Latest studies
- Active not recruitingPHASE2
A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With Achondroplasia
This study is testing a medicine called vosoritide for babies up to one year old with achondroplasia. We want to see if it's safe and helps improve a tricky neck problem often seen in achondroplasia, especially in babies who might need surgery.
AchondroplasiaAustralia - Recruiting
Atypical Hemolytic-Uremic Syndrome (aHUS) Registry
This study is gathering important information about a rare kidney disease called atypical Hemolytic-Uremic Syndrome (aHUS). It looks at how the disease progresses and how safe new treatments like eculizumab and ravulizumab are for patients of all ages.
Atypical Hemolytic-Uremic SyndromeAustralia - Active not recruitingPHASE3
A Study of Zanidatamab in Combination With Chemotherapy Plus or Minus Tislelizumab in Patients With HER2-positive Advanced or Metastatic Gastric and Esophageal Cancers
This study looks at a new drug, zanidatamab, combined with chemotherapy, and sometimes another drug called tislelizumab. It aims to see if this treatment works better and is safer than standard chemotherapy for advanced stomach and gullet cancers that have HER2.
Gastric NeoplasmsGastroesophageal AdenocarcinomaEsophageal AdenocarcinomaAustralia - RecruitingPHASE3
Maridebart Cafraglutide in Heart Failure With Preserved or Mildly Reduced Ejection Fraction and Obesity
This study looks at a new medicine, Maridebart cafraglutide, for people with heart failure and obesity. It aims to see if the medicine can reduce heart-related problems like hospital stays, improve symptoms, and help people live longer, alongside their usual heart failure care.
Heart Failure With Preserved Ejection FractionHeart Failure With Mildly Reduced Ejection FractionObesityAustralia - Active not recruitingPHASE1
A Study of XL092 as Single-Agent and Combination Therapy in Subjects With Solid Tumors
This study looks at a new drug called XL092 for advanced solid tumours like kidney, breast, prostate, or bowel cancer. Researchers are testing it alone and with other drugs (Atezolizumab, Avelumab) to see if it's safe, how it works, and if it can help slow down or shrink tumours.
Neoplasm MalignantRenal Cell CarcinomaHormone Receptor Positive Breast CarcinomaMetastatic Castration-resistant Prostate CancerAustralia - RecruitingPHASE1, PHASE2
A Phase I/II Study of AZD4512 Monotherapy or in Combination With Anticancer Agents in Participants With Relapsed/Refractory B-cell Non-Hodgkin Lymphoma
This study is testing a new medicine, AZD4512, for people with a type of blood cancer called B-cell non-Hodgkin lymphoma that has come back or not responded to past treatments. It looks at how safe and effective the medicine is, sometimes on its own, and sometimes with other cancer drugs.
B-cell Non-Hodgkin LymphomaAustralia - RecruitingPHASE2
A Study to Investigate the Safety and Efficacy of GSK4532990 Compared With Placebo in Adult Participants Aged 18 to 70 Years With Alcohol-related Liver Disease
This study is testing a new medicine, GSK4532990, for people aged 18-70 with liver damage caused by alcohol. We want to see if it's safe and if it helps the liver. Some people will get the medicine, others a dummy pill.
Liver Diseases, AlcoholicAustralia - Active not recruitingPHASE3
Quizartinib or Placebo Plus Chemotherapy in Newly Diagnosed Patients With FLT3-ITD Negative AML
This study is looking at a new drug, quizartinib, combined with standard chemotherapy for adults recently diagnosed with a specific type of leukaemia called AML. We want to see if this combination improves treatment outcomes compared to chemotherapy alone.
LeukemiaAustralia - Recruiting
Lysosomal Acid Lipase (LAL) Deficiency Registry
This study is gathering information on a rare genetic condition called LAL Deficiency. It aims to understand the disease better and improve care for affected individuals, from babies to adults. It's a worldwide effort to collect health data over time.
Lysosomal Acid Lipase DeficiencyCholesterol Ester Storage DiseaseWolman DiseaseAcid Cholesteryl Ester Hydrolase Deficiency, Type 2Australia - RecruitingPHASE2
Imeroprubart in Adult Participants With Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)
This study is testing a new medicine, Imeroprubart, for adults with a nerve condition called Chronic Inflammatory Demyelinating Polyneuropathy (CIDP). Researchers want to see if it's safe and helps improve symptoms compared to a dummy treatment.
Chronic Inflammatory Demyelinating PolyneuropathyAustralia - Active not recruitingPHASE2
Phase 2 Safety and Efficacy Study of Tulisokibart (MK-7240/PRA023) in Subjects With Systemic Sclerosis Associated With Interstitial Lung Disease (SSc-ILD) (MK-7240-007)
This study looks at a new drug, tulisokibart, for people with a lung condition called interstitial lung disease (ILD) linked to scleroderma. We want to see if it's safe and helps improve the condition.
Diffuse Cutaneous Systemic SclerosisInterstitial Lung DiseaseAustralia - Active not recruitingPHASE3
Efficacy and Safety of Selumetinib in Adults With NF1 Who Have Symptomatic, Inoperable Plexiform Neurofibromas
This study is testing a new medicine, selumetinib, for adults with a condition called Neurofibromatosis Type 1 (NF1). It focuses on people who have painful, non-removable growths called plexiform neurofibromas. We want to see if this medicine can help reduce symptoms and safely shrink these growths.
Neurofibromatosis 1Plexiform Neurofibroma (PN)Australia - Active not recruitingPHASE3
A Study to Compare Chemotherapy Alone Versus Chemotherapy Plus Nivolumab or Nivolumab and BMS-986205, Followed by Continued Therapy After Surgery With Nivolumab or Nivolumab and BMS-986205 in Participants With Muscle Invasive Bladder Cancer
This study looks at treating muscle-invasive bladder cancer. It compares standard chemotherapy with chemotherapy plus a new immunotherapy drug called nivolumab, given before and after surgery. The aim is to see if adding nivolumab helps more people.
Urinary Bladder NeoplasmsMuscle-Invasive Bladder CancerAustralia - Active not recruitingPHASE3
Long-term Safety and Efficacy of Efanesoctocog Alfa (BIVV001) in Previously Treated Patients With Hemophilia A
This study is looking at a new medicine called efanesoctocog alfa (BIVV001) for people with haemophilia A. It aims to see if it's safe and effective over a long time in preventing and treating bleeds, improving joint health and quality of life.
Hemophilia AAustralia - Active not recruitingPHASE3
An Efficacy and Safety Study of Luspatercept (ACE-536) Versus Placebo in Subjects With Myeloproliferative Neoplasm-Associated Myelofibrosis on Concomitant JAK2 Inhibitor Therapy and Who Require Red Blood Cell Transfusions
This study looks at a new drug, Luspatercept, for people with myelofibrosis and anaemia who need regular blood transfusions and are already taking a JAK2 inhibitor. We want to see if it helps reduce the need for transfusions.
Myeloproliferative DisordersMyelofibrosisPrimary MyelofibrosisPost-Polycythemia Vera MyelofibrosisAustralia - Active not recruitingPHASE2
A Study of the Safety and Tolerability in Participants With PIK3CA-related Overgrowth Spectrum or Proteus Syndrome Who Are Being Treated With Miransertib (MK-7075) in Other Studies (MK-7075-006)
This study looks at how safe and well a medicine called Miransertib is for children and adults with certain growth conditions (PROS or Proteus Syndrome). It's for people already in other Miransertib studies or about to start, exploring long-term effects.
PIK3CA-Related Overgrowth Spectrum (PROS)/Proteus Syndrome (PS)Australia - Recruiting
Real-World Study to Assess Subcutaneous Epcoritamab in Adult Participants With Diffuse Large B-Cell and Follicular Lymphoma
This study looks at how a new drug called epcoritamab works for adults with Diffuse Large B-cell Lymphoma (DLBCL) or Follicular Lymphoma (FL) in real-life settings. It aims to see how well it treats these blood cancers when given as part of regular care.
Diffuse Large B-cell LymphomaFollicular LymphomaAustralia - Available
Expanded Access Program of Neladalkib (NVL-655) for Patients With Advanced ALK+ NSCLC or Other ALK+ Solid Tumors
This program offers people with advanced ALK-positive lung cancer or other ALK-positive tumours an opportunity to receive an investigational medicine called neladalkib (NVL-655). It is for those who can't join a regular trial and have run out of other good treatment options.
Non Small Cell Lung CancerALK-positive Non-small Cell Lung Cancer (NSCLC)Australia - RecruitingPHASE3
Extension Study for the Port Delivery System With Ranibizumab (Portal)
This study looks at a long-term eye implant to treat 'wet' age-related macular degeneration (nAMD). It checks the safety of the implant and how well it works over time in people who have been in previous studies. It also explores ways to manage potential side effects.
Neovascular Age-Related Macular DegenerationAustralia - Active not recruitingPHASE3
Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia
This study is testing a new medicine, vosoritide, for children aged 3 to 17 with hypochondroplasia (a type of short stature). It will compare vosoritide to a dummy medicine (placebo) to see if it helps children grow taller over 52 weeks.
HypochondroplasiaAustralia - RecruitingPHASE3
Randomized, Embedded, Multifactorial Adaptive Platform Trial for Community- Acquired Pneumonia
This study, called REMAP-CAP, looks at different treatments for serious lung infections like pneumonia, flu, and COVID-19 in adults needing intensive care. It aims to find out which treatments work best to help patients recover.
Community-acquired Pneumonia, Influenza, COVID-19Australia - Active not recruitingPHASE2
A Study of TAR-200 in Combination With Cetrelimab, TAR-200 Alone, or Cetrelimab Alone in Participants With Non-Muscle Invasive Bladder Cancer (NMIBC) Unresponsive to Intravesical Bacillus Calmette-Guérin Who Are Ineligible for or Elected Not to Undergo Radical Cystectomy
This study looks at new ways to treat a type of bladder cancer that hasn't responded to usual treatments. It tests two medicines, TAR-200 and cetrelimab, alone or together, for people who can't or don't want major surgery.
Urinary Bladder NeoplasmsAustralia - RecruitingPHASE2
WISPer: Evaluation of MTX-463 in Participants With Idiopathic Pulmonary Fibrosis (IPF)
This study, called WISPer, is testing a new medicine called MTX-463 for Idiopathic Pulmonary Fibrosis (IPF). It's comparing MTX-463 to a dummy medicine (placebo) to see if it's safe and effective for people with this lung condition.
Idiopathic Pulmonary FibrosisAustralia - Active not recruitingPHASE3
A Study of Nonsteroidal Aromatase Inhibitors Plus Abemaciclib (LY2835219) in Postmenopausal Women With Breast Cancer
This study looks at a new combination of medicines for postmenopausal women with a certain type of breast cancer. It tests if abemaciclib with standard hormone therapy works better than hormone therapy alone.
Breast CancerAustralia - RecruitingPHASE2
A Study of Elritercept Alone or Together With Ruxolitinib in Adults With Myelofibrosis
This study looks at a new medicine called Elritercept for myelofibrosis, a bone marrow condition. Researchers want to see if it's safe and effective when used alone or with another medicine called Ruxolitinib. They're also checking how it affects symptoms and red blood cell counts.
MyelofibrosisAustralia - Active not recruitingPHASE3
HAELO: A Phase 3 Study to Evaluate NTLA-2002 in Participants With Hereditary Angioedema (HAE)
This study is testing a new medicine, NTLA-2002, for people with Hereditary Angioedema (HAE). We want to see how well it works and if it's safe compared to a placebo (a dummy medicine) to reduce HAE attacks.
Hereditary AngioedemaAustralia - RecruitingPHASE2
A Clinical Study of Calderasib (MK-1084) in People With Advanced Solid Tumors (MK-1084-014)
This study is looking into a new medicine, calderasib, to treat certain advanced cancers. Researchers want to see if it can shrink these cancers, either alone or with another medicine called cetuximab, in people whose cancers have a specific genetic change (KRAS G12C mutation).
Neoplasm MalignantAustralia - Active not recruitingNA
Ryme Medical TLD Pilot Study
This study is looking at a new treatment called Targeted Lung Denervation (TLD) for people with a lung condition called COPD. It wants to see if the TLD treatment, using the Ryme Medical system, is safe and works well. This is a pilot study, meaning it's an early look at the treatment.
Chronic Obstructive Pulmonary DiseaseAustralia - Active not recruitingPHASE3
Study Comparing Blinatumomab Alternating With Low-intensity Chemotherapy Versus Standard of Care Chemotherapy for Older Adults With Newly Diagnosed Philadelphia-negative B-cell Precursor Acute Lymphoblastic Leukemia
This study looks at a new way to treat a type of leukaemia (blood cancer) called B-cell precursor acute lymphoblastic leukaemia (ALL) in older adults. It compares a drug called Blinatumomab combined with gentler chemotherapy against the usual chemotherapy treatment to see which works best.
Newly Diagnosed Philadelphia (Ph)-Negative B-cell Precursor Acute Lymphoblastic Leukemia (ALL)Australia - RecruitingPHASE3
A Study to Evaluate the Efficacy of Pumitamig Versus Pembrolizumab in Participants With Previously Untreated Advanced Non-Small Cell Lung Cancer and PD-L1 ≥ 50%. (ROSETTA Lung-202)
This study looks at a new treatment called Pumitamig for advanced non-small cell lung cancer that hasn't been treated before. It compares Pumitamig to an existing treatment, Pembrolizumab, in people whose cancer cells have a high level of a specific marker called PD-L1.
Non-Small Cell Lung Cancer (NSCLC)Australia - Recruiting
Patient Retrospective Outcomes (PRO)
This study looks back at anonymous medical records of adults with long-lasting pain. It compares how well different treatments worked, including those from Boston Scientific, to help doctors understand what works best for patients.
Chronic PainAustralia - RecruitingPHASE3
A Study Evaluating Efruxifermin in Subjects With Non-Cirrhotic Nonalcoholic Steatohepatitis (NASH)/Metabolic Dysfunction-Associated Steatohepatitis (MASH) and Fibrosis
This study is looking into a new medication called efruxifermin for people with a kind of liver disease called NASH/MASH and scarring (fibrosis). It's a large study where some people get the medication, and others get a dummy treatment, to see if it's safe and helps improve the liver condition.
NASH With FibrosisMASH With FibrosisAustralia - RecruitingPHASE2, PHASE3
(HARBOR) Study to Evaluate Efficacy and Safety of BLU-263 Versus Placebo in Patients With Indolent Systemic Mastocytosis
This study is testing a new medicine, elenestinib, for a rare blood condition called indolent systemic mastocytosis. It compares the new medicine to a dummy pill (placebo) to see if it helps reduce symptoms that current treatments aren't fully controlling.
Indolent Systemic MastocytosisSmoldering Systemic MastocytosisAustralia - Active not recruitingPHASE3
A Study to Evaluate Mezigdomide in Combination With Carfilzomib and Dexamethasone (MeziKD) Versus Carfilzomib and Dexamethasone (Kd) in Participants With Relapsed or Refractory Multiple Myeloma (SUCCESSOR-2)
This study is looking into a new combination of medicines for people in the UK with multiple myeloma that has come back or not responded to previous treatments. It compares a new medicine combination with a standard one to see if the new option works better.
Relapsed or Refractory Multiple MyelomaAustralia - RecruitingPHASE3
Study of Casdatifan and Cabozantinib Versus Placebo and Cabozantinib in Patients With Advanced Clear Cell Renal Cell Carcinoma
This study is looking at a new drug, casdatifan, combined with an existing one, cabozantinib, for advanced clear cell kidney cancer. It compares this combination to cabozantinib with a dummy drug (placebo) to see if it stops cancer growth for longer in patients who've already had immunotherapy.
Metastatic Clear Cell Renal Cell CarcinomaAdvanced Clear Cell Renal Cell CarcinomaAustralia - RecruitingPHASE2, PHASE3
Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia
This study is looking for children with achondroplasia, a type of dwarfism, who haven't had prior growth treatment. It compares a new medicine called BMN 333 with an existing one, Vosoritide, to see how well they help children grow and if they are safe.
AchondroplasiaAustralia - Active not recruitingPHASE2
Phase 2 Study of EDG-5506 in Becker Muscular Dystrophy (GRAND CANYON)
This study is testing a new medicine called sevasemten (also known as EDG-5506) for adults with Becker muscular dystrophy. It wants to see if the medicine is safe and if it helps people. Early parts of the study are now full, but one large part, GRAND CANYON, has also finished taking new participants.
Becker Muscular DystrophyAustralia - RecruitingPHASE3
A Study of Adagrasib Plus Pembrolizumab Plus Chemotherapy vs. Placebo Plus Pembrolizumab Plus Chemotherapy in Participants With Previously Untreated Non-squamous Non-small Cell Lung Cancer With KRAS G12C Mutation (KRYSTAL-4)
This study looks at a new combination of drugs, adagrasib, pembrolizumab, and chemotherapy, for people with a specific type of advanced non-small cell lung cancer (NSCLC) that hasn't been treated before and has a KRAS G12C gene change.
Carcinoma, Non-Small-Cell LungAustralia - RecruitingPHASE1
A Study to Investigate APL-4098 Alone and in Combination in Adults With AML or MDS
This study is testing a new drug, APL-4098, alone and with other medicines, for adults with certain types of blood cancers like Acute Myeloid Leukaemia (AML) and Myelodysplastic Syndrome (MDS). Doctors want to see if it's safe, how well it's tolerated, and if it helps.
Acute Myeloid Leukemia RefractoryMyelodysplastic Syndrome Acute Myeloid LeukemiaMyelodysplastic Syndrome With Excess BlastsAcute Myeloid Leukemia, in RelapseAustralia - RecruitingPHASE3
Study Comparing AAA817+ARPI Versus Standard of Care in Adult Participants With PSMA-positive mCRPC
This study is looking at a new treatment called AAA817, combined with standard prostate cancer drugs, for men with advanced prostate cancer. It compares this new combination to existing treatments to see which works best at slowing the cancer's growth.
Prostate CancerAustralia - Active not recruitingPHASE3
Phase 3 Study of MRTX849 With Cetuximab vs Chemotherapy in Patients With Advanced Colorectal Cancer With KRAS G12C Mutation (KRYSTAL-10)
This study, KRYSTAL-10, is for people with advanced bowel cancer that has a specific genetic change (KRAS G12C). It compares a combination of new drugs, MRTX849 and cetuximab, against standard chemotherapy to see which works better for those who have already had initial treatment.
Advanced Colorectal CancerMetastatic Colorectal CancerAustralia - RecruitingPHASE1
A Study of VERVE-102 in Patients With Familial Hypercholesterolemia or Premature Coronary Artery Disease
This study is testing a new gene-editing treatment called VERVE-102 for people with high cholesterol due to a genetic condition (Familial Hypercholesterolemia) or early heart disease. It aims to see if VERVE-102 is safe and can help lower 'bad' cholesterol.
Heterozygous Familial HypercholesterolemiaPremature Coronary Heart DiseaseAustralia - RecruitingPHASE3
A Study to Evaluate Pumitamig Versus Durvalumab Following Concurrent Chemoradiation Therapy in Participants With Unresectable Stage III Non-small Cell Lung Cancer (NSCLC) (ROSETTA Lung-201)
This study is looking at two different treatments, pumitamig and durvalumab, for people with a type of lung cancer called non-small cell lung cancer (NSCLC) that can't be removed by surgery. It's for patients who have already had chemotherapy and radiation.
Non-small Cell Lung Cancer (NSCLC)Australia - RecruitingPHASE3
A Study to Compare the Efficacy of Nivolumab and Relatlimab Plus Chemotherapy vs Pembrolizumab Plus Chemotherapy for Stage IV/Recurrent Non-squamous Non-small Cell Lung Cancer With PD-L1 Expression ≥ 1%
This study compares new ways to treat advanced lung cancer. It looks at two drug combinations, Nivolumab and Relatlimab plus chemotherapy, versus Pembrolizumab plus chemotherapy. The goal is to find the best treatment for a specific type of lung cancer.
Non-small Cell Lung CancerAustralia - RecruitingPHASE3
A Study to Evaluate the Safety and Efficacy of Tacabrutideg (BGB-16673) Compared to Pirtobrutinib in Adults With Relapsed/Refractory Chronic Lymphocytic Leukemia or Small Lymphocytic Lymphoma
This study compares two medicines, BGB-16673 and pirtobrutinib, for adults with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) when previous treatments haven't worked well enough. It aims to see which medicine is safer and more effective.
Chronic Lymphocytic LeukemiaSmall Lymphocytic LymphomaAustralia - RecruitingPHASE3
A Study of Vepugratinib (LY3866288) in Participants With Cancer in the Urinary Tract
This study is testing a new medicine, vepugratinib, for advanced or spread bladder cancer. It will be given with two other drugs, enfortumab vedotin and pembrolizumab, to see if it's safe and helps. It’s compared to a dummy medicine (placebo).
Carcinoma, Transitional CellUrinary Bladder NeoplasmsNeoplasm MetastasisAustralia - Active not recruitingPHASE3
REDEFINE 3: A Research Study to See the Effects of CagriSema in People Living With Diseases in the Heart and Blood Vessels
This study, called REDEFINE 3, investigates a new medicine, CagriSema, for people with heart and blood vessel diseases. It aims to see if CagriSema can reduce the risk of serious events like heart attacks and strokes.
Cardiovascular DiseaseAustralia - RecruitingPHASE3
A Study Comparing AZD0120, a Dual-targeted CAR-T Against B-cell Maturation Antigen (BCMA) and CD19, Versus Standard Regimens in Participants With Relapsed Refractory Multiple Myeloma (DURGA-4)
This study compares a new treatment, AZD0120, with standard treatments for multiple myeloma that has come back or isn't responding well. It aims to see if the new medicine works better and is safe for patients.
Relapsed Refractory Multiple MyelomaAustralia - RecruitingPHASE3
Study of Sacituzumab Govitecan Versus Standard of Care in Participants With Previously Treated Extensive Stage Small Cell Lung Cancer
This study is looking at a new medicine called sacituzumab govitecan (SG) for people with a type of lung cancer called extensive stage small cell lung cancer (ES-SCLC) that has been treated before. We want to see if SG works better than standard treatments to help people live longer.
Extensive Stage Small Cell Lung Cancer (ES-SCLC)Australia - Active not recruitingPHASE3
A Study to Evaluate the Efficacy, Safety, and Tolerability of BMS-986278 in Participants With Progressive Pulmonary Fibrosis
This study is looking into a new medication called BMS-986278 for people with progressive pulmonary fibrosis. It aims to see if the medication is safe and effective in slowing down this lung condition.
Progressive Pulmonary FibrosisAustralia