Long-Term, Open Label Extension Study of Pemziviptadil (PB1046) in PAH Subjects Following Completion of Study PB1046-PT-CL-0004

• Register: ClinicalTrials.gov
• Last refreshed on: 20 September 2021
• Main ID: NCT03795428
• Date of registration: 18/12/2018
• Prospective Registration: Yes
• Primary sponsor: PhaseBio Pharmaceuticals Inc.
• Public title: Long-Term, Open Label Extension Study of Pemziviptadil (PB1046) in PAH Subjects Following Completion of Study PB1046-PT-CL-0004 VIP Extend
• Scientific title: A Long-Term, Open Label Extension Study of Pemziviptadil (PB1046) Subcutaneous Injections in Pulmonary Arterial Hypertension Subjects Following Completion of Study PB1046-PT-CL-0004
• Date of first enrolment: April 10, 2019
• Target sample size: 63
• Recruitment status: Recruiting
• URL: https://clinicaltrials.gov/show/NCT03795428
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Sequential Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

United States

Key inclusion & exclusion criteria

Inclusion Criteria:

- Subjects must have completed Week 17 / End of Study of PB1046-PT-CL-0004;

- Willing and able to sign a written Informed Consent (IC) prior to all study-related
procedures;

- Agrees to use a medically acceptable method of contraception (both male and female
patients) throughout the entire study period and continuing for 30 days after their
last dose of study drug. if the possibility of conception exists. Medically acceptable
methods of contraception include the following: abstinence (not having sex), vasectomy
(with confirmed negative sperm counts), condoms and partner using vaginal spermicide
and/or cervical cap with spermicide or sponge; oral, implantable, or injectable
contraceptives (starting ?2 months before dosing), diaphragm with vaginal spermicide,
intrauterine device, surgical sterilization (?6 months after surgery). Female subjects
?45 years of age of non-childbearing potential are defined as being surgically sterile
by bilateral tubal ligation, bilateral oophorectomy, or hysterectomy. Female subjects
45to-60 years of age, inclusive, who are post-menopausal for at least 1 year, and have
a follicle-stimulating hormone (FSH) level confirmation indicating post-menopausal
status, will be considered to be of non-childbearing potential. Female subjects > 60
years of age are considered post-menopausal and of non-childbearing potential;

- Willing and able to understand and follow instructions, return to the study unit for
specified study visits; and, be able to participate in the study through the Stable
Dose Maintenance Period, at a minimum.

Exclusion Criteria:

- Concomitant medical disorder, condition, or history, that in the opinion of the
Investigator, would impair the subject's ability to participate in or complete the
requirements of the study;

- Pregnant or lactating female subjects;

- Significant liver dysfunction as measured by any one of the following during
participation in PB1046-PT-CL-0004. (If exclusionary laboratory results become
available after the subject has enrolled in PB1046-PT-CL-0006 they should be
discontinued. a. alanine aminotransferase (ALT) > 3.0 times upper limit of normal
(ULN) or; b. aspartate aminotransferase (AST) > 3.0 times ULN or; c. serum bilirubin =
1.6 mg/dL.

- Recent history of substance abuse that, in the opinion of the Investigator, would
impair the subject's ability to participate in or complete the requirements of the
study;

- In the opinion of the principal investigator (PI), any major surgical procedure within
90 days, or a planned surgical procedure during the study period; which would impact
participation in PB1046-PT-CL-0006.

- Other new medical or psychiatric conditions which, in the opinion of the Investigator,
would place the subject at increased risk, would preclude obtaining voluntary consent,
or would confound the objectives of the study;

- Known hypersensitivity to study drug or any of the excipients of the drug formulation.



Age minimum: 18 Years
Age maximum: 79 Years
Gender: All

Health Condition(s) or Problem(s) studied

Pulmonary Arterial Hypertension

Intervention(s)

Drug: Pemziviptadil (PB1046) Injection

Primary Outcome(s)

Incidence of Immunogenicity [Time Frame: Duration of extension study - Starting up to 30 days prior to first dose of study drug in original study (PB1046-PT-CL-0004/0005) and completing 28 days after last dose.]

12-Lead ECG - Incidence of clinically significant abnormal ECG findings as measured by 12 Lead ECG [Time Frame: Duration of extension study - Starting the day of first dose and completing 28 days after last dose.]

Change in Diastolic Blood Pressure from baseline [Time Frame: Duration of extension study - Starting the day of first dose and completing 28 days after last dose.]

Change in Oral Body Temperature from baseline [Time Frame: Duration of extension study - Starting the day of first dose and completing 28 days after last dose.]

Incidence and Severity of Adverse Events [Time Frame: Duration of extension study - Starting the day of first dose and completing 28 days after last dose.]

Change in Systolic Blood Pressure from baseline [Time Frame: Duration of extension study - Starting the day of first dose and completing 28 days after last dose.]

Incidence of Clinical Laboratory Abnormalities [Time Frame: Duration of extension study - Starting the day of first dose and completing 28 days after last dose.]

Change in Heart Rate from baseline [Time Frame: Duration of extension study - Starting the day of first dose and completing 28 days after last dose.]

Change in Respiratory Rate from baseline [Time Frame: Duration of extension study - Starting the day of first dose and completing 28 days after last dose.]

Secondary Outcome(s)

Change from baseline in NT-proBNP [Time Frame: Duration of extension study - Starting the day of first dose and completing 28 days after last dose.]

Incidence of Clinical Worsening [Time Frame: Duration of extension study - Starting the day of first dose and completing 28 days after last dose.]

Change from baseline in NYHA/WHO Functional Class (FC) [Time Frame: Duration of extension study - Starting the day of first dose and completing 28 days after last dose.]

Change from baseline in 6MWD (6 minute walk distance test) [Time Frame: Duration of extension study - Starting the day of first dose and completing 28 days after last dose.]

Change from baseline in Borg Dyspnea Index (BDI) [Time Frame: Duration of extension study - Starting the day of first dose and completing 28 days after last dose.]

Survival [Time Frame: Duration of extension study - Starting the day of first dose and completing 28 days after last dose.]

Change from baseline in emPHasis-10 (Health Related Quality of Life) score [Time Frame: Duration of extension study - Starting the day of first dose and completing 28 days after last dose.]

Change in REVEAL Registry Risk Calculator Score [Time Frame: Duration of extension study - Starting the day of first dose and completing 28 days after last dose.]

Secondary ID(s)

PB1046-PT-CL-0006

Source(s) of Monetary Support

Please refer to primary and secondary sponsors