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NCT ID: NCT06136819 Recruiting - Clinical trials for Lower Urinary Tract Symptoms

RT-310 Dose Escalation BPH Study

Start date: April 19, 2024
Phase: Phase 1
Study type: Interventional

RT-310, a novel implant, is intended to minimally invasive treat locally the prostate gland for the management of prostate disease, while minimizing side-effects. The objectives of the study are to assess whether RT-310 is safe and feasible for the participant population.

NCT ID: NCT06136650 Recruiting - Prostatic Neoplasms Clinical Trials

A Study of Opevesostat (MK-5684) Versus Alternative Next-generation Hormonal Agent (NHA) in Metastatic Castration-resistant Prostate Cancer (mCRPC) Post One NHA (MK-5684-004)

Start date: December 18, 2023
Phase: Phase 3
Study type: Interventional

The purpose of this study is to assess the efficacy and safety of opevesostat plus hormone replacement therapy (HRT) compared to alternative abiraterone acetate or enzalutamide in participants with Metastatic Castration-resistant Prostate Cancer (mCRPC) previously treated with one next-generation hormonal agent (NHA). The primary study hypotheses are that opevesostat is superior to alternative abiraterone acetate or enzalutamide with respect to radiographic progression free survival (rPFS) per Prostate Cancer Working Group (PCWG) Modified Response Evaluation Criteria in Solid Tumors (RECIST 1.1) as assessed by Blinded Independent Central Review (BICR) and overall survival (OS), in androgen receptor ligand binding domain (AR LBD) mutation positive and negative participants.

NCT ID: NCT06131983 Recruiting - Clinical trials for Facio-Scapulo-Humeral Dystrophy

Study of ARO-DUX4 in Adult Patients With Facioscapulohumeral Muscular Dystrophy Type 1

Start date: February 26, 2024
Phase: Phase 1
Study type: Interventional

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of ARO-DUX4 in participants with facioscapulohumeral muscular dystrophy Type 1 (FSHD1). In Part 1 of the study, participants will receive a single dose of ARO-DUX4 or placebo. In Part 2 of the study, participants will receive 2 or 4 doses of ARO-DUX4 or placebo. Participants who complete Part 1 will have the option to re-screen and re-randomize into Part 2. All participants will undergo pre- and post-dose MRI-guided muscle biopsies (a total of 2 biopsies). Participants who complete Part 1 and enroll in Part 2 will be required to undergo an additional screening biopsy. Participants completing Part 1 or Part 2 may have the option to continue to receive drug in an open-label extension study or may be eligible to participate in later-stage clinical studies.

NCT ID: NCT06128629 Recruiting - Clinical trials for Transthyretin Amyloidosis (ATTR) With Cardiomyopathy

MAGNITUDE: A Phase 3 Study of NTLA-2001 in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM)

Start date: December 13, 2023
Phase: Phase 3
Study type: Interventional

To evaluate the efficacy and safety of a single dose of NTLA-2001 compared to placebo in participants with ATTR-CM.

NCT ID: NCT06099782 Recruiting - Clinical trials for Non-Small Cell Lung Cancer

A Study of Participant Reported Preference for Subcutaneous Pembrolizumab Coformulated With Hyaluronidase (MK-3475A) Over Intravenous Pembrolizumab (MK-3475) Formulation in Multiple Tumor Types (MK-3475A-F11)

Start date: December 26, 2023
Phase: Phase 2
Study type: Interventional

The purpose of this study is to evaluate participant preference for coformulated hyaluronidase/pembrolizumab (MK-3475A) administered subcutaneously (SC) over pembrolizumab (MK-3475) administered intravenously (IV) in participants with multiple tumor types. There will be no hypothesis testing in this study.

NCT ID: NCT06097260 Recruiting - Clinical trials for Idiopathic Pulmonary Fibrosis

Randomized, Double-blind Study of Efficacy and Safety of Bexotegrast (PLN-74809) for Idiopathic Pulmonary Fibrosis

Start date: November 16, 2023
Phase: Phase 2
Study type: Interventional

A randomized, double-blind, dose-ranging, placebo-controlled study to evaluate the efficacy and safety of bexotegrast (PLN-74809) for the treatment of idiopathic pulmonary fibrosis (BEACON-IPF).

NCT ID: NCT06089837 Recruiting - Healthy Subjects Clinical Trials

Safety, Tolerability, and Pharmacokinetics of Multiple Doses of Oral EC5026 in Healthy Subjects

Start date: November 28, 2023
Phase: Phase 1
Study type: Interventional

EC5026 has been shown to be effective in preclinical pain models of pain, including inflammatory and neuropathic pain subtypes. Two Phase 1a studies - a Phase 1a single ascending dose (SAD) study and a Phase 1a fed-fasted study - have been conducted, evaluating the safety, tolerability, PK, and food effects of single oral doses of EC5026 ranging 0.5 to 24 mg. The present study will evaluate the safety, tolerability, and PK of 2 sequential ascending dose regimens of EC5026, administered once daily for 7 consecutive days, in healthy volunteers.

NCT ID: NCT06081075 Recruiting - Genetic Disease Clinical Trials

Newborn Genomics Programme

Start date: January 15, 2024
Phase:
Study type: Observational

Genomic methods can significantly contribute to all facets of precision medicine, from diagnosis to prevention, therapeutic intervention, and management of acute and chronic illnesses. DNA based methods are already having a considerable impact across healthcare in fields that include: public health, infectious disease monitoring, acute and chronic disease, pharmacogenomics, prenatal testing and diagnosis, and therapeutic development. In this proposal, investigators are focusing on the application of genomic methods in precision medicine - specifically on rapid whole-genome sequencing of parents and children (i.e. a trio) for the identification of diseases that have genetic components. Goals Primary goal: is to provide safe rapid whole genome sequencing to Neonatal Intensive Care Unit/Pediatric Intensive Care Unit patients. Secondary goals: 1) Although several groups globally are implementing rapid sequencing of rare disease, these are predominantly in the research space, with many unanswered questions regarding the best way to implement them into a national healthcare system. Each country and their healthcare systems are unique, and valuable knowledge will be gained by implementing this process within a New Zealand context. As part of this the study will measure the impact on the individuals and families. 2) to expand the research team's understanding of non-coding disease-causing variants and methylation changes that contribute to severe disease in early life. Primary Aims 1. To incorporate long-read RNA sequencing data into the diagnostic rapid Whole Genome Sequencing pipeline to provide a direct measure of the functional outcome of the variants of clinical concern. 2. To measure the clinical utility of analysing non-coding variants in the diagnosis of critically ill children who do not have pathogenic, likely pathogenic, or variants of unknown significance for mendelian disorders. 3. To identify, in a real-world setting within the New Zealand health-care system, the clinical and economic effects of deploying rapid Whole Genome Sequencing-informed rapid precision medicine for critically ill children.

NCT ID: NCT06079879 Recruiting - Clinical trials for Essential Thrombocythemia

A Study of Bomedemstat (IMG-7289/MK-3543) Compared to Best Available Therapy (BAT) in Participants With Essential Thrombocythemia and an Inadequate Response or Intolerance of Hydroxyurea (MK-3543-006)

Start date: December 31, 2023
Phase: Phase 3
Study type: Interventional

This is a study evaluating the safety and efficacy of bomedemstat (MK-3543) compared with the best available therapy (BAT) in participants with essential thrombocythemia (ET) who have an inadequate response to or are intolerant of hydroxyurea. The primary study hypothesis is that bomedemstat is superior to the best available therapy with respect to durable clinicohematologic response (DCHR).

NCT ID: NCT06077864 Recruiting - Obesity Clinical Trials

A Study to Test the Effect of Survodutide (BI 456906) on Cardiovascular Safety in People With Overweight or Obesity (SYNCHRONIZE™ - CVOT)

Start date: November 17, 2023
Phase: Phase 3
Study type: Interventional

This study is open to adults who are at least 18 years old and have a body mass index (BMI)bof 27 kg/m2 or more. People can take part if they have cardiovascular or chronic kidney disease. People who have at least 2 health problems related to their weight or risks of cardiovascular disease can participate. Participants must have previously tried to lose weight by changing their diet. The purpose of this study is to find out whether people with overweight or obesity who take a medicine called survodutide (BI 456906) are less or more likely to develop serious cardiovascular problems. It also aims to find out whether health parameters like blood pressure improve. Overweight and obesity are linked to cardiovascular disease. Survodutide is a medicine that is developed to help people with obesity or overweight to lose weight. Participants are divided into 3 groups of almost equal size. 2 groups get different doses of survodutide and 1 group gets placebo. Placebo looks like survodutide but does not contain any medicine. Every participant has a 2 in 3 chance of getting survodutide. Participants inject survodutide or placebo under the skin once a week. All participants also receive counselling on diet and physical activity. Participants are in the study for up to 2 years and 3 months. During this time, it is planned that participants visit the study site up to 21 times and attend remote visits by video calls. During these visits, the doctors check participants' cardiovascular and overall health. The results are compared between survodutide and placebo groups. The study staff also takes note of any unwanted effects.