A Global Prospective Observational Registry of Patients With Pompe Disease
NCT06121011
Recruiting
This is a global, multicenter, prospective, observational registry of patients with Pompe disease, including those with late-onset pompe disease (LOPD) and infantile-onset pompe disease (IOPD). Both untreated patients and those being treated with an approved therapy for Pompe disease are eligible to participate. The objectives of the registry are: * To evaluate the long-term safety of Pompe disease treatments through collection of data that describe the frequency of adverse events (AEs)/seriou... Read More
Gender:
ALL
Ages:
All
Trial Updated:
08/11/2025
Locations: University of Arkansas Medical Science, Little Rock, Arkansas +39 locations
Conditions: Pompe Disease
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Vercise™ DBS Dystonia Prospective Study
NCT02686125
Recruiting
To compile characteristics of real-world outcomes of Boston Scientific Corporation's commercially approved VerciseTM Deep Brain Stimulation (DBS) Systems for the treatment of dystonia.
Gender:
ALL
Ages:
7 years and above
Trial Updated:
08/11/2025
Locations: AZ Sint-Lucas, Ghent, Not set +35 locations
Conditions: Dystonia
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RELIEF - A Global Prospective Observational Post-Market Study to Evaluate Long-Term Effectiveness of Neurostimulation Therapy for Pain
NCT01719055
Recruiting
To compile characteristics of real-world clinical outcomes for Boston Scientific commercially approved neurostimulation systems for pain in routine clinical practice, when used according to the applicable Directions for Use - and - To evaluate the economic value and technical performance of Boston Scientific commercially approved neurostimulation systems for pain in routine clinical practice
Gender:
ALL
Ages:
18 years and above
Trial Updated:
08/11/2025
Locations: Physicians Research Group, LLC, Phoenix, Arizona +111 locations
Conditions: Pain
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Registry of Deep Brain Stimulation With the VERCISE™ System: Vercise DBS Registry
NCT02071134
Recruiting
The purpose of this registry is to compile characteristics of world-wide outcomes for the use of Boston Scientific's commercially available Vercise DBS System in the treatment of Parkinson's disease. The utilization of Image Guided Programming (IGP), and other commercially available programming features, used as planning tools for the programming of patients with Boston Scientific's Vercise DBS System are also evaluated. Additionally, the utilization of the DBS Illumina 3D feature that may be... Read More
Gender:
ALL
Ages:
18 years and above
Trial Updated:
08/11/2025
Locations: Neurology and Pain Specialty, Aliso Viejo, California +81 locations
Conditions: Parkinson's Disease
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A Study to Evaluate the Effect of Aficamten in Pediatric Patients With Symptomatic Obstructive Hypertrophic Cardiomyopathy (oHCM).
NCT06412666
Recruiting
The purpose of this study is to evaluate the efficacy, safety and PK of aficamten in a pediatric population with symptomatic obstructive hypertrophic cardiomyopathy (oHCM).
Gender:
ALL
Ages:
Between 12 years and 17 years
Trial Updated:
08/11/2025
Locations: Children's Hospital Los Angeles, Los Angeles, California +30 locations
Conditions: Pediatric, Symptomatic Obstructive Hypertrophic Cardiomyopathy
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A Study to Assess the Effectiveness and Safety of Pacritinib in Patients With VEXAS Syndrome (PAXIS)
NCT06782373
Recruiting
This study is to assess the effectiveness and safety of pacritinib in patients with VEXAS (i.e., Vacuoles in myeloid progenitors, E1 ubiquitin-activating enzyme, X-linked, autoinflammatory manifestations, and somatic) syndrome. 78 patients will be enrolled, randomized to either pacritinib dose A, pacritinib dose B + placebo, or placebo. Randomization will be stratified by prescribed GC dose on the day of randomization.
Gender:
ALL
Ages:
18 years and above
Trial Updated:
08/11/2025
Locations: Mayo Clinic - Scottsdale, Scottsdale, Arizona +39 locations
Conditions: VEXAS, VEXAS Syndrome
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Phase III Study Investigating Heart Failure and Cardiovascular Death With Baxdrostat in Combination With Dapagliflozin
NCT06677060
Recruiting
Participants include men and women ≥ 40 years of age with T2DM, established CV disease, a history of HTN with an SBP of at least 130 mmHg at screening, who meet the predefined serum potassium level, and with at least one additional risk factor for HF. The study will include an optional pre-screening period to facilitate sites' identification of potentially eligible participants to enter the full screening assessments. Participants will not be required to visit the site and no informed consent i... Read More
Gender:
ALL
Ages:
40 years and above
Trial Updated:
08/11/2025
Locations: Research Site, Birmingham, Alabama +862 locations
Conditions: Heart Failure
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Patient Retrospective Outcomes (PRO)
NCT01550575
Recruiting
This study will evaluate de-identified (anonymous) data in subject medical charts to review the clinical outcomes of various treatment approaches in the treatment of chronic pain.
Gender:
ALL
Ages:
18 years and above
Trial Updated:
08/11/2025
Locations: Lakeside Spine & Pain, Lake Havasu City, Arizona +141 locations
Conditions: Chronic Pain
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Study to evaLuate the effIcacy and Safety of abeLacimab in High-risk Patients With Atrial Fibrillation Who Have Been Deemed Unsuitable for Oral antiCoagulation (LILAC-TIMI 76)
NCT05712200
Recruiting
A study to evaluate the effect of abelacimab relative to placebo on the rate of ischemic stroke or systemic embolism (SE) in patients with Atrial Fibrillation (AF) who have been deemed by their responsible physicians or by their own decision to be unsuitable for oral anticoagulation therapy.
Gender:
ALL
Ages:
65 years and above
Trial Updated:
08/11/2025
Locations: Anthos Investigative Site 1040, Birmingham, Alabama +564 locations
Conditions: Atrial Fibrillation (AF)
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Open-Label Extension Study to Assess the Long-Term Safety and Tolerability of KarXT in Subjects With Psychosis Associated With Alzheimer's Disease (ADEPT-3)
NCT05980949
Recruiting
This is a Phase 3 global, multicenter, 52-week, open-label extension (OLE) rollover study for subjects completing study CN012-0026, CN012-0027 or CN012-0056. Subjects (randomized or non-randomized) who complete the 38-week CN012-0026 study, 14-week CN012-0027 study or 14-week CN012-0056 study will be eligible to enroll in CN012-0028. The primary objective of the study is to assess the long-term safety and tolerability of KarXT in subjects with psychosis associated with Alzheimer's Disease.
Gender:
ALL
Ages:
Between 55 years and 90 years
Trial Updated:
08/11/2025
Locations: Local Institution - 1029, Homewood, Alabama +382 locations
Conditions: Psychosis Associated With Alzheimer's Disease
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A Clinical Trial Investigating the Safety, Tolerability, and Therapeutic Effects of BNT113 in Combination With Pembrolizumab Versus Pembrolizumab Alone for Patients With a Form of Head and Neck Cancer Positive for Human Papilloma Virus 16 and Expressing the Protein PD-L1
NCT04534205
Recruiting
An open-label, controlled, multi-site, interventional, 2-arm, Phase II/III trial of BNT113 in combination with pembrolizumab vs pembrolizumab monotherapy as first line treatment in patients with unresectable recurrent or metastatic HPV16+ HNSCC expressing programmed cell death ligand-1 (PD-L1) with combined positive score (CPS) ≥1. This trial has two parts. Part A, is an initial non-randomized Safety Run-In Phase to confirm the safety and tolerability at the selected dose range level of BNT113... Read More
Gender:
ALL
Ages:
18 years and above
Trial Updated:
08/11/2025
Locations: California Research Institute, Los Angeles, California +186 locations
Conditions: Unresectable Head and Neck Squamous Cell Carcinoma, Metastatic Head and Neck Cancer, Recurrent Head and Neck Cancer
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LEGEND Study: EG-70 in NMIBC Patients BCG-Unresponsive and High-Risk NMIBC Incompletely Treated With BCG or BCG-Naïve
NCT04752722
Recruiting
This study will evaluate the safety and efficacy of intravesical administration of EG-70 in the bladder and its effect on bladder tumors in patients with NMIBC. This study study consists of two phases; a Phase 1 dose-escalation to establish safety and recommended the phase 2 dose, followed by a Phase 2 study to establish how effective the treatment is. The Study will include patients with NMIBC with Cis for whom BCG therapy is unresponsive and patients with NMIBC with Cis who are BCG-naïve or... Read More
Gender:
ALL
Ages:
18 years and above
Trial Updated:
08/11/2025
Locations: The University of Alabama at Birmingham Clinical Research Unit (CRU), Birmingham, Alabama +98 locations
Conditions: Superficial Bladder Cancer, Non-muscle Invasive Bladder Cancer With Carcinoma in Situ
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