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Epoprostenol for Injection in Pulmonary Arterial Hypertension

Phase 4
Completed
Conditions
Pulmonary Arterial Hypertension
Interventions
Drug: ACT-385781A (Actelion Epoprostenol)
Registration Number
NCT01105091
Lead Sponsor
Actelion
Brief Summary

This is a prospective, multi-center, open-label, randomized, Phase IV exploratory study comparing safety, tolerability, pharmacokinetics, and effectiveness of ACT-385781A and Flolan (epoprostenol sodium) in patients with pulmonary arterial hypertension who are naïve to injectable prostanoid treatment and in need of such treatment. Approximately 30 patients from 8 U.S. clinical sites will be randomized to receive either ACT-385781A or Flolan (2:1 respectively) for 28 days of treatment.

Detailed Description

Not available

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
30
Inclusion Criteria
  1. Male or female subjects aged 18-65 years

  2. Patients with the following types of pulmonary arterial hypertension (PAH) belonging to WHO Group I:

    • Idiopathic (IPAH)

    • Heritable (HPAH)

    • Associated (APAH) with

      • Connective tissue diseases
      • Drugs and toxins
  3. Patients with PAH in modified NYHA functional class III or IV at the time of enrollment in need of injectable epoprostenol.

  4. Patients must be injectable prostanoid treatment-naïve and either

    • newly diagnosed and not yet treated with specific PAH therapies or

    • currently treated with existing background PAH therapy with one or more of the following medications for 90 days prior to enrollment and on a stable dose for 30 days prior to enrollment:

      • Bosentan
      • Ambrisentan
      • Sildenafil
      • Tadalafil
  5. Women of childbearing potential must use a reliable method of contraception.

Exclusion Criteria
  1. Patients with respiratory and/or cardiovascular distress in need of emergency care including i.v. epoprostenol administration or any vasopressive i.v. drugs

  2. Known pulmonary veno-occlusive disease (PVOD)

  3. Current use of i.v. inotropic agents

  4. Tachycardia with heart rate > 120 beats/min

  5. Pulmonary arterial hypertension related to any condition other than those specified in the inclusion criteria

  6. Known hypersensitivity to the formulations of ACT-385781A or any of its excipients, and Flolan or any of its excipients

  7. Use of inhaled iloprost or treprostinil during the week prior to screening

  8. Cerebrovascular events (e.g., transient ischemic attack or stroke) within 6 months of screening

  9. History of myocardial infarction

  10. History of left-sided heart disease, including any of the following:

    • hemodynamically significant aortic or mitral valve disease
    • restrictive or congestive cardiomyopathy
    • left ventricular ejection fraction < 40% by multigated radionucleotide angiogram(MUGA),angiography, or echocardiography
    • unstable angina pectoris
    • life-threatening cardiac arrhythmias
  11. Chronic bleeding disorder

  12. Infection(s) within the past month that in the mind of the investigator would contraindicate the use of epoprostenol

  13. Pregnancy or breast-feeding

  14. Participation in another clinical trial, except observational (noninterventional), or receipt of an investigational product within 30 days prior to randomization

  15. Any known factor or disease that might interfere with treatment compliance, study conduct or interpretation of the results such as drug or alcohol dependence or psychiatric disease

  16. Known concomitant life-threatening disease other than PAH with a life expectancy < 12 months

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
1ACT-385781A (Actelion Epoprostenol)ACT-385781A (Actelion Epoprostenol)
2Flolan®Flolan®
Primary Outcome Measures
NameTimeMethod
Dose Normalized Pharmacokinetics of 6,15-diketo-13,14-dihydro-Prostacyclin F1alpha at 2 ng/kg/MinDay 1

The plasma concentration for the epoprostenol metabolite 6,15-diketo-13,14-dihydro-Prostacyclin F1alpha was measured at 2 ng/kg/min just prior to the next up-titration. Dose-normalized concentrations are used to summarize the results.

Dose Normalized Pharmacokinetics of 6,15-diketo-13,14-dihydro-Prostacyclin F1alpha at 4 ng/kg/MinDay 1

The plasma concentration for the epoprostenol metabolite 6,15-diketo-13,14-dihydro-Prostacyclin F1alpha was measured at 4 ng/kg/min just prior to the next up-titration. Dose-normalized concentrations are used to summarize the results.

Dose Normalized Pharmacokinetics of 6-keto-Prostacyclin F1alpha at 2 ng/kg/MinDay 1

The plasma concentration for the epoprostenol metabolite 6-keto-Prostacyclin F1alpha was measured at 2 ng/kg/min just prior to the next up-titration. Dose-normalized concentrations are used to summarize the results.

Dose Normalized Pharmacokinetics of 6-keto-Prostacyclin F1alpha at 4 ng/kg/MinDay 1

The plasma concentration for the epoprostenol metabolite 6-keto-Prostacyclin F1alpha was measured at 4 ng/kg/min just prior to the next up-titration. Dose-normalized concentrations are used to summarize the results.

Six-minute Walk Distance (6MWD) - Baseline and Day 28Baseline and 28 days (+3 days)

The 6-minute walk test (6MWT) was to be performed prior to initiating study treatment either during the screening visit or on Day 1 prior to drug initiation, and Day 28 (End of treatment (EOT)). This assessment is a non-encouraged test that measures the distance walked for a duration of 6 minutes. The 6MWD was recorded in the Case Report Form (CRF).

Patients With New York Heart Association (NYHA) Functional Class Change (Improved or Worsened) From Baseline to Day 28From baseline to 28 days (+3 days)

Disease severity was assessed by NYHA classification of PAH criteria: Class I: no limitation of physical activity (PA). Ordinary PA: no undue dyspnea/fatigue, chest pain, near syncope. Class II: slight limitation of PA. Comfortable at rest. Ordinary PA: undue dyspnea/fatigue, chest pain, near syncope. Class III: marked limitation of PA. Comfortable at rest. Less than ordinary PA: undue dyspnea/fatigue, chest pain, near syncope. Class IV: inability to carry out PA without symptoms. Right heart failure. Dyspnea/fatigue may even have been present at rest. Discomfort increased by any PA.

Percentage Central Venous Blood Oxygen Saturation (ScVO2) - Baseline and Day 28Baseline and 28 days

Central venous blood oxygen saturation assessment was performed only in specific centers. Measurements for ScVO2 were performed during the inpatient hospitalization period on Day 1 (prior to drug initiation) and on Day 28 (EOT). Samples for ScVO2 were obtained by aspirating blood from the indwelling central venous catheter. After the sample had been drawn, the catheter was primed with study drug in order to refill the lumen to avoid interruption in treatment and sudden decompensation.

Blood Pressure - Baseline and Day 28Baseline and 28 days

Blood pressure (systolic and diastolic) were measured indirectly using an automatic oscillometric device, on the same arm for each measurement. The Blood Pressure was assessed at baseline and at Day 28 (End of Study Treatment visit).

Heart Rate - Baseline and Day 28Baseline and 28 days

Heart rate was measured indirectly using an automatic oscillometric device, on the same arm for each measurement. The Heart Rate was assessed at Baseline and at Day 28 (End of Study Treatment visit).

Body Weight - Baseline and Day 28Baseline and 28 days

Body weight was measured both at baseline and day 28.

Secondary Outcome Measures
NameTimeMethod

Trial Locations

Locations (7)

Vanderbilt Medical Center

🇺🇸

Nashville, Tennessee, United States

Baylor College of Medicine

🇺🇸

Houston, Texas, United States

University of Pennsylvania

🇺🇸

Philadelphia, Pennsylvania, United States

Duke University Medical Center

🇺🇸

Durham, North Carolina, United States

University of California - San Diego

🇺🇸

La Jolla, California, United States

University of Colorado - Denver

🇺🇸

Aurora, Colorado, United States

University of Texas Southwestern Medical Center

🇺🇸

Dallas, Texas, United States

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