Park Ridge

Researchers are studying the long-term safety of vedolizumab given as a subcutaneous injection to children and teenagers aged 2 to 17 years with moderate to severe active ulcerative colitis UC or Crohns disease CD. The study aims to understand medical problems that may arise from extended use of vedolizumab SC, as well as its impact on hospital visits due to bowel inflammation and on the quality of life for these young participants. This Phase 3b extension study follows participants from an earlier study VedolizumabSC-3003 who have responded well or not to treatment. Participants who responded well to vedolizumab SC in the parent study will continue treatment in this extension study, receiving the same dose and frequency. They will be randomly assigned to receive vedolizumab 108 mg either via a prefilled syringe with an autoinjector pen or with a needle safety device. Dosage is every two weeks for participants weighing at least 30 kg and every four weeks for those weighing between 10 and under 30 kg. Those who did not respond well or recently used corticosteroids will not receive vedolizumab in this study but will be observed in an observational cohort. Throughout the study, participants will visit their study clinic multiple times over up to two years. Researchers will monitor adverse events and serious adverse events up to 18 weeks after the last dose, as well as special safety events in the observational group. They will also assess time to major inflammatory bowel disease-related events and changes in quality of life using the IMPACT-III questionnaire at regular intervals. Participants will have a safety follow-up visit after treatment ends, and those in the observational group will be followed for about two years after their last dose in the parent study.

Researchers are studying the use of unlicensed cryopreserved cord blood units CBUs for transplantation in both children and adults with blood cancers and other related disorders. This observational study involves patients with hematologic malignancies and various inherited and acquired disorders affecting the blood and immune system. The main goal is to monitor how well neutrophil recovery occurs after transplantation using these unlicensed CBUs in multiple institutions. Participants receive unlicensed cryopreserved CBUs as part of their transplant treatment. The study includes patients of any age receiving these CBUs for approved indications. The protocol focuses on the access and distribution of these unlicensed units rather than a specific treatment intervention. The study gathers data from recipients who receive these CBUs, tracking outcomes after transplantation. Participants are monitored for neutrophil recovery at 60 and 100 days after transplant, defined by a neutrophil count of at least 500mm3. Researchers also collect information on infection transmission, infusion reactions, survival rates at one year, and incidence of acute and chronic graft versus host disease. Platelet recovery is also evaluated. Safety and efficacy outcomes are followed over time to better understand the effects of unlicensed CBUs in this patient population.

Researchers are evaluating AZD0120, a dual-targeted CAR-T therapy aimed at BCMA and CD19, compared with standard treatment regimens for participants with relapsed refractory multiple myeloma RRMM. This Phase III, randomized, open-label global study aims to assess how AZD0120 performs relative to established therapies including DKd, DPd, PVd, or Kd. The study focuses on participants who have received previous treatments and now require additional therapy due to disease progression. Participants will receive either AZD0120 or one of four standard regimens chosen by their investigator, including combinations of daratumumab, carfilzomib, pomalidomide, bortezomib, and dexamethasone. The treatment period and dosing depend on the assigned regimen, and the study compares these approaches over time. The trial is designed to measure progression-free survival and response rates to evaluate the benefits of AZD0120 compared to standard care. During the study, participants will undergo regular assessments to monitor disease status and treatment effects. These include laboratory tests to measure disease markers, imaging, and safety evaluations. The primary outcomes include progression-free survival over three years and minimal residual disease negativity at nine months. Secondary outcomes assess response rates and overall survival over several years. The study duration extends up to 2030 with ongoing monitoring and follow-up to collect comprehensive data on participant health and treatment impact.

Researchers are evaluating the safety and effectiveness of ozanimod RPC1063 in helping children and teenagers with moderate to severe active ulcerative colitis UC who have not responded well to standard treatments. The study focuses on whether ozanimod can help achieve and maintain clinical remission in this young population. This research is conducted as a phase 2 and phase 3 trial sponsored by Bristol-Myers Squibb. Participants will receive ozanimod orally in either a high dose or low dose as part of the study. The treatment is given on specified days, and participants are randomly assigned to one of these dosing groups. The study uses a quadruple masking design, meaning that participants, caregivers, investigators, and assessors do not know which dose is given. The main study period will last up to 52 weeks, with ongoing assessments for up to 6 years to monitor longer-term outcomes and safety. During the study, participants will be monitored regularly for clinical remission, symptomatic remission, clinical response, and endoscopic improvement at various time points including weeks 10 and 52. Researchers will also track corticosteroid-free remission and measure blood levels of the drug and its metabolites. Safety is closely observed by recording adverse events, serious adverse events, and events leading to treatment discontinuation. The study involves clinical visits, endoscopic exams, and laboratory tests throughout the treatment and follow-up periods.

Researchers are evaluating remternetug, an investigational drug, to study its effects on asymptomatic individuals at risk for dominantly inherited Alzheimers disease DIAD caused by genetic mutations. The study aims to assess whether remternetug can prevent or reduce amyloid beta accumulation, a protein linked to Alzheimers progression, and to evaluate its impact on various disease biomarkers and safety. This research involves two stages Stage 1 focuses on amyloid beta changes, while Stage 2 examines downstream biomarkers related to disease progression compared to control groups. Participants will receive remternetug or a matching placebo as a subcutaneous injection every 12 weeks in a randomized, double-blind design. Following Stage 1, an open-label phase will begin after the last dose, allowing participants to receive remternetug openly. The study uses brain imaging, blood, and spinal fluid tests to monitor biomarker changes and treatment effects over time, with assessments scheduled up to 192 weeks. Participants are involved in regular visits for cognitive and clinical evaluations, brain scans using PET imaging, and collection of fluid samples to track biomarker changes. Researchers will closely monitor safety, tolerability, and cognitive measures throughout the study. The total participation duration extends over multiple years, with ongoing assessments to understand the drugs effect on disease progression and participant well-being.

Researchers are evaluating camizestrant against standard endocrine therapy for patients with ER-positive, HER2-negative early breast cancer who have an intermediate or high risk of disease recurrence. These patients must have completed locoregional therapy and at least 2 to 5 years of standard adjuvant endocrine therapy. The study is a Phase III open-label trial focused on improving outcomes for these patients over a long-term period. Participants are randomly assigned to receive either camizestrant orally or continue with the standard endocrine therapy chosen by their investigator, which may include aromatase inhibitors exemestane, letrozole, anastrozole or tamoxifen. Treatment in each group lasts for 60 months. The study allows prior use of CDK46 inhibitors and includes a follow-up period extending up to 10 years from the last patient randomization. During the study, participants will undergo regular assessments to monitor invasive breast cancer-free survival and other outcomes such as invasive disease-free survival, distant relapse-free survival, overall survival, and safety. Researchers will also evaluate symptoms like joint pain, hot flushes, and vaginal dryness using specific scales, along with quality of life measures and pharmacokinetics. Safety monitoring continues up to 28 days after the last dose, and participants remain under observation for up to 10 years total.

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 nipocalimab to reduce the risk of fetal anemia and other serious complications in pregnancies at high risk for severe Hemolytic Disease of the Fetus and Newborn HDFN. The study compares nipocalimab to a placebo in pregnant participants to see if it can decrease risks like fetal loss, the need for intrauterine transfusions, hydrops fetalis, or neonatal death. This phase 3 trial focuses on pregnancies with maternal alloantibody presence and previous severe HDFN history. Participants receive either nipocalimab or a matching placebo through weekly intravenous infusions starting at randomization until gestational week 35. The study is randomized and triple-masked, meaning neither participants nor researchers know who receives the drug or placebo. The treatment period covers the pregnancy phase where risk is highest, with careful monitoring throughout. During the study, participants undergo various assessments including lab tests, antibody titers, fetal antigen testing, and physical exams to monitor health. Researchers track pregnancy outcomes through delivery and up to 4 weeks after birth or 41 weeks postmenstrual age for newborns. Long-term infant health, including development and complications related to HDFN, is followed for up to 104 weeks. Safety and maternal outcomes are also closely observed until 24 weeks postpartum.

Researchers are evaluating the efficacy and safety of nipocalimab compared to intravenous immunoglobulin IVIG in pregnant women at risk of fetal and neonatal alloimmune thrombocytopenia FNAIT, a condition where maternal antibodies attack fetal platelets. This Phase 3 trial aims to reduce the risk of severe bleeding or low platelet counts in affected fetuses and newborns. The study is sponsored by Janssen Research & Development, LLC and focuses on pregnancies complicated by specific maternal alloantibodies and fetal genotypes. Participants are randomly assigned to receive either nipocalimab or IVIG treatments during pregnancy. Nipocalimab is given intravenously starting between 13 to 18 weeks of gestation until delivery. Those receiving IVIG start at gestational week 12 for high-risk pregnancies or week 20 for standard-risk pregnancies, with oral prednisone added per protocol and tapered off after delivery based on tolerance and investigator judgment. Both treatments are studied for their impact on fetal and neonatal outcomes. During the study, participants undergo assessments including physical exams, vital signs, ECGs, and laboratory tests to monitor health and response. Researchers will track outcomes such as fetal or neonatal death, severe bleeding, and platelet counts up to one week after birth. Long-term follow-up includes developmental assessments at 52 and 104 weeks and monitoring for treatment-emergent adverse events in mothers and infants. The overall participation period extends through delivery and includes postnatal evaluations for both mother and child.

Researchers are evaluating investigational study drugs for people with a genetic mutation that causes Alzheimers disease AD. The study focuses on those who either carry this mutation or are at risk but unaware of their genetic status. It aims to assess whether these drugs can prevent or slow the buildup of amyloid beta Ab2 plaques in the brain and later evaluate the impact on other Alzheimers disease biomarkers and disease progression. Participants will receive subcutaneous injections of remternetug or a matching placebo every 12 weeks during Stage 1, which is a blinded, placebo-controlled period lasting up to 4 years. After this, Stage 2 is an open-label period of 4 years where all mutation carriers will receive the active drug. Those initially on placebo will follow the same dose escalation as Stage 1, while those on active drug will continue their established dose. The design includes randomization and blinding for treatment and mutation status, with a shared placebo group across study arms. During the study, participants will undergo regular clinical and cognitive assessments, magnetic resonance imaging MRI, amyloid PET imaging, and cerebrospinal fluid CSF analysis. Researchers will monitor amyloid beta accumulation, downstream biomarkers like tau proteins and neurofilament light, and subtle cognitive changes. Safety, tolerability, and adherence will also be tracked. The total participation can last up to 8 years, including both treatment stages and follow-up assessments.