Ignite Creation Date:
2024-05-06 @ 5:15 PM
Last Modification Date:
2024-10-26 @ 2:25 PM
Study NCT ID:
NCT05241444
Status:
RECRUITING
Last Update Posted:
2024-07-08
First Post:
2022-01-12
Brief Title:
CD4LVFOXP3 in Participants With IPEX
Sponsor:
Bacchetta Rosa MD
Organization:
Stanford University
Study Overview
Official Title:
Phase 1 Study of Autologous CD4LVFOXP3 in Participants With Immune Dysregulation Polyendocrinopathy Enteropathy X-linked IPEX Syndrome
Status:
RECRUITING
Status Verified Date:
2024-07
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
This first-in-human Phase 1 clinical trial will test the feasibility of the manufacturing and the safety of the administration of CD4LVFOXP3 in up to 30 evaluable human participants with IPEX and evaluate the impact of the CD4LVFOXP3 infusion on the disease
Detailed Description:
Treatment with CD4LVFOXP3 is expected to replace the defective Treg cells of the participants and restore control of the immune system and therefore ameliorate symptoms of IPEX
We expect to learn the following from this study
1 That CD4LVFOXP3 can be consistently produced and be of expected quality to be used in humans
2 That CD4LVFOXP3 are safe in children and young adults with IPEX and determine its effects both good and bad
3 That CD4LVFOXP3 can improve overall health and allow reduction of medications
This Phase 1 feasibility and safety trial will gather data about CD4LVFOXP3 in vivo persistency and early signs of impact on symptoms of IPEX
We expect to learn the following from this study
1 That CD4LVFOXP3 can be consistently produced and be of expected quality to be used in humans
2 That CD4LVFOXP3 are safe in children and young adults with IPEX and determine its effects both good and bad
3 That CD4LVFOXP3 can improve overall health and allow reduction of medications
This Phase 1 feasibility and safety trial will gather data about CD4LVFOXP3 in vivo persistency and early signs of impact on symptoms of IPEX
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| CLIN2-13259 | OTHER_GRANT | California Institute of Regenerative Medicine CIRM | httpsreporternihgovquickSearch1R01FD007540-01 |
| DRU-2020-7764 | OTHER | None | None |
| RPD-2020-470 | OTHER | None | None |
| 1R01FD007540-01 | FDA | None | None |