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

Study Overview
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Brief Summary
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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.

Official Title
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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

Conditions
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Healthy Volunteers

Intervention / Treatment
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* Drug: BPN-14967
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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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.

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 65 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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Accepts Healthy Volunteers

Study Plan
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How is the study designed?
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Design Details

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


Arms and Interventions

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

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | 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 |