A Study Evaluating the Safety and Efficacy of LentiGlobin BB305 Drug Product in β-Thalassemia Maj… (NCT02151526) | Clinical Trial Compass
CompletedPhase 1/2
A Study Evaluating the Safety and Efficacy of LentiGlobin BB305 Drug Product in β-Thalassemia Major (Also Referred to as Transfusion-dependent β-Thalassemia [TDT]) and Sickle Cell Disease
France7 participantsStarted 2013-06-07
Plain-language summary
This is a Phase 1/2, open label, safety, and efficacy study of the administration of LentiGlobin BB305 Drug Product to participants with either transfusion dependent beta-thalassemia (TDT) or sickle cell disease (SCD).
Who can participate
Age range
5 Years – 35 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
. Be between 5 and 35 years of age, inclusive.
. Have severe SCD or transfusion dependent beta-thalassemia major, regardless of the genotype with the diagnosis confirmed by Hb studies. Transfusion dependence is defined as requiring at least 100 mL/kg/year of packed red blood cells (pRBCs).
. Be eligible for allogeneic hematopoietic stem cell transplant (HSCT) based on institutional medical guidelines, but without a matched related donor.
. Be willing and able, in the Investigator's opinion, to comply with the study procedures outlined in the study protocol.
. Have been treated and followed for at least the past 2 years in a specialized center that maintained detailed medical records, including transfusion history.
. Have failed to achieve adequate clinical benefit following hydroxyurea treatment with sufficient dosage, for at least 4 months unless this treatment was not indicated or not well tolerated.
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.
What they're measuring
1
Number of Treated Participants With Successful Neutrophil and Platelet Engraftment
Timeframe: From time of drug product infusion through Month 24
2
Time to Successful Neutrophil and Platelet Engraftment
Timeframe: From time of drug product infusion through Month 24
3
Incidence of Transplant Related Mortality
Timeframe: From screening through 365 days post-transplant
4
Number of Participants With Overall Survival (OS) Events
Timeframe: From time of drug product infusion through Month 24
5
Percentage of Participants With Vector-Derived Replication-Competent Lentivirus (RCL)
Timeframe: From time of drug product infusion through Month 24
6
Number of Treated Participants With Greater Than (>) 30 Percent (%) Contribution of an Individual Clone As Per Integration Site Analysis (ISA)
Timeframe: From time of drug product infusion through Month 24
. Have 1 or more of the following poor prognostic risk factors:
. Participants with severe SCD and cerebral vasculopathy (defined by overt stroke; abnormal transcranial Doppler \[\> 170 cm/sec\]; or occlusion or stenosis in the polygon of Willis; or presence of Moyamoya disease) may be enrolled only with approval by the Comite de Surveillance after review of safety and efficacy data from \>or= 2 SCD participants without cerebral vasculopathy treated with LentiGlobin BB305 Drug Product
Exclusion criteria
. Availability of a willing 10 /10 matched human leukocyte antigen (HLA) identical sibling hematopoietic cell donor, unless recommendation for enrollment is provided by the Comite de Surveillance following a review of the case.
. Clinically significant, active bacterial, viral, fungal, or parasitic infection.
. Contraindication to anesthesia for bone marrow harvesting.
. Any prior or current malignancy, myeloproliferative or immunodeficiency disorder.
. A white blood cell (WBC) count \<3×10\^9/L and/or platelet count \<120×10\^9/L.
. History of major organ damage including:
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Timeframe: From date of Informed Consent signing up to Month 24