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NCT06647979 · ClinicalTrials.gov registry record · Phase 1
Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe β-Hemoglobinopathies
A Phase 1 study of Sickle Cell Disease and Beta-Thalassemia, sponsored by Daniel Bauer.
- Recruiting
- Registry status
- Phase 1
- Development phase
- 10
- Enrollment target
- 1
- Study location
NCT06647979: Recruiting Phase 1 study of Sickle Cell Disease and Beta-Thalassemia, sponsored by Daniel Bauer.
NCT06647979 is a Phase 1 study of Sickle Cell Disease and Beta-Thalassemia that is actively recruiting participants, run by Daniel Bauer. The registered enrollment target is 10 participants, below the 293-participant average among 213 other Sickle Cell Disease trials with a reported enrollment target (97% lower). The trial reports 1 study location across 1 state. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT06647979, a Phase 1 study of Sickle Cell Disease and Beta-Thalassemia, is actively recruiting participants, sponsored by Daniel Bauer.
- RECRUITING
- Registry status
- Phase 1
- Development phase
- 10 participants
- Enrollment target
- 1
- Study location
Study Summary
A promising approach for the treatment of genetic diseases is called gene therapy. Gene therapy is a relatively new field of medicine that uses genetic material (mostly DNA) from the patient to treat his or her own disease. In gene therapy, the investigators introduce new genetic material in order to fix or replace a diseased gene, with the goal of curing the disease. The procedure is similar to a bone marrow transplant, in that the patient's malfunctioning blood stem cells are reduced or eliminated using chemotherapy, but it is different because instead of using a different person's (donor) blood stem cells for the transplant, the patient's own blood stem cells are given back after the new genetic material has been introduced into those cells. This approach has the advantage of eliminating any risk of Graft-Versus-Host Disease (GVHD), reducing the risk of graft rejection, and may also allow less chemotherapy to be utilized for the conditioning portion of the transplant procedure. The method used to fix or replace a diseased gene is called gene editing. A person's own cells are edited using a specialized biological medicine that has been formulated for use in human beings. Fetal hemoglobin (HbF) is a healthy, non-sickling kind of hemoglobin. Investigators have recently discovered a gene called BCL11A that is very important in the control of fetal hemoglobin expression. Increasing the expression of this gene in sickle cell patients could increase the amount of fetal hemoglobin while simultaneously reducing the amount of sickle hemoglobin in their blood, and therefore potentially cure the condition.
Primary Outcome
Successful hematopoietic reconstitution after conditioning (defined by absolute neutrophil count (ANC) greater than or equal to 0.5 x 10\^9 /L for three consecutive days without growth factor support), achieved by day 42 after day 0 of stem cell infusion (i.e., "primary engraftment").
Conditions Studied
Interventions
- BIOLOGICAL autologous bone marrow derived CD34+ HSPCs electroporated with BCL11A enhancer targeting Cas9 ribonucleoprotein
- DEVICE Sequencing Assay for Variant rs114518452
Study Locations (1)
Massachusetts
- Boston Children's Hospital - Boston
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 10 participants |
| Start Date | 2025-12-01 |
| Est. Completion | 2030-12 |
| Phase | Phase 1 |
What NCT06647979 shows while recruiting
NCT06647979 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 10 participants, a relatively small participant target, below the 293-participant average among 213 other Sickle Cell Disease trials with a reported enrollment target (97% lower).
The record links to 4 conditions, with Sickle Cell Disease appearing as the primary indexed condition, and to 2 interventions - of which autologous bone marrow derived CD34+ HSPCs electroporated with BCL11A enhancer targeting Cas9 ribonucleoprotein is the first listed.
NCT06647979 reports a single indexed study location in Massachusetts.
Frequently Asked Questions
What is clinical trial NCT06647979 about?
NCT06647979 is a clinical study titled "Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe β-Hemoglobinopathies". A promising approach for the treatment of genetic diseases is called gene therapy. Gene therapy is a relatively new field of medicine that uses genetic material (mostly DNA) from the patient to treat his or her own disease. In gene therapy, the investigators introduce new genetic material in order t...
What is the current status of trial NCT06647979?
This trial is currently recruiting. It is a Phase 1 study. The enrollment target is 10 participants. The study started on 2025-12-01. Estimated completion is 2030-12.
What conditions does trial NCT06647979 study?
This clinical trial studies the following conditions: Sickle Cell Disease, Beta-Thalassemia, Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0), Transfusion Dependent Beta-Thalassaemia.
What interventions are being tested in trial NCT06647979?
The interventions under investigation include: autologous bone marrow derived CD34+ HSPCs electroporated with BCL11A enhancer targeting Cas9 ribonucleoprotein (BIOLOGICAL), Sequencing Assay for Variant rs114518452 (DEVICE).
Who is sponsoring clinical trial NCT06647979?
This trial is sponsored by Daniel Bauer, which has 1 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT06647979 being conducted?
This trial has 1 study location across Massachusetts. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
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Where NCT06647979's enrollment target sits among peer trials
10 196th of 213 higher than 16 of 213 other Sickle Cell Disease trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Sickle Cell Disease 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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