Safety and Tolerability of Escalating Doses of BPN-14967 in Healthy Adults

A Phase 1, Randomized, Double-Blind, Placebo-Controlled, Single Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Food Effect of BPN-14967 in Healthy Adult Subjects

This is single center, randomized, double-blind, placebo-controlled, single ascending dose study to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics and food effect of BPN-14967 in healthy adult subjects.

Study Overview

• Status: Completed
• Conditions: Healthy Volunteers
• Intervention / Treatment: Drug: Placebo; Drug: BPN-14967

• Study Type: Interventional
• Enrollment (Actual): 40
• Phase: Phase 1

Contacts and Locations

Study Locations

• United States
  • Texas
    • Austin, Texas, United States, 78744
      • PPD Phase I Clinic

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 65 years (Adult, Older Adult)
• Accepts Healthy Volunteers: Yes
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• The subject is male or female (not of childbearing potential), 18 to 65 years of age, inclusive, at screening.
• The subject voluntarily consents to participate in this study and
• provides written informed consent before the start of any study-specific procedures.
• The subject is willing and able to remain in the study unit for the entire duration of the confinement period and return for outpatient visits.
• Female subjects must be of non-childbearing potential (defined as surgically sterile [i.e., had a bilateral tubal ligation, hysterectomy, or
• bilateral oophorectomy at least 6 months before the dose of study drug] or postmenopausal for at least 1 year before study drug administration confirmed by FSH test at screening).
• Male subjects must be surgically sterile (i.e., vasectomy) for at least 3 months before screening; or agree to use a condom with spermicide when sexually active with a female partner. Male subjects must also agree to refrain from sperm donation for 90 days after study drug administration.
• The subject has a body mass index (BMI) of 18 to 30 kg/m2, inclusive, at screening and weighs 50 to 100 kg (110-220 pounds), inclusive, at screening and Check-in.
• The subject is considered to be in stable health by the investigator

Exclusion Criteria

• Any significant acute or chronic medical illness including history or presence of clinically significant cardiovascular, pulmonary, hepatic, renal, hematologic, gastrointestinal, endocrine, immunologic, dermatologic, neurologic, oncologic, or psychiatric disease
• Any recent viral or bacterial infection.
• Participated in any clinical study in last 6 weeks.
• History of significant drug allergy
• History of significant vision, ocular or retinal disorder.
• Recent surgery, blood transfusion, drug or alcohol abuse and use of tobacco or nicotine containing products in past month.
• Evidence of organ dysfunction or any clinically significant deviation from normal in physical examination, vital signs, ECGs, or clinical laboratory determinations

Other protocol-defined inclusion/exclusion criteria could apply.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Basic Science
• Allocation: Randomized
• Interventional Model: Sequential Assignment
• Masking: Double

Number of Arms

5

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Cohort 1 (fasted condition); 10 mg BPN-14967 or placebo	Drug: Placebo; Oral capsules; Drug: BPN-14967; BPN-14967 oral capsules
Experimental: Cohort 2 (fasted condition); 25 mg BPN-14967 or placebo	Drug: Placebo; Oral capsules; Drug: BPN-14967; BPN-14967 oral capsules
Experimental: Cohort 3 (fasted condition); 50 mg BPN-14967 or placebo	Drug: Placebo; Oral capsules; Drug: BPN-14967; BPN-14967 oral capsules
Experimental: Cohort 4 (fed condition); 10 mg BPN-14967 or placebo	Drug: Placebo; Oral capsules; Drug: BPN-14967; BPN-14967 oral capsules
Experimental: Cohort 5 (high-fat fed condition); 10 mg BPN-14967 or placebo	Drug: Placebo; Oral capsules; Drug: BPN-14967; BPN-14967 oral capsules

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Area under the plasma concentration versus time curve from time 0 to the last timepoint with quantifiable concentration [AUC(0-t)]	Up to Day 8
Area under the plasma concentration versus time curve from time 0 extrapolated to infinity [AUC(0-inf)]	Up to Day 8
Maximum observed plasma concentration (Cmax)	Up to Day 8
Time to maximum observed plasma concentration (Tmax)	Up to Day 8
Terminal elimination rate constant	Up to Day 8
Terminal phase half-life (t1/2)	Up to Day 8
Apparent total body clearance (CL/F)	Up to Day 8
Apparent volume of distribution (Vz/F)	Up to Day 8
Number of participants with Adverse Events (AEs), Serious Adverse Events (SAEs) and AEs leading to discontinuation	Up to Day 10
Number of participants with Clinical Laboratory Results Abnormalities	Up to Day 10
Number of participants with Vital Sign Abnormalities	Up to Day 10
Number of participants with Physical Examination Abnormalities	Up to Day 10
Number of participants with 12-lead Electrocardiogram (ECG) Abnormalities	Up to Day 10
Number of participants with changes in visual acuity	Baseline and Day 10
Number of participants with Ocular Examination Abnormalities	Up to Day 10
Number of participants with changes in color vision	Baseline and Day 10

Collaborators and Investigators

• Sponsor: Belite Bio, Inc
• Investigators: Study Director: Konstantin Petrukhin, PhD, Professor of Ophthalmic Science, Department of Ophthalmology, Columbia University Medical Center

Study record dates

Study Major Dates

• Study Start (Actual): July 19, 2019
• Primary Completion (Actual): December 20, 2019
• Study Completion (Actual): December 20, 2019

Study Registration Dates

• First Submitted: July 1, 2019
• First Submitted That Met QC Criteria: July 1, 2019
• First Posted (Actual): July 2, 2019

Study Record Updates

• Last Update Posted (Actual): June 18, 2021
• Last Update Submitted That Met QC Criteria: June 15, 2021
• Last Verified: June 1, 2021

More Information

Terms related to this study

• Other Study ID Numbers: BPN-14967-001

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO
• IPD Plan Description: After completion of the study, data may be considered for reporting at a scientific meeting or for publication in a scientific journal. In these cases, the sponsor will be responsible for these activities and will work with the investigators to determine how the manuscript is written and edited, the number and order of authors, the publication to which it will be submitted, and any other related issues. The sponsor has final approval authority of all such issues. Data are the property of the sponsor and cannot be published without their prior authorization, but data and any publication thereof will not be unduly withheld.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No