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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: NCT05524649 Recruiting - Clinical trials for Microbial Colonization

Effect of Probiotics on Infant's Fecal Microbiota Composition

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

Randomized clinical trial to evaluate the effect of two probiotic strains which belong to Bifidobacterium Longum and Pediococcus pentosaceus species on fecal microbiota composition in healthy infants. Secondary outcomes comprise evaluation of anthropometric growth, digestive tolerance, sleeping habits, incidence of functional gastrointestinal disorders, incidence of gastrointestinal and respiratory infections, allergic reactions and safety and tolerability of the product.

NCT ID: NCT05524571 Recruiting - Thyroid Eye Disease Clinical Trials

Study to Assess Batoclimab in Participants With Active Thyroid Eye Disease

Start date: January 5, 2023
Phase: Phase 3
Study type: Interventional

To evaluate the efficacy of batoclimab 680 milligrams (mg) subcutaneous (SC) once a week (QW) for 12 weeks followed by 340 mg SC QW for 12 weeks versus placebo on proptosis responder rate at Week 24.

NCT ID: NCT05524051 Recruiting - Clinical trials for Acute Kidney Injury Following Cardiac Surgery

A Multi-center Study to Evaluate the Safety, Tolerability and Efficacy of TIN816 in Patients at Risk for Acute Kidney Injury Following Cardiac Surgery.

Start date: March 3, 2023
Phase: Phase 2
Study type: Interventional

This is a randomized, multi-centric, placebo-controlled, participant and investigator-blinded study to evaluate the safety, tolerability and efficacy of TIN816 in adult patients at risk for acute kidney injury following cardiac surgery.

NCT ID: NCT05523895 Recruiting - Clinical trials for Irritability Associated With Autism Spectrum Disorder

Pimavanserin for the Treatment of Irritability Associated With Autism Spectrum Disorder

Start date: August 9, 2022
Phase: Phase 2/Phase 3
Study type: Interventional

6-week, randomized, double-blind, fixed-dose, placebo-controlled, parallel group study in children and adolescents (aged 5 to17 years) with autism spectrum disorder (ASD) with irritability, agitation, or self-injurious behaviors to study the efficacy and safety of pimavanserin

NCT ID: NCT05523557 Recruiting - Clinical trials for Pulmonary Hypertension

Urban-training Intervention in Pulmonary Hypertension (UTHAP)

UTHAP
Start date: February 1, 2023
Phase: N/A
Study type: Interventional

Pulmonary rehabilitation is effective in the treatment of pulmonary hypertension (PH). However, the beneficial effects of such intervention have been seen to disappear over time in other chronic diseases. The objective of the project is to evaluate the efficacy of an urban exercise program after a rehabilitation program and to identify possible determinants of sustainability of the effect, both in patients and in a murine experimental model. Methodology: 1) Study in humans: randomized study (urban training) in 80 patients with PH. The main variable is the improvement in physical activity measured by accelerometry at 12 months. Improvements in bioimpedance and aerobic capacity will be analysed as secondary variables, as well as possible determinants of vascular function that guarantee the sustainability of the effect (pulse wave velocity, endothelial function, metabolic profile and other plasma biomarkers), and all of them will be correlated with the evolution of the disease (admission due to clinical deterioration). 2) Studies in a murine experimental model: Mice with pulmonary hypertension induced by the administration of SU5416 (sugen) and exposure to hypoxia for 3 weeks will be studied after a three-week rehabilitation program. Half of them will exercise 1-2 days a week for 4 more weeks. At the end of the program, the right ventricular pressure will be measured and subsequently the animals will be sacrificed. Morphometric studies will be performed on lung, cardiac and muscular tissue. Vascular endothelial function and autophagy will be also measured. The differences in these variables between the different experimental groups will be analysed.

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: NCT05522374 Recruiting - Clinical trials for Neurodegeneration With Brain Iron Accumulation (NBIA)

TIRCON International NBIA Registry

TIRCON
Start date: June 14, 2012
Phase:
Study type: Observational [Patient Registry]

TIRCON-reg aims to - continue the provision of a global registry and natural history study for NBIA disorders - harmonize and cover existing national and single site registries - enable participation of countries and single sites that so far have no access to an NBIA registry - join forces in order to recruit sufficient numbers of patients - define the natural history of NBIA disorders - define the most appropriate outcome measures - inform the design and facilitate the conduction of clinical trials

NCT ID: NCT05521672 Recruiting - Crohn Disease Clinical Trials

Clinical Trial to Evaluate if the Laparoscopic Administration of Cells Derived From the Fatty Tissue Can Improve the Inflammatory Stenosis in Patient With Crohn's Disease

MEIC
Start date: October 24, 2022
Phase: Phase 1/Phase 2
Study type: Interventional

A phase II, national, multicenter, uncontrolled and open trial to evaluate the feasibility and safety of laparoscopic administration of adipose derived allogenic mesenchymals stem cells (adMSC), for the treatment of patients with a single inflammatory stenosis in the context of Crohn's disease.

NCT ID: NCT05520450 Recruiting - Loneliness Clinical Trials

Telematic Support Group in Patients With Feelings of Loneliness and Social Isolation

Start date: September 1, 2022
Phase:
Study type: Observational

Loneliness can have negative consequences, both physically and psychologically. The aim of this study is to evaluate the effects of a telematic group intervention in reducing feelings of loneliness and social isolation, as well as improving the emotional well-being of people in home care. The sample is made up of patients aged 60 to 99 years resident in Sabadell health area who receive regular home care in the ATDOM Programme by the Primary Care Team of the Corporació Sanitària Parc Taulí, who agree to participate in the study and meet the eligibility criteria. The study includes clinical assessments, emotional well-being, social support and satisfaction with the intervention.