Your session is about to expire
← Back to Search
Gene Therapy
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
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 moreAwards & 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.
Find a Location
Who is running the clinical trial?
Children's Hospital of PhiladelphiaLead Sponsor
731 Previous Clinical Trials
8,472,822 Total Patients Enrolled
Janet Kwiatkowski, MDPrincipal InvestigatorChildren's Hospital of Philadelphia
6 Previous Clinical Trials
1,091 Total Patients Enrolled