- Active not recruiting
A Study of Lanadelumab in Teenagers and Adults With Hereditary Angioedema (HAE)
This study looks at how a medicine called lanadelumab helps teenagers and adults in the UK with a swelling condition called Hereditary Angioedema (HAE). We're comparing their HAE attacks before and after starting this treatment to see how well it works.
For: Hereditary Angioedema (HAE)United Kingdom - Recruiting
A Pilot Study to Assess Body Mass Composition Measurement Using BIA and Muscle Ultrasound in IPF and PPF Patients on Anti-fibrotic Medications
This study looks at how anti-fibrotic medicines for lung scarring might affect your body shape, specifically your muscle and fat. It uses simple tests like body scanners and muscle scans to see if these medicines cause changes not picked up by just weighing yourself.
For: Idiopathic Pulmonary Fibrosis (IPF) · Progressive Pulmonary FibrosisUnited Kingdom - Recruiting
A Study Measuring Markers of Airway Inflammation in Breath Samples From People With Respiratory Diseases and Healthy Volunteers.
This study looks for early signs of lung cancer and other lung problems by testing breath. We're trying to find a simple, quick way to help doctors decide who needs more tests, especially for people with ongoing breathing issues.
For: Lung Cancer (Diagnosis) · Lung Cancer (Suspected or Confirmed) · HealthyUnited Kingdom - Recruiting
A Study to Learn How Nusinersen (Spinraza) Affects Participants With Spinal Muscular Atrophy (SMA) Who Took it Before or During Pregnancy And About The Health of Their Babies
This study looks at pregnant women with Spinal Muscular Atrophy (SMA) who have taken the drug nusinersen (Spinraza). Researchers want to understand how the drug affects their pregnancy and the health of their babies, as there's not enough information yet.
For: Muscular Atrophy, SpinalUnited States · United Kingdom - Enrolling by invitation
A Study to Evaluate Safety of Exposure to Wegovy During Pregnancy
This study looks at how babies and mothers are doing when the mother has taken Wegovy for obesity or being overweight during pregnancy. It compares their health outcomes with mothers who didn't take Wegovy but had similar health conditions.
For: Pregnancy · Obesity · OverweightUnited States · Spain · United Kingdom - Recruiting
Reproductive Endocrinology Oxford Study (RepOx)
This study is looking at how Polycystic Ovary Syndrome (PCOS) develops and causes problems like difficulty getting pregnant or complications during pregnancy. Researchers will study different biological information to understand PCOS better.
For: Polycystic Ovary SyndromeUnited Kingdom - Enrolling by invitation
Somatic Mutation in Chronic Liver Disease
This study looks at how liver cells change genetically during chronic liver disease, a problem that is becoming more common. Researchers hope to understand what causes these changes to help develop better ways to detect, treat, and predict outcomes for liver conditions and liver cancer.
For: Chronic Liver Disease · Somatic MutationUnited Kingdom - Active not recruiting
Long Term Follow Up to Evaluate DTX301 in Adults With Late-Onset OTC Deficiency
This study is checking on the long-term safety of a treatment called DTX301 for adults with a condition called OTC deficiency. It's for people who've already taken part in an earlier study of DTX301. Researchers want to see how participants are doing over time.
For: Ornithine Transcarbamylase (OTC) DeficiencyUnited States · Canada · France - Recruiting
Long-term Follow-up (LTFU) Study of Participants in Any iECURE Protocol Using an Investigational Product (IP)
This study is checking on the long-term well-being and health progress of children who previously took part in a study for a rare liver condition called Ornithine Transcarbamylase Deficiency (OTCD). It also looks at how the condition naturally develops in those who didn't receive the study treatment.
For: Ornithine Transcarbamylase Deficiency · Ornithine Transcarbamylase Deficiency Disease · Urea Cycle Disorders, InbornUnited Kingdom - Enrolling by invitation
Retrospective WGS Study
This study looks back at records of children and young people with cancer who had a special genetic test called whole genome sequencing. The aim is to see if this test helped doctors choose better treatments for them, to understand its benefits.
For: Cancer · Pediatric Cancer · NeoplasmUnited Kingdom - Active not recruiting
Development of Novel Clinical Endpoints in Intermediate AMD
This study looks at how to best measure changes in intermediate Age-related Macular Degeneration (AMD). It aims to find new ways to track the condition's progression, understand how it affects vision, and identify risks for it getting worse. There are no treatments involved.
For: Age Related Macular Degeneration (AMD)Denmark · France · Germany - Recruiting
A Study of Real-Life Current Standards of Care in Participants With Relapsed and/or Refractory Multiple Myeloma
This study looks at how treatments for multiple myeloma, a type of blood cancer, work in real life when the disease comes back or doesn't respond to previous treatments. It tracks patient experiences for two years to see how standard care affects them.
For: Relapsed/Refractory Multiple MyelomaAustria · Belgium · France - Recruiting
Chemotherapy Induced Cognitive Impairment
This study looks into 'chemo fog' (memory/thinking problems after chemotherapy for breast cancer). Researchers want to understand why it happens, possibly due to brain inflammation, so they can find ways to help patients in the future.
For: Breast CancerUnited Kingdom - Recruiting
Movement Dynamics of Knee Osteoarthritis and Total Knee Replacement
This study looks at how people with knee arthritis walk and move, both before and after knee replacement surgery, compared to healthy people. We want to understand why some people still struggle with activity after surgery, hoping to find better ways to help them.
For: Knee Osteoarthritis · Total Knee ReplacementUnited Kingdom - Not yet recruiting
Barriers and Enablers to Accessing Medical Care for Urinary Incontinence and Prolapse in GLobal Majority Women
This study wants to find out why women from certain ethnic backgrounds might find it hard to get help for bladder leakage (urinary incontinence) and pelvic organ prolapse. We want to understand what stops them and what helps them get the care they need.
For: Pelvic Prolapse ConditionsUnited Kingdom - RecruitingPHASE3
A Study of Vedolizumab in Children With Ulcerative Colitis (UC) or Crohn's Disease (CD)
This study looks at the long-term safety of a medicine called vedolizumab for children and young people with Crohn's disease or ulcerative colitis. It's for those who have already taken part in a previous study with vedolizumab.
For: Ulcerative Colitis · Crohn's DiseaseUnited States · Australia · Belgium - RecruitingPHASE2
A Safety and Efficacy Study of GTX-102 in Subjects With Deletion- or Nondeletion-type Angelman Syndrome (AS)
This study is looking at a new treatment called GTX-102 for Angelman Syndrome. It aims to see if the treatment is safe and if it helps improve the condition in people of different ages and genetic types. Some people will get the treatment, and some won't at first.
For: Angelman SyndromeUnited States · Argentina · Brazil - Recruiting
A Study Investigating the Effectiveness and Safety of Garadacimab for Treating Patients With Hereditary Angioedema (HAE)
This study looks at how a new medicine, garadacimab, works for people in the UK with a rare swelling condition called Hereditary Angioedema (HAE). We want to see how well it prevents swelling attacks and if it is safe to use in everyday life.
For: Hereditary AngioedemasUnited States · Austria · Canada - Recruiting
A Study of Lanadelumab in Children With Hereditary Angioedema (HAE) in Multiple Countries
This study looks at how well a medicine called lanadelumab (Takhzyro) works for children aged 2-11 with a rare swelling condition called Hereditary Angioedema (HAE). We're checking how long children go without swelling attacks when taking this medicine in their daily lives, using existing medical records.
For: Hereditary Angioedema (HAE)Argentina · France · Germany - Recruiting
DiGESTnewborn Study
This study looks at how a mother's blood sugar during pregnancy affects her baby's blood sugar after birth. It's for mothers and babies already part of the DiGest Trial. We want to understand more about sugar levels in new babies.
For: GDM · HypoglycemiaUnited Kingdom - Recruiting
Danon Disease Natural History Study
This study aims to understand Danon disease better by looking at health information from people living with it. Researchers want to learn how the condition progresses over time in males and females. There are no new treatments given; it's purely about observing the disease.
For: Danon DiseaseUnited States · Germany · Israel - Recruiting
Detecting EARLY Heart Failure in Greater Manchester
This study aims to improve early detection of heart failure in Greater Manchester. Researchers are testing a new tool that identifies people at high risk before they show symptoms. The goal is to help doctors spot heart problems sooner, potentially allowing for earlier treatment and better health outcomes.
For: Heart FailureUnited Kingdom - Active not recruiting
COVID-19 Symptom Tracker
This study uses a free app to track COVID-19 symptoms and how the illness progresses. It also looks at how isolation impacts people's well-being. The goal is to learn more about the virus and help tell it apart from common colds.
For: COVID-19United States · United Kingdom - Recruiting
A Study of Maribavir in Adults With Kidney Failure Who Have a Cytomegalovirus (CMV) Infection After Transplantation
This study looks at how safe a medicine called maribavir is for adults with serious kidney problems and a CMV germ infection after they've had an organ transplant. We're reviewing existing medical records to understand its effects without changing anyone's care.
For: Cytomegalovirus (CMV) · Kidney Disease · Kidney FailureBelgium · Estonia · France - Recruiting
InvEstigating oeStrogen Signalling and the Effect upoN the exTracelluar Matrix In pAtients With Obstructive Lung Disease
This study looks at how female hormones, particularly oestrogen, might affect lung conditions like asthma and COPD. Researchers want to understand if changes in oestrogen levels contribute to these diseases and why women might be more affected.
For: Chronic Obstructive Lung Disease · Respiratory Ilness · Estrogen DeficiencyUnited Kingdom - Recruiting
A Study to Check Liver Health in Boys With XLMTM, a Serious Genetic Muscle Condition
This study looks at liver health in boys under 18 with X-linked myotubular myopathy (XLMTM). It's an observational study, meaning researchers will collect information over about a year to understand liver, gallbladder, and bile duct issues in XLMTM.
For: X-Linked Myotubular MyopathyUnited States · United Kingdom - Recruiting
OverTTuRe: Characteristics, Treatment Patterns and Outcomes of Patients With ATTR Amyloidosis
This study looks at how ATTR amyloidosis affects people in real life. Researchers will collect information from existing medical records to understand how the condition progresses, how it's treated, and its impact on daily life and healthcare use.
For: ATTR AmyloidosisUnited States · Canada · China - Recruiting
An International Observational Study of Adults With Acute Infection
This study looks at adults admitted to hospital with infections. Researchers will collect information and samples to better understand different types of infections, how they affect people, and how best to treat them. This helps plan future studies and improve care.
For: Infectious DiseaseUnited States · Argentina · Australia - Recruiting
PIBD-SETQuality: the Inception Cohort and Safety Registry
This study looks at how well current treatments work and their safety for children and teenagers with Inflammatory Bowel Disease (IBD). It wants to understand how individual factors might affect treatment outcomes and serious complications.
For: Inflammatory Bowel Diseases · Crohn Disease · Ulcerative ColitisNetherlands · United Kingdom - Active not recruiting
A Registry for Participants With Chronic Hypoparathyroidism
This study looks at the long-term safety and how well a treatment called rhPTH(1-84) works for chronic hypoparathyroidism. It's for people of all ages who have had the condition for at least six months and are already receiving a treatment.
For: HypoparathyroidismUnited States · Austria · Canada - Active not recruitingNA
A Study About Antibody Levels and Biomarkers in the Blood in People With Late-onset Pompe Disease
This study looks at blood markers and antibody levels in people with late-onset Pompe disease. It helps researchers understand the disease better and how bodies react to treatments, even without new medicines given during the study.
For: Pompe Disease (Late-onset)United States · Australia · Brazil - Not yet recruiting
Sepsis in Oncology Patients
This study looks at how the body's defence system (immune system) works in cancer patients who get a serious infection called sepsis. Researchers want to understand why some cancer patients get sepsis and how their bodies react.
For: Sepsis in Cancer PatientsUnited Kingdom - Recruiting
ChemoINTEL Assay Algorithm Development Study: In-Vitro Cytotoxic Drug Induced Apoptosis Correlation with Patient Clinical Response to Administered Chemotherapy in Patients
This study aims to create a tool to predict how well ovarian cancer patients will respond to chemotherapy. It uses samples from surgery and tracks their treatment to help doctors choose the best personal treatment plan.
For: Ovarian CancerUnited Kingdom