Actively Recruiting
Phase 1/2 Study Evaluating Safety and Efficacy of ALS20 Lentiviral Gene Therapy in Adults with Transfusion-Dependent Beta Thalassemia
Led by Children's Hospital of Philadelphia · Updated on 2026-04-15
12
Participants Needed
1
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are studying transfusion-dependent beta thalassemia, a hereditary blood disorder that requires lifelong blood transfusions and leads to serious health issues and reduced quality of life. The trial aims to evaluate the safety and effectiveness of a new gene therapy using autologous blood stem cells modified with a lentiviral vector called ALS20, which carries a healthy beta globin gene. This gene therapy is experimental and has not been approved by the FDA. The study involves collecting hematopoietic stem and progenitor cells from participants, modifying them with the ALS20 lentiviral vector, and then infusing these cells back after the participant undergoes myeloablative conditioning with busulfan chemotherapy. This single-arm pilot trial will include up to 12 subjects aged 18 to less than 36 years with transfusion-dependent beta thalassemia. The treatment is designed to reduce the need for blood transfusions by increasing healthy hemoglobin production. Participants will be closely monitored with tests measuring blood cell recovery, survival, transplant complications such as graft versus host disease, and possible genetic risks related to the therapy over a follow-up period of up to 24 months. Researchers will track neutrophil and platelet engraftment within the first 42 days to one year after infusion and assess hemoglobin levels to evaluate treatment impact. Safety assessments include monitoring for infections, liver function, and blood cancer risks. The total study duration spans from treatment through long-term observation.
CONDITIONS
Brief Title
ALS20-101 Lentiviral Gene Therapy for Beta Thalassemia
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age 18 to less than 40 years at the time of consent
- Diagnosis of transfusion-dependent beta thalassemia with specific genotypes
- Genetic confirmation of alpha and beta thalassemia by a certified laboratory
- Clinically stable with Karnofsky score of at least 70 and eligible for stem cell transplant
- Females of childbearing potential must use contraception from consent through 6 months after infusion
- Males of reproductive capacity must use contraception from start of mobilization through 6 months after infusion
- Discussion and agreement to proceed after reviewing all treatment options including stem cell transplant and approved gene therapies
You will not qualify if you...
- Prior receipt of stem cell transplant or gene therapy
- More than one alpha globin gene deletion or mutation
- Any prior or current cancer except certain treated skin cancers
- Known cancer predisposition syndrome
- Positive for HIV, HTLV, or active hepatitis B or C infection
- Active significant infections including COVID-19 or influenza
- Significant bleeding disorders
- Evidence of cardiac dysfunction or arrhythmia
- Advanced liver disease or abnormal liver tests not due to iron therapy
- High liver iron concentration
- Lung diffusion capacity less than 50%
- Low oxygen saturation in room air
- Evidence of kidney dysfunction
- Cardiac MRI T2 less than 10 ms
- Low platelet or neutrophil counts except benign ethnic neutropenia
- Unable to receive red cell transfusions
- Uncontrolled high blood pressure or seizure disorder
- Significant psychiatric disorder affecting participation
- Immediate family history of familial cancer syndrome
- Contraindication to anesthesia
- Pregnancy or breastfeeding
- Participation in another investigational drug trial within 30 days or five drug half-lives
- Other conditions making mobilization or transplant unsafe as judged by investigator
AI-Screening
AI-Powered Screening
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Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Until stem cell engraftment, approximately 1 year of active treatment and monitoring
Participants receive myeloablative conditioning with busulfan followed by infusion of their own hematopoietic stem and progenitor cells transduced with the ALS20 lentiviral vector.
Multiple visits during conditioning and infusion, followed by weekly visits for up to 12 weeks and additional visits through 1 year
Duration - Up to 2 years after treatment ends
Participants are monitored for safety and efficacy outcomes including blood counts, survival, and potential complications for up to 2 years after treatment.
Periodic visits over 2 years for long-term safety and efficacy monitoring
Trial Site Locations
Total: 1 location
1
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
Research Team
J
Janet Kwiatkowski, MD
J
Jaladhikumar Patel
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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