A Phase 2, Multi-Center, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Safety, Phage Kinetics, and Efficacy of Inhaled AP-PA02 Multi-Phage Therapeutic in Subjects with Non-Cystic Fibrosis Bronchiectasis and Chronic Pulmonary Pseudomonas aeruginosa Infection

Date Added
June 14th, 2023
PRO Number
Pro00128304
Researcher
Patrick Flume

List of Studies


Keywords
Bronchiectasis
Summary

This is a Phase 2, double-blind, randomized, placebo-controlled study to evaluate the safety, tolerability, and efficacy of AP-PA02 administered by inhalation. This study will evaluate AP-PA02 administration in stable NCFB (non-cystic fibrosis bronchiectasis) patients. Subjects will either be included in Cohort A or Cohort B. For Cohort A, subjects will be randomized to receive either inhaled AP-PA02 or placebo. Cohort A will include individuals with NCFB and confirmed chronic P. aeruginosa infection but not on chronic inhaled antibiotics. These individuals will receive wither AP-PA02 or placebo for 10 days twice a day.
Cohort B will include individuals who with NCFB and confirmed P. aeruginosa infection but who are on chronic antibiotics. These individuals will receive either AP-PA02 or placebo for 10 days plus their current inhaled antibiotics for 28 days.

Institution
MUSC
Recruitment Contact
Zerlinna Teague
8437920965
recruitment@musc.edu

A randomized, participant- and investigator-blinded, placebo-controlled study to investigate efficacy, safety and tolerability of LTP001 in participants with pulmonary arterial hypertension

Date Added
July 6th, 2023
PRO Number
Pro00128867
Researcher
Rahul Argula

List of Studies


Keywords
Pulmonary Hypertension
Summary

This study is exploring the safety and efficacy of the study drug LTP001 in subjects with pulmonary arterial hypertension. This is a randomized study and the subjects will be be given the study drug or placebo in a capsule form which they are to take once daily in the morning. Participants will be screened for up to 8 weeks followed by 24 weeks of daily dosing- study visits to the site will occur approximately every 4 weeks. During these study visits there will be study procedures that may include labs for blood and urine, ECGs, vital signs, 6MWD, echocardiogram, or right heart catherization. There is the opportunity for an open-label portion of the study as well.

Institution
MUSC
Recruitment Contact
Zerlinna Teague
8437920965
recruitment@musc.edu

A Non-Interventional Study Evaluating the Impact of TRIKAFTA® Initiation on Children and Caregivers of Children With Cystic Fibrosis Aged 2-5 Years in a Real-World Setting

Date Added
July 17th, 2023
PRO Number
Pro00128569
Researcher
Sylvia Szentpetery

List of Studies


Keywords
Cystic Fibrosis
Summary

This is a intervention longitudinal study for children with CF and caregivers. Children aged 2-5 who are diagnosed with CF and are eligible for TRIKAFTA will be invited to participate in this study. The treating physicians will manage children with CF in accordance to their medical judgement and data will be collected throughout the study. Children with CF and caregivers may be enrolled up to 3 months before their child initiates TRIKAFTA and site personnel will record date for the study in that time (such as pulmonary exacerbations, medical history, and medication history.) Caregivers will also report PROS (patient reported outcomes) and CGRO (caregiver-reported outcomes).

Institution
MUSC
Recruitment Contact
Zerlinna Teague
8437920965
recruitment@musc.edu

A Phase 2, Randomized, Double-blind, Placebo-controlled, 24-Week Study to Evaluate the Efficacy, Safety, and Tolerability of BBT-877, as mono- or add-on therapy, in Patients with Idiopathic Pulmonary Fibrosis (IPF)

Date Added
July 25th, 2023
PRO Number
Pro00128976
Researcher
Rachana Krishna

List of Studies


Keywords
Interstitial Lung Disease (ILD)
Summary

This is a Phase 2, multicenter, randomized, double-blind, placebo-controlled, study to evaluate the efficacy, safety, and tolerability of 200 mg twice daily (BID) of BBT-877 in patients with IPF, with or without AF approved background therapies (pirfenidone or nintedanib). For both the placebo and BBT-877 groups, patients will be selected so that 40% to 60% of the patients in each group will be treated with pirfenidone or nintedanib in order to investigate if BBT-877 could be used as either monotherapy or add-on therapy to AFs, to further reduce the decline in lung function and/or improve symptoms as compared to those drugs alone. As a result, 60% to 40% of the patients will receive BBT-877 or placebo, alone.

Institution
MUSC
Recruitment Contact
Zerlinna Teague
8437920965
recruitment@musc.edu

A Phase 3, Open-Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Fazirsiran in Participants with Alpha-1 Antitrypsin Deficiency-Associated Liver Disease

Date Added
September 12th, 2023
PRO Number
Pro00129829
Researcher
Charlie Strange

List of Studies


Keywords
Genetics, Liver, Pulmonary, Rare Diseases
Summary

Individuals with Alpha-1 Antitrypsin Deficiency Associated Liver Disease who were enrolled in and completed the AROAAT2001 or AROAAT2002 study will be invited to participate in this study. This is a 2 year open label extension study of the drug Fazirsiran. Participants will receive 200mg of Fazirsiran every 12 weeks at the study site. Study visits will include blood work, pulmonary function test, questionnaires and liver evaluation testing. A liver biopsy will be performed at end of study, week 102. Potential risk include but are not limited to, allergic reaction to study medication or risk of infection or bleeding from biopsy. Safety and side effects of all assessments and therapies will be monitored throughout the study.

Institution
MUSC
Recruitment Contact
Mary Hayden
843-792-8432
blantonm@musc.edu

TEMPUS GEMINI NSCLC SURVEILLANCE STUDY: A Longitudinal Multi-Omic Biomarker Profiling Study of Patients with Non-Small Cell Lung Cancer (NSCLC)

Date Added
September 27th, 2023
PRO Number
Pro00129366
Researcher
Christopher Gilbert

List of Studies

Keywords
Cancer, Cancer/Lung, Lung, Pulmonary
Summary

The purpose of this study is to identify whether investigational blood and tissue testing can detect cancer cells in the blood stream can tell if subjects are responding to their individual treatment plans.

Participation will last as long as the subject's individual treatment plan and will consist of collecting tissue biopsies (10 slides), which will be taken during the subject's standard of care procedure, as well as blood draws (between 1 and 2 tablespoons), which will be taken during each of the subject's standard of care clinic appointments throughout their care journey.

Institution
MUSC
Recruitment Contact
Michael Balassone
843-792-6696
balassom@musc.edu

A Phase 4, Prospective, Multicenter, Single-Arm Study of a Mean Pulmonary Artery Pressure-Targeted Approach with Early and Rapid Treprostinil Therapy to Reverse Right Ventricular Remodeling in Patients with Pulmonary Arterial Hypertension: ARTISAN (Afterload Reduction To Improve Right Ventricular Structure And FuNction)

Date Added
October 5th, 2023
PRO Number
Pro00129381
Researcher
Rahul Argula

List of Studies


Keywords
Critical Care, Pulmonary Hypertension
Summary

This is a phase 4, prospective, multicenter, single-arm, open-label study designed
to evaluate the effect of early and rapid treprostinil therapy on mean pulmonary artery pressure (mPAP) reduction to improve Right ventricular (RV) function and reverse RV remodeling in patients with PAH. This study will use the CardioMEMS™ HF System (CardioMEMS) to measure and monitor mPAP, but may allow mPAP monitoring via RHC (right heart catheterization), if CardioMEMS is NOT available at a subject's Baseline Visit (Day 1) or if the CardioMEMS™ PA Sensor implantation is unsuccessful.

Participation is expected to be up to 37 months and will include about 10 office visits to the study doctor. This will be divided up into a 30-day Screening Period, a 12-month Treatment Period, and a 24-month Extended Treatment Period.

Institution
MUSC
Recruitment Contact
Zerlinna Teague
8437920965
recruitment@musc.edu

Alpha-1 Foundation Therapeutic Development Network

Date Added
October 23rd, 2023
PRO Number
Pro00132722
Researcher
Charlie Strange

List of Studies


Keywords
Liver, Lung, Non-interventional, Pulmonary, Rare Diseases
Summary

The Alpha-1 Foundation Therapeutic Development Network (TDN) aims to make it easier to design and carry out clinical trials that enhance the treatment of patients with Alpha-1 Antitrypsin Deficiency (AATD). To achieve this, the TDN will establish a network of clinical trial centers that have enough patients to gather a comprehensive database of clinical and genetic information. This data will be crucial in determining the criteria for including or excluding participants in the trials and in recruiting suitable subjects.

Specifically, this study will enroll participants by in person or remote consent who will allow collection of medical records to be entered into an Alpha-1 TDN database. Participants will then be invited to future clinical trials.

Institution
MUSC
Recruitment Contact
Kristin Neff
843-792-1219
neffk@musc.edu

3P-100-ILD-00 Early Feasibility Study Protocol: A Within-subject Device-setting Escalation Early Feasibility Study Evaluating the Safety, Tolerability, and Functionality of 3P-100, an Electric Nitric Oxide (NO) Ambulatory Production and Delivery System, Delivering Nitric Oxide for Inhalation to Subjects with Pulmonary Hypertension (PH) Accompanying Interstitial Lung Disease (ILD), PH-ILD

Date Added
October 31st, 2023
PRO Number
Pro00128998
Researcher
Rahul Argula

List of Studies


Keywords
Interstitial Lung Disease (ILD), Lung, Pulmonary Hypertension
Summary

This is a multi-center early feasibility study evaluating the safety and tolerability of the 3P-100 device which creates and delivers iNO for the treatment of subjects with PH-ILD. All subjects will use the 3P-100 device and receive iNO via the 3P-100 device, aiming for ~4-4.5 hours of treatment. All subjects will remain on their prescribed Oxygen (O2). The study consists of an SV, an iNO treatment period at V1 (Day 0), and a Telephone Call at V1 + 1 Day for safety follow-up.

Institution
MUSC
Recruitment Contact
Zerlinna Teague
8437920965
recruitment@musc.edu

A Phase 2 Multicenter, Randomized, Double-blind, Placebocontrolled Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Efficacy of TTI-101 in Participants with Idiopathic Pulmonary Fibrosis

Date Added
November 6th, 2023
PRO Number
Pro00132082
Researcher
Rachana Krishna

List of Studies


Keywords
Interstitial Lung Disease (ILD)
Summary

This multicenter, randomized, double-blind, placebo-controlled, parallel-group clinical
study is designed to evaluate the safety and tolerability of 3 dose levels of TTI-101 vs placebo in participants with idiopathic pulmonary fibrosis (IPF). a screening period of up to 28 days, 100 participants will be randomly assigned to receive 1 of 3 dose levels of TTI-101 (25 participants per dose level) or matching placebo (25 participants). Enrollment will be stratified by current use of nintedanib. The assigned study drug will be self-administered orally in tablet form twice daily (BID) with a glass of water after a meal. Once randomized, each participant will maintain their current standard of care supportive medications, including cough treatment, medications for symptom relief or quality of life improvements, oxygen therapy where indicated, respiratory physiotherapy, and treatment of comorbidities. Following randomization, all participants will enter the 12-week, double-blind treatment period.

Institution
MUSC
Recruitment Contact
Zerlinna Teague
8437920965
recruitment@musc.edu



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