CHOP-ALS20 Lentiviral gene Therapy for Beta Thalassemia

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This clinical trial aims to test a new gene therapy in adults (18 to 35 years old) with beta thalassemia who require regular red cell transfusions. The study will evaluate the safety and efficacy of genetically engineered blood stem cells using a novel lentiviral vector that produces a high levels of a healthy beta globin. Several tests and procedures such as review of medical history, blood and urine tests, bone marrow aspirate/biopsy, heart ultrasound, electrocardiogram, magnetic resonance imaging (MRI) of the heart and liver, and lung tests will be done to find out if subjects are eligible to participate in this trial. If eligible, blood stem cells will be collected by apheresis. These stem cells will then have a healthy beta globin gene inserted. Subjects will then be hospitalized, and chemotherapy will be given to empty out the bone marrow. The stem cells, with the inserted gene (CHOP-ALS20), will then be given back to the subject. Subjects will be followed for side effects and to see if the inserted gene helps make hemoglobin and reduce or stop blood transfusions.

Who Do I Contact?

If you are interested in participating in the study or want to learn more please contact our study team at PatelJ23 [at] chop.edu or 267-426-5602.

Eligibility & Criteria

IRB #:
22-020309
Official Title:
Phase 1/2 study evaluating the safety and efficacy of gene therapy employing the lentiviral vector ALS20- transduced hematopoietic progenitor cells in subjects with transfusion-dependent beta thalassemia.
Study Phase:
Phase I
Phase II
Eligible Age Range:
18 - 35 Years
Gender:
All
Study Categories:

Visit Criteria

There are 4 main parts to the study:

  1. Screening period to see if a subject is eligible for the study
  2. Collection of the blood stem cells
  3. Gene transfer to the blood stem cells, conditioning chemotherapy and infusion of modified blood stem cells and,
  4. Follow up. 

Screening may last up to three months, while mobilization, apheresis, and drug product manufacturing is estimated to take between 3-9 months, conditioning chemotherapy, drug product infusion, and inpatient follow up is expected to take around two months and follow up after infusion of the stem cells will last for two years.