Cystic Fibrosis Patient Registry

Date Added
December 9th, 2002
PRO Number
Pro00120872
Researcher
Patrick Flume

List of Studies


Keywords
Cystic Fibrosis
Summary

The CF Patient Registry is an observational study. It does not involve medical treatments and serves only as an epidemiologic record of demographics, characteristics of disease and treatment, utilization patterns, and clinical outcomes.

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

The Core Center for Clinical Research, Improving Minority Health in Rheumatic Diseases

Date Added
January 15th, 2013
PRO Number
Pro00021985
Researcher
James Oates

List of Studies


Keywords
Autoimmune disease, Environmental Factors, Ethnicity and Disease, Genetics, Healthy Volunteer Studies, Lupus, Minorities, Rare Diseases, Scleroderma
Summary

Systemic lupus erythematosus (lupus; SLE), Systemic Sclerosis (scleroderma; SSc) and Systemic Vasculitis (SV) are relatively rare rheumatic diseases that disproportionately impact the African American community, and particularly African American women. The causes of lupus, scleroderma and vasculitis are unknown, but thought to include both genetic and environmental factors. We are enrolling lupus, scleroderma, and vasculitis patients, and healthy control subjects. This is not a drug study. The purpose of this study is to better understand the factors that predispose people to develop lupus, scleroderma, and vasculitis. Information about medical, social and family history, medications, physical exam findings, and laboratory tests will be collected for analysis. This study will involve approximately 1360 volunteers.

Institution
MUSC
Recruitment Contact
Jonathan Flume
843-792-3926
jof63@musc.edu

Multiple Patient Program for Lamprene® (clofazimine) for the treatment of Non-Tuberculous Mycobacterial (NTM) Infections

Date Added
March 21st, 2017
PRO Number
Pro00065207
Researcher
Patrick Flume

List of Studies


Keywords
Nontuberculous mycobacteria (NTM)
Summary

Lamprene®/Clofazimine, is a product of the pharmaceutical company named Novartis Pharmaceuticals Corporation. Lamprene®/Clofazimine is approved by FDA (the U.S Food and Drug Administration) for the treatment of leprosy. It is being tested in non-Novartis clinical studies for drug resistant tuberculosis and non-tuberculous mycobacteria (NTM).

If you have been diagnosed with NTM, then your doctor may decide that this infection can be treated with Lamprene®/Clofazimine. This medicine is provided to you in an expanded access program. This means that this medicine is not registered for the treatment of NTM, but it can be used in special situations where there are no other possible treatments. For example, this may be because you have a type of Mycobacterial infection that is resistant or failed to respond optimally to other drugs, or because you have had side effects that prevent the use of other drugs.

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

Study to Understand Disease Course Inflammation and Endotype of South Carolinians with Nontuberculous Mycobacteria

Date Added
June 18th, 2019
PRO Number
Pro00089284
Researcher
Patrick Flume

List of Studies


Keywords
Non-interventional, Nontuberculous mycobacteria (NTM)
Summary

The goal of this cohort and biorepository is to collect data and blood specimens on individuals with Nontuberculous Mycobacteria (NTM), pulmonary disease, and healthy adults to better understand the illness and ultimately improve the care and survival of those with these conditions.

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

Multicenter Randomized Pragmatic Clinical Trial Comparing Two-versus-Three Antibiotic Therapy for Pulmonary Mycobacterium Avium Complex Disease

Date Added
July 16th, 2019
PRO Number
Pro00089685
Researcher
Patrick Flume

List of Studies


Keywords
Nontuberculous mycobacteria (NTM)
Summary

The purpose of this study is to better understand treatment practices for Mycobacterium avium complex (MAC) disease. This study is comparing the effectiveness and tolerability of taking 2 drugs compared to 3 drugs in people with MAC disease.

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

ENCORE - A Randomized, Double-Blind, Placebo-Controlled, Active Comparator, Multicenter Study to Evaluate the Efficacy and Safety of an Amikacin Liposome Inhalation Suspension (ALIS)-Based Regimen in Adult Subjects with Newly Diagnosed Nontuberculous Mycobacterial (NTM) Lung Infection Caused by Mycobacterium avium Complex (MAC)

Date Added
February 8th, 2021
PRO Number
Pro00107820
Researcher
Patrick Flume

List of Studies


Keywords
Nontuberculous mycobacteria (NTM)
Summary

The purpose of this study is to evaluate the effect and safety of amikacin liposome inhalation suspension (ALIS) study treatment on patient-reported symptoms in subjects newly diagnosed with NTM lung infection caused by MAC who have not started standard treatment. Around 250 subjects 18 years and older with newly diagnosed NTM lung infection caused by MAC who have not started standard treatment are expected to participate in this study. The study will last around 17.5 months from the Screening visit to the end of the study.

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

A Prospective Study Evaluating Maternal and FetaL Outcomes in the ERa of ModulatorS (MAYFLOWERS)

Date Added
December 1st, 2021
PRO Number
Pro00116448
Researcher
Patrick Flume

List of Studies


Keywords
Cystic Fibrosis
Summary

This is a prospective, multi-center, observational study in pregnant women with cystic fibrosis (CF) to characterize forced expiratory volume in 1 second (FEV1) changes based on exposure to highly effective cystic fibrosis transmembrane conductance regulator (CFTR) modulators. The key factors contributing to the change in lung function during pregnancy and for 2 years post-delivery will be evaluated along with assessment of fetal and maternal outcomes.
The total duration of participation for each participant is expected to be about 3 years (up to 35 months). Women will be enrolled in the first trimester of pregnancy and assessed every 3 months during pregnancy and during the first year after delivery, then every 6 months for an additional year. Over the course of the study, CF clinical data, patient-reported outcomes, questionnaires, obstetrical outcomes, infant growth, child development outcomes, baseline CF-related therapies and co-morbidities will be collected to enable evaluation of changes from before pregnancy to during pregnancy and post-delivery.
MAYFLOWERS participants will be provided an opportunity to participate in an optional continuous glucose monitoring (CGM) sub-study to evaluate glucose control in pregnancy. Participants will undergo CGM sensor placement and data collection as part of the MAYFLOWERS study.

Institution
MUSC
Recruitment Contact
Zerlinna Teague
(843) 792-0965
shannonz@musc.edu

A Phase 2, Double-Blind, Randomized, Parallel-Group, Placebo-Controlled, Multi-Center Study to Evaluate the Efficacy, Safety, and Tolerability of Oral Omadacycline in Adult Subjects with Nontuberculous Mycobacterial (NTM) Pulmonary Disease Caused by Mycobacterium abscessus Complex (MABc)

Date Added
January 26th, 2022
PRO Number
Pro00115910
Researcher
Patrick Flume

List of Studies


Keywords
Nontuberculous mycobacteria (NTM)
Summary

The purpose of this study is to learn more about the safety and effectiveness of the drug omadacyline for people with Nontuberculous Mycobacterial (NTM) pulmonary disease, caused by a type of mycobacteria called Mycobacterium abscessus complex (MABc). Participants will be randomly assigned to receive either omadacycline or placebo, with a 60% chance of being treated with omadacycline and a 40% chance of being treated with placebo. About 75 men and women with NTM lung infection caused by MABc, ages 18 and up, will be in this study, with an estimated 4 participants being enrolled from the MUSC site. Participants will be in this study for approximately 5 months in total, beginning with the Screening visit and continuing until their last study visit.

Institution
MUSC
Recruitment Contact
Zerlinna Teague
(843) 792-1221
recruitment@musc.edu

Development of a nontuberculous mycobacterial pulmonary disease symptom scale (NTM-SS)

Date Added
July 12th, 2022
PRO Number
Pro00119468
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. We seek to develop and test a disease-specific patient-reported outcome measure (PROM) in well-characterized populations of patients with NTM pulmonary disease. Ultimately, a validated PROM for individuals with NTM pulmonary disease will fill a critical need for effective NTM treatments by improving clinical trial design and identifying a meaningful endpoint that can guide therapeutic development.

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

A Phase 2/3, Randomized, Double-blind, Placebo-controlled, Multicenter, Prospective Study to Assess the Efficacy, Safety, and Pharmacokinetics of Orally Administered Epetraborole in Patients with Treatment-refractory Mycobacterium avium Complex Lung Disease (MACrO2)

Date Added
December 14th, 2022
PRO Number
Pro00124613
Researcher
Patrick Flume

List of Studies


Keywords
Nontuberculous mycobacteria (NTM)
Summary

This is a pivotal Phase 2/3, double-blind, placebo-controlled study of oral study drug epetraborole + OBR (Optimized background regimen) versus placebo + OBR in patients with treatment-refractory MAC lung disease. A total of approximately 314 patients will be enrolled in this Phase 2/3 study. For the Phase 2 and Phase 3 parts of the study, the duration of study participation for each patient, not including
Screening, will be up to approximately 19 months. During the conduct of the study, an independent Data and Safety Monitoring Board (DSMB) will be responsible for periodic review of unblinded study safety data by performing a qualitative and quantitative safety assessment.

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



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