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NTLA-2001 in Transthyretin Amyloidosis With Cardiomyopathy

The purpose of this study is to understand how an experimental drug called NTLA-2001 affects a heart condition called transthyretin (TTR) amyloidosis with cardiomyopathy (ATTR-CM), a condition in which the heart muscle does not pump ...


Unknown Sites

Children's Hospital of Philadelphia, EIIT-LIFFT, Open label, PhI/II, FET-Fused Tumors, Lurbinectedin

Primary Objective: Phase 1: To determine the safety, tolerability, pharmacokinetic profile and recommended phase 2 dose (RP2D) of lurbinectedin given on a Day 1, 4 schedule every 21 days in patients with FET fusion tumors. Phase 2: ...


Eye and Orbit, Liver, Melanoma

RP2 in Combination With Nivolumab Versus Ipilimumab in Combination With Nivolumab in Metastatic Uveal Melanoma

This study focuses on immune checkpoint inhibitor-naive adults with inoperable metastatic uveal melanoma (a type of aggressive eye cancer that has not been treated with immune checkpoint inhibitor therapies). The purpose of the study is ...


Leukemia, other

Venetoclax and HMA-based Therapies for Adults With FLT3-mutated Acute Myeloid Leukemia: A MyeloMATCH Treatment Trial

This study focuses on adults diagnosed with acute myeloid leukemia (AML) who have previously participated in another study called the myeloMATCH study. The purpose of this research is to compare the usual drug combination treatment ...


Esophagus, Stomach

Nivolumab + Paclitaxel + Ramucirumab in Advanced Gastric and Esophageal Adenocarcinoma

This study focuses on people who have advanced stomach or esophageal cancer that has spread. The purpose of the study is to compare the usual treatment alone (the combination of 2 drugs, paclitaxel and ramucirumab) ...