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NCT ID: NCT04856982 Recruiting - Clinical trials for Amyotrophic Lateral Sclerosis Associated With a SOD1 Gene Mutation

A Study of BIIB067 (Tofersen) Initiated in Clinically Presymptomatic Adults With a Confirmed Superoxide Dismutase 1 Mutation

ATLAS
Start date: May 17, 2021
Phase: Phase 3
Study type: Interventional

The primary objective of this study is to evaluate the efficacy of tofersen in presymptomatic adult carriers of a superoxide dismutase 1 (SOD1) mutation with elevated neurofilament (NF). The secondary objectives of this study are to evaluate the safety and tolerability tofersen and to evaluate the effect of tofersen on pharmacodynamics (PD)/treatment response biomarkers when initiated prior to versus at the time of emergence of clinically manifest amyotrophic lateral sclerosis (ALS).

NCT ID: NCT04856085 Recruiting - Clinical trials for Hepatitis B, Chronic

Study of VIR-2218, VIR-3434, and/or PEG-IFNα in Subjects With Chronic Hepatitis B Virus Infection

MARCH
Start date: July 11, 2021
Phase: Phase 2
Study type: Interventional

This is a phase 2 study in which participants with chronic hepatitis B virus (HBV) infection will receive VIR-2218, VIR-3434 and/or PEG-IFNα and be assessed for safety, tolerability, and efficacy

NCT ID: NCT04855890 Recruiting - Atrial Fibrillation Clinical Trials

HIgh Power Short Duration Radiofrequency Ablation or Cryoballoon Ablation for Paroxysmal Atrial Fibrillation

HIPAF
Start date: May 1, 2021
Phase: N/A
Study type: Interventional

Pulmonary vein isolation (PVI) using the cryoballoon has been proven equal to RF-PVI and is widely used. High Power Short Duration Ablation in RF-PVI has been successfully tested in several trials. Prospective data comparing both strategies is lacking. This trial will compare acute success rates, safety and foremost long-term outcome of two different PVI strategies (HPSD-PVI vs. cryo-PVI).

NCT ID: NCT04855045 Recruiting - Eye Diseases Clinical Trials

An Open-label, Dose Escalation and Double-masked, Randomized, Controlled Trial Evaluating Safety and Tolerability of Sepofarsen in Children (<8 Years of Age) With LCA10 Caused by Mutations in the CEP290 Gene.

BRIGHTEN
Start date: March 23, 2021
Phase: Phase 2/Phase 3
Study type: Interventional

PQ-110-005 (BRIGHTEN) is an open-label, dose escalation and double-masked, randomized, controlled study evaluating safety and tolerability of sepofarsen administered via intravitreal (IVT) injection in pediatric subjects (<8 years of age) with LCA10 due to the c.2991+1655A>G mutation over 24 months of treatment.

NCT ID: NCT04854226 Recruiting - Infant Development Clinical Trials

Protein Turnover in Preterm Infants - Feeding of Target Fortified Breast Milk With Different Macronutrient Composition to Improve Growth

Start date: April 21, 2021
Phase: N/A
Study type: Interventional

The purpose of this study is to measure protein turnover rates in preterm infants.

NCT ID: NCT04850105 Recruiting - Clinical trials for Hereditary Transthyretin Amyloidosis With Polyneuropthy

A Non-interventional Cohort Safety Study of Patients With hATTR-PN

Start date: September 21, 2021
Phase:
Study type: Observational

This is a prospective, non-interventional, Long-term, multinational cohort safety study of patients with Hereditary Transthyretin Amyloidosis with Polyneuropathy (hATTR-PN). The overarching goal of this study is to further characterize the long-term safety of TEGSEDI (inotersen) in patients with hATTR-PN under real-world conditions.

NCT ID: NCT04849936 Recruiting - Clinical trials for Inflammatory Bowel Diseases

A Diagnostic Accuracy Study Testing Fecal Biomarkers In Comparison To Endoscopic Examination

Start date: July 13, 2020
Phase:
Study type: Observational

In this study it will be explored whether the levels of fecal biomarkers are associated with histologic inflammation of the intestinal mucosa and concomitant alteration of the mucosal permeability. The aim of the study is to assess the diagnostic accuracy of fecal biomarkers especially to detect the potential of differentiation between inflammatory and functional gastrointestinal diseases. The following biomarkers will be examined in the stool of the participants: calprotectin, lactoferrin, pmn-elastase, human beta-defensin, zonulin, alpha-antitrypsin.

NCT ID: NCT04849806 Recruiting - COPD Clinical Trials

Sympathetic Nerve Activity Predictors in Patients With Chronic Obstructive Pulmonary Disease

SNAP-COPD
Start date: May 10, 2022
Phase:
Study type: Observational

The project will be pursued in our respiratory, autonomic nervous system physiology laboratory (Respiratory, autonomic nervous system physiology laboratory, Department of Pneumology and Intensive Care Medicine, RWTH Aachen University Hospital; Head of Department: Professor Michael Dreher). Overactivity of the sympathetic nerve activity (SNA) axis with "centrally" increased heart rate and peripheral vasoconstriction is a known phenomenon in patients with systolic heart failure (HF) and has recently been described in patients with primary lung disease as seen in chronic obstructive pulmonary disease (COPD). However, systematic analyses on this clinically relevant topic are currently lacking. Thus, using a comprehensive, multimodal approach and state-of-the-art technology, this research project is designed to determine the extent and nature of increased SNA in COPD (AIM 1) and evaluate the underlying mechanisms (AIM 2). The project will address the following hypotheses: 1. In COPD, concomitant obstructive sleep apnea is independently associated with increased SNA. 2. Precapillary pulmonary hypertension (PH), inspiratory muscle dysfunction and systemic inflammation describe a COPD phenotype characterised by increased SNA with a different subtype.

NCT ID: NCT04849728 Recruiting - Clinical trials for NASH - Nonalcoholic Steatohepatitis

A Phase 3 Study Evaluating Efficacy and Safety of Lanifibranor Followed by an Active Treatment Extension in Adult Patients With (NASH) and Fibrosis Stages F2 and F3 ( NATiV3 )

NATiV3
Start date: August 19, 2021
Phase: Phase 3
Study type: Interventional

This Phase 3 study is conducted to evaluate lanifibranor in adults with NASH and liver fibrosis histological stage F2 or F3

NCT ID: NCT04848844 Recruiting - Heart Failure Clinical Trials

The PAtients pResenTing With COngenital HeaRt DIseAse Register (ARTORIA-R)

ARTORIA-R
Start date: September 2, 2020
Phase:
Study type: Observational [Patient Registry]

Advances in surgical and medical care have led to improved outcomes in patients with congenital heart disease (CHD). As a consequence, the majority of patients nowadays survives to adulthood (adults with CHD, that is, adult CHD [ACHD]) with good quality of life. Despite the surgical success, the morbidity and mortality of ACHD is higher than in the general population and is linked to the development of heart failure (HF) in adulthood. HF occurs in approximately 25% of patients with ACHD, even in those patients in whom the congenital mal-formation has been corrected successfully in childhood. The time course and presentation are heterogeneous owing to variable congenital malformation and limitation of treatment options. ACHD with an anatomic right ventricle as the systemic ventricle (e.g., atrial switch operation in patients with transposition of the great arteries [TGAs]) and those with a functional single ventricle (e.g., Fontan circulation) appear to be at higher risk of developing HF. Young age at initial corrective surgery-often in the first 2 years of life-and lack of specific medical therapies can contribute to a high and early demand for heart transplantation in patients with ACHD.