Participant Flow Module
The Participant Flow Module provides information about the flow of participants through each stage of the clinical trial. This includes pre-assignment details, recruitment information, and the types of units analyzed, helping researchers understand how participants move through the study.
Participant Flow Module path is as follows:
Study -> Results Section -> Participant Flow Module
Participant Flow Module
Ignite Creation Date:
2025-12-24 @ 10:42 PM
Ignite Modification Date:
2025-12-25 @ 8:12 PM
NCT ID:
NCT04669535
Pre Assignment Details:
The study was initially designed as a 2-stage study with dose escalation in stage 1 and safety and efficacy in stage 2, with the optimal dose identified in stage 1 however due to lack of funding only stage 1 was completed in this study. No subjects were enrolled in stage 2.
Recruitment Details:
This study is a dose-escalation study in children affected with GM2 gangliosidosis who were treated in four cohorts at dosage levels ranging from 1.42E+14 to 3.56E+14 vg per patient. All subjects are treated with a 1:1 ratio of AAVrh8-HEXA and AAVrh8-HEXB, administered via bilateral thalamic (BiTh) and dual intracisterna magna (ICM)/intrathecal (IT) administration into the cerebrospinal fluid (CSF). The BiTh injection volume and vector dose was doubled between each cohort.
Study:
NCT04669535
Study Brief:
Results Section:
NCT04669535