North Miami

Researchers are studying the treatment of accelerated or blast phase Philadelphia chromosome-negative myeloproliferative neoplasms MPNs, a type of blood cancer, by comparing two drug regimens. This phase II trial evaluates whether combining ASTX727, which includes the drugs cedazuridine and decitabine, with iadademstat is more effective than ASTX727 alone. ASTX727 works by helping bone marrow produce normal blood cells and attacking abnormal ones, while iadademstat may stop tumor growth by blocking enzymes needed for cell growth. Patients are randomly assigned to one of two treatment groups. One group takes ASTX727 orally once daily for five days in a 28-day cycle. The other group takes ASTX727 on the same schedule plus iadademstat orally on days 1-5, 8-12, 15-19, and 22-26 of each 28-day cycle. Treatment cycles continue unless the disease progresses or side effects are unacceptable. During the study, patients provide buccal swabs, blood samples, and bone marrow samples for analysis. Participants are monitored throughout treatment with various tests including blood and bone marrow examinations. After stopping treatment for reasons other than disease progression, patients have follow-up visits every three months if they stop due to progression, follow-up occurs every six months. Researchers measure the rate of complete acute leukemia response within four treatment cycles and track event-free survival, overall survival, and stem cell transplantation rates over up to two years.

Researchers are evaluating the safety and effects of disitamab vedotin for treating adults with advanced breast cancer that is difficult to treat and has spread in the body. The study focuses on patients whose tumors express HER2 and who have previously received treatment for their advanced breast cancer. This open-label, non-randomized study is sponsored by Pfizer and includes multiple groups based on HER2 and hormone receptor status. All participants will receive disitamab vedotin as an intravenous infusion every two weeks at the study clinic. The treatment continues until either the participant or doctor decides to stop, which may be due to cancer progression, side effects, or personal choice. After stopping treatment, participants will have follow-up visits about every six weeks, followed by phone calls every twelve weeks to monitor their health. During the study, participants will attend visits every two weeks for treatment and assessments. Researchers will evaluate tumor response, duration of response, disease control, progression-free survival, overall survival, and drug levels in the blood. Safety will be monitored for up to two years, and participants can expect regular checkups and tests throughout the study period, which may last up to three years.

Researchers are studying two surgical procedures to reduce the risk of ovarian cancer in women with BRCA1 genetic mutations. This trial compares bilateral salpingectomy, which removes only the fallopian tubes, with bilateral salpingo-oophorectomy, which removes both fallopian tubes and ovaries. The goal is to find out if removing just the fallopian tubes with delayed ovary removal is nearly as effective as removing both from the start. Participants choose between two groups one undergoes bilateral salpingectomy with the option of later ovary removal, and the other undergoes bilateral salpingo-oophorectomy. Both groups have imaging tests like pelvic ultrasounds or pelvic MRIs during screening and provide blood samples throughout the study. Follow-up visits occur at multiple time points, including 10 to 60 days, 6 months, 12 months, 24 months, and then yearly for up to 20 years. During the study, researchers track if ovarian or related cancers develop and assess symptoms related to estrogen loss, quality of life, cancer-related distress, sexual function, menopausal symptoms, medical decision making, and any adverse events. Various questionnaires and imaging tests support these evaluations. Long-term safety and cancer risk reduction are monitored for up to two decades after surgery.

Researchers are evaluating the potential of GB-0895, an investigational drug, as an additional treatment for adolescents and adults with severe asthma that is not well controlled by inhaled corticosteroids and other common asthma medications. This global, multicenter, randomized, double-blind, placebo-controlled Phase 3 trial aims to assess the efficacy and safety of GB-0895 over one year, with an optional extension phase. The study includes participants aged 12 to 80 years with documented severe uncontrolled asthma despite current therapy. Participants will be randomly assigned to receive either GB-0895 or a placebo through subcutaneous injections every six months during the 52-week treatment period. The study also includes phases for screening and run-in before treatment, followed by a follow-up period lasting up to 38 weeks after treatment ends. An optional open-label extension allows participants to continue receiving GB-0895 at specified weeks after the initial treatment phase. During the study, participants will attend visits every 1 to 2 months after the first month to monitor health and treatment effects. Assessments include lung function tests, asthma symptom questionnaires, quality of life evaluations, and tracking of asthma exacerbations. Researchers will closely monitor safety and treatment responses through these evaluations and collect data on various health measures to understand the drugs impact on severe asthma over the course of the study.

Researchers are evaluating a new approach to adjuvant treatment for patients with early-stage endometrial cancer characterized by POLE mutations or p53 wildtypeno specific molecular profile. This Phase II study compares this tailored treatment approach to the usual care, which typically involves surgery followed by additional therapy decisions based on pathology results. The study includes two sub-studies focusing on these molecular types of endometrial cancer. Participants may receive one of several interventions including observation alone, observation combined with adjuvant radiotherapy, or observation combined with vaginal brachytherapy. Radiotherapy uses specific photon energies and imaging for treatment planning, while vaginal brachytherapy is delivered with a vaginal cylinder or ovoids. Treatment starts within 10 weeks after surgery, and patients are assigned to groups based on their tumor molecular status. During the study, participants are monitored for recurrence rates in the pelvis, vagina, para-aortic area, and distant sites over 3 years, as well as recurrence-free survival, cancer-specific survival, and overall survival over 9 years. Patient-reported outcomes on fear of recurrence are also collected. Careful follow-up and data collection continue throughout the study, which concludes in 2029, ensuring thorough evaluation of this tailored treatment approach.

Researchers are investigating the use of photobiomodulation PBM, a non-invasive infrared light therapy, to reduce stress in people with epilepsy. Epilepsy affects over 50 million individuals worldwide and is often treated with medications that can have significant side effects. This study aims to assess whether PBM can improve brain function, reduce oxidative stress, and promote cellular repair, potentially improving the quality of life for people with epilepsy by lowering their perceived stress levels. Participants will receive PBM using the Vielight Pro 2 device, which delivers pulsed 810nm near-infrared light through a helmet placed on the head and a light emitter clipped to the nostril. The study involves 45 sessions, each lasting 20 minutes, conducted three times a week over 15 to 20 weeks. Some participants will receive active PBM, while others will receive sham fake sessions, with all other procedures identical between groups. The study includes intake, baseline, mid-treatment, post-treatment, and six-month follow-up assessments. Throughout the study, participants will undergo extensive metabolic and behavioral testing, including EEG recordings and various blood, urine, and hair analyses. They will also wear an 5ura ring to monitor sleep and activity continuously. Researchers will measure stress levels using validated psychological inventories and correlate these with objective brain and metabolic data. The total participation lasts about 3 to 4 months, with ongoing monitoring to assess the effects of PBM on stress and brain function in epilepsy.

Researchers are evaluating PF-07994525, an oral drug, in participants with relapsed or refractory multiple myeloma RR MM and advanced malignancies. This open-label Phase 1 study aims to assess the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary antitumor activity of PF-07994525. The trial is sponsored by Pfizer and includes participants who have undergone at least three prior lines of therapy and have measurable disease according to established criteria. The study has two parts Part 1 involves dose escalation to determine the safety, tolerability, and recommended dose of PF-07994525 as a monotherapy. Part 2 expands the dose evaluation in additional participants to further assess safety and drug effects. Participants receive oral doses of PF-07994525, and in some assessments, midazolam is also administered orally to study drug interactions. The dose escalation and expansion phases will help guide future development of this treatment. Participants are involved in regular safety monitoring including assessment of adverse events and laboratory tests from the first day of treatment through up to approximately two years after the last dose. Researchers will measure response rates, time to response, duration of response, progression-free survival, overall survival, and detailed pharmacokinetic parameters. The study also tracks dose modifications and dose-limiting toxicities to understand treatment tolerability. Participation duration may vary depending on treatment response and safety outcomes.

Researchers are evaluating the addition of a stem cell transplant with melphalan after chemotherapy with daratumumab, cyclophosphamide, bortezomib, and dexamethasone Dara-VCD compared to Dara-VCD chemotherapy alone for patients newly diagnosed with amyloid light chain AL amyloidosis. This phase III trial aims to assess outcomes such as major organ deterioration progression-free survival, overall survival, organ response rates, and quality of life. The study also investigates minimal residual disease negativity and treatment-related side effects. Participants initially receive induction therapy with Dara-VCD drugs over a series of 28-day cycles, including daratumumab and hyaluronidase-fihj subcutaneously, bortezomib subcutaneously, cyclophosphamide orally or intravenously, and dexamethasone orally or intravenously. After induction, those with a partial response or better are randomized to one of two consolidation arms either continued Dara-VCD chemotherapy or high-dose melphalan chemotherapy followed by autologous stem cell transplant. Following consolidation, patients receive maintenance daratumumab and hyaluronidase-fihj therapy every 28 days for up to 18 cycles, unless the disease progresses or unacceptable toxicity occurs. Throughout the study, participants undergo multiple assessments including CT, MRI, or PET-CT scans, fat pad biopsies, echocardiography, bone marrow aspiration and biopsies, and blood and urine sample collections at regular intervals. Patient-reported outcomes related to physical function, fatigue, and symptoms are collected using standardized questionnaires. After completing study treatment, patients are followed up every 3 to 6 months for up to 4 years to monitor progression and overall health.

The trial focuses on patients with metastatic or unresectable gastroenteropancreatic neuroendocrine tumors GEPNET that have spread and cannot be removed by surgery. It compares retreatment using 177Lu-DOTATATE peptide receptor radionuclide therapy PRRT with the usual treatments of everolimus, sunitinib, or cabozantinib. The study aims to evaluate whether retreatment with 177Lu-DOTATATE can better delay tumor progression compared to these established therapies. This is a phase II trial sponsored by the National Cancer Institute NCI. Participants are randomly assigned to one of two treatment groups. One group receives 177Lu-DOTATATE intravenously every 8 weeks for two cycles if no disease progression or unacceptable side effects occur. The other group receives daily oral treatment with everolimus, sunitinib for pancreatic NET patients only, or cabozantinib for U.S. patients only, continuing until disease progression or unacceptable toxicity. Patients in the oral treatment group whose cancer worsens may switch to the 177Lu-DOTATATE group. Both groups undergo CT or MRI scans and blood sample collections during the study. Throughout the study, participants are monitored with imaging scans and blood tests to assess tumor response and safety. Researchers measure progression-free survival as the primary outcome, looking at the time from randomization until tumor progression or death, tracked for up to three years. Secondary measures include overall survival, response rates, quality of life assessments, and post-progression outcomes. After treatment ends, participants have follow-up visits every 12 weeks until disease progression, then every 6 months until death, to continue monitoring their health and study outcomes.

Researchers are comparing two monoclonal antibody treatments, rituximab and mosunetuzumab, for adults with previously untreated follicular lymphoma that has a low tumor burden. The study aims to understand which treatment better controls disease progression and improves outcomes over time. Both drugs target cancer cells but work differently, and this phase III trial evaluates their effectiveness and safety. Participants are randomly assigned to one of two groups. One group receives rituximab intravenously and subcutaneously in cycles repeating every 56 days for up to five cycles unless the disease progresses or side effects become unacceptable. The other group receives mosunetuzumab subcutaneously in cycles repeating every 21 days for up to eight cycles under similar conditions. During treatment, patients undergo CT or PETCT scans and blood tests to monitor their disease and health. After completing the treatment phase, participants are followed with visits every six months for five years, then annually up to ten years. Researchers collect imaging and blood samples during and after treatment to assess progression-free survival and other health outcomes, including overall survival and treatment side effects. This long-term follow-up helps understand the treatments lasting effects and safety.