Summary Information For: LV, FVIII, YUVA-GT-F801, NCT03217032 * (no results published)
LV, FVIII, YUVA-GT-F801, NCT03217032 * (no results published)
Haemophilia A
Shenzhen Geno-Immune Medical Institute
General Study Information
  • ClinicalTrials.gov Identifier: NCT03217032 *
  • Phase I, Single Group Assignment, Non-randomized, open label trial *
  • Not yet recruiting *,
  • Last Update Posted: May 23, 2019 *
  • 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 FVIII *

Not reported

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

Not reported

One year *

Not reported

Efficacy details

Not reported

Not reported

Not reported

Not reported

Safety Details

Not reported

Not reported

Not reported

Not reported

Not reported

Not reported

Not reported

Not reported

Not reported

References:

* ClinicalTrials.gov

AAV, Adeno-associated virus; AEs, Adverse events; 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

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