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.

This study aims to identify which supplemental intervention components could be added to an online physical activity program that contribute to the greatest increases in physical activity in adults with knee replacement. The 4 supplemental intervention components include phone coaching, progress reports to physicians, tailored modules, and personalized feedback. Using the most effective components, an optimized Energize! Exercise intervention will be developed for testing in a subsequent trial.

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.

This study is for patients who have been diagnosed with a solid tumor cancer that has continued to grow despite treatments patients have already received (non-small cell lung cancer or urothelial cancer). The study drug is FF-10832 (gemcitabine liposome injection). Gemcitabine is a cancer treatment registered in the US for the treatment of ovarian, breast, non-small cell lung, and pancreatic carcinomas. The study drug is a new, liposomal formulation of Gemcitabine. This new formula was developed to increase the amount of gemcitabine that goes to tumor cells. The study drug will be given to patients by itself, or in combination with pembrolizumab. Pembrolizumab is an approved treatment for many types of cancer. There are two groups that a participant may be assigned to, which group a participant is assigned on will be determined randomly, in a 1:1 ratio, like flipping a coin. The drugs will be given via an infusion. There is a 50% chance of being assigned to either group. Participation in the study will likely last 12 months, but participants may stay on the study longer if the study treatment continues to benefit them. The study consists of a screening visit, treatment visits, end of study visit, and a long-term follow-up.

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.

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.

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.

The purpose of this study is to evaluate the effectiveness of varenicline (sometimes known as Chantix) compared to placebo (an inactive substance) for the treatment of cigarettes and cannabis (marijuana). Varenicline is not FDA approved for the combination treatment of cigarette abstinence and cannabis reduction or abstinence. All participants will also receive counseling and access to online treatment modules during a quit attempt for cigarettes and a reduction attempt for cannabis. This study is being conducted by the Medical University of South Carolina. All procedures are conducted remotely; therefore, no in-person visits are needed.

To qualify, participants must be 18 or older, live in South Carolina, use cigarettes and cannabis, and are interested in quitting cigarettes and reducing cannabis.

This study is an open label extension study for those who participated in the ION 682884-CS2 clinical trial for transthyretin-mediated amyloid cardiomyopathy (ATTR-CM). ATTR-CM is a disease caused by change in a protein called transthyretin (TTR) which can result in a build up of this protein in parts of the body including the heart. This build up is called an amyloid deposit, and when this occurs in the heart it can lead to a condition called cardiomyopathy. This study involves the medication eplontersen, which is considered investigational meaning it is not approved for commercial use by the Food and Drug Administration (FDA). Eplontersen is aimed at preventing production of the TTR protein to slow or reverse disease progression. Eplontersen is given as an injection under the skin in the upper arm, stomach or thigh. This study will last about 3 1/2 years and include 16 clinic visits. Study procedures include physical exams, blood work, questionnaires, hall walk tests, electrocardiograms (tracing of the heart's electrical activity), echocardiogram (ultrasound test of the heart) and taking a Vitamin A supplement.