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NCT ID: NCT05883748 Enrolling by invitation - Clinical trials for Erythropoietic Protoporphyria

Open-Label, Long-Term Extension Study to Investigate the Safety, Tolerability, and Efficacy of DISC-1459 (Bitopertin) in Participants With EPP

Start date: August 31, 2023
Phase: Phase 2/Phase 3
Study type: Interventional

This is an open-label, long-term extension study to investigate the safety, tolerability and efficacy of DISC-1459 in participants with EPP.

NCT ID: NCT05844449 Enrolling by invitation - Cystic Fibrosis Clinical Trials

Evaluation of Long-Term Safety and Efficacy of Vanzacaftor/Tezacaftor/Deutivacaftor in Cystic Fibrosis Participants 1 Year of Age and Older

Start date: August 11, 2023
Phase: Phase 3
Study type: Interventional

The purpose of this study is to evaluate the long-term safety, tolerability, and efficacy of vanzacaftor/tezacaftor/deutivacaftor (VNZ/TEZ/D-IVA) in participants with cystic fibrosis (CF).

NCT ID: NCT05796440 Enrolling by invitation - Clinical trials for Growth Hormone Deficiency

A Long-Term Safety Trial of LUM-201 in Children With Idiopathic Growth Hormone Deficiency Who Have Previously Completed a LUM-201 Clinical Trial (OraGrowtH211)

OraGrowtH211
Start date: August 1, 2023
Phase: Phase 2
Study type: Interventional

This is a multi-national trial. The trial aims to study the long-term safety of LUM-201 in subjects with Idiopathic Pediatric Growth Hormone Deficiency (iPGHD). This study will also assess pharmacodynamics and efficacy response to therapy with LUM-201.

NCT ID: NCT05759013 Enrolling by invitation - Clinical trials for Respiration, Artificial

Pivotal Evaluation of Abdominal Neuromuscular Electrical Stimulation (VentFree) for Weaning From Mechanical Ventilation

PREVENT
Start date: February 15, 2024
Phase: N/A
Study type: Interventional

The objective of this study is to evaluate the efficacy of the VentFree Respiratory Muscle Stimulator (VentFree) in critically ill adult patients who require invasive mechanical ventilation, when compared to sham.

NCT ID: NCT05752032 Enrolling by invitation - Clinical trials for Osteoarthritis, Knee

A Long Term Follow-up Study of Subjects Who Received ICM-203 or Matching Placebo

Start date: March 16, 2023
Phase:
Study type: Observational

This is an observational study of the long term safety and efficacy of ICM-203.

NCT ID: NCT05685303 Enrolling by invitation - Heart Failure Clinical Trials

Alleviant ALLAY-HF Study

Start date: January 10, 2023
Phase: N/A
Study type: Interventional

Prospective, multicenter, randomized, sham-controlled, double blinded, adaptive study designed to evaluate the safety and efficacy of a percutaneously created interatrial shunt using the Alleviant ALV1 System in patients with HFpEF/HFmrEF.

NCT ID: NCT05606991 Enrolling by invitation - Dementia Clinical Trials

The Effect of OSA on Brain Waste Clearance

Start date: July 18, 2023
Phase: N/A
Study type: Interventional

Recent ground-breaking research has shown that clearance of toxic neuro-metabolites from the brain including the proteins β-Amyloid (Aβ) and tau that form dementia causing plaques and tangles is markedly impaired when sleep is disturbed. This suggests that dementia risk may be increased in people with sleep disorders such as obstructive sleep apnea (OSA). Longitudinal studies have linked OSA with a 70-85% increased risk for mild cognitive impairment and dementia. Despite this strong link, little is known about the OSA-specific mechanistic underpinnings. It is not fully understood as to how sleep disturbance in OSA inhibit brain glymphatic clearance. However, it is known that OSA inhibits slow wave sleep, profoundly activates sympathetic activity, and elevates blood pressure - particularly during sleep. These disturbances have, in turn, been shown to independently inhibit glymphatic function. Previous studies have attempted to sample human cerebrospinal fluid (CSF) involved in glymphatic clearance for dementia biomarkers during sleep. However, these studies were severely limited by the need for invasive CSF sampling. To address this problem, a set of newly available, highly sensitive blood based SIMOA assays will be used to study glymphatic function in people treated for severe OSA who undergo CPAP withdrawal. Furthermore, novel methods will be utilized to capture changes in slow wave sleep, blood pressure and brain blood flow together with sleep-wake changes in blood levels of excreted neuro-metabolites to define the pathophysiological mechanisms that inhibit brain cleaning in OSA.

NCT ID: NCT05604170 Enrolling by invitation - Clinical trials for Tuberous Sclerosis Complex

Open-label Study of Adjunctive GNX Treatment in Children and Adults With TSC-related Epilepsy

Start date: May 16, 2022
Phase: Phase 3
Study type: Interventional

This is a Phase 3, global, open-label extension (OLE) study of adjunctive GNX treatment in children and adults with TSC who previously participated in either Study 1042-TSC-3001 or Study 1042-TSC-2001

NCT ID: NCT05517564 Enrolling by invitation - Clinical trials for Non-alcoholic Steatohepatitis

First-in-Human Study of GM-60106 in Healthy Adults and Otherwise Healthy Adults With an Increased Body Mass Index and Markers of Non-Alcoholic Fatty Liver Disease

Start date: August 1, 2022
Phase: Phase 1
Study type: Interventional

This is a Phase 1a/1b, randomised, double-blind, placebo-controlled single- and multiple-ascending dose study to evaluate the safety, tolerability, PK, and PD of GM-60106 in healthy adult male and female participants and otherwise healthy adults who have an increased BMI and markers of NAFLD.

NCT ID: NCT05515536 Enrolling by invitation - Friedreich Ataxia Clinical Trials

A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia

Start date: December 8, 2022
Phase: Phase 3
Study type: Interventional

The primary objective of this study is to assess the long-term safety of vatiquinone in participants with Friedreich ataxia (FA) previously exposed to vatiquinone.