Dose Selection and Study Design for B-LIEVE, A Phase 1/2 Dose Confirmation Clinical Trial of FLT180A Gene Therapy for Patients with Hemophilia B
Highlights From the 15th Annual Congress of EAHAD

Dose Selection and Study Design for B-LIEVE, A Phase 1/2 Dose Confirmation Clinical Trial of FLT180A Gene Therapy for Patients with Hemophilia B

G.Young1,P.Chowdary, 2,3,S.Barton4, A.Long4

1Children’s Hospital Los Angeles, University of Southern California Keck School of Medicine, Los Angeles, California, United States;2 Katharine Dormandy Haemophilia and Thrombosis Centre, Royal Free Hospital;3 University College London,United Kingdom;4 Freeline, Stevenage, United Kingdom

Key Data Points

B-AMAZE Dose Response

The B-LIEVE study is a phase 1/2 study of commercially manufactured FLT180a designed to confirm a target dose for a phase 3 study. The starting dose for B-LIEVE was based on results from the B-AMAZE trial, which demonstrated that FIX levels at Day 21 following infusion were highly predictive of FIX levels at Day 182 (week 26). An Emax dose-response model indicated that a dose of 7.7 x 1011 vg/kg should produce a mean FIX level of 67% of normal at Day 21 corresponding to a mean FIX level of 115% of normal at Day 182.

Dosing Plan for B-LIEVE Study Design

The adaptive study design for B-LIEVE includes a starting FLT180a dose of 7.7 x 1011 vg/kg in 3 participants. The starting dose will be adjusted in subsequent cohorts of 3 participants, each based on the Day 21 FIX levels in the previous cohort. Up to 9 participants (3 cohorts) will be enrolled with a 52-week follow-up period in order to confirm a dose for a phase 3 study.

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Supported by educational grants from Bayer, BioMarin, Freeline Therapeutics Limited, Pfizer Inc., Shire, Spark Therapeutics, and uniQure, Inc.

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