A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of C16TR for Inhalation With Tyvaso® Cohort in Healthy Participants

NCT06193031 | Completed Phase 1 DMC FDA Drug

• 1 other identifier: INS1009-101
• Study Type: interventional
• Target: 24
• Locations: 1 country
• Sites: 1

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.

Conditions

Healthy Volunteers

Outcome Measures

Primary Outcomes (1)

• Number of Participants who Experienced an Adverse Event (AE)

  Safety and tolerability of escalating doses of C16TR for inhalation in healthy participants.

  Up to 32 days

Secondary Outcomes (3)

• Area Under the Plasma Concentration-time Curve (AUC) of Treprostinil and C16TR Post C16TR for Inhalation Dose

  At multiple timepoints post dose on Days 2 to 4

• Cohort 1: AUC of Treprostinil Post Tyvaso® Dosing

  At multiple timepoints post dose on Days 1 and 2 for Cohort 1

• Mean Change From Baseline in Corrected QT Interval by Fridericia (QTcF) for C16TR

  Baseline up to Day 4 (Cohort 1) and Day 3 (Cohorts 2, 3, 4, and 5)

Study Arms (3)

Cohort 1: Tyvaso®+C16TR Dose A or Tyvaso®+Placebo

EXPERIMENTAL

Participants received a single dose of Tyvaso® on Day 1, then randomized to receive either a single dose of C16TR for inhalation (Dose A) or matching placebo on Day 2 in Cohort 1.

Drug: C16TR; Drug: Placebo; Drug: Tyvaso®

Cohort 2: C16TR Dose B or Placebo

EXPERIMENTAL

Participants were randomized to receive a single dose of C16TR for inhalation (Dose B) or matching placebo on Day 1 in Cohort 2.

Drug: C16TR; Drug: Placebo

Cohort 3: C16TR Dose C or Placebo

EXPERIMENTAL

Participants were randomized to receive a single dose of C16TR for inhalation (Dose C) or matching placebo on Day 1 in Cohort 3.

Drug: C16TR; Drug: Placebo

Interventions

C16TR DRUG

Administered as inhalation using a Philips Micro device inhaler.

Also known as: Hexadecyl-treprostinil

Cohort 1: Tyvaso®+C16TR Dose A or Tyvaso®+Placebo; Cohort 2: C16TR Dose B or Placebo; Cohort 3: C16TR Dose C or Placebo

Placebo DRUG

Phosphate buffered saline (PBS) administered using Philips Micro device inhaler.

Cohort 1: Tyvaso®+C16TR Dose A or Tyvaso®+Placebo; Cohort 2: C16TR Dose B or Placebo; Cohort 3: C16TR Dose C or Placebo

Tyvaso® DRUG

Administered as inhalation.

Also known as: Inhaled treprostinil

Cohort 1: Tyvaso®+C16TR Dose A or Tyvaso®+Placebo

Eligibility Criteria

• Age: 18 Years - 45 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Insmed Incorporated: lead

Study Sites (1)

USA001

Los Angeles, California, 91206, United States

Location

Related Links

• Related Info

MeSH Terms

Interventions

hexadecyl-treprostinil; treprostinil

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 21, 2023
• First Posted: January 5, 2024
• Study Start: November 17, 2015
• Primary Completion: December 18, 2015
• Study Completion: December 18, 2015
• Last Updated: January 5, 2024

Record last verified: 2023-12

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)