Clinical trial NCT06193031: A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of C16TR for Inhalation With Tyvaso® Cohort in Healthy Participants
Official title: A Phase 1, Randomized, Double-Blind, Placebo-Controlled Single Ascending Dose Study of C16TR for Inhalation to Determine Its Safety, Tolerability, and Pharmacokinetics With an Open-label Tyvaso® Cohort in Healthy Volunteers
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Healthy Volunteers
Lead sponsor: Insmed Incorporated
Sponsor class: INDUSTRY
First posted: 2024-01-05
Enrollment: 24
Sex: ALL
Minimum age (years): 18
Maximum age (years): 45
Standard ages: ADULT
Accepts healthy volunteers: yes
Allocation: RANDOMIZED
Intervention model: SEQUENTIAL
Primary purpose: TREATMENT

Brief summary: The primary purpose of this study is to determine the safety and tolerability of escalating doses of C16TR for inhalation in healthy participants.


Eligibility criteria: Inclusion Criteria:

* Have a body weight between 50 and 120 kg (females) or between 55 and 120 kg (males), inclusive, with a body mass index (BMI) between 19.0 and 32.0 kilograms per square meter (kg/m\^2), inclusive, at screening.
* Have a medical history, physical examination, vital signs, electrocardiogram (ECG) and clinical laboratory results within normal limits or considered not clinically significant by the Investigator at screening.
* Do not take any systemic or topical prescription, or nonprescription (over-the-counter \[OTC\]) medication (acetaminophen or ibuprofen are permitted upon principal investigator \[PI\] discretion) within 2 weeks or 5 half-lives (whichever is longer) before first dose of the study drugs until discharge from the study (unless prescribed by the Investigator to treat an AE).
* Agree to abstain from consuming alcohol at least 3 days prior to in-clinic confinement until discharge from the study.

Exclusion Criteria:

* Have a history of anaphylaxis, a previous documented hypersensitivity reaction, or a clinically significant idiosyncratic reaction to any drug.
* Have a clinically significant history of neurological, cardiovascular, respiratory, endocrine, hematological, hepatic, renal, gastrointestinal, genitourinary, pulmonary, and/or musculoskeletal disease; glaucoma; a psychiatric disorder, or any other chronic disease, whether controlled by medication or not.
* Have a history of orthostatic hypotension, or unexplained syncope.
* Have a history of additional risk factors for Torsades de Pointes (eg, heart failure, family history of Long QT Syndrome).
* Are positive for human immunodeficiency virus (HIV) infection, hepatitis B surface antigen (HBsAg), or the hepatitis C virus (HCV) antibody at screening.
* Are users or former users of nicotine-containing products with \> 10 pack-years of tobacco use history (including but not limited to cigarettes, cigars, and chewing or dipping tobacco), or users who stopped use or consumption (i.e., smoking, chewing, or pinching) of these nicotine-containing products less than 6 months before study drug administration or were using or had used topical or oral nicotine preparations for smoking cessation within the past 3 months before study drug administration.
* Have a history of alcohol abuse or a history of or current impairment of organ function reasonably related to alcohol abuse.
* Have a history or current evidence of abuse of licit or illicit drugs or a positive urine test for drugs of abuse.
* Have a history of abnormal bleeding tendencies.
* Donated any plasma within 7 days prior to first dosing, or has donated blood in excess of 450 mL, or had significant blood loss within 56 days prior to first dosing.
* Have any flu-like syndrome or other respiratory infection within 2 weeks of Day 1 or having been vaccinated with an attenuated live virus within 4 weeks of Day 1.
* Have a history of major surgery within 4 weeks or minor surgery within 2 weeks of screening.

Note: Other inclusion/exclusion criteria may apply.

Primary outcomes:
measure: Number of Participants who Experienced an Adverse Event (AE)
timeFrame: Up to 32 days
Secondary outcomes:
measure: Area Under the Plasma Concentration-time Curve (AUC) of Treprostinil and C16TR Post C16TR for Inhalation Dose
timeFrame: At multiple timepoints post dose on Days 2 to 4
measure: Cohort 1: AUC of Treprostinil Post Tyvaso® Dosing
timeFrame: At multiple timepoints post dose on Days 1 and 2 for Cohort 1
measure: Mean Change From Baseline in Corrected QT Interval by Fridericia (QTcF) for C16TR
timeFrame: Baseline up to Day 4 (Cohort 1) and Day 3 (Cohorts 2, 3, 4, and 5)
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: USA001
state: California
Study officials:
References: