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 @ 3:28 AM
Ignite Modification Date:
2025-12-25 @ 3:28 AM
NCT ID:
NCT05224505
Brief Summary:
Phase 1 dose escalation study to assess tolerability and safety of ATA-100 with 5-year follow-up
Detailed Description:
Multicenter, Phase 1 study evaluating safety, pharmacodynamic, and immunogenicity of ATA-100, an Adeno-Associated Virus (AAV) vector carrying the human FKRP transgene.
This study is an open-label dose escalation phase with long-term follow-up (LTFU) period.
Two dose cohorts will be enrolled sequentially and enrollment. An initial cohort of three (3) patients will receive a potentially effective dose, followed by a 2nd higher dose cohort of 3 patients.
All subjects will be followed for up to 5 years after active IMP (ATA-100) administration.
Study:
NCT05224505
Study Brief:
Protocol Section:
NCT05224505