Expanded Access Protocol for Gene Therapy Utilizing shmiR Lentivirus Vector to Induce Fetal Hemog… (NCT07640815) | Clinical Trial Compass
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Expanded Access Protocol for Gene Therapy Utilizing shmiR Lentivirus Vector to Induce Fetal Hemoglobin in Sickle Cell Disease
United States
Plain-language 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.
36 patients have received the gene therapy product, and the data so far has shown that the treatment has not caused any unexpected safety problems, and that it increases HbF within the red cells.
Who can participate
Age range
13 Years – 55 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Age ≥13-55 years.
. Patients with severe phenotype sickle cell disease (HbSS and other genotypes including HbSC); severity defined by VOE events in previous 2 years prior to enrollment on EAP
. Patients who meet the above criteria for disease severity, but have experienced poor mobilization, RBC alloimmunization as described below:
. Adequate hematologic parameters (regardless of therapy) including:
. White blood cell (WBC) count within the range of 2.5 - 25.0 x 109 /L
. Hemoglobin within the range of 5 - 11 g/dL
. Platelet count above 150 x 109 /L
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
. Patients who have concomitant condition or illness including, but not limited to:
. Ongoing or active infection
. Active malignancy
. 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.
. Patients with chronic pain defined as pain requiring opioids on a majority of days in the past 6 months before consent or patients taking long-acting daily opioids for longer than 6 months prior to enrollment or patients requiring blood transfusion to manage chronic pain prior to consent.
. Patients with history of overt CNS stroke at any time. Patients with imaging evidence of silent stroke but not on a chronic transfusion regimen are not excluded.
. Receiving a chronic transfusion regimen for primary or secondary stroke prophylaxis.