A Phase 3 Study to Evaluate the Efficacy and Safety of Pegozafermin in Subjects with Compensated Cirrhosis due to Metabolic Dysfunction-Associated Steatohepatitis (MASH)
The purpose of this study is to learn about the safety, effectiveness, and long-term outcomes of the research drug, pegozafermin, for patients with compensated cirrhosis due to MASH when compared to a placebo.
A Phase 3 Superiority Study Comparing the Safety and Efficacy of SNP-ACTH (1-39) Gel compared to Rituximab and FDA approved biosimilars in Adults with Primary Membranous Nephropathy (PMN) in a Two-Phase Adaptive Trial Design
This trial is recruiting individuals with Primary Membranous Nephropathy (PMN) - a kidney-specific, autoimmune disease in which your body's defense system turns against you and harms your body. PMN presents with increased protein in the urine. Currently, rituximab is recommended to treat PMN. In this study, SNP-ACTH (1-39) Gel will be evaluated as the study drug because other synthetic ACTH (Adrenocorticotropic hormone) therapies had been shown to be efficacious in PMN treatment as ACTH is suggested to directly protect kidneys (more specifically, podocytes of the kidneys) while modulating the over-active immune system of the body. The active ingredient of the study drug is an ACTH hormone that’s similar to the ACTH hormone in the body, but it’s synthesized in a laboratory and combined with gelatin (of porcine origin) for a more sustained release formulation. Cerium Pharmaceuticals, Inc. is funding this research study. The overall trial is divided into two Phases: Phase 3a (this ICF applies to the 3a only) Phase 3b (a separate ICF will be provided for the 3b) The purpose of this overall research study is: For Phase 3a: To determine the optimum dose of SNP-ACTH (1-39) Gel in subjects with PMN. For Phase 3b: To test the safety and effectiveness of SNP-ACTH (1-39) Gel relative to rituximab in subjects with PMN. SNP-ACTH (1-39) Gel of Cerium Pharmaceuticals, Inc. is a study drug product. It has not been approved by the United States Food and Drug Administration (FDA) for marketing in the USA for the treatment of PMN. Other long-acting ACTH therapies are used in the USA or other countries for the treatment of PMN. Currently, rituximab therapy is suggested as a starting treatment for reduction of the activation of the immune system against the body for high risk PMN subjects.
A Phase 3 Two-stage Randomized Multi-center Controlled Open-label Study Comparing Iberdomide Maintenance to Lenalidomide Maintenance Therapy after Autologous Stem Cell Transplantation (ASCT) in Participants with Newly Diagnosed Multiple Myeloma (NDMM)
This is a two-stage, Phase 3, randomized, multi-center, controlled, open-label study comparing iberdomide maintenance to lenalidomide maintenance therapy after ASCT in participants with newly diagnosed multiple myeloma.The primary objective of this study is to compare the efficacy of iberdomide to that of lenalidomide maintenance after ASCT in participants with NDMM, as measured by PFS.
A PHASE I FIRST-IN-HUMAN OPEN-LABELTRIALTO INVESTIGATE THE SAFETY TOLERABILITY PHARMACOKINETICS AND PRELIMINARY ANTITUMOR ACTIVITY OF SIM0500 A HUMANIZE GPRC5D-BCMA-CD3 TRISPECIFIC ANTIBODY IN PARTICIPANTS WITH RELAPSED OR REFRACTORY MULTIPLE MYELOMA
This is a clinical trial that aims to test the effectiveness and how the body processes a drug called SIM0500 in adult patients with relapsed or refractory multiple myeloma (RRMM). The trial has two parts: Part 1 focuses on finding the right doses (dose escalation), and Part 2 looks at optimizing those doses. In both parts, patients will receive SIM0500 until their disease worsens, they experience severe side effects, they decide to stop, or the trial ends. In Part 1, researchers will start with a low dose of 0.15 µg/kg and may lower it to 0.05 µg/kg if patients have serious side effects. The treatment will be given once a week, and each treatment cycle will last 28 days.
A Phase I Multicenter Open-label First-in Human Dose Escalation and Expansion Study of AZD9592 as Monotherapy and in Combination with Anti-cancer Agents in Patients with Advanced Solid Tumors
This is a first-in-human modular Phase 1, open-label, multi-center study to evaluate the safety and tolerability and identify a recommended phase 2 dose (RP2D) of AZD9592 alone and with a specific combination treatment, in EGFR and cMET expressing tumors, initially NSCLC EGFR mut (L858R/ex19del) and wild type, as well as HNSCC. The study will also evaluate the preliminary efficacy, PK, PD and immunogenicity of AZD9592.
A phase I open-label multi-center study of KFA115 as a single agent and in combination with pembrolizumab in patients with select advanced cancers
This study is a FIH, open-label, phase I, multi-center study that consists of two treatment arms in dose escalation: single-agent KFA115 (Arm A escalation) and KFA115 in combination with pembrolizumab preceded by a KFA115 run-in for 1 cycle (Arm B escalation). In expansion, the study consists of three treatment arms: single-agent KFA115 (Arm A expansion), KFA115 in combination with pembrolizumab after single-agent KFA115 run-in (Arm B expansion), and KFA115 to be initiated with pembrolizumab concurrently (Arm C expansion).
A PHASE I RANDOMIZED DOUBLE-BLIND PLACEBO-CONTROLLED SINGLE-CENTER STUDY OF SAFETY AND EFFECTIVENESS OF INNATE IMMUNITY STIMULATION VIA TLR9 IN MILD COGNITIVE IMPAIRMENT OR EARLY AD
This single-center, double-blind, placebo-controlled study will recruit in total 39 participants with either Mild Cognitive Impairment due to Alzheimer’s disease (MCI) or Mild Alzheimer’s disease dementia (mild AD). There will be 3 Dose levels. An initial cohort of 13 subjects will be randomized to a Dose level 1 (0.1 mg/kg vs. placebo) lasting 8 weeks. An additional 13 subjects will be recruited and randomized into Dose level 2 (0.25 mg/kg vs. placebo) for 8 weeks and 13 subjects for the last Dose level 3 (0.5 mg/kg vs. placebo) for 8 weeks. Primary ObjectivesEvaluate the safety and tolerability of 3 escalating dose levels of CpG 1018 (dose level 1: 0.1 mg/kg vs. placebo; dose level 2: 0.25 mg/kg vs. placebo; dose level 3: 0.5 mg/kg vs. placebo) as 3 subcutaneous (s.c.) injections in patients with MCI or mild AD.Secondary ObjectivesKey secondary objectives:• To evaluate drug effect on immunostimulatory responses• To evaluate drug effect on disease progression An independent unblinded Data Safety Monitoring Board (DSMB) will convene at regular intervals to monitor the overall safety of the study and to make recommendations to the PI related to study safety as appropriate.
A PHASE I/IB SINGLE ARM STUDY OF TWO FRACTION SBRT WITH DOMINANT LESION SIB FOR THE TREATMENT OF LOCALIZED PROSTATE CANCER
Phase I/IB, single arm trial of Two-Fraction SBRT with an MRI directed, dominant intraprostatic lesion, simultaneous integrated boost based on genomic classification in the treatment of localized prostate cancer. Primary endpoint will be physician-reported grade 2 or higher CTCAE toxicity. Secondary endpoints are: EPIC quality of life, PSA Nadir, and Phoenix Definition Biochemical failures as well as Disease Free Survival, Overall Survival, and MFS.
A Phase I/IIa Dose Escalation Study Evaluating the Safety Tolerability and Preliminary Efficacy of Intraductal Administration of RXRG001 to Parotid Gland(s) in Adults with Radiation-Induced Xerostomia and Hyposalivation
This is a Phase 1/2a study testing whether administering the new study medicine RXRG001 (a lipid nanoparticle containing circular mRNA) directly into the ducts of the parotid glands (salivary glands) is a safe and effective treatment for patients with radiation-induced dry mouth (xerostomia) and reduced saliva production (hyposalivation). This study has two parts. In Part 1 of the study, patients will be divided into two groups. Patients in Group 1 will be subdivided into three groups - each group will receive a single dose of study medicine at three different dose levels. Patients in Group 2 will also be subdivided into three groups to receive three monthly doses of the study medicine at three different dose levels. In Part 2 of the study, patients will be randomly assigned to 3 groups. Each group of patients will receive multiple doses of study medicine RXRG001 at three different dose levels. The study team will have blood samples taken from all patients to see how their bodies are handling the study medicine. All patients will be closely monitored for side effects and safety concerns.
A PHASE IB STUDY TO ASSESS SAFETY OF CONCURRENT AZELIRAGON WITH CRANIOSPINAL IRRADIATION IN PATIENTS WITH LEPTOMENGINAL METASTASIS FROM SOLID TUMOR MALIGNANCIES OR HIGH-GRADE GLIOMAS
This study is a Phase 1b clinical trial at a single hospital to test the tolerability and safety of combining the drug Azeliragon with craniospinal irradiation (CSI) in patients with leptomeningeal metastasis from solid tumors or high grade gliomas. Patients will take Azeliragon for 7 days before starting CSI, continue it during CSI, and take it for 7 more days afterward. The study will test different doses to find the highest dose of Azeliragon that is tolerated with CSI, based on any issues during the first 4 weeks of treatment. Once the best dose is found, additional patients will be treated at that level. Patients will have regular check-ups, including brain and spine MRIs and spinal fluid tests, during treatment and at 4 weeks, 3 months, 6 months, 9 months, and 12 months after finishing CSI. The study will also track survival, disease progression, and patient-reported symptoms. Patients can continue other treatments, like chemotherapy or immunotherapy, after finishing radiation, and their progress will be followed until death