Compassionate Use of Beraprost Sodium 314d Modified Release for Patients With Pulmonary Arterial Hypertension (PAH)

Compassionate Use of Beraprost Sodium 314d Modified Release (BPS-314d-MR) for Three Patients With Pulmonary Arterial Hypertension (PAH).

The purpose of this study is to see if Lung LLC's new experimental formulation of the medicine Beraprost Sodium, called Beraprost Sodium 314d Modified Release (BPS-314d-MR), can improve the symptoms of pulmonary arterial hypertension (PAH) in patients. An experimental drug is one that has not been approved by the U.S. Food and Drug Administration for use in the general public. This research study is for patients who have pulmonary arterial hypertension (PAH) and have just completed taking part in an earlier research study and received an older experimental formulation of Beraprost Sodium, called Beraprost Sodium Modified Release (BPS-MR). That earlier study was being done to see if BPS-MR could improve their PAH.

Patients may also be taking Tyvaso (treprostinil), Tracleer (bosentan), Letairis (ambrisentan), Adcirca (tadalafil) and/or Viagra or Revatio (sildenafil) to treat their PAH. The diagnosis of PAH means that the blood pressure in their lungs is higher than normal. The increased blood pressure in the lungs places a strain on the heart. The strain causes the heart to pump less blood into the lungs, causing shortness of breath and tiredness. The strain on the heart weakens the heart muscle making it less able to pump blood, a condition called heart failure. As heart failure develops, swelling in the feet and abdomen may occur.

Study Overview

• Status: Completed
• Conditions: Pulmonary Arterial Hypertension
• Intervention / Treatment: Drug: BPS-314d-MR

Detailed Description

According to Lung LLC, and from my review of summary results contained within the most recent version of the BPS-314d-MR Investigators' Brochure I concur, BPS-314d- MR has been shown to have a similar safety and pharmacokinetic profile in healthy volunteers to BPS-MR. It is also my understanding that at an End of Phase II meeting in April of this year, FDA acknowledged Lung LLC's intention to continue the development of BPS-314d-MR by conducting a Phase III pivotal study in PAH patients. This study, BPS-314d-MR-PAH-302 was subsequently initiated this past June.

In my opinion, and based upon the similarities of the two formulations, it is in the best interest of our patients to transition their treatment to BPS-314d-MR.

Lung LLC has agreed to supply BPS-314d-MR for the patients.

• Study Type: Interventional
• Enrollment (Actual): 1
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • California
    • Torrance, California, United States, 90502
      • Los Angeles Biomedical Research Institute

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 75 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Prior participation in Beraprost-MR study at Harbor-UCLA

Exclusion Criteria:

• No prior participation in Beraprost study at Harbor-UCLA

Study Plan

How is the study designed?

Design Details

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

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Beraprost open label; Compassionate use access to open label BPS-314d-MR	Drug: BPS-314d-MR; 15-150 mcg po BID,; Other Names: Beraprost 314-d modified release

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of participants experiencing Adverse Events	To determine the overall safety of open label study drug by tabulating the number of patients experiencing Adverse Events	2 years

Collaborators and Investigators

• Sponsor: Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
• Collaborators: Lung Biotechnology PBC
• Investigators: Principal Investigator: Ronald J Oudiz, MD, Los Angeles BRI

Study record dates

Study Major Dates

• Study Start (Actual): November 1, 2013
• Primary Completion (Actual): June 2, 2019
• Study Completion (Actual): June 2, 2019

Study Registration Dates

• First Submitted: October 15, 2013
• First Submitted That Met QC Criteria: October 16, 2013
• First Posted (Estimate): October 21, 2013

Study Record Updates

• Last Update Posted (Actual): October 20, 2020
• Last Update Submitted That Met QC Criteria: October 16, 2020
• Last Verified: October 1, 2020

More Information

Terms related to this study

• Keywords: oral; Pulmonary Arterial Hypertension; prostacyclin
• Additional Relevant MeSH Terms: Cardiovascular Diseases; Vascular Diseases; Respiratory Tract Diseases; Lung Diseases; Hypertension, Pulmonary; Hypertension; Pulmonary Arterial Hypertension; Familial Primary Pulmonary Hypertension; Vasodilator Agents; Platelet Aggregation Inhibitors; Beraprost
• Other Study ID Numbers: Compassionate use BPS-314d-MR