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A Phase 3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study Evaluating the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Subjects With α- or β- Transfusion-Dependent Thalassemia
The primary objective of this study is to compare the effect of mitapivat versus placebo on transfusion burden in pediatric participants with α- or β-transfusi…
A Randomized, Placebo-controlled, Double-blind Trial of Suzetrigine in Total Hip Arthroplasty
Total hip and knee joint replacements are among the most common and painful orthopedic procedures performed worldwide, often requiring intensive analgesia to s…
Matched Related Donor Non-myeloablative Hematopoietic Cell Transplantation With Alemtuzumab, 400 cGy TBI, and Abatacept for Sickle Cell Disease and Beta-Thalassemia
This is a non-ablative (partial) stem cell transplant for patients with severe sickle cell disease or beta-thalassemia requiring red cell transfusions. The int…
The Low-Down on Methamphetamine Isomers: Prevalence and Pharmacology in Humans
This study is being done to understand the metabolism and impairment profile of methamphetamine (meth). Meth exists as two chemical structures that are mirror …
A Phase 2b, Randomized, Blinded Trial Investigating the Efficacy and Safety of Visugromab in Combination With Nivolumab and Lenvatinib Compared to Double Placebo and Lenvatinib in Participants With Unresectable or Metastatic Hepatocellular Carcinoma and Compensated Liver Function (Child-Pugh A) After Failure of First-Line Treatment That Included an Approved Anti PD-(L)1 Compound (GDFATHER HCC-01)
This is a Phase 2b, randomized, blinded clinical trial investigating the efficacy and safety of visugromab in combination with nivolumab and Lenvatinib compare…
Role of Estrogen Formulation and Route of Delivery on Skeletal Outcomes in Functional Hypothalmic Amenorrhea
The purpose of this study is to assess whether the natural form of estrogen (17-beta estradiol) given as a patch so that it is absorbed through your skin, is b…
Understanding and Improving Access to Section 504 Among Students With Disabilities
The goal of this training program is to learn if the intervention (i.e., training program) can improve access to Section 504 among students with disabilities a…
A Phase II Trial Evaluating Chemotherapy Followed by Response-Based Reduced Radiation Therapy for Patients With Central Nervous System Germinomas
This phase II trial studies how well lower dose radiotherapy after chemotherapy (Carboplatin \& Etoposide) works in treating children with central nervous syst…
Improving Secure Storage of Firearms to Reduce Suicide Deaths: A Hybrid Type 1 Implementation Effectiveness Randomized Controlled Trial of the Family Firearms Secure Storage Training (FFAST) Intervention
Firearms are the lethal means used in over 70% of Veteran suicides with rates of Veteran deaths via self-induced firearm injury rising dramatically (by 64.8%) …
A Pilot Trial of Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Sickle Cell Disease
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…
The Role of Bimatoprost in Graves' Periorbitopathy
Testing whether Bimatoprost, a topical eye drop used for glaucoma, can be used to induce periorbital fat atrophy in patients with Graves' disease
A Phase 2, Double-Blind, Placebo-Controlled Study to Investigate the Safety and Efficacy of Mibavademab in Adult Women With Functional Hypothalamic Amenorrhea
This study is researching an experimental drug called mibavademab (also called "study drug"). The study is focused on patients with a condition called Function…