Gene Therapy for Beta Thalassemia

Phase 1 & 2

Recruiting

Led By Janet Kwiatkowski, MD

Research Sponsored by Children's Hospital of Philadelphia

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be between 18 and 65 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 1 year after infusion

Awards & highlights

No Placebo-Only Group

Summary

This trial aims to investigate whether a new treatment method can safely help patients with a blood disorder called transfusion-dependent beta thalassemia. The treatment involves modifying blood stem cells by adding a healthy

Who is the study for?

This trial is for individuals with transfusion-dependent beta thalassemia, a blood disorder. Participants must have a history of needing regular blood transfusions. Specific eligibility criteria are not provided, but typically include factors like age range, overall health status, and the severity of the condition.

What is being tested?

The study is testing an experimental gene therapy called CHOP-ALS20 (study drug) to treat beta thalassemia by modifying patients' own blood stem cells with a healthy beta globin gene and returning them to the patient's body.

What are the potential side effects?

Since this is the first time CHOP-ALS20 is being tested in humans, potential side effects are unknown. However, common risks may include immune reactions, infection risk from procedures, or complications related to stem cell transplantation.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 1 year after infusion

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~1 year after infusion

This trial's timeline: 3 weeks for screening, Varies for treatment, and 1 year after infusion for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Clonal Predominance

Incidence of Graft Versus Host Disease

Incidence of Vector-Derived Replication Competent Lentivirus

+6 more

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: beta thalassemiaExperimental Treatment1 Intervention

This arm will evaluate the safety and efficacy of infusing autologous hematopoietic stem and progenitor cells (HSPC) transduced with the novel lentiviral vector ALS20 that encodes the human βA-T87Q-globin gene, following myeloablative conditioning with busulfan.

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