ANZCTR search results

This is a multicenter, randomized, double-blind, controlled Phase 2/3 trial of zanzalintinib in combination with pembrolizumab versus zanzalintinib-matched placebo in combination with pembrolizumab in subjects with programmed death-ligand 1 (PD-L1) positive recurrent or metastatic head and neck squamous cell carcinoma (R/M HNSCC) incurable by local therapies who have not received prior systemic therapy for recurrent or metastatic disease.

FOCUS is a dyadic, psychoeducational intervention developed in the USA, shown to improve the wellbeing and quality of life (QoL) of patients with advanced cancer and their primary family carers. The intervention consists of five core components underpinning the FOCUS acronym: (F) supporting Family involvement, (O) supporting Outlook and meaning, (C) increasing Coping effectiveness, (U) reducing Uncertainty, and (S) Symptom management. Originally a nurse-delivered in-person intervention, FOCUS has been translated into a self-administered web-based intervention as part of an European study. The overall aim of this project is to determine the effectiveness and sustainability of a digital health intervention (FOCUSau) aimed at improving the wellbeing and self-efficacy of patients with advanced cancer and their primary support person/carer. A primary support person/carer is an unpaid individual identified by the person with advanced cancer (not necessarily a partner or family member) who is providing them with physical, social or emotional support. Hereafter referred to as a "carer". The term "dyad" refers to the patient and primary support person/carer. The project objectives are: 1. adapt FOCUS to the Australian context and develop FOCUSau; 2. examine the effectiveness of FOCUSau in improving the wellbeing (primary outcomes: QoL and self-efficacy) of patients with advanced cancer and their primary family carer; 3. compare the type and costs of health service use by participants in the intervention and control group; and 4. assess the acceptability, feasibility and scalability of FOCUSau in order to inform sustainable implementation of the intervention within the Australian health care system. A pragmatic phase III hybrid effectiveness-implementation trial with an integrated research design that includes digital health evaluation will be used in patients with advanced cancer and their primary support person/carer. Data will be collected three times from patient-carer dyads: 1. at baseline (T0) after which the dyad will immediately be randomised to one of the study arms, 2. first follow-up at 12 weeks after baseline (T1) and, 3. second follow-up at 24 weeks after baseline (T2).

This double-blind, randomized, placebo-controlled, multinational, multicenter, parallel-group, Phase 3, 2-arm, study will investigate the efficacy and safety of belumosudil compared with placebo, both administered on top of azithromycin and standard-of-care regimen of immunosuppression in male or female participants at least 1 year after bilateral lung transplant, who are at least 18 years of age and who have evidence of progressive CLAD despite azithromycin therapy. Study details include: The study duration will be up to 31 weeks for participants not entering the open-label extension (OLE) period and up to 57 weeks for participants entering the OLE period but not the long-term OLE. The treatment duration will be up to 26 weeks for participants not entering the OLE period and up to 52 weeks for participants entering the OLE period but not the long-term OLE. The number of visits will be up to 10 visits for participants not entering the OLE period and up to 16 visits for participants entering the OLE period but not the long-term OLE. For participants who enter the long-term OLE, treatment and study participation will continue with visits every 12 weeks per protocol specifications.

This clinical trial will study the effects of aficamten (versus placebo) on the quality of life, exercise capacity, and clinical outcomes of patients with non-obstructive hypertrophic cardiomyopathy.

This is a randomized, double-blind, placebo-controlled phase I study to evaluate the safety, tolerability and PK profiles of orally administered VG290131 in healthy subjects. The main questions it aims to answer are: 1. The safety and tolerability of VG290131 when administered orally as a single dose and multiple doses in healthy subjects. 2. The pharmacokinetic (PK) profiles of VG290131 and the food effect on the PK profiles of VG290131 in healthy subjects. Approximately 86 subjects will be enrolled in the study.

This is a study evaluating the safety and efficacy of bomedemstat (MK-3543) compared with the best available therapy (BAT) in participants with essential thrombocythemia (ET) who have an inadequate response to or are intolerant of hydroxyurea. The primary study hypothesis is that bomedemstat is superior to the best available therapy with respect to durable clinicohematologic response (DCHR).

A Phase 1, Open-Label, Drug-drug Interaction, and Randomized, Double-blind, Controlled, Multiple-dose Pharmacokinetics and Safety Study of Xeruborbactam Oral Prodrug (QPX7831) in Combination with Ceftibuten in Healthy Adult Participants

This trial is a Phase 2, multicenter, double-blind, randomized (ratio 2:1 TransCon CNP vs. placebo), placebo-controlled trial, designed to evaluate the safety, tolerability, and efficacy of 100 µg CNP/kg of Navepegritide (TransCon CNP) administered SC once-weekly for 52 weeks in infants with genetically verified heterozygous ACH, aged 0 to \< 2 years at the time of randomization.

The primary purpose of this study is to assess the safety and tolerability of ALXN1850 versus asfotase alfa in pediatric participants with HPP previously treated with asfotase alfa.

The primary purpose of this study is to evaluate the efficacy of ALXN1850 versus placebo on radiographic outcomes in pediatric participants with HPP who have not previously been treated with asfotase alfa.