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 15 clinical trials
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Clinical Study for IOPD Participants Less Than or Equal to 6 Months of Age to Evaluate Efficacy and Safety of Enzyme Replacement Therapy (ERT) With Avalglucosidase Alfa

This is a single group, treatment, Phase 3, open-label study to assess efficacy, safety, pharmacokinetic (PK), pharmacodynamics (PD) of avalglucosidase alfa in male and female participants less than or equal to 6 months of age with IOPD. Study details include: Study duration: Screening - up to 4 weeks; Primary Analysis …

deficiency
replacement therapy
  • 0 views
  • 30 Nov, 2021
  • 4 locations
None
GOLDEN STUDY: A Study to Assess Safety and Efficacy of Multiple Doses of IONIS-FB-LRx in Participants With Geographic Atrophy Secondary to Age-Related Macular Degeneration (AMD)

The purpose of the study is to evaluate the effect of IONIS-FB-LRx on the rate of change of the area of geographic atrophy (GA) secondary to age-related macular degeneration (AMD) measured by

geographic atrophy
macular degeneration
mydriasis
vaccination
fundus autofluorescence
  • 521 views
  • 26 Nov, 2021
  • 167 locations
None
  • 10 views
  • 05 Dec, 2021
  • 49 locations
None
  • 57 views
  • 05 Dec, 2021
  • 30 locations
None
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

tubal ligation
reticular pseudodrusen
ophthalmic solution
eye exam
macular degeneration
  • 111 views
  • 24 Nov, 2021
  • 1 location
None
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
  • 3 views
  • 05 Dec, 2021
  • 2 locations
None
Study of Efficacy and Safety of Brolucizumab vs. Aflibercept in Chinese Patients With Neovascular Age-Related Macular Degeneration

To compare brolucizumab to aflibercept in Chinese patients with untreated active choroidal neovascularization (CNV) secondary to age-related macular degeneration (AMD)

neovascularization
aflibercept
age-related macular degeneration
brolucizumab
  • 2 views
  • 05 Dec, 2021
  • 14 locations
None
Efficacy and Safety of Two Different Brolucizumab 6 mg Dosing Regimens in Neovascular Age-related Macular Degeneration

(AMD) who have previously received anti-VEGF (vascular endothelial growth factor) treatment.

diabetic retinopathy
spectral domain optical coherence tomography
neovascularization
macular edema
retinal pigment
  • 0 views
  • 05 Dec, 2021
  • 21 locations
None
A Study Investigating the Efficacy and Safety of Intravitreal Injections of ANX007 in Patients With Geographic Atrophy

This study is being conducted in participants with geographic atrophy (GA) secondary to age-related macular degeneration (AMD) to determine if intravitreal (IVT) injections of ANX007 reduce GA

diabetic retinopathy
geographic atrophy
fundus autofluorescence imaging
atrophy
corrected visual acuity
  • 0 views
  • 05 Dec, 2021
  • 45 locations
None
Vitamin A Palmitate Supplementation in Patients With Reticular Pseudodrusen (RPD) 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

eye exam
reticular pseudodrusen
  • 28 views
  • 28 Nov, 2021
  • 1 location