subjects will be enrolled at the same site To optimise participants safety, BF844 will be administered in a staggered manner as per protocol .
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Triple blind (participant, investigator, outcomes assessor)
- Number of arms
- 9
- Model description
- This is a first-in-human, single center, randomized, double-blinded, single and multiple ascending doses (SAD and MAD) and food effect Phase I study in healthy adult volunteers (HV). The SAD cohorts will consist of five cohorts of eight participants (6 randomized to treatment + 2 randomized to placebo) in each cohort (Total 40 HV). Additional cohorts may be added. The food effect (FE) cohort will consist of 12 participants who receive a single dose of BF844 in a cross-over manner. Each participants will receive the same single dose of BF844 under two separate conditions: one after an overnight fast, and the second following a high-fat breakfast 30 minutes prior to dosing. The MAD cohorts will consist of 3 cohorts of eight participants (6 randomized to treatment + 2 randomized to placebo) in each cohort (Total 24 HV). The subjects in MAD cohorts will be dosed once daily for 7 consecutive days. Additional cohorts may be added.
| Arm | Type | Receives |
|---|---|---|
| Cohort-1 First dose of SAD cohort (6 treatment + 2 placebo) | Experimental | Drug: BF844Drug: BF844 |
| Cohort-2 Second dose of SAD cohort (6 treatment + 2 placebo) | Experimental | Drug: BF844Drug: BF844 |
| Cohort-3 Third dose of SAD cohort (6 treatment + 2 placebo) | Experimental | Drug: BF844Drug: BF844 |
| Cohort-4 Fourth dose of SAD cohort (6 treatment + 2 placebo) | Experimental | Drug: BF844Drug: BF844 |
| Cohort-5 Fifth dose of SAD cohort (6 treatment + 2 placebo) | Experimental | Drug: BF844Drug: BF844 |
| Cohort-6 Using one of the BF844 doses(with/without food) that were tested in SAD | Experimental | Drug: BF844Drug: BF844 |
| Cohort-7 First dose of MAD cohort (6 treatment + 2 placebo) | Experimental | Drug: BF844Drug: BF844 |
| Cohort 8 Second dose of MAD cohort (6 treatment + 2 placebo) | Experimental | Drug: BF844Drug: BF844 |
| Cohort 9 Third dose of MAD cohort (6 treatment + 2 placebo) | Experimental | Drug: BF844Drug: BF844 |
Who may qualify
- Ages
- 18 to 45 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult
Key inclusion criteria (7 listed)
- 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:
- 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).
Key exclusion criteria (23 listed)
- Participants meeting ANY of the following criteria at time of Screening and/or pre-dosing will be excluded from enrollment:
- 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.
- Has received any investigational compound within 30 days or 5 half-lives, whichever is longer, prior to the first dose of study drug.
- 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.
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| BF844BF844, a small molecule developed by Usheriii Initiative to prevent or delay the progressive hearing and vision loss in patients with Usher syndrome type 3. | Drug | Cohort 8 Second dose of MAD cohort (6 treatment + 2 placebo), Cohort 9 Third dose of MAD cohort (6 treatment + 2 placebo), Cohort-1 First dose of SAD cohort (6 treatment + 2 placebo), Cohort-2 Second dose of SAD cohort (6 treatment + 2 placebo), Cohort-3 Third dose of SAD cohort (6 treatment + 2 placebo), Cohort-4 Fourth dose of SAD cohort (6 treatment + 2 placebo), Cohort-5 Fifth dose of SAD cohort (6 treatment + 2 placebo), Cohort-6 Using one of the BF844 doses(with/without food) that were tested in SAD, Cohort-7 First dose of MAD cohort (6 treatment + 2 placebo) |
Primary outcome
To determine the safety and tolerability of BF844 when administered orally at ascending single and multiple doses to healthy adult participants.
Time frame: Baseline and 14 Days
Percentage of participants who experience at least 1 treatment-emergent adverse event (TEAE).
To determine the safety and tolerability of BF844 when administered orally at ascending single and multiple doses to healthy adult participants.
Time frame: Baseline and 14 Days
Percentage of participants who discontinue due to an adverse event (AE).
To determine the safety and tolerability of BF844 when administered orally at ascending single and multiple doses to healthy adult participants.
Time frame: Baseline and 14 Days
Percentage of participants who meet the clinically significant criteria for safety laboratory tests at least once post dose.
To determine the safety and tolerability of BF844 when administered orally at ascending single and multiple doses to healthy adult participants.
Time frame: Baseline and 14 Days
Percentage of participants who meet the clinically significant criteria for vital sign measurements at least once post dose.
To determine the safety and tolerability of BF844 when administered orally at ascending single and multiple doses to healthy adult participants.
Time frame: Baseline and 14 Days
Percentage of participants who meet the clinically significant criteria for safety electrocardiogram (ECG) parameters at least once post dose.
To determine the safety and tolerability of BF844 when administered orally at ascending single and multiple doses to healthy adult participants.
Time frame: Baseline and 14 Days
Treatment-emergent hearing, vision, or balance abnormalities, as measured by audiometry, visual acuity, and clinical examination.
13 secondary outcomes
To determine the effect of food on the absorption of BF844 when administered as a single oral dose in healthy participants.
Time frame: Baseline and 24 hours
To determine the PK of BF844 when administered as multiple oral doses at escalating dose levels in healthy participants.
Time frame: Baseline and 7 days
To determine the PK of BF844 when administered as multiple oral doses at escalating dose levels in healthy participants.
Time frame: Baseline and 7 days
To determine the PK of BF844 when administered as multiple oral doses at escalating dose levels in healthy participants.
Time frame: Baseline and 7 days
To determine the PK of BF844 when administered as multiple oral doses at escalating dose levels in healthy participants.
Time frame: Baseline and 7 days
To determine the PK of BF844 when administered as multiple oral doses at escalating dose levels in healthy participants.
Time frame: Baseline and 7 days
To determine the effect of food on the absorption of BF844 when administered as a single oral dose in healthy participants.
Time frame: Baseline and 24 hours
To determine the effect of food on the absorption of BF844 when administered as a single oral dose in healthy participants.
Time frame: Baseline and 24 hours
To determine the effect of food on the absorption of BF844 when administered as a single oral dose in healthy participants.
Time frame: Baseline and 24 hours
To determine the effect of food on the absorption of BF844 when administered as a single oral dose in healthy participants.
Time frame: Baseline and 24 hours
To determine the PK of BF844 when administered as multiple oral doses at escalating dose levels in healthy participants.
Time frame: Baseline and 7 days
To determine the PK of BF844 when administered as multiple oral doses at escalating dose levels in healthy participants.
Time frame: Baseline and 7 days
To determine the PK of BF844 when administered as multiple oral doses at escalating dose levels in healthy participants.
Time frame: Baseline and 7 days
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Sep 2024.
- Background: PubMed 27110679Alagramam KN, Gopal SR, Geng R, Chen DH, Nemet I, Lee R, Tian G, Miyagi M, Malagu KF, Lock CJ, Esmieu WR, Owens AP, Lindsay NA, Ouwehand K, Albertus F, Fischer DF, Burli RW, MacLeod AM, Harte WE, Palczewski K, Imanishi Y. A small molecule mitigates hearing loss in a mouse model of Usher syndrome III. Nat Chem Biol. 2016 Jun;12(6):444-51. doi: 10.1038/nchembio.2069. Epub 2016 Apr 25.
- Individual participant dataSharing planned: No. The results of the study will be published without referring to individual participant data or compromising confidentiality.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Benedict Tan, MBBS, FRACP principal investigator
Linear Clinical Research
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing BF844 in people with Usher Syndrome Type 3, run by EyeXCel Pty. Ltd. at 1 site in Australia.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 23 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 19 Sep 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Australia. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Rachelle Kirk-Burnnand (+61 439-615-368, rachelle@basebio.com.au), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
To determine the safety and tolerability of BF844 when administered orally at ascending single and multiple doses to healthy adult participants. (time frame: Baseline and 14 Days). The study lists 6 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT06592131, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Sep 2024.