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C16TR

Phase 1

Healthy Volunteers | Small molecule | Other |Insmed Incorporated|Last Updated: Jan 5, 2024

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
RandomizedDouble-BlindCONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment24
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT06193031A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of C16TR for Inhalation With Tyvaso® Cohort in Healthy ParticipantsPHASE1 COMPLETED 24Nov 17, 2015Dec 18, 2015Jan 5, 20241 United States
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Study Endpoints
Primary Endpoints
Number of Participants who Experienced an Adverse Event (AE)
Up to 32 days

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

Secondary Endpoints
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)
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelSEQUENTIAL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Cohort 1: Tyvaso®+C16TR Dose A or Tyvaso®+PlaceboEXPERIMENTALParticipants 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.
Cohort 2: C16TR Dose B or PlaceboEXPERIMENTALParticipants were randomized to receive a single dose of C16TR for inhalation (Dose B) or matching placebo on Day 1 in Cohort 2.
Cohort 3: C16TR Dose C or PlaceboEXPERIMENTALParticipants were randomized to receive a single dose of C16TR for inhalation (Dose C) or matching placebo on Day 1 in Cohort 3.
Interventions
NameTypeDescription
C16TRDRUGAdministered as inhalation using a Philips Micro device inhaler.
PlaceboDRUGPhosphate buffered saline (PBS) administered using Philips Micro device inhaler.
Tyvaso®DRUGAdministered as inhalation.
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Eligibility Criteria
Age Range18 Years — 45 Years
SexALL
Healthy VolunteersYes
Study Sites1

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, electr...

Countries:United States
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