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A Gene Transfer Study Inducing Fetal Hemoglobin in Sickle Cell Disease (GRASP, BMT CTN 2001)
NCT05353647 · View on ClinicalTrials.gov ↗
Study Summary
A promising approach for the treatment of genetic diseases is called gene therapy. Gene therapy is a relatively new field of medicine in which genetic material (mostly DNA) in the patient is changed to treat his or her own disease. In gene therapy, we introduce new genetic material in order to fix or replace the patient's disease 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. To introduce new genetic material into the patient's own blood stem cells we use a modified version of a virus (called a 'vector') that efficiently inserts the "correcting" genetic material into the cells. The vector is a specialized biological medicine that has been formulated for use in human beings. Fetal hemoglobin (HbF) is a healthy, non-sickling kind of hemoglobin. The investigators have discovered a gene that is very important in controlling the amount of HbF. Decreasing 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, specifically the amount in red blood cells where sickle hemoglobin causes damage to the cell, and therefore potentially cure or significantly improve the condition. The gene we are targeting for change in this study that controls the level of fetal hemoglobin is called BCL11A. In summary, the advantages of a gene therapy approach include
Conditions Studied
Interventions
- BIOLOGICAL Autologous CD34+ HSC cells transduced with the lentiviral vector containing a shRNA targeting BCL11a
Study Locations (9)
California
- Children's Hospital of Los Angeles — Los Angeles
- UCLA Medical Center — Los Angeles
- UCSF Benioff Children's Hospital Oakland — Oakland
- UC Davis Medical Center — Sacramento
Massachusetts
- Boston Children's Hospital — Boston
- Dana-Farber Cancer Institute/Brigham and Women's Hospital — Boston
Georgia
- Children's Healthcare of Atlanta/Emory University — Atlanta
Illinois
- Lurie Children's Hospital of Chicago — Chicago
Wisconsin
- Medical College of Wisconsin — Milwaukee
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 25 participants |
| Start Date | 2022-07-12 |
| Est. Completion | 2027-07 |
| Phase | Phase 2 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT05353647
The ClinicalTrials.gov registry entry for NCT05353647 describes a study currently listed as active not recruiting. It is categorized as Phase 2, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 25 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is David Williams, which has 3 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 1 condition, with Sickle Cell Disease appearing as the primary indexed condition, and to 1 intervention — of which Autologous CD34+ HSC cells transduced with the lentiviral vector containing a shRNA targeting BCL11a is the first listed. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT05353647 reports 9 study locations spanning 5 distinct geographic areas — top geographies include California, Massachusetts, Georgia. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
Frequently Asked Questions
What is clinical trial NCT05353647 about?
NCT05353647 is a clinical study titled "A Gene Transfer Study Inducing Fetal Hemoglobin in Sickle Cell Disease (GRASP, BMT CTN 2001)". A promising approach for the treatment of genetic diseases is called gene therapy. Gene therapy is a relatively new field of medicine in which genetic material (mostly DNA) in the patient is changed to treat his or her own disease. In gene therapy, we introduce new genetic material in order to fix o...
What is the current status of trial NCT05353647?
This trial is currently active not recruiting. It is a Phase 2 study. The enrollment target is 25 participants. The study started on 2022-07-12. Estimated completion is 2027-07.
What conditions does trial NCT05353647 study?
This clinical trial studies the following conditions: Sickle Cell Disease. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT05353647?
The interventions under investigation include: Autologous CD34+ HSC cells transduced with the lentiviral vector containing a shRNA targeting BCL11a (BIOLOGICAL). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT05353647?
This trial is sponsored by David Williams, which has 3 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT05353647 being conducted?
This trial has 9 study locations across California, Georgia, Illinois, Massachusetts, Wisconsin. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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