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NCT ID: NCT04581408 Recruiting - Long QT Syndrome Clinical Trials

Mutation-specific Therapy for the Long QT Syndrome

MAST2
Start date: June 15, 2021
Phase: Phase 2
Study type: Interventional

Novel therapy for the Long QT Syndrome based on the mechanism of action of the disease-causing mutations Long QT syndrome type 2 (LQT2) accounts for ~ 35% of all LQTS cases and is difficult to manage, as beta-blockers frequently fail to provide full protection. Most LQT2 patients (pts) have a Class 2 mutation, which implies defective "trafficking". Lumacaftor (LUM) is a drug developed and currently indicated for the treatment of cystic fibrosis (CF) in patients homozygous for the F508del mutation in the CFTR gene. LUM corrects protein folding and trafficking defects of mutant and misfolded CFTR channels, restoring their cell surface expression. The investigators recently demonstrated that LUM can rescue in vitro the LQTS phenotype observed in human induced pluripotent stem cell- derived cardiomyocytes (hiPSC-CMs) from pts with LQT2 Class 2 mutations (PMID: 29020304) and in these same two patients Orkambi administrated for 7 days at the same dosage approved for cystic fibrosis showed to reduce their QTc (PMID: 30753398). With the present phase II clinical trial (MAST2) the investigators will enroll 20 LQT2 patients (see inclusion and exclusion criteria) and they will test in vivo the efficacy of Orkambi in shortening their QTc. Patients will be admitted to hospital for a maximum of 7 days (minimum in-hospital stay based on evidence of QTc shortening). Orkambi will be administered at the dose approved for cystic fibrosis and during the entire period continuous ECG monitoring through both telemetry and 12-lead 24-hr Holter monitoring will be performed and QTc length and morphology will be analyzed.

NCT ID: NCT04581187 Recruiting - Clinical trials for Hematologic Malignancies

An Online-platform to Improve Patient-centered Care During the COVID-19 Pandemic: a GIMEMA Surveillance Program in Hematologic Malignancies

Start date: December 2, 2020
Phase:
Study type: Observational [Patient Registry]

This is a national multicenter prospective observational study led by the GIMEMA. The GIMEMA-ALLIANCE Platform is also an online monitoring system for patients with hematologic malignancies aiming at helping hematologists in the early recognition and timely management of problems of their patients. Based on patient's rating of specific items (i.e. on the presence of clinically relevant problems or problems with adherence to therapy or risk of SARS-CoV-2 infection), the Platform will automatically send alerts to the treating hematologist (and/or appointed members of the local Team). Physicians will be free to make any action they feel appropriate for the best care of their patients.

NCT ID: NCT04580940 Completed - Pancreatic Diseases Clinical Trials

SpyGlass™ Discover Percutaneous

Start date: July 27, 2021
Phase:
Study type: Observational [Patient Registry]

To document the clinical utility of percutaneous cholangiopancreatoscopy using a thin, disposable, flexible endoscope for evaluation and treatment of complex pancreaticobiliary disease in a prospective, multi-center case series

NCT ID: NCT04580914 Completed - Clinical trials for Paroxysmal Atrial Fibrillation

Clinical Evaluation of the StablePoint Catheter and Force Sensing System for Paroxysmal Atrial Fibrillation

NEwTON AF
Start date: April 12, 2021
Phase: N/A
Study type: Interventional

The NEwTON AF study is a multi-center, global, prospective, single arm study to establish the safety and effectiveness of the IntellaNav StablePoint Catheter and Force-Sensing System in subjects with symptomatic, drug refractory, recurrent paroxysmal atrial fibrillation.

NCT ID: NCT04580680 Recruiting - Sepsis Clinical Trials

Extracorporeal Blood Purification Therapy in Critically Ill Patients (GlobalARRT)

GlobalARRT
Start date: November 1, 2020
Phase:
Study type: Observational [Patient Registry]

Worldwide, the use of Extracorporeal Blood Purification (EBP) in everyday clinical practice is becoming increasingly common, particularly in critical care settings. The efficacy of most of these treatments on removal of inflammatory mediators is the main rationale behind the use of EBP in critically ill patients with multiorgan dysfunction. Nonetheless, there are still some doubts as to the clinical efficacy of bacterial toxins and cytokines removal and many clinical trials aiming at exploring the effect of EBP on long-term outcomes of septic patients have failed to demonstrate consistent results regarding 28 day- or hospital-mortality rates. The primary aim of this observational prospective web-based registry is to define the possible clusters of critically ill patients - treated with extracorporeal blood purification therapies worldwide - who are homogeneous regarding both clinical and treatment characteristics and seem to benefit the most from EBP.

NCT ID: NCT04580485 Completed - Colorectal Cancer Clinical Trials

INCB106385 Alone or in Combination With Immunotherapy in Advanced Solid Tumors

Start date: February 3, 2021
Phase: Phase 1
Study type: Interventional

This is a multicenter, open-label, dose-escalation/dose-expansion Phase 1 clinical study to investigate the safety, tolerability, PK profile, pharmacodynamics, and preliminary clinical efficacy of INCB106385 when given as monotherapy or in combination with INCMGA00012 in participants with selected CD8 T-cell-positive advanced solid tumors including SCCHN, NSCLC, ovarian cancer, CRPC, TNBC, bladder cancer, and specified GI malignancies (defined as CRC, gastric/GEJ cancer, HCC, PDAC, or SCAC)

NCT ID: NCT04580420 Recruiting - Clinical trials for End Stage Renal Disease

Safety & Efficacy of DCR-PHXC in Patients With PH1/2 and ESRD

PHYOX7
Start date: April 15, 2021
Phase: Phase 2
Study type: Interventional

The aim of this study is to evaluate DCR-PHXC in participants with PH1 or PH2 and severe renal impairment, with or without dialysis.

NCT ID: NCT04580121 Completed - Clinical trials for Acute Myeloid Leukemia

A Dose Escalation and Expansion Study Evaluating the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of RO7283420.

Start date: November 4, 2020
Phase: Phase 1
Study type: Interventional

This open-label, entry-into-human (EIH) study will evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics of RO7283420. Escalating doses of RO7283420 will be administered to participants with Acute Myeloid Leukemia (AML) in order to determine the maximum tolerated dose (MTD) and/or recommended Phase II dose (RP2D).

NCT ID: NCT04580069 Completed - Knee Pain Swelling Clinical Trials

Role of Two Rehabilitative Treatments and Diet in the Modulation of Inflammation in Patients With Knee Prosthesis.

Start date: September 30, 2019
Phase: N/A
Study type: Interventional

The investigators studied the influence of two aproved rehabilitative treatments, connective tissue techniques and lymphatic drainage, on acute inflammation markers. Furthermore, the role of mediterranean diet on the same values was investigated in the first postoperative period

NCT ID: NCT04579952 Recruiting - Pain, Chronic Clinical Trials

Effects of Transcranial Direct Current Stimulation on Pain Control in Chronic Painful Total Knee Arthroplasty

TSEF-PTG
Start date: January 25, 2021
Phase: N/A
Study type: Interventional

The TSEF-PTG study aims to evaluate the effects of transcranial Direct Current Stimulation (tDCS) and exercise versus sham tDCS (placebo) and exercise on pain control in chronic painful total knee arthroplasty (TKA) patients.