Efficacy of Remodulin in Adults With Congenital Heart Disease (ACHD) and Pulmonary Hypertension

September 19, 2017 updated by: Jamil Aboulhosn, University of California, Los Angeles
The purpose of this study is to evaluate the efficacy of Remodulin in the treatment of adult patients with congenital heart disease and pulmonary hypertension. Baseline and post-treatment cardiopulmonary exercise tests will be performed.

Study Overview

Status

Withdrawn

Conditions

Intervention / Treatment

Detailed Description

A prospective, open-label, non-randomized multi-center study is proposed to assess the efficacy of Remodulin in adults with congenital heart disease and pulmonary hypertension. Subjects will be enrolled at UCLA and Ohio State University (PI: Curt Daniels, MD). The study will involve a six month trial of continuous subcutaneous Remodulin therapy, with assessments conducted prior to initiation of therapy and at 1 and 6 months following initiation of therapy. A separate initiation visit will be scheduled after the baseline visit in order to provide subjects with comprehensive training in the use and care of the Remodulin drug delivery system.

Baseline and post-treatment (6 month) assessments will include a history and physical examination, cardiopulmonary exercise test, six minute walking distance, serum brain natriuretic peptide (BNP), a Cambridge Pulmonary Hypertension Outcome Review (CAMPHOR) quality of life survey, and subjective assessment of functional capacity (New York Heart Association (NYHA) scale). The interim (1 month) follow-up visit will include a thorough review of adverse events associated with Remodulin therapy, functional class assessment, six minute walk distance, and serum BNP.

Remodulin will be initiated at 1.25 ng/kg/min and increased by 2-6 ng/kg/min weekly to a target dose of 40 ng/kg/min. If the initial infusion rate cannot be tolerated it will be reduced to 0.625 ng/kg/min. Changes in drug dose will be at the discretion of the investigators following review of interim events. If necessary, the dose may be decreased by 2 ng/kg/min every two days as tolerated.

Study Type

Interventional

Phase

  • Not Applicable

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • California
      • Los Angeles, California, United States, 90095
        • UCLA Medical Center
    • Ohio
      • Columbus, Ohio, United States, 43205
        • Ohio State University/Nationwide Children's Hospital

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

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • Age ≥ 18 years
  • Congenital heart disease with Pulmonary Arterial (PA) hypertension (repaired or unrepaired) defined as a mean resting directly measured Pulmonary Artery Pressure (PAP) of ≥35 mm Hg and/or doppler echo estimated PA systolic pressure ≥ 60 mm Hg.
  • Patients already on phosphodiesterase type 5 inhibitor (PDE-5), Endothelin Receptor Antagonist (ERA), or inhaled prostacyclin are not excluded

Exclusion Criteria:

  • Age < 18 years
  • Current intravenous or subcutaneous prostacyclin therapy
  • Resting systemic hypotension (Systolic blood pressure < 80 mm Hg)
  • Women who are pregnant or may become pregnant (unwilling to utilize effective contraception), as well as nursing mothers
  • Inability to ambulate

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: Supportive Care
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Remodulin
Subcutaneous (SQ) remodulin will be initiated at 1.25 ng/kg/min, and increased by 2-6 ng/kg/min weekly to a target dose of 40 ng/kg/min. If the initial infusion rate cannot be tolerated it will be reduced to 0.625 ng/kg/min. If subjects cannot tolerate the SQ therapy, we will attempt to switch to IV therapy.
Drug: Remodulin
Subcutaneous (SQ) remodulin will be initiated at 1.25 ng/kg/min, and increased by 2-6 ng/kg/min weekly to a target dose of 40 ng/kg/min. If the initial infusion rate cannot be tolerated it will be reduced to 0.625 ng/kg/min. If subjects cannot tolerate the SQ therapy, we will attempt to switch to IV therapy.
Other Names:
  • treprostinil, tyvaso

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Improved cardiopulmonary exercise test
Time Frame: 6 months
Change in 6 minute walk distance after 6 months of escalating Subcutaneous (SQ) remodulin as compared to baseline.
6 months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Improved overall cardiopulmonary variables
Time Frame: 1-6 months
  • Change in exercise duration (modified Bruce protocol)
  • Change in maximal oxygen consumption (VO2 max)
  • Change in minute ventilation- carbon dioxide production (VE/VCO2) ratio
  • Change in serum Brain Natriuretic Peptide (BNP) level
  • Change in quality of life as assessed by the Cambridge Pulmonary Hypertension Outcome Review (CAMPHOR) survey
  • Change in resting oxygen saturation
  • Tricuspid Annular Plane Systolic Excursion (TAPSE)
  • Heart rate recovery
1-6 months

Collaborators and Investigators

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

Sponsor

University of California, Los Angeles

Collaborators

United Therapeutics

Investigators

  • Principal Investigator: Jamil A Aboulhosn, MD, UCLA Health System

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

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

November 1, 2013

Primary Completion (Actual)

December 1, 2015

Study Completion (Actual)

December 1, 2015

Study Registration Dates

First Submitted

September 24, 2013

First Submitted That Met QC Criteria

November 4, 2013

First Posted (Estimate)

November 11, 2013

Study Record Updates

Last Update Posted (Actual)

September 21, 2017

Last Update Submitted That Met QC Criteria

September 19, 2017

Last Verified

September 1, 2017

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • Remodulin in ACHD
  • IRB#12-001968 (Other Identifier: IRB#12-001968)

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

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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