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 11 clinical trials
A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)

The purpose of this study is to evaluate the safety, tolerability, and efficacy of a single intravenous infusion of SPK-3006 in adults with clinically moderate, late-onset Pompe disease

replacement therapy
gene therapy
  • 26 views
  • 25 Jul, 2021
  • 21 locations
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

mydriasis
macular degeneration
geographic atrophy
fundus autofluorescence imaging
pupillary dilation
  • 515 views
  • 19 Jul, 2021
  • 156 locations
Study to Gather Information on Safety and Use of High Dose Aflibercept Injection Into the Eye in Patients With an Age Related Eye Disorder That Causes Blurred Vision or a Blind Spot Due to Abnormal Blood Vessels That Leak Fluid Into the Light Sensitive Lining Inside the Eye

-related macular degeneration (nAMD). Neovascular AMD is an eye disease that causes blurred vision or a blind spot due to abnormal blood vessels that leak fluid or blood into the light sensitive lining

  • 0 views
  • 01 Aug, 2021
  • 310 locations
Restoration of Central Vision With the PRIMA System in Patients With Atrophic AMD

The objective of this study is to evaluate the efficacy and safety of the PRIMA System in patients with atrophic AMD. Eligible subjects will be implanted with the PRIMA Implant. The subjects

geographic atrophy
corrected visual acuity
atrophy
vision test
  • 0 views
  • 01 Aug, 2021
  • 6 locations
Efficacy and Safety of Two Different Brolucizumab 6 mg Dosing Regimens in Neovascular Age-related Macular Degeneration

The purpose of this study is to evaluate the efficacy and safety of two different brolucizumab 6 mg dosing regimens in patients with visual impairment due to age-related macular generation (AMD

detachment
diabetic retinopathy
vegf
neovascularization
retinopathy
  • 0 views
  • 01 Aug, 2021
  • 10 locations
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 …

ophthalmic solution
detachment
fundus photography
fundus autofluorescence imaging
geographic atrophy
  • 421 views
  • 31 Jul, 2021
  • 1 location
Intrathecal Administration of scAAV9/JeT-GAN for the Treatment of Giant Axonal Neuropathy

Background The Gigaxonin gene lets the body make a protein chemical called Gigaxonin. Nerves need Gigaxonin to work properly. Giant Axonal Neuropathy (GAN) causes a shortage of functional Gigaxonin. Nerves stop working normally in people with GAN. This causes problems with walking and sometimes with eating, breathing, and many other …

weakness
MRI Scan
skin biopsy
  • 43 views
  • 01 Aug, 2021
  • 1 location
Effect of Enzyme Replacement Therapy in Patients With Juvenile-onset Pompe Disease

Pompe disease is known as glycogen storage disease type II, an autosomal recessive disease that results from acid alpha-glucosidase (GAA) deficiency leading to lysosomal glycogen accumulation

  • 0 views
  • 17 Jul, 2021
  • 1 location
Daily Testing at Home by NV-AMD Subjects With Notal Home OCT.

The purpose of this study is to evaluate the ability of subjects with NV-AMD to perform sequential daily self-imaging of their eyes with the self-operated Notal Home OCT device in their homes

  • 0 views
  • 18 Jul, 2021
  • 1 location
Cell Collection to Study Eye Diseases

Background Best Vitelliform Dystrophy (Best disease), Late-Onset Retinal Degeneration (L-ORD), and Age-Related Macular Degeneration (AMD) all affect the retina

retinal degeneration
macular degeneration
retinal disease
retinal diseases
retinopathy
  • 90 views
  • 01 Aug, 2021
  • 1 location