Latest studies
- 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 SyndromeUnited States - 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 FractionObesityUnited States - RecruitingPHASE3
Study to evaLuate the effIcacy and Safety of abeLacimab in High-risk Patients With Atrial Fibrillation Who Have Been Deemed Unsuitable for Oral antiCoagulation (LILAC-TIMI 76)
This study looks at a new medicine, abelacimab, for people with an irregular heartbeat (atrial fibrillation) who can't take usual blood thinners. It compares abelacimab to a dummy medicine to see if it can prevent strokes.
Atrial Fibrillation (AF)United States - 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 CancerUnited States - 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 LymphomaUnited States - 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, AlcoholicUnited States - RecruitingPHASE2
A Phase II Study of Ensifentrine in Non-Cystic Fibrosis Bronchiectasis
This study is testing a new medicine, ensifentrine, for people with a lung condition called non-cystic fibrosis bronchiectasis. We want to see if it helps reduce flare-ups, improve breathing, and make people feel better, compared to a dummy medicine.
Non-cystic Fibrosis BronchiectasisUnited States - RecruitingPHASE1
First in Human Study of CDR404 in HLA-A*02:01 Participants With MAGE-A4 Expressing Solid Tumors
This study is testing a new experimental medicine, CDR404, for hard-to-treat solid cancers that have a specific marker called MAGE-A4. It's the first time CDR404 is being used in people, and doctors want to see if it's safe and if it can help fight the cancer.
Select Advanced Solid TumorsUnited States - 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.
LeukemiaUnited States - RecruitingPHASE3
A Global Study of Volrustomig (MEDI5752) for Participants With Unresected Locally Advanced Head and Neck Squamous Cell Carcinoma Following Definitive Concurrent Chemoradiotherapy
This study is testing a new medicine, volrustomig, for people with a type of head and neck cancer that hasn't spread and wasn't removed by surgery. It aims to see if volrustomig helps prevent the cancer from coming back after standard treatment like chemotherapy and radiotherapy.
Locally Advanced Head and Neck Squamous Cell CarcinomaUnited States - 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 2United States - 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 PolyneuropathyUnited States - 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 DiseaseUnited States - RecruitingPHASE2
A Study of Novel Agents or Combinations as Perioperative Treatment in Participants With Locally Advanced Resectable Gastroesophageal Adenocarcinoma
This study looks at new treatments for stomach or food pipe cancer that can be removed by surgery. It aims to see how safe and effective these new drugs or drug combinations are before and after the operation.
Gastroesophageal AdenocarcinomaUnited States - 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)United States - Active not recruitingPHASE3
A Study of Atezolizumab Versus Placebo as Adjuvant Therapy in Participants With High-risk Muscle-invasive Bladder Cancer (MIBC) Who Are ctDNA Positive Following Cystectomy
This study looks at a medicine called atezolizumab for people with a type of bladder cancer that has spread into the muscle. It checks if this medicine, given after surgery, can stop the cancer from coming back in those who show signs of tiny cancer cells in their blood.
Muscle-invasive Bladder CancerUnited States - 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 CancerUnited States - 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 AUnited States - Active not recruitingNA
Evaluation of the Safety and Efficacy of TLD in Patients with COPD
This study is looking at a new treatment called Targeted Lung Denervation (TLD) for people with a lung condition called COPD. It aims to see if TLD is safe and works better than standard care to reduce flare-ups of COPD symptoms.
COPDUnited States - 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 MyelofibrosisUnited States - 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)United States - Active not recruitingPHASE1
Phase I Study of 131-I mIBG Followed by Nivolumab & Dinutuximab Beta Antibodies in Children With Relapsed/Refractory Neuroblastoma
This study is looking at new ways to treat neuroblastoma in children, a type of cancer. It combines standard treatment with new medicines (Nivolumab and Dinutuximab Beta) to see if they are safe and helpful. The goal is to find better treatments for children whose cancer has come back or is hard to treat.
NeuroblastomaUnited States - RecruitingPHASE1
A First-In-Human Study of LY3954068 in Participants With Early Symptomatic Alzheimer's Disease
This study looks at a new medicine called LY3954068 for people with early Alzheimer's disease. Researchers want to see if it's safe, how it moves through the body, and if it affects signs of the disease. Participants will receive the medicine or a dummy treatment through an injection into the spinal fluid.
Alzheimer DiseaseUnited States - Active not recruitingPHASE3
A Study to Investigate the Efficacy and Safety of OTL-203 in Subjects With MPS-IH Compared With Standard of Care With Allogeneic HSCT
This study looks at a new gene therapy, OTL-203, for children with Hurler Syndrome (MPS-IH). It compares this new treatment to the standard stem cell transplant to see which works better and is safer for these young patients.
MPS-IH (Hurler Syndrome)United States - Active not recruitingPHASE3
Safety, Efficacy and Tolerability of Ianalumab Versus Placebo, Combination With SoC Therapy, in Participants With Active Lupus Nephritis
This study is testing a new medicine, ianalumab, for adults with active lupus nephritis. It compares ianalumab given every 4 or 12 weeks to a dummy medicine, alongside standard treatments, to see if it's safe and effective.
Lupus NephritisUnited States - Active not recruitingPHASE1
A Study to Assess the Safety and Tolerability of AZD1390 Given With Radiation Therapy in Patients With Brain Cancer
This study is testing a new drug, AZD1390, alongside standard radiation for brain cancer. It's the first time AZD1390 has been given to patients. The main goal is to check its safety and how the body handles it, especially when combined with different types of radiation.
Recurrent Glioblastoma MultiformePrimary Glioblastoma MultiformeBrain Neoplasms, MalignantLeptomeningeal Disease (LMD)United States - 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)United States - 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 DegenerationUnited States - RecruitingPHASE1
A Phase 1/1b Study of IAM1363 in HER2 Cancers
This study is testing a new medicine called IAM1363 for advanced cancers with a specific change called HER2. It aims to find a safe and effective dose, including for breast, lung, and stomach cancers that have spread to the brain.
HER2 Mutation-Related TumorsHER2HER2-positive Breast CancerHER2 + Breast CancerUnited States - 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.
HypochondroplasiaUnited States - 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-19United States - 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 NeoplasmsUnited States - 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 FibrosisUnited States - RecruitingPHASE2
A Clinical Study of Tulisokibart (MK-7240) to Treat Radiographic Axial Spondyloarthritis (MK-7240-013)
This study is looking for a new way to treat a type of arthritis called radiographic axial spondyloarthritis (r-axSpA). Researchers want to see if an experimental medicine, tulisokibart, can help reduce pain, stiffness, and swelling in the spine and hips for people with this condition.
Radiographic Axial SpondyloarthritisUnited States - Active not recruitingPHASE2
A Study to Evaluate the Efficacy and Safety of Concomitant Use of Eplontersen and ALXN2220 Compared With Eplontersen and Placebo for Adults Participants With ATTR-CM
This study is testing two medicines, eplontersen and ALXN2220, to see if they are safe and help adults with a heart condition called ATTR-CM. Participants will take eplontersen along with either ALXN2220 or an inactive substance (placebo).
Transthyretin Amyloid Cardiomyopathy (ATTR-CM)United States - RecruitingPHASE2
A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease
This study is looking at a new gene therapy called RP-A501 for boys and men with Danon Disease. It aims to check if the treatment is safe and helps improve their condition. It's a Phase 2 trial, meaning it's still being tested.
Danon DiseaseUnited States - 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 CancerUnited States - RecruitingPHASE3
A Phase III Study of AZD0780 on Major Adverse CV Events in Patients With a History of ASCVD Events or at High Risk for a First Event
This study is looking into a new medication called AZD0780. It aims to see if this medicine can lower the risk of serious heart problems like heart attacks or strokes in people who have already had them, or in those at high risk of having one for the first time.
Cardiovascular DiseaseUnited States - RecruitingPHASE1, PHASE2
Clinical Study to Assess the Safety and Efficacy of the SpectraCure P18 System
This study is testing a new light-based treatment for men with prostate cancer that has come back. It uses a special light system called SpectraCure P18 and a drug called verteporfin to see if it's safe and helps shrink the cancer.
Recurrent Prostate CancerUnited States - 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 AngioedemaUnited States - 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 MalignantUnited States - Active not recruitingNA
F15 Recharge Free Axonics SNM System Clinical Study
This study is looking at a new device for people with bladder control issues (leaking, needing to go often) or bowel control issues. It's a small implant that helps nerves work better, and it doesn't need recharging. Researchers want to see how well it works and if it's safe.
Urinary Urge Incontinence (UUI)Urinary Frequency (UF)Fecal Incontinence (FI)United States - RecruitingPHASE2
A Study to Test Whether BI 1815368 Helps People With an Eye Condition Called Diabetic Macular Edema
This study tests a new medicine, BI 1815368, for people with diabetic macular edema (DME), an eye condition linked to diabetes. It aims to see if the medicine improves sight and to find the best dose. Participants take tablets for about 11 months.
Macular EdemaUnited States - Active not recruitingPHASE2
A Study to Test Whether BI 764524 Helps People With an Eye Condition Called Diabetic Retinopathy
This study is testing a new medicine, BI 764524, for people with an eye condition called diabetic retinopathy. It aims to see if it improves their vision and how often it should be given.
Diabetic RetinopathyUnited States - RecruitingPHASE1, PHASE2
A Phase 1/2 Study to Investigate CRB-701 in Solid Tumors
This study is testing a new medicine, CRB-701, for people with certain advanced solid cancers. It aims to find a safe and effective dose, and see which cancers it can treat. Participants will receive the medicine and have tests to check how it works.
Solid Tumor, AdultUnited States - 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)United States - 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)United States - RecruitingPHASE2
Dose-Ranging Safety, Tolerability, and Efficacy Study of AZD2373 in Participants With APOL1-Mediated Kidney Disease
This study is testing a new drug, AZD2373, for a specific type of kidney disease called APOL1-Mediated Kidney Disease. It's looking at how well the drug works and if it's safe in people aged 18-65 with this condition.
APOL1-Mediated Kidney DiseaseUnited States - 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 PainUnited States - Active not recruitingPHASE1, PHASE2
A Study of TAK-788 in Adults With Non-Small Cell Lung Cancer
This study is testing a new medicine, TAK-788 (also known as mobocertinib), for adults with a type of lung cancer. Researchers want to find the best dose, check for side effects, and see how it works in the body.
Carcinoma, Non-Small-Cell LungUnited States