ABI-009, an mTOR Inhibitor, for Patients With Severe Pulmonary Arterial Hypertension
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mTOR activation has been shown to be relevant in the development and progression of pulmonary hypertension. Inhibition of mTOR has been shown to reverse or regress pulmonary hypertension in animal models. ABI-009 is an albumin-bound mTOR inhibitor with improved penetration in lung tissue.
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Layout table for eligibility information
Ages Eligible for Study:
18 Years and older (Adult, Older Adult)
Sexes Eligible for Study:
Accepts Healthy Volunteers:
Male or female age >18 year old with a current diagnosis of WHO Group 1 PAH including idiopathic pulmonary arterial hypertension (IPAH), heritable pulmonary arterial hypertension (HPAH), drug and toxin induced PAH, or PAH associated with connective tissue disease, or congenital heart defects (repaired greater than 1 year prior to Screening)
Must meet following hemodynamic definition prior to initiation of study drug
Mean PAP of ≥ 25 mm Hg
PCWP or left ventricular end diastolic pressure (LVEDP) of ≤ 12 mm
PVR >5 mmHg/L/min (Woods unit)
Functional class III or IV according to the WHO set forth at the Dana Point Classification 2008 Meeting
On 2 or more specific standard PAH therapies (for ≥ 12 consecutive weeks and at stable dose for ≥ 8 consecutive weeks) unless documented inability to tolerate 2 standard therapies
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
6MWD ≥150 meters and ≤450 meters
Negative serum pregnancy test
Female of childbearing age either surgically sterilized or using acceptable method of contraception
Ability to provide written informed consent by the patient or legal guardian
History of heart disease including left ventricular ejection fraction (LVEF) ≤ 40% or clinically significant valvular constrictive or atherosclerotic heart disease (myocardial infarction, angina, cerebrovascular accident)
History of malignancy in 2 years prior to enrollment
Pulmonary hypertension (PH) belonging to groups 2 to 5 of the 2013 Nice classification
Current or recent (< 3 months) use of inotropic or vasopressor agents for the treatment of PAH
Recent (< 3 months) PAH related hospital admission
History of allergic reactions attributed to compounds of similar chemical or biologic composition including macrolide (eg, azithromycin, clarithromycin, dirithromycin, and erythromycin) and ketolide antibiotics
Uncontrolled diabetes mellitus as defined by HbA1c >8% despite adequate therapy
Prior use of study drug within previous 6 months from enrollment
Previous lung transplant
Naïve to available standard PAH therapy
Concomitant genetic or acquired immunosuppressive diseases (such as HIV, AIDS)
Uncontrolled intercurrent illness that in the opinion of the investigator would limit compliance and tolerance to study requirements (eg, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, diabetes, uncontrolled hypertension, coronary artery disease, or psychiatric illness/social situations)
Concomitant enrollment in another investigational treatment protocol for PAH
Use of strong inhibitors and inducers of CYP3A4 within the 14 days prior to receiving the first dose of ABI-009. Additionally, use of any known CYP3A4 substrates with narrow therapeutic window (such as fentanyl, alfentanil, astemizole, cisapride, dihydroergotamine, pimozide, quinidine, terfanide) within the 14 days prior to receiving the first dose of ABI-009