Endocrinology Trials

  • 30/09/2019

    Setmelanotide (RM-493) Phase 2 Treatment Trial in Patients with rare genetic disorders of obesity

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  • 31/10/2023

    COMET

    A phase 3 randomized multicenter multinational double-blinded study comparing the efficacy and safety of repeated biweekly infusions of neoGAA (GZ402666) and alglucosidase alfa in treatment-naïve patients with late onset Pompe disease

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  • 31/07/2022

    ASCEND

    A phase 2/3 multicenter randomized double-blinded placebo-controlled repeat-dose dose-comparison study to evaluate the efficacy safety pharmacodynamics and pharmacokinetics of olipudase alfa in patients with acid sphingomyelinase deficiency

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  • 13/07/2016

    Acceptability of Glycomacropeptide (GMP) Protein Substitutes for Adults With PKU

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  • 01/08/2040

    RAPSODI GD

    Remote assessment of Parkinsonism supporting ongoing development of interventions in Gaucher’s disease

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  • 30/11/2023

    A Long-Term Follow-up Study to Evaluate the Safety and Efficacy of Adeno-Associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Human Ornithine Transcarbamylase (OTC) in Adults with Late-Onset OTC Deficiency

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  • 31/12/2026

    A PROSPECTIVE OBSERVATIONAL REGISTRY OF PATIENTS WITH FABRY DISEASE

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  • 31/03/2020

    MODIFY

    A multicenter double-blind randomized placebo controlled parallel-group study to determine the efficacy and safety of
    lucerastat oral monotherapy in adult subjects with Fabry disease.

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  • 25/10/2015

    The I-DSD Registry

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  • 30/09/2020

    DIUR-005

    A phase III study of efficacy safety and tolerability of Chronocort compared with standard glucocorticoid replacement therapy in the treatment of congenital adrenal hyperplasia (CAH)

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