Found 185 matching trials
TASO-001 in Combination With Recombinant Interleukin-2(Aldesleukin) in Advanced or Metastatic Solid Tumor PHASE1 To evaluate safety, tolerance and efficacy of TASO(TGF-β2 targeting anti-se...
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Seoul National University Hospital
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Antisense Oligonucleotide for Spinal Muscular Atrophy N/A This is a longitudinal, multiple-center, observational study of patients ge...
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Department of Neurology, Peking Union Medical College Hospital
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Clinical Trial of BP1001 in Combination With With Venetoclax Plus Decitabine in AML PHASE2 The primary objectives of this study are to assess: (1) whether the combina...
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UCLA Medical Center
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Phase 1 Study of EZN-2968 Weekly in Adult Patients With Advanced Solid Tumors or Lymphoma PHASE1 This is a Phase 1, open-label, non-randomized, dose-escalation study to det...
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Duke University Medical Center
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Safety, Tolerability and Pharmacokinetics of Multiple Ascending Doses of NIO752 in Progressive Supranuclear Palsy PHASE1 This is a phase 1, multi-center, double-blind, placebo-controlled, multiple...
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University of California San Diego
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Investigation of Individualised Antisense Oligonucleotides (ASOs) in People With Unique Genetic Variants Causing Severely Debilitating, Life Threatening (SDLT) Central Nervous System (CNS) Conditions PHASE1 This study is being conducted to evaluate individualised antisense oligonuc...
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Active Not Recruiting
Location
Great Ormond Street Hospital
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Personalized Antisense Oligonucleotide Therapy for A Single Participant With ATN1 Gene Mutation PHASE1 This research project entails delivery of a personalized antisense oligonuc...
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Active Not Recruiting
Location
Columbia University
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Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Donidalorsen (IONIS-PKK-LRx) Administered Subcutaneously to Healthy Volunteers PHASE1 The purpose is to assess the Safety, Tolerability, Pharmacokinetics, and Ph...
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Ionis Investigative Site
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An Open-Label Extension Study to Evaluate the Long-term Safety and Efficacy of BMN 351 in Participants With Duchenne Muscular Dystrophy PHASE2 This open-label extension study aims to evaluate the long-term safety and t...
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Enrolling By Invitation
Location
Fondazione Serena ETS - Centro Clinico NeMO Milano
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Open-label Extension Study to Evaluate the Safety and Tolerability of WVE-120102 in Patients With Huntington's Disease PHASE1 WVE-HDSNP2-002 is an open-label extension (OLE) study to evaluate the safet...
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Westmead Hospital
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