Actively Recruiting
ALS20-101 Lentiviral Gene Therapy for Beta Thalassemia
Led by Children's Hospital of Philadelphia · Updated on 2026-04-15
12
Participants Needed
1
Research Sites
141 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
The main goal of this study is to find out if the blood disorder called transfusion-dependent beta thalassemia can be safely treated by modifying blood stem cells. This is done by collecting blood stem cells from the subject, modifying those cells, adding a healthy beta globin gene, and then giving them back to the subject. It is hoped that these modified cells will decrease the need for blood transfusions. The gene modified blood stem cells are called CHOP-ALS20 ("study drug"). This experimental gene therapy has not been tried on human beings before and is not FDA approved.
CONDITIONS
Official 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 time of consent
- Diagnosis of transfusion-dependent beta thalassemia with specific genotypes
- History of at least 120 mL/kg/year or 8 transfusions per year over past two years
- Genetic confirmation of alpha and beta thalassemia diagnosis by a certified laboratory
- Clinically stable with Karnofsky score of at least 70 and eligible for stem cell transplant
- Female participants of childbearing potential must agree to use contraception from consent through 6 months post-infusion
- Male participants of reproductive capacity must agree to use contraception from start of mobilization through 6 months post-infusion
- Thorough discussion of all treatment options including stem cell transplant and FDA approved gene therapies with a hematologist or transplant physician and agreement to proceed with this trial
You will not qualify if you...
- Prior hematopoietic stem cell transplant or gene therapy
- More than one alpha globin gene deletion or mutation
- Current or prior malignancy except treated basal or squamous cell skin cancer
- Known cancer predisposition syndrome
- Positive for HIV-1, HIV-2, HTLV-1, HTLV-2, or active hepatitis B or C infection
- Clinically significant active infections including COVID-19 or influenza
- Significant bleeding disorder
- Heart dysfunction or significant arrhythmia
- Advanced liver disease or significant liver iron overload
- Lung diffusion capacity less than 50% predicted
- Pulse oximetry below 92% in room air
- Kidney dysfunction based on creatinine or glomerular filtration rate
- Cardiac T2 MRI less than 10 ms
- Low platelet or neutrophil counts except benign ethnic neutropenia
- Unable to receive red blood cell transfusion
- Uncontrolled high blood pressure or seizure disorder
- Significant psychiatric disorders impacting participation
- Immediate family history of familial cancer syndrome
- Contraindication to anesthesia
- Pregnancy or breastfeeding for female participants
- Participation in another investigational drug trial within 30 days or 5 drug half-lives
- Any other condition making participant ineligible for mobilization, apheresis, or stem cell transplant as determined by investigator
AI-Screening
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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
CONTACT
J
Jaladhikumar Patel
CONTACT
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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