Pulmonary Arterial HypertensionPhase 1NCT03315507

A Study to Assess the Safety, Tolerability, and Hemodynamic Response of PB1046 in Subjects With PAH

A Phase 1 study testing PB1046 Subcutaneous Injection in people with Pulmonary Arterial Hypertension, run by PhaseBio Pharmaceuticals Inc. at 2 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

PB1046-PT-CL-0005 is an open-label, dose-titration study to assess the safety, tolerability, and hemodynamic effects of individually dose-titrated PB1046 administered by weekly subcutaneous injection for 8 weeks in adult subjects with PAH who have a permanently implanted hemodynamic monitor in the distal pulmonary… Read more about this study

Who is it for?: Ages 18 years and older
Conditions the study is for: Pulmonary Arterial Hypertension
What is being tested?: PB1046 Subcutaneous Injection
Type of treatment: Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 3 participants
Where: 2 sitesUnited States
Study dates: 20 Oct 2017to 8 Aug 2019
Phase: Phase 1
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 years and older
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

Willing and able to provide written informed consent and follow all study-related procedures;
Confirmed diagnosis of Pulmonary Arterial Hypertension (WHO Group 1) and WHO Functional Class II or III by clinical diagnostic criteria assessed by the Investigator and have a permanently implanted pulmonary artery hemodynamic monitor (IHM);
Adult subjects ≥18 years of age willing and able to utilize contraception as needed for 30 days after their last dose of study drug;
Body mass index ≥ 18 kg/m2 and ≤ 47 kg/m2;
Receipt of Investigator-directed stable (no change in dose or addition or removal of a therapy) medical-therapy in accordance with local standard of care for the management of PAH for 30 days prior to screening and between screening and first dose and are in stable clinical condition;
Screening hemoglobin ≥ 9.0 g/dL secondary to the volume of blood to be collected during the study period;
Willing and able to return to the study unit for specified study visits, or accommodate home visits;
Willing and able to transmit hemodynamics via IHM and monitor systemic blood pressure while at home and record results.

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;
Concomitant medical disorder that is expected to limit the subject's life-expectancy to ≤ 1 year;
Pregnant or lactating female subjects;
First positive result from serology testing at visit 1 (screening labs) for human immunodeficiency virus, hepatitis B surface antigen, or hepatitis C virus prior to first dose;
Participation in another investigational study within 30 days prior to screening or are taking part in a non-medication study which, in the opinion of the Investigator, would interfere with the study compliance or outcome assessments;
Use of bosentan therapy for PAH within 30 days prior to screening or during study participation;

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Sustained systolic blood pressure (SBP) < 95 mmHg and/or diastolic blood pressure (DBP) < 50 mmHg (confirmed by a duplicate seated reading) on at least 3 consecutive readings (self-monitored or office) prior to first dose, or overt symptomatic hypotension;
Sustained resting heart rate >110 beats per minute (BPM) at screening (V1) or prior to first dose (confirmed by duplicate assessments of office vital signs or consecutive ECG assessments) on at least 3 consecutive readings prior to first dose;
Clinically significant renal dysfunction as measured by the estimated glomerular filtration rate (eGFR) of < 40 mL/min/1.73m2 as calculated by the MDRD equation: eGFR = 175 x (Creat / 88.4)-1.154 x (Age)-0.203 x (0.742 if female) x (1.212 if African American) (conventional units);
Clinically significant liver dysfunction as measured by any one of the following: a. alanine aminotransferase (ALT) >3.0 time ULN or; b. aspartate aminotransferase (AST) >3.0 time ULN or; c. serum bilirubin ≥ 1.6 mg/dL;
Known 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;
Any major surgical procedure within 30 days prior to screening or planned surgical procedure during the study period;
In-patient hospitalization (defined as greater than 23 hours) within 30 days of subject dosing;
Enrollment within the past 3 months prior to screening or plans to enroll during the study into a cardiopulmonary rehabilitation program;
Other medical or psychiatric condition which, in the opinion of the Investigator, would place the subject at increased risk or would preclude obtaining voluntary consent or would confound the objectives of study;
Known hypersensitivity to study drug or any of the excipients of the drug formulation.

Trial locations and contacts

2 sites in 1 country.

United States2 sites

The Ohio State University Wexner Medical Center
Columbus, Ohio, 43210
Allegheny General Hospital
Pittsburgh, Pennsylvania, 15212

Other trials in United States

About this study

Official title: An Open-Label, Dose Titration Study to Assess the Safety, Tolerability, and Hemodynamic Response of PB1046, A Sustained-Release Analogue of Vasoactive Intestinal Peptide, In Adult Subjects With Pulmonary Arterial Hypertension

Conditions: Pulmonary Arterial Hypertension
Being tested: PB1046 Subcutaneous 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.

PB1046 Subcutaneous InjectionDrug
Eight weekly doses of PB1046.

Study groups in NCT03315507
Study group	Role	What they receive
PB1046 Injection	Experimental	PB1046 Subcutaneous Injection Receives: Drug: PB1046 Subcutaneous Injection

What is being measured

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

Adverse Events
Incidence and severity of AEs (described descriptively) and their relationship to study drug
Measured: Pre-dose to 28 days after last dose
Vital Signs (Blood pressure)
Changes from baseline in blood pressure and their relationship to study drug
Measured: Prior to eight weekly doses and 1 and 3 hours after the final dose and 1, 2, 3, 5, 7 and 28 days after final dose.
Vital Signs (Heart rate)
Changes from baseline in heart rate and their relationship to study drug
Measured: Prior to eight weekly doses and 1 and 3 hours after the final dose and 1, 2, 3, 5, 7 and 28 days after final dose.
Laboratory Parameters (Lipids)
Changes from baseline in lipids and their relationship to study drug
Measured: Pre-dose and 7 and 28 days after last dose
Laboratory Parameters (Serum chemistry)
Changes from baseline in serum chemistry and their relationship to study drug
Measured: Pre-dose and prior to doses 2-8 and 7 and 28 days after last dose
Laboratory Parameters (Urinalysis)
Changes from baseline in urinalysis and their relationship to study drug
Measured: Pre-dose and prior to doses 2-8 and 7 and 28 days after last dose
Laboratory Parameters (NT-pro-BNP)
Changes from baseline in NT-pro-BNP and their relationship to study drug
Measured: Prior to doses 1-8 and 7 and 28 days after last dose
Mean Pulmonary Artery Pressure
Change from baseline in mean pulmonary artery pressure
Measured: Prior to eight weekly doses and 1 and 3 hours after the final dose and 1, 2, 3, 5, 7, 8 and 28 days after final dose
Cardiac Index
Change from baseline in cardiac index
Measured: Prior to eight weekly doses and 1 and 3 hours after the final dose and 1, 2, 3, 5, 7, 8 and 28 days after final dose
Total pulmonary resistance
Change from baseline in total pulmonary resistance
Measured: Prior to eight weekly doses and 1 and 3 hours after the final dose and 1, 2, 3, 5, 7, 8 and 28 days after final dose
Laboratory Parameters (Fasting Plasma Glucose)
Changes from baseline in fasting plasma glucose and their relationship to study drug
Measured: Pre-dose and 7 and 28 days after last dose
Laboratory Parameters (Hematology)
Changes from baseline in hematology and their relationship to study drug
Measured: Pre-dose and prior to doses 2-8 and 7 and 28 days after last dose

Secondary outcomes (10)

Pharmacokinetic Dose Exposures (AUC (0-t))
Area under the curve over the dosing interval (AUC(0-t))
Measured: Prior to eight weekly doses and at 1, 3, 24, 48, 72,120, 168 and 192 hours after final dose.
Pharmacokinetic Dose Exposures (AUC (0-tmax))
Area under the curve concentration-time profile (AUC(0-tmax))
Measured: Prior to eight weekly doses and at 1, 3, 24, 48, 72,120, 168 and 192 hours after final dose.
Pharmacokinetic Dose Exposures (Cmax)
Maximum serum concentration (Cmax)
Measured: Prior to eight weekly doses and at 1, 3, 24, 48, 72,120, 168 and 192 hours after final dose.
Pharmacokinetic Dose Exposures (Tmax)
Time to Cmax (Tmax)
Measured: Prior to eight weekly doses and at 1, 3, 24, 48, 72,120, 168 and 192 hours after final dose.
Pharmacokinetic Dose Exposures (Ctrough)
Pre-dose serum concentrations at Weeks 1 through 7 and the concentrations after the last dose
Measured: Prior to eight weekly doses and at 1, 3, 24, 48, 72,120, 168 and 192 hours after final dose.
Pharmacokinetic Dose Exposures (t1/2)
Half-life (t1/2)
Measured: Prior to eight weekly doses and at 1, 3, 24, 48, 72,120, 168 and 192 hours after final dose.
Pharmacokinetic Dose Exposures (Lambda z)
Lambda z
Measured: Prior to eight weekly doses and at 1, 3, 24, 48, 72,120, 168 and 192 hours after final dose.
Pharmacokinetic Dose Exposures (Cl/F)
Apparent clearance (Cl/F)
Measured: Prior to eight weekly doses and at 1, 3, 24, 48, 72,120, 168 and 192 hours after final dose.
Pharmacokinetic Dose Exposures (Vd/F)
Volume of distribution (Vd/F)
Measured: Prior to eight weekly doses and at 1, 3, 24, 48, 72,120, 168 and 192 hours after final dose.
Immunogenicity
Incidence of immunogenicity
Measured: Prior to eight weekly doses and on study days 59, 77 and 105.

Timeline

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

20 Oct 20178 Aug 2019

First posted20 Oct 2017
Study start20 Oct 2017
Primary completion8 Aug 2019
Study completion8 Aug 2019

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 1
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Model description: Open Label
Masking: None (open label)
Enrolment: 3 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT03315507
Sponsor's protocol ID: PB1046-PT-CL-0005
Status last verified: Oct 2019
First submitted: 2 Oct 2017
Last updated: 8 Oct 2019
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 8 Oct 2019. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: An Open-Label, Dose Titration Study to Assess the Safety, Tolerability, and Hemodynamic Response of PB1046, A Sustained-Release Analogue of Vasoactive Intestinal Peptide, In Adult Subjects With Pulmonary Arterial Hypertension
ClinicalTrials.gov ID: NCT03315507
Acronym / sponsor ID: PB1046-PT-CL-0005
Conditions: Pulmonary Arterial Hypertension
Phase: Phase 1
Study type: Interventional
Recruitment status: Completed
Enrolment: 3 participants (actual)
Lead sponsor: PhaseBio Pharmaceuticals Inc.Industry (pharma, biotech, devices)
Study start: 20 Oct 2017 (actual)
Primary completion: 8 Aug 2019 (actual)
Study completion: 8 Aug 2019 (actual)
First posted: 20 Oct 2017 (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.

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1
Model description: Open Label

Arm	Type	Receives
PB1046 Injection	Experimental	Drug: PB1046 Subcutaneous InjectionPB1046 Subcutaneous Injection

Who may qualify

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

Key inclusion criteria (8 listed)

Willing and able to provide written informed consent and follow all study-related procedures;
Confirmed diagnosis of Pulmonary Arterial Hypertension (WHO Group 1) and WHO Functional Class II or III by clinical diagnostic criteria assessed by the Investigator and have a permanently implanted pulmonary artery hemodynamic monitor (IHM);
Adult subjects ≥18 years of age willing and able to utilize contraception as needed for 30 days after their last dose of study drug;
Body mass index ≥ 18 kg/m2 and ≤ 47 kg/m2;
Receipt of Investigator-directed stable (no change in dose or addition or removal of a therapy) medical-therapy in accordance with local standard of care for the management of PAH for 30 days prior to screening and between screening and first dose and are in stable clinical condition;

Key exclusion criteria (16 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;
Concomitant medical disorder that is expected to limit the subject's life-expectancy to ≤ 1 year;
Pregnant or lactating female subjects;
First positive result from serology testing at visit 1 (screening labs) for human immunodeficiency virus, hepatitis B surface antigen, or hepatitis C virus prior to first dose;
Participation in another investigational study within 30 days prior to screening or are taking part in a non-medication study which, in the opinion of the Investigator, would interfere with the study compliance or outcome assessments;

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
PB1046 Subcutaneous InjectionEight weekly doses of PB1046.	Drug	PB1046 Injection

Primary outcome

Adverse Events
Time frame: Pre-dose to 28 days after last dose
Incidence and severity of AEs (described descriptively) and their relationship to study drug
Vital Signs (Blood pressure)
Time frame: Prior to eight weekly doses and 1 and 3 hours after the final dose and 1, 2, 3, 5, 7 and 28 days after final dose.
Changes from baseline in blood pressure and their relationship to study drug
Vital Signs (Heart rate)
Time frame: Prior to eight weekly doses and 1 and 3 hours after the final dose and 1, 2, 3, 5, 7 and 28 days after final dose.
Changes from baseline in heart rate and their relationship to study drug
Laboratory Parameters (Lipids)
Time frame: Pre-dose and 7 and 28 days after last dose
Changes from baseline in lipids and their relationship to study drug
Laboratory Parameters (Serum chemistry)
Time frame: Pre-dose and prior to doses 2-8 and 7 and 28 days after last dose
Changes from baseline in serum chemistry and their relationship to study drug
Laboratory Parameters (Urinalysis)
Time frame: Pre-dose and prior to doses 2-8 and 7 and 28 days after last dose
Changes from baseline in urinalysis and their relationship to study drug
Laboratory Parameters (NT-pro-BNP)
Time frame: Prior to doses 1-8 and 7 and 28 days after last dose
Changes from baseline in NT-pro-BNP and their relationship to study drug
Mean Pulmonary Artery Pressure
Time frame: Prior to eight weekly doses and 1 and 3 hours after the final dose and 1, 2, 3, 5, 7, 8 and 28 days after final dose
Change from baseline in mean pulmonary artery pressure
Cardiac Index
Time frame: Prior to eight weekly doses and 1 and 3 hours after the final dose and 1, 2, 3, 5, 7, 8 and 28 days after final dose
Change from baseline in cardiac index
Total pulmonary resistance
Time frame: Prior to eight weekly doses and 1 and 3 hours after the final dose and 1, 2, 3, 5, 7, 8 and 28 days after final dose
Change from baseline in total pulmonary resistance
Laboratory Parameters (Fasting Plasma Glucose)
Time frame: Pre-dose and 7 and 28 days after last dose
Changes from baseline in fasting plasma glucose and their relationship to study drug
Laboratory Parameters (Hematology)
Time frame: Pre-dose and prior to doses 2-8 and 7 and 28 days after last dose
Changes from baseline in hematology and their relationship to study drug

10 secondary outcomes

Pharmacokinetic Dose Exposures (AUC (0-t))
Time frame: Prior to eight weekly doses and at 1, 3, 24, 48, 72,120, 168 and 192 hours after final dose.
Pharmacokinetic Dose Exposures (AUC (0-tmax))
Time frame: Prior to eight weekly doses and at 1, 3, 24, 48, 72,120, 168 and 192 hours after final dose.
Pharmacokinetic Dose Exposures (Cmax)
Time frame: Prior to eight weekly doses and at 1, 3, 24, 48, 72,120, 168 and 192 hours after final dose.
Pharmacokinetic Dose Exposures (Tmax)
Time frame: Prior to eight weekly doses and at 1, 3, 24, 48, 72,120, 168 and 192 hours after final dose.
Pharmacokinetic Dose Exposures (Ctrough)
Time frame: Prior to eight weekly doses and at 1, 3, 24, 48, 72,120, 168 and 192 hours after final dose.
Pharmacokinetic Dose Exposures (t1/2)
Time frame: Prior to eight weekly doses and at 1, 3, 24, 48, 72,120, 168 and 192 hours after final dose.
Pharmacokinetic Dose Exposures (Lambda z)
Time frame: Prior to eight weekly doses and at 1, 3, 24, 48, 72,120, 168 and 192 hours after final dose.
Pharmacokinetic Dose Exposures (Cl/F)
Time frame: Prior to eight weekly doses and at 1, 3, 24, 48, 72,120, 168 and 192 hours after final dose.
Pharmacokinetic Dose Exposures (Vd/F)
Time frame: Prior to eight weekly doses and at 1, 3, 24, 48, 72,120, 168 and 192 hours after final dose.
Immunogenicity
Time frame: Prior to eight weekly doses and on study days 59, 77 and 105.

Results and safety

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 8 Oct 2019.
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.

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

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing PB1046 Subcutaneous Injection in people with Pulmonary Arterial Hypertension, run by PhaseBio Pharmaceuticals Inc. at 2 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 8 inclusion and 16 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: finished, as of the sponsor's last update on 8 Oct 2019. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 2 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

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 is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

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?

Adverse Events (time frame: Pre-dose to 28 days after last dose). The study lists 12 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 NCT03315507.

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 8 Oct 2019.