The Myelin Disorders Biorepository Project (MDBP) seeks to collect and analyze clinical data and biological samples from leukodystrophy patients worldwide to support ongoing and future research projects. The MDBP is one of the world's largest leukodystrophy biorepositories, having enrolled nearly 2,000 affected individuals since it was launched over a decade ago. Researchers working in the biorepository hope to use these materials to uncover new genetic etiologies for various leukodystrophies, develop biomarkers for use in future clinical trials, and better understand the natural history of these disorders. The knowledge gained from these efforts may help improve the diagnostic tools and treatment options available to patients in the future.

Type: Observational [Patient Registry]

Start Date: Dec 2016

open study

The NICHD Neonatal Research Network's Follow-Up study is a multi-center cohort in which surviving extremely low birth-weight infants born in participating network centers receive neurodevelopmental, neurosensory and functional assessments at 22-26 months corrected age (Infants born prior to July 1, 2012 were seen at 18-22 months corrected age). Data regarding pregnancy and neonatal outcome are collected prospectively. The goal is to identify potential maternal and neonatal risk factors that may affect infant neurodevelopment.

Type: Observational

Start Date: Jan 1993

open study

This study is being done to collect data from treatment of patients who have diabetes with non-healing foot wounds and are being treated with a resorbable and biocompatible borate-based bioactive glass fiber matrix. A borate-based bioactive glass fiber matrix is used to cover the ulcer for wound management. The primary objective of this study is to evaluate the safety and efficacy of the borate-based bioactive glass fiber matrix in the treatment of diabetic foot ulcers in a real-world setting. The secondary objective is to evaluate the clinical and financial benefits in terms of quality of healing, pain, and treatment cost.

Type: Observational

Start Date: Sep 2024

open study

This Phase 2 open-label trial investigates the pharmacokinetics (PK), pharmacodynamics, and safety profile of TLC590 across various surgical procedures. Researchers aim to determine the maximum tolerated dose (MTD) of TLC590 via Safety Monitoring Committee (SMC). The study evaluates TLC590 in bunionectomy, laparoscopy-assisted open ventral hernia repair, breast augmentation, abdominoplasty, and total knee arthroplasty models. Additionally, it determines the relative bioavailability of TLC590 to ropivacaine injection.

Type: Interventional

Start Date: Sep 2024

open study

The main goal of this clinical trial is to check if the treatment is safe and well-tolerated. Researchers will compare the MR-301 active drug group with the placebo group to evaluate the safety and tolerability of the drug. Other measurements include assessing the patient's overall outcome, neurological responses, time spent in the intensive care unit, time in the hospital, and mortality. Participants will receive either MR-301 BID IV dosing or a matching placebo for a total of 3 weeks.

Type: Interventional

Start Date: Jun 2024

open study

There is a fundamental gap in understanding the maternal and neonatal effects of antenatal corticosteroid (ACS) administration in women with threatened preterm birth (PTB) who have diabetes. Since the initial discovery of ACS for neonatal benefit in 1972, more than 40 randomized controlled trials have been performed evaluating its efficacy. However, none of these trials have included women with T2DM, and there is limited data among women with gestational diabetes. While ACS have been shown to reduce neonatal morbidity associated with PTB in non-diabetic women, the side effects of ACS (maternal hyperglycemia and fetal hyperinsulinemia) may mitigate the neonatal benefit of ACS in women with diabetes. Before neonatal benefit of ACS can be evaluated in this population, the first step is to optimize maternal glycemic control after ACS. Previous studies evaluating maternal hyperglycemia after ACS have been limited by small sample size, retrospective study design, or insufficient glucose data. Use of continuous glucose monitoring (CGM) in a randomized clinical trial provides a unique opportunity to overcome these challenges. Our long-term goal is to improve maternal and child health among women with diabetes as an independently funded clinical researcher. The research objectives of this proposal are to test the efficacy of three treatment strategies at achieving maternal glycemic control after ACS and evaluate the association between maternal glycemic control and neonatal outcomes. Our central hypothesis is that treatment with a continuous insulin infusion will improve maternal glycemic control, which is key to improving neonatal outcomes, but at the cost of less patient satisfaction and more health resource utilization. This hypothesis will be tested by pursuing the following specific aims: 1) Test the efficacy of three treatment strategies (addition of sliding scale insulin, up-titration of home insulin, and continuous insulin infusion) at achieving maternal glycemic control after ACS and 2) Quantify the association between maternal glycemic control after ACS and neonatal morbidity. Completion of these aims will determine the optimal strategy to achieve maternal glycemic control after ACS and inform a larger, multicenter trial to improve neonatal outcomes among women with diabetes and threatened PTB.

Type: Interventional

Start Date: Feb 2022

open study

Study ROR-PH-301, ADVANCE OUTCOMES, is designed to assess the efficacy and safety of ralinepag when added to pulmonary arterial hypertension (PAH) standard of care or PAH-specific background therapy in subjects with World Health Organization (WHO) Group 1 PAH.

Type: Interventional

Start Date: Aug 2018

open study

The objective of this prospective, randomized, blinded clinical trial is to assess the safety and efficacy of the Carillon Mitral Contour System in treating heart failure with functional regurgitation (FMR).

Type: Interventional

Start Date: Jan 2018

open study

PRIZM is a Phase 2b randomized, double-blind, placebo-controlled, 3-treatment, 2-period, crossover study evaluating the efficacy and safety of oral zagociguat 15 and 30 mg vs. placebo when administered daily for 12 weeks in participants with genetically and phenotypically defined MELAS.

Type: Interventional

Start Date: Sep 2024

open study

A randomized, sham-controlled, double-blind clinical trial to examine the safety and effectiveness of tRNS on unmedicated pediatric patients (7-12 years) with ADHD. Subjects will undergo either tRNS or sham treatment for 10 days during a two-week period in a home-simulated environment. Each treatment session is 20 minutes, during which their attention will be maintained using a software game.

Type: Interventional

Start Date: Jul 2024

open study

The purpose of this study is to evaluate the effectiveness of small phlebotomy tubes to reduce RBC transfusions in medical intensive care unit (ICU) and Intermediate care unit (IMU) patients with low hemoglobin compared with standard size tubes, to compare the intervention and the control groups in regards to: ICU length of stay (LOS), ICU mortality, hospital LOS, and hospital mortality and to assess the acceptability of small phlebotomy tubes in adult ICU and IMU patients.

Type: Interventional

Start Date: Jul 2023

open study

This study intends to explore feasibility, acceptability, and outcomes related to the use of a digital health coaching intervention for individuals who have completed primary therapy for cancer. Up to 625 individuals with diverse cancer diagnoses will be enrolled across up to 8 clinical sites to participate in a randomized wait-list control study. Those in the intervention group will receive 6 months of digital coaching up front followed by 6 months of ongoing monitoring via patient reported and clinical outcomes, as well as wearable data. Those in the control group will be monitored via patient reported and clinical outcomes as well as wearable data for the first 6 months followed by 6 months of digital health coaching. Both groups will collect fecal microbiome samples at enrollment and month 6. The study aims to explore if and how digital health coaching may be used to enhance outcomes for individuals following completion of primary cancer therapy.

Type: Interventional

Start Date: Jun 2022

open study

The purpose of this study is to see if the addition of IV Ketorolac to usual care multi-modal pain therapy compared to usual care will improve oral intake post-operatively

Type: Interventional

Start Date: Sep 2021

open study

The purpose of this study is to test the safety, tolerability, and effects of choline in people with increased risk of Alzheimer's Disease (AD), also known as pre-symptomatic AD. Choline is a dietary supplement, but is being investigated to see if it has any effects on the progression to AD.

Type: Interventional

Start Date: Jun 2023

open study

Nonalcoholic fatty liver disease (NAFLD) is a spectrum of liver conditions associated with fat accumulation that ranges from benign, non-progressive liver fat accumulation to severe liver injury, cirrhosis, and liver failure. NAFLD is the most common liver disease in US adults and the second leading cause for liver transplantation in the US. The natural history of NAFLD in the general population has been well described, with those with non-alcoholic fatty liver (NAFL, or simple steatosis) destined to have rare incidence of hepatic events compared to those with non-alcoholic steatohepatitis (NASH), who are at high risk for future development of cirrhosis, liver cancer and liver failure. The NASH Clinical Research Network (NASH CRN) was established by the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) in 2002, through the mechanism of RFA-DK-01-025, to further the understanding of diagnosis, mechanisms, progression and therapies of NASH. The NASH CRN effort has resulted in numerous seminal studies in the field. However, NASH CRN studies have systematically excluded persons living with HIV (PLWH), as NAFLD in these persons was thought to be different from that in the general population due to HIV, ART, concomitant medications, and co-infections. This has resulted in major knowledge gaps regarding NAFLD in the setting of HIV. This ancillary study of NAFLD and NASH in Adults with HIV (HIV NASH CRN), HNC 001 goal is to examine the prevalence of hepatic steatosis and NAFLD in a large, multicenter, and multiethnic cohort of PLWH (Steatosis in HIV Study)

Type: Observational

Start Date: Jul 2021

open study

A multicenter, randomized, adaptive allocation clinical trial to determine if increasing durations of induced hypothermia are associated with an increasing rate of good neurological outcomes and to identify the optimal duration of induced hypothermia for neuroprotection in comatose survivors of cardiac arrest.

Type: Interventional

Start Date: May 2020

open study

Patients with schizophrenia spectrum disorder (SSD) will be exposed to active repetitive transcranial magnetic stimulation (rTMS) from H coil for improving white matter integrity.

Type: Interventional

Start Date: May 2024

open study

The goal of Parts A and B of this Phase 1, first-in-human, randomized study is to assess the safety, tolerability, and pharmacokinetics (PK) of single (SAD) and multiple (MAD) ascending doses of oral JNT-517 in healthy participants. In Part C, the goal is to evaluate the differences in bioavailability between a tablet and suspension formulation of JNT-517 and the food effect in healthy volunteers. All participants in Part C will receive JNT-517. The goal of Part D is to assess the safety, tolerability, PK, and effect on urinary Phe and other amino acids of JNT-517 in participants with phenylketonuria (PKU). Participants in Part D will receive either JNT-517 or placebo and will be blinded to their treatment assignment. The study consists of 4 parts: - Part A: SAD in healthy participants -randomized, double-blind, placebo-controlled - Part B: MAD in healthy participants (14 days)-randomized, double-blind, placebo-controlled - Part C: Relative bioavailability of 2 formulations and food effect in healthy participants-randomized, open-label - Part D: Phase 1b in participants with PKU (4 weeks)-randomized, double-blind, placebo-controlled In each part, participants will complete a Screening Period, a Treatment Period, and a Follow-up Period for safety.

Type: Interventional

Start Date: Oct 2022

open study

A prospective, retrospective, non-randomized, multicenter study with two independent hernia study cohorts (Ventral / Incisional Hernia Repair and Diaphragmatic / Hiatal Hernia Repair). The primary objective of this study is to collect GORE® ENFORM Biomaterial product commercial-use data on device functional performance.

Type: Interventional

Start Date: May 2021

open study

A multicenter, prospective, single arm, non randomized clinical trial to evaluate the safety and efficacy of the C2 CryoBalloon Focal Ablation System (CbFAS) for the treatment of persistent dysplasia or intestinal metaplasia (IM) in the tubular esophagus after 3 or more radiofrequency ablations (RFA) for dysplastic BE, or <50% eradication of Barrett's Esophagus (BE) after 2 RFA treatments.

Type: Interventional

Start Date: Sep 2018

open study

The purpose of the study is to test the hypothesis that functionally navigated repetitive TMS stimulations to the prefrontal cortex (PFC) modulate aberrant cortical electrical activities at PFC circuitry. The TMS location of the PFC site will be individually localized by the symptom-related functional connectivity between PFC and symptom related areas (such as the auditory and language processing cortex). The investigators predict that such modulation will correct abnormal activities in patients with schizophrenia, reduce symptoms, especially auditory hallucination, and improve working memory/sustained attention performance.

Type: Interventional

Start Date: Oct 2016

open study

Multi-center open-label randomized controlled trial to assess if early intervention (12.0-14.0 weeks) (study group) improves the outcome of TRAP sequence as compared to late intervention (16.0-19.0 weeks) (control group). The investigators will randomly assign women diagnosed with TRAP sequence diagnosed between 12.0 and 13.6 weeks to an early or late intervention group (1:1), using a web-based application and a computer-generated list with random permuted blocks of sizes 2 or 4 (www.sealedenvelope.com), stratified by gestational age (GA) at inclusion (11.6 -12.6 weeks versus 13.0-13.6 weeks). Analysis will be by intention to treat.

Type: Interventional

Start Date: May 2016

open study

The objective is to test the efficacy of a laparotomy-assisted fetoscopic surgical approach to cover spina bifida spinal cord developmental defects using cryopreserved human umbilical cords (NEOX Cord 1K®) as a meningeal and skin patch.

Type: Interventional

Start Date: Sep 2023

open study

The purpose of this study is to investigate whether transcranial direct-current stimulation (tDCS) will engage reward-related brain circuitry, more specifically the uncinate fasciculus (UF) tract, which connects the orbitofrontal cortex (OFC) and nucleus accumbens (NAcc) regions. Also to evaluate whether the changes in the fractional anisotropy (FA) of the UF tract are associated with changes of clinical symptoms of anhedonia and finally to investigate the moderation role of simulated electric fields (EFs) in an association between FA of the UF and symptoms of anhedonia.

Type: Interventional

Start Date: Oct 2022

open study

The objectives of this study are to determine the feasibility, tolerability, and treatment effect of endoscopic ultrasound (EUS) radiofrequency ablation (RFA) plus standard-of-care neoadjuvant chemotherapy (NAC) in the treatment of pancreatic ductal adenocarcinoma (PDAC). Endoscopic ultrasound (EUS) radiofrequency ablation (RFA) and neoadjuvant chemotherapy (NAC) will be performed before tumor resection surgery, with the goal of shrinking a tumor or stopping the spread of cancer so that surgery might be less invasive and more effective.

Type: Interventional

Start Date: Aug 2021

open study