Park Ridge

This research involves both pediatric and adult patients with various blood-related cancers and other disorders affecting the blood and immune system. It focuses on using unlicensed cryopreserved cord blood units (CBUs) for transplantation, aiming to study how well these unlicensed CBUs support recovery after transplant. The study also looks at important outcomes such as infection transmission, infusion reactions, survival rates, and graft-versus-host disease. Participants will receive transplants using these unlicensed cord blood units as part of a multicenter access and distribution protocol. The study is conducted at multiple U.S. transplant centers under the care of transplant physicians. The transplantation process involves administering these CBUs to patients with hematologic malignancies and other relevant conditions. Patients will be monitored for neutrophil recovery at 60 and 100 days post-transplant to assess engraftment success. Researchers will also evaluate infection rates, serious infusion reactions, survival one year after transplant, and incidences of acute and chronic graft-versus-host disease. Platelet recovery will be tracked as well. The study involves regular assessments to follow patients’ health and transplant outcomes over time.

Researchers are evaluating the safety and effectiveness of zodasiran injections in adolescents and adults aged 12 years and older who have genetically or clinically diagnosed Homozygous Familial Hypercholesterolemia (HoFH). This phase 3, randomized, placebo-controlled study aims to understand how the treatment affects levels of LDL cholesterol, a harmful type of cholesterol, in the blood over time. Participants receive either zodasiran or a placebo through subcutaneous injections during the double-blind treatment period. After completing this phase, those who wish can enter an optional open-label extension where all placebo participants have the chance to switch to the active drug. The study involves ongoing monitoring to compare the effects of zodasiran versus placebo. Throughout the study, participants will have their LDL cholesterol levels measured at the start and after 12 months to assess changes. Researchers will also monitor safety through regular clinical assessments and laboratory tests. The total duration includes the double-blind period plus the optional extension, providing detailed information on treatment impact and participant health over time.

Researchers are evaluating AZD0120, a dual-targeted CAR-T therapy against BCMA and CD19, compared to standard treatments for participants with relapsed refractory multiple myeloma (RRMM). This Phase III, randomized, open-label global study compares AZD0120 with established regimens including DKd (daratumumab, carfilzomib, dexamethasone), DPd (daratumumab, pomalidomide, dexamethasone), PVd (pomalidomide, bortezomib, dexamethasone), and Kd (carfilzomib and dexamethasone). The study aims to assess the safety and effectiveness of AZD0120 in this patient population. Participants will receive either AZD0120 CAR-T cell therapy or one of the standard drug regimens. The treatments involve combinations of biologic and drug therapies targeting multiple myeloma. The study is designed to evaluate outcomes such as progression-free survival and minimal residual disease negativity rate over periods of up to three years. During the study, participants will be closely monitored with regular assessments to evaluate disease progression and treatment response. Researchers will track progression-free survival over three years and measure minimal residual disease negativity rates at nine months, among other safety and efficacy evaluations. Participants will have laboratory tests and clinical evaluations to ensure safety and to measure treatment effects throughout the study duration.

This research aims to evaluate the effectiveness and safety of an oral drug called ozanimod (RPC1063) in treating children and teenagers aged 2 to 17 years who have moderate to severe active ulcerative colitis (UC). These young participants have not responded well to standard treatments, and the study focuses on helping them achieve and maintain clinical remission of their condition. Participants will receive specified doses of ozanimod by mouth according to a set schedule. The study is a Phase 2/3, randomized, double-blind trial conducted at multiple centers. The goal is to assess how well ozanimod works and how safe it is in this pediatric population with UC that extends beyond the rectum. During the study, researchers will monitor participants closely through medical assessments, including endoscopy to confirm disease extent and other evaluations to track disease remission. The main outcome measured is the proportion of participants who reach clinical remission by Week 52. Safety and drug behavior in the body will also be observed throughout the trial period.

Researchers are evaluating remternetug, a monoclonal antibody, to see if it can help prevent or slow the buildup of amyloid beta protein in people who carry genetic mutations that cause dominantly inherited Alzheimer's disease (DIAD) but do not yet show symptoms. This study focuses on individuals at risk for early-onset Alzheimer's, aiming to understand how remternetug affects brain changes related to the disease, safety, and tolerability. The trial has two stages: the first tests if remternetug can prevent or reverse amyloid accumulation, and the second looks at its effects on other disease markers compared to control groups. Participants receive either remternetug or a matching placebo as subcutaneous injections every 12 weeks. The study follows a two-stage design; Stage 1 assesses changes in amyloid levels using brain imaging over about 192 weeks, while Stage 2 evaluates the impact on other Alzheimer's disease biomarkers and disease progression. The study drug works by targeting and removing amyloid plaques in the brain through immune system activation. During the trial, participants undergo brain scans, blood and spinal fluid tests, and cognitive assessments to monitor disease markers and any early cognitive changes. Safety data and participants' ability to tolerate the treatment are also closely tracked. The total participation duration includes baseline and follow-up visits, with periodic evaluations to measure the drug's effects and monitor any side effects throughout the study period.

Researchers are evaluating the effectiveness of camizestrant compared to standard endocrine therapy in patients with early breast cancer that is estrogen receptor positive (ER+) and human epidermal growth factor receptor 2 negative (HER2-). These patients have an intermediate or high risk of cancer recurrence and have already completed local treatments such as surgery and possibly chemotherapy, alongside at least 2 years and up to 5 years of standard adjuvant endocrine therapy. The study is a Phase III, open-label trial designed to assess outcomes over a long term. Participants will be randomly assigned to receive either camizestrant, an oral selective estrogen receptor degrader, or one of several standard endocrine therapies including tamoxifen, anastrozole, letrozole, or exemestane, administered according to local approved guidelines. The treatment duration for both groups is planned to last 60 months. Eligible patients may have previously used CDK4/6 inhibitors, and the study will specifically include those with intermediate or high risk of recurrence as determined by clinical and biological markers. During the study, participants will be monitored for up to 10 years from the last patient's randomization to evaluate invasive breast cancer-free survival. Additional outcomes include invasive disease-free survival, distant relapse-free survival, overall survival, safety, and clinical outcome assessments. The study involves ongoing assessments of health status, treatment effects, and safety to determine the long-term benefits and risks of camizestrant compared to standard therapies.

Researchers are evaluating combination chemotherapy treatments for patients with newly diagnosed stage II-IV diffuse anaplastic Wilms tumors (DAWT) and relapsed favorable histology Wilms tumors (FHWT). This phase II trial aims to assess how adding vincristine and irinotecan to standard chemotherapy regimens affects event-free survival and overall survival compared to historical data. The study also explores kidney toxicity, tumor genetics, and radiation therapy techniques to reduce side effects in children with lung and liver metastases. Two chemotherapy regimens are studied. Arm I (Regimen UH-3) involves cycles of vincristine, doxorubicin, cyclophosphamide, carboplatin, etoposide, and irinotecan given intravenously on specific days every 21 days. Radiation therapy is given around week 7 of cycle 3 if needed. Arm II (Regimen ICE/Cyclo/Topo) includes cycles of ifosfamide, carboplatin, etoposide, cyclophosphamide, and topotecan given intravenously every 21 days, with surgery and/or radiation during certain cycles. Both arms include multiple cycles in absence of disease progression or unacceptable side effects. Participants undergo various scans like CT, PET, MRI, chest x-rays, ultrasounds, and bone scans throughout the trial. Blood samples and biopsies may be collected periodically. After treatment, patients are followed up every 3 months for the first 2 years, every 6 months for years 3-4, and once at year 5. The main outcomes measured are event-free survival and overall survival up to 5 years, along with monitoring kidney health and treatment effects.

Researchers are evaluating the effectiveness of nipocalimab compared to a placebo in reducing the risk of fetal anemia in pregnant participants at risk for severe Hemolytic Disease of the Fetus and Newborn (HDFN). This Phase 3 study focuses on pregnancies with a history of severe HDFN and aims to improve outcomes for live neonates by addressing complications related to red blood cell volume during fetal development. Participants will receive either nipocalimab or a placebo through intravenous infusions. The study monitors the effects of these treatments on reducing fetal anemia and related severe outcomes such as fetal loss, intrauterine transfusion, hydrops fetalis, or neonatal death. Treatment is given during pregnancy, with careful tracking of maternal and fetal health. During the study, participants will undergo lab tests, physical exams, and monitoring of vital signs and heart activity. Researchers will assess the percentage of pregnancies that avoid adverse outcomes from randomization through the neonatal period, up to 4 weeks of age or 41 weeks postmenstrual age. Safety and efficacy will be closely observed throughout the study period.

Researchers are evaluating the effectiveness and safety of nipocalimab in reducing the risk of severe fetal and neonatal alloimmune thrombocytopenia (FNAIT) in pregnant women. This phase 3 study focuses on pregnancies at risk of FNAIT, a condition where the mother's immune system attacks fetal platelets, potentially leading to serious bleeding complications in the fetus or newborn. Participants will receive either nipocalimab or intravenous immunoglobulin (IVIG), both administered intravenously. Additionally, prednisone will be given orally. The study includes careful monitoring and treatment during pregnancy to assess how well these therapies work in preventing severe outcomes related to FNAIT. During the study, participants will be closely monitored through physical exams, medical history reviews, vital signs, ECGs, and lab tests. Researchers will also track the health of the fetus and newborn, focusing on outcomes such as death, severe bleeding, or low platelet counts up to one week after birth. The study requires ongoing follow-up and adherence to treatment protocols until the last visit, ensuring thorough evaluation of safety and treatment effects.

Researchers are studying potential disease-modifying treatments for individuals who carry or are at risk of carrying genetic mutations that cause dominantly inherited Alzheimer's disease (DIAD), a rare form of early-onset Alzheimer's disease. The study aims to evaluate how investigational drugs affect biomarkers related to the disease, focusing on safety, tolerability, and whether these drugs can prevent or slow the build-up of amyloid beta (Ab2) plaques, which are associated with Alzheimer's progression. This study includes participants who are asymptomatic but within a specific time window before expected symptom onset based on their family mutation history. Participants will receive either the investigational drug remternetug or a matching placebo, administered as subcutaneous injections every 12 weeks. The study has two treatment stages: Stage 1 is a blinded, placebo-controlled period lasting 4 years, where mutation carriers are randomized 1:1 to active drug or placebo, and mutation-negative participants receive placebo. Stage 2 is a 4-year open-label period where all mutation carriers receive active drug, with dosing and safety monitoring schedules designed to maintain blinding to original treatment assignments. Throughout the study, participants will undergo regular assessments including clinical evaluations, cognitive testing, MRI and amyloid imaging, and cerebrospinal fluid analysis. Researchers will measure the drugs' effects on amyloid and other Alzheimer's biomarkers, subtle cognitive changes, and clinical outcomes. Safety and treatment adherence will be monitored carefully. The total treatment and observation period spans approximately 8 years, with interim analyses and long-term follow-up to evaluate disease progression and treatment impact.