Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe β-Hemoglobinopathies
Public ClinicalTrials.gov record NCT06647979. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
An Adaptive Design Basket Trial of Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe β-Hemoglobinopathies
Study identification
- NCT ID
- NCT06647979
- Recruitment status
- Recruiting
- Study type
- Interventional
- Phase
- Phase 1
- Lead sponsor
- Daniel Bauer
- Other
- Enrollment
- 10 participants
Conditions and interventions
Conditions
Interventions
- Sequencing Assay for Variant rs114518452 Device
- autologous bone marrow derived CD34+ HSPCs electroporated with BCL11A enhancer targeting Cas9 ribonucleoprotein Biological
Device · Biological
Eligibility (public fields only)
- Age range
- 13 Years to 40 Years
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Nov 30, 2025
- Primary completion
- Nov 30, 2028
- Completion
- Nov 30, 2030
- Last update posted
- Feb 2, 2026
2025 – 2030
United States locations
- U.S. sites
- 1
- U.S. states
- 1
- U.S. cities
- 1
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Boston Children's Hospital | Boston | Massachusetts | 02115 | Recruiting |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT06647979, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Feb 2, 2026 · Synced May 16, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT06647979 live on ClinicalTrials.gov.