The purpose of the study is to assess the efficacy, safety, and tolerability of zeleciment basivarsen (DYNE-101) for the treatment of myotonic dystrophy 1 (DM1).

Type: Interventional

Start Date: Apr 2026

open study

A prospective, open-label, single arm interventional trial to establish normative reference ranges for urodynamics parameters in healthy adult participants using using the Glean Urodynamics System.

Type: Interventional

Start Date: Mar 2026

open study

The goal of this clinical study is to test whether a structured prehabilitation program can be successfully used by adults with pancreatic cancer who are receiving chemotherapy before planned surgery. Prehabilitation is a program designed to improve a person's physical fitness, nutrition, and symptom control before surgery. The study's main hypothesis is that patients with pancreatic cancer can take part in and adhere to a multimodal prehabilitation program during chemotherapy, and that doing so may improve physical function, patient experience, and early recovery after surgery.

Type: Interventional

Start Date: Mar 2026

open study

The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics of HJB647 at two different doses in participants with chronic stable heart failure with reduced or mildly reduced ejection fraction (HFrEF/HFmrEF).

Type: Interventional

Start Date: Mar 2026

open study

The goal of this observational study is to establish a patient registry and a biorepository (sample collection and storage) to investigate health disparities, access, and barriers to cancer screening and early detection technologies. The registry and biorepository will serve as a resource to support Cancer Early Detection (CED) screenings and future research focused on communities at increased risk for cancer. The study seeks to address: •Barriers and disparities in cancer prevention, screening, and treatment, particularly in historically underrepresented populations. Participants will: - Attend a minimum of five clinic visits over a five-year period for scheduled annual assessments while actively enrolled in the study. - Complete questionnaires at each visit that collect information on their medical history, cancer history, and family cancer history. - Allow relevant health information from their electronic health records (EHR) to be collected and reviewed. - Provide blood, saliva, and stool samples for research purposes. - Enter a long-term follow-up period for an additional five years.

Type: Observational [Patient Registry]

Start Date: Mar 2025

open study

The goal of this clinical trial is to learn if drug CADI-05, when used together with pembrolizumab (an FDA approved immunotherapy), can help treat locally advanced head and neck squamous cell carcinoma (LA-HNSCC) in adults. It will also learn about the safety of drug CADI-05. The main questions it aims to answer are: - Does using CADI-05 together with pembrolizumab help the immune system fight cancer better and lead to better results for patients? - What side effects or health problems might happen when people receive these two treatments? Participants will: - Get pembrolizumab by IV (through a vein) once on day 1 of week 1 and again day 1 of week 4. This is standard of care treatment. - Get CADI-05 as a small injection into the skin once a week for 5 weeks. This is the experimental (research) treatment. - Visit the clinic every week for treatments, checkups and tests for 5 weeks. - Have surgery between week 6 and week 7. - Return to the clinic once for a follow-up visit about 30 days after surgery.

Type: Interventional

Start Date: Apr 2026

open study

This is a phase 3 randomized, double -masked study comparing the efficacy of EYP-1901 against Aflibercept.

Type: Interventional

Start Date: Feb 2026

open study

This research is being done to compare the red blood circulation survival in healthy adult volunteers between the 51 chromium (51Cr) red blood cell (RBC) labeling method and the Biotin (BioRBC) red blood cell (RBC) labeling method to determine if biotinylated red blood cells (BioRBC) is an acceptable non radioactive alternative to 51 chromium (51Cr) radiolabeling for regulatory pharmacokinetic studies of red blood cell products.

Type: Interventional

Start Date: Apr 2026

open study

The purpose of this trial is to evaluate safety, tolerability, pharmacokinetics and pharmacodynamic impact of PrP-siRNA in symptomatic prion disease patients.

Type: Interventional

Start Date: Apr 2026

open study

The purpose of this study is to evaluate the absorption, metabolism, and excretion of DSP-5336 following a single oral administration of the study drug in patients with hematologic malignancies whose disease has progressed after available standard therapies.

Type: Interventional

Start Date: Mar 2026

open study

The purpose of this study is to find better ways to help support families in their hopes during cancer treatment. Primary Objective - To characterize themes related to how patients and parents/caregivers narrate their experience of 'hope' when receiving cancer therapy on a phase 1/2 clinical trial, with a focus on whether, why, when, and how patients' and caregivers' hopes adapt to changing circumstances. - To engage patients, caregivers, and clinicians in focus groups to identify strengths, weaknesses, opportunities, and threats to hope during phase 1/2 clinical trial participation and facilitate the co-design of a stakeholder-driven supportive intervention related to hope based on focus group recommendations. Secondary Objective - To describe health care provider perspectives on patient and family hope and goal-care concordance in the context of phase 1/2 clinical trials.

Type: Observational

Start Date: Apr 2026

open study

This will be a 3-visit, single-site, randomized, single-masked, bilateral wear, non-dispensing, crossover study which will evaluate visual performance with different magnitudes of scatter.

Type: Interventional

Start Date: Feb 2026

open study

The goal of this study is to learn about the effects of eating kimchi on the gut health of healthy adults in the USA. The investigators will be researching the changes in the gut microbiome, biomarkers of gut health and cardiometabolic health after consuming fermented and unfermented cabbage. The main questions it aims to answer are: Does eating kimchi (fermented cabbage) result in enrichment of lactic acid bacteria in the stools of participants? Does eating kimchi result in metabolic changes in the gut microbiome, biomarkers of gut and cardiometabolic health of participants? Researchers will compare a group of participants eating fermented cabbage (kimchi) daily to a group of participants eating non-fermented cabbage daily. Participants will: Eat kimchi or cabbage daily for 3 weeks. Visit the study site for brief visits up to 5 times. Have blood drawn and provide a fecal sample 2 times - at beginning and end of the 3 week study. Keep occasional records of food intake and questionnaires about any gastrointestinal symptoms that participants may have.

Type: Interventional

Start Date: Mar 2026

open study

The purpose of this study is to assess the effectiveness and safety of a single dose of IPN10200 compared to placebo (double-blind phase) and how well and safely repeat doses of IPN10200 work over time (open-label phase) in adult participants with moderate to severe glabellar lines. Glabellar lines are wrinkle-like lines that appear between the eyebrows and can become more noticeable with age or repeated facial expressions. They may affect a person's appearance and confidence. All participants in the double-blind phase will receive IPN10200 or placebo during the first treatment cycle. De novo participants in the open-label phase will receive IPN10200 during the first treatment cycle. Some participants may receive additional treatment cycles with IPN10200 depending on their eligibility. There will be 3 periods in this study: - A screening period (up to 20 days) to assess whether the participant can take part, requiring at least 1 visit to the study centre. - A treatment period where participants may receive up to 4 treatment cycles. In the double-blind phase, participants receive a single treatment of IPN10200 or placebo. In the open-label phase (rollover participants from double-blind), eligible participants may receive additional cycles of IPN10200. In the open-label phase (de novo participants), participants will receive IPN10200 in the first cycle and eligible participants may receive additional cycles of IPN10200. Requires multiple visits during the first month followed by 1 visit every month. - A follow-up period (24 weeks) after the last injection where participants' health will be monitored. Participants will undergo health measurements and observation, including blood sampling, physical examinations, clinical evaluations and electrocardiograms (ECG: recording of the electrical activity of heart). They will also be asked to fill in questionnaires and keep a diary. Each participant will be in this study for up to 107 weeks. Participants may withdraw consent to participate at any time.

Type: Interventional

Start Date: Feb 2026

open study

Macular dystrophies are a group of inherited eye conditions that affect the macula. The macula is in the center of the retina, the light sensitive part at the back of the eye. In people with macular dystrophies, some of the cells in the macula gradually stop working and may die over time. This leads to vision loss in the center of the eye. Side vision (peripheral vision) is mostly unaffected. Stargardt disease (STGD) is a type of macular dystrophy which is caused by 1 faulty gene (ABCA4). Vision loss most typically happens in childhood, but many people do not develop it until they are adults. As well as STGD, there are other macular dystrophies that look very similar to STGD but that are caused by many other different genes. Together, STGD and STGD-like conditions can be called STGD-type macular dystrophies. This is because they look the same clinically and have similar symptoms. Since different genes can cause these conditions, genetic testing is the only way to be sure which specific condition a person has. In this study, researchers want to learn if the disease progresses in a similar way in people with STGD and STGD-like macular dystrophies. People taking part in the study will continue to manage their condition, as agreed with their own doctor. People will visit their clinic every 6 months to have various standard eye tests and imaging. The information collected will include questions about people's wellbeing, general health, medication and supplements taken, and daily activities. Children over 6 years old and adults with STGD-type macular dystrophies may take part in this study. They will be in the study for up to 24 months (2 years). The study sponsor (Astellas) will not decide how people's condition is managed. However, the sponsor will provide instructions on when people visit their clinic and what is recorded during the study. If available, medical records, clinical and imaging data from previous visits going back 24 months will also be reviewed.

Type: Observational

Start Date: Feb 2026

open study

This trial will study the safety and tolerability and disease survival rates in adult patients with recurrent/metastatic (R/M) HNSCC when treated with carboplatin or cisplatin, paclitaxel, and toripalimab.

Type: Interventional

Start Date: Apr 2026

open study

This study evaluates the efficacy and safety of investigational study drug ALK-001 in participants 8 to 45 years of age, inclusive, with symptoms and signs of autosomal recessive Stargardt disease (STGD)

Type: Interventional

Start Date: Apr 2026

open study

The purpose of this study is to assess the efficacy and safety of brenipatide when administered with standard of care (SoC) compared to placebo plus SoC for treatment of schizophrenia. The trial is divided into three periods as follows: Screening period will last approximately 1 month, treatment period will last a maximum of 12 months, and the follow up period will last approximately 2 months. The length of time of your study participation may last up to approximately 15 months.

Type: Interventional

Start Date: Feb 2026

open study

Pancreatic cancer is difficult to diagnose early. By the time people have been diagnosed, the cancer has usually spread to other parts of the body (metastatic). The standard treatment is chemotherapy, but other treatments are needed to improve outcomes in people with pancreatic cancer. The first treatment that people usually receive is chemotherapy. At the time this study started, some of the main standard chemotherapies for pancreatic cancer were mFOLFIRINOX or NALIRIFOX. Genes give your body instructions on how to make proteins. Proteins are needed to keep the body working properly. Many types of cancer are caused by changes in certain genes, making them faulty. Many people with pancreatic cancer have a faulty KRAS gene. One such change in the KRAS gene is called a G12D mutation. Researchers are looking for ways to stop the actions of abnormal proteins made from the KRAS G12D mutation. This study is about setidegrasib given with chemotherapy in people with pancreatic cancer who have the KRAS G12D mutation. Before setidegrasib can become an approved treatment, clinical studies need to be completed to understand how it works and how safe it is. The main aim is to learn if people who are given setidegrasib with chemotherapy live for longer than people who are given placebo with chemotherapy. Other aims are to learn if setidegrasib delays the cancer and symptoms returning, how the body processes setidegrasib, and its safety, when given with chemotherapy. People in this study will be adults with metastatic pancreatic cancer with the G12D mutation in their KRAS gene. Surgery or radiotherapy will not be an option to cure their cancer. People cannot take part if the cancer cells have spread to the thin tissue covering the brain and spinal cord (leptomeningeal disease), have symptoms of cancer in the brain or nervous system, or have recently had some other cancers that required treatment. In this study, people are given either setidegrasib with mFOLFIRINOX or NALIRIFOX chemotherapy, or a placebo with mFOLFIRINOX or NALIRIFOX chemotherapy. Whether people receive setidegrasib or placebo is decided by chance. The study doctor decides which chemotherapy (mFOLFIRINOX or NALIRIFOX) people receive. All of the study treatments are given slowly through a tube into a vein (infusion). People will continue to receive study treatment until their cancer gets worse, they can't tolerate the study treatment, they start other cancer treatment, they or the doctor decides the person should stop receiving study treatment, or sadly they pass away. There will be safety checks at each visit, and the doctors will continue to check for medical problems and people's wellbeing throughout the study.

Type: Interventional

Start Date: Feb 2026

open study

This phase II trial studies how well giving zanidatamab before surgery (neoadjuvant) works in treating patients with colon and rectal cancer that is human epidermal growth factor receptor 2 positive (HER2+ve) who are planned for curative intent treatment. Zanidatamab is a monoclonal antibody that may interfere with the ability of tumor cells to grow and spread. A monoclonal antibody is a type of protein that can bind to certain targets in the body, such as molecules that cause the body to make an immune response (antigens).

Type: Interventional

Start Date: Apr 2026

open study

The primary purpose of this study is to determine the impact of whole-fat dairy consumption on cardiovascular and metabolic risk factors and peripheral vascular function in adults with metabolic syndrome.

Type: Interventional

Start Date: Apr 2026

open study

The goal of this clinical trial is to compare transgluteal sciatic nerve block to standard of care to treat sciatic back pain in adult patients who present to the emergency department. The main question it aims to answer is: Is a transgluteal sciatic nerve block better than standard of care in improving pain in ER patients with sciatic back pain? If there is a comparison group: Researchers will compare patients who receive transgluteal sciatic nerve blocks to patients who receive standard of care to see if pain scores improve in ER patients with sciatic back pain. Participants will be randomized into the transgluteal sciatic nerve block group or the control group (standard of care). Participants will be asked to answer questions about their pain and will be asked to walk a timed short distance.

Type: Interventional

Start Date: Apr 2026

open study

The purpose of this clinical study is to find out if UBT251 is safe and effective for treating people who are living with overweight or obesity. Participants will get either UBT251 (the treatment being tested) or Placebo (a treatment that has no active medicine in it), which treatment participants get is decided by chance.

Type: Interventional

Start Date: Feb 2026

open study

This is a pilot randomized controlled trial to assess the feasibility, acceptability, appropriateness, and structure of the SONATA intervention. In addition, it will assess the preliminary efficacy of SONATA compared to enhanced usual care among 70 older adults with advanced cancer.

Type: Interventional

Start Date: Apr 2026

open study

This study is being done to learn more about a new medicine called PF-08634404 and how well it works when given with chemotherapy to people with gastroesophageal cancer that is locally advanced (spread to nearby tissues) or has spread to other parts of the body. To join the study, participants must meet the following conditions: Be 18 years or older. Have locally advanced or metastatic gastric, gastroesophageal junction or esophageal adenocarcinoma Be treatment naïve for advanced or metastatic disease Be in good physical condition and have healthy organs based on medical tests. The study has two parts: - In the first part, researchers will check how safe the study medicine in combination with chemotherapy is and how well people respond to it. - In the second part, they will compare study medicine plus chemotherapy to another approved treatment (nivolumab plus chemotherapy) to see which works better. The treatment will be given in repeated time periods called cycles.

Type: Interventional

Start Date: Apr 2026

open study