- Active not recruitingPHASE3
Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy
This study is testing a new gene therapy, PF-06939926, for boys aged 4-7 with Duchenne muscular dystrophy. It aims to see if the treatment is safe and effective in improving muscle function. Some boys will get the treatment right away, others will get a dummy treatment first, then the real treatment later.
For: Duchenne Muscular DystrophyUnited States · Australia · Belgium - RecruitingPHASE2
Efficacy and Safety Study of OATD-01 in Patients With Active Pulmonary Sarcoidosis
This study is testing a new medicine, OATD-01, for people with active sarcoidosis in their lungs. We want to see if it helps improve symptoms and is safe to use. Some people will get the new medicine, and others will get a dummy medicine.
For: Pulmonary SarcoidosisUnited States · Denmark · France - RecruitingPHASE2
A Phase 2 Trial of ALN-APP in Patients With Cerebral Amyloid Angiopathy
This study is looking at a new medicine called ALN-APP for a condition called Cerebral Amyloid Angiopathy (CAA). It aims to see how well ALN-APP works to slow down CAA and if it's safe. Participants will either receive the new medicine or a dummy treatment.
For: Cerebral Amyloid AngiopathyUnited States · Australia · Canada - RecruitingPHASE1
A Research Study on the Effects of NNC0537-1482 in Participants With Heart Failure
This study is testing a new medicine called NNC0537-1482 for people with heart failure. We're looking at its safety and how it works. Some will get the new medicine, others a dummy medicine. It lasts about two months, plus a check-up period.
For: Heart FailureUnited Kingdom - Active not recruitingPHASE3
A Study to Investigate the Safety and Efficacy of mRNA-1403 in Participants ≥18 Years of Age for the Prevention of Acute Gastroenteritis
This study is testing a new vaccine (mRNA-1403) to see if it can prevent severe stomach flu, especially the kind caused by norovirus. It will also check how safe the vaccine is and if it causes side effects in adults aged 18 and over.
For: Acute Gastroenteritis · Norovirus Acute GastroenteritisUnited States · Australia · Canada - Active not recruitingPHASE3
Study of Ianalumab Versus Placebo in Addition to First-line Corticosteroids in Primary Immune Thrombocytopenia (ITP)
This study is looking at a new medicine called ianalumab for adults with a blood condition called ITP. We want to see if ianalumab, when given with standard treatment (steroids), can help keep platelet counts up. Half the participants will get ianalumab, and half will get a dummy medicine.
For: Primary Immune Thrombocytopenia (ITP)United States · Argentina · Australia - Active not recruitingPHASE3
Brentuximab Vedotin Plus Lenalidomide and Rituximab for the Treatment of Relapsed/Refractory DLBCL
This study is for people in the UK with a type of blood cancer called Diffuse Large B-cell Lymphoma (DLBCL) that has returned or not responded to previous treatments. It aims to see if adding an experimental drug, brentuximab vedotin, to two existing treatments (rituximab and lenalidomide) works better than the two existing treatments alone.
For: Diffuse Large B-cell LymphomaUnited States · Australia · Belgium - AuthorisedTherapeutic confirmatory (Phase III)
A Phase 3, Randomized, Double-blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of Azetukalner in Depressive Episodes Associated With Bipolar I or II Disorder (Bipolar Depression)
This study is looking for people with bipolar depression aged 18 or over. It's testing a new medicine, Azetukalner, against a dummy treatment, to see if it helps improve mood over six weeks. The main goal is to check how much depression symptoms change.
For: Depressive episodes associated with Bipolar disorder I or II (Bipolar Depression)Germany · Bulgaria · Spain - RecruitingPHASE3
A Study With Eptinezumab in Adolescents (12-17 Years) With Chronic Migraine
This study is testing a new medicine, eptinezumab, for young people aged 12 to 17 who get chronic migraines. It wants to see if this medicine can reduce the number of migraine days compared to a dummy treatment.
For: Chronic Migraine in ChildrenUnited States · Argentina · Canada - RecruitingPHASE4
A Study to Investigate the Effect of Budesonide/Glycopyrronium/Formoterol Fumarate Metered Dose Inhaler (BGF MDI) Compared With Placebo MDI on Heart and Lung Function in Participants With Chronic Obstructive Pulmonary Disease (COPD) and Hyperinflation
This study looks at a new inhaler (BGF MDI) for people with COPD and lung hyperinflation. We want to see how it affects heart and lung function compared to a dummy inhaler. It's for people aged 40-80 who smoke or used to smoke.
For: Chronic Obstructive Pulmonary Disease · HyperinflationGermany · United Kingdom - Active not recruitingPHASE2
Efficacy and Safety of GSK4527226 [AL101] in Participants With Early Alzheimer's Disease
This study is testing a new medicine, GSK4527226, for people in the early stages of Alzheimer's disease. Researchers want to see if it's safe and helps improve or slow down symptoms compared to a dummy medicine.
For: Alzheimer's DiseaseUnited States · Argentina · Australia - Active not recruitingPHASE2
Long-term Follow-up Study to Evaluate Durability of Treatment Response in Previous Bepirovirsen Study Participants (B-Sure)
This study is checking if the benefits of bepirovirsen for Hepatitis B last over time. It's for people who previously took part in a bepirovirsen trial and showed good results. No new treatment is given in this follow-up study, just monitoring.
For: Hepatitis BUnited States · Argentina · Australia - Active not recruitingPHASE3
A Study of Navenibart in Participants With Hereditary Angioedema
This study is testing a new medicine, navenibart, for people with Hereditary Angioedema (HAE). Researchers want to see if it safely and effectively prevents swelling attacks in adults and teenagers with HAE.
For: Hereditary Angioedema (HAE)United States · Australia · Austria - Active not recruitingPHASE2, PHASE3
Efficacy and Safety of M281 in Adults With Warm Autoimmune Hemolytic Anemia
This study is looking into a new medication, M281, for adults with a blood condition called warm autoimmune hemolytic anemia (wAIHA). It aims to see if the medicine is effective and safe compared to a dummy treatment. Participants will be treated for a long time, with close monitoring.
For: Warm Autoimmune Hemolytic AnemiaUnited States · Brazil · China - RecruitingPHASE3
Botulinum Toxin Type A Blockade of the Sphenopalatine Ganglion in Treatment-refractory Chronic Cluster Headache
This study looks at a new way to treat very bad headaches called 'cluster headaches.' We're testing if injecting a medicine called botulinum toxin (similar to Botox) near a special nerve in the face can help stop these painful attacks when other treatments haven't worked.
For: Cluster HeadacheGermany · Italy · Norway - Active not recruitingPHASE3
A Study to Determine the Efficacy and Safety of Deucravacitinib Compared With Placebo in Participants With Active Psoriatic Arthritis (PsA) Who Are Naïve to Biologic Disease Modifying Anti-rheumatic Drugs or Had Previously Received TNFα Inhibitor Treatment
This study is testing a new medication called deucravacitinib for active psoriatic arthritis. It compares the new drug to a dummy pill to see if it helps people feel better and if it's safe. It's for people new to certain treatments or who have tried one specific type before.
For: Psoriatic ArthritisUnited States · Argentina · Australia - RecruitingPHASE2
A Study to Learn About How Well BAY 3401016 Works in Adults With Alport Syndrome
This study is looking into a new medication called BAY 3401016 for adults with Alport Syndrome, a genetic kidney condition. It aims to see if this medicine can help slow down kidney damage by blocking a specific protein.
For: Alport SyndromeUnited States · Argentina · Canada - Active not recruitingPHASE3
A Study of Efficacy and Safety of Ianalumab in Previously Treated Patients With Warm Autoimmune Hemolytic Anemia
This study looks at a new medicine, ianalumab, for people with warm autoimmune hemolytic anemia (wAIHA) who haven't responded to previous treatments. It aims to see if ianalumab can increase healthy red blood cells and if it is safe.
For: Warm Autoimmune Hemolytic Anemia (wAIHA)United States · Argentina · Australia - Active not recruitingPHASE2, PHASE3
An Extension Study of Treprostinil Palmitil Inhalation Powder (TPIP) for Pulmonary Arterial Hypertension (PAH)
This study looks at the long-term safety of a new inhaled medicine, Treprostinil Palmitil (TPIP), for people with a lung condition called Pulmonary Arterial Hypertension (PAH). It's for those who have already tried TPIP in earlier studies.
For: Pulmonary Arterial HypertensionUnited States · Argentina · Austria - Active not recruitingPHASE2
Study of RV001V in Biochemical Failure Following Curatively Intended Therapy For Localized Prostate Cancer
This study is for men who have had treatment for prostate cancer, but their PSA levels are now rising. It aims to test a new vaccine (RV001V) against a dummy treatment to see if it can help prevent the cancer from returning.
For: Prostate Cancer RecurrentUnited States · Belgium · Denmark - Active not recruitingPHASE2, PHASE3
Pharmacodynamic EffIcacy and Clinical Benefit of AT 007 in Patients With Sorbitol Dehydrogenase (SORD) Deficiency
This study is testing a new medicine called AT-007 for people with a genetic condition that affects their nerves, called SORD deficiency. Researchers want to see if AT-007 is safe and helps improve symptoms or slows the condition's progression.
For: Hereditary Neuropathy Caused by SORD DeficiencyUnited States · Czechia · Italy - Active not recruitingPHASE3
Evaluation of Maralixibat in Pruritus Associated With General Cholestatic Liver Disease (EXPAND)
This study is looking into a new medication, maralixibat, for children and adults with a specific type of chronic liver disease that causes severe itching. It aims to see if maralixibat is safe and effective when other treatments haven't worked.
For: Cholestatic Liver Disease (Except ALGS, PFIC, PBC and PSC)United States · Brazil · Canada - Active not recruitingPHASE2
Study Evaluating AZD7798 for Treatment in Crohn's Disease Patients With an Ileostomy
This study is investigating a new medication called AZD7798 for people with Crohn's disease who have had an ileostomy. We want to see if it's safe, well-tolerated, and helps heal the gut lining compared to a dummy drug.
For: Crohn's DiseaseBelgium · Italy · Netherlands - RecruitingPHASE2
Treatment of Ectopic Calcification in Fahr's Disease or Syndrome
This study is looking into a new treatment for Fahr's disease, a condition where calcium builds up in the brain. Researchers want to see if drugs called bisphosphonates, which help with bone strength, can also help people with Fahr's disease feel better.
For: Fahr Disease · Fahr Syndrome · Primary Familial Brain CalcificationNetherlands · United Kingdom - Active not recruitingPHASE2
A Phase 2, Dose Ranging Study Assessing Rocatinlimab in Moderate-to-severe Asthma
This study is looking at a new medicine called rocatinlimab for people aged 18-75 with moderate to severe asthma. It wants to see if the medicine can reduce asthma attacks. Participants will receive either rocatinlimab or a dummy medicine called a placebo.
For: AsthmaUnited States · Argentina · Australia - Active not recruitingPHASE1, PHASE2
Fulvestrant +/- Akt Inhibition in Advanced Aromatase Inhibitor Resistant Breast Cancer
This study is for post-menopausal women with advanced breast cancer that has grown despite standard hormone treatment. It's testing a new drug, AZD5363, alongside an existing treatment, Fulvestrant, to see if it can stop cancer growth more effectively.
For: Estrogen Receptor Positive Breast CancerUnited Kingdom - Not yet recruitingPHASE2
A Study to Investigate the Efficacy and Safety of SC0032, a Magnesium Salt Oral Spray, in Adults With Refractory or Unexplained Chronic Cough
This study is testing a new magnesium oral spray, SC0032, for adults with a stubborn, long-lasting cough that hasn't responded to other treatments or has no clear cause. Researchers want to see if it's safe and helps improve coughs.
For: Refractory Chronic Cough and Unexplained Chronic CoughUnited Kingdom - RecruitingPHASE2, PHASE3
A Phase 2/3 Study of Brepocitinib in Adults With Lichen Planopilaris
This study is testing a new medicine, brepocitinib, for a hair loss condition called lichen planopilaris (LPP). Researchers want to see if it's safe and helps improve symptoms. It's for adults with active LPP and involves taking tablets or a dummy pill.
For: Lichen PlanopilarisUnited States · Australia · Canada - RecruitingPHASE2
A Study to Assess the Effectiveness and Safety of GSK3862995B in Adults With Bronchiectasis
This study looks at a new drug, GSK3862995B, for adults with bronchiectasis. It aims to see if the drug is safe and effective in improving this long-term lung condition, compared to a dummy drug. We also check how people's bodies handle the medication.
For: BronchiectasisUnited States · China · Germany - RecruitingPHASE3
Pridopidine Phase 3 Study to Evaluate Efficacy and Safety in ALS
This study is testing a drug called pridopidine for people with Amyotrophic Lateral Sclerosis (ALS), also known as Motor Neurone Disease. We want to see if pridopidine can slow down how ALS progresses and if it's safe to use. Participants will take the drug or a dummy pill daily.
For: Amyotrophic Lateral SclerosisUnited States · Belgium · Canada - RecruitingPHASE3
A Study to Evaluate XEN1101 as Adjunctive Therapy in Primary Generalized Tonic-Clonic Seizures
This study is testing a new medicine, XEN1101, alongside usual treatments for a specific type of epilepsy seizure called Primary Generalized Tonic-Clonic Seizures (PGTCS). It aims to see if the new medicine helps reduce these seizures and if it's safe for people aged 12 and older.
For: Primary Generalized Tonic-Clonic SeizuresUnited States · Argentina · Australia - RecruitingPHASE1, PHASE2
A Study to Evaluate ALN-CIDEB in Adult Participants With Metabolic Dysfunction-Associated Steatotic Liver Disease or With Metabolic Dysfunction-Associated Steatohepatitis (MASLD/MASH)
This study is testing a new drug called ALN-CIDEB for people with fatty liver conditions, MASLD and MASH. It aims to check how safe the drug is, if it causes side effects, and how it affects liver fat.
For: Metabolic Dysfunction-Associated Steatotic Liver Disease · Metabolic Dysfunction-Associated SteatohepatitisUnited States · United Kingdom - RecruitingPHASE3
A Study Evaluating the Efficacy and Safety of Pegozafermin in Participants With MASH and Fibrosis (ENLIGHTEN-Fibrosis)
This study is looking into a new medicine called pegozafermin for people with a liver condition called MASH, which includes scarring (fibrosis). It compares pegozafermin to a dummy medicine to see if it's safe and helps improve the liver. We want to find better ways to treat this condition.
For: Metabolic Dysfunction-Associated Steatotic Liver Disease (MASH) / Nonalcoholic Steatohepatitis (NASH) With FibrosisUnited States · Argentina · Australia - RecruitingPHASE3
A Study to Learn About the Study Medicine (PF-08653944) in People With Obesity or Overweight and Type 2 Diabetes (T2D)
This study looks at a new medicine for adults with overweight or obesity and type 2 diabetes. It aims to see if the medicine helps with weight loss and is safe. The medicine is given as a weekly injection under the skin.
For: Obesity · Overweight and/or Obesity · OverweightUnited States · Argentina · Bulgaria - RecruitingPHASE3
Efficacy, Safety, and Tolerability of Zeleciment Basivarsen (DYNE-101) in Participants With Myotonic Dystrophy Type 1
This study looks at a new medicine called zeleciment basivarsen (DYNE-101) for Myotonic Dystrophy Type 1. We want to see if it's safe, works well, and how people tolerate it. Participants will receive either the study medicine or a dummy medicine for nearly a year.
For: Myotonic Dystrophy Type 1 (DM1) · DM1 · Myotonic DystrophyUnited States · Belgium · Denmark - RecruitingPHASE3
A Study to Evaluate the Efficacy and Safety of Pegozafermin in Participants With Compensated Cirrhosis Due to MASH
This study is testing a new medicine, Pegozafermin, for people with a severe liver condition called MASH with cirrhosis. It will check how well the medicine works and if it's safe compared to a dummy medicine.
For: Metabolic Dysfunction-Associated Steatohepatitis (MASH) / Nonalcoholic Steatohepatitis (NASH) With Compensated CirrhosisUnited States · Argentina · Australia - Active not recruitingPHASE3
A Phase 3 Study to Evaluate the Effect of Resmetirom on Clinical Outcomes in Patients With Well-compensated NASH Cirrhosis (MAESTRO-NASH-OUTCOMES)
This study is looking at a new medicine called resmetirom for people with a type of liver scarring called NASH cirrhosis. It aims to see if the medicine can prevent serious liver problems or death over about three years, compared to a dummy pill.
For: NASH · Cirrhosis, LiverUnited States · Belgium · Canada - Active not recruitingPHASE3
A Randomized, Double-blind 2-arm, Followed by an Open-label 1-arm, NEPTUNUS Extension Study to Assess the Long-term Safety and Efficacy of Ianalumab in Patients With Sjogrens Syndrome.
This study looks at the long-term safety and effectiveness of a new medicine, ianalumab, for people with Sjögren's Syndrome. It's for those who have already completed an earlier study and aims to see how well the treatment works and if it has any long-term side effects.
For: Sjogrens SyndromeUnited States · Argentina · Australia - Active not recruitingPHASE1
A Study to Assess THN391 in Subjects With Alzheimer's Disease
This study is for people aged 65-85 with early Alzheimer's and small vessel disease. It tests a new drug, THN391, or a dummy drug, over 6 months to see if it's safe and how it behaves in the body. It involves clinic visits, scans, and memory tests.
For: Alzheimer Disease, Early OnsetNetherlands · United Kingdom - Active not recruitingPHASE2
A Study to Evaluate the Effectiveness and Safety of Treatments, Either Alone or in Combination, for the Treatment of Moderate to Severe Atopic Dermatitis
This study looks at new treatments for moderate to severe eczema (atopic dermatitis). It checks how well a drug called Lutikizumab works, either alone or with other treatments, and if it's safe. Participants will get injections and have regular check-ups.
For: Atopic DermatitisUnited States · Japan · South Korea - RecruitingPHASE3
A Study of Tirzepatide in Adolescents With Obesity and Weight-Related Comorbidities (SURMOUNT-ADOLESCENTS-2)
This study looks at a medicine called tirzepatide for teenagers aged 12-17 who are overweight and have related health problems. Researchers want to see if it helps with weight and heart health when combined with healthy eating and exercise.
For: Obesity · Weight GainUnited States · Argentina · Australia - 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.
For: Carcinoma, Non-Small-Cell LungUnited States · Argentina · Australia - Active not recruitingPHASE2
Study of Bepirovirsen in Participants Living With Human Immunodeficiency Virus and Chronic Hepatitis B Virus Infection (B-Focus)
This study is looking into a new medication called Bepirovirsen for people who have both HIV and long-term Hepatitis B. It will compare Bepirovirsen to a dummy treatment (placebo) to see if it's safe and effective in improving their health.
For: Hepatitis BUnited States · Argentina · Brazil - Active not recruitingPHASE2
Proof of Concept Study on Pitolisant Effect on Autism Spectrum Disorders in Children and Adolescents
This study is looking into a new medication called Pitolisant for boys aged 6 to 17 with Autism Spectrum Disorder (ASD). It aims to see if Pitolisant helps manage ASD symptoms and if it's safe to use, compared to a dummy pill.
For: Autism Spectrum DisorderFrance · Italy · Spain - RecruitingPHASE3
Study of Oral Deucrictibant XR Tablet for Prophylaxis and Deucrictibant IR Capsule for On-Demand Treatment of Angioedema Attacks in Adults With Acquired Angioedema Due to C1 Inhibitor Deficiency
This study is testing a new medicine, deucrictibant, for adults with a rare swelling condition called acquired angioedema (AAE). It looks at whether the medicine can prevent swelling attacks and also treat them when they happen, compared to a dummy pill.
For: Acquired Angioedema Due to C1-Inhibitor Deficiency (AAE-C1-INH)United States · Australia · Austria - RecruitingPHASE2
A Study to Investigate Efficacy and Safety of SAR442970 in Patients With Crohn's Disease
This study is testing a new medicine, SAR442970, for people with moderate to severe Crohn's disease. Researchers want to see if it's safe and helps improve symptoms compared to a dummy pill. It's a key step in developing new treatments.
For: Crohn's DiseaseUnited States · Australia · Belgium - Active not recruitingPHASE2, PHASE3
ChariotMS - Cladribine to Halt Deterioration in People With Advanced Multiple Sclerosis
This study is testing a new use for a drug called Cladribine (Mavenclad) for people with advanced Multiple Sclerosis (MS). We want to see if it can slow down MS progression and improve quality of life.
For: Advanced Multiple Sclerosis · Progressive Multiple SclerosisUnited Kingdom - RecruitingPHASE3
Study to Evaluate Efficacy and Safety of Inclisiran in Children With Heterozygous Familial Hypercholesterolemia
This study is testing a new medicine, inclisiran, for children aged 6-11 with a genetic condition called heterozygous familial hypercholesterolemia (HeFH). It aims to see how well inclisiran lowers 'bad' cholesterol and if it's safe for them.
For: Familial Hypercholesterolemia - HeterozygousUnited States · Argentina · Austria - RecruitingPHASE1, PHASE2
A Study to Evaluate BMS-986470 in Healthy Volunteers and Participants With Sickle Cell Disease
This study is testing a new medicine, BMS-986470, for people with sickle cell disease and healthy volunteers. Researchers want to see if it's safe, how it works in the body, and if it helps with sickle cell symptoms. It's an early-stage study.
For: Anemia, Sickle Cell · Healthy VolunteersUnited States · Canada · France - RecruitingEARLY_PHASE1
Losartan and Social Processing
This study looks at how a single dose of Losartan, a blood pressure tablet, might affect how healthy people understand and react to social situations. Scientists want to see if it changes how we process social information.
For: Social CognitionUnited Kingdom