Ignite Creation Date:
2024-05-06 @ 12:44 PM
Last Modification Date:
2024-10-26 @ 1:03 PM
Study NCT ID:
NCT03837483
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2024-01-31
First Post:
2019-02-08
Brief Title:
A Clinical Study to Evaluate the Use of a Cryopreserved Formulation of OTL-103 in Subjects With Wiskott-Aldrich Syndrome
Sponsor:
Fondazione Telethon
Organization:
Fondazione Telethon
Study Overview
Official Title:
A Single Arm Open-label Clinical Trial of Hematopoietic Stem Cell Gene Therapy With Cryopreserved Autologous CD34 Cells Transduced With Lentiviral Vector Encoding WAS cDNA in Subjects With Wiskott-Aldrich Syndrome WAS
Status:
ACTIVE_NOT_RECRUITING
Status Verified Date:
2023-03
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
This is an open-label single arm study to evaluate the cryopreserved formulation of OTL-103 Gene Therapy OTL-103 consists of autologous CD34 hematopoietic stem cells in which the gene encoding for the Wiskott-Aldrich Syndrome is introduced by means of a third generation lentiviral vector
Detailed Description:
None
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 |
|---|---|---|---|
| 2018-003842-18 | EUDRACT_NUMBER | None | None |