ANZCTR search results

This is a long-term follow-up safety and efficacy study of participants in clinical trials for spinal muscular atrophy (SMA) who were treated with onasemnogene abeparvovec-xioi. Participants will roll over from their respective previous (parent) study into this long-term study for continuous monitoring of safety as well as monitoring of continued efficacy and durability of response to onasemnogene abeparvovec-xioi treatment.

The aim of this clinical investigation is to collect data on objective and subjective hearing performance, quality of life and safety in adult subjects with conductive hearing loss, mixed hearing loss or single-sided sensorineural deafness.

This is a Phase II, Randomized, Blinded, Sham Procedure-Controlled, Parallel-Group Trial to Evaluate the Efficacy, Safety and Tolerability of LYR-210 in Adult Subjects with Chronic Sinusitis.

SN1011 (the study drug), is currently being developed by Sinomab as a new drug for treating autoimmune disease (diseases occurring when your body's natural immune/defence mechanism attacks healthy tissue and nerves), such as rheumatoid arthritis (RA). RA causes recurrent joint pain and swelling, particularly in the hands and feet, and can lead to bone erosion and joint deformity. SN1011 is known as a BTK inhibitor. Bruton's tyrosine kinase (BTK) is an enzyme that plays a key role in B-cell development, and B-cells play an important role in immunity throughout the body. It is thought that blocking the BTK signal may inhibit disease progression in people with RA and may even resolve the disease. The purpose of this research study is to assess the safety and tolerability of SN1011 as well as the pharmacokinetics (PK - how your body handles the study drug) and pharmacodynamics (PD - how the study drug affects your body) of the study drug. The investigators are doing this study in healthy men and women.

XNW3009 is a small molecule hURAT1 inhibitor developed independently by Sinovent Pty Ltd., and is intended to treat gout-related hyperuricemia. Uricosuric drugs increase urinary uric acid excretion by blocking renal tubular reabsorption of urate. The human urate transporter 1( hURAT1) is responsible for the majority of the reabsorption of filtered urate, and the mutations in the hURAT1 gene have been demonstrated to be responsible for urate non-homeostasis. This is a randomized, double-blind, placebo-controlled, dose-escalation study to investigate the safety, tolerability, PK and PD of XNW3009 after administration of single (Part A) and multiple (Part B) oral doses in healthy adult subjects. Approximately six sequential dose panels (single oral doses of 1, 5, 10, 20, 35 and 50 mg XNW3009) will be evaluated in SAD and approximately three sequential dose panels (ten consecutive days for respectively daily oral doses of 10, 20,35 mg, QD) will be evaluated in MAD.

This study will evaluate crisaborole therapy once daily (QD) as a long-term topical maintenance therapy for the reduction of flare in responders to crisaborole twice daily (BID) treatment.

The study is designed to be able to prove if the Molemap Artificial Intelligence (AI) algorithm can be used as a diagnostic aid in a clinical setting. This study will determine whether the diagnostic accuracy of the Molemap AI algorithm is comparable to a specialist dermatologist, teledermatologist and registrar (as a surrogate for a general practitioner). The study patient population will be adult patients who require skin cancer assessment. The use of AI as a diagnostic aid may assist primary care physicians who have variable skill in skin cancer diagnosis and lead to more appropriate referrals (rapid referral for lesions requiring treatment and fewer referrals for benign lesions), thereby improving access and reducing waiting times for specialist care.

The main purpose of this study is to compare the overall survival (OS) of nivolumab plus ipilimumab versus standard of care (SOC) (sorafenib or lenvatinib) in all randomized participants with advanced hepatocellular carcinoma (HCC) who have not received prior systemic therapy.

This is a Phase 2 randomized, dose-blind, multicenter study designed to evaluate the safety, tolerability, efficacy, Pharmacokinetics (PK), and Pharmacodynamics (PD) of treatment with KZR-616 in patients with active Autoimmune Hemolytic Anemia or Immune Thrombocytopenia.

The main purpose of this study is to characterize the long-term safety and efficacy of the drug Deucravacitinib (BMS-986165) in patients who have been previously enrolled in an applicable Phase 3 psoriasis study.