Study of a New Thermo Stable Formulation of Epoprostenol Sodium to Treat Pulmonary Arterial Hypertension (PAH)

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
GlaxoSmithKline
ClinicalTrials.gov Identifier:
NCT01462565
First received: October 13, 2011
Last updated: May 15, 2014
Last verified: October 2013
  Purpose

The purpose of this multicentre, open label, single-arm study in approximately 20 adult patients is to evaluate the Impact on lifestyle of a new thermo stable formulation of epoprostenol sodium in subjects with Pulmonary Arterial Hypertension (PAH).


Condition Intervention Phase
Hypertension, Pulmonary
Drug: current marketed FLOLAN (epoprostenol sodium)
Drug: new thermo stable formulation of epoprostenol sodium
Phase 4

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Single-arm, Open Label Study Evaluating the Impact on Lifestyle of a New Thermo Stable Formulation of FLOLAN® in Subjects With Pulmonary Arterial Hypertension (PAH). (FLOLAN® is a Registered Trademark of the GlaxoSmithKline Group of Companies.)

Resource links provided by NLM:


Further study details as provided by GlaxoSmithKline:

Primary Outcome Measures:
  • Quality of life assessment [ Time Frame: week 4 ] [ Designated as safety issue: No ]
    Quality of life assessment using SF-36 questionnaire

  • Time required to prepare medication and effect of treatment on daily activities [ Time Frame: week 4 ] [ Designated as safety issue: No ]
    study specific questionnaire to assess ease of administration and changes in quality of life particularly activities of daily living assessment

  • change from baseline in dose of thermo stable epoprostenol sodium [ Time Frame: a minimum of 6 hours upto 48 hours after start of thermo stable epoprostenol sodium ] [ Designated as safety issue: No ]
    Change from baseline in the dose of thermo stable epoprostenol sodium formulation at the time of discharge at a minimum of 6 hours or per local guidelines/practices


Secondary Outcome Measures:
  • safety and tolerability (number of subjects with adverse events, infusion site reactions and haematology and clinical chemistry parameters outside the normal range, change from baseline in vital signs). [ Time Frame: up to week 4 ] [ Designated as safety issue: Yes ]
  • efficacy (change from baseline in 6-minute walk distance, breathlessness after the 6MWD - Borg Dyspnoea Index and WHO functional class). [ Time Frame: at week 4 ] [ Designated as safety issue: No ]

Enrollment: 16
Study Start Date: November 2011
Study Completion Date: November 2012
Primary Completion Date: May 2012 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: All subjects
Subjects enter the study already taking current marketed FLOLAN (epoprostenol sodium) and continue for 4 weeks (run-in). At baseline, they will be swopped to the new thermo stable formulation of epoprostenol sodium for 4 weeks (or longer if they continue in the extension phase of the study).
Drug: current marketed FLOLAN (epoprostenol sodium)
continuous intravenous infusion
Drug: new thermo stable formulation of epoprostenol sodium
continuous intravenous infusion

Detailed Description:

This is a multicentre, open label, single-arm study in approximately 20 adult patients (18 - 75 years old) designed to evaluate the Impact on lifestyle of a new thermo stable formulation of epoprostenol sodium in subjects with Pulmonary Arterial Hypertension (PAH). The co-primary objectives are 1) to describe the effect of the new thermo stable formulation of epoprostenol sodium on quality of life and 2) to determine the dose titration requirement in patients switching from the currently marketed FLOLAN (epoprostenol sodium) to the new thermo stable formulation. Secondary objectives include assessing the safety, tolerability and efficacy of the thermo stable formulation of epoprostenol sodium and the exploratory objective is to evaluate the effect of the new thermo stable formulation of epoprostenol sodium on haemodynamic parameters in a subset of subjects.

Subjects who are already receiving FLOLAN (epoprostenol sodium) for the treatment of PAH and have been on a stable dose for at least 3 months and on stable doses of other PAH treatments for at least 30 days prior to screening will be enrolled. After a screening visit, eligible subjects will have a 4-week run-in period with their existing FLOLAN (epoprostenol sodium) treatment. At the end of the 4-week period, they will be admitted to the clinic for baseline assessments and for switching to study medication (the new thermo stable formulation of epoprostenol sodium). Subjects will remain in hospital for a minimum of 6 hours to ensure clinical and hemodynamic stability prior to discharge. Subjects may stay in hospital for up to 24-48 hours after switching to the new thermo stable formulation of epoprostenol sodium at the discretion of the investigator. Dose titration requirement will be assessed at the time of discharge. Haemodynamic parameters will be obtained in a subgroup of subjects enrolled in centres where the collection of haemodynamic data is considered part of the standard of care. Subjects will receive the study medication as a continuous intravenous infusion for a 4-week treatment period. Those who complete the 4-week treatment period will have the option of entering an extension phase of the study to continue receiving the new formulation.

  Eligibility

Ages Eligible for Study:   18 Years to 75 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Adult male or female at least 18 to 75 years at the time of screening.
  • Subjects must have been on FLOLAN (epoprostenol sodium) therapy for pulmonary arterial hypertension (PAH) as approved in the product label.
  • Subjects must be on stable doses of their existing FLOLAN (epoprostenol sodium) treatment for a minimum of 3 months prior to screening.
  • Subjects must be on stable doses of any current PAH treatments other than FLOLAN (epoprostenol sodium) in the last 30 days.
  • Subjects must walk a distance of at least 150 meters during six-minute walk distance test (6MWD). This test must be completed during the Screening Visit.
  • A female subject is eligible to participate if she is of non-childbearing potential or of childbearing potential, has a negative pregnancy test at screen, and agrees to use one of the contraception methods listed in the protocol.
  • Subjects must be competent to understand the information given in the Institutional Review Board (IRB) or Independent Ethics Committee (IEC) approved informed consent form and must sign the form prior to the initiation of any study procedures.

Exclusion Criteria:

  • Subjects who are given FLOLAN (epoprostenol sodium) for a condition or in a manner that is outside the approved indication.
  • Subjects with congestive heart failure arising from severe left ventricular dysfunction.
  • Subjects, with or without supplemental oxygen, who have a resting arterial oxygen saturation (SaO2) <90% as measured by pulse oximetry at screening.
  • Subjects have been hospitalized as an emergency or visited the emergency room for a condition related to PAH or treatment for PAH in the last 3 months.
  • The subject's clinical condition is such that they are not expected to remain clinically stable for the duration of the study.
  • Female subjects who are pregnant or breastfeeding.
  • Subjects who have demonstrated noncompliance with previous medical regimens.
  • Subjects who have a history of abusing alcohol or illicit drugs within 1 year.
  • Subjects with a diagnosis of active hepatitis (hepatitis B surface antibody and hepatitis C antibody).
  • Subjects who have participated in a clinical study involving another investigational drug or device within four weeks before screening.
  • Subjects who had history malignancies within the past 5 years, with the exception of basal cell carcinoma of the skin or in situ carcinoma of the cervix.
  • Any concurrent condition that would affect the safety of the subject or in the opinion of the investigator it is not in the best interest of the patient to participate in the study.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01462565

Locations
United States, Florida
GSK Investigational Site
Miami Beach, Florida, United States, 33140
United States, Illinois
GSK Investigational Site
Chicago, Illinois, United States, 60637
United States, Maryland
GSK Investigational Site
Baltimore, Maryland, United States, 21205
United States, Massachusetts
GSK Investigational Site
Boston, Massachusetts, United States, 02118-2393
United States, Ohio
GSK Investigational Site
Columbus, Ohio, United States, 43221
United States, Texas
GSK Investigational Site
Dallas, Texas, United States, 75390-8550
Canada
GSK Investigational Site
Quebec, Canada, G1V 4G5
Netherlands
GSK Investigational Site
Amsterdam, Netherlands, 1081 HV
Sponsors and Collaborators
GlaxoSmithKline
Investigators
Study Director: GSK Clinical Trials GlaxoSmithKline
  More Information

No publications provided

Responsible Party: GlaxoSmithKline
ClinicalTrials.gov Identifier: NCT01462565     History of Changes
Other Study ID Numbers: 115332
Study First Received: October 13, 2011
Last Updated: May 15, 2014
Health Authority: United States: Food and Drug Administration
Canada: Health Canada
Netherlands: Medische Ethische Toestings Commissie (medical ethical review committee) Vrije Universiteit, Amsterdam

Keywords provided by GlaxoSmithKline:
flolan
pulmonary arterial hypertension
thermo stable formulation
epoprostenol sodium
modified sterile diluent

Additional relevant MeSH terms:
Hypertension
Hypertension, Pulmonary
Vascular Diseases
Cardiovascular Diseases
Lung Diseases
Respiratory Tract Diseases
Tezosentan
Epoprostenol
Vasodilator Agents
Cardiovascular Agents
Therapeutic Uses
Pharmacologic Actions
Antihypertensive Agents
Platelet Aggregation Inhibitors
Hematologic Agents

ClinicalTrials.gov processed this record on September 30, 2014