Ignite Creation Date:
2024-05-06 @ 8:28 PM
Last Modification Date:
2024-10-26 @ 3:28 PM
Study NCT ID:
NCT06399107
Status:
RECRUITING
Last Update Posted:
2024-05-03
First Post:
2024-04-29
Brief Title:
Investigation Into the Use of BAH243 Lentiviral Vector for Gene Therapy in Treating Sickle Cell Disease
Sponsor:
Essen Biotech
Organization:
Essen Biotech
Study Overview
Official Title:
Lentiviral Vector Gene Therapy in Sickle Cell Disease Using Autologous CD34 Hematopoietic Stem Cells Collected Via Apheresis and Modified With a Lentiviral Vector
Status:
RECRUITING
Status Verified Date:
2024-08
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:
BAH243
Brief Summary:
This study is an open-label non-randomized single-dose Phase 12 trial involving around 85 adult and pediatric participants aged between 2 and 50 years with sickle cell disease SCD It aims to assess the effectiveness of hematopoietic stem cell transplantation HSCT using BAH243 for SCD
Detailed Description:
Participants in this study will be involved for two years following their transplant Those who join will also be invited to partake in a subsequent long-term follow-up study which will continue to evaluate the safety and effectiveness of the treatment for an additional 13 years culminating in a total of 15 years of observation post-infusion of the drug product This clinical trial is structured as a single-arm single-dose single-center open-label study without any dose escalation involved Its main goal is to investigate the safety of the study drug in treating sickle cell disease SCD The treatment regimen including myeloablative conditioning will only commence for subsequent participants once the initial participant has successfully completed dosing and undergone a safety evaluation
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