ANZCTR search results

This is a Phase 2, randomized, double-blind, placebo-controlled, multicenter, multinational, dose-finding study evaluating the efficacy of three treatment doses of CC-90001 compared with placebo, in Non-alcoholic Steatohepatitis (NASH) participants with Stage 2, Stage 3 liver fibrosis. This study is designed to assess response to treatment on measures of fibrosis and other efficacy parameters. It will also assess dose response and overall safety.

The primary objectives of this study were: to assess the safety and tolerability, to determine the maximum tolerated dose (MTD) or maximum administered dose (MAD) and to determine the recommended Phase 2 dose (RP2D) of BGB-A1217 (known as ociperlimab) in combination with tislelizumab in participants with advanced solid tumors in phase 1. Primary objective of Phase 1b was to assess overall response rate (ORR) determined by Investigator per Response Evaluation Criteria in Solid Tumors (RECIST) version (v).1.1 for patients in each dose-expansion cohort.

This study is to characterize the safety, tolerability, pharmacokinetics (PK), immunogenicity, pharmacodynamics (PD) and anti-tumor activity of AK112, a PD-1/VEGF bispecific antibody, as a single agent in adult subjects with advanced solid tumor malignancies. The study consists of a dose escalation phase (Phase 1a) to determine the maximum tolerated dose (MTD), or recommended Phase 2 dose (RP2D) for AK112 as a single agent, and a dose expansion phase (Phase 1b) in subjects with specific tumor types which will characterize treatment of AK112 as a single agent at the MTD or RP2D.

This is the first in human treatment with ST-920, a recombinant AAV2/6 vector encoding the cDNA for human a-Gal A. The purpose of this study is to evaluate the safety and tolerability of ascending doses of ST-920. ST-920 aims to provide stable, long-term production of a-Gal A at therapeutic levels in subjects with Fabry disease. The constant production of a-Gal A in humans should, importantly, enable reduction and potentially clearance of Fabry disease substrates Gb3 and lyso-Gb3. On Day 1, patients will be infused intravenously with a single dose of ST-920 and followed for a period of 52 weeks.

Primary Objectives: * To evaluate the safety and tolerability of isatuximab administered subcutaneously (SC) versus intravenously (IV) * To assess the safety and tolerability (including local injection site tolerability) of isatuximab using the (investigational) isatuximab injector device * To evaluate the pharmacokinetics (PK) of SC and IV isatuximab Secondary Objectives: * To estimate absolute bioavailability of SC and IV isatuximab * To measure receptor occupancy (RO) after isatuximab SC versus IV administration * To assess efficacy of isatuximab after SC and IV administration * To assess patient expectations prior to and patient experience and satisfaction after SC administration * To evaluate potential immunogenicity of SC or IV isatuximab

The purpose of this study was to evaluate efficacy of derazantinib monotherapy or derazantinib-atezolizumab in combination in patients with advanced urothelial cancer harboring fibroblast growth factor receptor (FGFR) genetic aberrations (GA) of various clinical stages of disease progression and prior treatments.

Current dosing regimens for vancomycin result in many young infants not reaching the target level of vancomycin in the blood at steady state (when the blood is in equilibrium at 24-48 hours).The purpose of this study is to assess an improved method of calculating the dose of vancomycin ('model-based dosing') in young infants with infections in order for them to achieve the target vancomycin level at steady state. A dosing calculator (which will be available through a web application) will be used for the dose calculation.

This study evaluated the long-term safety and tolerability of elexacaftor (ELX), tezacaftor (TEZ), and ivacaftor (IVA) triple combination (TC) treatment in participants with cystic fibrosis (CF).

The purpose of the study is to collect information on how Ryzodeg® works in real world patients and to see if Ryzodeg® can lower blood sugar levels. Participants will get Ryzodeg® as prescribed to them by their doctor. The study will last for about 6 to 9 months. Participants will be asked questions about their health and their diabetes treatment as part of their normal doctor's appointment.

The proposed study is designed to provide patients previously enrolled in Phase 1 and 2 studies of DCR-PHXC and their siblings (\<18 years old) long-term access to DCR-PHXC, and to evaluate the long-term safety and efficacy of DCR-PHXC in patients with PH.