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 35 clinical trials
Study to Evaluate Efficacy and Safety in Chinese Patients With Late Onset Pompe Disease With Alglucosidase Alfa Treatmen

)] 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
  • 25 Mar, 2021
  • 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
deficiency
myozyme
alglucosidase alfa
  • 0 views
  • 22 Jan, 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 …

hysterectomy
vitamin
eye drops
vasectomy
macular degeneration
  • 111 views
  • 19 Jan, 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.

atrophy
vitamin
macular degeneration
  • 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
  • 07 Dec, 2021
  • 2 locations
Pivotal 1 Study of RGX-314 Gene Therapy in Participants With nAMD

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 …

vascular endothelial growth factor
macular degeneration
vegf
anti-vegf therapy
gene therapy
  • 1 views
  • 26 Sep, 2021
  • 26 locations
In Utero Enzyme Replacement Therapy for Lysosomal Storage Diseases

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.

  • 0 views
  • 18 Apr, 2021
  • 1 location
Exercise Capacity in Response to Enzyme Replacement Therapy in Pediatric Pompe Disease.

Our aims are to investigate the acute and long term effect of ERT on exercise capacity; comparing the effect of different ERT dosages (as prescribed by the clinician according to clinical judgment) and assessing the relationship between enzyme blood level and exercise capacity. Such evaluation may allow a more objective …

  • 0 views
  • 24 Feb, 2021
  • 1 location
Pompe Disease Registry Protocol

The Pompe Registry is a global, multicenter, international, longitudinal, observational, and voluntary program for patients with Pompe disease, designed to track the disease's natural history

gaa gene
alglucosidase alfa
deficiency
  • 0 views
  • 22 Jan, 2022
  • 250 locations
Pompe Disease Registry Protocol

The Pompe Registry is a global, multicenter, international, longitudinal, observational, and voluntary program for patients with Pompe disease, designed to track the disease's natural history

replacement therapy
gaa gene
deficiency
myozyme
alglucosidase alfa
  • 367 views
  • 23 Jan, 2022
  • 250 locations