ALS20-101 Lentiviral Gene Therapy for Beta Thalassemia
This study is testing a new gene therapy called ALS20 for people with transfusion-dependent beta thalassemia. This condition requires regular blood transfusions. Researchers will collect your blood stem cells, add a healthy beta globin gene to them, and then give these modified cells (ALS20) back to you. The main goal is to see if ALS20 is safe and if it can reduce your need for blood transfusions. This is the first time ALS20 is being tested in humans and it is not yet approved by the FDA. You may be able to join if you are between 18 and 40 years old and have transfusion-dependent beta thalassemia. The study will enroll up to 12 participants.
- Study design
- This is a single-arm study, meaning all participants will receive the ALS20 treatment. It is a pilot Phase 1/2 study and plans to enroll up to 12 participants.
- What's involved
- Not specified in the trial record.
- Compensation
- Not stated in the trial record.
- Follow-up
- You will be followed for platelet engraftment through the end of treatment (an average of 1 year), and for overall survival for 2 years after treatment ends.
AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.
ALS20-101 Lentiviral Gene Therapy for Beta Thalassemia
At a glance
Conditions
Where it's being run
1 sites across 1 statesStudy leadership
- Janet Kwiatkowski, MD · PRINCIPAL_INVESTIGATOR · Children's Hospital of Philadelphia
Who to contact
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What this trial measures
- Neutrophil Engraftmentwithin 42 days after infusion
time to neutrophil engraftment
- Platelet Engraftmentthrough end of treatment, an average 1 year
time to platelet engraftment
- Overall Survival at 2 years2 years after treatment ends
Survival status after treatment ends
- Incidence of transplant related mortality1 year after infusion
Incidence of transplant related mortality within 100 days and within 1 year after infusion
- Incidence of Graft Versus Host Diseasethrough end of treatment, an average of 1 year
any clinical evidence of graft versus host disease (GVHD)
- Incidence of Vector-Derived Replication Competent Lentivirusthrough end of treatment, an average of 1 year
The detection of vector-derived replication competent lentivirus in any subject throughout the study until end of treatment.
- Insertional Oncogenesisthrough the end of the study, up to 24 months
The number of subjects with insertional oncogenesis
- Clonal Predominancethrough the end of the study, up to 24 months
The number of subjects with clonal predominance
- maintain total hemoglobin level of 9.0 g/dL or higherthrough the end of the study, up to 24 months
The proportion of subjects able to discontinue regular red cell transfusions and maintain total hemoglobin level of 9.0 g/dL or higher (average over 1-year period) in the absence of red cell transfusion(transfusion independence). Success is defined as a minimum of 4 to 6 subjects achieving this endpoint.