Downey

Researchers are evaluating a culturally-tailored home-based physical activity program designed for Hispanic or LatinoLatina adolescent and young adult childhood cancer survivors. These survivors may face long-term effects like weight gain, fatigue, and reduced physical fitness after cancer treatment. The study aims to see if this culturally-relevant program can help increase physical activity and improve overall health compared to using a Fitbit tracker alone. The study has two stages. In Stage 1, 20 Latinx survivors participate in developing the intervention using Fitbit trackers, text messages, social media support, wearable activity devices, and interviews over 9 months. In Stage 2, 170 survivors who do not meet physical activity guidelines are randomized to either the intervention group, which includes Fitbit use, weekly goal-setting, peer support via social media and Zoom meetings, and optional activity partners, or a control group that only uses Fitbit trackers for 12 weeks. The intervention includes an intensive phase with weekly sessions followed by a 4-week maintenance phase. Participants will wear Fitbit trackers daily and engage in goal-setting, peer discussions, and physical activity reminders. Researchers will measure moderate to vigorous physical activity, sedentary time, and health-related quality of life over 12 weeks. Additional evaluations include physiological markers of heart and metabolic health and qualitative interviews to improve the program. The study lasts through the intervention phases with ongoing monitoring and support for participants.

Researchers are evaluating the clinical efficacy, safety, and tolerability of XEN1101 as an additional treatment for people with focal-onset seizures in a Phase 3 randomized, double-blind, placebo-controlled study. This trial aims to compare two doses of XEN1101 with a placebo to see how well the medication can reduce seizure frequency in patients who continue their current antiseizure medications. The study involves adults diagnosed with focal epilepsy who have tried at least two antiseizure medicines without achieving seizure freedom. About 360 participants will be randomly assigned to receive either 25 mg or 15 mg of XEN1101 or a placebo once daily with an evening meal. The study includes up to 9.5 weeks of baseline monitoring to track seizure frequency followed by 12 weeks of blinded treatment. Participants maintaining the study drug can then join an open-label extension to continue treatment or enter an 8-week follow-up after treatment ends. Throughout the study, participants will keep accurate seizure diaries and continue their stable antiseizure medications. Researchers will measure the median percentage change in seizure frequency from baseline through the 12-week treatment period, along with secondary outcomes like the proportion of participants with at least a 50% reduction in seizures and patient-reported improvement. Safety will be monitored from screening until 56 days after the last dose. Overall, participants are involved for the baseline, treatment, and follow-up phases lasting several months.

Researchers are investigating the best way to combine chemotherapy and radiation therapy for patients aged 3 to 29 years with localized non-germinomatous germ cell tumors NGGCT in the brain. This phase II trial aims to optimize treatment based on how well the tumor responds to initial chemotherapy, with the goal of reducing spinal cord relapses and adjusting therapy for better disease control. The study also compares different radiation types and examines cognitive and physical effects in children and young adults with NGGCT. Participants first receive induction chemotherapy consisting of carboplatin, etoposide, and ifosfamide over six cycles every 21 days. Based on tumor response, patients are assigned to one of two plans Plan A involves whole ventricular plus spinal canal irradiation WVSCI, delivered daily for 6 weeks, while Plan B includes high-dose chemotherapy with stem cell transplant followed by radiation therapy to the whole brain and spine. Some patients may undergo second-look surgery depending on tumor response before continuing treatment. Throughout the study, participants undergo MRI scans, collection of cerebrospinal fluid and blood samples, and questionnaires assessing cognitive, social, and behavioral functioning. Researchers monitor tumor response, progression-free survival, overall survival, and patterns of disease recurrence for up to 10 years. Safety and side effects are also evaluated to better understand long-term outcomes of these treatment approaches.

Researchers are evaluating how well combination chemotherapy works in treating patients with newly diagnosed stages 2 to 4 diffuse anaplastic Wilms tumor DAWT and patients with relapsed favorable histology Wilms tumor FHWT. This phase II trial compares the effects of two chemotherapy regimens, UH-3 and ICECycloTopo, on event-free survival and overall survival, aiming to improve outcomes based on different relapse risk groups and prior treatments. The study also explores kidney toxicity, genetic markers, surgery impacts, and radiation therapy techniques to reduce side effects and better understand tumor behavior. Participants are assigned to one of two treatment groups. In Arm I Regimen UH-3, patients receive cycles of vincristine, doxorubicin, cyclophosphamide, carboplatin, etoposide, and irinotecan intravenously over various days in a 21-day cycle, with radiation therapy at week 7 of cycle 3 if needed. In Arm II Regimen ICECycloTopo, patients receive cycles of carboplatin, etoposide, ifosfamide, cyclophosphamide, and topotecan intravenously over 10 cycles every 21 days, with surgery andor radiation therapy during certain cycles as clinically indicated. Throughout the trial, patients undergo multiple imaging tests including CT scans, PET scans, chest x-rays, MRIs, abdominal ultrasounds, and bone scans, along with blood sample collections and biopsies. After completing treatment, follow-up visits occur every 3 months for the first 2 years, then every 6 months for years 3 and 4, and once at year 5. The main outcomes measured are event-free survival and overall survival up to 5 years from study entry, with ongoing monitoring for treatment effects and safety.

Researchers are evaluating STM-416, a drug studied for safety and tolerability in patients with recurrent high-grade papillary non-muscle-invasive bladder cancer NMIBC without carcinoma in situ CIS. This first-in-human, Phase 12a, multi-center study includes patients who have completed standard of care SOC and have visible recurrent disease, focusing on those undergoing transurethral resection of bladder tumor TURBT without perioperative intravesical chemotherapy. The study aims to determine the safety of STM-416 administered during surgery and to explore its effects alongside SOC therapy given afterward. The study has two parts. Phase 1 is an open-label, dose-escalation phase where up to six increasing doses of STM-416 are given intraoperatively in 3 to 6 patients per dose to assess safety and tolerability. Phase 2a is a randomized, single-blind, dose-expansion phase comparing two doses of STM-416 administered during TURBT followed by SOC therapy. All participants receive SOC treatment after surgery. The study plans to enroll about 30 patients in Phase 1, with additional participants in Phase 2a. Participants will undergo TURBT surgery with STM-416 treatment during the procedure, followed by SOC therapy. Researchers will monitor dose-limiting toxicities and adverse events for up to 90 days in Phase 1 and track recurrence-free survival for up to 24 months in Phase 2a. Pharmacokinetics and pharmacodynamics of STM-416 will be studied at various time points after administration. Overall, participants will be followed regularly to assess safety, treatment response, and disease recurrence as part of the study lasting up to two years.

This trial studies children, adolescents, and young adults with Philadelphia chromosome positive Ph or ABL-class Philadelphia chromosome-like Ph-like B-cell acute lymphoblastic leukemia B-ALL. It evaluates the combination of blinatumomab with dasatinib or imatinib alongside standard chemotherapy. The study aims to estimate 3-year event-free survival and overall survival, describe safety and toxicity, and explore treatment responses and immune function in these patients. Participants receive a modified chemotherapy regimen including multiple cycles of blinatumomab without traditional consolidation chemotherapy combined with continuous tyrosine kinase inhibitors dasatinib or imatinib depending on fusion subtype. Treatment includes induction phases, blinatumomab blocks, interim maintenance, delayed intensification, and maintenance cycles over two years, with various drugs administered orally, intravenously, or intrathecally. Radiation therapy may be given in some cases. Throughout the study, participants undergo blood and cerebrospinal fluid sample collection, bone marrow biopsies, and heart function tests such as echocardiography or multigated acquisition scans. Researchers monitor minimal residual disease, treatment-related side effects, and long-term outcomes up to three years. The study involves regular assessments to evaluate treatment effectiveness and safety over the full duration of therapy.

Researchers are evaluating the safety and effectiveness of different doses of Corabotase also called IPN10200 for treating adults with upper limb spasticity. This study aims to understand how Corabotase affects the body and to determine which doses provide the best balance of safety and benefit. The trial involves adult participants aged 18 to 70 years who have spastic hemiparesis following stroke or traumatic brain injury and meet other specific health criteria. The study includes several groups receiving different doses of Corabotase, Dysport, or placebo as single injections into various upper limb muscles. Participants are assigned randomly to groups, with some in dose-escalation cohorts and others in fixed-dose groups. The injections are given once, and doses are carefully adjusted to assess safety and response. The trial uses a double-blind design, meaning neither participants nor researchers know who receives which treatment. Participants will be monitored over nine months with regular assessments including physical exams, vital signs, lab tests, and evaluations of muscle spasticity using the Modified Ashworth Scale. Researchers will track any side effects, antibody development, and changes in muscle tone and disability. Patient and physician impressions of treatment effects and pain levels will also be recorded. This long-term follow-up helps researchers understand how the treatments work and their safety over time.

Researchers are evaluating the combination of nivolumab and blinatumomab compared to blinatumomab alone in children and young adults aged 1 to under 31 years with first relapse of CD19 B-cell acute lymphoblastic leukemia B-ALL, including patients with Down syndrome. This phase II trial aims to compare event-free survival after reinduction and consolidation therapy, assess safety and tolerability, and explore various outcomes such as remission rates and toxicity, with follow-up up to 10 years after enrollment. Participants receive treatments based on their assigned groups and arms, involving cycles of immunotherapy including blinatumomab, nivolumab, dexamethasone, methotrexate, and other chemotherapy drugs given by various routes such as intravenous infusion, intrathecal injection, and oral administration. Treatment cycles repeat every 36 or 37 days for up to two cycles, with some groups receiving radiation therapy or maintenance chemotherapy afterward. Patients with high white blood cell counts or specific disease locations may receive pre-immunotherapy treatments. Throughout the study, participants undergo lumbar punctures, bone marrow biopsies and aspirations, and collection of blood, urine, and cerebrospinal fluid for monitoring. Researchers measure outcomes such as minimal residual disease negative remission rates and event-free survival. After completing treatment, participants are followed every three months for one year to monitor their health and any long-term treatment effects.

Researchers are evaluating a new treatment approach for children and young adults newly diagnosed with acute myeloid leukemia AML, including those with and without FLT3 gene mutations. The trial compares standard chemotherapy using daunorubicin, cytarabine, and gemtuzumab ozogamicin to therapy with CPX-351, a liposome-encapsulated form of daunorubicin and cytarabine, andor the drug gilteritinib which may block abnormal FLT3 gene function. The study aims to understand which treatment works better and to monitor heart function changes during and after therapy. Participants are assigned to different treatment groups based on their risk and FLT3 mutation status. Treatments include various chemotherapy regimens delivered intravenously and intrathecally, with some groups receiving CPX-351 and others receiving standard drugs. Patients with FLT3 mutations receive additional oral gilteritinib for extended periods, including maintenance therapy up to one year. Hematopoietic stem cell transplantation is also part of the treatment for some high-risk patients following chemotherapy courses. During the study, participants will undergo multiple assessments including blood tests, bone marrow biopsies, imaging scans, and neuropsychological testing to monitor leukemia status and treatment effects. Cardiac function is closely followed using echocardiography and biomarkers. Patient outcomes such as event-free survival, overall survival, minimal residual disease, relapse rates, and treatment safety are tracked for up to three years. The total study participation may extend over several years with ongoing evaluations.

Researchers are evaluating whether adding immunotherapy drugs brentuximab vedotin and nivolumab to the standard treatment of chemotherapy with or without radiation improves survival for patients aged 5 to 60 with early stage classical Hodgkin lymphoma. This phase III trial compares progression-free survival and overall survival between the standard therapy and the immunotherapy-enhanced approach, as well as patient-reported outcomes and long-term side effects. Participants initially receive two cycles of ABVD chemotherapy every 28 days and then undergo imaging to classify their early response. Based on risk level and response, patients are assigned to one of eight treatment arms that include either continuing standard chemotherapy, receiving immunotherapy drugs, or combinations with involved-site radiation therapy. Treatments are delivered intravenously or orally in cycles lasting 28 days. Imaging and blood samples are collected throughout the trial. Participants are monitored regularly with PET scans, CT or MRI imaging, and blood tests. Follow-up visits occur every 3 months in the first year, then every 6 months for years two and three, and annually up to 12 years from registration. Researchers assess survival outcomes, adverse events, patient-reported symptoms and quality of life, and metabolic tumor burden. Long-term effects such as cardiovascular and pulmonary health are also evaluated using questionnaires and clinical assessments.