Clinical Trial

Investigation Into the Use of BAH243 Lentiviral Vector for Gene Therapy in Treating Sickle Cell Disease

Study acronym: BAH243
Recruiting Phase 1/2
View on ClinicalTrials.gov →
Record status
This record was last updated November 5, 2024 (before its estimated November 10, 2025 completion). Its status may not reflect the trial's current state.
Summary
This study is an open-label, non-randomized, single-dose Phase 1/2 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.
Protocol Amendment History 2 amendments
This ClinicalTrials.gov record has been amended 2 times since 2024-05-01; most recent amendment 2024-11-04.
Status change: Not Yet Recruiting → Recruiting 2024-08-06
Trial Details
NCT Number NCT06399107
Lead Sponsor Essen Biotech
Conditions Sickle Cell Disease, Sickle-Cell Disease With Crisis
Enrollment 85 participants
Start Date 2024-08-01
Primary Completion 2025-11-10 (estimated)
Study Completion 2025-12-28 (estimated)
Updated on ClinicalTrials.gov 2024-11-05