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A Phase 1/2 Study of SHP648 an Adeno-Associated Viral Vector for Gene Transfer in Hemophilia B Subjects

A Phase 1/2 Study of SHP648 an Adeno-Associated Viral Vector for Gene Transfer in Hemophilia B Subjects

Recruiting
18-75 years
Male
Phase 1/2
  • Simplified description (AI rewritten)
  • Technical description (Original)

This study will consists of 3 dose cohorts with 2-7 participants in each of the three ascending dose cohorts. Initially 2 participants will be dosed in Cohort 1, followed by dosing of up to 5 additional participants if the cohort is expanded. Participants in cohort 2 and 3 will receive 2-fold or 3-fold dose escalation to their respective preceding cohort doses if required.

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Study details
    Factor IX Deficiency

NCT04394286

Baxalta now part of Shire

19 February 2024

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