Clinical trial for Sickle Cell Disease
Official title Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Sickle Cell Disease
ClinicalTrials.gov ID: NCT07708350
What this study is testing
What is autologous bone marrow derived CD34+ HSPCs electroporated with BCL11A enhancer targeting Cas9 ribonucleoprotein?
An investigational cell‑based therapy being studied for sickle cell disease is designed to edit the patient’s own blood stem cells to reduce the activity of the BCL11A gene, which may increase fetal hemoglobin production
Plain-language explanation of the investigational treatment. It is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- The study wants to see if editing a patient’s own blood stem cells can raise healthy fetal hemoglobin and lower sickle hemoglobin.
- Who it's for
- It is looking for adults who have sickle cell disease (HbSS or HbSβ‑thalassemia).
- What's involved
- Participants will give a blood sample, have their stem cells edited in a lab, and then receive the edited cells back. They will have several clinic visits and blood tests to monitor safety and blood changes.
- Time commitment
- The study lasts about 12 months, with regular visits for blood draws and monitoring after the cell infusion.
- Phase 1: an early, usually small safety study
- Which group you join is decided by chance.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 13 to 35
You may be able to join if
- Diagnosis of either a) sickle cell disease with genotype HbSS, HbS/B0 thalassemia, HbSD, or HbSO
- Age 13-35 years.
- Clinically severe disease, defined as: The presence of one or more of the following clinical complications: i) Minimum of two episodes of acute chest...
- Adequate hematologic parameters including: a. White blood cell (WBC) count within the range of 2.5 - 25.0 x 109 /L b.
- Karnofsky performance status ≥70%
You likely can't join if
- Uncontrolled infection, such as current febrile illness, infection requiring parenteral antibiotics, or systemic fungal infection.
- Active malignancy.
- Active complication of underlying hemoglobinopathy that would place the patient at unacceptable risk for participation, in the judgment of the...
- Major surgery in the past 30 days.
- Medical/psychiatric illness/social situations that would limit compliance with study requirements as determined by the treating physician.
- Severe cerebral vasculopathy (defined by occlusion or stenosis in the circle of Willis; or presence of Moyamoya disease)
See the full eligibility criteria
- Diagnosis of either a) sickle cell disease with genotype HbSS, HbS/B0 thalassemia, HbSD, or HbSO
- Age 13-35 years.
- Clinically severe disease, defined as: The presence of one or more of the following clinical complications: i) Minimum of two episodes of acute chest syndrome (ACS) in the 2 years before study entry.
- Adequate hematologic parameters including: a. White blood cell (WBC) count within the range of 2.5 - 25.0 x 109 /L b. Platelet count within the range of 150 - 700 x 109 /L
- Karnofsky performance status ≥70%
- Serum creatinine \</=1.5 times the upper limit of normal for age, and calculated creatinine clearance or Glomerular Filtration Rate (GFR) \</= 60 mL/min/1.73 m2.
- Direct bilirubin ≤ 2.0 mg/dL
- Diffusing Capacity of the Lungs for Carbon Monoxide (DLCO) (corrected for hemoglobin), Forced Expiratory Volume in 1 Second (FEV1), Forced Vital Capacity (FVC) \>50% of predicted
- Left ventricular ejection fraction \>40% or shortening fraction \>25%
- Failure of hydroxyurea therapy due to lack of clinical improvement, inability to tolerate due to side effects (e.g., myelosuppression, gastrointestinal symptoms, or hepatic enzyme elevations) or not clinically indicated...
- Confirmed sickle cell disease diagnosis by molecular genetic testing.
- No Human Leukocyte Antigen (HLA) genotypically-identical related appropriate bone marrow donor available.
- Parental/guardian/patient signed informed consent.
- Willingness to return for follow-up for 15 years.
- Uncontrolled infection, such as current febrile illness, infection requiring parenteral antibiotics, or systemic fungal infection.
- Active malignancy.
- Active complication of underlying hemoglobinopathy that would place the patient at unacceptable risk for participation, in the judgment of the Investigators.
- Major surgery in the past 30 days.
- Medical/psychiatric illness/social situations that would limit compliance with study requirements as determined by the treating physician.
- Severe cerebral vasculopathy (defined by occlusion or stenosis in the circle of Willis; or presence of Moyamoya disease)
- Receiving a chronic transfusion regimen for primary or secondary stroke prophylaxis. (Note: patients with a history of abnormal transcranial Doppler (TCD) who have transitioned from transfusions to hydroxyurea for...
- History of overt stroke or any neurologic event lasting \> 24 hours. (Note: patients with imaging evidence of silent stroke but not on a chronic transfusion regimen are not excluded.) 6.
The study team makes the final eligibility decision.
Where it is enrolling
- Boston, Massachusetts, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part, but confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 13 years to 35 years. The study team makes the final eligibility decision.
Where is this trial taking place?
This study is enrolling now at Boston, Massachusetts, United States. Enter your location above to see the nearest enrolling site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool that is independent of the study sponsor. Details come from ClinicalTrials.gov. The study team decides eligibility, and your own doctor gives medical advice.