A randomized, double-blind, vehicle-controlled, multi-center, parallel-group study of APL-9 (second generation complement-3 inhibitor) in mild to moderate acute respiratory distress syndrome due to COVID-19.
Principal Investigator: Matthew R. Baldwin, MD, MS
This is a prospective, multicenter, randomized, controlled, pivotal trial conducted under an FDA approved IDE to validate the safety and efficacy of the Hemolung® Respiratory Assist System, an investigational Class III medical device, for COPD patients experiencing an acute exacerbation requiring ventilatory support.
Principal Investigator: Daniel Brodie, MD
Contact: Cara Agerstrand or Alexis Serra, 646-317-2269
This is an international, multicenter, prospective observational cohort study that aims to describe current effective blood product usage during VV-ECMO as well as the current anticoagulation strategies and bleeding episode incidence in a large cohort of ECMO patients.
Principal Investigator: Cara Agerstrand, MD
Trial Steering Committee Member: Daniel Brodie, MD
Contact: Cara Agerstrand or Alexis Serra, 646-317-2269
This is an international, multicenter, prospective observational cohort study that aims to describe bleeding, thromboembolic complications, blood management and anticoagulation strategies in ECMO for mechanical circulatory support (MCS). This study seeks to identify risk factors for bleeding and thromboembolic complications as well as investigate any associations with morbidity and mortality.
Principal Investigator: Koji Takeda, MD
Trial Steering Committee Member: Daniel Brodie, MD
Contact: Daniel Brodie or Alexis Serra, 646-317-2269
Principal Investigator: Jeanine D'Armiento, MD, PhD
This DoD funded study will investigate the tolerability of nilotinib in patients with LAM both on and off sirolimus therapy, and evaluate the effects of nilotinib on biomarkers of LAM disease activity.
Alpha-1 Antitrypsin Deficiency Clinical Trials
Alpha-1 Antitrypsin Disease Cohort: Longitudinal Biomarker Study of Disease
NIH/NHLBI UG3 HL152323-01
Principal Investigator: Jeanine D'Armiento, MD, PhD
The first phase of this grant will establish a multi-center research infrastructure that will allow the construction of a longitudinal cohort of patients across the disease spectrum of AATD. In the second phase, we will test the hypothesis that biomarkers in serum and sputum of a population of patients with alpha-1 antitrypsin deficiency will correlate with imaging biomarkers that can predict prognosis and outcome of disease.
Alvelestat (MPH966) for the Treatment of ALpha-1 ANTitrypsin Deficiency (ATALANTa)
NIH/NCATS UH3 TR002450-02
Principal Investigator: Dransfield at UAB
CUIMC Site Principal Investigator: Jeanine D'Armiento, MD, PhD
Alvelestat (MPH966, formerly AZD9668) is a potent, selective, and reversible, oral inhibitor of human NE, and suppression of NE is expected to reduce lung damage and may slow disease progression. This study is to establish proof of clinical concept by investigating the mechanistic effect and safety of alvelestat (MPH966) in patients with alpha-1 antitrypsin deficiency.
Alpha-1 Foundation Research Grant
Principal Investigator: Monica Goldklang, MD
“In vivo imaging of lung apoptosis in alpha-1 antitrypsin deficiency”
This project seeks to examine the role of apoptosis targeted lung imaging in the alpha-1 antitrypsin deficiency patient population. *Currently in no cost extension.
Alpha-1 Foundation Gordon L. Snider Scholar Award
Principal Investigator: Monica Goldklang, MD
Mentor: Jeanine D'Armiento, MD, PhD
“A Role for MMP-13 in COPD Exacerbations in the Alpha-1 Community”
This project seeks to understand the interaction between MMP-13 and alpha-1 antitrypsin in the lung destruction that occurs during COPD exacerbations. *Currently in no cost extension.
Arrowhead Pharmaceuticals
Site Principal Investigator Columbia University: Monica Goldklang, MD
“A Placebo-Controlled, Multi-dose, Phase 2/3 Study to Determine the Safety, Tolerability and Effect on Liver Histologic Parameters in Response to ARO-AAT in Patients With Alpha-1 Antitrypsin Deficiency (AATD) [SEQUOIA]”
This study seeks to evaluate the tolerability and efficacy of interfering RNA technology for the treatment of AATD related liver disease.
Vertex Pharmaceuticals
Site Principal Investigator Columbia University: Monica Goldklang, MD
“A Phase 2, Randomized, Double-blind, Placebo-controlled Study of the Efficacy and Safety of VX-864 in PiZZ Subjects”
This study will evaluate the efficacy of the small molecule corrector drug VX-814 on AAT serum levels.
COVID-19 Clinical Trials
GC 2006: Study to Evaluate the Safety and Efficacy of Liquid Alpha1-Proteinase Inhibitor (Human) in Hospitalized Participants With Coronavirus Disease (COVID-19)
Site Principal Investigator: Monica Goldklang, MD
Hospitalized COVID-19 patients who are not yet on mechanical ventilation or in the ICU will be randomized to receive alpha-1 antitrypsin or placebo control on days 1 and 8 of the trial. This is based on data suggesting that alpha-1 antitrypsin has anti-viral effects with regards to COVID-19 viral entry into the epithelium.
GC2007: Study to Evaluate the Safety and Efficacy of High Dose Intravenous Immune Globulin (IVIG) Plus Standard Medical Treatment (SMT) Versus SMT Alone in Participants in Intensive Care Unit (ICU) With Coronavirus Disease (COVID-19)
Site Principal Investigator: Monica Goldklang, MD
This trial enrolls new (< 48 hours) ICU patients requiring supplemental oxygen or on mechanical ventilation into a randomized trial comparing high dose IVIG to placebo for a 4-5 day treatment regimen.
TXA Trial
Principal Investigator: Jeanine D'Armiento, MD, PhD
COVID-19 reduces levels of ACE-2, the enzyme that converts angiotensin II to angiotensin 1-7. This trial utilizes TXA127, the pharmaceutical formulation of angiotensin 1-7 to determine if normalizing the renin-angiotensin-aldosterone axis reduces acute kidney injury and multi-organ system failure in COVID-19 infection.
Validation of new COVID-19 screening test
Principal Investigator: Jeanine D'Armiento, MD, PhD
This study will validate a new diagnostic method for detecting SARS-CoV-2 using an isothermal Loop-Mediated Amplification (LAMP) assay to develop a quick potential at-home PCR screening method for diagnosis of COVID-19 infection. It will compare the LAMP assay to standard qPCR techniques using the CDC primers with an established, FDA-EUA approved protocol. Newly diagnosed patients with COVID-19 will provide saliva samples and nasopharyngeal swabs for concurrent LAMP assay and qPCR testing.
