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GENE THERAPY, OPEN-LABEL, DOSE-ESCALATION STUDY OF SPK-9001 [ADENO-ASSOCIATED VIRAL VECTOR WITH HUMAN FACTOR IX GENE] IN SUBJECTS WITH HEMOPHILIA B

Trial Profile

GENE THERAPY, OPEN-LABEL, DOSE-ESCALATION STUDY OF SPK-9001 [ADENO-ASSOCIATED VIRAL VECTOR WITH HUMAN FACTOR IX GENE] IN SUBJECTS WITH HEMOPHILIA B

Status: Completed
Phase of Trial: Phase I/II

Latest Information Update: 17 Jan 2024

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At a glance

  • Drugs Fidanacogene-elaparvovec (Primary)
  • Indications Haemophilia B
  • Focus Adverse reactions; Therapeutic Use
  • Sponsors Pfizer; Spark Therapeutics
  • Most Recent Events

    • 12 Dec 2023 Results reporting descriptive data on impact on HRQoL for participants in the fidanacogene elaparvovec phase 1/2a study (NCT02484092) and its LTU study (NCT03307980) presented at the 65th American Society of Hematology Annual Meeting and Exposition
    • 14 Dec 2021 Results assessing Follow-up of More Than 5 Years presented at the 63rd American Society of Hematology Annual Meeting and Exposition
    • 07 Jan 2020 Status changed from active, no longer recruiting to completed.

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