Estab Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1)
NCT03981575
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
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Observational Study Protocol: LIVER-R
NCT06252753
Recruiting
Given the number of anticipated durvalumab-based treatment launches in the hepatobiliary cancer space over the next 3 years, there is a need to capture contemporary real-world data across these indications. LIVER-R is a multicountry, multicenter, observational study of patients with a confirmed diagnosis of hepatobiliary cancer treated with a durvalumab-based regimen as part of routine clinical practice or early access program (EAP). The study design will include primary and secondary data colle... Read More
Gender:
ALL
Ages:
Between 18 years and 130 years
Trial Updated:
06/04/2025
Locations: Research Site, Birmingham, Alabama +112 locations
Conditions: Hepatobiliary Cancers
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Evaluating Buprenorphine/Naloxone Microdosing vs. Standard Dosing in Emergency Departments
NCT04893525
Recruiting
This is a multi-centre, open-label RCT at four Emergency Departments (EDs) in British Columbia and Alberta. The purpose of the current study is to compare the effectiveness of buprenorphine/naloxone microdosing and standard dosing take-home induction regimens at enabling patients to successfully complete the induction regimen, and at retaining patients on opioid agonist therapy. We will randomize our enrolled patients to receive take-home microdosing or standard dosing packages of buprenorphine... Read More
Gender:
ALL
Ages:
18 years and above
Trial Updated:
06/04/2025
Locations: Foothills Medical Centre, Calgary, Alberta +5 locations
Conditions: Opioid-use Disorder
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Fluids in Septic Shock (FISSH)
NCT03677102
Recruiting
Despite evidence of the physiologic benefits and possible lower mortality associated with low chloride solutions, normal saline remains the most wildly used fluid in the world. Given uncertainty about the impact of lower chloride versus higher chloride solutions on mortality, it is unlikely that clinical practice will change without new and direct randomized controlled trial (RCT) evidence. Editorials published in leading critical care journals have called for RCT's to address this important cli... Read More
Gender:
ALL
Ages:
16 years and above
Trial Updated:
06/03/2025
Locations: University of Calgary - Foothills Medical Centre, Calgary, Alberta +28 locations
Conditions: Sepsis, Septic Shock
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Transcatheter Mitral Valve Repair for Inotrope Dependent Cardiogenic Shock
NCT05298124
Recruiting
Mitral regurgitation may be seen in the setting of cardiogenic shock. Transcatheter edge-to-edge repair (TEER) has been shown to improve outcomes in patients with chronic heart failure. Observational studies suggest improvements in clinical outcomes in patients with mitral regurgitation in the setting of cardiogenic shock; however, there remains a lack of randomized clinical data to support the use of TEER in cardiogenic shock. This study will be a multicenter, open-label, randomized-controlled... Read More
Gender:
ALL
Ages:
18 years and above
Trial Updated:
06/03/2025
Locations: Mayo Clinic, Rochester, Minnesota +3 locations
Conditions: Cardiogenic Shock, Mitral Regurgitation
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Gene Therapy Trial for Otoferlin Gene-mediated Hearing Loss
NCT05821959
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
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MED-EL Remote Care Multi-Center Feasibility Study
NCT05973669
Recruiting
MED-EL Remote Care is a way for MED-EL cochlear implant users to check their hearing and cochlear implant device from any location, without the need for a scheduled, in-person appointment with their audiologist. This study will assess the effectiveness, efficiencies, and useability of MED-EL Remote Care.
Gender:
ALL
Ages:
All
Trial Updated:
06/03/2025
Locations: University of Iowa Hospitals and Clinics, Iowa City, Iowa +4 locations
Conditions: Hearing Loss, Sensorineural, Cochlear Implant, Cochlear Implant Recipients, Hearing Disorders, Cochlear Implants
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A Phase 2b Study of Zagociguat in Patients With MELAS
NCT06402123
Recruiting
PRIZM is a Phase 2b randomized, double-blind, placebo-controlled, 3-treatment, 2-period, crossover study evaluating the efficacy and safety of oral zagociguat 15 and 30 mg vs. placebo when administered daily for 12 weeks in participants with genetically and phenotypically defined MELAS.
Gender:
ALL
Ages:
Between 18 years and 75 years
Trial Updated:
06/03/2025
Locations: UC San Diego - Altman Clinical and Translational Research Institute, La Jolla, California +24 locations
Conditions: Mitochondrial Encephalopathy, Lactic Acidosis and Stroke-Like Episodes (MELAS Syndrome)
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A Study to Evaluate How Safe Pozelimab + Cemdisiran Combination Therapy is and How Well it Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Not Recently Received or Have Not Received Complement Inhibitor Treatment
NCT05133531
Recruiting
This study is researching a clinical treatment combination with two experimental drugs called pozelimab and cemdisiran. The study is focused on people with paroxysmal nocturnal hemoglobinuria (PNH). The aim of the study is to see how safe and effective the pozelimab + cemdisiran combination is for people with PNH and how the combination compares with 2 existing treatments: ravulizumab and eculizumab. The pozelimab + cemdisiran combination may be referred to as "study drugs". Ravulizumab and ecu... Read More
Gender:
ALL
Ages:
18 years and above
Trial Updated:
06/03/2025
Locations: The Oncology Institute of Hope & Innovation, Whittier, California +62 locations
Conditions: Paroxysmal Nocturnal Hemoglobinuria
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Inspiratory Work of Breathing Before and After Extubation
NCT07017608
Recruiting
Critically ill patients who (1) are not able to maintain their airway, (2) cannot breathe on their own, or (3) both, are ones who often require tracheal intubation and support from a breathing machine (mechanical ventilator). When the patient is ready to be liberated from the mechanical ventilator because the initial insult for intubation has been resolved, the patient is screened using the readiness to wean test in preparation for extubation. As the patient passes this screening, a spontaneous... Read More
Gender:
ALL
Ages:
18 years and above
Trial Updated:
06/03/2025
Locations: Toronto General Hospital, University Health Network, Toronto, Ontario
Conditions: Lung Transplant; Complications, Ventilator-Induced Lung Injury, Ventilator Associated Pneumonia
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Cancer Exercise: Evaluation of a Mobile App in Breast Cancer Survivors
NCT06791018
Recruiting
This study will examine whether a mobile app increases exercise levels in breast cancer survivors after treatments, reduces fatigue, and improves physical functions. This experimental study has 200 participants placed into one of two groups. One group will be asked to use the cancer-specific exercise app over 12 weeks, and the other group will receive the cancer exercise guidelines. Exercise will be measured by self-report questionnaires before and after the intervention and three months later.... Read More
Gender:
ALL
Ages:
18 years and above
Trial Updated:
06/03/2025
Locations: Exercise Oncology Research Laboratory, Faculty of Kinesiology, Sport and Recreation, University of Alberta., Edmonton, Alberta
Conditions: Breast Cancer, Breast Cancer Early Stage Breast Cancer (Stage 1-3)
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A Study to Investigate the Safety, Tolerability, Pharmacokinetics (PK), and Preliminary Anticancer Activity of GSK4524101 Alone or With Niraparib in Participants With Solid Tumors
NCT06077877
Recruiting
The primary purpose of this study is to determine the maximum tolerated dose of GSK4524101 monotherapy (MTD) and GSK4524101 in combination with niraparib (MTDc). The study consists of two parts - Part 1 (Dose Escalation) and Part 2 (Dose Expansion).
Gender:
ALL
Ages:
18 years and above
Trial Updated:
06/03/2025
Locations: GSK Investigational Site, San Francisco, California +8 locations
Conditions: Neoplasms
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