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This is a Phase 1/Phase 2 open-label single arm, multicenter, and multinational study with SAR446268 for treatment of male and female participants 10 to 50 years old with non-congenital myotonic dystrophy (DM) type 1 (DM1). The purpose of this study is to evaluate the safety and…

A Phase 3, Randomized, Double-Masked, Active-Controlled Trial in Adults with Macular Neovascularization Secondary to Age-Related Macular Degeneration

The goal of this clinical study is to learn more about the study drug lenacapavir (LEN), safety, tolerability, and pharmacokinetics (how LEN is absorbed, modified, distributed, and removed from the body of the participants) of once-yearly intramuscular for HIV pre-exposure prophy…

The purpose of this study is to evaluate the efficacy and safety of KarXT + KarX-EC for cognitive impairment in Alzheimer's Disease

Researchers are looking for other ways to treat breast cancer (BC) that is hormone receptor-positive and human epidermal growth factor receptor 2-negative (HR+/HER2-) and either unresectable locally advanced or metastatic. * HR positive (HR+) means the cancer cells have proteins…

How does magnetic resonance-guided laser interstitial thermal therapy (MRgLITT) impact clinical prognosis in mesial temporal lobe epilepsy (mTLE)?

Early Decompressive Hemicraniectomy for High-Risk Large Ischemic Core Stroke Post-EVTAcute Ischemic Stroke (AIS), particularly Anterior Circulation Large Vessel Occlusion (LVO), is a major cause of global disability and death. While endovascular thrombectomy (EVT) is the standard…

The main objective of this study is to evaluate the differences in objective response rate and overall survival between the Purinostat mesylate for injection and selinexor, as assessed by blinded independent central review (BICR), in patients with relapsed or refractory diffuse l…

The purpose of this study is to evaluate the efficacy and safety of SKB264 in patients with unresectable locally advanced, recurrent, or metastatic HR+/HER2- breast cancer who have previously failed endocrine therapy.

The main goal of this clinical trial is to learn if the drug eRapa works to slow down the progression of disease in patients diagnosed with Familial Adenomatous Polyposis (FAP). Researchers will compare eRapa to Placebo. The questions to be answered by this trial are: * Does tak…

This is a prospective, single-arm, open-label and dose-escalation Investigator Initialed study to evaluate LUCAR-G79 in adult subjects with relapsed/refractory autoimmune diseases.

Multicenter, randomized, controlled, open-label, Phase 3 study designed to demonstrate that neladalkib (NVL-655) is superior to alectinib in prolonging progression-free survival (PFS) in patients with treatment-naïve, Anaplastic Lymphoma Kinase (ALK) positive, advanced Non-Small …

This phase III trial compares the effect of adding gemcitabine to intravesical Bacillus Calmette Guerin (BCG) versus intravesical BCG alone in patients with non-muscle invasive bladder cancer that has come back after a period of improvement (recurrent). Gemcitabine is a chemother…

The goal of this clinical trial is to evaluate the safety, tolerability, and recommended Phase 2 Dose (RP2D) of KLN-1010 in patients with relapsed or refractory multiple myeloma.

Researchers are looking for other ways to treat locally advanced or metastatic colorectal cancer (mCRC) that is unresectable and has a gene mutation called KRAS G12C. Standard (or usual) treatments for this type of colorectal cancer may include mFOLFOX6 with or without bevacizum…

Background: X-linked hyper-IgM (HIGM) syndrome is caused by a mutation in the CD40 ligand (CD40L) gene. People with this disease have white blood cells that do not work properly. These people are at risk of severe infections and autoimmune diseases. Researchers want to know if t…

The purpose of this study is to evaluate the efficacy and safety of KarXT + KarX-EC for cognitive impairment in Alzheimer's Disease

The purpose of this study is to evaluate the efficacy, safety and drug levels of CC-97540 in participants with active systemic lupus erythematosus (SLE) including lupus nephritis with inadequate response to glucocorticoids and at least 2 immunosuppressants.

A clinical study to explore the safety, efficacy and cell metabolic kinetics of universal CD19/20 car-t cell injection in moderate to severe refractory systemic lupus erythematosus. This study is a single arm, open, exploratory dose increasing clinical study, which aims to evalu…

This study is an open-label, randomized controlled, multicenter, phase III clinical trial