New York

Researchers are investigating CGT9486, also known as bezuclastinib, in an open-label Phase 2 study for patients with Advanced Systemic Mastocytosis AdvSM. This includes those diagnosed with 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, pharmacokinetics, and pharmacodynamics of bezuclastinib in this patient population. Participants will receive bezuclastinib tablets orally, taken continuously in 28-day cycles. The study is divided into two parts Part I focuses on identifying effective and tolerable dosing exposures over 18 months, while Part II evaluates the drugs efficacy by measuring objective response rates and confirming the exposure-response relationship, also over 18 months. Additional assessments include effects on mutation allele burden, serum tryptase levels, histopathologic changes, spleen and liver volume, and safety monitoring. During the study, participants will undergo various clinical evaluations, including laboratory tests, imaging to monitor organ size changes, and assessments of disease response and progression. Researchers will track adverse events and pharmacokinetic profiles throughout the 18 months. The study involves continuous monitoring of participants to understand the treatments impact on survival and disease progression over this period.

Researchers are evaluating the safety and effectiveness of elenestinib BLU-263 combined with symptom directed therapy SDT compared to placebo plus SDT in adults with indolent systemic mastocytosis ISM whose symptoms are not well controlled by SDT alone. This randomized, double-blind, placebo-controlled Phase 23 study includes multiple parts to assess different doses and durations of elenestinib treatment, including an open-label extension for participants finishing earlier phases. The study also enrolls participants who have previously received an approved selective KIT inhibitor and includes pharmacokinetic groups. Participants receive oral elenestinib or placebo once daily alongside SDT, which is personalized based on individual symptom management needs. Part 1 focuses on short-term treatment lasting up to 12 weeks, while Part 2 extends treatment to approximately 48 weeks. Part 3 and other parts allow treatment for up to about 5 years. The study monitors participants through these phases to evaluate how elenestinib affects symptoms, disease markers, and safety over time. During the study, participants undergo regular assessments of symptoms using the ISM-Symptom Assessment Form ISM-SAF, laboratory tests including serum tryptase and KIT D816V allele levels, bone marrow evaluations, and quality of life measures. Researchers track adverse events and changes in disease-related factors at various points up to 5 years. This thorough monitoring helps measure treatment effects and safety over both short and long-term periods, with total participation lasting several years depending on the study part.

Researchers are evaluating the combination of CGT9486 and sunitinib compared to sunitinib alone in patients with locally advanced, unresectable, or metastatic Gastrointestinal Stromal Tumors GIST. This Phase 3, open-label international trial involves multiple parts, including dose confirmation, drug interaction assessments, and efficacy comparisons. The study also includes substudies focusing on drug-drug interaction potential and first-line treatment in patients with specific genetic mutations KIT exon 9. Approximately 482 patients will participate across these parts.

Researchers are investigating the minimum effective dose of 131I-apamistamab as part of a conditioning regimen for patients with advanced sickle cell disease SCD undergoing hematopoietic stem cell transplantation. This Phase 1, single-center study focuses on patients aged 12 to 50 years with sickle cell anemia and an available HLA-matched sibling donor. The study aims to replace the typical conditioning regimen, which includes chemotherapy, total body irradiation, and Campath antibody, with 131I-apamistamab to potentially reduce long-term side effects such as secondary cancers, infertility, cataracts, and lung toxicity. Participants will receive 131I-apamistamab as an intravenous infusion approximately ten days before receiving donor stem cell infusion. The dose of 131I-apamistamab will be personalized and prepared in advance, with target dose levels of either 100 mCi or 150 mCi. Additional treatments may include oral sirolimus to suppress immune response and intravenous Campath antibody. Other procedures such as total body irradiation and red blood cell exchange transfusions may also be part of the overall treatment process, following institutional practices. During the study, participants will undergo various assessments including blood tests, hormone level measurements, and imaging scans to monitor drug absorption and treatment effects. Researchers will evaluate outcomes such as graft failure rate 42 days after transplant, event-free survival, engraftment times for neutrophils and platelets, incidence of graft-versus-host disease, transplant-related toxicities and infections, donor cell presence, and immune recovery. Hormonal evaluations related to fertility will be measured at study start and one year post-transplant. The study includes long-term follow-up of up to seven years to assess these outcomes and safety.

This research aims to explore how a dietary supplement called the Insulin Balance Formula may help support healthy insulin levels and its effects on weight, fatigue, and sugar cravings. The study focuses on generally healthy adults who want to maintain healthy insulin levels and improve overall metabolic health using this nutritional approach. It is an observational study where participants act as their own control in a single-arm design to detect initial signals of benefit and gather safety data. Participants will take the Insulin Balance Formula supplement daily for 30 days, starting with one capsule to assess tolerance and increasing to three capsules, ideally one before each meal or all at once. The formula contains five ingredients Berberine, Beta Alanine, Zinc Citrate, Folate, and Fenugreek Extract. During the study, participants will follow a balanced diet and avoid introducing new supplements or health interventions. Throughout the 30-day period, participants will complete several assessments, including body composition measurement, the PROMIS Sleep Disturbance Scale, the Carbohydrate Craving Questionnaire, and evaluations of hunger and fatigue after meals. These will be done at baseline and study completion, with some hunger and fatigue assessments repeated randomly during the study. Participants are encouraged to provide honest feedback, and their privacy is protected. The study aims to generate meaningful data on how this supplement may support metabolic health and guide future research.

Researchers are evaluating the clinical performance of 3M Filtek Supreme Flowable Composite for dental restorations, focusing on Class IV cavities and veneers on anterior maxillary teeth. This non-randomized clinical study aims to provide data on the survival and aesthetic and functional outcomes of this flowable composite material, which has been developed to improve ease of use and adaptability compared to traditional paste composites. The study includes follow-ups at 7 days, 6 months, 12 months, and 24 months, using a modified Fdration Dentaire Internationale FDI score updated in 2023 to assess the restorations. Participants will receive 3M Filtek Supreme Flowable Composite treatment applied to either Class IV restorations or veneers, with veneers performed only on paired contralateral teeth. The composite contains a mix of resins and various fillers designed for improved handling and performance. The study is conducted at a single site and includes at least 50 Class IV and 25 veneer restorations. Evaluations will be performed by two calibrated examiners different from the treating operator to ensure objective scoring. During the study, participants will have their dental restorations photographed and assessed at each follow-up visit for up to 24 months. The primary outcome is the survival of the treated site over this period. Secondary outcomes include evaluations of esthetic and functional properties. Participants must attend all scheduled follow-up visits and maintain good oral hygiene. Safety and performance data will be collected throughout the study duration, which lasts up to 24 months post-treatment.

Researchers are evaluating the use of psilocybin, a 5-HT2A receptor agonist, for helping adults with tobacco use disorder quit smoking. This phase 2, multi-site, double-blind, randomized clinical trial involves 66 participants across four experienced research sites. The study aims to compare psilocybin with niacin, an active placebo, both combined with cognitive behavior therapy CBT, to assess smoking cessation effectiveness and related cognitive changes over 12 months. Participants will be randomly assigned to receive either oral psilocybin or oral niacin in two sessions spaced one week apart. Psilocybin doses are 30 mg in the first session and either 30 mg or 40 mg in the second, depending on responses to a mystical experience questionnaire. Niacin doses are 150 mg in the first session and either 150 mg or 200 mg in the second, following the same dosing schedule. Both groups receive CBT alongside the medication sessions to support quitting smoking. During the study, participants will undergo assessments including cognitive control tasks and questionnaires about smoking urges at screening and shortly after a target quit date. Smoking abstinence will be biochemically confirmed and measured for up to 12 months. Safety and health status will be monitored through interviews, physical exams, ECGs, and lab tests. The trial also investigates psychological factors that may influence quitting success, with overall participation lasting about one year.

Researchers are evaluating the long-term safety and tolerability of LB-102 in adults with stable schizophrenia who have had inadequate responses, side effects, or issues with their current antipsychotic medications, or who have completed prior LB-102 studies. This Phase 3, open-label, multicenter trial focuses on patients aged 18 to 65 years with stable disease and aims to provide extended monitoring of this treatment. Participants will receive LB-102 with flexible dosing ranging from 50 mg to 100 mg. This single-group study involves administering the drug openly over 52 weeks to assess how well patients tolerate it and to monitor safety during this period. Throughout the study, participants will undergo evaluations including monitoring adverse events and treatment-emergent events. Effectiveness will be assessed using the Positive and Negative Syndrome Scale PANSS. The study lasts up to 52 weeks, during which safety and tolerability are carefully observed and recorded.

Researchers are evaluating the safety and effectiveness of two drugs, 64Cu-SAR-bisPSMA and 67Cu-SAR-bisPSMA, in men with prostate cancer that has spread and no longer responds to hormone-lowering treatments. This study focuses on cancers that express a protein called PSMA and aims to find safe and effective dosing while monitoring how the drugs behave in the body. Participants will go through different study phases. In the initial dosimetry phase, a single dose of 200 MBq of 64Cu-SAR-bisPSMA is given. Later phases involve receiving multiple doses of 64Cu-SAR-bisPSMA or 67Cu-SAR-bisPSMA, with doses and number of administrations varying by phase and participant group. The dose escalation phase tests increasing doses of 67Cu-SAR-bisPSMA, followed by a cohort expansion phase with up to six doses of the recommended 67Cu-SAR-bisPSMA dose. During the study, participants will undergo PETCT scans to track drug distribution and dosimetry within 48 hours of dosing. Researchers will measure prostate-specific antigen PSA levels and radiographic responses over up to five years to evaluate treatment effects. Safety will be monitored through assessments of vital signs, ECGs, laboratory tests, and recording any adverse events. The total study duration can extend up to five years for long-term follow-up.

Researchers are evaluating the use of Gallium-68-DOTATATE PETMRI in patients with somatostatin receptor-positive SSTR-positive central nervous system CNS tumors, mainly focusing on meningioma but also including other tumor types such as esthesioneuroblastoma, hemangioblastoma, medulloblastoma, paraganglioma, pituitary adenoma, and SSTR-positive systemic cancers metastatic to the brain. The study aims to assess the diagnostic usefulness of this imaging technique, especially in distinguishing tumor recurrence from post-treatment changes, with a particular interest in cases where tumor location limits surgical removal or where patients have higher-grade disease or previous radiation treatment. This is a Phase 4 interventional study sponsored by Weill Medical College of Cornell University.