Miami

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 evaluating the use of 18F-Fluciclovine positron emission tomography (PET) to monitor brain metastases in adults treated with stereotactic radiosurgery (SRS). This pilot imaging study focuses on whether 18F-Fluciclovine PET can serve as a biomarker to measure tumor response or progression after SRS in patients with brain metastases. The trial is a phase 1 study involving participants with diagnosed brain metastases from cancer. Participants will receive a 5-mCi dose of 18F-fluciclovine administered intravenously just before PET scanning. PET imaging will be performed up to 25 minutes after injection, capturing both static images from 10 to 20 minutes and dynamic series divided into four 5-minute frames from 5 to 25 minutes post-injection. This imaging approach allows for assessment of movement and time-dependent changes in the brain metastases. During the study, participants will undergo PET scans to measure changes in standardized uptake values (SUV-peak, SUVmean, and SUVmax) over 8 weeks. The study will include assessments such as MRI scans and safety monitoring. Participants must adhere to contraception requirements if of reproductive potential and avoid breastfeeding temporarily if applicable. The overall goal is to determine the ability of 18F-Fluciclovine PET to track treatment effects following SRS in brain metastasis patients.

Researchers are evaluating remibrutinib (LOU064) in adolescents aged 12 to under 18 years who have chronic spontaneous urticaria (CSU) that is not well controlled by H1-antihistamines. This Phase 3 trial aims to assess the effectiveness, how the drug is processed in the body, and safety of remibrutinib compared to a placebo. The study also intends to gather long-term data on how well remibrutinib works and its safety over several years after treatment ends. The trial includes three periods. First, the core period is a 24-week double-blind phase where about two-thirds of participants receive remibrutinib and one-third receive placebo, with about 10 site visits over approximately 32 weeks. Next is an optional open-label extension lasting from one to three years, where participants who completed the core period may receive remibrutinib or enter an observational treatment-free phase depending on their symptoms. Participants may cycle through treatment and observational periods up to six times. Finally, an optional long-term treatment-free follow-up can last up to three years with one site visit and up to four phone calls. During the study, participants undergo assessments including changes in urticaria activity scores (UAS7), itching severity (ISS7), and hive severity (HSS7) measured from baseline to 12 weeks. Regular visits monitor safety, symptoms, and drug effects. The study tracks these measures to understand remibrutinib's impact on CSU symptoms and overall safety profile during and after treatment, with total participation potentially lasting several years.

This study consists of 2 week screening period in which subjects who have consented will be evaluated for eligibility per protocol requirements. During this 2 week screening period subjects will be given access to ediary in which they will be required to self report symptoms of IBS-C daily. Information in ediary will also be used to determine eligibility prior to enrollment. During the 4 week RTP (Randomized treatment period), subjects will be randomized in in a ratio of 5:1 to receive tenapanor or matching placebo for 4 weeks. During the RTP, patients will continue recording daily assessments in the eDiary system as instructed and compliance with eDiary entries will be monitored. Patients will return for study visit every weeks (Visits 3-6) and will undergo safety assessments as per the protocol. At the end of this 4 week period, subjects will complete 2 week treatment free follow-up period and safety assessments per protocol will be conducted at the end of this 2 week period. The study plans to enroll up to 6 cohorts of eligible patients sequentially, starting from Cohort 1 with 12 patients randomized in to receive tenapanor 2 mg BID or matching placebo for 4 weeks. Subsequent cohorts will assess increasing tenapanor doses, following a dose escalation order. The study will proceed to the next dosing cohort if the current cohort completes the 4-week RTP and does not meet any of the dose escalation stopping criteria pre-specified in the protocol.

Researchers are evaluating 4D-710, an investigational gene therapy, in adults with cystic fibrosis (CF) lung disease who cannot use or tolerate CFTR modulator therapy. This Phase 1/2, multicenter, open-label trial also includes a sub-study assessing 4D-710 in adults with advanced CF lung disease or frequent lung flare-ups despite using CFTR modulators. The study aims to assess the safety, tolerability, and early effectiveness of this gene therapy in these populations. The trial involves a single dose of 4D-710, which is a gene therapy using a specialized virus to deliver a modified CFTR gene. Participants receive this treatment once, and those in the sub-study must be on a stable CFTR modulator regimen for at least 60 days before screening and continue it during a 24-month observation period. The study monitors participants with CF lung disease ranging from moderate to advanced stages. During the study, participants undergo regular evaluations including lung function tests, oxygen level checks, and monitoring for adverse effects. Researchers track the occurrence and severity of any side effects over a 60-month period. The study also includes assessments of lung health, medication adherence, and clinical status to understand the therapy's impact and safety over time.

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.

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, how the body processes, and effects on body weight of the investigational drug CRB-913 in participants with obesity. This Phase 1b study includes two parts: Part 1 focuses on healthy adults to measure drug levels in the blood after a single dose, while Part 2 involves obese participants to assess safety and weight effects using different doses compared to placebo. Part 2 is blinded so that participants, doctors, and the sponsor do not know who receives the drug or placebo. In Part 1, healthy adults receive a single dose of CRB-913 tablets to study how much of the drug enters the bloodstream and how long it stays. In Part 2, obese participants take one of three doses of CRB-913 or placebo once daily for 12 weeks. After treatment ends, participants are monitored for 28 days. The study includes a randomized, double-blind, placebo-controlled design for Part 2. Participants will attend study visits for drug administration, safety assessments, and blood tests to measure drug levels and effects on body weight. Researchers will monitor for side effects and adverse events from the first dose through 28 days after final dosing. The total participation time includes the 12-week treatment period plus the 28-day follow-up phase to evaluate safety and drug behavior in the body.

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 rilzabrutinib compared to placebo in adults with active Immunoglobulin G4 Related Disease (IgG4-RD). This Phase 3, randomized, double-blind study aims to measure the time until the first IgG4-RD clinical disease flare during a 52-week treatment period. Additional goals include assessing disease control, flare-free rates, use of glucocorticoid rescue, and monitoring safety through adverse events, laboratory tests, and electrocardiograms. Participants will be randomly assigned to receive either oral rilzabrutinib tablets or placebo for 52 weeks. Glucocorticoids may be used as rescue medication if needed. The study includes a screening period lasting 4 to 6 weeks before treatment begins, followed by the 52-week double-blind treatment phase, and a 2-week follow-up after treatment. An optional open-label extension lasting up to 108 weeks is also available for participants. During the study, participants will attend 16 visits for assessments, which may include clinical evaluations, imaging tests such as CT, MRI, PET, or ultrasound to monitor disease activity, and laboratory tests. Researchers will track time to disease flare and collect data on flare-free rates, safety parameters, and medication use. Participants' vaccination status and contraceptive use will be monitored according to local guidelines, and overall study participation could last up to 60 weeks or longer if joining the extension phase.