Drug KineticsPhase 1NCT05366283
Study to Evaluate Pharmacokinetics, Pharmacodynamics, and Safety of Sargramostim Administered Subcutaneously, Intravenously, or by Inhalation
A Phase 1 study testing sargramostim in people with Drug Kinetics and Drug Effects on Physiology, run by Partner Therapeutics, Inc. at 1 site in Canada.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 55 yearsHealthy volunteers can take part
- Conditions the study is for
- Drug Kinetics, Drug Effects on Physiology
- What is being tested?
- sargramostim
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 42 participants
- Where
- 1 siteCanada
- Study dates
- 13 Apr 2022to 16 Jun 2022
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 55 years
- All sexes
- Healthy volunteers accepted
- Adult
Who may be able to join
- 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.
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
About this study
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
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
- Being tested
- sargramostim
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
sargramostimBiological
Also called Leukine®, rhu-GM-CSF
Sargramostim is a glycosylated, yeast-derived recombinant human granulocyte-macrophage colony-stimulating factor.
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 (Part 1) | Experimental | Single dose of 250 microgram (mcg) sargramostim IV over 2 hours Receives: Biological: sargramostim |
| Cohort 2 (Part 1) | Experimental | Single dose of 25 mcg sargramostim SC Receives: Biological: sargramostim |
| Cohort 3 (Part 1) | Experimental | Single dose of 125 mcg sargramostim SC Receives: Biological: sargramostim |
| Cohort 4 (Part 1) | Experimental | Single dose of 250 mcg sargramostim SC Receives: Biological: sargramostim |
| Cohort 5 (Part 1) | Experimental | Single dose of 500 mcg sargramostim SC Receives: Biological: sargramostim |
| Cohort 6 (Part 1) | Experimental | Single dose of 250 mcg sargramostim IH Receives: Biological: sargramostim |
| Cohort 7 (Part 2) | Experimental | Two doses of 500 mg sargramostim SC, weekly Receives: Biological: sargramostim |
What is being measured
The primary outcome is the main question the study is designed to answer.
maximum concentration
Measured: Blood samples collected up to 24 hours post dose
time to maximum
Measured: Blood samples collected up to 24 hours post dose
Area under the concentration-time curve (AUC) from time 0 to 24 hours
Measured: Blood samples collected up to 24 hours post dose
AUC from time 0 to infinity
Measured: Blood samples collected up to 24 hours post dose
AUC from time 0 to 24 hours/infinity
Measured: Blood samples collected up to 24 hours post dose
Elimination half-life
Measured: Blood samples collected up to 24 hours post dose
Clearance
Measured: Blood samples collected up to 24 hours post dose
Steady state volume of distribution (IV administration only)
Measured: Blood samples collected up to 24 hours post dose
Secondary outcomes (4)
Number of subjects with adverse events in Part 1
Measured: Up to Day 15 in Part 1
Number of subjects with adverse events in Part 2
Measured: Up to Day 22 in Part 2
Ratio of T-regulatory cells to T-effector cells in the blood.
Measured: 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
Measured: Up to Day 15 in Part 1, up to Day 22 in Part 2
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 May 2022
- Study start13 Apr 2022
- Primary completion27 May 2022
- Study completion16 Jun 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Drug KineticsStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Basic science
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 42 participants (actual)
Sponsor and investigators
- Lead sponsor
- Partner Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Ila Joshi, PhD, Study director, Partner Therapeutics, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05366283
- Sponsor's protocol ID
- PTX-001-005
- Status last verified
- May 2022
- First submitted
- 4 May 2022
- Last updated
- 3 Oct 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 Oct 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT05366283
- Acronym / sponsor ID
- PTX-001-005
- Conditions
- Drug Kinetics, Drug Effects on Physiology
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 42 participants (actual)
- Lead sponsor
- Partner Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- 13 Apr 2022 (actual)
- Primary completion
- 27 May 2022 (actual)
- Study completion
- 16 Jun 2022 (actual)
- First posted
- 9 May 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Basic science
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 7
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 (Part 1) | Experimental | Biological: sargramostimSingle dose of 250 microgram (mcg) sargramostim IV over 2 hours |
| Cohort 2 (Part 1) | Experimental | Biological: sargramostimSingle dose of 25 mcg sargramostim SC |
| Cohort 3 (Part 1) | Experimental | Biological: sargramostimSingle dose of 125 mcg sargramostim SC |
| Cohort 4 (Part 1) | Experimental | Biological: sargramostimSingle dose of 250 mcg sargramostim SC |
| Cohort 5 (Part 1) | Experimental | Biological: sargramostimSingle dose of 500 mcg sargramostim SC |
| Cohort 6 (Part 1) | Experimental | Biological: sargramostimSingle dose of 250 mcg sargramostim IH |
| Cohort 7 (Part 2) | Experimental | Biological: sargramostimTwo doses of 500 mg sargramostim SC, weekly |
Who may qualify
- Ages
- 18 to 55 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (8 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| sargramostimAlso: Leukine®, rhu-GM-CSFSargramostim is a glycosylated, yeast-derived recombinant human granulocyte-macrophage colony-stimulating factor. | Biological | 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) |
Primary outcome
maximum concentration
Time frame: Blood samples collected up to 24 hours post dose
time to maximum
Time frame: Blood samples collected up to 24 hours post dose
Area under the concentration-time curve (AUC) from time 0 to 24 hours
Time frame: Blood samples collected up to 24 hours post dose
AUC from time 0 to infinity
Time frame: Blood samples collected up to 24 hours post dose
AUC from time 0 to 24 hours/infinity
Time frame: Blood samples collected up to 24 hours post dose
Elimination half-life
Time frame: Blood samples collected up to 24 hours post dose
Clearance
Time frame: Blood samples collected up to 24 hours post dose
Steady state volume of distribution (IV administration only)
Time frame: Blood samples collected up to 24 hours post dose
4 secondary outcomes
Number of subjects with adverse events in Part 1
Time frame: Up to Day 15 in Part 1
Number of subjects with adverse events in Part 2
Time frame: Up to Day 22 in Part 2
Ratio of T-regulatory cells to T-effector cells in the blood.
Time frame: 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
Time frame: Up to Day 15 in Part 1, up to Day 22 in Part 2
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Oct 2022.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Ila Joshi, PhD study director
Partner Therapeutics, Inc.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing sargramostim in people with Drug Kinetics and Drug Effects on Physiology, run by Partner Therapeutics, Inc. at 1 site in Canada.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 3 Oct 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
maximum concentration (time frame: Blood samples collected up to 24 hours post dose). The study lists 8 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Oct 2022.