San Carlos

Researchers are evaluating the safety, tolerability, and how the body processes and responds to VX-670 in adults with Myotonic Dystrophy Type 1 DM1. This study is a Phase 12 trial that compares different doses of VX-670 to placebo in people diagnosed with DM1 who have a confirmed genetic test showing a specific repeat in their DNA. Participants will be randomly assigned to receive either single or multiple doses of VX-670 or matching placebo. The study has two parts Part A focuses on single ascending doses, while Part B includes both single and multiple ascending doses. The dosing levels in Part B will be based on results from Part A. Both VX-670 and placebo are given as intravenous solutions. During the study, participants will be monitored for adverse events up to 42 days in Part A and 168 days in Part B to assess safety and tolerability. Researchers will measure drug concentrations in blood plasma and muscle at various time points, including baseline, days 15, and 120. Muscle biopsies will be used to analyze changes in gene splicing. The total participation duration varies by study part, with detailed monitoring of drug effects and safety throughout.

Researchers are studying the effects of the drug ZT-01 on nighttime low blood sugar hypoglycemia in adults with type 1 diabetes who experience low blood sugars at night. The study aims to find out if ZT-01 can reduce the number of nighttime hypoglycemia episodes and how it affects blood sugar levels. The safety of ZT-01 will also be evaluated during this phase 2a trial sponsored by Zucara Therapeutics Inc. Participants will receive both placebo and ZT-01 at one of three dose levels 7 mg, 15 mg, or 22 mg, injected under the skin daily for 28 days each. The study uses a randomized, double-blind, crossover design, so participants will receive ZT-01 during one period and placebo during another, without knowing which they are receiving. They will wear a continuous glucose monitor CGM provided by the study during treatment periods and continue their usual blood sugar monitoring and insulin use. During the approximately 16-week study, participants will have up to 6 visits and 2 phone calls. They will provide blood and urine samples to assess eligibility and effects of treatment, have blood pressure and temperature checked at visits, and receive ECGs at 4 visits to monitor heart activity. Some participants may join a sub-study requiring overnight stays for blood level tests of ZT-01 and glucagon. Participants will complete daily diaries and upload CGM data to a study phone for ongoing monitoring. The main outcome measured is the number of nighttime hypoglycemia episodes during each 28-day treatment period.

This research aims to develop and validate a single-gene Non-Invasive Prenatal Test sgNIPT to detect serious health conditions like cystic fibrosis, spinal muscular atrophy, sickle cell disease, and thalassemias in unborn babies. It focuses on pregnant people with higher risk pregnancies due to carrier status or affected conditions, including cases without reproductive partner screening. The study will gather blood samples and medical information from pregnant participants and, when applicable, their partners and newborns. Participants will undergo the investigational sgNIPT, which is designed for pregnant people whose fetus is at increased risk for a single-gene disorder. This includes situations where there is no partner screening, positive partner screening but no prenatal diagnostic testing, or ultrasound findings suggesting a single-gene disorder regardless of carrier status. The study will collect newborn cheek swabs and health data within six months after delivery as part of the research. During the study, participants will provide blood samples after nine weeks of pregnancy, and researchers will collect medical and genetic information from participants and their partners. Newborn health information and cheek swabs will be collected post-delivery to assess the tests performance. The primary outcome is the accuracy of the sgNIPT in detecting four main autosomal recessive disorders approximately two years after study launch, followed by evaluation of other single gene disorders about six months later. Participation involves consenting to these procedures and ongoing information sharing throughout the study period.

Researchers are evaluating whether stereotactic body radiotherapy SBRT given to all sites of disease in patients with 1 to 5 metastatic cancer lesions can delay the progression of their condition. This study focuses on patients with metastatic breast cancer or non-small cell lung cancer, aiming to see if adding SBRT to standard treatments can extend the time before the disease worsens. Participants are assigned to one of two groups. One group receives the standard care treatment determined by their oncologist, which may include chemotherapy, targeted therapy, immunotherapy, or hormonal therapy. The other group receives SBRT targeted to all visible metastatic sites, with radiation doses tailored by the radiation oncologist, alongside standard systemic therapies. SBRT is delivered concurrently to all metastatic sites, and after completion, standard care continues. Radiation doses vary depending on the location of metastases, with specific dosing recommendations for lung and bone metastases. Throughout the study, participants will be monitored for how long their cancer remains stable without worsening, with follow-up lasting up to two years to assess progression-free survival and overall survival. Researchers will perform imaging studies and clinical assessments to measure disease status. Patients may continue systemic therapy during SBRT as advised by their doctors. Safety and effectiveness of the combined treatments will be carefully tracked during the study period.

Researchers are comparing the rates of surgical and minimally invasive interventions, as well as any harms, in Medicare beneficiaries treated with the MILD procedure versus those treated with interspinous process decompression IPD for lumbar spinal stenosis with neurogenic claudication. This observational study uses Medicare claims data to follow patients for 24 months after their initial procedure starting from January 1, 2017. The purpose is to evaluate outcomes between these two types of procedures without requiring prior patient enrollment or consent. The study includes two groups patients who received MILD, which is a percutaneous image-guided lumbar decompression performed under fluoroscopic guidance through a dorsal approach to the spine, and patients who received IPD, a different device-based decompression procedure. Data on reoperations and complications will be collected for both groups over a 24-month follow-up period using Medicare claims. Enrollment continues until the sponsor decides to stop. Participants involvement is passive as the study uses existing Medicare claims data. Researchers will monitor rates of harms related to the initial procedure and subsequent surgical or minimally invasive interventions over two years. No direct patient visits or interventions are conducted, and the study is exempt from institutional review board oversight. The total study duration extends to December 2026, covering cases treated since early 2017.