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A Study of CTX460 in Adults with Emphysema Associated with Severe Alpha-1 Antitrypsin Deficiency (AATD).

This study aims to enroll 58 pre-adolescent (\<13 years) pediatric participants with sickle cell disease (SCD) who have a pre-adolescent sibling bone marrow donor. All participants will go through a pre-transplant evaluation to find out if there are health problems that will keep…

Patients with unresectable liver metastases (LM) from colorectal cancer (CRC)have a poor prognosis. In patients with resectable disease, surgery offers a distinct survival benefit. This study will offer live donor liver transplantation (LDLT) to select patients with unresectable …

This project seeks to perform a Phase 3 clinical trial to test whether an FDA-approved cancer drug called trametinib is effective in treating young infants with genetic conditions called RASopathies and a severe, life-threatening heart problem called hypertrophic cardiomyopathy. …

Hypoxic-ischemic brain injury (HIBI) is a leading cause of death and lifelong disability in near-term and term infants, most often caused by perinatal arterial ischemic stroke (PAIS, incidence approximately 1:5000 live births) or perinatal asphyxia (PA, incidence 1-2:1000 live bi…

Patients whose colorectal cancer has spread to the liver, and whose liver metastases cannot be removed by surgery or destroyed by ablation, currently receive palliative chemotherapy in Germany. Fewer than one in ten of these patients survive five years. International studies have…

The goal of this study is to collect clinical data and samples and to evaluate the feasibility of allogeneic hematopoietic cell transplantation (HCT) in children and young adults with refractory Systemic Juvenile Idiopathic Arthritis (sJIA), including those with sJIA-associated l…

This clinical study is being conducted to evaluate the safety and effectiveness of zenagamtide in people with atherosclerotic cardiovascular disease (ASCVD) and excess body weight compared with placebo. The purpose of this clinical study is to find out if zenagamtide is safe and …

Bundibugyo virus (BDBV) causes severe and often fatal outbreaks of Bundibugyo virus disease (BVD). No vaccine against BDBV is licensed. There is an urgent need to identify vaccines that are safe, immunogenic, and effective in preventing disease among people at highest risk of inf…

The purpose of this study is to measure the change in bad cholesterol (LDL-C) with VERVE-102 compared to placebo in adults who have plaque buildup in their arteries or are at high risk of developing it.

This study is testing VV-14300 in adults with long-standing type 1 diabetes (T1D) whose blood sugar is not well controlled despite using an automated insulin delivery (AID) system. VV-14300 is an investigational gene therapy that is injected into muscle and is designed to help re…

The purpose of this phase I/II clinical trial is to test CARCIK-CD123/33 cells to treat patients with R/R AML/BPDCN, who have otherwise exhausted conventional treatment options. In particular, this study will assess the safety and feasibility of CARCIK-CD123/33 cells infusion in …

Antiretroviral therapy (ART) has significantly extended the life expectancy of people living with HIV-1 (PLWH), but it does not cure the infection. Broadly neutralizing antibodies (bNAbs), such as 3BNC117 and 10-1074, not only neutralize circulating virus but also engage the immu…

Autoimmune diseases refer to a common category of diseases caused by the immune system reacting to self-antigens, leading to tissue damage. Autoimmune diseases encompass a wide variety of conditions, such as systemic lupus erythematosus（SLE）, Sjögren's syndrome (SS), systemic scl…

Hepatitis B virus (HBV) infection is a major public health threat in China. At present, a functional cure, also known as clinical cure or sustained Hepatitis B surface antigen (HBsAg) loss, is recommended as the ideal endpoint of HBV treatment. However, HBsAg loss can be achieved…

This phase III trial compares denosumab to placebo for the prevention of breast cancer in women with a BRCA1 germline mutation. A germline mutation is an inherited gene change which, in the BRCA1 gene, is associated with an increased risk of breast and other cancers. Denosumab is…

This is a phase II clinical study to evaluate the safety and efficacy of pCAR-19 B cell autologous infusion preparation in the treatment of CD19-positive relapsed/refractory B-cell acute lymphoblastic leukemia.

GNT0004 is a recombinant adeno-associated viral (AAV) vector gene therapy composed of an AAV8 serotype capsid containing a sequence-optimized gene for a human microdystrophin. Duchenne muscular dystrophy (DMD) is a neuromuscular disorder caused by dystrophin gene mutations leadin…

The purpose of this study is to test the safety and efficacy of GD2-CART01, a CAR T cell treatment targeting GD2 in paediatric or young adult patients with High Risk and/or relapsed/refractory Neuroblastoma. A small exploratory cohort of patients with GD2-positive tumors other t…

The purpose of this study is to find out whether it is better to receive a new drug, MEDI4736, or better to receive no further treatment after surgery (and possibly chemotherapy) for lung cancer.