GS-4997 in Adults With Pulmonary Arterial Hypertension

Overview

About this study

This study will compare the efficacy, safety, and tolerability of three doses of GS-4997 to placebo in adults with pulmonary arterial hypertension (PAH). The study will consist of a 24-week placebo-controlled treatment period and a long-term GS-4997 treatment period. Participants completing the 24-week placebo-controlled period will be eligible to receive active treatment with GS-4997 in the long-term treatment period.

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. There is no guarantee that every individual who qualifies and wants to participate in a trial will be enrolled. Contact the study team to discuss study eligibility and potential participation.

Inclusion Criteria:

  • Diagnosis of idiopathic pulmonary arterial hypertension (IPAH), heritable pulmonary arterial hypertension (HPAH), drug- and toxin-induced PAH, or PAH associated with connective tissue disease, HIV infection, or congenital heart defects (repaired greater than 1 year prior to Screening)

  • Meet all of the following hemodynamic criteria by means of a screening right heart catheterization (RHC) completed prior to randomization:

    • Mean pulmonary artery pressure (mPAP) of ≥ 25 mm Hg
    • Pulmonary vascular resistance (PVR) ≥ 400 dyne•sec/cm^5
    • Pulmonary capillary wedge pressure (PCWP) or left ventricular end diastolic pressure (LVEDP) of ≤ 12 mm Hg if PVR ≥ 400 and < 500 dynes•sec/cm^5, or PCWP/LVEDP ≤ 15 mm Hg if PVR ≥ 500 dynes•sec/cm^5
  • Be able to walk a distance of at least 100 m
  • Have World Health Organization (WHO) Functional Class II or III symptoms

  • Meet the following criteria determined by pulmonary function tests completed no more than 24 weeks prior to screening, performed with or without bronchodilation:

    • Forced expiratory volume in one second (FEV1) ≥ 55% of predicted normal
    • FEV1:forced vital capacity (FVC) ratio ≥ 0.60
  • Receiving treatment with one or more drugs approved for PAH for ≥ 12 consecutive weeks and at stable dose for ≥ 8 consecutive weeks

Exclusion Criteria:

  • Diagnosis of PAH associated with significant venous or capillary involvement (PCWP > 15 mm Hg), pulmonary capillary hemangiomatosis, portal hypertension, or unrepaired congenital heart defects
  • Pulmonary hypertension (PH) belonging to groups 2 to 5 of the 2013 NICE classification
  • Left ventricular ejection fraction (LVEF) ≤ 40% or clinically significant ischemic, valvular or constrictive heart disease
  • Uncontrolled hypertension (≥ 180/110 mm Hg) at Screening
  • End stage renal disease (receiving peritoneal dialysis, hemodialysis, or status after renal transplantation)
  • Severe liver disease (Child-Pugh Class C, with or without cirrhosis)

Individuals may be rescreened one additional time with prior notification to and approval by the sponsor.

Participating Mayo Clinic locations

Study statuses change often. Please contact the study team for the most up-to-date information regarding possible participation.

Mayo Clinic Location Status

Rochester, Minn.

Mayo Clinic principal investigator

Robert Frantz, M.D.

Closed for enrollment

More information

Publications

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Additional contact information

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