ANZCTR search results

The primary objective of the study is to evaluate the long-term safety profile of GTX-102 in participants with Angelman Syndrome (AS)

This is a Phase II open-label, two-arm randomised non-comparative, multi-centre study to evaluate the efficacy of Epcor-only (Epcoritamab alone) or Epcor-R2 (Epcoritamab, lenalidomide and rituximab) as consolidation post anti-CD19 CAR T-cell therapy for patients that have responded by conventional criteria but who are at high risk of progression by virtue of being Minimal Residual Disease (MRD) positive as determined by a Circulating Tumour DNA (ctDNA) assay.

The purpose of this study is to measure the benefit of adding abemaciclib to the chemotherapy, temozolomide, for newly diagnosed high-grade glioma following radiotherapy. Your participation could last approximately 11 months and possibly longer depending upon how you and your tumor respond.

The goal of this study (iMMagine-3) is to compare the study drug, anitocabtagene autoleucel to standard of care therapy (SOCT) in participants with relapsed/refractory multiple myeloma who have received 1 to 3 prior lines of therapy, including an anti-CD38 monoclonal antibody and an immunomodulatory drug. The primary objective of this study is to compare the efficacy of anitocabtagene autoleucel versus SOCT in participants with RRMM.

Posttraumatic stress disorder (PTSD) is a common and debilitating mental illness. Current treatments for PTSD include psychotherapy and antidepressant medications. Many patients are unable to tolerate psychotherapy for PTSD and drop out of it. In addition, its effectiveness is limited. Up to 50 percent of patients who receive psychotherapy do not benefit from it. Antidepressant medications have only small benefits in PTSD. They also have unpleasant side effects that can make patients unwilling to take them. There is an urgent need to develop new treatments for PTSD that work and are well-tolerated. Silexan has the potential to provide an important alternative treatment for PTSD. Silexan is derived from lavender oil. It is taken orally in the form of capsules. It is currently available over-the-counter in 14 countries, including Australia and the United States. Previous research has shown that it is an effective treatment for anxiety disorders, including Generalized Anxiety Disorder. It is also well-tolerated by patients. The only side effects that have been identified so far are mild gastrointestinal symptoms (including burping and breath odour) and these are uncommon. The results of a small pilot study suggest that Silexan may also be effective and well-tolerated in PTSD. The STOP trial is a clinical trial that aims to investigate whether adding Silexan to treatment-as-usual improves PTSD symptoms in adults with PTSD. The trial will recruit 278 participants. Participants will be randomly assigned to take Silexan or a placebo (look-alike dummy pills) daily in addition to their usual medications for 12 weeks. The severity of their PTSD symptoms will be assessed prior to and at the end of this 12-week period. The STOP trial has the potential to obtain definitive evidence regarding whether Silexan helps treat symptoms of PTSD. If Silexan is found to be an effective treatment for PTSD, the pool of patients who could potentially benefit from this treatment includes any adults with PTSD. Silexan is already available over-the-counter at a relatively low cost so there will be few barriers to accessing this treatment.

Gastroparesis is a chronic and debilitating gastric disease associated with poor quality of life, psychological distress, frequent hospitalisations, and high healthcare utilization and associated costs. It is defined by persistent upper gastrointestinal symptoms and delayed gastric emptying with no mechanical gastric outlet obstruction. Gastric emptying scintigraphy (GES) is the current gold standard for diagnosing gastroparesis but its clinical utility is currently being questioned. Current management strategies have often been found to be ineffective, largely due to an incomplete understanding of the disease's pathophysiology. There is a critical need for more advanced diagnostic testing that can better diagnose patients and guide personalized targeted therapy. Body surface gastric mapping (BSGM) using Gastric Alimetry (Alimetry Ltd., New Zealand) is a new FDA-cleared medical device to assess gastric function by non-invasively assessing gastric motility using simultaneous high-resolution electrogastrography and symptom profiling. BSGM has demonstrated clinical utility in the assessment of gastric function through patient phenotyping in a variety of cohorts, including patients with nausea and vomiting disorders, diabetes, delayed gastric emptying, and post-gastric surgery. Previous research revealed that the detection of gastric motility abnormality rates through patient phenotyping were higher using Gastric Alimetry compared to GES (43% vs 23%). Clinical application of these phenotypes has also aided in changing management decisions, which reduced healthcare utilization and associated costs. However, how GES and BSGM test results differentially influence clinical management in patients is uncertain. This exploratory pilot study proposes a two-arm, prospective trial to assess whether BSGM-guided care could change clinical outcomes compared to the standard of care (GES) in patients with suspected gastroparesis. The trial consists of two phases. Phase 1 involves participants separately undertaking a GES and BSGM test. Based on these results, the referring clinician will devise management plans for treatment using a standardized form: 1) unblinded to one test (GES or BSGM) but blinded to the other test; and 2) unblinded to both tests (GES + BSGM). They will be asked to recommend any changes to interventions (medications, diet, endoscopic/surgical referral or other) and additional testing. In phase 2, those in Phase 1 will undergo BSGM-guided care based on their combined management plan (GES + BSGM) and followed up over a 12 month period. A separate set of participants will be recruited to undergo standard of care (GES only) in parallel with Phase 1 participants. After 12 months, those on the standard of care arm will be crossed over to BSGM-guided care, undergo a BSGM test, treated according to the new management plan, and followed up over 6 months. Questionnaires will assess symptoms, quality of life, health psychology, sleep, and work impact. If validated, this may change clinical practice by reducing the need for invasive or radioactive-based procedures to diagnose these patients and facilitating a more targeted treatment approach.

This is a long-term, multicenter, non-interventional study of children ages 2.5 to \<17 years with hypochondroplasia (HCH).

This is a prospective, open label, multicenter, single arm, first in human clinical study. Patients with infra-inguinal peripheral arterial disease appropriate for treatment with a femoro-popliteal stent will be enrolled. The patients will be treated with the ChampioNIR Stent System. All implanted patients will be followed up at 30 days and 6, 12, 24 and 36 months. The follow-up visits will include patency evaluation by duplex ultrasound

A multi-centre, prospective, blinded, randomised clinical trial of fludrocortisone compared with placebo in patients presenting with aneurysmal subarachnoid haemorrhage. The study aim is to determine if early administration of enteral fludrocortisone in aneurysmal subarachnoid haemorrhage reduce death and dependency at six months.

The study will look at the effects of NNC0194-0499, cagrilintide and semaglutide, on liver damage and alcohol use in participants with alcoholic liver disease. Participants will get NNC0194-0499, semaglutide, cagrilintide or ''dummy" medicine in different treatment combinations. Which treatment participants get is decided by chance. The study will last for about 39 weeks.