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An Observational Study of Individuals With Isolated Dystonia
This is an observational study in individuals with isolated (or primary) dystonia that involves more than one body region. The purpose of the study is to colle…
FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases
FLOWER is a completely virtual, nationwide, real-world observational study to collect, annotate, standardize, and report clinical data for rare diseases. Patie…
PRIMROSE: A Modular Phase I/IIa, Multi-centre, Dose Escalation, and Expansion Study of AZD3470, a MTA Cooperative PRMT5 Inhibitor, as Monotherapy and in Combination With Anticancer Agents in Patients With Advanced/Metastatic Solid Tumors That Are MTAP Deficient
This is a first time in human (FTiH) Phase I/IIa, open-label, multi-centre study of AZD3470 in participants with advanced or metastatic solid tumors with MTAP …
Rural Community Peer Partnerships for Improving Methamphetamine-Associated Heart Failure Screening and Engagement
The goal of PEER-Heart is to implement and assess feasibility and preliminary effectiveness of a community peer-facilitated screening for methamphetamine-assoc…
An Adaptive Design Basket Trial of Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe β-Hemoglobinopathies
A promising approach for the treatment of genetic diseases is called gene therapy. Gene therapy is a relatively new field of medicine that uses genetic materia…
New Approach to Objectively Measure Social Engagement in Older Adults With Hearing Loss
The goal of this clinical trial is to learn if hearing devices, like hearing aids, affect social and cognitive function of older adults. The main questions the…
A PHASE 2, TWO-PART, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, MULTICENTER STUDY TO EVALUATE THE EFFICACY, SAFETY, AND TOLERABILITY OF SUBCUTANEOUSLY ADMINISTERED REGN7999 (AN INHIBITOR OF TMPRSS6) IN PARTICIPANTS WITH IRON OVERLOAD DUE TO NON-TRANSFUSION DEPENDENT β-THALASSEMIA
This study is researching an experimental drug called REGN7999 (called "study drug"). The study is focused on patients with non-transfusion dependent beta-thal…
Implementing a Contingency Management Program Addressing Methamphetamine Use For and With the People of Hawaii
The goal of this clinical trial is to use contingency management (CM) as an intervention tool to address methamphetamine use. The main objectives are to: * G…
Assessment of the Ocular Microbiome in Health and Disease
The objective of this application is to illustrate the core constituents of the ocular surface microbiome, describe factors that promote colonization, and asse…
Advancing Troiluzole as a Treatment for Methamphetamine Use Disorder: A Human Laboratory Study
This will be a human laboratory study evaluating the influence of troriluzole treatment on the effects of methamphetamine. Supported by and included in the Hel…
2 x 2 Factorial, Double-blind, Randomized Trial of 'Set and Setting': a Translational Study in Healthy Volunteers
One hundred twenty healthy participants, ages 21 to 70, who experience moderate-to-lower-than-average mental well-being will be evenly randomized into four dif…
A Phase I Study to Evaluate the Safety and Tolerability of Escalating Doses of Fostamatinib in Subjects With Stable Sickle Cell Disease
Background: Sickle cell disease (SCD) is a genetic disease that causes the body to produce abnormal ( sickled ) red blood cells. SCD can cause anemia and life…