A RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, ASCENDING DOSE STUDY TO EVALUATE PLASMA GELSOLIN IN HEALTHY VOLUNTEERS

Overview

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.

Full Title of Study: “A PHASE 1, SINGLE-CENTER, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, MULTIPLE ASCENDING DOSE STUDY FOR THE EVALUATION OF THE SAFETY, TOLERABILITY, AND PHARMACOKINETICS OF RECOMBINANT HUMAN PLASMA GELSOLIN (Rhu-pGSN) FOLLOWING INTRAVENOUS ADMINISTRATION TO HEALTHY VOLUNTEERS”

Study Type

• Study Type: Interventional
• Study Design
  • Allocation: Randomized
  • Intervention Model: Parallel Assignment
  • Primary Purpose: Treatment
  • Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
• Study Primary Completion Date: May 31, 2023

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.

Interventions

• 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
• Other: placebo
  • intravenous administration of saline control at time 0, 12, 36, 60 and 84 hours

Arms, Groups and Cohorts

• Experimental: rhu-pGSN
  • Treated with 5 doses of rhu-pGSN
• Placebo Comparator: normal saline
  • Treated with 5 doses of saline

Clinical Trial Outcome Measures

Primary Measures

• Safety — SAE frequency
  • Time Frame: 28 days
  • serious adverse events
• Safety — severe AE frequency
  • Time Frame: 28 days
  • Grade 3/4 severe adverse events

Secondary Measures

• pharmacokinetics — pGSN levels over time after dosing
  • Time Frame: 108 hours
  • pGSN levels
• antidrug antibodies — frequency before and 28 days post-dose
  • Time Frame: 28 days
  • anti-pGSN antibodies

Participating in This Clinical Trial

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

Gender Eligibility: All

Minimum Age: 18 Years

Maximum Age: 55 Years

Are Healthy Volunteers Accepted: Accepts Healthy Volunteers

Investigator Details

• Lead Sponsor
  • BioAegis Therapeutics Inc.
• Provider of Information About this Clinical Study
  • Sponsor
• Overall Official(s)
  • Mark J DiNubile, MD, Study Director, BioAegis Therapeutics
• Overall Contact(s)
  • Mark J DiNubile, MD, 609-706-5866, mdinubile@bioaegistx.com