A Study to Evaluate Plasma Gelsolin in Healthy Volunteers

Single-center, Randomized, Double-blind, Placebo-controlled, Ascending-Dose Study to Evaluate the Safety and Pharmacokinetics of Recombinant Human Plasma Gelsolin (Rhu-pGSN) After Intravenous Administration to Healthy Volunteers

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.

Study Overview

• Status: Completed
• Conditions: Healthy; Volunteers
• Intervention / Treatment: Drug: Recombinant human plasma gelsolin; Other: placebo

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 Type: Interventional
• Enrollment (Actual): 32
• Phase: Phase 1

Contacts and Locations

• Study Contact: Name: Mark J DiNubile, MD; Phone Number: 609-706-5866; Email: mdinubile@bioaegistx.com
• Study Contact Backup: Name: Susan L Levinson, PhD; Email: slevinson@bioaegistx.com

Study Locations

• United States
  • Minnesota
    • Saint Paul, Minnesota, United States, 55114
      • Nucleus Network

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 55 years (Adult)
• Accepts Healthy Volunteers: Yes

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: rhu-pGSN; Treated with 5 doses of rhu-pGSN	Drug: Recombinant human plasma gelsolin; intravenous administration of either 6, 12, 18, or 24 mg/kg at time 0, 12, 36, 60 and 84 hours
Placebo Comparator: normal saline; Treated with 5 doses of saline	Other: placebo; intravenous administration of saline control at time 0, 12, 36, 60 and 84 hours

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Safety -- SAE frequency	serious adverse events	28 days
Safety -- severe AE frequency	Grade 3/4 severe adverse events	28 days

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
pharmacokinetics -- pGSN levels over time after dosing	pGSN levels	108 hours
antidrug antibodies -- frequency before and 28 days post-dose	anti-pGSN antibodies	28 days

Collaborators and Investigators

• Sponsor: BioAegis Therapeutics Inc.
• Investigators: Study Director: Mark J DiNubile, MD, BioAegis Therapeutics

Study record dates

Study Major Dates

• Study Start (Actual): March 14, 2023
• Primary Completion (Actual): May 24, 2023
• Study Completion (Actual): May 24, 2023

Study Registration Dates

• First Submitted: March 16, 2023
• First Submitted That Met QC Criteria: March 16, 2023
• First Posted (Actual): March 29, 2023

Study Record Updates

• Last Update Posted (Actual): June 29, 2023
• Last Update Submitted That Met QC Criteria: June 27, 2023
• Last Verified: June 1, 2023

More Information

Terms related to this study

• Keywords: safety; pharmacokinetics
• Other Study ID Numbers: BTI-101

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

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