Phoenix

Researchers are evaluating the safety, tolerability, and effectiveness of CYB003, a Deuterated Psilocin Analog, compared to a placebo when added to current antidepressant treatment in adults with moderate to severe Major Depressive Disorder (MDD). This Phase III trial focuses on participants aged 18 to 85 years who have had inadequate response to a stable antidepressant dose, aiming to better understand how CYB003 might improve depressive symptoms. Participants receive oral doses of CYB003 or matching placebo along with manualized psychological support provided by trained facilitators. The treatment period includes multiple dosing sessions with monitoring and assessments throughout. Placebo is used as a comparator to evaluate the combined safety and efficacy of CYB003 in this population. During the study, participants undergo evaluations using the Montgomery-Åsberg Depression Rating Scale (MADRS) at several time points, including screening, baseline, and multiple days up to the end of treatment at Day 42. Researchers monitor symptoms, side effects, and overall safety. Participants provide informed consent and are assessed regularly to track changes in depression severity and any adverse events over the course of the study.

Researchers are investigating the drug bezuclastinib in an open-label, two-part Phase 2 study for patients with Advanced Systemic Mastocytosis (AdvSM), including Aggressive Systemic Mastocytosis (ASM), Systemic Mastocytosis with an Associated Hematologic Neoplasm (SM-AHN), and Mast Cell Leukemia (MCL). The study aims to evaluate the safety, effectiveness, and how the drug behaves in the body for these serious conditions. Bezuclastinib is given orally as tablets taken continuously in 28-day cycles. The study has two parts: Part I focuses on identifying safe and tolerable doses of bezuclastinib over 18 months, while Part II evaluates its effectiveness by measuring the objective response rate and confirming the relationship between drug exposure and response during another 18-month period. Participants will undergo assessments including clinical evaluations, laboratory tests, and monitoring of their disease status to determine treatment effects and safety. Researchers will track the drug's impact on the disease and patient health throughout the study, which involves continuous treatment and follow-up over the specified time frames.

Researchers are evaluating (Z)-endoxifen as a potential treatment for premenopausal women with estrogen receptor-positive (ER+) and HER2-negative breast cancer. This phase 2 open-label study includes two parts: a pharmacokinetic (PK) phase to understand how the body processes the drug and a treatment phase to assess the drug's effects on tumor growth. The study aims to see if (Z)-endoxifen can slow or stop tumor growth by measuring changes in a biomarker called Ki-67. Participants are premenopausal women who meet specific cancer and health criteria. Participants in the PK part will take (Z)-endoxifen capsules daily at varying doses (20 mg, 40 mg, or 80 mg). Some will also receive a monthly injection of goserelin, a drug that temporarily stops estrogen production in the ovaries. The treatment cohort will receive both (Z)-endoxifen and goserelin. Tumor tissue samples will be collected by breast biopsy after about 4 weeks to assess the Ki-67 biomarker. Participants showing tumor response may continue treatment for up to 24 weeks or until they undergo surgery. Throughout the study, participants will have blood draws to measure drug levels and tumor markers, breast biopsies, imaging scans, and safety assessments. The main outcomes include measuring (Z)-endoxifen levels after 4 weeks, the rate of Ki-67 reduction, and tumor response after 24 weeks. Study participation lasts up to 6 months, including treatment, surgery, and a follow-up visit one month after surgery.

Researchers are investigating the safety and effects of 4D-150 gene therapy in adults aged 50 and older with neovascular (wet) age-related macular degeneration (AMD) who are already receiving anti-VEGF treatment and have shown a clinical response. This Phase 1/2 trial includes dose-escalation and randomized, controlled, masked expansion phases, aiming to evaluate 4D-150 administered by intravitreal injection in one eye, with additional substudies assessing dosing in the second eye and vector shedding. Participants will receive a one-time dose of 4D-150 by injection into the study eye, followed by monthly assessments for 24 months to monitor safety and effectiveness. Those who receive 4D-150 will then enter a long-term follow-up period up to 5 years to assess ongoing safety and the duration of treatment effects. Substudies include one for contralateral eye dosing and another to characterize vector shedding, with participants monitored regularly for safety through one year and continuing long-term follow-up through year 5. Throughout the study, participants will undergo tests of visual and retinal function and structure, with assessments performed monthly initially and safety monitored for up to five years. Researchers will track treatment-emergent adverse events, serious adverse events, and any significant changes in safety parameters. Participants must comply with study procedures and visits, and males receiving 4D-150 will be advised to use barrier methods during intercourse for six months to prevent fluid transmission.

Researchers are conducting an open-label, multi-center, non-randomized pivotal Phase 3 study to evaluate the effectiveness and safety of PET imaging using [18F]PI-2620 for detecting tau protein buildup in people with Alzheimer's disease and control subjects. The study compares PET imaging results during life with brain tissue analysis obtained after death through autopsy, aiming to improve diagnosis of tau-related brain changes. Participants will receive an intravenous injection of the radioligand [18F]PI-2620 at a dose of 185 MBq 20%. The PET imaging will be performed to visualize tau deposits in the brain. This study focuses on assessing the diagnostic accuracy of this imaging method by comparing it to post-mortem histopathology findings. Throughout the study, participants will undergo PET scans and assessments to determine the presence and extent of tau pathology. The primary outcome measure is the ability of visual assessment of [18F]PI-2620 PET images to correctly distinguish tau neurofibrillary pathology associated with Alzheimer's disease, confirmed at autopsy within about one year. Safety and tolerability during imaging procedures will also be monitored, with a total participation period depending on the timing of brain autopsy after death.

Researchers are assessing the safety and effects of Ritlecitinib, a study medicine, for treating hidradenitis suppurativa (HS), a condition causing long-lasting, painful red skin lumps. This phase 2 study focuses on adults with moderate to severe HS who have not responded well to or cannot tolerate antibiotics. The goal is to compare experiences and outcomes between those receiving Ritlecitinib and those receiving a placebo. Participants will be randomly assigned to take either Ritlecitinib or a placebo pill once daily at home. The treatment involves an initial loading dose of Ritlecitinib for 8 weeks, followed by an 8-week maintenance dose, totaling 16 weeks of treatment. The placebo group will receive a matching pill with no active medicine. Over approximately 24 weeks, including screening and follow-up, participants will attend around 10 clinic visits for health evaluations, including physical exams, blood and urine tests, vital signs, chest X-rays, ECGs, hearing tests, and questionnaires. They will also track their medication intake and HS symptoms daily using an electronic diary on a mobile phone. The study will measure how many patients achieve at least a 50% improvement in HS symptoms by week 16 to evaluate treatment response and safety.

This research aims to evaluate the effects of litifilimab (BIIB059), a monoclonal antibody, in adults with active subacute or chronic cutaneous lupus erythematosus (CLE), with or without systemic lupus erythematosus (SLE). Participants have active skin symptoms of CLE that have not improved with antimalarial therapy or had difficulties continuing that treatment. The study focuses on reducing skin disease activity using several scores including CLA-IGA-R and CLASI, while also assessing safety, immune response, and quality of life. Participants will be randomly assigned to receive either litifilimab or a placebo injection under the skin every four weeks during a 24-week double-blind period where neither participants nor researchers know which treatment is given. After this, all participants will receive litifilimab injections every four weeks for an additional 28 weeks. Those who complete the treatment may join a long-term extension study or enter a follow-up safety period lasting up to 24 weeks. Total participation may last up to 80 weeks. Throughout the study, researchers will monitor skin disease activity using the CLA-IGA-R erythema score and the CLASI-A activity score to see how many participants improve. They will also assess safety, tolerability, immune system effects, and participants' quality of life using questionnaires. These evaluations occur regularly during both treatment periods and follow-up to understand the impact of litifilimab on CLE symptoms and overall health.

Researchers are evaluating the safety and effectiveness of tenapanor in adults with Chronic Idiopathic Constipation (CIC) in this 26-week phase 3 study. The study is randomized, double-blind, and placebo-controlled, involving multiple centers. It aims to compare three doses of tenapanor (5 mg, 25 mg, and 50 mg taken twice daily) against a placebo, with a focus on improving spontaneous bowel movements. Participants will first undergo a 2-week screening where their eligibility is assessed through medical history, physical exams, lab tests, ECG, and self-reported constipation symptoms using an electronic diary (eDiary). Eligible patients will then be randomly assigned to receive one of the three doses of tenapanor or placebo twice daily for 26 weeks. During this treatment period, patients will continue daily and weekly symptom reporting via the eDiary and attend regular safety visits at weeks 2, 4, 8, 12, 16, 20, and 26. After completing the 26-week treatment, patients enter a 4-week treatment-free safety follow-up period to monitor any adverse events. A final visit occurs at the end of this follow-up to assess safety. The main outcome measured is the durable complete spontaneous bowel movements response over 12 weeks. Overall, the study involves careful monitoring of symptoms, safety, and treatment effects over approximately 32 weeks.

Researchers are evaluating the safety and effectiveness of the drug LY4065967 for treating diabetic peripheral neuropathic pain (DPNP). This study is part of a larger chronic pain master protocol aimed at speeding up the development of new treatments for chronic pain. Participants have diabetic peripheral neuropathy mainly affecting their lower limbs and have had this condition for at least six months. The study compares oral LY4065967 to a placebo, with participants randomly assigned to either group. The trial is a Phase 2, randomized, placebo-controlled clinical trial. Treatments are given by mouth, and participants continue their usual diabetes care with stable treatment for at least 90 days before screening. During the study, researchers monitor changes in average pain intensity using a numeric rating scale from baseline to week 8. Participants undergo assessments including blood sugar control (HbA1c), body mass index measurement, and safety monitoring for heart and vitamin B12 status. The trial is designed for adults aged 18 years and older and includes close observation to ensure participant safety throughout the study period.

Researchers are studying a medicine called enlicitide to reduce low-density lipoprotein cholesterol (LDL-C) in adults with high cholesterol (hyperlipidemia). This trial aims to find out if taking enlicitide together with rosuvastatin, a standard cholesterol-lowering drug, works better than a placebo in lowering LDL-C levels. The study is a Phase 3 trial that is randomized, double-blind, and placebo-controlled to ensure accurate and unbiased results. Participants will receive oral tablets of enlicitide or placebo along with oral capsules of rosuvastatin or placebo. The study compares the effect of enlicitide plus rosuvastatin against placebo to evaluate their impact on LDL-C. The treatment period lasts 8 weeks, during which participants take their assigned medications as directed. During the study, researchers will measure the average percent change in LDL-C from the start of the trial to week 8. Participants will be monitored for safety and any side effects throughout the study. The total participation time includes screening, treatment, and follow-up assessments to evaluate the medicines' effects and safety in adults aged 18 to 64 with hyperlipidemia.