ANZCTR search results

The lysosomal storage disorders (LSDs) are monogenic disorders associated with inflammation affecting multiple organs, and early death. Few treatments are available that can modify the disease course, and there is an urgent need to identify new steps in pathogenesis that can be targeted therapeutically. The complement system is novel and highly plausible as a primary driver of inflammation and cellular injury in the LSDs. This study assesses the complement activation state in patients with Fabry disease (FD), Gaucher disease (GD) and Niemann-Pick disease, type C (NPC), with comparison to healthy controls. This has the potential for immense clinical benefit through targeted complement inhibition across the full spectrum of lysosomal storage disorders, in which key pathophysiological processes including the inflammatory response to lysosomally 'stored' materials are shared.

The reason for this study is to see if the study drug LY3484356 alone or in combination with other anticancer therapies is safe and effective in participants with advanced or metastatic breast cancer or endometrial cancer.

Study of NGM395 in adult participants.

The study will be conducted to evaluate the therapeutic response (combined per participant microbiological and clinical response) of oral gepotidacin compared to oral nitrofurantoin for treatment of uncomplicated UTI (acute cystitis) in adolescent and adult female participants.

This is a cohort-based, open-label dose escalation and expansion study in adults with advanced solid tumors or lymphoma, refractory or resistant to standard therapy, or without available standard or curative therapy.

This is a retrospective study evaluating the safety and performance of the MINITAC Suture anchors for extremities repair. Data will be used to support Notified Body (BSI) requirements for extremities repair indications.

To evaluate the safety and tolerability of sotorasib administered in investigational regimens in adult participants with KRAS p.G12C mutant advanced solid tumors.

The study evaluates the long-term safety, tolerability, efficacy, and pharmacodynamics of elexacaftor (ELX, VX-445) in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in participants with cystic fibrosis (CF).

This is a Phase I, open-label, two-part in healthy adult males. There will be up to 12 subjects with 6 subjects in each part of the study. Subjects from Part A are eligible to participate in Part B. For Part A, each of the 6 subjects will complete two periods of the study with washout period of 7 days between. Each subject during participation in the study will receive a dose of PXS 5382A orally in a fed state and a fasted state. For Part B, repeated oral BID administration of PXS-5382A will be performed in the Fed state and dose will be dependent on analysis of Part A. In both part A and B PK, PD and safety assessments will be collected.

The purpose of this study is to compare the efficacy of ciltacabtagene autoleucel (cilta-cel) with standard therapy, either Pomalidomide, Bortezomib and Dexamethasone (PVd) or Daratumumab, Pomalidomide and Dexamethasone (DPd).