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NCT04046224 · ClinicalTrials.gov registry record · Phase 1

Dose-Ranging Study of ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy in Subjects With Fabry Disease (STAAR)

A Phase 1 study, sponsored by Sangamo Therapeutics.

Completed
Registry status
Phase 1
Development phase
36
Enrollment target

NCT04046224: Completed Phase 1 study, sponsored by Sangamo Therapeutics.

NCT04046224 is a Phase 1 study that has completed, run by Sangamo Therapeutics. The registered enrollment target is 36 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (40% lower). According to ClinicalTrials.gov, the official US trial registry.

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The verdict

NCT04046224, a Phase 1 study, has completed, sponsored by Sangamo Therapeutics.

COMPLETED
Registry status
Phase 1
Development phase
36 participants
Enrollment target

Study Summary

This is the first in human treatment with ST-920, an adeno-associated virus (AAV2/6) vector encoding the complementary deoxyribonucleic acid (cDNA) for human a-Gal A. The purpose of this study is to evaluate the safety and tolerability of ascending doses of ST-920. ST-920 aims to provide stable, long-term production of α-Gal A at therapeutic levels in subjects with Fabry disease. The constant production of α-Gal A in humans should, importantly, enable reduction and potentially clearance of Fabry disease substrates Gb3 and lyso-Gb3. On Day 1, patients will be infused intravenously with a single dose of ST-920 and followed for a period of 52 weeks.

Primary Outcome

All incidences of Treatment-Emergent Adverse Events (TEAEs) in subjects who receive ST-920 as assessed by Common Terminology Criteria for Adverse Events (CTCAE)

Interventions

  • BIOLOGICAL ST-920

Trial Details

FieldValue
Enrollment Target 36 participants
Start Date 2019-07-23
Est. Completion 2025-04-10
Phase Phase 1
Sangamo Therapeutics

25 total trials

What the finished NCT04046224 record still lists

NCT04046224 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 36 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (40% lower).

The record links to 0 conditions, and to 1 intervention - of which ST-920 is the first listed.

NCT04046224 does not publish any study locations in the registry export this page uses.

Frequently Asked Questions

What is clinical trial NCT04046224 about?

NCT04046224 is a clinical study titled "Dose-Ranging Study of ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy in Subjects With Fabry Disease (STAAR)". This is the first in human treatment with ST-920, an adeno-associated virus (AAV2/6) vector encoding the complementary deoxyribonucleic acid (cDNA) for human a-Gal A. The purpose of this study is to evaluate the safety and tolerability of ascending doses of ST-920. ST-920 aims to provide stable, lon...

What is the current status of trial NCT04046224?

This trial is currently completed. It is a Phase 1 study. The enrollment target is 36 participants. The study started on 2019-07-23. Estimated completion is 2025-04-10.

What interventions are being tested in trial NCT04046224?

The interventions under investigation include: ST-920 (BIOLOGICAL).

Who is sponsoring clinical trial NCT04046224?

This trial is sponsored by Sangamo Therapeutics, which has 25 total clinical trials registered on ClinicalTrials.gov.

How this trial's enrollment target compares

Where NCT04046224's enrollment target sits among peer trials

36 1210th of 2000 higher than 743 of 2,000 other Phase 1 trials

participants (enrollment target), bucketed by value

Each bar is a band; taller bars hold more other Phase 1 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.

Source ClinicalTrials.gov registry export · 2026-08-08

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Source: ClinicalTrials.gov NCT04046224, the US trial registry maintained by the National Library of Medicine. NCT04046224 (small enrollment · none site footprint · completed) retrieved and formatted by PlainTrial, see methodology.