Stopped: Terminated: Study drug resupply delayed (Covid-19)
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.
Age range
18 Years – 79 Years
Sex
ALL
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Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
Incidence and severity of AEs
Timeframe: 172 days - Starting up to 30 days prior to first dose and completing 28 days after last dose.
Incidence of Clinical Laboratory Abnormalities
Timeframe: 172 days - Starting up to 30 days prior to first dose and completing 28 days after last dose.
Changes in Diastolic Blood Pressure
Timeframe: 172 days - Starting up to 30 days prior to first dose and completing 28 days after last dose.
Changes in Systolic Blood Pressure
Timeframe: 172 days - Starting up to 30 days prior to first dose and completing 28 days after last dose.
Changes in Oral Body Temperature
Timeframe: 172 days - Starting up to 30 days prior to first dose and completing 28 days after last dose.
Changes in Respiratory Rate
Timeframe: 172 days - Starting up to 30 days prior to first dose and completing 28 days after last dose.
Changes in Heart Rate
Timeframe: 172 days - Starting up to 30 days prior to first dose and completing 28 days after last dose.
12-Lead ECG - Incidence of clinically significant findings
Timeframe: 172 days - Starting up to 30 days prior to first dose and completing 28 days after last dose.
Immunogenicity
Timeframe: 172 days - Starting up to 30 days prior to first dose and completing 8 weeks after last dose. May be extended in the event that result does not return to baseline in time allotted.
Change in baseline in pulmonary vascular resistance (PVR)
Timeframe: 142 days - Pre-dose (up to 30 days prior to first dose) and post-dose 16.