RecruitingPhase 1Phase 2NCT06364774

ALS20-101 Lentiviral Gene Therapy for Beta Thalassemia

Phase 1/2 Study Evaluating the Safety and Efficacy of Gene Therapy Employing Lentiviral Vector ALS20-transduced Hematopoietic Progenitor Cells in Subjects With Transfusion-dependent-thalassemia

Sponsor

Children's Hospital of Philadelphia

Enrollment

12 participants

Start Date

Apr 14, 2025

Study Type

INTERVENTIONAL

Conditions

Beta-Thalassemia

Summary

The main goal of this study is to find out if the blood disorder called transfusion-dependent beta thalassemia can be safely treated by modifying blood stem cells. This is done by collecting blood stem cells from the subject, modifying those cells, adding a healthy beta globin gene, and then giving them back to the subject. It is hoped that these modified cells will decrease the need for blood transfusions. The gene modified blood stem cells are called CHOP-ALS20 ("study drug"). This experimental gene therapy has not been tried on human beings before and is not FDA approved.

Eligibility

Min Age: 18 YearsMax Age: 40 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying a biological treatment called ALS20 for people with beta-thalassemia. The study is currently recruiting participants at 1 location. People eligible for this study include aged 18 Years to 40 Years.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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