The purpose of the study is to evaluate the effect of mivelsiran in adult participants with early-stage DS-AD and to characterize the safety, tolerability, and pharmacodynamics (PD) of mivelsiran. The study will be conducted over 2 periods: a 24-month double-blind period and an optional 12-month open-label treatment extension (OLE) period. The estimated duration of study participation, inclusive of screening, treatment, and additional safety follow-up, is up to 39 months.

Type: Interventional

Start Date: Aug 2026

open study

Researchers are looking for new ways to treat children with relapsed or refractory solid tumors: - Relapsed means the cancer came back after treatment - Refractory means the cancer did not respond (get smaller or go away) to treatment - Solid tumors are cancers mostly in body organs and tissues, not in the blood or other body liquids The study treatment I-DXd (also known as MK-2400 or ifinatamab deruxtecan) is an antibody-drug conjugate (ADC). An ADC attaches to a protein on cancer cells and delivers treatment to destroy those cells. The goals of this study are to learn: - About the safety of I-DXd and if children younger than 12 years old tolerate it - How many children who receive I-DXd have the cancer get smaller or go away

Type: Interventional

Start Date: Jul 2026

open study

Skylark Bio is conducting a study of an investigational new drug called SKY-GJB2 and its delivery device, the SKY-CAT. SKY-GJB2 is a gene therapy that is being developed to treat children who have hearing loss due to changes in the GJB2 gene (also referred to as Connexin 26). The SKY-CAT is a device that delivers the gene therapy. The purpose of this study is to: Learn about the safety and tolerability (does not cause ongoing discomfort) of SKY-GJB2 Evaluate the efficacy of SKY-GJB2 (how well is works)

Type: Interventional

Start Date: May 2026

open study

The goal of this clinical trial is to evaluate the safety and efficacy of privosegtor, a neuroprotective candidate, in patients diagnosed with optic neuritis (ON). Researchers will compare privosegtor and the standard of care (methylprednisolone) to a placebo and standard of care (methylprednisolone).

Type: Interventional

Start Date: May 2026

open study

This study is being conducted to evaluate INCA033989 versus best available therapy in participants with essential thrombocythemia and a CALR mutation previously treated with cytoreductive therapy.

Type: Interventional

Start Date: Sep 2026

open study

The purpose of this research study is to examine how well a medication called lumateperone (Caplyta) works to relieve depression in older adults with treatment-resistant depression. Lumateperone (Caplyta) is approved by the U.S. Food and Drug Administration to treat Major Depressive Disorder in adults who are also taking another antidepressant medication. This study will compare lumateperone (Caplyta) to placebo (a sugar pill without medication).

Type: Interventional

Start Date: Aug 2026

open study

The purpose of this observational research study is to learn more about the natural history of fucosidosis, its symptoms, and how it develops over time. This study intends to collect information from participants diagnosed with fucosidosis; however, this study does not include any medication or treatment other than the usual medical care provided to study participants. The information collected in this study will be used to help understand the disease characteristics of fucosidosis; with this information potentially being able to help design future studies and treatments for this disease. There is currently no approved treatment for patients with fucosidosis. The study consists of 2 parts: a) Part A - retrospective data collection, and b) Part B - prospective data collection.

Type: Observational

Start Date: Mar 2026

open study

The main purpose of this study is to assess the amount of mirikizumab in breast milk in women who are breastfeeding who are receiving stable maintenance doses of mirikizumab for an approved indication. Participation in this study could last up to 94 days, including screening and follow-up period.

Type: Interventional

Start Date: Aug 2026

open study

The intention of the study is to demonstrate superiority of AZD2265 relative to standard of care treatments by assessment of radiographic progression-free survival (rPFS) and overall survival (OS) in participants with mCRPC.

Type: Interventional

Start Date: May 2026

open study

The goal of this study will be piloting the Parents and Babies perinatal depression prevention intervention to examine its feasibility and acceptability and to obtain preliminary data on its efficacy. This includes exploring/understanding users' experiences navigating the interventions (e.g., from an implementation perspective) and preliminary evaluations of clinical outcomes, including mental health and minority stress. With data gathered from this pilot study, we will make final adaptations to the Parents and Babies manuals and plan to develop a randomized-control trial to evaluate efficacy of the intervention.

Type: Interventional

Start Date: Aug 2026

open study

The goal of this clinical trial is to investigate biomarkers of non-IgE-mediated immediate hypersensitivity reactions during infusion of intravenous vancomycin. The main question it aims to answer is: • Identifying novel biomarkers in blood that occur during infusion reaction Participants will: - Have allergy skin testing for vancomycin - Receive an infusion of vancomycin

Type: Interventional

Start Date: May 2026

open study

This phase II trial compares standard consolidation with daratumumab, carfilzomib, lenalidomide, and dexamethasone to consolidation with teclistamab following standard induction therapy and autologous hematopoietic stem cell transplant for improving overall survival of patients with plasma cell leukemia. Consolidation therapy is treatment given after initial therapy to kill any cancer cells that may remain in the body. Daratumumab is in a class of medications called monoclonal antibodies. It binds to a protein called CD38, which is found on some types of immune cells and cancer cells, including myeloma cells. Daratumumab may block CD38 and help the immune system kill cancer cells. Carfilzomib inhibits protein complexes called proteasomes, which inhibits cancer cell growth and leads to cancer cell death. Lenalidomide may help kill cancer cells and prevents the growth of blood vessels that cancer cells need to survive. Dexamethasone is in a class of medications called corticosteroids. It is used to reduce inflammation and lower the body's immune response to help lessen the side effects of chemotherapy drugs. Teclistamab is a monoclonal antibody that may interfere with the ability of cancer cells to grow and spread. A monoclonal antibody is a type of protein that can bind to certain targets in the body, such as molecules that cause the body to make an immune response (antigens). Giving teclistamab as consolidation therapy after induction and autologous hematopoietic stem cell transplant may improve survival outcomes in patients with plasma cell leukemia, compared to standard consolidation with daratumumab, carfilzomib, lenalidomide, and dexamethasone.

Type: Interventional

Start Date: Apr 2027

open study

The purpose of this study is to develop a prospective cohort of patients with and without cardiac sarcoidosis as well as active and inactive disease to develop a prospective biobank from which exosome analysis of peripheral blood will derive unique biomarkers of disease activity in cardiac sarcoidosis.

Type: Observational

Start Date: Jul 2026

open study

This research is being done to determine if an image-based deep learning model (Sybil) can accurately predict the likelihood of future lung cancer based on chest computed tomography (CT) imaging from individuals with a family history of lung cancer.

Type: Observational

Start Date: Jun 2026

open study

This is a phase 2b, multicenter, randomized, double-blind, placebo-controlled study.

Type: Interventional

Start Date: May 2026

open study

The design is a prospective, observational registry for subjects with CHE under the care of dermatology or other qualified healthcare provider. Longitudinal data are collected from both subjects and their treating provider during routine clinical encounters using a structured and standardized data collection method

Type: Observational

Start Date: Jul 2026

open study

The goal of this clinical trial is to learn if a single dose of the study drug, JST-018, is safe and tolerable when administered by injection into the arm or thigh muscle of healthy men and women aged 18 to 55. The main questions it aims to answer are: - Is a single administration of JST-018 safe? - What is the concentration of the JST-018 in the blood over time? - Do antibodies to JST-018 develop following a dose of JST-018? Researchers will compare JST-018 to Placebo to see if there are any differences in the safety and tolerability of a single dose at different dose levels. Participants will be confined to the clinic for the first 3 days. They will receive an injection on the second day, and then return for 9 more visits over the period of 1 year for: - Physical exam with vital signs - Electro-cardiogram (ECG) - Bood collection for clinical labs and research samples - Urine sample - Assessment of potential adverse effects and medications taken

Type: Interventional

Start Date: Jun 2026

open study

This is a first-in-human Phase 1 study evaluating the safety and tolerability of C.001 in patients with retinal diseases involving degeneration of the retinal pigment epithelium. Participants will receive a single administration of C.001 delivered by subretinal injection. The study will evaluate three dose levels in a sequential manner. The primary objective is to assess the safety of C.001 over a 3-month period following administration. Participants will be followed for up to 12 months to further evaluate safety and explore changes in visual function and retinal structure.

Type: Interventional

Start Date: Apr 2026

open study

Single arm- subject treated with Tegoprubart and everolimus. The purpose of this research is to gather information on the safety and effectiveness of investigational regimen containing 2 experimental components: - An investigational drug called Tegoprubart and - Human pancreatic islet cells Both Tegoprubart and human pancreatic islet cells are considered investigational because they are not approved for use in the United States by the Food and Drug Administration (FDA). Participation in this research will last about 5 years. Assess safety, tolerability, and efficacy of transplanted islet cells and immunomodulation with Tegoprubart in combination with anti-thymocyte globulin (ATG), etanercept and with everolimus in adults with brittle T1D and chronic kidney disease (stage 2-3a).

Type: Interventional

Start Date: Apr 2027

open study

The purpose of the study is to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics, and preliminary efficacy of multiple anti-cancer agents in participants with metastatic prostate cancer.

Type: Interventional

Start Date: Jun 2026

open study

The main purpose of this study, performed under the master protocol W8M-MC-CWMM (NCT06143956), is to examine the body weight-lowering efficacy and safety of macupatide plus eloralintide compared with placebo in participants with obesity or overweight and without type 2 diabetes. Participation will last about 64 weeks.

Type: Interventional

Start Date: May 2026

open study

This study will assess the safety, tolerability, and pharmacokinetics (PK) of intravitreal (IVT) injections of RO7663498 in participants with diabetic retinopathy (DR).

Type: Interventional

Start Date: Jun 2026

open study

Cancer is a condition where cells in a specific part of the body grow and reproduce uncontrollably. Acute myeloid leukemia (AML) is a cancer of the blood and bone marrow (the spongy tissue inside the bones) that affects white blood cells that helps to fight infections and also prevents normal blood cell production. This study will assess the adverse events and changes in the disease activity when Pivekimab Sunirine (PVEK) is given in combination with Venetoclax (VEN) and Azacitidene (AZA) in adult participants with AML ineligible to receive intensive chemotherapy. Pivekimab sunirine is a drug being evaluated in the treatment of AML.This is a Phase 2/Phase 3, study of PVEK. Phase 2 is open-label and randomized. Phase 3 is double-blind, randomized. Phase 2 and Phase 3 studies test potential new treatments in patients with a condition or disease. Open-label means that both patients and study doctors know which study treatment is given to patients in Phase 2 of the study. Double-blind means that neither the patients nor the study doctors know who is given which study treatment in Phase 3 of the study. Approximately 660 adult participants will be enrolled in 180 sites worldwide. In Phase 2 of the study, patients will be randomized to receive PVEK + VEN + AZA or standard of care treatment with VEN + AZA. In Phase 3, patients will be randomized to receive PVEK + VEN + AZA or a matching-placebo for PVEK plus VEN + AZA. PVEK is given as an infusion into the vein, AZA is given as an injection under your skin (subcutaneous) or as an infusion into the vein (intravenous) (depending on country where patient enrolls), and VEN is a tablet given by mouth. The total study duration is approximately 71 months. There may be higher treatment burden for participants in this trial compared to their standard of care. Participants will attend regular visits during the study at a hospital or clinic. The effect of the treatment will be checked by medical assessments, blood tests, and checking for side effects.

Type: Interventional

Start Date: Jun 2026

open study

This study is being done to learn about the safety and effects of the study drugs, PF-08653945 and PF-08653944, when given alone or together for weight loss, compared to a placebo (a dummy drug that has no active ingredient in it).

Type: Interventional

Start Date: May 2026

open study

The main purpose of this study is to look at how efgartigimod affects thyroid function in adults with Graves' Disease (GD). The study will also check whether efgartigimod is safe and well tolerated. It will look at how efgartigimod is distributed and eliminated in the body, how it changes antibody levels, and how the immune system responds to it. The study consists of a part A double-blinded treatment period, a part B treatment/observation period and a part C open-label treatment/observation period. During the part A and part B treatment periods, participants will receive efgartigimod PH20 SC via Prefilled Syringe (PFS) or placebo. During the part C open-label treatment period, participants will receive efgartigimod PH20 SC PFS. Participation in the different parts of the study will depend on the participant's response to treatment. The total study duration for participants ranges from 63 to 135 weeks, depending on the response to treatment. More information can be found here: https://clinicaltrials.argenx.com/vitalithy

Type: Interventional

Start Date: Jun 2026

open study