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NCT ID: NCT05651113 Recruiting - Clinical trials for Severe Combined Immunodeficiency

The Experience of Screening for SCID

Start date: November 9, 2022
Phase:
Study type: Observational

This project will evaluate the impact of including Severe Combined Immunodeficiency into the newborn bloodspot screening panel. It will recruit parents and health professionals primarily from the sites where this new form of screening is being trialled well as additional sites where clinicians will be involved in the care of these babies and comparator groups are needed. The proposed work will consist of two work packages. The first, a mixed-methods study conducted with families from the point of screening information being returned through to the child's fifth birthday. The second, a qualitative interview study conducted with health professionals during the clinical evaluation phase of the national pilot programme.

NCT ID: NCT05650632 Recruiting - Multiple Myeloma Clinical Trials

A Study to Assess Adverse Events of Intravenously (IV) Infused ABBV-383 in Adult Participants With Relapsed or Refractory Multiple Myeloma

Start date: March 21, 2023
Phase: Phase 1
Study type: Interventional

Multiple Myeloma (MM) is a cancer of the blood's plasma cells ( blood cell). The cancer is typically found in the bones and bone marrow (the spongy tissue inside of the bones) and can cause bone pain, fractures, infections, weaker bones, and kidney failure. Treatments are available, but MM can come back (relapsed) or may not get better (refractory) with treatment. This is a study to determine adverse events and change in disease symptoms of ABBV-383 in adult participants with relapsed/refractory (R/R) MM. ABBV-383 is an investigational drug being developed for the treatment of R/R Multiple Myeloma (MM). This study is broken into 2 Arms; Arm A (Parts 1 and 2) and Arm B. Arm A includes 2 parts: step-up dose optimization (Part 1) and dose expansion (Part 2). In Part 1, different level of step-up doses are tested followed by the target dose of ABBV-383. In Part 2, the step-up dose identified in Part 1 (Dose A) will be used followed by the target dose A of ABBV-383. In Arm B a flat dose of ABBV-383 will be tested. Around 120 adult participants with relapsed/refractory multiple myeloma will be enrolled at approximately 30 sites across the world. Participants will receive ABBV-383 as an infusion into the vein in 28 day cycles for approximately 3 years. There may be higher treatment burden for participants in this trial compared to their standard of care. Participants will attend regular visits during the study at a hospital or clinic. The effect of the treatment will be checked by medical assessments, blood tests, checking for side effects and questionnaires.

NCT ID: NCT05650567 Recruiting - Dermatomyositis Clinical Trials

Study of M5049 in DM and PM Participants (NEPTUNIA)

Start date: January 19, 2023
Phase: Phase 2
Study type: Interventional

The purpose of this study is to evaluate the efficacy and safety of orally administered M5049 in idiopathic inflammatory myopathies, specifically dermatomyositis (DM) and polymyositis (PM) participants for 24 weeks.

NCT ID: NCT05650177 Recruiting - Clinical trials for Persistent Depressive Disorder

Effects of Mindfulness on Brain Functioning in Depressed Patients

Start date: June 6, 2022
Phase: N/A
Study type: Interventional

Depression is a common disorder that often takes a recurrent or chronic course. Recent research has indicated that such courses are associated with significant changes in brain functioning including changes in strength of functional connectivity between regions of the default mode network, a large-scale brain network involved in spontaneous thinking and rumination, and the ability to suppress this network. Training in mindfulness has been found to be an effective treatment for patients with recurrent and chronic courses of depression and there is evidence for beneficial effects on brain structure and functioning. However, it remains unclear whether and in how far the training may serve to reverse brain changes in recurrent and chronic courses of depression. The aim of this research is to test the effects of sustained training in mindfulness meditation on default mode network connectivity and suppression in patients suffering from recurrent or chronic depression. The investigators will invite currently depressed patients with a recurrent or chronic course of the disorder (N = 36) to take part in a 6-month mindfulness intervention and investigate changes in default-mode network connectivity and suppression over repeated assessments. Building on our previous research, the intervention will use a blended format that combines an online app offering psychoeducational materials and meditation guidance with brief individual therapy sessions delivered via videoconference. Eligible participants will be asked to complete questionnaires and take part in brain scans before the start of the treatment, 3 months after the start of the treatment and after the end of treatment.

NCT ID: NCT05650125 Recruiting - Cancer Clinical Trials

A Platform Study for solId orgaN CancERs

PINCER
Start date: January 1, 2022
Phase:
Study type: Observational

Generating bespoke biosampling protocols for individual tumour specific translational projects is onerous, cumbersome and inefficient. This study aims to provide a broad platform to maximise the unique access to biopsy and resected tumour specimens available from cancer patients to provide a high-quality and efficient source of biosamples for specific translational projects. Access to historical FFPE samples and clinical data, as well as prospective fresh tissue samples, will allow interrogation of the underlying biology of these cancers. Matched pseudoanonymised clinical and radiological data will allow the development of rich high-yield datasets.

NCT ID: NCT05649800 Recruiting - Stroke Clinical Trials

Vascular mEchanisms in, Stroke, dePression, dementiA, and deliRum: The VESPAR Project

VESPAR
Start date: March 17, 2023
Phase:
Study type: Observational

The goal of this observational study is to determine the feasibility of using integrated Transcranial Doppler Ultrasonography or Near Infrared Spectroscopy to detect changes in cerebral autoregulation and neurovascular coupling in healthy, stroke, dementia, depression and delirium populations. We also aim to: - Determine the optimal stimulus for neurovascular coupling - To derive sample size estimates for a future study - To develop a multilevel, multivariate model that can be applied to future datasets

NCT ID: NCT05649748 Recruiting - Clinical trials for Pulmonary Arterial Hypertension

An Extension Study of Treprostinil Palmitil Inhalation Powder (TPIP) for Pulmonary Arterial Hypertension (PAH)

Start date: March 7, 2023
Phase: Phase 2/Phase 3
Study type: Interventional

The primary purpose of the study is to evaluate the safety and tolerability of the long-term use of TPIP in participants with PAH from studies INS1009-201 (NCT04791514), INS1009-202 (NCT05147805) and other lead-in studies of TPIP in participants with PAH.

NCT ID: NCT05649722 Recruiting - Clinical trials for Pulmonary Hypertension

An Extension Study of Treprostinil Palmitil Inhalation Powder (TPIP) for Pulmonary Hypertension Associated With Interstitial Lung Disease (PH-ILD)

Start date: May 11, 2023
Phase: Phase 2/Phase 3
Study type: Interventional

The primary objective of this study is to evaluate the safety and tolerability of the long-term use of TPIP in participants with PH-ILD from Study INS1009-211 (NCT05176951) and other lead-in studies of TPIP in participants with PH-ILD.

NCT ID: NCT05649384 Recruiting - Clinical trials for NSTEMI - Non-ST Segment Elevation MI

Emergency Department Triage of Patients With Acute Chest Pain Based on the ESC 0/1-hour Algorithm (PRESC1SE-MI)

PRESC1SE-MI
Start date: December 1, 2020
Phase:
Study type: Observational

The PRESC1SE-MI study compares two algorithms for triage of patients presenting with chest pain and symptoms of heart attack (myocardial infarction) to the emergency department. Both algorithms are recommended by the European Society of Cardiology: the 0/3-hour algorithm and the 0/1-hour algorithm. Currently, most emergency departments worldwide use the 0/3-hour troponin algorithm. Cardiac troponin (cTn) is a heart-specific biomarker which indicates damage of the heart muscle and which increases after a heart attack. In the 0/3-hour algorithm, the amount of troponin in the bloodstream is measured with a high-sensitivity assay at admission and 3 hours thereafter. Likewise, the 0/1-hour algorithm means that the blood sample in which the troponin is measured is collected at admission and 1 hour later. Since recent clinical studies suggest that the 0/1-hour algorithm is superior to the 0/3-hour algorithm, many hospitals consider switching to the 0/1-hour algorithm. The aim of this study is to assess how feasible the time-saving 0/1-hour algorithm would be in reality and whether it provides the same accuracy and safety in the diagnosis of myocardial infarction as the current practice the 0/3-hour algorithm.

NCT ID: NCT05648968 Recruiting - Clinical trials for Warm Autoimmune Hemolytic Anemia (wAIHA)

A Study of Efficacy and Safety of Ianalumab in Previously Treated Patients With Warm Autoimmune Hemolytic Anemia

VAYHIA
Start date: December 30, 2022
Phase: Phase 3
Study type: Interventional

The purpose of this study is to evaluate efficacy and safety of ianalumab compared to placebo in patients with warm autoimmune hemolytic anemia, who failed at least one line of treatment.