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
  • 0 views
  • 07 Oct, 2022
  • 6 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)

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 fundus autofluorescence

corrected visual acuity
pneumoniae
mydriasis
fundus autofluorescence imaging
vaccination
  • 1382 views
  • 04 Oct, 2022
  • 84 locations
  • 73 views
  • 04 Oct, 2022
  • 63 locations
A Phase 2, Double-Masked, Placebo-Controlled, Dose Range Finding Study of Danicopan (ALXN2040) in Patients With Geographic Atrophy (GA) Secondary to Age-Related Macular Degeneration (AMD)

This is a dose finding study designed to evaluate the efficacy, safety, and pharmacokinetics of danicopan in participants with GA secondary to AMD. The study consists of a Screening Period of up

geographic atrophy
atrophy
  • 1 views
  • 15 Oct, 2022
  • 59 locations
Restoration of Central Vision With the PRIMA System in Patients With Atrophic Age-Related Macular Degeneration (PRIMAvera)

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

vision test
geographic atrophy
corrected visual acuity
atrophy
  • 0 views
  • 21 Oct, 2022
  • 20 locations
Avalglucosidase Alfa French Post-trial Access for Participants With Pompe Disease (PTA Avalglucosidase)

Pompe disease in France who have completed Study EFC14028, LTS13769, or ACT14132, from market authorization until reimbursement of avalglucosidase alfa in France or until May 2023, whichever comes first

replacement therapy
beta-human chorionic gonadotropin
  • 0 views
  • 07 Oct, 2022
  • 10 locations
Avalglucosidase Alfa French Post-trial Access for Participants With Pompe Disease (PTA Avalglucosidase)

Pompe disease in France who have completed Study EFC14028, LTS13769, or ACT14132, from market authorization until reimbursement of avalglucosidase alfa in France or until May 2023, whichever comes first

replacement therapy
beta-human chorionic gonadotropin
  • 0 views
  • 20 Jan, 2023
  • 10 locations
A Single Arm, Prospective, Open-label, Multi-center Study to Evaluate Efficacy and Safety in Chinese Patients With Late Onset Pompe Disease With Alglucosidase Alfa Treatment (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
alglucosidase alfa
myozyme
deficiency
beta-human chorionic gonadotropin
  • 4 views
  • 28 Oct, 2022
  • 1 location
Single Arm, Multicenter, Open and Dose-escalation Clinical Study on Safety, Tolerance, and Efficacy of GC301, an AAV-Delivered Gene Transfer Therapy in Patients With Infantile-onset Pompe Disease

therapy for Pompe disease. Patients diagnosed with infantile-onset Pompe disease who are younger than 6 months old will be studied.

  • 0 views
  • 25 Oct, 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
vitamin
retinal diseases
blindness
hysterectomy
  • 115 views
  • 20 Oct, 2022
  • 1 location