In Our DNA SC: A Helix Research Network Study

Date Added
October 19th, 2021
PRO Number
Pro00115183
Researcher
Daniel Judge

List of Studies


Keywords
Genetics, Healthy Volunteer Studies
Summary

The purpose of this project is to study DNA and its connection to your health. DNA is in your blood, your saliva, and other tissues in your body. DNA is the unique instructions you are born with that tells your body how to work. By looking at DNA, you can learn information about your health, certain traits, and even your ancestral roots. DNA is also called your genetic information. DNA is mostly the same from person to person. But everyone's DNA is slightly different. We are still learning how DNA impacts health. The study will look at the DNA of many different people from many different backgrounds and compare it to information in their health records. The goal is to understand how learning about DNA can help improve health care for individuals, families, and the community. Participants will provide a sample for DNA sequencing. Sequencing is the process of reading the letters of your DNA. This study may sequence your whole genome. Over time, you may be asked to provide additional samples for research purposes. The research team will collect health information about you from your medical record and may ask you questions about your health using surveys or other data collection methods

Institution
MUSC
Recruitment Contact
Joseph Baierl
(843) 876-0582
InOurDNASC@musc.edu

An Open-Label Extension and Safety Monitoring Study of Acoramidis (AG10) in Participants with Symptomatic Transthyretin Amyloid Cardiomyopathy Who Completed the Phase 3 ATTRibute-CM Trial (AG10-301)

Date Added
October 12th, 2021
PRO Number
Pro00114829
Researcher
Daniel Judge

List of Studies


Keywords
Drug Studies, Heart
Summary

Amyloidosis (ATTR) Cardiomyopathy (CM) is a progressive, fatal disease in which amyloid deposits build up slowly, over the course of many years to cause organ damage. This study will continue to evaluate the safety and tolerability of a new drug called acoramidis given to adult patients with symptomatic transthyretin amyloid cardiomyopathy. All participants will be given the study medication, which is taken by mouth twice a day.

This study is enrolling subjects who participated in the AG10 301 study, and are eligible to enroll in this study within 30 days of completing the 30 month visit for the AG10 301 study. This study is expected to last approximately 5 years and will have in person visits at day 1, month 1 and then every 6 months along with phone visits quarterly when an in person visit is not required.

Institution
MUSC
Recruitment Contact
Hannia Ramos
843-876-4806
ramosha@musc.edu

A Phase 3 Global, Double-Blind, Randomized, Placebo‑Controlled Study to Evaluate the Efficacy and Safety of ION-682884 in Patients with Transthyretin‑Mediated Amyloid Cardiomyopathy (ATTR CM)

Date Added
June 8th, 2021
PRO Number
Pro00110317
Researcher
Daniel Judge

List of Studies


Keywords
Cardiovascular, Heart
Summary

The purpose of this study is to evaluate the safety and effectiveness of ION-682884 and determine if it can help people with transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) which is a disease caused by change in a protein called transthyretin (TTR). ION-682884 is an investigational drug. "Investigational" means that ION-682884 is not yet approved for use in any settings outside of clinical research studies like this one and is considered experimental. ION-682884 has been shown to reduce the level of TTR in the blood of animals and healthy volunteers tested to date. Reducing the amount of TTR in your blood may reduce the amount of amyloid deposits in your body and may keep your cardiomyopathy from getting worse over time. However, it is not known if ION-682884 will help your cardiomyopathy.

If you meet the requirements and agree to participate in this study, you will receive subcutaneous (under the skin) injections of either ION-682884 or placebo once every 4 weeks from week 1 through week 117.
Your participation in this study will last approximately 36 months and will consist of clinic visits and follow up visits at home.

Institution
MUSC
Recruitment Contact
Hannia Ramos
843-876-4806
ramosha@musc.edu

Data Capture of 6-Minute Walk Test Performance by a Wearable Monitoring Device in Patients With Transthyretin Amyloid Cardiomyopathy and Heart Failure.

Date Added
April 16th, 2021
PRO Number
Pro00107809
Researcher
Daniel Judge

List of Studies


Keywords
Cardiovascular
Summary

This study is assessing a wearable monitoring device to be used by the study participants in the ATTRibute-CM (AG10-301) study while they complete the 6 minute hall walk at their in clinic study visits. The results of the hall way data captured by the wearable monitor will be compared to the data captured by the study

Institution
MUSC
Recruitment Contact
Hannia Ramos
843-876-4806
ramosha@musc.edu

A Phase 3, Multinational, Randomized, Placebo-controlled Study of ARRY-371797 (PF-07265803) in Patients with Symptomatic Dilated Cardiomyopathy Due to a Lamin A/C Gene Mutation

Date Added
April 10th, 2018
PRO Number
Pro00075889
Researcher
Daniel Judge

List of Studies


Keywords
Cardiovascular, Drug Studies, Genetics
Summary

The purpose of this research study is to test if ARRY-371797 (PF-07265803) is safe and effective in treating dilated cardiomyopathy caused by a genetic mutation to the LMNA gene. This will be demonstrated by the functional capacity of patients in their 6-minute walk tests and quality of life questionnaires.

Institution
MUSC
Recruitment Contact
Kavin Panneerselvam
843 792 0464
panneeer@musc.edu



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