Pulmonary Arterial HypertensionPhase 2VIP ExtendNCT03795428

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

A Phase 2 study testing Pemziviptadil (PB1046) Injection in people with Pulmonary Arterial Hypertension, run by PhaseBio Pharmaceuticals Inc. at 16 sites in United States.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record

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Trial summary

This is a multi-center, Phase 2 Long-Term, Open Label Extension (OLE) Study to assess the safety and tolerability of pemziviptadil (PB1046) at an optimally titrated dose. This is a Long-Term, Open label Extension (OLE) Study for subjects with (PAH), having participated in double-blind Study PB1046-PT-CL-0004. Read more about this study

Who is it for?: Ages 18 to 79 years
Conditions the study is for: Pulmonary Arterial Hypertension
What is being tested?: Pemziviptadil (PB1046) Injection
Type of treatment: Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 25 participants
Where: 16 sitesUnited States
Study dates: 10 Apr 2019to 11 Jan 2022
Phase: Phase 2
Are there results?: None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 18 to 79 years
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

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.

Who cannot join

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.

Trial locations and contacts

16 sites in 1 country.

City, postcode or hospital

16 sites in 1 country

United States16 sites

Emory University, The Emory Clinic
Atlanta, Georgia, 30322
Tufts Medical Center
Boston, Massachusetts, 02111
Brigham and Women's Hospital
Boston, Massachusetts, 02115
The Lindner Center for Research and Education at The Christ Hospital
Cincinnati, Ohio, 45219
University of Cincinnati
Cincinnati, Ohio, 45267
University of Texas Southwestern Medical Center
Dallas, Texas, 75390
University of Iowa Hospitals and Clinics
Iowa City, Iowa, 52242
University of Kansas Medical Center
Kansas City, Kansas, 66160
University of California San Diego
La Jolla, California, 92037
University of Miami - Pulmonary Research Center
Miami, Florida, 33125
NYU Langone Health
New York, New York, 10016
INTEGRIS Baptist Medical Center
Oklahoma City, Oklahoma, 73112
Allegheny General Hospital
Pittsburgh, Pennsylvania, 15212
UPMC Presbyterian
Pittsburgh, Pennsylvania, 15213
University of Rochester Medical Center
Rochester, New York, 14642
University of California - Davis
Sacramento, California, 95817

Other trials in United States

About this study

Official 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

During this period, subjects will continue to be followed for safety and tolerability, as well as for periodic efficacy, quality of life data and immunogenicity. The study will continue per the schedule of events until such time when pemziviptadil (PB1046) is able to be self-administered, becomes commercially available to the subjects in a particular country or region, or the sponsor terminates the study due to lack of efficacy, safety or other reasons.

Read the detailed description

Subjects entering this study will enter from the double-blind Study PB1046-PT-CL-0004. The starting dose level of pemziviptadil (PB1046) for all subjects in this parent study was a sub-therapeutic or minimally effective dose (MED) of 0.2 mg/kg, administered by SC injection.

Subjects were randomized into the MED) Group or a dose-titration group. In the dose-titration group, individual subjects were titrated up to their maximum tolerated dose (MTD) in a blinded fashion, with the objective of titrating subjects up to a dose of at least 1.2 mg/kg or higher in the MTD Group, while subjects in the MED Group remained at the MED level of 0.2 mg/kg, and underwent "sham dose-titration" to maintain the blind.

Subjects entering the 0006 trial prior to implementation of this protocol amendment will remain blinded until such time that open label dosing will not unblind the 0004 study.

Conditions: Pulmonary Arterial Hypertension
Being tested: Pemziviptadil (PB1046) Injection
Medical subject headings: Pulmonary Arterial Hypertension

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Pemziviptadil (PB1046) InjectionDrug
Once-weekly subcutaneous injection

Study groups in NCT03795428
Study group	Role	What they receive
Pemziviptadil (PB1046) Injection-OL Active Drug-Up-Titration to Stable Dose	Experimental	Pemziviptadil (PB1046) Injection: Regardless of dose assignment, all subjects will be up-titrated in 0.2 mg/kg weekly increments, beginning with 0.4 mg/kg at Week 1, to the target dose of 1.2 mg/kg or higher depending on safety and tolerability. Receives: Drug: Pemziviptadil (PB1046) Injection

What is being measured

The primary outcome is the main question the study is designed to answer.

Incidence and Severity of Adverse Events
Measured: Duration of extension study - Starting the day of first dose and completing 28 days after last dose.
Incidence of Clinical Laboratory Abnormalities
Measured: Duration of extension study - Starting the day of first dose and completing 28 days after last dose.
Change in Diastolic Blood Pressure from baseline
Measured: Duration of extension study - Starting the day of first dose and completing 28 days after last dose.
Change in Systolic Blood Pressure from baseline
Measured: Duration of extension study - Starting the day of first dose and completing 28 days after last dose.
Change in Oral Body Temperature from baseline
Measured: Duration of extension study - Starting the day of first dose and completing 28 days after last dose.
Change in Respiratory Rate from baseline
Measured: Duration of extension study - Starting the day of first dose and completing 28 days after last dose.
Change in Heart Rate from baseline
Measured: Duration of extension study - Starting the day of first dose and completing 28 days after last dose.
12-Lead ECG - Incidence of clinically significant abnormal ECG findings as measured by 12 Lead ECG
Measured: Duration of extension study - Starting the day of first dose and completing 28 days after last dose.
Incidence of Immunogenicity
Incidence of positive immunogenicity results after receipt of study drug
Measured: 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.

Secondary outcomes (8)

Survival
Measured: 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)
Measured in meters walked in 6 minutes.
Measured: Duration of extension study - Starting the day of first dose and completing 28 days after last dose.
Change from baseline in NT-proBNP
Measured: 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)
Measured: 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
Scores, which assess breathlessness, fatigue, control and confidence, range from 0 to 50, higher scores indicate worse quality of life.
Measured: 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)
BDI scale as measured from 0 to 10 (0 being no breathlessness and 10 being maximal breathlessness)
Measured: Duration of extension study - Starting the day of first dose and completing 28 days after last dose.
Incidence of Clinical Worsening
As defined by any one of the following: 1. All cause mortality; 2. Hospitalization due to worsening PAH; 3. Initiation of parenteral prostacyclin; 4. Any three of the following: 15% decrease in 6MWD, Functional class III or IV symptoms, Addition of PAH therapy, Worsening right heart failure
Measured: Duration of extension study - Starting the day of first dose and completing 28 days after last dose.
Change in REVEAL Registry Risk Calculator Score
Risk scores range from 0 (Lowest risk) to 22 (Highest risk) in PAH subjects
Measured: Duration of extension study - Starting the day of first dose and completing 28 days after last dose.

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

10 Apr 201911 Jan 2022

First posted7 Jan 2019
Study start10 Apr 2019
Primary completion11 Jan 2022
Study completion11 Jan 2022

Why it stopped: Terminated: Study drug resupply delayed (Covid-19)

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Other trials for Pulmonary Arterial HypertensionStudies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 2
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Sequential
Model description: To protect the blind of the parent study (PB1046-PT-CL-0004), all subjects entering PB1046-PT-CL-0006 will commence dosing at Week 1 on 0.4 mg/kg and will be up-titrated in 0.2 mg/kg increments in an open label fashion for 9 weeks.
Masking: None (open label)
Enrolment: 25 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT03795428
Sponsor's protocol ID: PB1046-PT-CL-0006
Acronym: VIP Extend
Status last verified: Feb 2022
First submitted: 18 Dec 2018
Last updated: 22 Feb 2022
Results posted: No
Data monitoring committee: Yes
FDA-regulated drug: Yes
FDA-regulated device: No
Individual data sharing: No

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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All Pulmonary Arterial Hypertension trials

Study at a glance

From the registry record, last updated by the sponsor on 22 Feb 2022. Recruitment status is as the sponsor reported it: confirm with the study team.

Official 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
ClinicalTrials.gov ID: NCT03795428
Acronym / sponsor ID: VIP Extend / PB1046-PT-CL-0006
Conditions: Pulmonary Arterial Hypertension
Phase: Phase 2
Study type: Interventional
Recruitment status: TerminatedReason stopped: Terminated: Study drug resupply delayed (Covid-19)
Enrolment: 25 participants (actual)
Lead sponsor: PhaseBio Pharmaceuticals Inc.Industry (pharma, biotech, devices)
Study start: 10 Apr 2019 (actual)
Primary completion: 11 Jan 2022 (actual)
Study completion: 11 Jan 2022 (actual)
First posted: 7 Jan 2019 (actual)
Results: Not posted in the source record

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Detailed description

Subjects entering the 0006 trial prior to implementation of this protocol amendment will remain blinded until such time that open label dosing will not unblind the 0004 study.

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Sequential
Masking: None (open label)
Number of arms: 1
Model description: To protect the blind of the parent study (PB1046-PT-CL-0004), all subjects entering PB1046-PT-CL-0006 will commence dosing at Week 1 on 0.4 mg/kg and will be up-titrated in 0.2 mg/kg increments in an open label fashion for 9 weeks.

Arm	Type	Receives
Pemziviptadil (PB1046) Injection-OL Active Drug-Up-Titration to Stable Dose	Experimental	Drug: Pemziviptadil (PB1046) InjectionPemziviptadil (PB1046) Injection: Regardless of dose assignment, all subjects will be up-titrated in 0.2 mg/kg weekly increments, beginning with 0.4 mg/kg at Week 1, to the target dose of 1.2 mg/kg or higher depending on safety and tolerability.

Who may qualify

Ages: 18 to 79 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (4 listed)

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.

Key exclusion criteria (7 listed)

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.

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
Pemziviptadil (PB1046) InjectionOnce-weekly subcutaneous injection	Drug	Pemziviptadil (PB1046) Injection-OL Active Drug-Up-Titration to Stable Dose

Primary outcome

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.
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 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 Systolic 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.
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.
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.
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.
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.
Incidence of positive immunogenicity results after receipt of study drug

8 secondary outcomes

Survival
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 NT-proBNP
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 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 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.
Incidence of Clinical Worsening
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.

Results and safety

The study was stopped early (reason given: Terminated: Study drug resupply delayed (Covid-19)). Results are not available in the source record.

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 22 Feb 2022.
Individual participant dataSharing planned: No

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Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

The record lists no contact. Use the official record to reach the sponsor.

16 sites in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Pemziviptadil (PB1046) Injection in people with Pulmonary Arterial Hypertension, run by PhaseBio Pharmaceuticals Inc. at 16 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 4 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: stopped early, as of the sponsor's last update on 22 Feb 2022. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 16 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study was stopped early (reason given: Terminated: Study drug resupply delayed (Covid-19)). Results are not available in the source record.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

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.). The study lists 9 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03795428.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Feb 2022.