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Long-term Follow-up After Adoptive Transfer of Genetically Modified Cell Products
Human gene therapy products are designed to achieve therapeutic effect through genetic modifications of human cells using retroviral or lentiviral vectors, res…
Clinical Genetics Branch (CGB) Eligibility Screening Survey
Background: Clinical Genetics Branch (CGB) researchers study individuals and populations at high genetic risk of cancer in order to improve our understanding …
The Natural History of GATA2 Deficiency and Related Disorders
Background: \- GATA2 deficiency is a genetic disorder that can cause problems with a person s immune system and other body systems. Some people who have this …
A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of KAI-9531 Administered Once Weekly in Participants Living With Obesity or Overweight and Diabetes
The primary objective of this study is to demonstrate that KAI-9531 subcutaneous (SC) injection once weekly is superior to placebo on: * Percent change in bod…
A Phase 2 Randomized, Placebo-Controlled Clinical Trial to Assess the Safety and Efficacy of Donanemab in Participants With Early Cognitive Decline, at Least One Core Clinical Feature of Dementia With Lewy Bodies, and Confirmation of Alpha-Synuclein and Amyloid Co-pathology
The main purpose of this study is to evaluate whether treatment with donanemab slows the progression of cognitive (how we think, learn, remember, pay attention…
Randomized Phase III Trial of Rapid-Plan Knowledge-Based Planning vs. Human-Driven Planning for Prostate Cancer Radiotherapy
This phase III trial compares the effects of radiation therapy using RapidPlan, trademark, knowledge-based planning to human-driven planning in treating patien…
A Phase 3 Randomized, Double-blind, Placebo-controlled Study to Evaluate the Impact of Maridebart Cafraglutide on Cardiovascular Outcomes in Participants With Atherosclerotic Cardiovascular Disease and Overweight or Obesity
The primary objective of this trial is to demonstrate that maridebart cafraglutide is superior to placebo when given as an adjunct to standard of care with res…
A Randomized Study Evaluating 2 Dose Regimens of Dupilumab in Children ≥6 Months of Age Weighing ≥5 to <15 kg With Active Eosinophilic Esophagitis
This study is researching an experimental drug called dupilumab (called "study drug"). The study is focused on children with active eosinophilic esophagitis (E…
A Phase 3b Open-Label Study of Long-Term Neurocognitive Outcomes in Children With Phenylketonuria Treated With Sepiapterin
The main purpose of this trial is to evaluate the long-term efficacy of sepiapterin on preserving neurocognitive functioning in children with PKU when treatmen…
Integrated Therapies for Alcohol Use in Alcohol-associated Liver Disease (ITAALD) Trial
This is a multicenter, randomized, double-blinded, placebo-controlled trial focused on the treatment of severe alcohol-associated hepatitis (sAH) and alcohol u…
Ketoconazole Effects on Cortisol Circadian Rhythm in Mild Autonomous Cortisol Secretion: A Pilot Study
Background: Cortisol is a hormone in the blood. Cortisol levels normally go down at night and up in the morning. Mild autonomous cortisol secretion (MACS) is …
A Randomized, Triple-blind, Placebo-controlled, Phase 3 Trial to Evaluate the Efficacy, Safety, and Tolerability of Intravenous AMBTX-01 (Neridronate) for Treatment of Complex Regional Pain Syndrome Type 1 (CRPS-RISE)
Phase 3 Trial of AMBTX-01 (neridronate) for Complex Regional Pain Syndrome Type 1