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ALS20-101 Lentiviral Gene Therapy for Beta Thalassemia
NCT06364774 · View on ClinicalTrials.gov ↗
Study Summary
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 Studied
Interventions
- BIOLOGICAL ALS20
Study Locations (1)
Pennsylvania
- Children's Hospital of Philadelphia — Philadelphia
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 12 participants |
| Start Date | 2025-04-14 |
| Est. Completion | 2027-12-31 |
| Phase | Phase 1 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT06364774
The ClinicalTrials.gov registry entry for NCT06364774 describes a study currently listed as recruiting. It is categorized as Phase 1, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 12 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Children's Hospital of Philadelphia, which has 492 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 1 condition, with Beta-Thalassemia appearing as the primary indexed condition, and to 1 intervention — of which ALS20 is the first listed. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT06364774 reports 1 study location spanning 1 distinct geographic area — top geographies include Pennsylvania. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
Frequently Asked Questions
What is clinical trial NCT06364774 about?
NCT06364774 is a clinical study titled "ALS20-101 Lentiviral Gene Therapy for Beta Thalassemia". 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 ...
What is the current status of trial NCT06364774?
This trial is currently recruiting. It is a Phase 1 study. The enrollment target is 12 participants. The study started on 2025-04-14. Estimated completion is 2027-12-31.
What conditions does trial NCT06364774 study?
This clinical trial studies the following conditions: Beta-Thalassemia. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT06364774?
The interventions under investigation include: ALS20 (BIOLOGICAL). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT06364774?
This trial is sponsored by Children's Hospital of Philadelphia, which has 492 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT06364774 being conducted?
This trial has 1 study location across Pennsylvania. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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