A Study to Evaluate Plasma Gelsolin in Healthy Volunteers

Phase 1

Completed

• Conditions: Healthy; Volunteers
• Interventions: Drug: Recombinant human plasma gelsolin; Other: placebo

• Registration Number: NCT05789745
• Lead Sponsor: BioAegis Therapeutics Inc.

Brief Summary

Study BTI-101 is a Phase 1, randomized, double-blind, placebo-controlled, dose escalation, parallel design study to evaluate the safety, tolerability, and pharmacokinetics of IV rhu-pGSN or saline placebo administered as 5 doses each of 6, 12, 18, or 24 mg/kg of body weight. Each of 4 dosing cohorts will include 8 subjects randomized 3:1 rhu-pGSN:placebo (6 rhu-pGSN subjects:2 placebo subjects). Subjects will be healthy adult volunteers 18-55 years of age.

Detailed Description

Doses will be administered at 0 hours (Day 1), 12 hours (Day 1), 36 hours (Day 2), 60 hours (Day 3), and 84 hours (Day 4). Subjects will be kept in-house until after the last blood sample is taken on Day 5. Subjects will return for follow-up 7 days after the initiation of therapy (Day 8) and on Day 28 for the End-of-Study (EOS) Visit. After each cohort has completed the Day 8 visit, review of the safety results (including labs) will be conducted (and unblinded where appropriate) before the initiation of the next higher dose cohort.

To assess safety and tolerability, subjects will undergo physical examinations (including vital sign measurements), adverse event (AE) assessments, concomitant medication assessments, and safety laboratory testing. Blood samples will be collected for analysis of pGSN levels and antibodies against pGSN.

Study Timeline

• Study Start: 2023-03-14
• Study First Submitted: 2023-03-16
• Study First Submitted QC: 2023-03-16
• Study First Posted: 2023-03-29
• Primary Completion: 2023-05-24
• Study Completion: 2023-05-24
• Status Verified: 2023-06
• Last Update Submitted: 2023-06-27
• Last Update Posted: 2023-06-29

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 32

Inclusion Criteria

1. Healthy male or female adults 18 to 55 years of age without chronic or active acute medical conditions
2. Informed consent obtained from subject
3. Weight ≤100 kg and body mass index (BMI) <30 kg/m2
4. Willingness to use contraception during the course of the study, starting at screening and for at least 3 months after their final study treatment

Exclusion Criteria

1. Pregnant or lactating women
2. Acute illness during the month prior to screening
3. Circumstances that may require any medications (including prescription medication, over-the-counter medication, vitamins, or supplements) during the during the inpatient days of the study other than acetaminophen
4. Hospitalization during the year prior to screening
5. History of cancer or treatment with systemic chemotherapy or radiation therapy at any time
6. Transplantation of hematopoietic or solid organs
7. History of diabetes mellitus; myocardial infarction, angina, or other cardiovascular disease; stroke or cerebrovascular disease; chronic obstructive pulmonary disease (COPD) or asthma; deep vein thrombosis (DVT)/pulmonary embolism (PE); liver or kidney disease; clinically significant psychiatric condition; or active or chronic infection
8. Receipt of blood products during the year prior to screening
9. Chronic mechanical ventilation or dialysis
10. Any clinically significant abnormalities in clinical chemistry, hematology, or urinalysis results as judged by the Investigator
11. Any clinically significant abnormalities of vital signs, EKG or physical examination findings as judged by the Investigator
12. Positive results for recreational drugs during screening
13. Any other condition deemed by the Investigator as possibly interfering with the conduct of the study

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
rhu-pGSN	Recombinant human plasma gelsolin	Treated with 5 doses of rhu-pGSN
normal saline	placebo	Treated with 5 doses of saline

Primary Outcome Measures

Name	Time	Method
Adverse Events	28 days	Number of subjects who had an Adverse Event
Serious Adverse Events	28 days	Number of subjects who had a Serious Adverse Event

Secondary Outcome Measures

Name	Time	Method
Pharmacokinetics: pGSN Concentration	108 hours	Concentration of pGSN at baseline, 12 hours after dose 1, and 24 hours after doses 2 and 5
Pharmacokinetics: pGSN Area Under the Curve (AUC)	108 hours	Area Under the Curve (AUC) of pGSN concentration at 12 hours after dose 1, and 24 hours after doses 2 and 5
Pharmacokinetics: pGSN Half-life	108 hours	Half-life of pGSN concentration 12 hours after dose 1, and 24 hours after doses 2 and 5
Presence of Anti-drug Antibodies	28 days	Number of participants who tested positive for anti-pGSN antibodies at baseline, and on day 28

Trial Locations

Locations (1)

Nucleus Network; 🇺🇸 Saint Paul, Minnesota, United States