A PHASE III RANDOMIZED DOUBLE-BLIND PLACEBO-CONTROLLED MULTICENTER BASKET STUDY TO EVALUATE THE EFFICACY SAFETY PHARMACOKINETICS AND PHARMACODYNAMICS OF SATRALIZUMAB IN PATIENTS WITH ANTI-N-METHYL-D-ASPARTIC ACID RECEPTOR (NMDAR) OR ANTI-LEUCINE-RICH GLIOMA-INACTIVATED 1 (LGI1) ENCEPHALITIS
The purpose of this study is to assess the efficacy, safety, pharmacokinetics, and pharmacodynamics of satralizumab in participants with anti-N-methyl-D-aspartic acid receptor (NMDAR) and anti-leucine-rich glioma-inactivated 1 (LGI1) encephalitis.Current treatment of these disorders exclusively use off-label immune therapies and is based on expert opinion, retrospective case series, and open-label studies. Several unmet needs exist, including the frequent occurrence of long-term cognitive deficits, insufficient seizure control, frequent dependence on high-dose corticosteroids, and faster-acting but durable immunotherapy. There is a need for prospectively generated evidence-based treatments to meaningfully lessen the acute and long-term consequences of these disorders.
A Phase III Randomized Double-Blind Placebo-Controlled Multicenter Global Study of Rilvegostomig in Combination With Chemotherapy as Adjuvant Treatment After Resection of Biliary Tract Cancer With Curative Intent (ARTEMIDE-Biliary01)
A global study to assess the efficacy and tolerability of rilvegostomig compared to placebo in combination with investigator's choice of chemotherapy in participants with BTC after surgical resection with curative intent.
A PHASE III RANDOMIZED DOUBLE-BLIND PLACEBO-CONTROLLED MULTICENTER STUDY TO EVALUATE THE EFFICACY AND SAFETY OF OBINUTUZUMAB IN PATIENTS WITH ISN/RPS 2003 CLASS III OR IV LUPUS NEPHRITIS
This is a parallel-group, double-blind, randomized, placebo-controlled study comparingthe efficacy and safety of obinutuzumab versus placebo among patients with ISN/RPS2003 Class III or IV LN treated with standard-of-care therapy with MMF andcorticosteroids. The study will enroll approximately 252 patients.Patients must be 18?75 years of age and have ISN/RPS Class III or IV proliferative LNas evidenced by renal biopsy performed in the 6 months prior to screening or duringscreening. Patients may have concomitant Class V disease (i.e., Class III/V orClass IV/V). Patients must exhibit significant proteinuria as evidenced by a UPCR ? 1based on a 24-hour urine collection during screening.Key exclusion criteria include evidence of severe renal impairment, defined by an eGFR? 30 mL/min per 1.73 m2 of body surface area or ESRD requiring dialysis ortransplantation; evidence of active infection; and other safety-related exclusions.The study consists of the following study periods: screening, blinded treatment,open-label treatment (OLT), and study follow-up.
A PHASE III RANDOMIZED DOUBLE-BLIND PLACEBO-CONTROLLED MULTICENTER STUDY TO EVALUATE THE EFFICACY SAFETY PHARMACOKINETICS AND PHARMACODYNAMICS OF SATRALIZUMAB AS MONOTHERAPY OR IN ADDITION TO BASELINE THERAPY IN PATIENTS WITH MYELIN OLIGODENDROCYTE GLYCOPROTEIN ANTIBODY-ASSOCIATED DISEASE (MOGAD)
This Phase III, randomized, double-blind (DB), placebo-controlled, multicenter study isdesigned to evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics ofsatralizumab compared with placebo as monotherapy or in addition (add-on) tobaseline/background ISTs for MOGAD relapse prevention.The study will include a screening period of up to 28 days, an event-driven DB treatment period, and an approximately 2-year open-label extension (OLE) period.
A Phase III Randomized Double-blind Placebo-controlled Multicenter Trial to Evaluate the Efficacy and Safety of Diamyd to Preserve Endogenous Beta Cell Function in Adolescents and Adults with Recently Diagnosed Type 1 Diabetes Carrying the Genetic HLA DR3-DQ2 Haplotype
The purpose of this study is to test the safety and effectiveness of an investigational drug called Diamyd®. Diamyd® is considered “investigational” in this research study, because it has not been approved for treatment of type 1 diabetes by regulatory authorities in any country, including the Food and Drug Administration (FDA) in the United States. It is hoped that Diamyd® will help the body preserve its ability to produce insulin by stopping or delaying the immune system’s attack on beta cells. In order to determine if Diamyd® is effective, a comparison must be made between Diamyd® and placebo. A placebo does not contain any active ingredient. Researchers use a placebo to see if a study drug works better or is safer than not taking anything at all.
A Phase III Randomized Double-Blind Placebo-Controlled Non-Inferiority Multi-Center Study of the Effects of Stopping Hydroxychloroquine in Elderly Lupus Disease
This study aims to develop evidence-based protocols for managing older patients with systemic lupus erythematosus (SLE), a topic that has been largely overlooked. Hydroxychloroquine (HCQ) is an effective medication for SLE, helping to reduce disease activity and keep symptoms stable. While HCQ is generally safer than traditional immunosuppressants regarding infections, long-term use can lead to retinal toxicity, with studies showing that nearly a third of patients may experience retinal damage. As lupus patients live longer, they may develop other health issues that affect how HCQ is processed in the body, potentially increasing the risk of toxicity compared to its benefits. Given that disease activity often decreases with age, this study will investigate whether HCQ can be safely stopped in stable patients aged 60 and older. Participants will be randomly assigned to receive either a placebo or continue with HCQ and will be monitored every two months for one year to track disease activity and flares.
A Pilot Phase II Open-Label Single-Center Study of Sodium-Glucose Cotransporter-2 Inhibitor Empagliflozin in Major Depressive Disorder
This is an open-label, single arm clinical trial preliminarily testing the antidepressant effects of empagliflozin in patients with major depressive disorder (MDD), and assessing its feasibility, safety and tolerability in this patient population. The study aims to enroll 16 subjects, and will collect clinical and laboratory data at screen, baseline, weeks 2, 4 and 6 (the endpoint). The primary outcome will be change in depression severity, compared to baseline, following drug administration as assessed by the Montgomery-Åsberg Depression Rating Scale (MADRS). In exploratory analysis, we will study the relationship between ketogenesis and response, as well as the effect of treatment on markers of brain bioenergetic metabolism and neural insulin resistance by studying differences in the molecular cargo of brain derived exosomes before and after administration of the study drug.
A pilot proof of concept study of the Effects of Administration of SCFA in Rheumatoid Arthritis Inadequate Responders (EASi-RAIR)
This study is a pilot, proof of concept study to determine the effects of administering an oral SCFA supplement to RA patients with inadequate response to methotrexate. We will include up to 65 participants to obtain a sample size of at least 25 participants taking the oral supplement. We hypothesize that oral SCFA will change the participants’ gut microbiome and regulatory immune responses. Clinical data to assess for adverse events, stool, urine samples and peripheral blood will be collected at baseline, 1 month, with an optional 2 month time-point and optional visit up to 12 months following starting treatments with the next line of therapy. Fecal microbiome will be analyzed. Adaptive immune responses will be analyzed from participant blood samples.
A pilot proof of concept study the contribution of eye-hand coordination impairment to functional deficits in stroke
We propose a quantitative assessment of eye-hand function in 30 stroke patients and 30 age- and sex-matched healthy adults. To explore structural and functional changes in frontoparietal networks are critical for eye-hand coupling and efficient performance of visually guided activities of daily living. We expect results from our proposal will provide a foundation for an improved understanding of eye-hand coordination in functional deficits and functional recovery, post-stroke. This advanced knowledge will provide the basis for future translational research to develop new clinical assessments and interventional approaches to address EHdC in stroke survivors. Achieving the aims of this proposal will also address the current gap between motor recovery and functional recovery in stroke survivors with EHdC. We seek to characterize frontoparietal microstructure associated with EHdC and to identify how they relate to the motor ability of the eye, the hand, and clinical measures of functional recovery in MCA stroke.
A Pilot Proof of Concept Study with Paired-Design on the Effectiveness of Metal Related Artifact Reduction of Hip Arthroplasty Implants or Other Metallic Hardware Using Radiofrequency Pulse Polarization Optimization in 3T MRI
This is a single-center single-arm clinical trial with a paired design which compares the effectiveness of 3T MRI with (modified) and without (standard) radiofrequency pulse polarization optimization in reducing metal-related artifacts in patients with hip arthroplasty implants or other metallic hardware. Twenty participants with symptomatic primary total hip arthroplasty or other metallic hardware will be imaged with both methods. The results of the two methods will be compared in reference to the size of the artifact-degraded regions, visibility of the normal structures and the rate of abnormality detection.