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.
Found 43 clinical trials
Pharmacogenomics of antiVEGF in Patients With Age-Associated Macular Degeneration (AMD)
This is a phase III, multicenter, randomized double-blinded clinical trial with two parallel groups (ranibizumab and bevacizumab) and an observational follow-up of patients who meet elegibility criteria and decline participation due to treatment randomization. It will be performed involving 630 eyes from patients with wet age-related macular degeneration (wAMD) diagnosis …
Treatment of Neovascular AMD: Artificial Intelligence in Real-world Setting
The purpose of this study is to implement quantitative assessment tools for the treatment of active neovascular AMD patients in a real-world setting in order to provide advantages for both patients (treatment burden) and healthcare system (scheduling visits/treatments).
Home OCT-Guided Treatment Versus Treat and Extend for the Management of Neovascular AMD
Home optical coherence tomography- guided treatment versus treat and extend for the management of neovascular age-related macular degeneration.
A Long-term Extension Study of JNJ-81201887 (AAVCAGsCD59) Parent Studies in Participants With Geographic Atrophy (GA) Secondary to Age-related Macular Degeneration (AMD)
The purpose of this study is to assess the long-term safety and tolerability after an intravitreal injection (a shot of medicine into the eye) of JNJ-81201887 administered in parent clinical studies.
Evaluation of the Safety and Efficacy of Late-onset Pompe Disease Gene Therapy Drug
This study is being conducted to evaluate the safety and effectiveness of GC301 adeno-associated virus vector expressing codon-optimized human acid alpha-glucosidase (GAA) as potential gene therapy for Pompe disease. Patients diagnosed with late-onset Pompe disease （LOPD） who are ≥ 6 years old will be studied.
China Post-approval Commitment (PAC) Study of Avalglucosidase Alfa in Participants With IOPD
This is a single group, 52-week treatment, Phase 4, open-label, single-arm study to assess the safety and efficacy of avalglucosidase alfa IV infusion in male and female Chinese participants with IOPD who are treatment-naïve or were previously treated with ERT. Study details include: The study duration: total study duration is …
Transcriptomic Analysis to Put an End to Misdiagnosis in Patients With Rare Muscle Diseases
Since 2017, more than 250 analyses performed at the Molecular Genetics Laboratory of the Timone Enfant Hospital have yielded negative results in patients with rare genetic muscle diseases. The researchers hypothesise that some of these misdiagnosed patients carry pathogenic RNA (transcript) disrupting variants that were not identified by DNA sequencing. …
Clinical Exploration of Adeno-associated Virus (AAV) Expressing Human Acid Alpha- Glucosidase (GAA) Gene Therapy for Patients With Infantile-onset Pompe Disease
This study is being conducted to evaluate the safety and effectiveness of GC301 adeno-associated virus vector expressing codon-optimized human acid alpha-glucosidase (GAA) as potential gene therapy for Pompe disease. Patients diagnosed with infantile-onset Pompe disease who are younger than 6 months old will be studied.
Gene Therapy(FT-003) for Wet AMD
The goal of this clinical trial is to evaluate the safety, tolerability and efficacy of disposable vitreous injection of FT-003 in subjects with neovascular age-related Macular Degeneration (nAMD). The study was divided into two phases, Phase 1 dose escalation and Phase II dose expansion.
Active AMD Study to Improve Function in Veterans
Age Related Macular Degeneration (AMD) is the leading cause of vision loss among Veterans aged 50 years and older. AMD also adversely affects mortality, physical and cognitive functioning, and activities of daily living. These debilitations negatively impact quality of life for US Veterans. Unfortunately, there are no cures and few …
