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Open-Label Phase 2 Trial of Vyxeos in Patients With Intermediate and High-Risk Acute Myeloid Leukemia Who Have Failed an Initial Cycle of Standard Cytarabine and Daunorubicin Chemotherapy
This phase II trial studies the side effects and how well Vyxeos works in treating patients with intermediate and high-risk acute myeloid leukemia who have fai…
Skeletal Muscle Myosin Binding Protein C in Fatigue and Aging
This study is investigating changes to the proteins in skeletal muscle that contribute to reduced muscle size and muscle function that occurs with aging.
Developing Digital Speech Biomarkers of Bulbar Disease Decline in Spanish-Speaking Persons With Amyotrophic Lateral Sclerosis
The goal of this observational study is to learn how speech and breathing change over time in Spanish-speaking individuals with amyotrophic lateral sclerosis (…
Characterization of Biliary Cell-derived Organoids From Bile of PSC and Non-PSC Patients
The purpose of this research is to create a collection of bile, bile duct brushings and medical information from people with Primary Sclerosing Cholangitis (PS…
Optimal Treatment by Invoking Biologic Clusters in Renal Cell Carcinoma (OPTIC RCC)
This phase II trial tests whether using genetic testing of tumor tissue to select the optimal treatment regimen works in treating patients with clear cell rena…
Promoting Prosocial Behavior in Syndromic Intellectual and Developmental Disabilities
The purpose of this study is to evaluate the effectiveness of an adapted, telehealth functional behavioral therapy (FBTsIDD) specifically focused on promoting …
A Phase 1/2a, Open-Label, Dose-Escalation Study to Evaluate the Safety and Preliminary Efficacy of TRX319 in Subjects With Primary or Secondary Progressive Multiple Sclerosis
The goal of this clinical trial is to treat male and female participants with two types of Multiple Sclerosis (MS) called primary progressive or secondary prog…
A Prospective Observational Patient Registry to Evaluate Disease Progression in Patients With ENPP1 Deficiency and Infantile-Onset ABCC6 Deficiency (GACI Type 2)
The purpose of this prospective registry is to characterize the natural history of ectonucleotide pyrophosphatase/phosphodiesterase1(ENPP1) Deficiency and the …
A Phase 1, Randomized, Double-blind, Placebo-controlled, Single Ascending Dose and Multiple Ascending Dose Study to Evaluate Safety, Tolerability, and Pharmacokinetics of Amisodin in Healthy Adult Subjects
Researchers will evaluate the safety, tolerability, and pharmacokinetics (PK) of orally administered Amisodin in healthy adult subjects through a randomized, d…
Post-Authorization Safety Study for Assessment of Pregnancy Outcomes in Patients Treated With Mayzent (Siponimod): An OTIS Observational Pregnancy Surveillance Study
This study will utilize a prospective, observational, exposure cohort design to examine pregnancy and infant outcomes in women and infants who are exposed to s…
Spastic Paraplegia - Centers of Excellence Research Network (SP-CERN) - Natural History Study Pilot
The Spastic Paraplegia - Centers of Excellence Research Network (SP-CERN) is a collaborative research consortium dedicated to advancing the understanding, diag…
A Phase 1 Dose Escalation and Expansion Study of the c-Kit Specific Antibody-Drug Conjugate NN3201 in Subjects With Advanced and/or Metastatic Solid Tumors Known to Express c-Kit
This open-label clinical trial will evaluate the safety and tolerability of NN3201 in subjects with advanced and/or metastatic solid tumors known to express c-…