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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 a new, cost-effective way to assess real-world function in people with multiple sclerosis (MS) using patient-recorded "selfie" videos. This study aims to confirm if videos of walking and speech taken remotely on a mobile phone provide information comparable to in-person clinical visits. The goal is to develop a patient-centered, low-burden method for monitoring changes in walking and speech over time in diverse people with MS. Participants will record five brief "selfie" videos at home during baseline, 3 months, 6 months, and 12 months visits, each taking about 15 minutes every 3 months. In addition, participants will attend in-person visits at baseline, 6 months, and 12 months, lasting about one hour each. Videos are captured using the participant's own device, such as a smartphone, tablet, or computer, and uploaded securely without needing special software. Throughout the study, researchers will collect and compare data from the videos and in-person visits to validate this digital tool. They will measure walking and speech function using the MS Functional Composite and assess the feasibility, usability, and accuracy of remote video assessments over one year. This approach aims to provide accessible, meaningful monitoring that reflects daily life activities for people with MS.

Researchers are evaluating the safety and effectiveness of combining a targeted radioactive drug called 177Lu-PSMA-617 with liver-directed therapy in men with metastatic castration-resistant prostate cancer (mCRPC) who have liver metastases and have progressed after at least one androgen pathway inhibitor. This phase 1b open-label study aims to characterize safety, tumor response, progression-free survival, overall survival, and other outcomes in this specific patient group. Participants receive up to six cycles of intravenous 177Lu-PSMA-617 every 6 weeks. Before starting the drug, they undergo a single session of liver-directed therapy, such as ablation or trans-arterial chemoembolization (TACE), targeting PSMA-negative liver lesions. Imaging with PET/CT scans and biopsies may be performed as part of the treatment process. Follow-up visits happen every 3 months for up to 5 years after the last treatment. During the study, participants complete questionnaires and undergo various assessments including imaging and biopsies to monitor tumor response and safety. Researchers track adverse events, objective response rates, hepatic disease responses, and PSA levels over 12 months and beyond. Safety and treatment effects are closely monitored throughout the study and follow-up periods.

Congenital hyperinsulinism (HI) is a condition causing persistent low blood sugar, often starting in early infancy. This condition may require surgery when medical treatments do not control symptoms. The study is focused on using 18F-fluoro-L-DOPA (FDOPA) PET imaging to better differentiate between focal and diffuse forms of HI and to accurately locate focal lesions in the pancreas before surgery. This approach aims to guide surgical decisions and improve treatment outcomes for children with this condition. The trial evaluates imaging with 18F-Fluoro Dopa PET as a diagnostic tool. Participants undergo this PET scan to help identify and locate abnormal areas in the pancreas. The imaging is performed after stopping certain medications to ensure clear results. This research is conducted in a Phase 2 setting, primarily involving infants and children up to 18 years old who have HI that is uncontrolled by medication or after surgery. Participants will have their hypoglycemia and related pancreatic lesions assessed using the FDOPA PET scan. Researchers will measure how sensitive and accurate the imaging is at detecting and locating lesions within four weeks. Participants must meet specific clinical and genetic criteria and have normal liver and kidney function. The study involves monitoring medication washout and evaluating imaging results to support better diagnosis and treatment planning.

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 investigating the effects of XYOSTED as a testosterone replacement therapy in adolescent males aged 12 to under 18 years who have primary or secondary hypogonadism. This Phase 3/4, open-label, multicenter study aims to evaluate how well XYOSTED supports puberty continuation or induction, as well as its dosage, safety, and testosterone level outcomes. Participants have a confirmed deficiency or absence of endogenous testosterone and will be assessed for pubertal development and hormone levels before starting treatment. Participants will receive XYOSTED injections with dosages tailored to their weight and targeted Tanner Stage of puberty. Dose adjustments will be made regularly based on serum total testosterone levels measured at specific intervals after dosing, with evaluations approximately every three months to reach desired testosterone levels. After completing the 52-week primary study, participants may join a 24-month long-term safety extension with clinic visits every six months for ongoing clinical, laboratory, and pharmacokinetic assessments. During the study, participants will undergo thorough clinical examinations including pubertal staging, multiple testosterone measurements, and monitoring for safety and pharmacokinetics throughout treatment and extension periods. Researchers will track changes in testosterone levels from enrollment through week 53 and monitor overall safety. The study includes detailed follow-up and dose management to support pubertal development and assess long-term effects of XYOSTED therapy in this population.

Researchers are evaluating the safety and usefulness of a new imaging method using a radiotracer called 64Cu-GRIP B, which targets granzyme B, a marker produced by immune cells during immunotherapy. The study focuses on adults with advanced cancers that have spread to nearby tissues or lymph nodes, particularly genitourinary cancers such as renal cell carcinoma, urothelial carcinoma, and metastatic castration-resistant prostate cancer. This phase I/II trial aims to explore how well this imaging technique can detect granzyme B and potentially serve as a biomarker to monitor early responses to immune-based cancer treatments. Participants are divided into four groups based on their cancer type. All receive an injection of 64Cu-GRIP B before undergoing positron emission tomography (PET) imaging to visualize granzyme B in tumors. Some groups will have additional PET scans at eight weeks and at disease progression. The study includes detailed safety monitoring immediately after the injection and throughout the trial. Imaging results will be compared with conventional methods to assess tumor detection and uptake patterns. During the study, participants will have tumor biopsies, blood tests, and PET imaging to measure radiotracer uptake and monitor adverse events. Researchers will track various pharmacokinetic measures for up to eight weeks and follow participants for up to two years to observe clinical outcomes, including treatment response and side effects. The goal is to understand the relationship between imaging findings and patient outcomes, which could inform future cancer treatment strategies.

Researchers are working to create a comprehensive reference database focused on intracranial aneurysms (IA). This project aims to gather detailed clinical history, imaging data, biological samples, and other related information to better understand risk markers for aneurysm formation and rupture, along with prognostic factors for different management strategies. The study also seeks to develop patient-specific management protocols and assess how the database and its tools can improve care, reduce costs, and support new discoveries and industrial developments. Participants include patients with newly diagnosed or known intracranial aneurysms, healthy volunteers, and family members of patients with a familial history of IA. Data collected includes demographic details, medical history, imaging scans such as MRI angiography and CT angiography, and various biological samples like blood, cerebrospinal fluid, saliva, and stool. Participants are asked to provide consent for data and sample use, including genetic analysis and potential future research applications. There are no limits on the number of participants for this database. During the study, participants will provide access to their health records, complete questionnaires, and undergo imaging and sample collection. Researchers will track clinical outcomes, imaging results, and quality of life measures over time. The primary outcome is disease model validation over 5 years. Consent includes provisions for confidentiality, withdrawal without impact on care, and possible re-contact for additional information or consent. The study ensures safety through ethical oversight and insurance coverage for any direct harm related to participation.

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 evaluating MDNA11, a long-acting "beta-only" recombinant interleukin-2 designed to activate immune cells that attack cancer while minimizing stimulation of cells that suppress immunity. This Phase 1/2 open-label study aims to assess the safety, tolerability, pharmacokinetics, pharmacodynamics, and early anti-tumor activity of MDNA11 alone or combined with pembrolizumab in patients with advanced or metastatic solid tumors. The study includes dose-escalation and dose-expansion parts for both monotherapy and combination therapy with pembrolizumab. MDNA11 is given intravenously every two weeks with doses ranging from 0.003 to 0.6 mg/kg for monotherapy, while dose ranges for combination therapy are also evaluated. Treatment continues until progression, withdrawal, or loss to follow-up, with tumor assessments by CT or MRI every 8 weeks. Participants will undergo regular imaging scans every 8 weeks to monitor tumor response and safety assessments throughout the 24-month study. Researchers will track recommended doses for expansion, treatment-related adverse events, and overall safety. The study involves up to 115 patients across multiple sites and includes long-term monitoring for up to 24 months.