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 125 clinical trials
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
beta-human chorionic gonadotropin
myozyme
alglucosidase alfa
deficiency
  • 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
beta-human chorionic gonadotropin
myozyme
alglucosidase alfa
deficiency
  • 4 views
  • 06 May, 2022
  • 1 location
An Investigation of Vitamin A Palmitate Supplementation in Patients 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

vasectomy
tubal ligation
reticular pseudodrusen
hysterectomy
eye drops
  • 115 views
  • 19 Aug, 2022
  • 1 location
Visual Telerehabilitation in AMD Patients

of the retina (PRL), to compensate for visual impairments. However, in many cases, the PRL lands on a sub-optimal retinal area and becomes useless. Modern low-vision rehabilitation procedures for AMD

  • 0 views
  • 08 Oct, 2021
  • 1 location
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

vegf
retinal diseases
macular degeneration
vascular endothelial growth factor
anti-vegf therapy
  • 2 views
  • 16 May, 2022
  • 59 locations
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

edema
  • 4 views
  • 11 Aug, 2022
  • 2 locations
Pivotal 2 Study of RGX-314 Gene Therapy in Participants With nAMD (ASCENT)

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

  • 0 views
  • 18 Jun, 2022
  • 17 locations
MMP-9 Inhibition for Recalcitrant Wet AMD

Wet (or neovascular) form of age-related macular degeneration (wAMD) is the most common cause of blindness in the Western world. Currently, anti-vascular endothelial growth factor (VEGF) intravitreal injections (IVI) remain the standard-of-care treatment for wAMD. Previous studies show that about 90% of treated patients lose minimal visual function after 2 …

  • 0 views
  • 18 Apr, 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