Summary Information For: LV, FVIII-ET3, CD68-ET3-LV CD34+, Expression Therapeutics, NCT04418414 (no results published)
LV, FVIII-ET3, CD68-ET3-LV CD34+, Expression Therapeutics, NCT04418414 (no results published)
Haemophilia A
Expression Therapeutics, LLC
General Study Information
  • ClinicalTrials.gov Identifier: NCT044184141
  • Phase 1, Single Group Assignment, Non-randomized, open label trial1

The status of the trial was reported on ClinicalTrials.gov1 as follows

  • Not yet recruiting *,
  • Last Update Posted: May 23, 2022
  • Male subjects who are >= 18 years of age
  • Diagnosis of severe hemophilia A (<1 IU/dL FVIII activity) based on one-stage coagulation assay
  • Documented history of more than 150 days of FVIII treatment
  • History of spontaneous central nervous system bleeding within the last 5 years
  • Subjects who have had prior cellular based therapy or gene editing/ gene therapy including a previous stem cell transplant

Serotype of the lentiviral vector not reported / manufacturing platform unknown

Hybrid human/porcine B-domain deleted FVIII variant (ET3, HP47)23, 4

Lentiviral FVIII gene therapy targeting expression in Macrophages

Hematopoietic Stem Cell Transplantation (HSCT) of CD34+ cells transduced with CD68-ET3 lentiviral vector (encoding human FVIII gene)

Dose for the 7 participants not reported

Not reported

Not reported

Efficacy details

Not reported

Not reported

Not reported

Not reported

Safety Details

Not reported

Not reported

Not reported

Not reported

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Not applicable

Not applicable

Not reported

Not reported

References:
  1. Hematopoietic Stem Cell Transplantation Gene Therapy for Treatment of Severe Hemophilia A. Available at: Study Details | Hematopoietic Stem Cell Transplantation Gene Therapy for Treatment of Severe Hemophilia A | ClinicalTrials.gov
  2. Brown, H.C., et al., Bioengineered coagulation factor VIII enables long-term correction of murine hemophilia A following liver-directed adeno-associated viral vector delivery. Mol Ther Methods Clin Dev, 2014. 1: p. 14036. Bioengineered coagulation factor VIII enables long-term correction of murine hemophilia A following liver-directed adeno-associated viral vector delivery - PubMed (nih.gov)
  3. Doering, C.B., et al., Identification of porcine coagulation factor VIII domains responsible for high level expression via enhanced secretion. J Biol Chem, 2004. 279(8): p. 6546-52. 37. Identification of Porcine Coagulation Factor VIII Domains Responsible for High Level Expression via Enhanced Secretion* - Journal of Biological Chemistry (jbc.org)
  4. Doering, C.B., et al., Preclinical Development of a Hematopoietic Stem and Progenitor Cell Bioengineered Factor VIII Lentiviral Vector Gene Therapy for Hemophilia A. Hum Gene Ther, 2018. 29(10): p. 1183-1201. Preclinical Development of a Hematopoietic Stem and Progenitor Cell Bioengineered Factor VIII Lentiviral Vector Gene Therapy for Hemophilia A - PMC (nih.gov)

CD68, Cluster of Differentiation 68 Marker for monocyte lineage and circulating macrophages; ET3, FVIII variant with porcine A1 and ap-A3 domain sequences; LV; Lentivirus