Trial intelligence / Profile preview

PRECIZN-1: BIVV003 for Severe Sickle Cell Disease

NCT03653247Phase 2

Key facts

Status: Completed
Start: Mar 2019
Primary completion: Jul 2025
Countries: 1
Sponsor: Sangamo Therapeutics
Modality: Gene Editing, Gene Silencing, Hematopoietic Stem Cells

Drugs: ST-400
Conditions: sickle cell disease (SCD)

Overview

Phase 2, open-label, single-arm trial of ST-400 in sickle cell disease (SCD), sponsored by Sangamo Therapeutics. Primary endpoints: Overall Survival at 104 weeks, Successful Engraftment at 42 days and 2 more. Started Mar 2019; status: completed.

Design

1 arm

Study design: Non-randomizedOpen-labelSingle group

BIVV003
Autologous HSCT with BIVV003

Who it enrols

Adults (18-40) with severe sickle cell disease (HbSS or HbSbeta0 genotype) defined by a history of stroke, acute chest syndrome, frequent pain crises, or other complications. Participants must be clinically stable for HSCT, have adequate organ function, and must not have received prior gene therapy or HSCT.

Endpoints

Primary

Overall Survival104 weeks
Successful Engraftment42 days
Adverse Events104 weeks
Serious Adverse Events104 weeks

Secondary

CD34+ HSPC Yield from Plerixafor Mobilization12 weeks
Time to Initial Neutrophil Recovery104 weeks
Time to Platelet Recovery104 weeks
Fetal Hemoglobin Levels104 weeks
Percent F-cells104 weeks
Sickle Hemoglobin Levels104 weeks
Total Hemoglobin Concentration104 weeks
PROMIS-57 Scale Score104 weeks

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