ALS20-101 Lentiviral Gene Therapy for Beta Thalassemia
ConditionBeta-Thalassemia
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age18-40
SponsorChildren's Hospital of Philadelphia
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Age 18 to < 40 years at the time of consent
Diagnosis of transfusion dependent beta thalassemia (β0 β0, β+β0, β+β+, βEβ0, βEβ+,β0 or β+ /βA + alpha triplication(s)). Transfusion-dependent is defined as a history of receiving at least 120 mL/kg/year packed red blood cells or at least 8 transfusions per year in the past two years. The first 2 subjects enrolled must have a non- β0 β0 genotype.
Genetic confirmation of α and β thalassemia diagnosis (β0β0, β+β0, β+β+, βEβ0, βEβ+, β0 or β+ /βA + alpha triplication(s)) by a Clinical Laboratory Improvement Amendments (CLIA) certified laboratory is required.
Clinically stable, Karnofsky score at least 70, and eligible to undergo Hematopoietic Stem Cell Transplantation (HSCT).
Disqualifiers
Prior receipt of HSCT or gene therapy
More than one alpha globin gene deletions/mutations.
Any prior or current malignancy (excluding adequately treated basal or squamous cell carcinoma of the skin)
Known cancer predisposition syndrome
Trial design
Design model
Single group
Treatments tested in this trial
ALS20
Biological/Vaccinenovel lentiviral vector ALS20
Treatment groups
12 Participants
are divided into 1 treatment groupGroup A: beta thalassemiaExperimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
Neutrophil Engraftment
time to neutrophil engraftment
Time frame
within 42 days after infusion2
Platelet Engraftment
time to platelet engraftment
Time frame
through end of treatment, an average 1 year3
Overall Survival at 2 years
Survival status after treatment ends
Time frame
2 years after treatment ends4
Incidence of transplant related mortality
Incidence of transplant related mortality within 100 days and within 1 year after infusion
Time frame
1 year after infusionSponsors and contacts
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Source ClinicalTrials.gov