• ClinicalTrials.gov Identifier: NCT03217032¹ (Lentiviral FVIII Gene Therapy)
• Phase I, Single Group Assignment, Non-randomized, open label trial¹

• Not yet recruiting *,
• Last Update Posted: 2019-05-23¹

Males ≥2 years with confirmed diagnosis of hemophilia A and endogenous FVIII ≤2 IU/dL or ≤2% of normal  

Participated in a gene transfer trial within  the last 6 months or in a clinical trial with an investigational drug within the last 12 weeks

• Lentiviral vector system NHP/TYF
• Serotype / manufacturing platform not reported

Human FVIII¹

Not reported

Infusion of lentiviral FVIII gene modified hematopoietic stem cells and mesenchymal stem cells ^*

Not reported

One year¹

Not reported

Not reported

Not reported

Not reported

Not reported

Not reported

Not reported

Not reported

Not reported

Not reported

Not reported

Not reported

Not reported

Not reported

1. Lentiviral FVIII Gene Therapy. Available at: Study Details | Lentiviral FVIII Gene Therapy | ClinicalTrials.gov

AAV, Adeno-associated virus; ABR, Annualized bleeding rate; AEs: adverse events; AIR, Annualized FVIII/FIX infusion rate; ALT, alanine aminotransferase; AST, aspartate aminotransferase; CH, Chromogenic Assay; Co., cohort; DOACs, Direct oral anticoagulants; D, days; EDs, exposure days; FIX, factor IX; FIX-Padua, gain of function FIX variant; FVIII, factor VIII; gc, genome copies; HEK cells, human embryonic kidney cells; IQR, interquartile range; IRR, Infusion-related reaction; NAbs, neutralizing antibodies; OS, One-stage clotting assay; Pop., population; pt., patient/participant; pts., patients/participants; P1, Participant 1; PI, phase I; PBGD, porphobilinogen deaminase; PBMC, peripheral blood mononuclear cells; SAEs, serious adverse events; SFU, spot-forming units; TAC, tacrolimus; ULN, upper limit of normal; VCN, vector copy number; vg, vector genomes; W, weeks; WT, wild type; Y, year