Description Module
The Description Module contains narrative descriptions of the clinical trial, including a brief summary and detailed description. These descriptions provide important information about the study's purpose, methodology, and key details in language accessible to both researchers and the general public.
Description Module path is as follows:
Study -> Protocol Section -> Description Module
Description Module
Ignite Creation Date:
2025-12-25 @ 3:24 AM
Ignite Modification Date:
2025-12-25 @ 3:24 AM
NCT ID:
NCT04174105
Brief Summary:
This is a phase 1/2 open-label, ascending dose, multicenter clinical study to evaluate the safety and efficacy of AT845 in adult (aged ≥ 18 years) subjects, ambulatory or nonambulatory, with Late Onset Pompe Disease (LOPD).
Detailed Description:
This study (FORTIS) will evaluate the safety and efficacy of an investigational gene replacement therapy, AT845, in adult subjects with LOPD. Subjects will receive a single dose of AT845 delivered via intravenous (IV) infusion.
Up to 3 nominal dose levels of AT845 are planned to be evaluated in this study. A single AT845 administration via IV infusion is planned for each subject. The initial dosing cohort received a single dose of 3x10\^13 vg/kg of AT845. The second dose cohort will receive a single dose of 6×10\^13 vg/kg. The third dose cohort will receive a single dose of 1×10\^14 vg/kg. Dose escalation between cohorts will be based on evaluations of safety and in consultation with the independent DMC.
There will be a core observation period of 48 weeks with scheduled visits and assessments. Following the conclusion of the core observation period, subjects will be seen every 6 months for a safety follow-up visit for up to 5 years postdose.
Study:
NCT04174105
Study Brief:
Protocol Section:
NCT04174105