Last updated on December 2019

Safety and Dose Escalation Study of an Adeno-Associated Viral Vector for Gene Transfer in Hemophilia A Participants

Brief description of study

The purpose of this study is to evaluate the safety and determine the dose of BAX 888

Detailed Study Description

This study consists of 3 cohorts. Participants will be assigned to 1 of 2 dose cohorts with a minimum of 24 hours between dosing of each participant. Initially, 2 participants will be dosed in a cohort, with up to a total of 5 participants if the cohort is expanded based on safety and FVIII expression data.

Dose escalation:

After dosing first 2 participants in cohort 1 or cohort 2 on day 0 if week 4 FVIII activity levels of both participants are approximately their baseline values, then dose escalation to cohort 2 will be triggered with no further dosing in cohort 1. If FVIII expression (greater than or equal to [>=] 2 [percent] %) is observed in at least 1 participant among the 2 participants the decision to escalate dose or expand the cohort with dosing of additional participants will be based on all available data through Week 14.

Dose expansion:

After dose escalation and administration of BAX 888 to the first 2 participants in 3 cohorts: If sustained Week 14 FVIII levels are lesser than (<) 30% in both participant (first 2 participant in cohort 1, 2) then escalation to immediate next cohort will be triggered after Data Monitoring Committee (DMC) review of all data inclusive of Week 14 for cohort 1 and cohort 2. For cohort 3 dosing of additional participants will be paused until further review of all available data. If sustained Week 14 FVIII levels are >= 30% in at least 1 of the 2 participants (first 2 participant in cohort 1, 2, 3) then expansion of cohorts 1, 2, 3 will be initiated with dosing of at least 3 additional participant.

Clinical Study Identifier: NCT03370172

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Semmelweis Egyetem

Budapest, Hungary
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