The Participant REgistry for Prevention-focused Aging/dementia REsearch (PREPARE) is a database of individuals with Mild Cognitive Impairment who are interested in participating in research on aging/Alzheimer's disease and related dementias conducted at the Medical University of South Carolina (MUSC). PREPARE will connect MUSC researchers with potential participants so they can provide information about studies for which they may be eligible. PREPARE will also collect survey data from people with Mild Cognitive Impairment and their study partners to better understand the impact of this condition on daily functioning.

Tetra Discovery Partners, Inc. has two studies that are recruiting males aged 9 to 45 to participate in a treatment clinical trial for Fragile X Syndrome. The treatment, called BPN14770, is a phosphodiesterase inhibitor that has the potential to address cognitive and neurological impairment in those with Fragile X Syndrome.

BPN14770-CNS-301 is a clinical trial for males aged 18 to 45 with Fragile X Syndrome. It is double-blind, meaning that neither the study doctor nor the enrolled participants know whether or not they are receiving study drug or placebo. A placebo is an inactive material that looks like the study drug, but does not contain any active study drug.

The main goals of this study are to learn how well the study drug works and how safe the study drug is compared with placebo.

After completing the double-blind study, participants have the opportunity to enroll in the open-label extension of this clinical trial, called BPN14770-CNS-302; "open-label" means that the study doctor and participants are all aware that participants are definitely getting the study medication.

This is a randomized, double-blind, parallel group, vehicle-controlled phase to evaluate the efficacy and safety of diacerein 1% ointment applied topically once daily for 8 weeks for the treatment of adult and pediatric (age ≥ 6 months) patients with generalized EBS. The duration of study participation is anticipated to be approximately ~16 to 20 weeks per patient consisting of a Screening Period of up to 4 weeks, a Treatment Period of 8 weeks and a No Treatment Follow-up Period of 8 weeks. Patients that complete this portion of the study will be eligible to participate in an open-label, 24-week extension phase to evaluate the long-term safety of diacerein 1% ointment for the treatment of generalized EBS.

This study is for participants who have acute leukemia, a type of cancer of the blood and bone marrow. This study will help us learn more about a newly discovered oral drug, DSP-5336, that is being tested in people with acute leukemia and other types of blood cancer. DSP-5336 is a drug that interferes with a protein (Menin) in the body that has been found to have a role in developing leukemia. The main purpose of this study is to evaluate how safe DSP-5336 is at different doses and to find a recommended dose that is safe for further study. The overall planned study duration, including the above visits and periods, is 50 months (4.2 years).

This study is for those with Transthyretin (TTR) Amyloidosis with Cardiomyopathy or ATTR-CM. ATTR-CM is a condition where the heart muscle does not pump your blood through your body as it should because of build up of abnormal protein between the muscle fibers in your heart. The specific protein is called transthyretin amyloid (TTR). The study involves an investigational drug called NTLA-2001. Investigational means it is not approved for commercial sale or use by the Food and Drug Administration. This study is a randomized study meaning participants will be assigned by chance to receive NTLA-2001 or placebo. A placebo looks like the investigational drug but contains no active ingredient. In this study, two out of three participants are assigned to receive NTLA-2001 and one out of three will receive placebo. NTLA-2001 or placebo are given by intravenous (IV) infusion meaning in your vein.

NTLA-2001 consists of a gene editing system which can "knockout" the TTR gene and permanently change the DNA (deoxyribonucleic acid, which contains information that determines parts in traits such as eye color or height, that are passed on from parent to child) so the gene stops making the TTR protein.

This study will last at least 18 months and include up to 17 visits. Study related procedures include physical exams, blood work including genetic testing, eye exam, hall walk test, electrocardiograms (ECGs), echocardiogram (echo) and vitamin A supplement. Study risks include infusion related risks like headache, fever, chills, or increased liver enzymes. Other study related risks include genetic risks, loss of confidentiality and unknown risks.

The study is for patients who have been diagnosed with an advanced/metastatic (meaning cancer has spread from where it started) solid tumor (cancer) with no standard treatment available. The investigational drug in this study is DB-1311. DB-1311 infusion is an antibody-drug combination composed of an anti-B7-H3 (a protein associated with worse overall survival and drug resistance) antibody and P1021 (novel topoisomerase I inhibitor). The antibody portion of the drug blocks a protein in the body that help cancer cells live, grow and spread. The purpose of this trial is to find a safe and tolerable dose of the study drug. Participation in this study will last about 24 months. The study consists of a screening visit, treatment visits, and a safety follow up visit.

For this study, we are recruiting 54 individuals with Parkinson's Disease and Freezing of Gait (FOG) who are planning to undergo Deep Brain Stimulation (DBS). The objective of this study is to better understand the FOG response to DBS. Prior to DBS study participants will undergo a MRI scan, behavioral assessment related to walking, a cognitive evaluation, and assessment of other Parkinson's disease symptoms. Following DBS participants will repeat these assessments at multiple timepoints over the period of three years. Overall, participants will complete a total of 9 visits over a period of approximately 3 years.

This is a research study to examine if and how patterns of use change over time among three different groups of tobacco users: 1) adults who exclusively smoke combustible cigarettes, 2) adults who exclusive use e-cigarettes, and 3) adults who use both. This is a naturalistic survey study, there is nothing required or requested of participants, other than to complete our surveys. Participants do not need to quit or reduce their smoking/e-cigarette use if they do not want to. They do not need to travel for this study; it is completely remote and they can participate from your home.
Participation in the study will take place over a period of 12 months in the form of: 1) Monthly Surveys (13 total), 2) Daily Diaries for the first 90 days, 3) Follow-Up Diaries in each of the 7 days leading up to each monthly follow-up survey, and 4) Submission of Breath Samples (13 total).

Assess the safety and effectiveness of the ASG device in
the treatment of lesions involving the ascending aorta and
aortic arch.

The purpose of this study is to learn more about long-term safety (good or bad effects) of avacopan and its efficacy (how well it is working) in treatment of antineutrophil cytoplasmic autoantibody (ANCA)-associated vasculitis.

Avacopan is currently approved as an adjunctive (another treatment used along with primary treatment) treatment in adult people with severe active ANCA-associated vasculitis in the US and is also approved in the EU, Canada, Japan, and other countries.

In this study, avacopan will be compared with a placebo (a drug that looks likes the study medication but does not contain any medication). This is a randomized study, meaning that you will be assigned by chance (like flipping a coin) into a treatment group. You will have an equal chance of being placed in either of the 3 following groups: treatment with avacopan for 5 years in group A, or treatment with avacopan for 1 year followed by placebo for 4 years in group B, or treatment with placebo for 5 years in group C. The study is also a double-blinded study, meaning you and your study doctor will not know what you are receiving, the avacopan or placebo.

The study is sponsored by Amgen, Inc. Participation in the study will require 27 visits to the MUSC main campus over approximately 63 months, and visits will include the following procedures: blood draw, urine collection, physician-led assessments of your disease (for example physical exam and medical history review), and health questionnaires. You will also be provided with a paper diary to record any missed doses/overdoses of the study drug.

Compensation is available for participation.