NCT04418414

Hematopoietic Stem Cell Transplantation Gene Therapy for Treatment of Severe Hemophilia A

ET3-201: Phase 1 Study of Hematopoietic Stem Cell Transplantation (HSCT) Gene Therapy Incorporating a Lentiviral Vector (LV) Encoding a High Expressing Factor VIII Transgene for Treatment of Severe Hemophilia A

• Status: Not Yet Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 7 (estimated)
• Sponsor: Expression Therapeutics, LLC · Industry
• Sex: Male
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This is a first-in-human, non-randomized, open label, single treatment, Phase 1 study in approximately 7 patients with severe hemophilia A. The study will evaluate gene therapy by transplantation of autologous CD34+ hematopoietic stem cells transduced ex vivo with the CD68-ET3 lentiviral vector.

Detailed description

Eligible subjects will undergo CD34+ hematopoietic stem cell collection. These cells will be transduced ex vivo with CD68-ET3 lentiviral vector and subsequently, following a conditioning regimen of busulfan and anti-thymocyte globulin, the transduced cells will be infused to patients. After completion of study treatment, patients are followed up periodically for up to 15 years.

Conditions

• Hemophilia A

Interventions

Type	Name	Description
DRUG	Gene therapy	CD34+ hematopoietic stem cells transduced with CD68-ET3 lentiviral vector (encoding human factor VIII gene) is administered by IV infusion following conditioning regimen with busulfan and anti-thymocyte globulin.
OTHER	Biological	G-CSF and Plerixafor are administered by subcutaneous injection prior to apheresis.

Timeline

• Start date: 2024-09-01
• Primary completion: 2029-08-01
• Completion: 2039-08-01
• First posted: 2020-06-05
• Last updated: 2024-02-20

Regulatory

• FDA-regulated drug study