Ignite Creation Date:
2025-12-25 @ 4:12 AM
Ignite Modification Date:
2025-12-26 @ 3:11 AM
Study NCT ID:
NCT03556020
Status:
TERMINATED
Last Update Posted:
2022-08-23
First Post:
2018-06-01
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
Phase 2 Study to Assess Safety, Tolerability and Efficacy of Once Weekly SC Pemziviptadil (PB1046) in Subjects With Symptomatic PAH
Sponsor:
PhaseBio Pharmaceuticals Inc.
Organization:
Study Overview
Official Title:
A Randomized, Double-Blind, Parallel Group, Phase 2 Study to Assess the Safety, Tolerability, and Efficacy of Once Weekly Subcutaneous (SC) Injections of a Sustained-Release Vasoactive Intestinal Peptide (VIP) Analogue, Pemziviptadil (PB1046), in Adult Subjects With Symptomatic Pulmonary Arterial Hypertension (PAH)
Status:
TERMINATED
Status Verified Date:
2022-08
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Terminated: Study drug resupply delayed (Covid-19)
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
VIP
Brief Summary:
This is a multi-center, randomized, double-blind, controlled, Phase 2 study to assess the safety, tolerability, and efficacy of pemziviptadil (PB1046) at the optimally titrated dose after 16 weeks of treatment. Subjects will be randomized in a 2:1 ratio to one of two parallel dose groups: a) high-dose group where PB1046 will be up-titrated from a 0.2 mg/kg minimally effective starting dose to a target high dose level of at least 1.2 mg/kg or higher to a maximally tolerated dose (MTD), or b) a low-dose group that will start at 0.2 mg/kg and remain at this minimally effective dose (MED) level with sham up-titration. The total treatment period will be comprised of 2 phases: 1) an initial 10 week dose titration phase in which weekly doses of PB1046 will be titrated (or sham titrated) up to a target dose level of at least 1.2 mg/kg or higher to the MTD, and 2) a maintenance of treatment phase that begins when subjects reach week 11 and continues for 6 weeks during which no further up-titration should occur.
Detailed Description:
The primary safety and tolerability objectives will be assessed by investigating the incidence and severity of adverse events (AEs) as well as changes from baseline in vital signs, laboratory parameters, ECGs and their relationship to pemziviptadil (PB1046). The primary efficacy objective will be assessed by investigating the change in PVR derived from right heart catheterization (RHC). Secondary efficacy objectives will be assessed by investigating the impact of pemziviptadil (PB1046) on change from baseline in 6 minute walk distance (6MWD) test and NT-proBNP, a prognostic biomarker for PAH, in the two groups (comparing the MTD and MED groups) at the end of the treatment period. In addition, the effect of pemziviptadil (PB1046) on other cardiopulmonary hemodynamic parameters (e.g. CI, mPAP, mRAP, wedge pressure and SvO2) as measured by RHC will be assessed. Changes in BDI, HRQoL, and NYHA/WHO (New York Heart Association/World Health Organization) FC will also be assessed.
An independent Data Safety Monitoring Board (DSMB) will periodically assess safety, efficacy and biomarker data to independently assess the overall safety profile of pemziviptadil (PB1046), to help adjudicate potential dose-limiting toxicities, and to monitor the overall benefit risk profile of pemziviptadil (PB1046) during the study. The DSMB will review the safety and tolerability data after the first 10 subjects while recruitment is ongoing.
An independent Data Safety Monitoring Board (DSMB) will periodically assess safety, efficacy and biomarker data to independently assess the overall safety profile of pemziviptadil (PB1046), to help adjudicate potential dose-limiting toxicities, and to monitor the overall benefit risk profile of pemziviptadil (PB1046) during the study. The DSMB will review the safety and tolerability data after the first 10 subjects while recruitment is ongoing.
Study Oversight
Has Oversight DMC:
True
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?: