Subcutaneous Treprostinil as a Bridge to Lung Transplantation in Severe Pulmonary Hypertension: A Single-Arm Retrospective Study

September 19, 2024 updated by: Zhibin Xu, Guangzhou Institute of Respiratory Disease
This single-arm, retrospective study aims to evaluate the safety and efficacy of subcutaneous treprostinil as a bridge therapy for lung transplantation in patients with severe pulmonary hypertension (PH). A total of 46 patients with either pulmonary arterial hypertension (PAH) or chronic lung disease-associated PH (CLD-PH) were included, all of whom were referred for lung transplantation between January 1, 2015, and September 1, 2024. The study examines changes in clinical, functional, and hemodynamic parameters following 3-6 months of treprostinil therapy.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Detailed Description

This study evaluates the use of subcutaneous treprostinil as a bridge therapy for lung transplantation in patients with severe pulmonary hypertension (PH), including both pulmonary arterial hypertension (PAH) and chronic lung disease-associated PH (CLD-PH). This retrospective, single-center study included 46 patients treated between January 1, 2015, and September 1, 2024. All patients experienced significant clinical and hemodynamic impairment at the time of treprostinil initiation. The primary aim was to assess improvements in NYHA functional class and 6-minute walk distance (6-MWD) following 3-6 months of therapy. Secondary outcomes included NT-proBNP levels, echocardiographic parameters, and post-transplantation survival rates. The study also analyzed the incidence of primary graft dysfunction (PGD) and the continued use of treprostinil post-transplantation.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
46

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 4

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Patients diagnosed with severe pulmonary hypertension (PAH or CLD-PH) requiring lung transplantation
  • Age 18 years or older
  • Able to provide informed consent

Exclusion Criteria:

  • Patients with PH caused by left heart disease
  • Patients unable to undergo lung transplantation due to medical conditions
  • Patients with a history of severe drug allergies

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: Subcutaneous Treprostinil Group
Patients in this group received subcutaneous treprostinil, starting at a dose of 1.25 to 4 ng/kg/min and gradually titrated according to patient response. The target dose was over 20 ng/kg/min after 3 months of treatment. All patients were referred for lung transplantation during or after treprostinil treatment.
Drug: Subcutaneous Treprostinil
Patients in this group received subcutaneous treprostinil, starting at a dose of 1.25 to 4 ng/kg/min and gradually titrated according to patient response. The target dose was over 20 ng/kg/min after 3 months of treatment. All patients were referred for lung transplantation during or after treprostinil treatment.

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Change in NYHA Functional Class
Time Frame: Baseline to 3-6 months after treatment initiation
The change in NYHA functional class will be assessed after 3-6 months of treatment with subcutaneous treprostinil. Improvement will be defined as a reduction in class (e.g., from class IV to class III or II).
Baseline to 3-6 months after treatment initiation
Improvement in 6-Minute Walk Distance (6-MWD)
Time Frame: Baseline to 3-6 months after treatment initiation
The 6-minute walk distance (6-MWD) will be measured at baseline and after 3-6 months of subcutaneous treprostinil treatment to evaluate the improvement in exercise capacity.
Baseline to 3-6 months after treatment initiation

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Change in NT-proBNP Levels
Time Frame: Baseline to 3-6 months after treatment initiation
NT-proBNP levels will be measured before and after treatment to assess changes in cardiac function.
Baseline to 3-6 months after treatment initiation

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Guangzhou Institute of Respiratory Disease

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
January 1, 2015

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
September 1, 2024

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
September 1, 2024

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
September 18, 2024

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
September 18, 2024

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
September 20, 2024

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
September 23, 2024

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
September 19, 2024

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
September 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • 2022K-48

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

IPD Plan Description

There is no plan to share individual participant data (IPD) from this study. Due to privacy concerns and the sensitive nature of the health data collected, the research team has decided not to make individual-level data available to external researchers. All data will remain confidential and will be used solely for the purposes of this study, in compliance with institutional and ethical guidelines.

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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