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

NCT01462565 | Completed Phase 4 Results

• 1 other identifier: 115332
• Study Type: interventional
• Target: 16
• Locations: 3 countries
• Sites: 8

Brief Summary

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).

Conditions

Hypertension, Pulmonary

Keywords

flolan; pulmonary arterial hypertension; thermo stable formulation; epoprostenol sodium; modified sterile diluent

Outcome Measures

Primary Outcomes (3)

• Change From Baseline in Medical Outcomes Study Short Form 36 (SF-36)

  Subject-rated measure of health status comprised of 36 items: 8 subscale scores (physical functioning, role limitations due to physical health problems, bodily pain, general health, vitality, social functioning, role limitations due to emotional problems, and mental health), 2 summary scores (physical component and mental component), and a self-evaluated change in health status. Subscale and summary scores range: 0-100. Higher subscale and summary scores was considered as better health status. Change from Baseline was calculated as score at observation minus score at baseline. Baseline was defined as Visit 2 i.e. Day-14 (+ or - 7 days).

  Baseline (Visit 2 i.e. Day-14 [+ or - 7 days]) and Week 4 (Visit 3)

• Change From Baseline in Study Specific Participant Acceptance Survey

  Study-specific questionnaire comprised the pre-defined15 questions which included activities of daily living assessment. Participants rated the question on a scale of 1 to 10, where 1 was do not agree and 10 was strongly agree. Change from Baseline was calculated as score at observation minus score at Baseline. Changes from Baseline was assessed for Questions 2 to 12. Baseline was defined as Visit 2 i.e. Day-14 (+ or - 7 days).

  Baseline (Visit 2 i.e. Day-14 [+ or - 7 days]) and Week 4 (Visit 3).

• Change From Baseline in Dose of Thermo Stable Epoprostenol Sodium at Week 4

  Dose titration requirement was assessed at the time of discharge. Change from Baseline was calculated as score at observation minus score at Baseline. Units- nanogram per kilogram per minute (ng/kg/min). Baseline was Visit 2 i.e . Day-14 (+ or - 7 days).

  Baseline (Visit 2 i.e. Day-14 [+ or - 7 days]) and Week 4

Secondary Outcomes (12)

• Number of Participants With Any Treatment Emergent Adverse Events (AEs) and Treatment Emergent Serious Adverse Events(SAEs)

  Up to visit 3 (Week 4)

• Number of Participants With Infusion Site Reactions During Treatment Period

  Baseline visit (Visit 2) to Week 4 (Visit 3)

• Change From Baseline in Vital Signs at Week 4 : Systolic and Diastolic Blood Pressure

  Baseline (Visit 2 i.e. Day-14 [+ or - 7 days]) and Week 4(Visit 3)

• Change From Baseline in Vital Signs at Week 4: Heart Rate

  Baseline (Visit 2 i.e. Day-14 [+ or - 7 days]) to Week 4

• Number of Participants With Abnormal Clinical Chemistry

  Up to 1 week after Week 4 (Follow-up)

Study Arms (1)

All subjects

EXPERIMENTAL

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); Drug: new thermo stable formulation of epoprostenol sodium

Interventions

current marketed FLOLAN (epoprostenol sodium) DRUG

continuous intravenous infusion

All subjects

new thermo stable formulation of epoprostenol sodium DRUG

continuous intravenous infusion

All subjects

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• GlaxoSmithKline: lead

Study Sites (8)

GSK Investigational Site

Miami Beach, Florida, 33140, United States

Location

GSK Investigational Site

Chicago, Illinois, 60637, United States

Location

GSK Investigational Site

Baltimore, Maryland, 21205, United States

Location

GSK Investigational Site

Boston, Massachusetts, 02118-2393, United States

Location

GSK Investigational Site

Columbus, Ohio, 43221, United States

Location

GSK Investigational Site

Dallas, Texas, 75390-8550, United States

Location

GSK Investigational Site

Québec, Quebec, G1V 4G5, Canada

Location

GSK Investigational Site

Amsterdam, 1081 HV, Netherlands

Location

Related Publications (1)

• Provencher S, Paruchuru P, Spezzi A, Waterhouse B, Gomberg-Maitland M; pH12 Flolan reformulation study group. Quality of life, safety and efficacy profile of thermostable flolan in pulmonary arterial hypertension. PLoS One. 2015 Mar 20;10(3):e0120657. doi: 10.1371/journal.pone.0120657. eCollection 2015.

  PMID: 25793960 DERIVED

MeSH Terms

Conditions

Hypertension, Pulmonary; Pulmonary Arterial Hypertension

Interventions

Epoprostenol

Condition Hierarchy (Ancestors)

Lung Diseases; Respiratory Tract Diseases; Hypertension; Vascular Diseases; Cardiovascular Diseases

Intervention Hierarchy (Ancestors)

Prostaglandins I; Prostaglandins; Eicosanoids; Fatty Acids, Unsaturated; Fatty Acids; Lipids; Autacoids; Inflammation Mediators; Biological Factors

Results Point of Contact

• Title: GSK Response Center
• Organization: GlaxoSmithKline

866-435-7343

Study Officials

• GSK Clinical Trials

  GlaxoSmithKline

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 13, 2011
• First Posted: October 31, 2011
• Study Start: November 1, 2011
• Primary Completion: May 16, 2012
• Study Completion: November 8, 2012
• Last Updated: October 16, 2017
• Results First Posted: October 16, 2017

Record last verified: 2017-05

Locations

NL (1); CA (1); US (6)