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A Study to Investigate the Pharmacokinetics (PK) and Safety and to Provide Proof of Mechanism of Alogabat in Children and Adolescents Aged 5-17 Years With Angelman Syndrome (AS) With Deletion Genotype.
Expand descriptionThis is a two-part, Phase IIa, multicenter, 12-week, open-label study. Up to 56 participants with deletion AS aged 5-17 years (inclusive) will be enrolled in the study.
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4D-310 in Adults With Fabry Disease and Cardiac Involvement
Expand descriptionThis is a prospective multicenter, open-label, dose-escalation trial to assess the safety, tolerability, and pharmacodynamics of 4D-310 following a single IV administration. The study population is comprised of adult males and females with Fabry Disease and cardiac involvement
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The Communicate Study Partnership
Expand descriptionThe vision of the Communicate Study Partnership is to ensure more Aboriginal patients receive culturally safe healthcare in their first language. The Communicate Study Partnership will implement and evaluate creative ways to embed cultural safety training and increase use of Aboriginal Interpreters and Aboriginal Health Practitioners at Northern Territory Top End hospitals. Quantitative outcomes (interpreter uptake, outcomes including leave against medical advice, costs) will be measured using time-series analysis. Qualitative outcomes derived from interviews with patient, healthcare provider and interpreter participants, will be informed by decolonising theory and participatory approaches. Successful project implementation will improve experience of care and health outcomes for Aboriginal people, build Aboriginal workforce, and improve healthcare provider satisfaction.
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A Multi-Centre, Randomised, Controlled, Masked Phase 2 Study of the Safety and Efficacy of KIO-101 Ophthalmic Solution, Versus Vehicle Control in the Treatment of Dry Eye Disease in Patients With Autoimmune Disease
Expand descriptionA Multi-Centre, Randomised, Controlled, Masked Phase 2 Study of the Safety and Efficacy of KIO-101 Ophthalmic Solution, versus Vehicle Control in the treatment of Dry Eye Disease in Patients with Autoimmune Disease
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AC-OLE-01-VA Tolerability Study in Healthy Participants Under Different Dosing Conditions
Expand descriptionThis is an open-label, 3-arm study to compare tolerability of three different titration and dosing schedules of the AC-OLE-01-VA formulation of tricaprilin. Following a screening of up to 28 days, eligible participants will be initially randomised to one of 2 arms (Arm 1 or Arm 2). Arms 1 and 2 will be completed in advance of Arm 3 with all subsequent participants allocated to Arm 3.
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Evaluating the Efficacy of NTI164 in Young People With Autism Spectrum Disorder
Expand descriptionThis is an 18 to 54 week study assessing the efficacy of Full-Spectrum Medicinal Cannabis Plant Extract 0.08% THC (NTI164) on the severity of autism spectrum disorder in young people.
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Open-label, Long-term Safety Study of LP352 in Subjects With Developmental and Epileptic Encephalopathy
Expand descriptionThe objective of this study is to assess the long-term safety, tolerability, and efficacy of adjunctive therapy of LP352 in subjects with developmental and epileptic encephalopathies who completed participation in Study LP352-201.
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A Study of BMS-986360/CC-90001 Alone and in Combination With Chemotherapy or Nivolumab in Advanced Solid Tumors
Expand descriptionThe aim of this study is to assess the safety and tolerability of BMS-986360 as monotherapy and in combination with chemotherapy or nivolumab in participants with advanced solid tumors.
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A Study of Subcutaneous Nivolumab + Relatlimab Fixed-dose Combination (FDC) in Previously Untreated Metastatic or Unresectable Melanoma
Expand descriptionThe purpose of this study is to demonstrate that the study drug exposure level of the nivolumab + relatlimab FDC subcutaneous (SC) formulation is not worse than nivolumab + relatlimab FDC intravenous (IV) administration in participants with previously untreated metastatic or unresectable melanoma.
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Phase 3 Study to Evaluate Ianalumab on Top of Standard-of-care Therapy in Patients With Systemic Lupus Erythematosus (SIRIUS-SLE 2)
Expand descriptionThe trial will evaluate efficacy, safety and tolerability of ianalumab compared to placebo, given as monthly subcutaneous (s.c.) injection on top of standard-of-care (SoC) treatment in participants with active systemic lupus erythematosus (SLE).