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Sheffield city is located in United Kingdom. Currently, 20 clinical trials are being conducted in this city.

Sheffield, located in South Yorkshire, England, is renowned for its rich industrial heritage, particularly in steel production, earning it the nickname “Steel City.” This city is the birthplace of stainless steel and crucible steel, revolutionizing industries worldwide. Surrounded by the natural beauty of the Peak District National Park, Sheffield is one of the greenest cities in the UK, boasting numerous parks and woodland areas. It also has a vibrant cultural scene, with the iconic Sheffield Theatres complex and the annual Sheffield Doc/Fest attracting global attention.

  • CT-EU-00112473

    Study on AOC 1020 for Facioscapulohumeral Muscular Dystrophy Treatment

    This study is aimed at exploring the safety, tolerability, and potential effectiveness of a novel treatment known as AOC 1020 for adults affected by Facioscapulohumeral Muscular Dystrophy (FSHD), covering both FSHD Type 1 and FSHD Type 2.

    The structure of the trial is randomized, double-blind, placebo-controlled, ensuring that participants are randomly allocated to receive either the AOC 1020 treatment or a placebo (a substance without therapeutic effect, serving as a control in the testing of new drugs) via an intravenous (IV) infusion. The objective is to keep both participants and study staff unaware of which treatment is being administered to maintain impartiality.

    The detailed study is organized into three segments: Part A is dedicated to dose titration utilizing both a single and multiple dose schedule for one cohort. Part B investigates single-ascending and multiple-ascending dose designs across two cohorts. Conclusively, Part C is designed with a parallel, multi-dose cohort approach for one cohort. Each segment of the study lasts for 12 months, encompassing approximately 9 months of active treatment followed by a 3-month follow-up period.

    After the conclusion of the active treatment and follow-up, participants are presented with the opportunity to enter a planned open-label extension, which allows continued access to the treatment being investigated. Those opting out of the extension are subjected to a 6-month safety follow-up period.

    The primary concern of the study is the monitoring of the incidence of treatment-emergent adverse events throughout the duration of the study, up to Day 365, to ensure the safety and well-being of all participants.

    United Kingdom
  • A non-invasive test to assess adrenal function in children with asthma using inhaled steroids

    The study is focused on developing a new way to check whether the body is producing enough of an essential stress hormone called cortisol, especially in children using inhaled steroids to treat asthma. Steroid inhalers are common, but there is concern that they may prevent the body from producing enough cortisol when it is really needed, such as during illness. This could be serious. Currently, doctors use a test called the **short synacten test (SST)** to check whether the adrenal glands (which produce cortisol) are functioning properly. But this test requires needles and can be stressful and unpleasant, especially for children.

    The goal of the study is to create a **non-invasive** version of this test. Instead of needles, doctors want to use a nasal spray of a drug called **Synacthen**, which stimulates the adrenal glands, and then measure cortisol levels in saliva. This new method could make testing easier, faster and more convenient for children.

    Doctors will start by testing this new approach in adults to understand how well it works and how it compares to the traditional test. They will look at how the body absorbs the drug and how it affects cortisol levels. They will then perform the same tests on children to make sure it is safe and effective for them too.

    United Kingdom
  • Clinical Study on Growth and Health in Children with Achondroplasia

    This is a long-term, multi-center, observational study for children aged 2.5 to <17 years who have been diagnosed with achondroplasia. The main objective of the study is to evaluate various aspects of the condition, including growth, medical complications related to achondroplasia, health-related quality of life, body pain, functional abilities, cognitive functions, and treatments received by the participants. The study will monitor changes in height Z score and upper to lower body segment ratio over a period of up to 2 years. No study medication will be administered to the participants during this observational study.

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  • CT-EU-00091315

    Testing infigratinib in children aged 3 to 11 years with achondroplasia

    This study focuses on evaluating the medication Infigratinib in young children aged 3 to 11 diagnosed with Achondroplasia who previously participated in the PROPEL study for at least 6 months. The primary objectives of this study are to assess the safety profile of Infigratinib in this specific age group, evaluate the children’s tolerance to potential side effects, and investigate the therapeutic efficacy of the medication for treating Achondroplasia. During the study, doses of Infigratinib will be incrementally increased to determine the optimal and most effective dosage for the participants.

  • A comprehensive evaluation of retatrutide in obesity and associated comorbidities

    This study evaluates the effectiveness and safety of a new drug, retatrutide, in individuals with obesity or overweight, including those with knee osteoarthritis or obstructive sleep apnea. Lasting about 89 weeks, the trial involves randomized assignment of participants to either receive retatrutide or a placebo. The main goals are to observe changes in body weight, knee pain in osteoarthritis, and sleep apnea severity. The study also examines various secondary outcomes like changes in BMI, waist circumference, and blood pressure. The trial aims to provide new insights into weight management and associated health conditions, offering hope for improved treatments.

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  • Testing safety and tolerability of a new Amyotrophic Lateral Sclerosis medication

    This study aims to assess the safety and tolerability of a novel medication, designated as QRL-201, in individuals diagnosed with Amyotrophic Lateral Sclerosis (ALS). The drug is administered via intrathecal administration, directly into the cerebrospinal fluid surrounding the spinal cord. The study comprises 64 participants divided into 8 groups. Within each group of 8 individuals, 6 will receive the active medication, while 2 will receive a placebo, a substance with no therapeutic effect. The investigation includes the monitoring and reporting of any observed side effects or serious health events. Additionally, assessments will be conducted to measure the peak concentration of the critical drug in the bloodstream, the time taken to reach this peak concentration, and the overall quantity of the drug present in the bloodstream.

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  • Evaluating the efficacy of macitentan in the treatment of high pulmonary artery blood pressure

    This study is checking if a 75 mg dose of a medicine called Macitentan can do a better job for patients with a lung condition called Pulmonary Arterial Hypertension (PAH) compared to a 10 mg dose. The main goal is to see if the 75 mg dose can better delay the patient’s first major health event related to PAH. Major events include things like unplanned hospital stays related to PAH, or their PAH getting worse. The researchers will check if patients’ PAH gets worse by looking at things like their physical exercise ability and signs of heart failure. The study also looks at how patients’ symptoms change from day to day.

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  • Testing gefurilimab treatment in patients with myasthenia gravis

    This study is looking at a potential treatment for a disease called generalized myasthenia gravis (gMG) in adults. It’s called gefurulimab (ALXN1720). For our study, researchers will divide participants into two groups, each of which will be treated differently. One group will receive the new drug. Importantly, the researchers conducting the study will not know who received which treatment. This ensures the fairness and accuracy of the results. Participants’ health will be closely monitored to ensure that ALXN1720 is safe. The main goal is to check whether new mediations are effective. It will be measured by checking whether patients’ condition has improved compared to when the study started. This will take approximately 26 weeks.

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  • Sotatercept treatment study for new Pulmonary Hypertension patients

    This study is an important test where doctors are looking at how a drug called Sotatercept can help people struggling with Pulmonary Arterial Hypertension (or PAH for short). The goal is to see whether this drug can delay or prevent the condition from getting worse. PAH makes it really hard for patients to breathe because it affects lungs and heart. The testing process is being done in a fair and careful way. Half of the patients will get the drug, and the others will get a ‘placebo’ which doesnt contain any medicine. The doctors will look at the effect on patients over time. The study is looking specifically at patients who were recently diagnosed with PAH and are at risk for the disease to get worse.

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  • Testing retatrutide’s effect on obesity and knee osteoarthritis

    This research is about a new medication called Retatrutide (LY3437943), which will be taken once a week by people who are overweight or suffer from obesity, and also have a condition where their knee hurts due to wear and tear, known as osteoarthritis of the knee. This is a third phase study, where we test how well the drug works and how safe it is. Unlike some studies, participants will not know whether they are taking the medication or a placebo (a pill that looks the same but doesn’t contain the medication). The study will last about 77 weeks.

    SpainUnited Kingdom
  • Testing zamaglutenase for gluten breakdown in celiac disease treatment

    This study focuses on a medicine called zamaglutenase (TAK-062) that may help people with celiac disease. Celiac disease is a condition where the body can’t process gluten, which is found in wheat, rye, and barley. When people with celiac disease eat gluten, their immune system reacts by damaging the small intestine. TAK-062 is meant to break down the gluten in a person’s stomach, possibly helping the body cope better. The study needs around 357 volunteers, who will be split into two groups at random. Both groups will involve adults over 18 years of age. The first group will get a placebo (a medicine-like substance with no actual medicine) and a gluten bar, and the other group will get a dose of TAK-062 alongside the gluten bar. After some time, a committee will check the results of the first group. Depending on what they find, the study may then include teenagers, and the second group will start. Just like any medicine, TAK-062 may or may not cause side effects. Even seemingly unrelated health problems that happen during the study are important, as they could be linked to the medicine, even if it’s not clear at first. This study aims to find clear and truthful results about how TAK-062 works for people with celiac disease.

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  • Long-term study of infigratinib for the treatment of achondroplasia in patients aged 3 years to 18 years

    In the PROPEL OLE study, researchers are evaluating the long-term efficacy and safety of Infigratinib in children with Achondroplasia (ACH). Infigratinib targets specific proteins known as FGFR 1-3, which play a crucial role in growth and development. This extension study involves children who have previously participated in a trial and potentially includes those who have not received prior treatment. Before initiating the study, new participants will undergo a six-month monitoring period in the PROPEL study to assess their growth. Additionally, researchers will evaluate the impact of the drug on the children’s quality of life.

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  • Fenfluramine Hydrochloride study for seizure control in cyclin-dependent kinase like-5 (CDKL5)

    This is studying a new substance called Fenfluramine Hydrochloride (ZX008) in children and adults experiencing seizures with cyclin-dependent kinase like-5 (CDKL5) deficiency disorder (CDD). The research has two parts. In the first part, lasting 20 weeks, some participants receive the new drug, while others receive a placebo (inactive substance). This part aims to determine if ZX008 is effective and safe. The second part lasts 54 weeks and is open-label, meaning everyone is aware they are receiving the active drug. It helps assess long-term effects. If participants complete the first part, they can enroll in the second part, including an open-label treatment period (52 weeks) and a taper period (2 weeks).

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  • Study on treating uncontrolled bleeding with bentracimab

    A trial investigating bentracimab’s ability to reverse ticagrelor’s effects in patients with severe bleeding or those needing urgent surgery. The study, involving at least 200 patients from various regions, tests if bentracimab can quickly counteract ticagrelor, improving patients’ conditions or allowing for safe surgeries. Patients receive a thorough follow-up to ensure the drug’s effectiveness and safety, aiming to provide a reliable treatment for critical, time-sensitive medical scenarios.

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  • Comparison of different treatments for follicular lymphoma

    This study involves comparing two treatments for a condition known as follicular or marginal zone lymphoma, both of which are types of cancer affecting lymph cells. The first treatment combines zanubrutinib with an antibody called Anti-CD20, while the second treatment involves lenalidomide and rituximab. These treatments are intended for patients who have not responded or have stopped responding to conventional treatment. One of the primary objectives of the study is to determine which treatment is more effective in preventing the cancer from progressing, referred to as progression-free survival. Additionally, the study aims to assess the impact of these treatments on the patients’ quality of life, evaluating various aspects through questionnaires related to physical and emotional well-being, symptoms, and the ability to perform normal activities.

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  • Testing bemarituzumab treatment on solid tumors

    This study is testing a new drug, bemarituzumab, for people with certain advanced solid tumors that are high in a protein called FGFR2b. It is for adults whose cancer has not responded to previous treatment or cannot be surgically removed. The main goal of this test is to see if bemarituzumab can help fight these cancers and to make sure it is safe for people to take. Participants receive the drug through a drip. This study will use special imaging methods, such as computed tomography or magnetic resonance imaging, to see how the tumors respond to the drug. If the drug helps and the tumor shrinks or doesn’t grow, researchers will track how long it lasts.

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  • Study on dazostinag & pembrolizumab for advanced solid tumors

    The purpose of this study is to test a new drug called dazostinag. A study is being conducted to see whether this drug is helpful in adults with advanced forms of solid cancer. Some people are given dazostinag alone, while others are given it with another medicine called pembrolizumab. Scientists’ focus here is on finding out whether these drugs cause any side effects, and finding out what the maximum dose is that people can take without serious side effects. The study consists of two parts, including a dose escalation phase and a dose escalation phase. In the first part, the dose of dazostinag will be gradually increased, given alone or in combination with pembrolizumab. In the second part, Dazostinag will be tested with pembrolizumab and other anticancer drugs. This section will focus on patients with specific cancers that are difficult to remove or have spread to other parts of the body.

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  • A study of lanifibranor treatment in adults with nonalcoholic fatty liver disease

    This study is a two-part trial looking at how well a drug called Lanifibranor works for adults suffering from liver diseases known as NASH and fibrosis stages F2 and F3. In the first part of the trial, the researchers will compare the effects of Lanifibranor with a placebo, which is a substance with no medical effect. The researchers will be looking at how much the drug can improve the health of the liver. The second part of the study will continue the use of Lanifibranor to see if there are any long-term safety issues. Apart from studying the drug’s effectiveness in resolving NASH and reducing live fibrosis, the trial will also be observing how it affects other aspects of liver health, diabetes, blood sugar and fat levels, liver stiffness and patients’ quality of life.

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  • A study on a new treatment for Hypophosphatasia

    This trial focuses is on evaluating the efficacy of a novel drug, ALXN1850, in assisting adolescents and adults diagnosed with Hypophosphatasia (HPP), a condition characterized by the deficiency of alkaline phosphatase enzyme. ALXN1850 aims to substitute this lacking enzyme and will be administered through subcutaneous injections. The primary objective is to assess the performance of the new drug in comparison to a placebo. The trial is conducted in a blinded manner, ensuring that participants remain unaware of whether they are receiving the actual drug or the placebo. The paramount goal is to ascertain the safety and effectiveness of the new treatment.

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  • Evaluating safety and effects of anifrolumab in systemic sclerosis treatment

    The DAISY trial, a phase III study, is focused on evaluating the use of anifrolumab in the treatment of systemic sclerosis in adults. Its purpose is to measure the effectiveness and safety of the drug and observe its effect on symptoms and progression of the disease. Participants undergo comprehensive screening, treatment and follow-up. The study specifically focuses on response to treatment, changes in lung function and potential side effects, providing important information on the treatment of systemic sclerosis.

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