Ignite Creation Date:
2024-05-06 @ 12:28 PM
Last Modification Date:
2024-10-26 @ 12:59 PM
Study NCT ID:
NCT03765632
Status:
COMPLETED
Last Update Posted:
2023-02-01
First Post:
2018-08-08
Brief Title:
Efficacy and Safety of the Cryopreserved Formulation of OTL-101 in Subjects With ADA-SCID
Sponsor:
Great Ormond Street Hospital for Children NHS Foundation Trust
Organization:
Great Ormond Street Hospital for Children NHS Foundation Trust
Study Overview
Official Title:
Efficacy and Safety of a Cryopreserved Formulation of Autologous CD34 Haematopoietic Stem Cells Transduced ex Vivo With Elongation Factor 1α Short Form EFS Lentiviral Vector Encoding for Human ADA Gene in Subjects With Severe Combined Immunodeficiency SCID Due to Adenosine Deaminase Deficiency
Status:
COMPLETED
Status Verified Date:
2023-01
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 is a prospective non-randomized single-cohort longitudinal single-center clinical study designed to assess the efficacy and safety of a cryopreserved formulation of OTL-101 autologous CD34 hematopoietic stemprogenitor cells transduced ex vivo with EFS Elongation Factor 1α Short form Lentiviral Vector LV encoding for the human ADA gene administered to ADA-SCID subjects between the ages of 30 days and 18 years of age who are not eligible for an Human Leukocyte Antigen HLA matched siblingfamily donor and meeting the inclusionexclusion criteria The OTL-101 product is infused after a minimal interval of at least 24 hours following the completion of reduced intensity conditioning For subjects who successfully receive the OTL-101 product pegademase bovine PEG-ADA Enzyme Replacement Therapy ERT is discontinued at Day30 -315 after the transplant After their discharge from hospital the subjects will be seen at regular intervals to review their history perform examinations and draw blood samples to assess immunity and safety
Detailed Description:
None
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
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 |
|---|---|---|---|
| 17IC04 | OTHER | UCL Great Ormond Street Institute of Child Health | None |