Mar 13th, 2023 - This is a 2-part, multi-center, Phase 2 study to evaluate the effect of EDG-5506 on safety, pharmacokinetics and biomarkers of muscle damage in approximately 27 children with DMD treated with oral, once-daily EDG-5506 for 12 months. This study will have up to a 4-week Screening period, a 12-week randomized, double-blind, placebo controlled treatment period (Part A), a 40-week open-label extensi...

Mar 13th, 2023 - PRIMARY OBJECTIVES: I. To determine the maximum-tolerated dose (MTD)/ the recommended phase 2 dose (RP2D) of venetoclax (VEN) with or without daratumumab subcutaneous (DARA SC) and dexamethasone (DEX), in previously treated light chain (AL) amyloidosis (PTAL) patients with t(11;14). (Phase Ia). II. To evaluate the safety and tolerability of VEN/DARA SC and DEX in the treatment of PTAL patients ...

Mar 13th, 2023 - A maximum of 25 clinical sites across the U.S. will participate in the study. One hundred and sixty-five subjects are intended to be implanted with the GORE® VIAFORT Vascular Stent in this study, with a limit of 33 treated subjects per site. Subjects will be evaluated through hospital discharge and return for follow-up visits at 1, 6, 12, 24, 36, 48, and 60 months post-treatment.

Mar 13th, 2023 - The proposed study will determine the feasibility, tolerability, and acceptability of a study that tests: 1) personalized treatment delivery (i.e., module sequencing and treatment discontinuation timing) aimed at increasing the efficiency of care, and 2) the research protocol designed to evaluate the effects of this personalized care. A sample of 60 participants with heterogeneous anxiety disor...

Mar 13th, 2023 - This study is a Phase I/IIa modular, open-label, multi-center study of AZD5305 administered orally, either as monotherapy or in combination with other anti-cancer agents in patients with advanced solid malignancies.

Mar 13th, 2023 - Secondary objectives: To assess the diagnostic performance of exercise lung ultrasound for the diagnosis of HFpEF. To identify the value of the B-lines score on exercise maximizing the sensitivity and specificity for the diagnosis of HFpEF. To evaluate the relationship of the delta of B-lines scores between rest and exercise with the diagnosis of HFpEF. To assess the diagnostic performance of t...

Mar 13th, 2023 - This is a multi-reader methodological study comparing the diagnostic value of 68Ga-PSMA-HBED-CC PET/CT or 68Ga-PSMA-HBED-CC PET/MRI over MRI alone, using histologic confirmation or serial follow-up for up to 2 years as the gold standard for determination of primary prostate cancer or prostatic cancer recurrence/metastasis. It is hypothesized that this will demonstrate the superiority of 68Ga-PS...

Mar 13th, 2023 - Background: The current pandemic of Coronavirus disease 2019 (COVID-19) caused by SARS-CoV-2 virus is a public health emergency of international concern. The severity of COVID-19 ranges from asymptomatic to severe. Patients with chronic lung disease such as primary ciliary dyskinesia (PCD) are thought to be at increased risk, but no data support this. Disease course and optimal treatment strate...

Mar 13th, 2023 - Approximately 250 patients who are referred for a nuclear stress testing of the heart with regadenoson (Lexiscan®) will be recruited to participate in the study. Following regadenoson (administered as part of a stress routine test protocol) participants will receive either aminophylline (75 mg - intravenously) or a matching inactive placebo (sterile salt water) injection. Participants will be s...

Mar 13th, 2023 - The primary objective of the study is to evaluate the cumulative incidence of atopic dermatitis in healthy infants at risk for dysbiosis at 12 months of age and to identify factors associated with the presence of atopic dermatitis.

Mar 13th, 2023 - This study will test the safety of BCG and gemcitabine in people who have BCG-relapsing Non-Muscle Invasive Bladder Cancer (NMIBC). The researchers will test increasing doses of gemcitabine to find the highest dose that causes few or mild side effects when combined with BCG. The study will also look at whether this combination of drugs is effective in treating BCG-relapsing NMIBC.

Mar 13th, 2023 - As diabetes technologies have become more innovative and effective in the management of pediatric type 1 diabetes (T1D), research and usage has preferentially increased only in those of higher socioeconomic status (SES). Studies have consistently demonstrated 50% lower rates of diabetes technology use in youth of lower SES. Although diabetes technology has the potential to reduce disparities in...

Mar 13th, 2023 - PRIMARY OBJECTIVE: I. To evaluate the proportion of patients with objective response (OR) to targeted study agent(s) in patients with advanced refractory cancers/lymphomas/multiple myeloma. SECONDARY OBJECTIVES: I. To evaluate the proportion of patients alive and progression free at 6 months of treatment with targeted study agent in patients with advanced refractory cancers/lymphomas/multiple m...

Mar 10th, 2023 - Objectives. The primary aim of this pilot study is to assess the effects of short-term, high-dose corticosteroid administration on the 12-week evolution of multiple sclerosis lesions with centripetal enhancement pattern on magnetic resonance imaging (MRI), in particular with respect to the development of a hypointense rim on 7-tesla phase images. In our prior work, the phase rim has been associ...

Mar 10th, 2023 - The intent of this study is to evaluate the efficacy and safety of pamrevlumab as monotherapy in participants with IPF. Participants who are not being treated with approved IPF therapies (that is, nintedanib or pirfenidone) may be eligible for screening. Examples of reasons participants may not be treated with approved IPF therapies include but are not limited to: Intolerant or not responsive t...

Mar 10th, 2023 - Aim. The aim of the current study is to investigate the feasibility of CPP for young children who have an increased risk of exposure to adverse and traumatic experiences with negative effects on their health and development. Feasibility will be conceptualized as acceptance of the method by therapists, caregivers, and children; practical applications of the method; any need for modifications; an...

Mar 10th, 2023 - Selective Internal Radiation Therapy (SIRT) is a technique where radiation is internally delivered to a tumor. In SIRT, small radioactive beads are deposited in the liver through a large blood vessel (hepatic artery). SIRT that uses the radioactive material Yttrium-90 is called Y-90 SIRT. Stereotactic Body Radiation Therapy (SBRT) is a technique where radiation is externally delivered to a tumo...

Mar 10th, 2023 - This is a phase 1 and 2 study. The phase I is a dose finding portion of the study and phase 2 will enroll additional patients at the dose found in phase I. Patients in phase I and phase 2 will have the same treatment and followup. Participants will undergo standard of care diagnostic imaging, complete blood count (CBC) with differential and chemistry labs initially. While receiving the GCSF, pa...

Mar 10th, 2023 - There are two conditioning regimens in this protocol for children >6 months. Alemtuzumab (Campath), Fludarabine and Melphalan are used. The regimens differ by the timing and dosing of Alemtuzumab (Campath). The two timings are distal and intermediate. Distal campath is initiated 22 days prior to the allogeneic transplant. Intermediate campath is initiated 14 days prior to allogeneic transplant....

Mar 10th, 2023 - Study participation will be offered to MS patients either on day of clinic visit. Patient screening, consent discussion and MSPT test completion is estimated to take approximately an hour prior to the appointment with the treating physician. Participants begin with an instructional overview, which acquaints them with all elements of the MSPT. The tests will be performed in the same order and if...