Table 1_VTX-PID as a novel recombinant immunoglobulin G–degrading enzyme (IdeS) for effici

The NAVIgATE study was a first-in-human, double-blind controlled trial involving 35 healthy men, stratified by baseline anti-AAV3B neutralizing antibody levels. It investigated the safety and efficacy of ascending doses of VTX-PID (imlifidase) as a pretreatment to enable AAV-based gene therapy. The dataset, authored by Bernard Benichou and last updated in May 2026, contains results from this phase I study, including primary safety and key secondary immunogenicity endpoints.

Use Cases

• Analyze dose-dependent effects on antibody reduction based on the described VTX-PID dose range (0.075-0.6 mg/kg).
• Model the time window for gene therapy administration based on the reported 2–5 day duration of antibody depletion at higher doses.
• Assess safety profiles for novel enzyme pretreatments based on the reported adverse events and liver function test elevations.
• Compare treatment efficacy between patient strata based on the described intermediate (≤1:45) and high (>1:45) baseline neutralizing antibody levels.

Strengths

• Data originates from a defined clinical trial protocol with a registered EUCT number (2023-503892-83-00).
• Includes results from 35 participants across multiple dose levels, allowing for dose-response analysis.
• Reports specific, quantifiable endpoints such as anti-AAV3B neutralizing antibody and total antibody levels.

Limitations

• Column-level documentation is absent; field semantics must be inferred after download.
• Row count is unknown, which may limit suitability assessment.
• The primary data file is a DOCX document (939.9 KB), which is a very small and unstructured format for quantitative analysis.

Provenance

Source

figshare, authored by Bernard Benichou.

Collection Method

Data was generated from a randomized, double-blind, placebo-controlled phase I clinical trial.

Freshness

Last updated 2026-05-20 13:21:05; freshness should be verified.