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A Non-interventional, Epidemiologic Study of XLMTM and Clinical Expression in the Liver
XLMTM (X-linked myotubular myopathy) is a serious genetic muscle condition. It is caused by changes in the MTM1 gene which stops or slows down normal muscle de…
A Phase 2b, Randomized, Double-blind, Placebo-controlled, Parallel-group, Multicenter, Dose-ranging Study Investigating the Efficacy and Safety Profile of KT-621 Administered Orally to Adult Participants With Uncontrolled Moderate to Severe Eosinophilic Asthma
This Phase 2b study is designed to evaluate the safety and efficacy of KT-621 in participants with uncontrolled moderate to severe eosinophilic asthma. The ma…
A Modular Phase I/II Open-label, Multicenter Study to Evaluate the Safety, Pharmacokinetics, Pharmacodynamics, Immunogenicity, and Efficacy of AZD4512 Monotherapy or in Combination With Other Anticancer Agent(s), in Participants With Relapsed/Refractory B-cell Non-Hodgkin Lymphoma (B-NHL) (Lumi-NHL)
This is a Phase I/II open-label, global multicenter study to evaluate the safety and efficacy of AZD4512 monotherapy or in combination with other anticancer ag…
Phase 2 Platform Trial to Assess the Efficacy and Safety of Long-acting Antibodies as Single Agents and in Combinations for Moderately to Severely Active Ulcerative Colitis
This is a Phase 2, multicenter, proof-of-concept platform study in adult participants with moderately to severely active ulcerative colitis (UC). The primary g…
Anatomical and Functional Imaging Correlates of Chronic Pain in Cerebral Palsy
The investigators hope to use MRI biomarkers to identify and characterize sensorimotor network disruption patterns associated with chronic pain and sensory def…
Deep Phenotyping of Hearing Instability Disorders: Cohort Establishment, Biomarker Identification, Development of Novel Phenotyping Measures, and Discovery of Therapeutic Targets
Background: Disorders of hearing instability (HI) are poorly characterized and ineffectively treated. HI can cause fluctuations in hearing thresholds and spee…
Randomized Trial of ELEVATEd Cardiac Pacing Rate for Personalized Treatment of Heart Failure With Preserved Ejection Fraction (ELEVATE-HFpEF)
ELEVATE-HFpEF is a prospective, randomized, controlled, double-blinded, multi-center, global, interventional pivotal study evaluating the safety and efficacy o…
A Phase 2, Randomized, Double-blind, Sham Surgery-controlled Study of the Efficacy and Safety of Intraputaminal AAV2-GDNF in the Treatment of Adults With Moderate Stage Parkinson's Disease
The objective of this randomized, surgically controlled, double-blinded, Phase 2 study is to evaluate the safety and efficacy of AAV2-GDNF delivered to the put…
Electrographic Seizure Management and Neurobehavioral Outcomes in Critically Ill Children
Electrographic seizures are common in critically ill patients leading to increased use of resource-intense continuous EEG monitoring for seizure identification…
Effect of Meal Timing During Adjuvant Treatment for Cancer: A Randomized Clinical Trial
The goal of this clinical trial is to test meal-timing as a novel and sustainable interventional approach during cancer treatment to improve therapeutic respon…
The CryoICE™ Cryoanalgesia Registry for Pain Management in Post-cardiothoracic Surgery Via Cryoablation of the Intercostal Nerves
This is a retrospective and prospective, multicenter, observational patient registry to record outcomes from patients undergoing cryoablation of the intercosta…
Prospective Early Patient Experience Initiative (IMPACT-HTN)
An estimated 120 million people have high blood pressure (hypertension) in the US, of which approximately 10% have resistant hypertension. This study aims to e…