Clinical trial NCT05366283: Study to Evaluate Pharmacokinetics, Pharmacodynamics, and Safety of Sargramostim Administered Subcutaneously, Intravenously, or by Inhalation
Official title: Single Ascending Dose and Repeat Dose Study to Evaluate Pharmacokinetics, Pharmacodynamics, and Safety of Sargramostim Administered Subcutaneously, by Intravenous Infusion, or by Inhalation in Healthy Adult Subjects
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Drug Kinetics
Drug Effects on Physiology
Lead sponsor: Partner Therapeutics, Inc.
Sponsor class: INDUSTRY
First posted: 2022-05-09
Enrollment: 42
Sex: ALL
Minimum age (years): 18
Maximum age (years): 55
Standard ages: ADULT
Accepts healthy volunteers: yes
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: BASIC_SCIENCE

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.


Eligibility criteria: 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

Primary outcomes:
measure: maximum concentration
timeFrame: Blood samples collected up to 24 hours post dose
measure: time to maximum
timeFrame: Blood samples collected up to 24 hours post dose
measure: Area under the concentration-time curve (AUC) from time 0 to 24 hours
timeFrame: Blood samples collected up to 24 hours post dose
measure: AUC from time 0 to infinity
timeFrame: Blood samples collected up to 24 hours post dose
measure: AUC from time 0 to 24 hours/infinity
timeFrame: Blood samples collected up to 24 hours post dose
measure: Elimination half-life
timeFrame: Blood samples collected up to 24 hours post dose
measure: Clearance
timeFrame: Blood samples collected up to 24 hours post dose
measure: Steady state volume of distribution (IV administration only)
timeFrame: Blood samples collected up to 24 hours post dose
Secondary outcomes:
measure: Number of subjects with adverse events in Part 1
timeFrame: Up to Day 15 in Part 1
measure: Number of subjects with adverse events in Part 2
timeFrame: Up to Day 22 in Part 2
measure: Ratio of T-regulatory cells to T-effector cells in the blood.
timeFrame: Up to Day 15 in Part 1, up to Day 22 in Part 2
measure: CD14/CD16/human leukocyte antigen-DR isotype (HLA-DR) absolute cell count
timeFrame: Up to Day 15 in Part 1, up to Day 22 in Part 2
Other outcomes:
Locations:
city: Mount-Royal
country: Canada
facility: AltaSciences
state: Quebec
Study officials:
affiliation: Partner Therapeutics, Inc.
name: Ila Joshi, PhD
role: STUDY_DIRECTOR
References: