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Found 150 clinical trials
A Prospective Study to Observe & Describe Clinical Outcomes of Alglucosidase Alfa Treatment in Patients ≤6 Months of Age With Infantile-onset Pompe Disease (IOPD)

treatment.

Secondary Objectives:

<ul>
  • To describe the effect of routine practice with alglucosidase alfa on invasive ventilation-free survival and survival at 12 and 18 months of age, as

    alglucosidase alfa
    alpha-glucosidase
    deficiency
    assisted ventilation
    replacement therapy
    • 0 views
    • 01 Mar, 2024
    • 14 locations
    Mucopolysaccharidosis I (MPS I) Registry

    -Iduronidase), or other treatment modalities.

    The objectives of the Registry are:

    <ul>
  • To evaluate the long-term effectiveness and safety of Aldurazyme® (laronidase)
  • To characterize

    • 0 views
    • 06 Feb, 2025
    • 171 locations
    Fabry Disease Registry & Pregnancy Sub-registry

    of the Registry are:

    <ul>
  • To enhance the understanding of the variability, progression, and natural history of Fabry disease, including heterozygous females with the disease;
  • To

    agalsidase beta
    fabrazyme
    deficit
    deficiency
    alpha-galactosidase
    • 0 views
    • 03 Dec, 2024
    • 256 locations
    Pompe Disease Registry Protocol

    research and non-research related purposes.

    The objectives of the Registry are:

    <ul>
  • To enhance understanding of the variability, progression, identification, and natural history of

    gaa gene
    alglucosidase alfa
    deficiency
    • 0 views
    • 03 Dec, 2024
    • 200 locations
    International Collaborative Gaucher Group (ICGG) Gaucher Disease Registry & Pregnancy Sub-registry

    of the Registry are:

    <ul>
  • To enhance understanding of the variability, progression, identification, and natural history of Gaucher disease, with the ultimate goal of better guiding and

    type 1 gaucher disease
    deficiency
    alglucerase
    imiglucerase
    eliglustat
    • 0 views
    • 06 Feb, 2025
    • 288 locations
    An Observational Study to Assess the Effectiveness and Safety of Cemiplimab in Patients With Advanced Non-small Cell Lung Cancer (NSCLC) in Routine Clinical Practice Settings in Europe (CEMI-LUNG)

    Primary Objective: Describe the overall survival rate (OS) rate in patients with advanced NSCLC treated with a cemiplimab-based regimen in routine clinical practice. Secondary

    lung carcinoma
    • 0 views
    • 06 Feb, 2025
    • 10 locations
    A Study to Evaluate the Quality of Life in Male and Female Adult Participants With Severe Asthma Treated With Dupilumab in a Real-world Setting

    Primary Objective:

    <ul>
  • To describe dupilumab health-related quality of life (HRQoL) effectiveness at 52 weeks compared to baseline.
    • ul>

    Secondary Objectives:

    <ul>
    • 0 views
    • 03 Dec, 2024
    • 1 location
    A Non-Interventional National Study in Pediatric Patients With Unexplained Enlarged Spleen

    (e.g. portal hypertension, haematological malignancy, hemolytic anemia, infection) based on clinical examination and routine biological tests (full blood count, reticulocytes, liver tests, abdominal

    • 0 views
    • 01 Mar, 2024
    • 40 locations
    Avalglucosidase Alfa Pregnancy Study

    avalglucsodiase alfa on pregnancy and maternal complications and adverse effects in the developing fetus, neonate, and infant.

    <ul>
  • Outcomes in exposed infants, including growth and development, will be

    • 0 views
    • 06 Feb, 2025
    • 1 location
    Long-term Safety and Efficacy of a Modified Suprachoroidal Silicone Tube (SST) Shunt

    The goal of this observational study is to evaluate the long-term safety and efficacy of a modified suprachoroidal silicone tube (SST) shunt in treating patients with primary open-angle glaucoma (POAG) and pseudo-exfoliative glaucoma (PXG). The main questions it aims to answer are: Does the SST shunt maintain intraocular pressure (IOP) …

    • 0 views
    • 06 Feb, 2025
    • 1 location