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Gene Transfer for Sickle Cell Disease
NCT03282656 · 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 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 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 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 introduce the gene into the patient's own blood stem cells is to engineer and 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. The investigators have recently discovered a gene 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. In summary, the advantages of a gene therapy approach include: 1) it can be used even if the patient does not have a matched donor available; 2) it may allow a reduction in the amount of chemotherapy required to prepare the patient for the transplant; and 3) it will avoid the strong medicines often required to prevent and treat GVHD and reje
Conditions Studied
Interventions
- BIOLOGICAL single infusion of autologous bone marrow derived CD34+ HSC cells transduced with the lentiviral vector containing a short-hairpin RNA targeting BCL11a
Study Locations (2)
California
- UCLA - Mattel Children's Hospital — Los Angeles
Massachusetts
- Boston Children's Hospital — Boston
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 10 participants |
| Start Date | 2018-02-13 |
| Est. Completion | 2026-11-13 |
| Phase | Phase 1 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT03282656
The ClinicalTrials.gov registry entry for NCT03282656 describes a study currently listed as active not recruiting. It is categorized as Phase 1, 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 10 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 single infusion of autologous bone marrow derived CD34+ HSC cells transduced with the lentiviral vector containing a short-hairpin RNA 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: NCT03282656 reports 2 study locations spanning 2 distinct geographic areas — top geographies include California, Massachusetts. 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 NCT03282656 about?
NCT03282656 is a clinical study titled "Gene Transfer for Sickle Cell Disease". 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 NCT03282656?
This trial is currently active not recruiting. It is a Phase 1 study. The enrollment target is 10 participants. The study started on 2018-02-13. Estimated completion is 2026-11-13.
What conditions does trial NCT03282656 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 NCT03282656?
The interventions under investigation include: single infusion of autologous bone marrow derived CD34+ HSC cells transduced with the lentiviral vector containing a short-hairpin RNA targeting BCL11a (BIOLOGICAL). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT03282656?
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 NCT03282656 being conducted?
This trial has 2 study locations across California, Massachusetts. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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