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NCT ID: NCT06041906 Enrolling by invitation - Clinical trials for Congenital Porto-Systemic Shunt (CPSS)

International Registry of Congenital Portosystemic Shunt (IRCPSS)

IRCPSS
Start date: April 26, 2018
Phase:
Study type: Observational [Patient Registry]

Congenital Portosystemic Shunt (CPSS) is a rare condition important by the multiplicity and severity of associated complications. CPSS is venous anomaly in which blood coming from the intestines only partially passes through the liver. This leads to the accumulation of potentially toxic factors that cause systemic effects. Complications vary among the individuals, and currently, it is challenging to predict which individuals will develop severe complications. The IRCPSS registry is established with the aim of centralizing detailed clinical follow-up and biological information from participants around the world who suffer from Congenital Portosystemic Shunt (CPSS). A multidisciplinary consortium of experts is collaborating to enhance our understanding of the prevalence, natural history, individual risks, and physiopathology of the disease through the IRCPSS registry.

NCT ID: NCT05929807 Enrolling by invitation - Achondroplasia Clinical Trials

A Clinical Trial to Investigate Long-term Safety, Tolerability, and Efficacy of Weekly Subcutaneous Doses With TransCon CNP in Children and Adolescents With Achondroplasia

AttaCH
Start date: June 21, 2023
Phase: Phase 2/Phase 3
Study type: Interventional

TransCon CNP administered once-weekly in children and adolescents with achondroplasia who have completed a prior TransCon CNP clinical trial. Participants who complete a prior TransCon CNP trial and meet all eligibility criteria will be invited to continue into the long-term open label extension trial to receive 100 µg CNP/kg/week of TransCon CNP. Trial treatment will be completed when the participant reaches 16 years of age for females and 18 years of age for males and have femur and tibial epiphyseal closure. TransCon CNP treatment will continue if femur and tibial epiphyseal closure is not confirmed at the age of 16 years for females, and 18 years for males. Treatment with TransCon CNP will be completed once femur and tibial epiphyseal closure is confirmed by radiographic imaging. The trial duration is individual for each trial participant. Visits will occur every 12-14 weeks throughout the trial.

NCT ID: NCT05889273 Enrolling by invitation - Clinical trials for Autism Spectrum Disorder

ML-004 Open-Label Extension Study in Adults and Adolescents With Autism Spectrum Disorders (ASD)

Start date: June 1, 2023
Phase: Phase 2
Study type: Interventional

ML-004-003 is a multi-center, open-label extension study that will enroll approximately 120 adolescent and adult subjects with ASD that have completed study ML-004-002. The primary objective of the study will be to evaluate the safety of ML-004 in subjects with ASD.

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.