There are about 2439 clinical studies being (or have been) conducted in New Zealand. The country of the clinical trial is determined by the location of where the clinical research is being studied. Most studies are often held in multiple locations & countries.
This is a Phase 1/2a double-blinded, placebo-controlled, dose-escalating study to evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of single and multiple ascending doses of ARO-DM1 compared to placebo in male and female subjects with Type 1 Myotonic Dystrophy (DM1). Participants who have provided written informed consent and met all protocol eligibility requirements will be randomized to receive single (Part 1) or multiple (Part 2) doses of ARO-DM1 or placebo.
This study is researching an experimental drug called REGN13335. This is the first time that REGN13335 will be given to people. This study will enroll healthy adults. The aim of the study is to see how safe and tolerable REGN13335 is in healthy volunteers. The study is looking at several other research questions, including: - What side effects may happen from taking the study drug - How much study drug is present in the blood of study participants at different times - Whether the body makes antibodies against the study drug (which could make the drug less effective or could lead to side effects)
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.
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.
To evaluate the efficacy and safety of a single dose of NTLA-2001 compared to placebo in participants with ATTR-CM.
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.
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).
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.
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.
This is a study evaluating the safety and efficacy of bomedemstat (MK-3543) compared with the best available treatment (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 treatment with respect to durable clinicohematologic response (DCHR).