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A 96-week, open-label, Phase 2 study to evaluate the efficacy and safety of Nizubaglustat (AZ-3102) in patients with Gaucher disease type 3 (GD3).

To find out if giving blinatumomab as injections under the skin can help to control MRD and keep the disease from coming back in participants with B-cell ALL.

To look at the effectiveness of sacituzumab tirumotecan (MK-2870) in treating participants with treatment-refractory unresectable and/or metastatic anal/rectal cancer.

Severe burn injury frequently requires prolonged mechanical ventilation because of inhalation injury, respiratory failure, systemic inflammation, and repeated surgical procedures. Patients with extensive burns are at high risk of ventilator-associated complications, prolonged int…

The goal of this clinical trial is to determine whether AN4035 is safe and tolerable in people with advanced or metastatic solid tumors that have Rat Sarcoma oncogene (RAS) mutated solid tumors and high levels of the CEACAM5 protein. RAS genes help control how cells grow and divi…

The purpose of this study is to find out if zenagamtide is safe and effective for treating participants who have excess body weight compared to treatment with semaglutide.

Autosomal dominant polycystic kidney disease (ADPKD), the most commonly inherited kidney disease, is characterized by the development of cysts in the kidney that impair function. Of those affected, half will progress to end-stage kidney disease by age 60, requiring dialysis or ki…

This phase II trial studies how well epcoritamab works in combination with the chemotherapy regimen dose adjusted etoposide, prednisone, Oncovin (vincristine), cyclophosphamide, hydroxydaunorubicin-rituximab (DA-EPOCH-R) in treating patients with high risk Burkitt lymphoma. Epcor…

Exposure to general anesthesia during early brain development has emerged as an important risk factor for long-term attention-deficit/hyperactivity disorder (ADHD)-like behaviors and learning difficulties in school-aged children. However, objective biomarkers for perioperative ne…

This is a randomized, phase II, multi-centre clinical trial. Sample size: 68 patients (Experimental Arm (Durvalumab + chemotherapy + FMT capsules): 34 patients, Control Arm (Durvalumab + chemotherapy): 34 patients) Population: Patients with stage IIA, IIB, IIIA and IIIB (only T…

The purpose of the study is threefold: 1. Assess the safety and efficacy of a single high-dose intravenous (LAmB 10mg/kg) compared to the SOC daily dosing (3mg/kg) of the same medication for induction therapy in moderate to severe histoplasmosis. 2. Assess the safety and efficac…

The aim of this trial is to assess the feasibility of EREG/AREG assessment as a clinical diagnostic standard, used to guide clinical decision making in right-PTL, RAS-wt aCRC. Further to this, the aim is to determine whether EREG/AREG status identifies right-PTL participants who …

The clinical trial is intended to assess for clinical evidence of Clemastine Fumarate as a myelin repair therapy in patients with acute inflammatory injury-causing demyelination as measured by multi-parametric MRI assessments. No reparative therapies exist for the treatment of a…

This is a single-arm, open-label and dose-escalation clinical study to evaluate the LVIVO-TaVec122 product in adult subjects with Relapsed/Refractory B-cell Malignancies.

Adults who are unconscious or severely ill and need a breathing tube connected to a ventilator are at high risk of developing a lung infection (pneumonia) within the first few days in the intensive care unit. This early pneumonia affects up to 30 to 50 % of certain high-risk pati…

The objective of this study is to evaluate the safety, tolerability, and pharmacokinetics (PK) of S241656 and to determine the recommended dose for expansion (RDE) of S241656 in participants with relapsed/refractory (R/R) acute myeloid leukemia (AML), myelodysplastic syndrome/acu…

This study will evaluate the efficacy and non-inferiority of a non-surgical approach for the treatment of patients with locally advanced breast cancer.

This is a multicenter, prospective, randomized controlled investigator-initiated trial (IIT) aimed at evaluating the efficacy and safety of switching to mazdutide therapy in patients with type 2 diabetes mellitus (T2DM) and moderate-to-severe non-alcoholic fatty liver disease (NA…

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and efficacy of KER-065 administered to adult and pediatric ambulatory and nonambulatory male participants with Duchenne Muscular Dystrophy (DMD) on stable background …

The purpose of this study is to assess KarXT + KarX-EC for the treatment of irritability associated with autism in children and adolescents.