Study Evaluating Safety of BT01001 Ophthalmic Solution

NCT07313722 | Recruiting Phase 1 FDA Drug

• 1 other identifier: BT01001-101
• Study Type: interventional
• Target: 32
• Locations: 1 country
• Sites: 1

Brief Summary

This Phase I study is designed to evaluate the safety, tolerability, and pharmacokinetic profile of BT01001 Ophthalmic Solution in healthy adult volunteers. The primary objectives are to assess the safety and tolerability of single and multiple ascending doses and to characterize the pharmacokinetics of BT01001 Ophthalmic Solution following topical ocular administration. This is a randomized, double-blind, placebo-controlled, dose-escalation trial consisting of four ascending dose cohorts. Each cohort will enroll eight participants, including six receiving BT01001 Ophthalmic Solution and 2 receiving Placebo.

Conditions

Healthy

Outcome Measures

Primary Outcomes (2)

• Number of Participants With Adverse Events as Assessed by CTCAE V5.0

  Any AE event related to study treatment

  after dosing up to 14 days/12 days

• Dose-Limiting Toxicity

  ≥CTCAE 2 ocular adverse events or· \>CTCAE 3 non-ocular· adverse events· assessed with·CTCAE·5.0

  SAD: after dosing up to 2 days; MAD: after dosing up to 8 days

Secondary Outcomes (3)

• Measured Area Under the Curve (AUC)

  SAD(dose 1-3): Day 1, Day 2; MAD(dose 1-3): Day 8, Day 9, Day 10; MAD(dose 4): Day 6, Day 7,Day 8;

• Time to Maximum Plasma Concentration (Tmax)

  SAD(dose 1-3): Day 1, Day 2; MAD(dose 1-3): Day 8, Day 9, Day 10; MAD(dose 4): Day 6, Day 7,Day 8;

• Maximum Plasma Concentration (Cmax)

  SAD(dose 1-3): Day 1, Day 2; MAD(dose 1-3): Day 8, Day 9, Day 10; MAD(dose 4): Day 6, Day 7,Day 8;

Study Arms (14)

BT01001 Ophthalmic Solution dose 1 SAD

EXPERIMENTAL

• BT01001 Ophthalmic Solution 5 mg/ml (0.5%） N = 6 Single Ascending Dose (SAD, Day 1): 2 drops administered once to the right eye. Elution/Washout Phase: 1 day following SAD.

Drug: BT01001 5 mg/ml（0.5%）

Placebo dose 1 SAD

PLACEBO COMPARATOR

• Placebo Ophthalmic Solution (0 mg/mL) N = 2 Single Ascending Dose (SAD, Day 1): 2 drops administered once to the right eye. Elution/Washout Phase: 1 day following SAD.

Drug: Placebo

BT01001 Ophthalmic Solution dose 1 MAD

EXPERIMENTAL

• BT01001 Ophthalmic Solution 5 mg/ml (0.5%） N = 6 Multiple Ascending Dose (MAD, Days 3-9): 2 drops administered twice daily (BID) from Days 3 to 8; on Day 9, 2 drops administered once in the morning only. Dose Administration: 2 drops with three minutes interval per dosing time.

Drug: BT01001 5 mg/ml（0.5%）

Placebo dose 1 MAD

PLACEBO COMPARATOR

• Placebo Ophthalmic Solution (0 mg/mL) N = 2 Multiple Ascending Dose (MAD, Days 3-9): 2 drops administered twice daily (BID) from Days 3 to 8; on Day 9, 2 drops administered once in the morning only. Dose Administration: 2 drops with three minutes interval per dosing time.

Drug: BT01001 15 mg/ml（1.5%）

BT01001 Ophthalmic Solution dose 2 SAD

EXPERIMENTAL

• BT01001 Ophthalmic Solution 10 mg/ml (1.0%） N = 6 Single Ascending Dose (SAD, Day 1): 2 drops administered once to the right eye. Elution/Washout Phase: 1 day following SAD.

Drug: BT01001 10 mg/ml（1.0%）

Placebo dose 2 SAD

PLACEBO COMPARATOR

• Placebo Ophthalmic Solution (0 mg/mL) N = 2 Single Ascending Dose (SAD, Day 1): 2 drops administered once to the right eye. Elution/Washout Phase: 1 day following SAD.

Drug: Placebo

BT01001 Ophthalmic Solution dose 2 MAD

EXPERIMENTAL

• BT01001 Ophthalmic Solution 10 mg/ml (1.0%） N = 6 Multiple Ascending Dose (MAD, Days 3-9): 2 drops administered twice daily (BID) from Days 3 to 8; on Day 9, 2 drops administered once in the morning only. Dose Administration: 2 drops with three minutes interval per dosing time.

Drug: BT01001 10 mg/ml（1.0%）

Placebo dose 2 MAD

PLACEBO COMPARATOR

• Placebo Ophthalmic Solution (0 mg/mL) N = 2 Multiple Ascending Dose (MAD, Days 3-9): 2 drops administered twice daily (BID) from Days 3 to 8; on Day 9, 2 drops administered once in the morning only. Dose Administration: 2 drops with three minutes interval per dosing time.

Drug: Placebo

BT01001 Ophthalmic Solution dose 3 SAD

EXPERIMENTAL

• BT01001 Ophthalmic Solution 15 mg/ml (1.5%） N = 6 Single Ascending Dose (SAD, Day 1): 2 drops administered once to the right eye. Elution/Washout Phase: 1 day following SAD.

Drug: BT01001 15 mg/ml（1.5%）

Placebo dose 3 SAD

PLACEBO COMPARATOR

• Placebo Ophthalmic Solution (0 mg/mL) N = 2 Single Ascending Dose (SAD, Day 1): 2 drops administered once to the right eye. Elution/Washout Phase: 1 day following SAD.

Drug: Placebo

BT01001 Ophthalmic Solution dose 3 MAD

EXPERIMENTAL

• BT01001 Ophthalmic Solution 15 mg/ml (1.5%） N = 6 Multiple Ascending Dose (MAD, Days 3-9): 2 drops administered twice daily (BID) from Days 3 to 8; on Day 9, 2 drops administered once in the morning only. Dose Administration: 2 drops with three minutes interval per dosing time.

Drug: BT01001 15 mg/ml（1.5%）

Placebo dose 3 MAD

PLACEBO COMPARATOR

• Placebo Ophthalmic Solution (0 mg/mL) N = 2 Multiple Ascending Dose (MAD, Days 3-9): 2 drops administered twice daily (BID) from Days 3 to 8; on Day 9, 2 drops administered once in the morning only. Dose Administration: 2 drops with three minutes interval per dosing time.

Drug: Placebo

BT01001 Ophthalmic Solution dose 4 MAD

EXPERIMENTAL

• BT01001 Ophthalmic Solution 15 mg/ml (1.5%） N = 6 Multiple Ascending Dose (MAD, Days 1-7): 2 drops administered three times daily (TID) from Days 1 to 6; on Day 7, 2 drops administered once in the morning only to the right eye. Dose Administration: 2 drops with three minutes interval per dosing time.

Drug: BT01001 15 mg/ml（1.5%）

Placebo dose 4 MAD

PLACEBO COMPARATOR

• Placebo Ophthalmic Solution (0 mg/mL) N = 2 Multiple Ascending Dose (MAD, Days 1-7): 2 drops administered three times daily (TID) from Days 1 to 6; on Day 7, 2 drops administered once in the morning only to the right eye. Dose Administration: 2 drops with three minutes interval per dosing time.

Drug: Placebo

Interventions

BT01001 5 mg/ml（0.5%） DRUG

BT01001 Ophthalmic Solution will be administered as topical instillation into the right eye of each subject according to the assigned dosing schedule.

Also known as: BT01001 Ophthalmic Solution

BT01001 Ophthalmic Solution dose 1 MAD; BT01001 Ophthalmic Solution dose 1 SAD

BT01001 10 mg/ml（1.0%） DRUG

BT01001 Ophthalmic Solution will be administered as topical instillation into the right eye of each subject according to the assigned dosing schedule.

Also known as: BT01001 Ophthalmic Solution

BT01001 Ophthalmic Solution dose 2 MAD; BT01001 Ophthalmic Solution dose 2 SAD

BT01001 15 mg/ml（1.5%） DRUG

BT01001 Ophthalmic Solution will be administered as topical instillation into the right eye of each subject according to the assigned dosing schedule.

Also known as: BT01001 Ophthalmic Solution

BT01001 Ophthalmic Solution dose 3 MAD; BT01001 Ophthalmic Solution dose 3 SAD; BT01001 Ophthalmic Solution dose 4 MAD; Placebo dose 1 MAD

Placebo DRUG

Placebo BT01001 Ophthalmic Solution will be administered as a topical instillation into the right eye of each subject following the same dosing schedule as the active treatment.

Also known as: BT01001 Ophthalmic Solution Placebo

Placebo dose 1 SAD; Placebo dose 2 MAD; Placebo dose 2 SAD; Placebo dose 3 MAD; Placebo dose 3 SAD; Placebo dose 4 MAD

Eligibility Criteria

• Age: 18 Years - 50 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64)

You may qualify if:

• Healthy male or female participant, 18 to 50 years of age at the time of screening, who are in good health based on medical history, physical examinations, vital signs, electrocardiogram (ECG), and clinical laboratory evaluations.
• Body Mass Index (BMI) between 18.0 and 27.0 kg/m² (inclusive).
• Negative alcohol breath test and negative urine drug screen at screening.
• Willing and able to comply with all study procedure and capable of good communication with study personnel.
• Participants of childbearing potential must agree to abstain from sexual intercourse or use an effective method of contraception from screening until 90 days after the final study drug administration; Female participants of childbearing potential must have a negative urine pregnancy test at screening.
• Able to understand the study procedure and voluntarily sign the written informed consent form.
• Corrected vision acuity ≥ 0.8 in both eyes.
• Intraocular pressure (IOP) \< 21 mmHg in each eye, with and inter-eye difference \< 4 mmHg.
• Slit-lamp and ophthalmoscopic examinations that are normal or show abnormalities considered not clinically significant by the investigator.

You may not qualify if:

• Known or suspected hypersensitivity to any component of the investigational product, or a history of multiple allergies two or more allergens).
• History or presence of any clinically significant diseases or abnormality, including but not limited to cardiovascular, cerebrovascular, respiratory, endocrine, metabolic, renal, hepatic, gastrointestinal, dermatologic, oncologic, hematologic, immunologic, infectious, neurologic or psychiatric conditions, or any acute or chronic condition that may interfere with study assessments.
• Participation in any investigational drug or medical device trial within 90 days prior to study drug administration.
• Use of prescription or over-the-counter medications within 14 days prior to dosing, or unwillingness to discontinue such medications during the study.
• Receipt of systemic corticosteroid therapy within 6 months prior to dosing.
• History of alcohol abuse or substance abuse within 2 years prior to dosing.
• Regular smoking of ≥ 5 cigarettes per day (or equivalent tobacco use) within 12 weeks prior to screening, or inability/unwillingness to abstain during the study.
• Average alcohol consumption \>14 units per week within 12 weeks prior to screening (1 unit approximately equivalent to 360 mL beer, 150 mL wine, or 45 mL of 40% spirits).
• History or evidence of intravenous illicit drug use; positive test for HIV, HCV, HBsAg, anti-HCV, anti-HIV, or Treponema pallidum antibody at screening.
• Blood donation or receipt of blood products within 30 days prior to dosing.
• History of bleeding disorders or coagulation abnormalities.
• Clinically significant abnormalities on physical examination, vital signs, 12-lead ECG, or laboratory results at screening.
• Abnormal findings that normalize on repeat testing may be accepted if assessed not clinically significant by the investigator.
• Current or past use of bariatric medications or history of bariatric surgery (e.g., gastric bypass).
• Impaired mental status or other factors that may compromise adherence to study requirements.

Sponsors & Collaborators

• Beyang Therapeutics Co., Ltd.: lead

Study Sites (1)

Shanghai General Hospital

Shanghai, 200080, China

RECRUITING

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 19, 2025
• First Posted: January 2, 2026
• Study Start: November 17, 2025
• Primary Completion: February 1, 2026
• Study Completion: April 1, 2026
• Last Updated: January 2, 2026

Record last verified: 2025-12

Locations

CN (1)