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

NCT05366283 | Completed Phase 1 FDA Drug

• 1 other identifier: PTX-001-005
• Study Type: interventional
• Target: 42
• Locations: 1 country
• Sites: 1

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.

Conditions

Drug Kinetics; Drug Effects on Physiology

Outcome Measures

Primary Outcomes (8)

• maximum concentration

  Blood samples collected up to 24 hours post dose

• time to maximum

  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

• AUC from time 0 to infinity

  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

• Elimination half-life

  Blood samples collected up to 24 hours post dose

• Clearance

  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

Secondary Outcomes (4)

• Number of subjects with adverse events in Part 1

  Up to Day 15 in Part 1

• Number of subjects with adverse events in Part 2

  Up to Day 22 in Part 2

• 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

• 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

Study Arms (7)

Cohort 1 (Part 1)

EXPERIMENTAL

Single dose of 250 microgram (mcg) sargramostim IV over 2 hours

Biological: sargramostim

Cohort 2 (Part 1)

EXPERIMENTAL

Single dose of 25 mcg sargramostim SC

Biological: sargramostim

Cohort 3 (Part 1)

EXPERIMENTAL

Single dose of 125 mcg sargramostim SC

Biological: sargramostim

Cohort 4 (Part 1)

EXPERIMENTAL

Single dose of 250 mcg sargramostim SC

Biological: sargramostim

Cohort 5 (Part 1)

EXPERIMENTAL

Single dose of 500 mcg sargramostim SC

Biological: sargramostim

Cohort 6 (Part 1)

EXPERIMENTAL

Single dose of 250 mcg sargramostim IH

Biological: sargramostim

Cohort 7 (Part 2)

EXPERIMENTAL

Two doses of 500 mg sargramostim SC, weekly

Biological: sargramostim

Interventions

sargramostim BIOLOGICAL

Sargramostim is a glycosylated, yeast-derived recombinant human granulocyte-macrophage colony-stimulating factor.

Also known as: Leukine®, rhu-GM-CSF

Cohort 1 (Part 1); Cohort 2 (Part 1); Cohort 3 (Part 1); Cohort 4 (Part 1); Cohort 5 (Part 1); Cohort 6 (Part 1); Cohort 7 (Part 2)

Eligibility Criteria

• Age: 18 Years - 55 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• Partner Therapeutics, Inc.: lead

Study Sites (1)

AltaSciences

Mount Royal, Quebec, H3P 3P1, Canada

Location

MeSH Terms

Interventions

sargramostim; Colony-Stimulating Factors

Intervention Hierarchy (Ancestors)

Glycoproteins; Glycoconjugates; Carbohydrates; Hematopoietic Cell Growth Factors; Cytokines; Intercellular Signaling Peptides and Proteins; Peptides; Amino Acids, Peptides, and Proteins; Proteins; Biological Factors

Study Officials

• Ila Joshi, PhD

  Partner Therapeutics, Inc.

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: BASIC SCIENCE
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 4, 2022
• First Posted: May 9, 2022
• Study Start: April 13, 2022
• Primary Completion: May 27, 2022
• Study Completion: June 16, 2022
• Last Updated: October 3, 2022

Record last verified: 2022-05

Data Sharing

• IPD Sharing: Will not share

Locations

CA (1)