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Histiocytosis, Langerhans-cell
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Histiocytosis, Langerhans-cell trials may be worth asking aboutClinical trial pipeline · Data from ClinicalTrials.gov
See which Histiocytosis, Langerhans-cell trials may be worth asking aboutNorth America
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This study will evaluate clinical and laboratory tests that might be useful in determining if an investigational drug can slow the progression of Niemann-Pick D…
This open-label study aims to gather long-term safety, tolerability, PK, biomarker, and clinical efficacy data relating to daily administration of Nizubaglustat…
An 18-month double-blind, randomized, placebo-controlled, multicenter, Phase 3 study to evaluate the safety and efficacy of oral nizubaglustat (AZ-3102) in late…
An 18-month double-blind, randomized, placebo-controlled, multicenter, Phase 3 study to evaluate the safety and efficacy of oral nizubaglustat (AZ-3102) in late…
Background: Niemann-Pick type C (NPC) disease is a rare, progressive neurodegenerative disease that affects mainly the brain, liver, and spleen but also other…
This phase II trial tests the safety, side effects, best dose and activity of tovorafenib (DAY101) in treating patients with Langerhans cell histiocytosis that…
A roll-over study to assess long-term effect in pediatric patients treated with dabrafenib and/or trametinib.
The purpose of this study is to use agnostic genomic evaluation using whole exome sequencing (WES) of a variety of rare hematologic diseases grouped under rare…