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A Phase 3 Study to Evaluate the Safety and Efficacy of Efgartigimod PH20 Subcutaneous in Adult Patients with Primary Immune Thrombocytopenia
A Phase 3 study to evaluate the safety and efficacy of efgartigimod PH20 subcutaneous in adult patients with primary immune thrombocytopenia
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Genotype-phenotype Correlations in Children and Adults With CTNNB1 Mutation
The aim of this study is to explore and define the correlations between genotype and phenotype in patients with CTNNB1 mutations as well as monitor and survey the natural history of the CTNNB1 syndrome.
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A Phase 1/2 Study of Bleximenib in Participants With Acute Leukemia (cAMeLot-1)
The purpose of this study is to determine the recommended Phase 2 dose(s) (RP2D\[s\]) of bleximenib in phase 1 Part 1 (Dose Escalation) and to determine the safety and tolerability at RP2D in Phase 1 Part 2 (Dose expansion). The purpose of the Phase 2 part of the study is to evaluate the efficacy of bleximenib at the RP2D.
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Safety, Tolerability and Pharmacokinetics of CBP-174 in Healthy Adults
The purpose of this study is to evaluate the safety, tolerability and pharmacokinetics of CBP-174 after a single oral dose in healthy adult subjects.
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Study of Sotatercept in Newly Diagnosed Intermediate- and High-Risk PAH Participants (MK-7962-005/A011-13)
The objective of this study is to evaluate the effects of sotatercept (MK-7962, formerly called ACE-011) treatment (plus background pulmonary arterial hypertension (PAH) therapy) versus placebo (plus background PAH therapy) on time to clinical worsening (TTCW) in participants who are newly diagnosed with PAH and are at intermediate or high risk of disease progression.
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Combination Study With Eftilagimod Alpha (a Soluble LAG-3 Fusion Protein) and Pembrolizumab in Patients With Recurrent or Metastatic HNSCC
Evaluate the safety and efficacy of eftilagimod alpha in combination with pembrolizumab against pembrolizumab alone in 1st line metastatic or recurrent HNSCC with programmed death-ligand 1 (PD-L1) positive (combined positive score \[CPS\] =1) tumors, and determine the efficacy and safety of efti plus pembrolizumab in patients with PD-L1 negative tumors.
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Phase Ib Study of Select Drug Combinations in Patients With Lower Risk MDS
The purpose of this study was to characterize the safety, tolerability and confirm the dose for select single agents and combinations in patients with lower risk (very low, low, and intermediate risk) MDS.
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Treatment Effects of Subcutaneous Injections of Pentosan Polysulfate Sodium vs Placebo in Participants With Knee OA Pain
The purpose of this study is to measure the change in pain and function with subcutaneous injections of pentosan polysulfate sodium (PPS) compared with subcutaneous injections of placebo in participants with knee osteoarthritis pain. Study details include: * The study duration will be up to 31 weeks per participant * The treatment duration will be 6 weeks. * The visit frequency will be twice weekly during treatment. * The visit frequency will be every 4 weeks during the follow-up period.
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Prisons Evaluation of a One-stop-shop InterVentiOn
A prospective historically controlled study to assess the effect of an intervention integrating point-of-care hepatitis C (HCV) RNA testing, non-invasive liver fibrosis assessment, fast-tracked direct-acting antiviral (DAA) prescription, and linkage to hepatitis care (a 'one-stop-shop' intervention), on the proportion of participants initiating DAA therapy among people who are recently incarcerated within reception correctional centre(s) in Australia.
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Real-world Treatment Patterns and Associated Outcomes in Patients With Resectable Early-stage Non-small Cell Lung Cancer
This is a non-interventional, multi-country, multicentre, retrospective study designed to determine the treatment patterns and associated survival rate in patients with primary stage IA to IIIB resectable NSCLC diagnosed between 01 January 2013 and 31 December 2017 and followed until at least 31 December 2020 The main objective of this study is to describe the treatment patterns and determine their associated 3-year survival rate according to clinical and pathologic staging in patients with resectable early-stage (IA to IIIB as per AJCC seventh edition) NSCLC.