Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Treprostinil Palmitil · 9 trials · 5 indications
Determination of the effect of mild, moderate, and severe hepatic impairment on the PK of TP and TRE following a single dose of TPIP, when compared to normal hepatic function.
Determination of the effect of mild, moderate, and severe hepatic impairment on the PK of TP and TRE following a single dose of TPIP, when compared to normal hepatic function.
Determination of the effect of mild, moderate, and severe hepatic impairment on the PK of TP and TRE following a single dose of TPIP, when compared to normal hepatic function.
Determination of the effect of mild, moderate, and severe hepatic impairment on the PK of TP and TRE following a single dose of TPIP, when compared to normal hepatic function.
Determination of the effect of mild, moderate, and severe hepatic impairment on the PK of TP and TRE following a single dose of TPIP, when compared to normal hepatic function.
Determination of the effect of mild, moderate, and severe hepatic impairment on the PK of TP and TRE following a single dose of TPIP, when compared to normal hepatic function.
Determination of the effect of mild, moderate, and severe hepatic impairment on the PK of TP and TRE following a single dose of TPIP, when compared to normal hepatic function.
Safety and tolerability of single and multiple doses of treprostinil inhalation powder will be determined in healthy participants.
| Arm | Type | Description |
|---|---|---|
| Treprostinil Palmitil Inhalation Powder | EXPERIMENTAL | Participants will receive TPIP, once daily (QD), at a starting dose of 80 micrograms (μg) up to maximum tolerated dose (up to 1280 μg) for 24 weeks. |
| Placebo | PLACEBO_COMPARATOR | Participants will receive a TPIP-matching placebo, QD, for 24 weeks. |
| Treprostinil Palmitil Inhalation Powder (TPIP) | EXPERIMENTAL | Participants transitioning from study INS1009-311 (NCT07179380) will undergo an initial double-dummy titration with the stable dose from the lead-in study and either TPIP or placebo for 4 weeks. They will then receive open-label TPIP at a stable maintenance dose with optional escalation (80-1280 micrograms once daily) for the remainder of the 104-week treatment period. |
| Treprostinil Palmitil | EXPERIMENTAL | Participants will be administered TPIP once per day at a starting dose of 80 micrograms (μg). Participants will be titrated up to the highest tolerated dose for each individual participant of between 80 μg and 640 μg during the initial 3 weeks of treatment. The overall treatment period will be 16 weeks. |
| Group 1: Treprostinil Palmitil Inhalation Powder | EXPERIMENTAL | Healthy participants with normal hepatic function will receive a single dose of TPIP on Day 1. |
| Group 2: Treprostinil Palmitil Inhalation Powder | EXPERIMENTAL | Participants with mild hepatic impairment (classified based on the numerical Child-Pugh total score of 5 to 6) will receive a single dose of TPIP on Day 1. |
| Group 3: Treprostinil Palmitil Inhalation Powder | EXPERIMENTAL | Participants with moderate hepatic impairment (classified based on the numerical Child-Pugh total score of 7 to 9) will receive a single dose of TPIP on Day 1. |
| Group 4: Treprostinil Palmitil Inhalation Powder | EXPERIMENTAL | Participants with severe hepatic impairment (classified based on the numerical Child-Pugh total score of 10 to 15) will receive a single dose of TPIP on Day 1. |
| Part A (SAD Cohort 1): TPIP | EXPERIMENTAL | Participants in the single ascending dose (SAD) Cohort 1 received a single dose of TPIP at Dose A, Dose B, or Dose C by oral inhalation on Day 1. |
| Part A (SAD Cohort 2): TPIP or Placebo | EXPERIMENTAL | Participants in SAD Cohort 2 received a single dose of TPIP at Dose D or matching placebo by oral inhalation on Day 1. |
| Part A (SAD Cohort 3): TPIP or Placebo | EXPERIMENTAL | Participants in SAD Cohort 3 received a single dose of TPIP at Dose E or matching placebo by oral inhalation on Day 1. |
| Part B (MAD Cohort 1): TPIP or Placebo | EXPERIMENTAL | Participants in the multiple ascending dose (MAD) Cohort 1 received TPIP at Dose B, Dose C or matching placebo, once daily (QD) by oral inhalation on Days 1 through 7. |
| Part B (MAD Cohort 2): TPIP or Placebo | EXPERIMENTAL | Participants in MAD Cohort 2 received TPIP up to Dose D or matching placebo, QD by oral inhalation on Days 1 through 7. |
| Name | Type | Description |
|---|---|---|
| Treprostinil Palmitil Inhalation Powder | DRUG | Oral inhalation using a capsule-based dry powder inhaler device. |
| Placebo | DRUG | Oral inhalation using a capsule-based dry powder inhaler device. |
| Treprostinil Palmitil | DRUG | Administered by oral inhalation, using a Plastiape capsule-based dry powder inhaler. |
Inclusion Criteria * Participants must have a diagnosis of World Health Organisation (WHO) Group 1 pulmonary hypertension (PAH) in any of the following subtypes, in accordance with European Society of Cardiology European Respiratory Society (ESC/ERS) Guidelines: * Idiopathic PAH * Heritable PA...