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Hypertension, PulmonaryPhase 4NCT01462565

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

A Phase 4 study testing current marketed FLOLAN (epoprostenol sodium) and new thermo stable formulation of epoprostenol sodium in people with Hypertension, Pulmonary, run by GlaxoSmithKline at 8 sites in 3 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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). Read more about this study

Who is it for?
Ages 18 to 75 years
Conditions the study is for
Hypertension, Pulmonary
What is being tested?
current marketed FLOLAN (epoprostenol sodium) and new thermo stable formulation of epoprostenol sodium
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
16 participants
Where
8 sitesin 3 countries
Study dates
1 Nov 2011to 8 Nov 2012
Phase
Phase 4
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 75 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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.

Who cannot join

  • 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.
Show all 12Show fewer
  • 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.

Trial locations and contacts

8 sites in 3 countries.

8 sites in 3 countries

United States6 sites

  • GSK Investigational Site

    Baltimore, Maryland, 21205

  • GSK Investigational Site

    Boston, Massachusetts, 02118-2393

  • GSK Investigational Site

    Chicago, Illinois, 60637

  • GSK Investigational Site

    Columbus, Ohio, 43221

  • GSK Investigational Site

    Dallas, Texas, 75390-8550

  • GSK Investigational Site

    Miami Beach, Florida, 33140

Other trials in United States

Canada1 site

  • GSK Investigational Site

    Québec, Quebec, G1V 4G5

Other trials in Canada

Netherlands1 site

  • GSK Investigational Site

    Amsterdam, 1081 HV

Other trials in Netherlands

About this study

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.)

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).

Read the 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.

Being tested
current marketed FLOLAN (epoprostenol sodium), new thermo stable formulation of epoprostenol sodium
Keywords
flolan, pulmonary arterial hypertension, thermo stable formulation, epoprostenol sodium, modified sterile diluent
Medical subject headings
Hypertension, Pulmonary, Pulmonary Arterial Hypertension

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • current marketed FLOLAN (epoprostenol sodium)Drug

    continuous intravenous infusion

  • new thermo stable formulation of epoprostenol sodiumDrug

    continuous intravenous infusion

Study groups in NCT01462565
Study groupRoleWhat they receive
All subjectsExperimentalSubjects 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).
Receives: Drug: current marketed FLOLAN (epoprostenol sodium); Drug: new thermo stable formulation of epoprostenol sodium

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. 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).

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

  2. 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).

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

  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).

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

Secondary outcomes (12)

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

    An AE was defined as any untoward medical occurrence that occurred during the course of the trial after study treatment had started. An adverse event was therefore any unfavourable and unintended sign, symptom, or disease temporally associated with the use of study drug, whether or not considered related to the study drug. An SAE is any untoward medical occurrence that at any dose results in death, are life threatening, requires hospitalization or prolongation of hospitalization or results in disability/incapacity, congenital anomaly/birth defect and medically significant and all events of possible drug-induced liver injury with hyperbilirubinaemia. Only treatment emergent AEs and SAEs were reported in this outcome measure. Specifically, this study reported 2 SAEs, but only 1 was categorized as treatment emergent.

    Measured: Up to visit 3 (Week 4)

  2. Number of Participants With Infusion Site Reactions During Treatment Period

    Infusion site reactions were reported during the treatment period. Infusion site was inspected for erythema, excoriation, induration, skin necrosis or signs of local sepsis.

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

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

    Systolic blood pressure is a measure of blood pressure while the heart is beating. Diastolic blood pressure is a measure of blood pressure while the heart is relaxed. Change from Baseline was calculated as the value at the indicated time points minus the value at Baseline. Baseline was Visit 2 i.e. Day-14 (+ or - 7 days).

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

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

    Summary mean change in heart rate measured in beats per minute (beats/min or BPM). Change from Baseline was calculated as the value at the indicated time points minus the value at Baseline. Baseline was Visit 2 i.e. Day-14 (+ or - 7 days).

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

  5. Number of Participants With Abnormal Clinical Chemistry

    Abnormal clinical chemistry was analyzed as follows: serum alanine aminotransferase (ALT/SGPT) >= 3 x upper limit of normal (ULN) , aspartate aminotransferase (AST/SGOT) >= 3 x ULN , total bilirubin >= 34.2, creatinine >= 176.8.

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

  6. Number of Participants With Abnormal Hematology

    Values for hemoglobin, hematocrit, and platelet count were analyzed. Participants with abnormal values have been reported. The low and high value concern were as follows: hemoglobin (Males < 98, >180.0) (females <91, >161.0)grams per litre (g/L); hematocrit (Males < 32.0, >54.0) (females <29.0, >50.6) fraction (1); platelet count (< 100, > 500) gram international units per litre (gI/L).

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

  7. Number of Participants With Abnormal Urinalysis

    Dipstick method was used to measure blood, glucose and protein. Data was analyzed up to 1 week after Week 4 (Follow-up visit).

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

  8. Change From Baseline in Six Minute Walk Distance Test (6MWD) After 4-weeks of Treatment

    This assessment was a non-encouraged test that measures the distance walked for a duration of 6 minutes. Change from Baseline was calculated as value at observation minus value at Baseline. Baseline visit was Visit 2 i.e. Day-14 (+ or - 7 days).

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

  9. Breathlessness After 6MWD - Borg Dyspnoea Index (BDI)

    The BDI was calculated by using a 10-point scale (0 = None, 10 = Maximum) and indicates the degree of breathlessness after completion of the 6-minute walk test. The BDI scale was assessed by each participant. Change from Baseline = score at observation minus score at Baseline. Baseline visit was Visit 2 i.e. Day-14 (+ or - 7 days).

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

  10. World Health Organization [WHO] Functional Class at Baseline and After 4- Weeks of Treatment

    World Health Organization functional class was analyzed as class I, class II, class III and class IV. World Health Organization functional class was analyzed at Baseline (Visit 2 i.e. Day-14 [+ or - 7 days]) and Week 4. The classed were defined as Class I: No symptoms of pulmonary arterial hypertension with exercise or at rest, Class II: No symptoms at rest but uncomfortable and short of breath with normal activity, Class III: May not have symptoms at rest but activities greatly limited by shortness of breath, fatigue, or near fainting and Class IV: Symptoms at rest and severe symptoms with any activity. Hence the severity increased from class I (better) to Class IV (worse).

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

  11. Mean Oxygen Saturation in Blood Over Time

    Pulse oximetry (oxygen saturation) was analyzed. Data for Pulse oximetry (oxygen saturation) was analyzed up to the treatment follow up (1 week after Visit 3 [Week 4]).

    Measured: up to the treatment follow up (1 week after Visit 3 [Week 4])

  12. Number of Participants With Urine Pregnancy Test Positive

    Urine samples were collected for urine pregnancy test. Urine samples were collected at up to the treatment follow up (1 week after Visit 3 [Week 4]). Number of participants with urine pregnancy test positive has been reported.

    Measured: up to the treatment follow up (1 week after Visit 3 [Week 4])

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted31 Oct 2011 (estimated)
  2. Study start1 Nov 2011
  3. Primary completion16 May 2012
  4. Study completion8 Nov 2012

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 4
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
16 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01462565
Sponsor's protocol ID
115332
Status last verified
May 2017
First submitted
13 Oct 2011
Last updated
16 Oct 2017
Data monitoring committee
No

Publications

  • 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.

    PubMed 25793960

Questions about this trial

What is this clinical trial studying?

A Phase 4 study testing current marketed FLOLAN (epoprostenol sodium) and new thermo stable formulation of epoprostenol sodium in people with Hypertension, Pulmonary, run by GlaxoSmithKline at 8 sites in 3 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 7 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 16 Oct 2017. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 8 sites in United States, Canada, Netherlands. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 16 Oct 2017). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Change From Baseline in Medical Outcomes Study Short Form 36 (SF-36) (time frame: Baseline (Visit 2 i.e. Day-14 [+ or - 7 days]) and Week 4 (Visit 3)). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01462565, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Oct 2017.

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