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Rett Syndrome Real World Data Observational Registry
The Rett Syndrome Registry is a longitudinal observational study of individuals with MECP2 mutations and a diagnosis of Rett syndrome. Designed together with t…
Phase II, Open-Label, Prospective Study of T Cell Receptor Alpha/Beta Depletion (A/B TCD) Peripheral Blood Stem Cell (PBSC) Transplantation for Children and Adults With Hematological Malignancies
This is a phase II, open-label, prospective study of T cell receptor alpha/beta depletion (TCR α/β TCD) peripheral blood stem cell (PBSC) transplantation for c…
Phase 1 Trial of Engineered HSV G207 in Children With Recurrent or Refractory Cerebellar Brain Tumors
This study is a clinical trial to determine the safety of inoculating G207 (an experimental virus therapy) into a recurrent or refractory cerebellar brain tumo…
Phase 1b/3 Global, Randomized, Controlled, Open-label Trial Comparing Treatment With RYZ101 to Standard of Care Therapy in Subjects With Inoperable, Advanced, SSTR+, Well-differentiated GEP-NETs That Have Progressed Following Prior 177Lu-SSA Therapy
This study aims to determine the safety, pharmacokinetics (PK) and recommended Phase 3 dose (RP3D) of RYZ101 in Part 1, and the safety, efficacy, and PK of RYZ…
Genetic and Environmental Contributions to Obesity Pathogenesis
The goal of this research is to investigate genetic and environmental factors that contribute to obesity through brain inflammation. The main questions are 1) …
Rifampin to Reduce Elevated Levels of Blood and Urine Calcium in Patients With Inactivating Mutations in the CYP24A1 Gene
This study evaluates the efficacy of rifampin in the treatment of hypercalcemia and/or hypercalciuria in participants with at least one inactivating mutation o…
Effect of Increasing Fluence on Efficacy of Low Level Laser Therapy for Androgenetic Alopecia, a Randomized Control Trial
Androgenetic alopecia (AGA) is a prevalent disease, occurring in 80% of Caucasian men and 50% of Caucasian women by age 701. Treatments for AGA are limited, an…
COMMENCER: COffee and Metabolites Modulating the Gut MicrobiomE in Colorectal caNCER
This is research study is assessing the effects of 6-g daily use of freeze-dried instant coffee on liver fat and fibrosis and the gut microbiome and metabolome…
Phase 1 First-in-Human, Dose Escalation and Dose Expansion Study of RYZ401, a Novel Radiopharmaceutical Therapy Labeled With Actinium-225, in Subjects With Neuroendocrine Tumors and Other Solid Tumors Expressing Somatostatin Receptors.
The primary objectives are to determine the recommended Phase 2 dose (RP2D) and optimal treatment regimen, characterize safety and tolerability, and evaluate p…
Application of a Systematic Developmental Assessment to a Novel Population: Infants With Rare Genetic Disorders
The main objective of this study is to apply a well-established model of developmental surveillance (which evolved to characterize the outcomes of very low bir…
A Prospective, Randomized Trial to Compare Telegenetics to Counseling Via a Novel Genetic Information Assistant in High-risk Cancer Patients.
The goal of this clinical trial is to learn about different ways cancer genetic screening can be provided to rural communities in participants at high risk for…
Phase 1, Double-Blinded, Placebo-Controlled, Single Ascending Dose (SAD) and Multiple Ascending Dose (MAD) Safety and Pharmacokinetics Trial of AVR-48
This is a Phase 1 (healthy adult volunteers), 2-part, double-blind, randomized, placebo controlled trial to evaluate the safety and pharmacokinetic (PK) profil…