Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe β-Hemoglobinopathies

Part of paid clinical trials in Boston, Massachusetts.

Sponsor
Daniel Bauer
Study ID
NCT06647979
Phase
PHASE1
Status
Recruiting
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Conditions

  • Beta-Thalassemia
  • Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0)
  • Sickle Cell Disease
  • Transfusion Dependent Beta-Thalassaemia

Eligibility Criteria

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

Interventions

  • autologous bone marrow derived CD34+ HSPCs electroporated with BCL11A enhancer targeting Cas9 ribonucleoprotein — BIOLOGICAL
    autologous bone marrow derived CD34+ HSPCs electroporated with BCL11A enhancer targeting Cas9 ribonucleoprotein
  • Sequencing Assay for Variant rs114518452 — DEVICE
    Device used to carry out the diagnostic testing for exclusion criteria number 12

Study Details

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.

Key Dates

Start date
Dec 1, 2025
Status verified
Jan 2026
Primary completion
Dec 31, 2028
Completion
Dec 31, 2030

Study Design

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

Arms

  • Experimental: Sickle Cell Disease and Transfusion-Dependent Beta-Thalassemia

Primary Outcome Measure

Primary engraftment [ Time Frame: 42 days ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
Boston Children's HospitalBostonMassachusetts02115
Emily Morris
[email protected]
617-355-8724

Find similar trials in Boston, MA

By condition
Sickle cell disease
By research site
Boston Children's Hospital· Boston, MA

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