Estab Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1)
Recruiting
Building on previous work of the Myotonic Dystrophy Clinical Research Network (DMCRN), the present study seeks to overcome insufficient data on natural history; lack of reliable biomarkers; and incomplete characterization and limited biological understanding of the phenotypic heterogeneity of Myotonic Dystrophy 1 by examining strategies to improve the reliability by making further refinements in our sample collection and analysis procedures by developing strategies for managing patient heterogen... Read More
Gender:
ALL
Ages:
Between 18 years and 70 years
Trial Updated:
06/04/2025
Locations: University of California, San Diego, La Jolla, California +16 locations
Conditions: Myotonic Dystrophy 1, DM1
BiomeBakery Project
Recruiting
This study aims to investigate the chronic effects of prebiotic fibre-fortified functional bakery product on cognitive, affective, inflammatory, metabolic, and anthropometric outcomes in healthy adults aged 60-75 years with mild to moderate subjective cognitive complaints.
Gender:
ALL
Ages:
Between 60 years and 75 years
Trial Updated:
06/04/2025
Locations: University of Reading, School of Psychology and Clinical Languages, Reading, Berkshire
Conditions: Cognition, Affect (Mental Function), C Reactive Protein
PRECOG Pilot Project
Recruiting
This pilot study aims to investigate the chronic effects of a prebiotic blend on cognitive, affective and gut microbiome outcomes in healthy adults aged 60-75 years.
Gender:
ALL
Ages:
Between 60 years and 75 years
Trial Updated:
06/04/2025
Locations: University of Reading, School of Psychology and Clinical Languages, Reading, Berkshire
Conditions: Cognition, Affect (Mental Function), Gut Microbiome
Fibre and Mental Wellbeing Feasibility Trial
Recruiting
This feasibility study will explore whether adding an extra 10 grams of fibre to participants' daily diet for two weeks would improve their mental health and wellbeing.
Gender:
ALL
Ages:
Between 18 years and 40 years
Trial Updated:
06/04/2025
Locations: University of Reading, School of Psychology and Clinical Languages, Reading, Berkshire
Conditions: Affect (Mental Function), Sleep, Anxiety, Stress
Otoferlin Gene-mediated Hearing Loss Natural History Study
Recruiting
This is a retrospective and prospective longitudinal study in participants with Otoferlin Gene-Mediated Hearing Loss.
Gender:
ALL
Ages:
44 years and below
Trial Updated:
06/04/2025
Locations: University of Iowa, Iowa City, Iowa +9 locations
Conditions: Sensorineural Hearing Loss, Bilateral
Experiences of Managing Sleep Disordered Breathing in Children With Cerebral Palsy.
Recruiting
Cerebral palsy (CP) refers to a non-progressive movement disorder, which occurs due to damage to the developing brain around the time of birth. Symptoms of sleep disordered breathing (SDB) include noisy breathing during sleep, increased day-time sleepiness and reduced energy levels. In the long term, SDB might have an effect on the brain and learning, as well as putting strain on the heart. Children with CP have a higher risk of sleep breathing problems compared to typically-developing children... Read More
Gender:
ALL
Ages:
Between 1 month and 16 years
Trial Updated:
06/04/2025
Locations: NHS Lothian, Edinburgh, Edinbrugh +1 locations
Conditions: Sleep Disordered Breathing (SDB), Cerebral Palsy (CP), Respiratory Support
A Study to Investigate the Safety and Efficacy of Belantamab for the Treatment of Multiple Myeloma When Used as Monotherapy and in Combination Treatments
Recruiting
The study consists of two parts: Part 1 The primary purpose of this part aims to evaluate the safety, tolerability, and clinical activity of escalating doses of single agent belantamab in participants with refractory multiple myeloma (RRMM) who have received at least 3 prior therapies (4L+). Part 2 The primary purpose of this part is to evaluate the safety, tolerability, and clinical activity of different dose ratios of belantamab mafodotin in combination with belantamab (delivered as separate d... Read More
Gender:
ALL
Ages:
18 years and above
Trial Updated:
06/04/2025
Locations: GSK Investigational Site, Grand Rapids, Michigan +20 locations
Conditions: Multiple Myeloma
A Study to Evaluate the Efficacy and Safety of Pegozafermin in Participants With Compensated Cirrhosis Due to MASH
Recruiting
The study will assess the efficacy and safety of pegozafermin administered in participants with compensated cirrhosis due to MASH (biopsy-confirmed fibrosis stage F4 MASH \[previously known as nonalcoholic steatohepatitis, NASH\]).
Gender:
ALL
Ages:
Between 18 years and 75 years
Trial Updated:
06/04/2025
Locations: 89bio Clinical Study Site, Homewood, Alabama +249 locations
Conditions: Metabolic Dysfunction-Associated Steatohepatitis (MASH) / Nonalcoholic Steatohepatitis (NASH) With Compensated Cirrhosis
A Study Evaluating the Safety and Tolerability of QRL-201 in ALS
Recruiting
The primary objective of this study is to determine the safety and tolerability of multiple doses of QRL-201 in people living with ALS
Gender:
ALL
Ages:
Between 18 years and 80 years
Trial Updated:
06/04/2025
Locations: Universitaire Ziekenhuizen Leuven (UZ Leuven), Leuven, Not set +14 locations
Conditions: Amyotrophic Lateral Sclerosis
Dual Endothelin Receptor Antagonism in Large Vessel Vasculitis (DERAIL-LVV)
Recruiting
Large vessel vasculitis is a disease that causes damage to blood vessels. This damage to blood vessels can increase the risk of patients with LVV developing cardiovascular disease, including heart attacks and strokes. A chemical produced in the body called endothelin may contribute to this increase in cardiovascular disease risk by causing the vessels to stiffen and blood pressure to increase. It has previously been shown that by blocking the effects of endothelin, vessel stiffness and blood pr... Read More
Gender:
ALL
Ages:
18 years and above
Trial Updated:
06/04/2025
Locations: University of Edinburgh, Edinburgh, Not set
Conditions: Large Vessel Vasculitis, Giant Cell Arteritis (GCA), Takayasu Arteritis
Fatigue in Lupus Intervention Programmes (FLIP)
Recruiting
Systemic Lupus Erythematosus (SLE) is an autoimmune rheumatic disease. Patients report that fatigue has a significant impact on their quality of life but is often not discussed in healthcare settings. Fatigue is more prevalent in SLE than other Rheumatic diseases. Management across the NHS is very variable ranging from a booklet to one to one appointments or, less often, a group intervention. Previous studies in other Rheumatic diseases have shown that a group cognitive behavioural approach can... Read More
Gender:
ALL
Ages:
Between 18 years and 85 years
Trial Updated:
06/04/2025
Locations: NHS Lothian, Edinburgh, Not set
Conditions: Systemic Lupus Erythematosus
Gene Therapy Trial for Otoferlin Gene-mediated Hearing Loss
Recruiting
Part A of this trial will evaluate the safety and tolerability of a single unilateral administration of one of two dose levels of AAVAnc80-hOTOF and will evaluate the Akouos delivery device to safely achieve the intended product performance.
Gender:
ALL
Ages:
All
Trial Updated:
06/03/2025
Locations: University of Iowa, Iowa City, Iowa +7 locations
Conditions: Sensorineural Hearing Loss, Bilateral