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 56 clinical trials
A Phase II, Multicenter, Randomized, Single-Masked, Sham-Controlled Study to Assess Safety, Tolerability, and Efficacy of Intravitreal Injections of FHTR2163 in Patients With Geographic Atrophy Secondary to Age-Related Macular Degeneration (GALLEGO) (GALLEGO)

participants with geographic atrophy (GA) secondary to age-related macular degeneration (AMD) compared with sham control. After completing the study's last visit (Week 76), eligible participants will have the

atrophy
macular degeneration
geographic atrophy
corrected visual acuity
age-related macular degeneration
  • 390 views
  • 15 Sep, 2022
  • 69 locations
In Utero Enzyme Replacement Therapy (ERT) for Prenatally Diagnosed Lysosomal Storage Disorders (LSDs). (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
  • 17 Oct, 2022
  • 1 location
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
  • 07 Oct, 2022
  • 1 location
A Phase 1b, Multicenter, Dose Escalation, Evaluation of Safety and Tolerability of ASP7317 for Geographic Atrophy Secondary to Age-related Macular Degeneration

-related macular degeneration (called dry AMD). The macula is the center part of the back of the eye that allows you to see fine detail. In an advanced stage of this disease, areas of the macula die

atrophy
geographic atrophy
corrected visual acuity
spectral domain optical coherence tomography
neovascularization
  • 272 views
  • 15 Oct, 2022
  • 2 locations
A Virtual Reality Platform Simulating Visual Impairment for Testing of Electronic Travel Aids and Performing Orientation and Mobility Training

widespread forms of visual impairment in the United States (US): age-related macular degeneration (AMD), diabetic retinopathy (DR), and glaucoma, each with three levels of severity, (mild, moderate, and

visual impairment
Accepts healthy volunteers
glaucoma
brimonidine tartrate ophthalmic solution
retinopathy
  • 0 views
  • 28 Oct, 2022
  • 1 location
Autologous Transplantation of Induced Pluripotent Stem Cell-Derived Retinal Pigment Epithelium for Geographic Atrophy Associated With Age-Related Macular Degeneration

Background Age-related macular degeneration is a common eye disease in people over 50. The "dry" form of the disease can worsen into geographic atrophy, causing blind spots. Researchers want to learn if replacing older eye cells with younger ones can help treat this disease. Objective To test the safety of …

fundus photography
drusen
atrophy
ophthalmic solution
geographic atrophy
  • 549 views
  • 04 Oct, 2022
  • 1 location
Efficacy of Lenses in Abolishing Photoparoxysmal Responses (PPRs)

Background. Blue lenses that filter out red light have been proposed as a new therapeutic alternative for patients with PSE, such as the lens Zeiss Clarlet Z1. This lens only allows a small overall quantity of visible light, and particularly a minimum percentage of red light, to pass through. However, …

  • 0 views
  • 04 Oct, 2022
  • 2 locations
A Multicenter, Prospective, Observational Study of the Progression of Intermediate Age-Related Macular Degeneration

the timeline and rates of conversion for high-risk iAMD at baseline to more advanced atrophic AMD stages. The other primary objective of this observational study is to assess the feasibility of

fundus photography
drusen
spectral domain optical coherence tomography
neovascularization
optical coherence tomography
  • 0 views
  • 23 Oct, 2022
  • 24 locations
High Resolution Retinal Imaging in Patients With Inherited Retinal Degenerations

The purpose of this study is to determine whether the structure and function of the human retina can be studied with high resolution in patients with inherited retinal degenerations using the Adaptive Optics Scanning Laser Ophthalmoscope (AOSLO).

retinal degeneration
Accepts healthy volunteers
  • 252 views
  • 24 Oct, 2022
  • 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
replacement therapy
deficiency
alglucosidase alfa
myozyme
  • 385 views
  • 07 Oct, 2022
  • 252 locations