Study to Evaluate Pharmacokinetics, Pharmacodynamics, and Safety of Sargramostim Administered Subcutaneously, Intravenously, or by Inhalation

Phase 1

Completed

• Conditions: Drug Kinetics; Drug Effects on Physiology
• Interventions: Biological: sargramostim

• Registration Number: NCT05366283
• Lead Sponsor: Partner Therapeutics, Inc.

Brief Summary

This single center, single ascending dose (SAD) and repeat dose study in healthy adults comprises 2 parts. Part 1 will consist of 6 SAD cohorts, in which different dose levels of sargramostim will be given by intravenous (IV) infusion, subcutaneous (SC) injection, or inhalation (IH) administration. Part 2 will consist of 1 repeat dose cohort in which sargramostim will begiven by SC administration.

Blood samples for PK assessment will be collected before and over 24 hours after each study drug administration. Blood samples for PD assessment will be collected before and up to 14 days after drug administration in Part 1, as well as before the first and up to 14 days after the second drug administration in Part 2. Safety and tolerability will be assessed throughout the study. For Cohort 5 of Part 1 only, 3 subjects will provide a CSF sample for PK assessmen tonce after SC drug administration.

Detailed Description

Not available

Study Timeline

• Study Start: 2022-04-13
• Status Verified: 2022-05
• Study First Submitted: 2022-05-04
• Study First Submitted QC: 2022-05-04
• Study First Posted: 2022-05-09
• Primary Completion: 2022-05-27
• Study Completion: 2022-06-16
• Last Update Submitted: 2022-09-30
• Last Update Posted: 2022-10-03

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 42

Inclusion Criteria

• Healthy adult male and female participants
• Nonsmoker or ex-smoker
• Able to provide informed consent and comply with all study procedures
• Body Mass Index within 18.5-30.0 kilograms/meter squared, inclusive
• Females of childbearing potential willing to use highly-effective method of birth control.

Exclusion Criteria

• Females who are pregnant or breastfeeding
• History of allergic to sargramostim or its excipients, other human GM-CSFs, or other yeast-derived products.
• History of severe allergic reactions to other drugs.
• History of or any current medical condition or laboratory finding that may jeopardize completion of the study, present an increased risk to the subject, or compromise interpretation of the study. .
• Immunization with COVID-19 vaccine within 14 days of the study.
• Scheduled immunization with COVID-19 vaccine during the study
• Use of prescription drugs within 28 days of the study or requirement for maintenance drugs during the study.
• Participation in another investigational drug study within 28 days

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Cohort 5 (Part 1)	sargramostim	Single dose of 500 mcg sargramostim SC
Cohort 1 (Part 1)	sargramostim	Single dose of 250 microgram (mcg) sargramostim IV over 2 hours
Cohort 2 (Part 1)	sargramostim	Single dose of 25 mcg sargramostim SC
Cohort 7 (Part 2)	sargramostim	Two doses of 500 mg sargramostim SC, weekly
Cohort 4 (Part 1)	sargramostim	Single dose of 250 mcg sargramostim SC
Cohort 6 (Part 1)	sargramostim	Single dose of 250 mcg sargramostim IH
Cohort 3 (Part 1)	sargramostim	Single dose of 125 mcg sargramostim SC

Primary Outcome Measures

Name	Time	Method
maximum concentration	Blood samples collected up to 24 hours post dose
Area under the concentration-time curve (AUC) from time 0 to 24 hours	Blood samples collected up to 24 hours post dose
Steady state volume of distribution (IV administration only)	Blood samples collected up to 24 hours post dose
time to maximum	Blood samples collected up to 24 hours post dose
AUC from time 0 to infinity	Blood samples collected up to 24 hours post dose
Elimination half-life	Blood samples collected up to 24 hours post dose
Clearance	Blood samples collected up to 24 hours post dose
AUC from time 0 to 24 hours/infinity	Blood samples collected up to 24 hours post dose

Secondary Outcome Measures

Name	Time	Method
CD14/CD16/human leukocyte antigen-DR isotype (HLA-DR) absolute cell count	Up to Day 15 in Part 1, up to Day 22 in Part 2
Number of subjects with adverse events in Part 2	Up to Day 22 in Part 2
Number of subjects with adverse events in Part 1	Up to Day 15 in Part 1
Ratio of T-regulatory cells to T-effector cells in the blood.	Up to Day 15 in Part 1, up to Day 22 in Part 2

Trial Locations

Locations (1)

AltaSciences; 🇨🇦 Mount-Royal, Quebec, Canada