NCT06399107

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

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
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 85 (estimated)
• Sponsor: Essen Biotech · Academic / Other
• Sex: All
• Age: 2 Years – 90 Years
• Healthy volunteers: Not accepted

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.

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.

Conditions

• Sickle Cell Disease
• Sickle-Cell Disease With Crisis

Interventions

Type	Name	Description
GENETIC	Drug Product is administered by IV infusion following myeloablative conditioning with busulfan	An autologous CD34+ cell-enriched population from patients with sickle cell disease (SCD), which includes hematopoietic stem cells (HSCs) that have been transduced with the BAH243 lentiviral vector (LVV) carrying the βA-T87Q-globin gene, is preserved in a cryopreservation solution.

Timeline

• Start date: 2024-08-01
• Primary completion: 2025-11-10
• Completion: 2025-12-28
• First posted: 2024-05-03
• Last updated: 2024-11-05

Locations

1 site across 1 country: China