Sep 30th, 2022 - The study is designed as a multicenter, single-arm, open label study to demonstrate the efficacy and safety of LNP023 (iptacopan) at a dose of 200 mg b.i.d. in adult patients with aHUS who are treatment naive to complement inhibitor therapy (including anti-C5 antibody). The study will enroll approximately 50 participants and assess the effects of iptacopan on a range of efficacy assessments rel...

Sep 30th, 2022 - This is an expanded access program in which the drug being tested is called pevonedistat, which is used in combination with azcitidine. This study will provide expanded access of pevonedistat (in combination with azacitidine) for the first-line treatment to participants with HR-MDS and option of real world data (RWD) collection for the benefit of future participants. All participants will recei...

Sep 30th, 2022 - Study Description: As a primary objective, this study represents an innovative investigative measure of the safety and tolerability of JAK inhibition in participants with primary Sj(SqrRoot)(Delta)gren's syndrome. Secondary objectives will include investigating the effects of Tofacitinib on target tissues (e.g., salivary glands), systemic inflammation, and on vascular function in SS participant...

Sep 30th, 2022 - PRIMARY OBJECTIVE: I. To collect preliminary data on the effectiveness of a single dose of pegloticase in lowering serum uric acid levels to less than 6.0 mg/dl within 24 hours in patients with hyperuricemia in the setting of tumor lysis syndrome. SECONDARY OBJECTIVES: I. To assess the effect of pegloticase on renal function in patients with hyperuricemia in the setting of tumor lysis syndrome ...

Sep 30th, 2022 - PRIMARY OBJECTIVES: I. Determine the maximum tolerated dose (MTD) and dose-limited toxicities (DLTs) of venetoclax in combination with low-intensity chemotherapy in patients with relapsed/refractory acute lymphoblastic leukemia (ALL) (Phase I). II. Evaluate the overall response rate (complete response [CR] + CR with inadequate count recovery [CRi]) of the regimen after 2 cycles. (Phase II) SECO...

Sep 30th, 2022 - PRIMARY OBJECTIVE: I. Determine the toxicities and maximum tolerated dose (MTD) of birinapant concurrent with intensity modulated re-irradiation therapy (IMRRT). SECONDARY OBJECTIVES: I. Determine the objective response rate of patients with locoregionally recurrent head and neck squamous cell carcinoma (HNSCC) treated with re-irradiation and birinapant. II. Determine the local-regional control...

Sep 30th, 2022 - Urticaria is a common skin disorder that is classified according to its chronicity into acute and chronic forms. It may occur spontaneously or on exposure to a physical factor. In the latter case, the urticaria is classified as a physical urticaria . Physical urticaria may be induced by mechanical and applied pressure, exercise, or exposure to cold, heat, sun, water, or vibration. The urticaria...

Sep 30th, 2022 - PRIMARY OBJECTIVES: I. To evaluate overall survival (OS) of adjuvant reirradiation plus concurrent pembrolizumab followed by pembrolizumab to complete 12 months total of pembrolizumab to adjuvant reirradiation plus concurrent platinum chemotherapy in high risk head and neck squamous cell carcinoma (HNSCC) patients. II. To evaluate OS of adjuvant pembrolizumab for 12 months compared to adjuvant ...

Sep 30th, 2022 - Surgical site infections (SSI) are serious complications accounting for 20% of all the healthcare-associated infections and are considered the second most frequent type of hospital-acquired infection in Europe and the United States. The incidence of SSI in England at 30-days is 8.6% for coronary artery bypass graft (CABG) and 2.2% for non-CABG operations. SSI after cardiac surgery is associated...

Sep 30th, 2022 - Aim 1 (Primary): Examine the feasibility and acceptability of TranS-C among adults with poor sleep health and excess weight. Aim 2 (Exploratory): Explore the effect of TranS-C on the sleep health of adults with poor sleep and excess weight. Aim 3 (Exploratory): Explore the effect of TranS-C on the cardiometabolic health of adults with poor sleep and excess weight.

Sep 30th, 2022 - Paired assessments will be made on patients who are consented; and on healthy controls who have verbally assented to participate. We plan to enroll 20 patients and 50 healthy controls. Assessments will be made at the convenience of the patient and healthy controls. Each assessment will include bilateral PLR reading within a 5 minute interval. The first assessment will be completed with the NPi-...

Sep 30th, 2022 - Principal Investigators: The principal investigators (PIs) will be transplant physicians at all participating U.S. transplant centers. Study Design: This study is an access and distribution protocol for unlicensed cryopreserved cord blood units (CBUs) in pediatric and adult patients with hematologic malignancies and other indications. Primary Objective: The primary objective of this study is to...

Sep 30th, 2022 - This is a clinical translational study on a prospective, interventional clinical cohort (defined as the AHCC12 study). Patient will receive adjuvant Atezolizumab plus Bevacizumab every 3-weekly for a year following surgery. Serially collected biosamples will be deeply-phenotyped and correlated with treatment outcomes (recurrence vs non-recurrence).

Sep 30th, 2022 - PRIMARY OBJECTIVES: I. Compare progression free survival of combination of olaparib and selumetinib sulfate (selumetinib) to selumetinib alone in patients with RAS mutant ovarian cancer. (Cohort 1) II. Compare progression free survival of combination of olaparib and selumetinib to selumetinib alone in patients with RAS mutant endometrial cancer. (Cohort 2) SECONDARY OBJECTIVES: I. Determine saf...

Sep 30th, 2022 - Patients admitted on this protocol will have, or be suspected of having, one of the filarial infections affecting humans. After routine clinical evaluation they will be studied in depth immunologically, and their blood cells and/or serum will be collected to provide reagents (eg., specific antibodies, T-cell clones, etc.) that will be used in the laboratory to address the broader questions of d...

Sep 30th, 2022 - This protocol concerns the approach to the localization and management of the tumor in patients with Zollinger-Ellison syndrome. It details the diagnostic imaging tests to be used to localize the tumor and the subsequent management of the tumor.

Sep 30th, 2022 - We are studying virologic and/or immunologic parameters of HIV infection and other infectious or non-infectious immune deficiency diseases in order to better understand the pathogenesis of HIV. Because of the lack of an adequate animal model it is generally necessary to utilize human peripheral blood cells for studying aspects of either in vivo or in vitro HIV infection. We wish to be able to c...

Sep 30th, 2022 - PRIMARY OBJECTIVE: I. To determine the efficacy of the regimen, as defined by the rate of complete remission (CR) or CR with incomplete count recovery (CRi). SECONDARY OBJECTIVES: I. To determine efficacy outcomes, including rate of minimal residual disease negativity by flow cytometry and polymerase chain reaction (PCR) for BCR-ABL1 transcripts, median relapse-free survival, and median overall...

Sep 30th, 2022 - A prospective, single-site, double-arm, parallel, interventional, randomized, controlled clinical trial to assess 90-day rate of joint infections in subjects undergoing primary hip replacements after administration of Next Science No-Rinse Solution (hereby referred to as NS) vs SOC (saline irrigation). The Next Science treatment arm will exhibit greater reduction in rate of 90-day post-operativ...

Sep 30th, 2022 - Study Description: The long-term neurological sequelae of Ebola virus disease are not welldescribed. Through the PREVAIL III (PIII) Ebola Natural History Study, a Neurology Substudy was undertaken to better understand the long-term neurologic sequelae of EVD, with 5 years of follow up planned. Unfortunately, due to the COVID-19 pandemic beginning in early 2020, the final study evaluations of th...