Auckland

Researchers are evaluating the effects of an investigational medicine called PGN-EDODM1 to understand how safe and tolerable multiple doses are for adults with myotonic dystrophy type 1 DM1. This phase 2 study compares PGN-EDODM1 to a placebo to assess its safety and tolerability in people with this condition. Participants will be randomly assigned to receive either PGN-EDODM1 or a placebo. The treatment is given by intravenous infusion once every 4 weeks for a total of 12 weeks. Those in the PGN-EDODM1 group will receive ascending doses, while the placebo group will receive saline infusions on the same schedule. During the study, participants will be monitored for safety by tracking adverse events from the start through Day 112. Researchers will measure drug levels in the blood, changes in muscle tissue, myotonia severity, hand grip strength, and mobility using specific tests. The total participation lasts through about 16 weeks, including treatment and follow-up assessments.

Researchers are evaluating ARO-ALK7, a drug given by injection, in adult participants with obesity, both with and without Type 2 Diabetes Mellitus T2DM. This Phase 12a study aims to understand the safety, tolerability, how the body processes the drug, and how the drug affects the body. The study is divided into two parts Part 1 focuses on adults with obesity without T2DM, and Part 2 includes adults with obesity both with and without T2DM, testing ARO-ALK7 alone or combined with tirzepatide. Participants receive either single or multiple doses of ARO-ALK7 or matching placebo injections. In Part 2, some participants also receive weekly tirzepatide starting from Day 15 through Day 253. The doses of ARO-ALK7 are escalated to explore different levels of the drug. The study compares ARO-ALK7 alone, placebo, ARO-ALK7 combined with tirzepatide, and placebo combined with tirzepatide to assess their effects. During the study, participants are monitored for treatment-emergent adverse events until Day 253. Researchers collect detailed information on how the drug moves and acts in the body, especially during Part 1. Participants will have scheduled visits to receive treatments and undergo safety and effectiveness assessments. The total study duration covers up to about 8 and a half months, including dosing and follow-up, to fully evaluate the drugs impact and safety profile.

Researchers are studying ARO-INHBE, a drug administered by subcutaneous injection, in adults with obesity, with and without type 2 diabetes. This Phase 12a trial evaluates the safety, tolerability, pharmacokinetics, and pharmacodynamics of single and multiple doses of ARO-INHBE. The study includes participants with obesity, defined by a body mass index between 30 and 50 kgm, and those with type 2 diabetes mellitus meeting specific criteria. The study phases explore different dosing regimens and combinations with tirzepatide. The trial has three parts Part 1 assesses single and multiple ascending doses of ARO-INHBE or placebo. Part 2 and Part 3 evaluate multiple doses of ARO-INHBE alone or combined with weekly tirzepatide injections at set dosages, or matching placebos, over periods ranging up to 365 days. Dosing schedules vary by study part and include injections on specific days such as Day 1, 29, 85, 169, 253, and weekly tirzepatide from Day 15 onwards. Participants will undergo regular assessments including monitoring for treatment-emergent adverse events up to Day 365. Pharmacokinetic measurements such as plasma concentration and clearance of ARO-INHBE are taken at various timepoints. Participants are expected to adhere to stable diet and exercise routines and comply with study visits and procedures throughout the study duration. Safety and drug activity are carefully evaluated during and after treatment periods.

Researchers are studying people with idiopathic pulmonary fibrosis IPF or progressive pulmonary fibrosis PPF who have previously taken nerandomilast in another study. The aim is to assess how well patients tolerate long-term treatment with nerandomilast and to evaluate whether it improves lung function and delays worsening symptoms, hospital visits, or death. This open-label extension trial is sponsored by Boehringer Ingelheim and focuses on treatment over an extended period. Participants take nerandomilast tablets for up to 1 year and 10 months while continuing their usual pulmonary fibrosis treatments. The study involves a single treatment group receiving the drug, and no placebo or comparison groups. Regular visits with doctors help monitor health and collect data during this extended treatment phase. Throughout the study, participants undergo regular lung function tests and health assessments to track any adverse events and changes in lung capacity. The main outcome measured is the occurrence of any adverse events for up to about 99 weeks. Secondary outcomes include changes in forced vital capacity and time to worsening of disease symptoms or hospitalization. The study includes ongoing safety monitoring with a total participation time of up to nearly two years.

Researchers are evaluating the long-term safety and tolerability of dazodalibep in people with Sjgrens Syndrome. This phase 3 open-label study extends previous trials by continuing to monitor participants who completed 48 weeks of treatment with dazodalibep or placebo. The study is sponsored by Amgen and aims to better understand the safety profile of dazodalibep over an extended period. Participants who finished the initial 48-week trials HZNP-DAZ-301 or HZNP-DAZ-303 will receive an assigned dose of dazodalibep intravenously for an additional 132 weeks. This extension study involves a single treatment group receiving dazodalibep without placebo, focusing on ongoing treatment effects and participant safety. During the study, participants will be monitored for treatment-emergent adverse events for up to 152 weeks. Researchers will also measure the presence of anti-drug antibodies and plasma concentrations of dazodalibep for up to 132 weeks. Participants need to be available for all study visits and procedures, with safety assessments conducted regularly throughout the long-term extension period.

Researchers are studying ZL-1503 in a phase 11b clinical trial to assess its safety, tolerability, and how the body processes the drug. The study includes both healthy adults and adults with moderate to severe atopic dermatitis AD, a skin condition characterized by inflammation and itching. This randomized, double-blind, placebo-controlled trial aims to better understand the effects of ZL-1503 in these groups. The trial has two parts Part A involves single increasing doses of ZL-1503 given to healthy volunteers, while Part B involves multiple increasing doses given to adults with moderate to severe AD. Participants will receive either ZL-1503 or a placebo. The study monitors participants closely during dosing to evaluate how the drug is tolerated and how it behaves in the body. Participants will undergo various assessments throughout the study, including monitoring for adverse events, vital signs, laboratory tests, and electrocardiograms ECGs for up to 48 weeks after the last dose. These evaluations help researchers track safety and any potential side effects. The total participation period can last up to 48 weeks following dosing to ensure thorough observation.

Researchers are evaluating AZD0780, an oral PCSK9 inhibitor, in a phase 3, randomized, placebo-controlled study. This trial focuses on patients with established atherosclerotic cardiovascular disease ASCVD or those at high risk for a first ASCVD event. The study aims to assess how AZD0780 compares to placebo in reducing the risk of major adverse cardiovascular events, also known as MACE-PLUS, over the course of the trial. Participants are randomly assigned to receive either oral AZD0780 once daily or a matching placebo once daily. The study continues until a primary analysis censoring date, which may be up to approximately 54 months from randomization. After this, a study closure visit will be conducted as the final visit for each participant. During the study, participants will be regularly monitored for cardiovascular events including heart attacks, strokes, urgent coronary revascularizations, and other related outcomes. Researchers will track the time to first occurrence of these events as the primary outcome. Safety and other secondary outcomes like all-cause mortality will also be assessed. The total participation time can last up to about 54 months, with ongoing evaluations throughout this period.

This trial focuses on elderly patients aged 80 years or older, or those 75 years and older who are considered frail, with untreated diffuse large B-cell lymphoma DLBCL and related lymphoma subtypes. The study is a phase III, randomized, open-label, multicenter trial conducted in several countries including Sweden, Norway, Finland, Denmark, Italy, Australia, and New Zealand. It aims to compare the standard chemotherapy regimen R-miniCHOP with an experimental treatment R-pola-miniCHP, where vincristine is replaced by polatuzumab vedotin, to assess differences in outcomes for this patient population. Participants will be randomly assigned to one of two treatment groups. One group will receive R-mini-CHOP consisting of rituximab, cyclophosphamide, doxorubicin, vincristine, and prednisone over six 21-day cycles. The other group will receive R-pola-mini-CHP, which includes rituximab, cyclophosphamide, doxorubicin, prednisone, and polatuzumab vedotin instead of vincristine, also given over six 21-day cycles. Both treatments last approximately 18 weeks. The study includes a screening period lasting up to 4 weeks before treatment begins. During the study, participants will be followed for up to 36 months after completing treatment to monitor progression-free survival over two years. Researchers will evaluate disease progression and safety outcomes through regular assessments during and after the treatment period. Participants will provide informed consent and undergo evaluations including health status and disease measurements to ensure eligibility and monitor treatment effects throughout the trial.

Researchers are studying BBT001, a drug candidate for treating moderate to severe atopic dermatitis AD, in a Phase 1 randomized, double-blinded, placebo-controlled trial involving healthy volunteers and adult patients with AD. The study aims to evaluate the safety, tolerability, pharmacokinetics, immunogenicity, pharmacodynamics, and exploratory clinical activity of BBT001. Participants will receive either a single or multiple ascending doses of BBT001 or placebo. Healthy volunteers will receive single and multiple doses, while adult patients with moderate to severe AD will receive multiple doses. The study includes several parts A, B, C, D, and E with different dosing regimens to assess these effects. Throughout the study, participants will be monitored for adverse events, changes in blood parameters, vital signs, physical exams, and ECG readings for up to 169 days after the first dose. Pharmacokinetic and immunogenicity measures will also be collected at specified time points. The total duration of monitoring varies by study part but can last up to approximately 24 weeks post-dose.

Researchers are evaluating how well combination chemotherapy works in treating patients with newly diagnosed stages 2 to 4 diffuse anaplastic Wilms tumor DAWT and patients with relapsed favorable histology Wilms tumor FHWT. This phase II trial compares the effects of two chemotherapy regimens, UH-3 and ICECycloTopo, on event-free survival and overall survival, aiming to improve outcomes based on different relapse risk groups and prior treatments. The study also explores kidney toxicity, genetic markers, surgery impacts, and radiation therapy techniques to reduce side effects and better understand tumor behavior. Participants are assigned to one of two treatment groups. In Arm I Regimen UH-3, patients receive cycles of vincristine, doxorubicin, cyclophosphamide, carboplatin, etoposide, and irinotecan intravenously over various days in a 21-day cycle, with radiation therapy at week 7 of cycle 3 if needed. In Arm II Regimen ICECycloTopo, patients receive cycles of carboplatin, etoposide, ifosfamide, cyclophosphamide, and topotecan intravenously over 10 cycles every 21 days, with surgery andor radiation therapy during certain cycles as clinically indicated. Throughout the trial, patients undergo multiple imaging tests including CT scans, PET scans, chest x-rays, MRIs, abdominal ultrasounds, and bone scans, along with blood sample collections and biopsies. After completing treatment, follow-up visits occur every 3 months for the first 2 years, then every 6 months for years 3 and 4, and once at year 5. The main outcomes measured are event-free survival and overall survival up to 5 years from study entry, with ongoing monitoring for treatment effects and safety.