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A Study to Evaluate a Novel Gene Therapy in Patients With Relapsed and Refractory Multiple Myeloma
The goal of this clinical trial is to evaluate the safety, tolerability, and recommended Phase 2 Dose (RP2D) of KLN-1010 in patients with relapsed or refractory multiple myeloma.
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Long-Term Extension Study of Vosoritide to Treat Children With Hypochondroplasia
The purpose of this study is to evaluate the long-term safety and efficacy of daily doses of vosoritide in participants with HCH
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Psilocybin-Assisted Psychotherapy in Cancer Patients With Adjustment Disorder
This study is assessing the efficacy and safety of PEX010 in psilocybin-assisted psychotherapy for the treatment of adjustment disorder due to cancer diagnosis. Who is it for? This study is for people who are aged between 18 and 80 years old and suffer from anxiety after adjusting to an acutely stressful event of their cancer diagnosis. This is called adjustment disorder. Study details Participants in this study will be randomly allocated by chance (similar to flipping a coin) to one of three groups: a 25mg PEX010 dose group, a 10 mg PEX010 dose group or a 1mg PEX010 dose group. Participants will be allocated a dose that will be administered during their psilocybin-assisted psychotherapy (PAP) dosing session. The PAP dosing session will run approximately 8 hours, with PEX010 administered at Day 14 (dosing day). At Week 10, non-responders that continue to meet the study eligibility criteria may commence an additional PAP cycle (at 25 mg PEX010). A maximum of 2 PAP cycles may be administered. Long term follow up will comprise of a study visit at 3 months post Week 10 (of the final cycle) to assess safety and tolerability of PEX010. It is hoped that this research will develop important scientific knowledge that could contribute to the development of a potential new treatment for anxiety and depression after adjusting to an acutely stressful event such as a cancer diagnosis.
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Clinical Trial to Evaluate Safety, Tolerability, and Pharmacokinetics of SOF-SKN in Healthy Participants
The goal of this study is to investigate the safety and tolerability of the topical application of SOF-SKN in healthy volunteers. The study will be divided into part 1 and part 2. Part 1, is a single ascending dose (SAD) design, while Part 2 is a multiple ascending dose (MAD) design.
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Study to Evaluate the Safety and Immunogenicity of Investigational Vaccines Using the RSV (Respiratory Syncytial Virus) Monovalent Antigen in Healthy Participants 18 to 49 Years of Age
The purpose of this study is to evaluate the safety and immunogenicity of different liponanaoparticle (LNP) formulations of modified ribonucleic acid (mRNA) vaccines using the RSV pre-F antigen in healthy participants 18 to 49 years of age. -Each participant will remain in the study for approximately 6 months. * The study intervention will be administered as a single intramuscular (IM) dose in the upper arm on Day 1 (D01). * The visit frequency will include 1 Screening Visit, 4 planned site visits to occur on D01, D03, D08, and D29, and 2 telephone calls to occur at Month (M)3 and M6.
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A Study to Test Vicadrostat (BI 690517) Taken Together With Empagliflozin in People With Type 2 Diabetes, High Blood Pressure, and Cardiovascular Disease
This study is open to adults with type 2 diabetes, high blood pressure, and cardiovascular disease. People can join the study if they have these conditions and do not have a history of heart failure. The purpose of this study is to find out if a medicine called vicadrostat, when taken with empagliflozin, helps reduce cardiovascular risk in people with these conditions. The study will compare this combination to a placebo version of vicadrostat with empagliflozin. Participants are put into 2 groups randomly, which means by chance. One group takes vicadrostat and empagliflozin tablets, and the other group takes placebo tablets with empagliflozin. Placebo tablets look like vicadrostat tablets but do not contain any medicine. Participants take a tablet once per day for 2 and a half years and up to 4 years and 3 months. All participants also continue their medication for type 2 diabetes, high blood pressure, and cardiovascular disease. Participants have an equal chance of receiving the study medicine or placebo. Participants are in the study for up to 4 years and 3 months. During this time, they visit the study site regularly. During these visits, doctors collect information about participants' health and take blood samples. The doctors document when participants experience cardiovascular events. The doctors also regularly check participants' health and take note of any unwanted effects.
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A Study of AZD2962, an IRAK4 Inhibitor (IRAK4 [a Body Protein] Blocker), in Participants With Haematologic Neoplasms (Blood Cancers)
The purpose of the study to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics, and preliminary efficacy of AZD2962, an Interleukin-1 Receptor-Associated Kinase 4 (IRAK4) inhibitor, as monotherapy and in combination with other agents in participants with haematologic neoplasms.
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Adding IL-2 to Tebentafusp to Eradicate Cancer Progression
A recent clinical trial found that after 36 months, patients taking tebentafusp had a median survival of 21.6 months, compared to 16.9 months for those in the control group. Since recruitment for tebentafusp in metastatic uveal melanoma (mUM) has ended, a new trial is starting to test whether adding IL-2 can help overcome resistance to tebentafusp and improve its effectiveness. This study aims to answer: 1. Can combining tebentafusp with IL-2 improve tumor response and overall survival? 2. What are the benefits and side effects of this combination therapy? All participants will receive both IL-2 and tebentafusp in a 28-day treatment cycle. The dosing schedule is as follows: Cycle1: Day1-3 IL-2 Day4 Tebentafusp Day 10 IL-2 Day 11 Tebentafusp Day 17 IL-2 Day 18 Tebentafusp Day 24 IL-2 Day 25 Tebentafusp Cycle 2 \& thereafter Day 1 IL-2 Day 2 Tebentafusp Day 8 IL-2 Day 9 Tebentafusp Day 15 IL-2 Day 16 Tebentafusp Day 22 IL-2 Day 23 Tebentafusp
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A Study to Compare the Combination of BMS-986504 With Pembrolizumab and Chemotherapy Versus Placebo Plus Pembrolizumab and Chemotherapy in First-line Metastatic Non-small Cell Lung Cancer Participants With Homozygous MTAP Deletion
The purpose of this study is to compare the clinical benefit of the combination of BMS-986504 (a selective MTA-cooperative inhibitor of PRMT5) plus pembrolizumab and chemotherapy versus placebo plus pembrolizumab and chemotherapy in first-line metastatic non-small cell lung cancer participants with homozygous MTAP deletion
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A Clinical Study to Determine What Effect the EyeCool Treatment Has on the Eye's Surface Anatomy and Inflammation Response After Being Treated for Having Chronic Ocular Surface Pain (COSP)
The goal of this clinical trial is to understand what effect a cold treatment with an investigational device, ETX-4143, has on the anatomical structures and the inflammatory response of the surface of the eye in those people who suffer from chronic ocular surface pain (COSP). The trial will also tell us more about the safety of this investigational device. The main questions this study aims to answer are: * What anatomical changes happen to corneal surface nerves after treatment with ETX-4143 * What changes in inflammatory mediators and cell response take place after treatment with ETX-4143 Researchers will obtain images of the corneal surface nerves for analysis using a confocal microscope and collect tear bio samples for analysis. Participants will: * Be screened for having chronic ocular surface pain * Will be treated with ETX-4143 cold treatment for 4 minuets * Fill out a weekly questionnaire on eye pain for 12 weeks * Be seen in the clinic 2 weeks, 6 weeks, and 12 weeks after treatment to have images of the corneal surface taken, and to collect a tear bio sample.