A Study to Evaluate the Safety and Pharmacokinetics of Single and Multiple Doses of Prosetin in Healthy Volunteers

Study Overview
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Brief Summary
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This trial is a Phase 1a/1b, randomized, double-blind, placebo-controlled study designed to assess the safety, tolerability, and pharmacokinetics of single and multiple ascending oral doses of prosetin administered to healthy adult subjects.

Official Title
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A Phase 1, Randomized, Double-Blind, Placebo-Controlled, Dose-Escalating Study to Evaluate the Safety and Pharmacokinetics of Single Ascending and Multiple Doses of Prosetin in Healthy Volunteers

Conditions
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Amyotrophic Lateral Sclerosis

Intervention / Treatment
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* Drug: prosetin
* Drug: placebo


Participation Criteria
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Eligibility Criteria
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Key Inclusion Criteria: Adults between 18 and 65 years of age, inclusive BMI within 18.0 to 32.0 kg/m2, inclusive In good health, in the opinion of the Investigator, as determined by a physical examination, vital signs, 12-lead ECG, and clinical laboratory assessments Females of childbearing potential must agree to an approved method of contraception Key Exclusion Criteria: History or clinical manifestation of any significant metabolic, allergic, dermatological, hepatic, renal, hematological, pulmonary, cardiovascular, gastrointestinal, musculoskeletal, neurological, or psychiatric disorder Any episodes of vertigo in the previous 12 months prior to screening or any medical history of seizures Active autoimmune conditions such as systemic lupus erythematosus A diagnosis of cancer or evidence of continued disease within five years before screening

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: Treatment
Allocation: Randomized
Intervention Model: Sequential Assignment
Masking: Double


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Prosetin<br>Part A: single-ascending dose; Part B: multiple doses (7 days) | Drug: prosetin<br>* oral solution<br>|
| Placebo Comparator: Placebo<br>Part A: single-ascending dose; Part B: multiple doses (7 days) | Drug: placebo<br>* oral solution<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Incidence and severity of adverse events (AEs) |  | Up to 42 days | 
| Incidence of significant clinical laboratory test abnormalities |  | Up to 42 days | 
| Incidence of significant vital signs abnormalities |  | Up to 42 days | 
| Incidence of significant electrocardiogram (ECG) abnormalities |  | Up to 42 days | 
| Incidence of significant physical, neurological, and/or ophthalmic examination abnormalities |  | Up to 42 days | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Maximum concentration (Cmax) of prosetin in plasma |  | Up to 42 days | 
| Maximum concentration (Cmax) of prosetin in cerebrospinal fluid (CSF) |  | Up to 42 days | 
| Area under the concentration versus time curve (AUC) of prosetin in plasma |  | Up to 42 days | 
| Area under the concentration versus time curve (AUC) of prosetin in CSF |  | Up to 42 days |