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 range5 Years – 35 Years
SexALL
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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.
✓. 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.
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
7
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Timeframe: From date of Informed Consent signing up to Month 24