Strength and Muscle Related Outcomes for Nutrition and Lung Function in CF (STRONG-CF)

Date Added
June 19th, 2023
PRO Number
Pro00129339
Researcher
Christina Mingora

List of Studies

Keywords
Cystic Fibrosis
Summary

This is a prospective, multicenter observational study to investigate clinical markers of nutritional status, physical frailty, and sarcopenia in adults with CF spanning a range of lung impairment and to identify barriers and risk factors to optimize nutrition and physical functioning in this patient population. Repeat measurements of body composition and physical function by various methods will be taken during the study and compared to clinical outcomes, as well as with each other. Smaller sub-set studies will be performed to assess bioimpedance analysis (BIA) and/or ultrasound as measures of body composition. Two cohorts matched by age, sex, race, and CFTR genotype severity will be enrolled: (1) those with FEV1 <60% and (2) those with FEV1 ≥60%.

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

Confidence Optimization through Resources for Education, Engagement, and Empowerment (CORE) Study

Date Added
June 29th, 2023
PRO Number
Pro00128946
Researcher
Erin Forster

List of Studies


Keywords
Inflammatory Bowel Disease
Summary

This research study is being conducted to gather information about patients with inflammatory bowel disease (IBD) to better understand their knowledge and preferences about self-help resources and learn how to help patients better manage their disease. Participation in this study will involve signing a research consent form, completing questionnaires, and the opportunity to participate in self-help resources. Some participants may be offered the opportunity to participate in a virtual focus group, as well. Participation in the study will last approximately 3 months.

Institution
MUSC
Recruitment Contact
Zerlinna Teague
843-792-0965
recruitment@musc.edu

Medical University of South Carolina Interstitial and Connective Tissue Associated Lung Disease Biobank (MUSICAL)

Date Added
August 9th, 2023
PRO Number
Pro00127647
Researcher
Aravind Menon

List of Studies

Keywords
Interstitial Lung Disease (ILD), Lung
Summary

The purpose of this study is to learn more about interstitial lung disease through collection of information and blood samples to be used in future research projects. Basic information and a blood sample will be collected in conjunction with clinical care for this study, approximately every 3 months.

Institution
MUSC
Recruitment Contact
Zerlinna Teague
843-792-0965
recruitment@musc.edu

Invasive Mechanical Ventilation (IMV) Strategies Domain

Date Added
September 27th, 2023
PRO Number
Pro00131378
Researcher
Andrew Goodwin

List of Studies


Keywords
Critical Care
Summary

This study is part of what is called a platform study. This platform study, called the "PRACTICAL" study is designed so that various interventions can be evaluated at the same time against standard therapy. This allows researchers to compare these newer interventions to each other as well as to the established usual practice and helps them explore different ways to potentially improve the management of lung injury. Within the platform study there are various different sub-studies that have their own interventions and procedures. This domain sub-study is the "Mechanical Ventilation Study" and it is a multi-centre, randomized, open-label trial that will evaluate multiple ventilation strategies in comparison to conventional lung-protective ventilation in patients with acute hypoxemic respiratory failure (AHRF). This domain will enroll perpetually, as interventions are added, continued, or discontinued. Researchers for this study are looking for different types of ventilation strategies (ways that the ventilators control settings can be adjusted) that may be the most helpful for people in their recovery, while also reducing lung damage caused by the ventilator.

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

Bone Marrow Mesenchymal Stem Cell Derived Extracellular Vesicles for Hospitalized Patients with Moderate-to-Severe ARDS: A Phase III Clinical Trial

Date Added
October 19th, 2023
PRO Number
Pro00132554
Researcher
Andrew Goodwin

List of Studies


Keywords
Critical Care
Summary

This study is a multicenter, randomized, double-blinded, placebo-controlled trial to evaluate the safety and efficacy of ExoFlo for the treatment of moderate-to-severe ARDS (Acute Respiratory Distress Syndrome). The purpose of this study is to research and evaluate the safety and efficacy of intravenous (IV) administration of bone marrow mesenchymal stem cell derived extracellular vesicles, ExoFlo, as treatment for Moderate-to-Severe ARDS. Bone Marrow Mesenchymal Stem Cell (bmMSC)-Derived Extracellular Vesicles is an investigational drug created from human bone marrow being studied for the treatment of moderate-to-severe ARDS. This is a research study that will involve monitoring oxygen and inflammation levels after taking the investigational product and assessing the safety of the investigational product. The experimental treatment is a biologic product called Bone Marrow Mesenchymal Stem Cell (bmMSC)-Derived Extracellular Vesicles Allograft Product ExoFlo, which is purified from the bone marrow of a healthy well-screened individual. Participants will receive either 15mL of the investigational product with 85 mL of normal saline or 100 mL of normal saline only (placebo). The expected duration of participation in the study is a maximum of 61 days, which includes 1-day screening prior to treatment and 60 days following the first treatment.

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

A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Oral Inhalation of Seralutinib for the Treatment of Pulmonary Arterial Hypertension (PAH)

Date Added
February 12th, 2024
PRO Number
Pro00134528
Researcher
Rahul Argula

List of Studies


Keywords
Pulmonary Hypertension
Summary

This Phase 3 study, comprising a double-blind, placebo-controlled treatment period of up to 48 weeks, will assess the efficacy and safety of seralutinib versus placebo when added to background PAH disease-specific medication in adult PAH subjects who are WHO FC II or III. The primary objective of the study is to assess the efficacy of seralutinib relative to placebo with a primary endpoint of change from baseline to Week 24 in 6MWD.

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

A randomized, double-blind, placebo-controlled, multicenter study to assess the efficacy and safety of a 12-week administration of OATD-01, an oral inhibitor of chitinase-1 (CHIT1), for the treatment of active pulmonary sarcoidosis (the KITE study)

Date Added
May 7th, 2024
PRO Number
Pro00136799
Researcher
Walter James

List of Studies


Keywords
Sarcoidosis
Summary

The study will test OATD-01, an experimental medicine, for the first time in patients with pulmonary sarcoidosis (swollen tissue in the lungs). The study goal is to evaluate OATD-01 in the reduction of inflammation and assessing OATD-01 safety.

Everyone who participates in this study will receive OATD-01 or matching placebo (inactive mock tablet). Subjects will be randomly assigned to receive either OATD-01 or placebo for 12 weeks.

The study will run in several hospitals or outpatient clinics, in different countries in Europe and in the USA. In this study, there will be about 98 study subjects in total.

For all patients whether taking OATD-01 or matching placebo, there will be a screening period, a treatment period which will commence after randomization has taken place and will last for 12 weeks and then a follow up visit approximately 4 weeks post last dose of OATD-01.

The total duration of the study is 18 weeks.

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

A Phase 1b/2a Study To Evaluate The Safety Of CMTX-101 In Combination With Inhaled Tobramycin In People With Cystic Fibrosis Chronically Infected With Pseudomonas aeruginosa

Date Added
June 10th, 2024
PRO Number
Pro00137048
Researcher
Patrick Flume

List of Studies


Keywords
Cystic Fibrosis
Summary

This is a double-blind, randomized, placebo-controlled study of CMTX-101 in pwCF who are 18 years of age or older and chronically infected with P. aeruginosa. This study will evaluate the safety and tolerability, PK, immunogenicity, reduction of pulmonary P. aeruginosa burden, and exploratory endpoints of CMTX-101 in up to 41 participants. This study has 2 parts.

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

Patient reported outcomes in patients with nontuberculous mycobacterial pulmonary disease

Date Added
July 29th, 2024
PRO Number
Pro00133747
Researcher
Patrick Flume

List of Studies


Keywords
Lung, Pulmonary
Summary

Nontuberculous mycobacteria (NTM) cause a chronic pulmonary infection associated with cough, fatigue, and shortness of breath. Our primary objectives are to better understand the trajectory of patient-reported outcomes (PROs), e.g.
respiratory symptoms and health-related quality of life (HRQoL), across the entire disease course and measure toxicity and tolerability using patient-reported symptomatic adverse events in treated patients with nontuberculous mycobacterial pulmonary disease (NTM-PD).

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

Health Outcomes of Parents with Cystic Fibrosis (HOPe:CF) Aim 2

Date Added
August 13th, 2024
PRO Number
Pro00136818
Researcher
Patrick Flume

List of Studies


Keywords
Cystic Fibrosis
Summary

We will follow 146 new parents of children <5 years of age at 18 participating US
adult CF centers to assess the primary outcome of ppFEV1 up to 5 years after
becoming a parent. A prospective approach will capture the immediate and longterm impact of the use of the highly effective CFTR modulator ETI by ~90% of US
adults with CF. By combining objective health measures and participant surveys,
we can comprehensively assess the psychosocial impacts of parenthood and
explore the interplay between the parenting role and physical and mental health.
We anticipate identifying modifiable factors that may ameliorate negative health
impacts of parenthood.

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



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