Pompe Disease Clinical Trials

A listing of Pompe Disease medical research trials actively recruiting patient volunteers. Search for closest city to find more detailed information on a research study in your area.

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Found 41 clinical trials
Avalglucosidase Alfa French Post-trial Access for Participants With Pompe Disease (PTA Avalglucosidase)

Pompe disease in France who have completed Study EFC14028, LTS13769, or ACT14132, from market authorization until reimbursement of avalglucosidase alfa in France or until May 2023, whichever comes first

beta-human chorionic gonadotropin
replacement therapy
  • 0 views
  • 08 Aug, 2022
  • 6 locations
Study to Evaluate Efficacy and Safety in Chinese Patients With Late Onset Pompe Disease With Alglucosidase Alfa Treatmen (APOLLO-LOPD)

)] among Chinese Late Onset Pompe Disease patients above 5 years old. To evaluate the safety of Myozyme 20mg/kg, IV biweekly in Chinese LOPD patients above 3 years old. Secondary Objective

  • 0 views
  • 10 May, 2022
  • 1 location
Development of an Assessment Tool for Health-related Quality of Life in Children and Adolescents With Pompe Disease (Pompe_HrQol)

Health related quality of life (HrQoL), is "the patient's subjective perception of the impact of his disease and its treatment on his daily life, physical, psychological and social functioning and well-being" and thus constitutes a patient reported outcome (PRO) of utmost importance. Generic HrQoL instruments can by definition not capture …

  • 0 views
  • 13 Jun, 2022
  • 1 location
Study to Evaluate Efficacy and Safety in Chinese Patients With Late Onset Pompe Disease With Alglucosidase Alfa Treatmen (APOLLO-LOPD)

)] among Chinese Late Onset Pompe Disease patients above 5 years old. To evaluate the safety of Myozyme 20mg/kg, IV biweekly in Chinese LOPD patients above 3 years old. Secondary Objective

gaa gene
alglucosidase alfa
deficiency
beta-human chorionic gonadotropin
myozyme
  • 4 views
  • 06 May, 2022
  • 1 location
Vitamin A Palmitate Supplementation in People With Age-Related Macular Degeneration (and Without Reticular Pseudodrusen) and Delayed Dark Adaptation

Background Age-related macular degeneration (AMD) is an eye disease. It is the leading cause of vision loss in people over 55 in the U.S. Changes in the eye can make it difficult for the eye to adjust to low light. This is known as dark adaptation. Identifying and watching the …

vitamin
hysterectomy
urine tests
eye disease
retinal diseases
  • 115 views
  • 20 Jul, 2022
  • 1 location
Study to Evaluate the Response to Supplementation With Postbiotics in Patients With Macular Degeneration.

A pilot study to establish the efficacy and safety of supplementation with postbiotics in patients with macular degeneration.

macular degeneration
atrophy
vitamin
  • 0 views
  • 01 Oct, 2021
  • 1 location
Retinal Imaging Using NOTAL-OCT V3.0

The study will include up to 250 AMD patients and up to 30 DR patients. In Tel Aviv medical center up to 100 AMD/ 20 DR patients will be recruited; In Assuta HaShalom up to 100 AMD/10 DR patients will be recruited; In Bnei Zion medical center up to 50 …

edema
  • 4 views
  • 22 Apr, 2022
  • 2 locations
Pivotal 1 Study of RGX-314 Gene Therapy in Participants With nAMD (ATMOSPHERE)

RGX-314 is being developed as a novel one-time gene therapy for the treatment of neovascular (wet) age-related macular degeneration (wet AMD). Wet AMD is characterized by loss of vision due to new, leaky blood vessel formation in the retina. Wet AMD is a significant cause of vision loss in the …

anti-vegf therapy
vegf
vascular endothelial growth factor
lucentis
retinal diseases
  • 1 views
  • 16 May, 2022
  • 34 locations
In Utero Enzyme Replacement Therapy for Lysosomal Storage Diseases (IUERT)

The investigators aims to determine the the maternal and fetal safety and feasibility of in utero fetal enzyme replacement therapy in fetuses with Lysosomal Storage Diseases.

replacement therapy
  • 11 views
  • 15 May, 2022
  • 1 location
Assessment of Safety and Acute Effects of a Lower-limb Powered Dermoskeleton in Patients With Neuromuscular Disorders (Exo-KGO1)

The aims of the current study are as follow: i) Evaluate the safety, usability, and acute efficiency of a programmable ambulation exoskeleton (KeeogoTM Dermoskeleton System, B-Temia Inc., Quebec, Canada) in patients with neuromuscular disorders, ii) Elaborate recommendations regarding usability criteria for safe and efficient use the device in patients with …

respiratory assessment
Accepts healthy volunteers
myopathy
  • 0 views
  • 24 Apr, 2022
  • 1 location