Latest studies
- Recruiting
Exploring the hidden burden of living with Glanzmann thrombasthenia
This study wants to hear from people living with Glanzmann thrombasthenia (GT) and their caregivers. We want to understand the daily challenges and feelings of those affected. Your stories can help improve support and understanding for everyone with GT.
Glanzmann thrombastheniaJordan - RecruitingPHASE3
LIVERAGE™: A Study to Test Whether Survodutide Helps People With a Liver Disease Called NASH/MASH Who Have Moderate or Advanced Liver Fibrosis
This study looks at a new medicine called survodutide for adults with a liver condition called MASH and moderate to advanced liver damage. It aims to see if the medicine can improve liver health and how safe it is.
Metabolic Dysfunction Associated Steatohepatitis (MASH)Liver FibrosisJordan - RecruitingPHASE3
LIVERAGE™ - Cirrhosis: A Study to Test Whether Survodutide Helps People With a Liver Disease Called NASH/MASH Who Have Cirrhosis
This study looks at a new medicine called survodutide for adults with a liver condition called NASH or MASH and scarring (cirrhosis). Participants will get either the medicine or a dummy treatment, along with diet and exercise advice, to see if it helps their liver.
Metabolic Dysfunction Associated SteatohepatitisJordan - Active not recruitingPHASE3
Study of Two Doses of Crizanlizumab Versus Placebo in Adolescent and Adult Sickle Cell Disease Patients
This study is looking at a new medicine called crizanlizumab to see if two different doses can help teenagers and adults with sickle cell disease. We want to find out if it's safe and if it can reduce pain crises (VOCs) compared to a dummy medicine (placebo).
Sickle Cell Disease (SCD)Jordan - Active not recruitingPHASE3
A Study to Evaluate How Safe Pozelimab + Cemdisiran Combination Therapy is and How Well it Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Not Recently Received or Have Not Received Complement Inhibitor Treatment
This study looks at a new drug combination, pozelimab and cemdisiran, for adults with a rare blood condition called PNH. We want to see how safe and effective it is compared to existing treatments, ravulizumab and eculizumab. It's for people who haven't recently had certain PNH treatments.
Paroxysmal Nocturnal HemoglobinuriaJordan - Recruiting
Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN) International Registry
This international study is gathering information on a very rare blood cancer called Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN). It aims to understand the disease better, look at different treatments, and improve care by collecting data from patients worldwide.
Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)Jordan - Active not recruitingPHASE2
A Study of Combination Therapy With Guselkumab and Golimumab in Participants With Moderately to Severely Active Crohn's Disease
This study is looking at a new combination treatment, JNJ-78934804, for people aged 18-65 with Crohn's disease that is moderate to severe. It compares this new treatment to existing medicines, guselkumab and golimumab, to see how well it works over 48 weeks.
Crohn's DiseaseJordan - Active not recruiting
Follow-up in Gynecological Cancer Survivors
This study aims to understand the physical and emotional challenges faced by women after treatment for gynaecological cancer. Researchers will ask around 1100 patients about their quality of life to identify common issues and how they might be linked to their treatment or personal circumstances.
Gynecologic CancerJordan - RecruitingPHASE1, PHASE2
Open-label Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy (FORWARD-53)
This study is looking at a new medicine called WVE-N531 for Duchenne muscular dystrophy. It wants to see if the medicine is safe, how the body uses it, and if it helps patients. It focuses on boys with a specific genetic change.
Duchenne Muscular DystrophyJordan - RecruitingPHASE3
Long-term Safety and Tolerability of Inclisiran in Participants With HeFH or HoFH Who Have Completed the Pediatric ORION-16, ORION-13, ORION-20, or ORION-19 Studies
This study looks at the long-term safety of a medicine called inclisiran for people aged 12-100 with high cholesterol due to a genetic condition (HeFH or HoFH). It's for those who have already been in earlier inclisiran studies.
Heterozygous or Homozygous Familial HypercholesterolemiaJordan - RecruitingPHASE3
A Study in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) to Evaluate How Safe Long-term Treatment With Pozelimab + Cemdisiran Combination Therapy is and How Well it Works
This study looks at two new drugs, pozelimab and cemdisiran, for a rare blood condition called PNH. Researchers want to see if the combination is safe and works well over a long time for adults with PNH.
Paroxysmal Nocturnal HemoglobinuriaJordan - Active not recruitingPHASE2
Activity of Lorlatinib Based on ALK Resistance Mutations Detected on Blood in ALK Positive NSCLC Patients
This study looks at a lung cancer drug called lorlatinib for people with a specific type of non-small cell lung cancer (NSCLC) where previous treatments have stopped working. It aims to see if specific changes in the cancer's genes, found in blood tests, can predict who will benefit most from lorlatinib.
Non Small Cell Lung CancerJordan - Active not recruitingPHASE3
Efficacy and Safety of Remibrutinib Compared to Teriflunomide in Participants With Relapsing Multiple Sclerosis (RMS)
This study is comparing a new medication called remibrutinib with an existing treatment, teriflunomide, for people with relapsing multiple sclerosis (RMS). It aims to see how well remibrutinib works and if it's safe to use in managing RMS symptoms.
Relapsing Multiple SclerosisJordan - Active not recruitingPHASE3
A Study of the Efficacy and Safety of Efgartigimod in Patients With Primary Sjögren's Syndrome
This study is looking into a new treatment called efgartigimod for people with Primary Sjögren's Syndrome. It aims to see how well it works and if it's safe. Participants will either receive the new medicine or a dummy treatment, and the study will last about two years.
Primary Sjogrens DiseaseJordan - Recruiting
Pompe Disease Registry Protocol
This registry tracks people with Pompe disease globally. It helps us understand the condition better, how it changes over time, and the effects of treatments. The goal is to improve care for everyone with Pompe disease.
Glycogen Storage Disease Type IIPompe DiseaseJordan - RecruitingPHASE3
A Study to Evaluate the Efficacy and Safety of Inavolisib in Combination With Phesgo Versus Placebo in Combination With Phesgo in Participants With PIK3CA-Mutated HER2-Positive Locally Advanced or Metastatic Breast Cancer
This study is looking at a new combination of treatments, Inavolisib and Phesgo, for HER2-positive breast cancer that has spread. We want to see if this new combination works better and is safe compared to Phesgo alone after initial treatment.
Metastatic Breast CancerJordan - Active not recruitingPHASE3
Cardiovascular Outcome Study to Evaluate the Effect of Obicetrapib in Patients With Cardiovascular Disease
This study is looking at a new medicine called Obicetrapib for people with heart disease. It aims to see if this medicine can help reduce serious heart problems like heart attacks and strokes, especially for those whose cholesterol isn't well-controlled with their usual treatments.
Atherosclerotic Cardiovascular DiseaseJordan - Active not recruitingPHASE3
A Study to Evaluate the Efficacy and Safety of Giredestrant in Combination With Phesgo (Pertuzumab, Trastuzumab, and Hyaluronidase-zzxf) Versus Phesgo in Participants With Locally Advanced or Metastatic Breast Cancer (heredERA Breast Cancer)
This study looks at a new drug, Giredestrant, combined with an existing treatment called Phesgo, for advanced breast cancer. It aims to see if this new combination is safe and works better than Phesgo alone for people with specific types of breast cancer.
Locally Advanced or Metastatic Breast CancerJordan - Recruiting
International Collaborative Gaucher Group (ICGG) Gaucher Disease Registry & Pregnancy Sub-registry
This study tracks the health of people with Gaucher disease over time, whether or not they are receiving treatment. There's also a sub-study for pregnant women with Gaucher disease to see how their pregnancies and babies' health progress. It helps doctors learn more about the condition.
Gaucher DiseaseCerebroside Lipidosis SyndromeGlucocerebrosidase Deficiency DiseaseGlucosylceramide Beta-Glucosidase Deficiency DiseaseJordan - RecruitingNA
Partial Breast Re-irradiation Using Ultra Hypofractionation (PRESERVE)
This study looks at a new, shorter radiation treatment for women whose breast cancer has come back, or who have developed a new cancer in the same breast after earlier treatment. It aims to see if this quicker, one-week course of radiation is safe and works well.
Breast CancerBreast Cancer RecurrentJordan - Active not recruitingPHASE2
A Global Study of Midostaurin in Combination With Chemotherapy to Evaluate Safety, Efficacy and Pharmacokinetics in Newly Diagnosed Pediatric Patients With FLT3 Mutated AML
This study is testing a new combination of treatments for children and teenagers with a certain type of acute myeloid leukemia (FLT3-mutated AML). It combines a drug called midostaurin with standard chemotherapy to see if it's safe and effective for these patients.
FLT3-mutated Acute Myeloid LeukemiaJordan - Active not recruitingPHASE3
A Study to Compare the Efficacy and Safety of Entrectinib and Crizotinib in Participants With Advanced or Metastatic ROS1 Non-small Cell Lung Cancer (NSCLC) With and Without Central Nervous System (CNS) Metastases
This study compares two medicines, entrectinib and crizotinib, for advanced lung cancer (non-small cell lung cancer) that has a specific genetic change called ROS1. It aims to see which medicine works better and is safer for patients, including those whose cancer has spread to the brain.
Carcinoma, Non-Small-Cell LungJordan - Active not recruitingPHASE3
DAY101 vs. Standard of Care Chemotherapy in Pediatric Participants With Low-Grade Glioma Requiring First-Line Systemic Therapy (LOGGIC/FIREFLY-2)
This study is for children and young people under 25 with a type of brain tumour called low-grade glioma. It compares a new medicine, tovorafenib, with standard chemotherapy to see which works best and is safest for those needing first-time treatment.
Low-grade GliomaRapidly Accelerated Fibrosarcoma (RAF) Altered GliomaPediatric Low-grade GliomaJordan - Active not recruitingPHASE3
A Study of Adding Apalutamide to Radiotherapy and LHRH Agonist in High-Risk Patients With Hormone-Sensitive Prostate Cancer
This study is looking at whether adding a drug called apalutamide to standard prostate cancer treatments (radiotherapy and hormone therapy) helps men with high-risk prostate cancer live longer without their cancer spreading. It compares the new combination to the standard treatment alone.
Prostatic NeoplasmsJordan - Active not recruitingPHASE2, PHASE3
A Study of Guselkumab in Participants With Moderately to Severely Active Ulcerative Colitis
This study is looking at a new medicine called guselkumab for people with ulcerative colitis that isn't well-controlled. We want to see if it helps reduce symptoms and if it's safe to use for those with moderate to severe forms of the condition.
Ulcerative ColitisJordan - RecruitingPHASE2
Safety and Efficacy of OD-07656 and of Subsequent Vedolizumab Therapy in Moderately to Severely Active Ulcerative Colitis
This study is testing a new medicine, OD-07656, for people with moderate to severe ulcerative colitis. It also looks at whether OD-07656 can improve the effects of another medicine called vedolizumab when used afterwards. The main goal is to check how well the medicines work and if they are safe.
Ulcerative Colitis (UC)UC - Ulcerative ColitisJordan - Active not recruitingPHASE3
A Phase 3 Study to Evaluate the Safety and Efficacy of Efgartigimod PH20 Subcutaneous in Adult Patients With Primary Immune Thrombocytopenia
This study looks at a new injection called efgartigimod for adults with primary immune thrombocytopenia (ITP). It aims to see how safe and effective it is. If you've been in a previous study for this treatment, you might be able to continue.
Primary Immune ThrombocytopeniaJordan - Enrolling by invitationPHASE2
Open-Label Extension Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy
This study is looking at the long-term safety and effects of a medicine called WVE-N531 for Duchenne muscular dystrophy. It's for boys who have already taken part in an earlier study of WVE-N531. They will continue to receive the treatment and be carefully checked.
Duchenne Muscular DystrophyJordan - Active not recruitingPHASE3
A Study to Investigate Tolerability and Efficacy of Asciminib (Oral) Versus Nilotinib (Oral) in Adult Participants (≥18 Years of Age) With Newly Diagnosed Philadelphia Chromosome Positive Chronic Myelogenous Leukemia in Chronic Phase (Ph+ CML-CP)
This study compared two oral medications, asciminib and nilotinib, for adults newly diagnosed with a type of blood cancer called Philadelphia chromosome-positive chronic myeloid leukemia (CML). It looked at how well people tolerated each drug.
Philadelphia Chromosome-Positive Chronic Myeloid LeukemiaJordan - Active not recruitingPHASE2, PHASE3
A Study of the Efficacy and Safety of Guselkumab in Participants With Moderately to Severely Active Crohn's Disease
This study looks at a new medicine called guselkumab for adults with moderately to severely active Crohn's disease. Researchers want to see how well it works and if it's safe compared to other treatments or a dummy medicine.
Crohn's DiseaseJordan - Active not recruitingPHASE3
A Study to Compare Safety and Efficacy of High Doses of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) (MIS51ON)
This study is looking at higher doses of a medicine called eteplirsen for boys with Duchenne muscular dystrophy (DMD). It's checking if these higher doses are safe and work better than the usual dose to help manage the condition.
Muscular Dystrophy, DuchenneJordan - Active not recruitingPHASE3
A Study of Guselkumab Therapy in Participants With Moderately to Severely Active Ulcerative Colitis
This study looks at a new drug, guselkumab, for people in the UK with moderate to severe ulcerative colitis. It aims to see if the drug can help improve their symptoms and lead to remission, compared to a dummy treatment. It's a key step in understanding this potential new medicine.
Colitis, UlcerativeJordan - Active not recruitingPHASE2
A Study of Combination Therapy With Guselkumab and Golimumab in Participants With Moderately to Severely Active Ulcerative Colitis
This study looks at new ways to treat ulcerative colitis, a condition causing gut inflammation. We're testing a combination of medicines (JNJ-78934804) for people whose current treatments aren't working well. The goal is to see if these new approaches are safe and effective.
Colitis, UlcerativeJordan - Recruiting
The K-Map Study, Global Prevalence of KC
The K-Map Study is looking for young people aged 6-23 to help understand how common a condition called keratoconus is. It uses a special camera to check eyes, even if they seem healthy, to see if more people have this eye problem than we currently think.
KeratoconusJordan - RecruitingPHASE2
A Study of Long-acting Antibodies Alone and in Combinations for Moderate to Severe Ulcerative Colitis
This study is testing new long-acting medicines for adults with moderate to severe ulcerative colitis. It aims to see how well they work and if they are safe, both on their own and in combinations, compared to a dummy treatment.
Ulcerative ColitisInflammatory Bowel DiseasesColitisColitis, UlcerativeJordan - Active not recruitingPHASE3
A Study of Guselkumab in Participants With Fistulizing, Perianal Crohn's Disease
This study is testing a new medication called guselkumab for people with Crohn's disease who have problems with fistulas around the back passage. It aims to see how well the medication works and if it's safe compared to a dummy treatment.
Fistulizing Crohns DiseasePerianal Crohns DiseaseJordan - Active not recruitingPHASE2
Adjuvant Palbociclib in Elderly Patients With Breast Cancer
This study looks at a new way to treat older patients (aged 70+) with early breast cancer. Instead of chemotherapy, it tests if a drug called palbociclib, combined with standard hormone therapy, can be just as good at preventing cancer from coming back.
Breast Cancer Stage IIBreast Cancer Stage IIIJordan