Acquired metabolic disease

This early study tests whether the drug CAEL-101/anselamimab can bind to and shrink abnormal protein deposits (amyloid) in people with AL amyloidoma. Five adults with measurable amyloid deposits will receive the drug and be monitored for up to 72 weeks. The goal is to see if the …

This Phase 2 trial tests a new drug called QLS4131, given under the skin, in combination with other medications for people with malignant plasma cell neoplasms like multiple myeloma. The study will compare different drug combinations to see which works best at shrinking tumors an…

This study tests an experimental drug called elranatamab in people newly diagnosed with AL amyloidosis who have not yet had treatment. The drug helps the body's immune cells find and destroy the abnormal cells causing the disease. Researchers want to see if it can produce deep re…

This study tests a new drug called NM8074 in 12 adults with paroxysmal nocturnal hemoglobinuria (PNH) who are already taking Soliris. PNH is a rare blood disorder where the immune system destroys red blood cells, causing anemia and fatigue. NM8074 is designed to block a different…

This early-phase study tests a new treatment called CAR19-BCMA CAR-T for people with certain blood cancers (plasma cell neoplasms or lymphomas/leukemias) that have returned or not responded to at least three prior treatments. The therapy uses a patient's own immune cells, modifie…

This study tests a new treatment for people with a rare disease called light-chain cardiac amyloidosis that has come back or not responded to other treatments. The treatment uses specially engineered immune cells (CAR-NK cells) from donated umbilical cord blood to target and atta…

This Phase 2 trial tests a new drug, belantamab mafodotin, combined with standard chemotherapy in 60 adults newly diagnosed with AL amyloidosis, a rare disease where abnormal proteins damage organs. The goal is to see if the combination improves blood and organ responses. The stu…

This phase II trial tests the drug SLN12140 in 10 adults with PNH who have never taken complement inhibitors. The goal is to see if SLN12140 can reduce red blood cell destruction (measured by LDH levels) and improve hemoglobin. Participants receive different doses over 12 to 64 w…

This early-phase trial tests a new type of immune cell therapy called GPRC5D CAR-T in 18 people with relapsed or treatment-resistant plasma cell disorders, such as multiple myeloma. The therapy involves giving patients a single infusion of their own modified immune cells designed…

This phase 2 trial tests a drug called sonrotoclax, combined with dexamethasone and sometimes daratumumab, in 39 people with a specific genetic form of AL amyloidosis (t(11;14)). The goal is to see if the treatment can quickly reduce harmful protein levels and improve organ funct…

This study looks at the long-term safety and effectiveness of an injection called LP-005 in adults with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder. It is for people who already completed an earlier LP-005 study. The goal is to see if LP-005 can help control …

This Phase 3 trial tests NTQ5082, an oral capsule, against the standard infusion eculizumab for paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder. About 78 adults who have not had complement inhibitor therapy will be enrolled. The main goal is to see if NTQ5082 can…

This Phase 3 trial tests NTQ5082 capsules, a new drug that blocks a protein called CFB, in people with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder that destroys red blood cells. The study will follow 78 adults who previously benefited from NTQ5082 or took ano…

This study explores whether a PET/MR scan using a radioactive tracer called 18F-Florbetaben can detect amyloid deposits—abnormal protein clumps that can damage organs—in people with certain blood disorders. Participants receive a single injection of the tracer followed by a whole…

This trial tests whether cabergoline, a drug that lowers the hormone prolactin, is safe for people with schizophrenia who have high prolactin levels due to antipsychotic medication. About 146 adults will receive either cabergoline or a placebo for 12 weeks, and researchers will c…

This study looks at 50 adults with AL amyloidosis who achieved complete remission after initial chemotherapy. It checks if tiny amounts of remaining disease (called minimal residual disease, or MRD) affect organ recovery and survival. Researchers also test whether a stem cell tra…

This 10-year observational study will follow 10,000 adults with diabetes, metabolic disorders, and related conditions like high blood pressure and fatty liver disease. Researchers will collect routine medical data to identify markers of disease severity and activity. The goal is …