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NCT06364774 · ClinicalTrials.gov registry record · Phase 1
ALS20-101 Lentiviral Gene Therapy for Beta Thalassemia
A Phase 1 study of Beta-Thalassemia, sponsored by Children's Hospital of Philadelphia.
- Recruiting
- Registry status
- Phase 1
- Development phase
- 12
- Enrollment target
- 1
- Study location
NCT06364774: Recruiting Phase 1 study of Beta-Thalassemia, sponsored by Children's Hospital of Philadelphia.
NCT06364774 is a Phase 1 study of Beta-Thalassemia that is actively recruiting participants, run by Children's Hospital of Philadelphia. The registered enrollment target is 12 participants, below the 180-participant average among 14 other Beta-Thalassemia trials with a reported enrollment target (93% lower). The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT06364774, a Phase 1 study of Beta-Thalassemia, is actively recruiting participants, sponsored by Children's Hospital of Philadelphia.
- RECRUITING
- Registry status
- Phase 1
- Development phase
- 12 participants
- Enrollment target
- 1
- Study location
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.
Primary Outcome
time to neutrophil engraftment
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 |
What NCT06364774 shows while recruiting
NCT06364774 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 12 participants, a relatively small participant target, below the 180-participant average among 14 other Beta-Thalassemia trials with a reported enrollment target (93% lower).
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.
NCT06364774 reports a single indexed study location in Pennsylvania.
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.
What interventions are being tested in trial NCT06364774?
The interventions under investigation include: ALS20 (BIOLOGICAL).
Who is sponsoring clinical trial NCT06364774?
This trial is sponsored by Children's Hospital of Philadelphia, which has 431 total clinical trials registered on ClinicalTrials.gov.
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.
Learn More About Clinical Trials
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Where NCT06364774's enrollment target sits among peer trials
12 14th of 14 higher than 1 of 14 other Beta-Thalassemia trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Beta-Thalassemia trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
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