In the effort to find better treatments for moderate acne, which often relies on long-term antibiotic use, researchers are exploring alternative options. While isotretinoin, a vitamin A derivative, is highly effective for severe acne, its side effects limit its use for milder cases. A recent study from our institution investigated a new approach: weekly isotretinoin dosing. The results were promising, with acne improvement and no major side effects. This suggests that weekly isotretinoin could be a successful alternative for moderate acne in both males and females. To validate these findings, we propose a randomized controlled trial comparing weekly isotretinoin to daily doxycycline over four months. This study could confirm the safety and effectiveness of weekly isotretinoin, as well as shed light on patient satisfaction, and long-term results compared to standard antibiotics. This research may offer a breakthrough in treating moderate acne while addressing concerns about antibiotic overuse.

The Patient Registry for Participation in Aging/Alzheimer's 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.

A clinical research study is an investigation in humans of a potential medical treatment that is designed to discover or verify whether it works and is safe. This study will test an investigational drug called DF9001. "Investigational" means that it has not been approved by any health authority such as the United States Food and Drug Administration (FDA) or the European Medicines Agency (EMA). Information gathered from this study will be sent to the various health authorities.
The study has two parts: a Dose Escalation part and an Efficacy Expansion part. The main purpose of the Efficacy Expansion part of this study is to test whether the study drug (DF9001) works (if it causes cancer to shrink or disappear). The Dose Escalation part of this study's main purpose is to test different doses of the study drug by itself, and also in combination with a drug called nivolumab, an FDA approved drug. Some foreseeable risks associated with DF9001 include Diarrhea,
Skin problems, Nail changes, Eye problems, Inflammation of the lungs, intestines and liver, as well as unforeseen side effects that may occur. There is no direct benefit in participating in this study.

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.

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 investigates a new treatment, V940, combined with pembrolizumab for people who've had surgery for non-small cell lung cancer (NSCLC). The goal is to check if this combination is safe, prevents cancer from returning, improves survival, and enhances quality of life compared to a placebo plus pembrolizumab. Participants will receive pembrolizumab, a drug that boosts the immune system to fight cancer. V940, a personalized treatment, uses genetic information from a patient's tumor to create a custom mRNA injection. It's experimental and not FDA-approved, but it offers a novel approach to empower the immune system against cancer, potentially bringing advancements in post-surgery NSCLC care.

This open-label research study will evaluate two investigational therapies, TransCon TLR7/8 Agonist and TransCon IL-2 β/γ, which are not approved by the U.S. Food and Drug Administration (FDA). Pembrolizumab, an FDA approved medication, also a study drug on this trial is given as standard of care for this type of cancer. This study begins with two study treatment groups, Groups A:TransCon TLR7/8 Agonist injected directed into the tumor once every 21 days and Group B: TransCon IL-2 β/γ which is given through a IV over 30 minutes once every 21 days. The surgery may occur after Cycle 2 of the medications at a scheduled time that the study doctor feels appropriate. Once 12 participants have completed study treatment, 6 in each group (A and B), an analysis will determine the continuation of the study. If it passes the criteria, the study will continue, and another study treatment group, Group C: Pembrolizumad alone given through an IV over 30 minutes once every 21 days, will open. Participants will be randomly assigned to one of the three groups. At the present time, limited information is available on the adverse side effects potentially related to TransCon IL-2 β/γ and TransCon TLR7/8. Very common side effects observed with TransCon IL-2 β/γ include changes with your immune system being stimulated, blood pressure, and heartbeat. Also flu-like symptoms (nausea, fever, and chills), changes in lung/liver/kidney function, and brain related symptoms including dizziness, weakness, confusion, difficulty speaking, and/or decreased brain function, which may become life-threatening. TransCon TLR7/8 Agonist requires more data collection to determine the extent of side effects in humans. At this time, there are no proven benefits in humans however, the animal studies have shown promising results. The national accrual goal for this study is 92 participants and MUSC projects to accrual approximately 5 participants per year over a span of 2 years.

This is a non-interventional, prospective, multinational, post-marketing, observational study designed to describe the real-world effectiveness, safety, and patterns of use of dupilumab in patients with PN. This study will be conducted globally and will collect data available from patients' medical files and other items routinely collected during disease management in clinical practice and PROs related to disease, work productivity, and QoL.