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NCT ID: NCT05526066 Recruiting - Clinical trials for Ornithine Transcarbamylase Deficiency

Study for Adolescent and Adult Participants With Ornithine Transcarbamylase Deficiency to Evaluate Safety and Tolerability of ARCT-810

Start date: July 6, 2022
Phase: Phase 2
Study type: Interventional

The primary objective is to evaluate the safety and tolerability of repeated doses of intravenously administered ARCT-810.

NCT ID: NCT05524883 Recruiting - Clinical trials for Duchenne Muscular Dystrophy (DMD)

Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping

DELIVER
Start date: August 12, 2022
Phase: Phase 1/Phase 2
Study type: Interventional

The primary purpose of this study is to evaluate the safety, tolerability, and dystrophin protein levels in muscle tissue following multiple intravenous (IV) doses of DYNE-251 in participants with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. The study consists of 3 periods: a multiple-ascending dose (MAD) / placebo-controlled period (24 weeks), an open-label period (24 weeks) and a long-term extension period (96 weeks).

NCT ID: NCT05524701 Recruiting - Sleep Restriction Clinical Trials

Short-term Sleep Restriction on Taste Preference and Perception

Start date: September 1, 2022
Phase: N/A
Study type: Interventional

The study aims is to investigate the effects of sleep restriction on sweet and fat taste perception and preference in both fasted and fed states. This is a randomised crossover trial with sleep restriction (4 hours per night for 2 nights) and normal sleep (at least 8 hours sleep for 2 nights) conditions, with a four-week washout period between conditions.

NCT ID: NCT05523804 Recruiting - Ovarian Cancer Clinical Trials

Patient Outcomes Following Interval and Delayed Cytoreductive Surgery in Advanced Ovarian Cancer

GO SOAR2
Start date: August 26, 2022
Phase:
Study type: Observational

To compare patient outcomes following interval and delayed cytoreductive surgeries and no surgery (neoadjuvant chemotherapy alone) and international variations in access to cytoreductive surgeries in women with advanced stage ovarian cancer.

NCT ID: NCT05523167 Recruiting - Dermatomyositis Clinical Trials

A Study to Investigate the Efficacy and Safety of Efgartigimod PH20 SC in Adult Participants With Active Idiopathic Inflammatory Myopathy.

ALKIVIA
Start date: October 12, 2022
Phase: Phase 2/Phase 3
Study type: Interventional

This study's purpose is to measure the treatment response from efgartigimod PH20 SC compared with placebo in participants with Idiopathic Inflammatory Myopathy (IIM). Participants with the IIM subtypes of dermatomyositis (DM), immune-mediated necrotizing myopathy (IMNM), or certain other subtypes of polymyositis (PM; including antisynthetase syndrome [ASyS]) will be included in the study. Treatment response will be measured by Total improvement score (TIS). Additional information can be found on https://myositis-study.com/.

NCT ID: NCT05522660 Recruiting - Clinical trials for Non-Small Cell Lung Cancer

Immunotherapy or Targeted Therapy With or Without Stereotactic Radiosurgery for Patients With Brain Metastases From Melanoma or Non-small Cell Lung Cancer

USZ-STRIKE
Start date: November 30, 2022
Phase: Phase 3
Study type: Interventional

The primary objective of the study is to assess the efficacy in terms of CNS-specific PFS of the combination of standard systemic treatment plus SRS vs. standard systemic treatment alone in patients with newly diagnosed and untreated (except for surgery) asymptomatic or oligosymptomatic brain metastases from melanoma or NSCLC. This proposed randomised phase III clinical study addresses one of the most controversial issues in the current approach to patients with brain mets: the timing of SRS in patients eligible for systemic immune checkpoint inhibition or targeted therapy in order to guide therapeutic options as to what strategy allows the best compromise between best survival and best QoL.

NCT ID: NCT05520398 Recruiting - Clinical trials for Obsessive-Compulsive Disorder

Neural Mechanisms of Obsessive-compulsive Disorder (OCD) Treatment Failure

ERP in Silico
Start date: November 10, 2021
Phase:
Study type: Observational

The aim of this study is to understand why patients with obsessive-compulsive disorder (OCD) fail in therapy. To understand the neural mechanisms involved in exposure therapy that support success and clinical improvement in order to improve therapy outcomes for OCD patients.

NCT ID: NCT05519995 Recruiting - Stress Clinical Trials

Sensate II Utilization and Perceived Stress in Adults

Start date: August 20, 2022
Phase:
Study type: Observational

Sensate® is A commercially marketed (FCC #: 2AS9ESEN231) stress management wellness device (BioSelf Technology; London, UK). This non-invasive wellness device is one component of a cross-modal (acoustic and aural) sensory experience designed to help an individual experience relaxation following a 10 to 30-minute session. The wellness device is paired with an in-app software program that offers infrasonic vibrations that are proprietarily synced to music or soundscapes. The wellness device is recommended in blogs, and online news articles as a possible modality for positive stress management (Bergland, 2017; Woodhall, 2018; Woolton, 2018), and anecdotal evidence from social media influencers suggest a relaxing response (Garth, 2021; Knight, 2021; Patterson, 2020). Moreover, a 2021 survey response from consumers of Sensate suggests an improvement in stress response during the COVID-19 pandemic (McDoniel & Chmelik, 2022). However, it is unknown to what extent, if any, the use of Sensate II correlated with validated measures of stress, affect, or sleep quality in adults.

NCT ID: NCT05519085 Recruiting - Clinical trials for Relapsed or Refractory Multiple Myeloma

A Study to Evaluate Mezigdomide, Bortezomib and Dexamethasone (MEZIVd) Versus Pomalidomide, Bortezomib and Dexamethasone (PVd) in Participants With Relapsed or Refractory Multiple Myeloma (RRMM)

SUCCESSOR-1
Start date: September 20, 2022
Phase: Phase 3
Study type: Interventional

The purpose of this study is to compare the efficacy and safety of mezigdomide (CC-92480), bortezomib and dexamethasone (MeziVd) versus pomalidomide, bortezomib and dexamethasone (PVd) in participants with relapsed or refractory multiple myeloma (RRMM) who received between 1 to 3 prior lines of therapy and who have had prior lenalidomide exposure.

NCT ID: NCT05519020 Recruiting - Cystic Fibrosis Clinical Trials

Inhaled Therapy Adherence and Outcomes to Kaftrio in Cystic Fibrosis

Start date: July 27, 2022
Phase:
Study type: Observational

Cystic fibrosis (CF) is a life-limiting and life-long genetic condition which requires intensive preventative treatment to manage the symptoms and progression of disease. While preventative treatments target the effects of cystic fibrosis, precision medicines target the underlying dysfunction of the cystic fibrosis transmembrane regulator (CFTR) protein at a cell level. The first of these expensive precision medicines also known as modulator therapies, Ivacaftor, was shown to be highly effective in clinical trials with an increase of over 10% in lung function. Real-world studies showed an increase of only 6% and a return to baseline lung function by year five of treatment. Preventative therapies were continued during the Ivacaftor clinical trials whereas there is real world evidence of declining inhaled preventative therapy use following Ivacaftor initiation. This is a potential explanation for the efficacy-effectiveness gap. The first study in the National Efficacy Effectiveness Modulator Optimisation (NEEMO) programme is exploring this (REC ref: 21/HRA/4940, IRAS 301975). Ivacaftor/Tezacaftor/Elexacaftor is the most recent modulator available, commissioned in the UK (United Kingdom) in 2020, and suitable for around 90% of people with cystic fibrosis. It is not yet known if the efficacy effectiveness gap seen with Ivacaftor also exists for Ivacaftor/Tezacaftor/Elexacaftor. There is also uncertainty about the continued need for preventative inhaled therapy alongside the prescription of Ivacaftor/Tezacaftor/Elexacaftor. This second study in the NEEMO programme is a cohort, observational study and will explore adherence to inhaled preventative therapies in adults with cystic fibrosis before and after commencing Ivacaftor/Tezacaftor/Elexacaftor, and in those not prescribed Ivacaftor/Tezacaftor/Elexacaftor. It will also look at the relationship between adherence to preventative inhaled therapy and outcome for adults with CF taking Ivacaftor/Tezacaftor/Elexacaftor. The analysis will use routinely collected pseudo anonymised data from the CFHealthHub learning health system (CFHealthHub), alongside anonymised data from the CF registry and routinely collected clinical data.