ABI-009, an mTOR Inhibitor, for Patients With Severe Pulmonary Arterial Hypertension

Study Purpose

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.

Recruitment Criteria

Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms

Accepts Healthy Volunteers
No

An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes.


An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes.


Searching Both is inclusive of interventional and observational studies.

Study Type
Interventional
Eligible Ages 18 Years and Over
Gender All
More Inclusion & Exclusion Criteria

Inclusion Criteria:

  • - 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

    Exclusion criteria:

    - 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 - Uncontrolled hyperlipidemia (serum triglyceride ≥300 mg/dL) - Serum cholesterol ≥350 mg/dL - Surgery within 3 months of start date of study drug - Baseline cytopenias: - Absolute Neutrophil Count ≤ 1.5 x 109/L - Hemoglobin ≤ 9 g/dL - Platelet count < 100,000/mm3 - Baseline liver disease: ALT/AST, total bilirubin, alkaline phosphatase >1.5 x ULN - Baseline renal disease: creatinine >1.5 ULN and/or creatinine clearance (Cockroft formula) ≤ 60 mL/min - Inability to attend scheduled clinic visits - 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

Trial Details

This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.

Trial ID:
NCT02587325

Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans.

Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data.

Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs.

Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.

Phase
Phase 1

The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.

Lead Sponsor
Aadi, LLC

The person who is responsible for the scientific and technical direction of the entire clinical study.

Principal Investigator
N/A
Principal Investigator Affiliation N/A

Category of organization(s) involved as sponsor (and collaborator) supporting the trial.

Agency Class
Industry
Overall Status Recruiting
Countries United States

The disease, disorder, syndrome, illness, or injury that is being studied.

Conditions
Pulmonary Hypertension

Contact a Trial Team

If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.

Inova Fairfax Hospital, Falls Church, Virginia

Status

Recruiting

Address

Inova Fairfax Hospital

Falls Church, Virginia, 22042

Site Contact

Vinita Gupta

vinita.gupta@inova.org

703-776-3697

University of Pittsburgh Medical Center, Pittsburgh, Pennsylvania

Status

Recruiting

Address

University of Pittsburgh Medical Center

Pittsburgh, Pennsylvania, 15213

Site Contact

Mary Kunkel

kunkelme@upmc.edu

412-647-4463

National Institutes of Health, Bethesda, Maryland

Status

Recruiting

Address

National Institutes of Health

Bethesda, Maryland, 20892

Site Contact

Keshia Thompson

keshia.thompson@nih.gov

301-827-6135

Indiana University, Indianapolis, Indiana

Status

Recruiting

Address

Indiana University

Indianapolis, Indiana, 46202

Site Contact

Annie Thorpe

Athorp2@iuhealth.org

317-962-9904

Harbor-UCLA Medical Center, Torrance, California

Status

Recruiting

Address

Harbor-UCLA Medical Center

Torrance, California, 90502

Site Contact

Ronald Oudiz, MD

roudiz@labiomed.org

310-222-3560

University of Arizona, Tucson, Arizona

Status

Recruiting

Address

University of Arizona

Tucson, Arizona, 85724

Site Contact

Valerie Bloss

vbloss@email.arizona.edu

520-626-8305

For more information, please contact PHA at Research@PHAssociation.org and refer to the terms of service below.

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