ALS20-101 Lentiviral Gene Therapy for Beta Thalassemia

Part of paid clinical trials in Philadelphia, Pennsylvania.

Sponsor
Children's Hospital of Philadelphia
Study ID
NCT06364774
Phase
PHASE1/PHASE2
Status
Recruiting
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Conditions

  • Beta-Thalassemia

Eligibility Criteria

Sex
ALL
Age
18 Years - 40 Years
Healthy Volunteers
Not accepted

Interventions

  • ALS20 — BIOLOGICAL
    novel lentiviral vector ALS20

Study Details

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.

Key Dates

Start date
Apr 14, 2025
Status verified
Apr 2026
Primary completion
Dec 31, 2027
Completion
Dec 31, 2027

Study Design

Enrollment
12 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: beta thalassemia
    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.

Primary Outcome Measure

Neutrophil Engraftment [ Time Frame: within 42 days after infusion ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
Children's Hospital of PhiladelphiaPhiladelphiaPennsylvania19104
Janet Kwiatkowski, MD
[email protected]
215-590-5286
Jaladhikumar Patel
[email protected]
267-426-5602
Janet Kwiatkowski, MD (PRINCIPAL_INVESTIGATOR)

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Children's Hospital of Philadelphia· Philadelphia, PA

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