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NCT ID: NCT05347147 Terminated - Clinical trials for Idiopathic Intracranial Hypertension

A Trial to Determine the Efficacy and Safety of Presendin in IIH

IIH EVOLVE
Start date: November 18, 2022
Phase: Phase 3
Study type: Interventional

Idiopathic intracranial hypertension (IIH) has significant associated morbidity and reduced quality of life. There is a significant risk of visual loss and patients also typically suffer with chronic disabling headaches. This trial has been designed to evaluate the efficacy and safety of a new formulation of exenatide (Presendin) in the reduction of intracranial pressure (ICP) in patients with IIH.

NCT ID: NCT05343949 Terminated - COPD Clinical Trials

High Intensity Interval Training and Technologies in COPD

Start date: May 8, 2019
Phase: N/A
Study type: Interventional

Trials in COPD have shown that HIIT leads to the same positive outcomes as constant load training but causes less breathlessness and leg discomfort during training. However, HIIT protocols in existing trials have all been different and use relatively long interval durations (30 s) and short rests. This is sub-optimal because long interval durations lead to greater breathlessness and patients may fear that they will not fully recover during short rests, potentially decreasing adherence. A novel HIIT protocol involving very brief intervals (e.g. 10 s) with longer rests may provide the same benefits with less distress due to breathlessness.

NCT ID: NCT05330286 Terminated - Healthy Volunteers Clinical Trials

Study to Compare the Pharmacokinetics, Safety and Tolerability of the Pediatric and Adult Branaplam Formulation in Healthy Adults and the Effect of Food on the Latter.

Start date: April 13, 2022
Phase: Phase 1
Study type: Interventional

This is a Phase 1 study designed to assess the relative bioavailability (BA), safety and tolerability and PK of the pediatric and adult formulations of branaplam.

NCT ID: NCT05323110 Terminated - Clinical trials for Chronic Inflammatory Disease

Study of Intravenously Administered Anti-LIGHT Monoclonal Antibody CBS001 in Healthy Volunteers

Start date: April 14, 2022
Phase: Phase 1
Study type: Interventional

A Phase 1, first in human (FIH), single-centre, double-blind, randomized, placebo-controlled, dose escalating trial to assess the safety and tolerability, pharmacokinetics (PK), immunogenicity and pharmacodynamics (PD) of CBS001 in healthy subjects. The study will be conducted in 2 parts: Part A: Single ascending intravenous (IV) doses of CBS001 Part B: Multiple ascending IV doses of CBS001

NCT ID: NCT05321524 Terminated - Biliary Atresia Clinical Trials

Obeticholic Acid in Pediatric Subjects With Biliary Atresia

CARE
Start date: July 1, 2015
Phase: Phase 2
Study type: Interventional

This is a Phase 2, multicenter, open-label, single dose and multi-dose, dose-finding study with an optional open-label extension (OLE) to assess the safety, tolerability, and pharmacokinetics of obeticholic acid (OCA) in pediatric subjects with biliary atresia with successful hepatoportoenterostomy (HPE, also known as a Kasai portoenterosomy). The OLE will continue to evaluate safety, tolerability, pharmacodynamics, and efficacy of OCA. In addition, a change in vitamin A and D levels, and where possible the degree of change in liver stiffness, will be assessed during the OLE.

NCT ID: NCT05307679 Terminated - Dup15q Syndrome Clinical Trials

A Study to Evaluate the Safety and Efficacy of Basmisanil Treatment in Children Aged 2-14 Years With Dup15q Syndrome

Start date: December 16, 2022
Phase: Phase 2
Study type: Interventional

This study consists of two parts. Part 1 will evaluate the safety, efficacy, and pharmacodynamics of 52-weeks of basmisanil treatment in children and adolescents (aged 2-14 years) with Dup15q syndrome. Part 1 will test the hypothesis that negative allosteric modulation of a GABAA receptor subtype can address excessive receptor function and positively impact core neurodevelopmental disease feature in individuals with Dup15q syndrome. Part 2 is an optional 2-year open-label extension to evaluate long-term safety, tolerability, and to provide supportive evidence of benefit of continued treatment with basmisanil in selected efficacy outcomes.

NCT ID: NCT05305040 Terminated - Clinical trials for Cytomegalovirus Infections

Study of Posoleucel (ALVR105,Viralym-M) for Multi-Virus Prevention in Patients Post-Allogeneic Hematopoietic Cell Transplant

Prevent
Start date: March 24, 2022
Phase: Phase 2/Phase 3
Study type: Interventional

This is a Phase 3 study to evaluate posoleucel (ALVR105, Viralym-M); an allogeneic, off-the-shelf multi-virus specific T cell therapy that targets six viral pathogens: BK virus, cytomegalovirus, adenovirus, Epstein-Barr virus, human herpesvirus 6 and JC virus.

NCT ID: NCT05298501 Terminated - Clinical trials for Mild Cognitive Impairment

Assessing Performance on Speech Tasks Via Crowdsourced Participants

AMYWEB
Start date: April 5, 2022
Phase:
Study type: Observational

The purpose of the study is to examine the properties of the Automatic Story Recall Test (ASRT) and its parallel variants, as well as letter fluency and category fluency cognitive tests. Tests will be completed in crowdsourced populations, to derive normative data, and examine test properties in demographically diverse and cognitively impaired participants recruited and tested online.

NCT ID: NCT05267574 Terminated - Clinical trials for Primary Mitochondrial Myopathy

An Open Label, Long Term Safety Study of REN001 in Primary Mitochondrial Myopathy Patients (Stride Ahead)

Start date: February 1, 2022
Phase: Phase 2/Phase 3
Study type: Interventional

This study is designed to evaluate the long-term safety and tolerability of REN001 administered once daily to subjects with PMM due to mitochondrial DNA mutations (mtDNA-PMM) or nuclear DNA mutations (nDNA-PMM). Subjects with mtDNA mutations will have previously completed Study REN001-201 or participated in Study REN001-101. Subjects with nDNA mutations who enroll in this study will be REN001- naïve.

NCT ID: NCT05256810 Terminated - Gout Clinical Trials

A Study to Evaluate ALN-XDH in Healthy Subjects and Patients With Gout

Start date: February 25, 2022
Phase: Phase 1/Phase 2
Study type: Interventional

The purpose of this study is to: - Part A: Evaluate the safety and tolerability of single ascending doses of ALN-XDH in healthy adult participants - Part B: Evaluate the safety, tolerability and efficacy of ALN-XDH as monotherapy in adult patients with gout - Part C: Evaluate the safety, tolerability and efficacy of ALN-XDH as add-on therapy in adult patients with gout