ANZCTR search results

The purpose of this study is to evaluate the efficacy of guselkumab in pediatric participants with moderately to severely active ulcerative colitis at the end of maintenance therapy among participants who were induction responders.

The purpose of this study is to find out whether an early three-day course of an oral steroid medication (dexamethasone) can improve the physical and mental recovery and wellbeing for children with Sydenham's chorea. Sydenham's chorea is a condition that impacts approximately 12% of children with acute rheumatic fever. It is caused by inflammation in the brain following an abnormal immune response to Group A streptococcus bacterial infection. Sydenham's chorea is a movement disorder that causes children's faces, hands, and feet to move quickly and uncontrollably, and can also affect mood and concentration. The physical recovery from Sydenham's chorea can take two to six months but the mental recovery (e.g. mood and concentration) can take longer to resolve. Sydenham's chorea remains endemic in Maori, Pacific Islander, Aboriginal and Torres Strait Islander children in New Zealand and Australia. There is limited evidence to direct treatment of Sydenham's chorea, and clinical practice differs widely around the world. Dexamethasone is an oral steroid which targets the abnormal immune response and successfully treats other immune-mediated brain disorders, with good tolerability. TREAT-SC is a randomized, double-blinded, placebo-controlled trial which will investigate whether a three day course of oral dexamethasone safely and effectively treats the movement disorder and psychiatric symptoms of Sydenham's chorea. The trial will recruit 80 participants from study sites in Australia and New Zealand.

Ulcerative colitis (UC) is a type of inflammatory bowel disease that causes inflammation and bleeding from the lining of the rectum and colon (large intestine). The purpose of this study is to assess how safe and effective lutikizumab is in adult participants with moderate to severe UC and how lutikizumab compares to adalimumab in the treatment of UC. Adverse events and changes in disease activity will be assessed. Lutikizumab is an investigational product being developed for the treatment of moderate to severe UC. Participants are placed in groups called treatment arms. Each group receives a different treatment. In the Induction Period, participants will be randomized into 1 of 3 arms receiving lutikizumab Dose 1, lutikizumab Dose 2, or adalimumab. In the Maintenance Period, participants who responded to lutikizumab will be randomized into 1 of 2 arms of lutikizumab maintenance and participants who responded to adalimumab will continue to receive adalimumab. All participants who did not achieve clinical response per modified Mayo Score at the end of the Induction period will receive lutikizumab. Around 200 adult participants with UC will be enrolled at approximately 200 sites worldwide. During the 12 week Induction Period, participants will be randomized to receive intravenous (IV) and subcutaneous (SC) lutikizumab or SC adalimumab. At the 12 week mark, participants who are on lutikizumab who have responded to treatment will be re-randomized to receive SC lutikizumab at different intervals until Week 52. Participants who are on adalimumab who are responding to treatment will continue to receive adalimumab. Participants who do not respond to treatment will receive SC lutikizumab. Participants who complete the Week 52 visit and in whom therapeutic benefit to study drug is confirmed by the investigator may roll over into an optional, 52-week long-term extension (LTE). There may be higher treatment burden for participants in this trial compared to their standard of care. Participants will attend regular visits during the study at a hospital or clinic. The effect of the treatment will be checked by medical assessments, blood tests, checking for side effects and completing questionnaires.

Transmural healing (TMH) is recognized as a potentially important measure of Crohn's disease (CD) activity but not a formal target. Observational studies suggest that TMH may be associated with better long-term outcomes. The study will evaluate TMH using noninvasive intestinal ultrasound (IUS), a patient-friendly technique that can be performed routinely in clinical practice. The aim of the study is to determine if treating to a target of corticosteroid-free (CS-free) IUS outcomes + clinical symptoms + biomarkers is superior to a target of clinical symptoms + biomarkers alone in achieving CS-free endoscopic remission measured by the Simple Endoscopic Score for Crohn's Disease (SES-CD). Qualified participants will be randomly assigned in a 1:1 ratio to one of 2 different target treatment groups. Group 1: Participants will be treated over 48 weeks to achieve a target of corticosteroid-free IUS-based outcomes + clinical remission + biomarker remission. At Week 22 and 30, the IUS-based component of the target will be IUS response and at Week 38, the final treatment target will be TMH. Group 2: Participants will be treated over 48 weeks to achieve a target of corticosteroid-free clinical remission + biomarker remission.

This study is a first-in-human (FIH), Phase 1a/1b study of BG-68501, a cyclin-dependent kinase-2 inhibitor (CDK2i), to assess the safety, tolerability, pharmacokinetics (PK), pharmacodynamics, and preliminary antitumor activity of BG-68501 in participants with advanced, nonresectable, or metastatic solid tumors as monotherapy and in combination with fulvestrant with or without BGB-43395, a selective CDK4 inhibitor, in adults with hormone receptor positive (HR+)/human epidermal growth factor receptor 2 negative (HER2-) breast cancer (BC). The study will also identify a recommended dose for expansion (RDFE) for BG-68501 as monotherapy and in combination for subsequent disease directed studies. The study will be conducted in 2 parts: Part 1 (dose escalation and safety expansion, including evaluation of food effect) and Part 2 (dose expansion).

The goal of this study is to assess the safety and effectiveness of Dostarlimab compared to Placebo in adult participants with Head and Neck Squamous Cell Carcinoma (HNSCC)

The purpose of this study is to evaluate the safety and preliminary efficacy of ATA3219 in participants with relapsed/refractory (R/R) B-cell non-Hodgkin Lymphoma (NHL).

Ornithine Transcarbamylase (OTC) deficiency, the most common urea cycle disorder, is an inherited metabolic disorder caused by a genetic defect in a liver enzyme responsible for detoxification of ammonia. Individuals with OTC deficiency can build-up excess levels of ammonia in their blood, potentially resulting in devastating consequences, including cumulative and irreversible neurological damage, coma and death. The severe form of the condition emerges shortly after birth and is more common in boys than girls. This is a Phase 1/2/3, open-label, multicenter, safety, efficacy, and dose finding study of ECUR-506 in male babies with neonatal onset OTC deficiency. The primary objective of this study is to evaluate the safety, tolerability, and efficacy of up to three dose levels of ECUR-506 following intravenous (IV) administration of a single dose.

The main aim of this study is to learn if TAK-279 reduces bowel inflammation and symptoms compared to placebo. Another aim is to compare any medical problems that participants have when they take TAK-279 or placebo and how well the participants tolerate any problems. The participants will take capsules of either TAK-279 or placebo for up to 3 months (12 weeks). Then all the participants will receive TAK-279 for the rest of the treatment part of the study (1 year or 52 weeks). During the study, participants will visit their study clinic several times.

The primary goal of this study is to evaluate the long-term safety and pharmacodynamic effects of votoplam in participants with HD.