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A Phase 1, Open-label Study to Evaluate the Safety and to Characterize the Pharmacokinetics of a Fixed-Dose Combination Formulation of Ceftibuten-Ledaborbactam Etzadroxil
This is a Phase 1, open-label, two-part, study in approximately 46 healthy adult participants between 18 and 55 years of age (both inclusive) (at least 16 part…
Deep TMS Neuromodulation of Neural Circuits Associated With Stimulant Use Disorder
This study aims to evaluate the efficacy of deep transcranial magnetic stimulation (dTMS) as a treatment for Veterans with a methamphetamine use disorder (MUD).
Video Education With Result Dependent dIsclosure
The overall study objective of this trial study is to identify and evaluate strategies to improve the accessibility of the video education with result dependen…
ACT-GEN (Adherence And Care Tracking In GENetic Cancer Syndromes)
To help people with high-risk cancer variants to follow cancer surveillance guidelines and lower their risk of developing the disease.
A Multicenter, Open Label Study of QRX003 Lotion in Subjects With Netherton Syndrome
To assess the safety, tolerability, and efficacy of QRX003 lotion (4%) when added to standard of care treatment regimen, including systemic therapy in subjects…
Pharmacokinetic (PK) Study of Tafenoquine in Healthy Adults
The goal of this clinical trial is to learn how people's different genetic makeups affects how their bodies convert the FDA approved drug ARAKODA (tafenoquine)…
Kaempferol Absorption and Pharmacokinetics Evaluation (K.A.P.E.)
This study is a multi-site clinical trial designed to evaluate how the body absorbs and processes Kaempferol, a naturally occurring compound found in many plan…
WiTNNess: An International Natural History Study of Autosomal Recessive TNNT1 Myopathy
WiTNNess is designed to accurately document the natural course and variation of muscle disease caused by pathogenic changes of the TNNT1 gene. The primary aim …
Genetic Investigation of Cancer Predisposition
Clinical information and samples (blood, saliva, and tumor) will be collected from patients with multiple cancers and/or a family history of cancer as well as …
Genetic Disorders of Obesity Program Database
This study collects data on children with severe, early-onset obesity.
A Phase 3b Study to Evaluate Efficacy and Safety of a Single Dose of Autologous CRISPR Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (CTX001) in Subjects With Transfusion-Dependent β-Thalassemia or Severe Sickle Cell Disease
This is a single-dose, open-label study in participants with transfusion-dependent β-thalassemia (TDT) or severe sickle cell disease (SCD). The study will eval…
An Adaptive Clinical Trial of BeginNGS Newborn Screening for Hundreds of Genetic Diseases by Genome Sequencing
The goal of this clinical trial is to test a new method for newborn screening using whole genome sequencing, called BeginNGS. Parents will be approached to pro…