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 59 clinical trials
OcuDyne System in the Treatment of AMD

This study evaluates the safety and feasibility of using the OcuDyne OPTiC System in patients with late-stage non-exudative age-related macular degeneration.

drusen
corrected visual acuity
atrophy
geographic atrophy
  • 0 views
  • 04 Oct, 2022
  • 2 locations
Treat-and-extend Using Aflibercept for Type 3 Neovascularization

Type 3 neovascularization is a subtype of neovascular age-related macular degeneration (AMD) that is characterized by intraretinal neovascularization. Treat-and-extend (TAE) regimen is a widely

  • 0 views
  • 04 Oct, 2022
  • 1 location
Safety and Tolerability of RPESC-derived RPE Transplantation in Patients With Dry Age-related Macular Degeneration (AMD)

The main objective of the study is evaluation of the safety and tolerability of RPESC-RPE-4W as therapy for dry AMD.

  • 0 views
  • 04 Oct, 2022
  • 1 location
A Randomized, Participant and Investigator Masked, Placebo-controlled, Multicenter, Proof-of-concept Study to Assess the Safety and Efficacy of LNP023 (Iptacopan) in Patients With Early and Intermediate Age-related Macular Degeneration

The purpose of this study is to assess the effect of Iptacopan to prevent conversion of early or intermediate age-related macular degeneration (AMD) eyes to new incomplete retinal pigment

atrophy
optical coherence tomography
retinal pigment
  • 2 views
  • 13 Oct, 2022
  • 10 locations
Long-Term Analysis of DImethyl Fumarate, to Slow the Growth of Areas of Geographic Atrophy (LADIGAGA)

Age-Related Macular Degeneration (AMD).

atrophy
geographic atrophy
retinal pigment
  • 0 views
  • 04 Oct, 2022
A Phase 2, Double-Masked, Randomized, Placebo-Controlled, Dose-Response Study Assessing the Safety and Efficacy of QA102 in Subjects With Dry Age-Related Macular Degeneration (AMD) (AMEND)

This is a phase 2, double-masked, randomized, placebo-controlled, dose-response study. The primary objective of the study is to evaluate the efficacy of QA102 oral capsules on the development of GA or CNV in high-risk eyes.

  • 0 views
  • 21 Oct, 2022
Povidone Iodine Efficacy Study (PIES)

This is a single center, randomized, double-blind, placebo-controlled Phase II clinical trial (RCT) to evaluate the efficacy of topical 10% Polyvinylpyrrolidone-Iodine (PVPI) to prevent new cavitated caries lesions when applied to the teeth of children with Severe Early Childhood Caries (S-ECC). At baseline, following oral rehabilitation in the operating room …

Accepts healthy volunteers
  • 0 views
  • 18 Oct, 2022
  • 1 location
ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD

)-experienced and ERT-naïve pediatric subjects with Pompe disease, aged 0 to < 18 years

alglucosidase alfa
replacement therapy
  • 17 views
  • 04 Oct, 2022
  • 13 locations
Clinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of ERT With Avalglucosidase Alfa (Baby-COMET)

This is a single group, treatment, Phase 3, open-label study to assess efficacy, safety, pharmacokinetic (PK), pharmacodynamics (PD) of avalglucosidase alfa in treatment-naïve male and female participants with IOPD. Study details include: Study duration: Screening - up to 4 weeks; Primary Analysis Period (PAP) - 52 weeks; Extended Treatment Period …

deficiency
replacement therapy
  • 0 views
  • 07 Oct, 2022
  • 9 locations
An Open-label, Multinational, Multicenter, Intravenous Infusion Study of the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Avalglucosidase Alfa in Treatment naïve Pediatric Participants With Infantile-Onset Pompe Disease (IOPD) (Baby-COMET)  

This is a single group, treatment, Phase 3, open-label study to assess efficacy, safety, pharmacokinetic (PK), pharmacodynamics (PD) of avalglucosidase alfa in treatment-naïve male and female participants with IOPD. Study details include: Study duration: Screening - up to 4 weeks; Primary Analysis Period (PAP) - 52 weeks; Extended Treatment Period …

deficiency
replacement therapy
  • 7 views
  • 04 Nov, 2022
  • 10 locations