A Research Study to Advance the CF Therapeutics Pipeline for People without Modulators

Date Added
September 18th, 2024
PRO Number
Pro00139279
Researcher
Patrick Flume

List of Studies


Keywords
Cystic Fibrosis
Summary

This is a prospective, longitudinal, multicenter, nonrandomized observational study to obtain research quality data across key outcome measures in people with Cystic Fibrosis who are ineligible and/or not taking a approved CFTR modulator and who are not receiving an investigational therapy.

Participants will be seen at study sites for research visits to include spirometry, patient reported outcomes (PROs), and blood collections on Day 0, Day 90 (3 months), Day 180 (6 months), and Day 360 (12 months). Participants will complete home spirometry as well during this period.

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

A Phase 3, Open-label, Efficacy-Assessor-Blinded Study, Comparing the Safety and Efficacy of Upadacitinib to Dupilumab in Children from 2 to Less than 12 Years of Age with Moderate to Severe Atopic Dermatitis (START UP)

Date Added
October 9th, 2024
PRO Number
Pro00138750
Researcher
Lara Wine Lee

List of Studies


Keywords
Skin
Summary

This study is being conducted at approximately 150 research centers worldwide and is expected to enroll approximately 675 pediatric subjects in total with moderately to severely AD. This study will have 2 cohorts, a Randomized Cohort, and a Dupilumab-Inadequate Responder / Dupilumab Medically Inadvisable Cohort. The study comprises a 35-day Screening Period; a 16-week, open-label, efficacy assessor blinded study treatment period for the subjects in the randomized cohort; an open-label period up to Week 160 for subjects in the upadacitinib study treatment arms across both cohorts (Randomized Cohort and Dupi-IR/Dupilumab Medically Inadvisable Cohort); an open label period up to Week 52 for subjects in the dupilumab arm; and a 30-day Follow up Visit/call after the last dose is administered for upadacitinib or dupilumab.

Institution
MUSC
Recruitment Contact
Courtney Rowley
843-792-9784
rowle@musc.edu

Evaluation of Low- and High-Nicotine Tobacco-Free Nicotine Pouches as a Harm Reducing Substitute for Combustible Cigarettes

Date Added
October 15th, 2024
PRO Number
Pro00139710
Researcher
Amanda Palmer

List of Studies

Keywords
Smoking
Summary

Tobacco-free oral nicotine pouches (such as Zyn brand) are a less harmful alternative to combustible tobacco (cigarette/cigarillo/little cigar) smoking. Pouches, however, contain nicotine, and addictive substance that is not risk-free. The present study is evaluating how well nicotine pouches, at different nicotine levels, help people switch away from smoking combustible tobacco. People who smoke will be asked to answer questions about their tobacco product use and provide breath samples and cheek swab samples at an in-person visit to MUSC Charleston. Participants will then be provided with a 28-day supply of nicotine pouches, and will be asked to switch from smoking to pouches over the course of 4 weeks. Finally, participants will complete a final visit at MUSC, and will answer more questions about their tobacco use 1-month later.

Institution
MUSC
Recruitment Contact
Talia Ogretmen
843-259-8111
ogretmet@musc.edu

Safety and Efficacy of Workflows of High-Volume Single Operators in a Left Atrial Appendage Occlusion Device Implant Procedural Day: SAFE HV

Date Added
October 30th, 2024
PRO Number
Pro00137571
Researcher
Loren Morgan

List of Studies

Keywords
Cardiovascular, Heart, Vascular
Summary

The purpose of this research is to gather information about the effectiveness and safety of Left Atrial Appendage Occlusion (LAAO) device procedures in patients using a Watchman device performed on days where doctors perform a large number of procedures.

The study primary outcome is to evaluate complications during the procedure up to 30 days after the procedure.

The final outcome is to determine successful placement within 31 - 90 days after implant.

Institution
MUSC Heart and Vascular Institute
Recruitment Contact
Jacqueline Sheriod-Scott
8032552927
sheriods@musc.edu

A PHASE III, OPEN-LABEL, MULTICENTER, RANDOMIZED STUDY EVALUATING GLOFITAMAB AS A SINGLE AGENT VERSUS INVESTIGATOR'S CHOICE IN PATIENTS WITH RELAPSED/REFRACTORY MANTLE CELL LYMPHOMA

Date Added
November 6th, 2024
PRO Number
Pro00135996
Researcher
Katherine Antel

List of Studies

Keywords
Cancer, Cancer/Lymphoma, Drug Studies, Men's Health, Women's Health
Summary

This study is for subjects that have been diagnosed with mantle cell lymphoma that has spread and has not responded to treatment. This study is testing an "investigational" (not yet FDA approved) study drug called glofitamab. The purpose of this study is to compare the effects, good or bad, of glofitamab (experimental arm) versus bendamustine plus rituximab (BR) or rituximab plus lenalidomide (R-Len;the control arm) on subjects with relapsed/refractory mantle cell lymphoma. Your total time in the study and the number of assessments in the follow up visits, will depend on how your MCL responds to study treatment. This could range from 1 day to more than 24 months. The screening period may last up to 28 days (4 weeks) and may involve more than one visit to the clinic.

Institution
MUSC
Recruitment Contact
HCC Clinical Trials Office
843-792-9321
hcc-clinical-trials@musc.edu

Portable Transcranial Magnetic Stimulation (TMS) in a Van for Treatment-Resistant Depression

Date Added
December 3rd, 2024
PRO Number
Pro00139739
Researcher
Mark George

List of Studies


Keywords
Anxiety, Depression
Summary

In this pilot study we will assemble a portable TMS(transcranial magnetic stimulation) unit, in a van, and then test out delivering TMS in Medical University of South Carolina (MUSC)-affiliated facilities in South Carolina, all within 90 minutes of driving from Charleston, SC. This work will hopefully open up access to TMS for millions of patients with treatment-resistant depression who cannot be treated in the current model.
We will recruit up to 30 treatment-resistant depression patients who live near one of the MUSC-affiliated clinics. We will treat these patients, open-label, with FDA-approved accelerated TMS (6 sessions each day, over 2 hours, for 5 days, spread over one or two weeks). We will measure TMS effectiveness using standard depression rating scales.

Institution
MUSC
Recruitment Contact
Alexandra Herron
843 876 9004
herronal@musc.edu

Portable Transcranial Magnetic Stimulation (TMS) in a Van for Treatment-Resistant Depression

Date Added
December 3rd, 2024
PRO Number
Pro00139739
Researcher
Mark George

List of Studies


Keywords
Anxiety, Depression
Summary

In this pilot study we will assemble a portable TMS(transcranial magnetic stimulation) unit, in a van, and then test out delivering TMS in Medical University of South Carolina (MUSC)-affiliated facilities in South Carolina, all within 90 minutes of driving from Charleston, SC. This work will hopefully open up access to TMS for millions of patients with treatment-resistant depression who cannot be treated in the current model.
We will recruit up to 30 treatment-resistant depression patients who live near one of the MUSC-affiliated clinics. We will treat these patients, open-label, with FDA-approved accelerated TMS (6 sessions each day, over 2 hours, for 5 days, spread over one or two weeks). We will measure TMS effectiveness using standard depression rating scales.

Institution
MUSC Health Florence Medical Center
Recruitment Contact
Alexandra Herron
843 876 9004
herronal@musc.edu

Portable Transcranial Magnetic Stimulation (TMS) in a Van for Treatment-Resistant Depression

Date Added
December 3rd, 2024
PRO Number
Pro00139739
Researcher
Mark George

List of Studies


Keywords
Anxiety, Depression
Summary

In this pilot study we will assemble a portable TMS(transcranial magnetic stimulation) unit, in a van, and then test out delivering TMS in Medical University of South Carolina (MUSC)-affiliated facilities in South Carolina, all within 90 minutes of driving from Charleston, SC. This work will hopefully open up access to TMS for millions of patients with treatment-resistant depression who cannot be treated in the current model.
We will recruit up to 30 treatment-resistant depression patients who live near one of the MUSC-affiliated clinics. We will treat these patients, open-label, with FDA-approved accelerated TMS (6 sessions each day, over 2 hours, for 5 days, spread over one or two weeks). We will measure TMS effectiveness using standard depression rating scales.

Institution
MUSC Health Orangeburg
Recruitment Contact
Alexandra Herron
843 876 9004
herronal@musc.edu

M22-574: Phase 3 ABBV-383 vs Standard Available Treatment in Subjects with RRMM with at Least 2 Prior Lines of Therapies

Date Added
December 6th, 2024
PRO Number
Pro00136949
Researcher
Kimberly Green

List of Studies

Keywords
Cancer
Summary

Multiple Myeloma (MM) is a cancer of the blood's plasma cells ( blood cell). The cancer is typically found in the bones and bone marrow (the spongy tissue inside of the bones) and can cause bone pain, fractures, infections, weaker bones, and kidney failure. Treatments are available, but MM can come back (relapsed) or may not get better (refractory) with treatment. This is a study to determine adverse events and change in disease symptoms of ABBV-383 in adult participants with relapsed/refractory (R/R) MM. ABBV-383 is an investigational drug being developed for the treatment of R/R Multiple Myeloma (MM). This study is broken into 2 Arms; Arm A (Parts 1 and 2) and Arm B. Arm A includes 2 parts: step-up dose optimization (Part 1) and dose expansion (Part 2). In Part 1, different level of step-up doses are tested followed by the target dose of ABBV-383. In Part 2, the step-up dose identified in Part 1 (Dose A) will be used followed by the target dose A of ABBV-383. In Arm B a flat dose of ABBV-383 will be tested. Around 120 adult participants with relapsed/refractory multiple myeloma will be enrolled at approximately 30 sites across the world. Participants will receive ABBV-383 as an infusion into the vein in 28 day cycles for approximately 3 years. There may be higher treatment burden for participants in this trial compared to their standard of care. Participants will attend regular visits during the study at a hospital or clinic. The effect of the treatment will be checked by medical assessments, blood tests, checking for side effects and questionnaires.

Institution
MUSC
Recruitment Contact
HCC Clinical Trials Office
843-792-9321
hcc-clinicaltrials@musc.edu

A Phase 1, Randomized, Double-Blind, Placebo-Controlled, Single Oral Dose Study Evaluating the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 349 in PiZZ and PiMZ/MASH Adult Participants

Date Added
December 10th, 2024
PRO Number
Pro00141286
Researcher
Charlie Strange

List of Studies


Keywords
Drug Studies, Genetics, Liver, Rare Diseases
Summary

Alpha-1 antitrypsin (AAT) deficiency is a condition in which the body does not make enough of AAT, a protein that protects the lungs and liver from damage. This condition is inherited, meaning you get the faulty gene from one or both of your parents.

The purpose of this study is to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of oral administration of BMN 349 in adult participants with the PiZZ genotype (also defined as having a severe deficiency or AAT ≤ 60mg/dL) or PiMZ genotype with metabolic dysfunction-associated steatohepatitis (MASH). Participants will receive an oral single dose of BMN 349 (250 mg), a medication designed to assist Z-alpha-1 antitrypsin to get out of the liver cell. The study drug BMN 349 has not been approved by the Federal Drug Administration (FDA).

This is a phase 1 study in which participants will get a single pill dose of drug or placebo to measure the amount of alpha-1 that gets out of the liver cells and into the bloodstream. Study details include:
• Study duration: up to 78 days
• Treatment duration: 1 day (single dose).
• Observations: The study will collect data on medical history, physical examination, vital signs, electrocardiogram readings, clinical laboratory parameters, pulmonary function tests, and drug distribution.
• Visit frequency: The Screening Visit, dosing, and post dosing evaluations will be conducted at MUSC on 3 consecutive days. Visits at days 8 and 36 days will occur at study site. Other procedures/assessments may be performed at the study site or at home by a healthcare professional and/or by telemedicine.

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



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