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NCT04693598 · ClinicalTrials.gov registry record · Phase 1

Gene Transfer Clinical Trial for Krabbe Disease

A Phase 1 study of Krabbe Disease, sponsored by Forge Biologics.

Active
Registry status
Phase 1
Development phase
6
Enrollment target
1
Study location

NCT04693598 is a Phase 1 study of Krabbe Disease that is active but no longer recruiting, run by Forge Biologics. The registered enrollment target is 6 participants, below the 417-participant average among 4 other Krabbe Disease trials with a reported enrollment target (99% lower). The trial reports 1 study location across 1 state.

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The verdict

NCT04693598, a Phase 1 study of Krabbe Disease, is active but no longer recruiting, sponsored by Forge Biologics.

ACTIVE NOT RECRUITING
Registry status
Phase 1
Development phase
6 participants
Enrollment target
1
Study location

Study Summary

This is a nonblinded, non-randomized dose escalation study of intravenous AAVrh10 after hematopoietic stem cell transplantation (HSCT) in which subjects will receive standard of care hematopoietic cell transplantation for Krabbe disease, followed by a single infusion of an adeno-associated virus gene therapy product. Extensive natural history subjects will be used to compare as control group.

Conditions Studied

Interventions

  • BIOLOGICAL FBX-101

Study Locations (1)

Michigan

  • University of Michigan Hospitals - Michigan Medicine - Ann Arbor

Trial Details

FieldValue
Enrollment Target 6 participants
Start Date 2021-11-05
Est. Completion 2026-11
Phase Phase 1

Sponsor

Forge Biologics

3 total trials

What the Registry Record Tells You About NCT04693598

The ClinicalTrials.gov registry entry for NCT04693598 describes a study currently listed as active not recruiting, categorized as Phase 1. The registered enrollment target is 6 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 417-participant average among 4 other Krabbe Disease trials with a reported enrollment target (99% lower). The listed sponsor is Forge Biologics, which has 3 total studies on file at ClinicalTrials.gov.

The record links to 1 condition, with Krabbe Disease appearing as the primary indexed condition, and to 1 intervention - of which FBX-101 is the first listed.

NCT04693598 reports 1 study location spanning 1 distinct geographic area - top geographies include Michigan.

Frequently Asked Questions

What is clinical trial NCT04693598 about?

NCT04693598 is a clinical study titled "Gene Transfer Clinical Trial for Krabbe Disease". This is a nonblinded, non-randomized dose escalation study of intravenous AAVrh10 after hematopoietic stem cell transplantation (HSCT) in which subjects will receive standard of care hematopoietic cell transplantation for Krabbe disease, followed by a single infusion of an adeno-associated virus gen...

What is the current status of trial NCT04693598?

This trial is currently active not recruiting. It is a Phase 1 study. The enrollment target is 6 participants. The study started on 2021-11-05. Estimated completion is 2026-11.

What conditions does trial NCT04693598 study?

This clinical trial studies the following conditions: Krabbe Disease.

What interventions are being tested in trial NCT04693598?

The interventions under investigation include: FBX-101 (BIOLOGICAL).

Who is sponsoring clinical trial NCT04693598?

This trial is sponsored by Forge Biologics, which has 3 total clinical trials registered on ClinicalTrials.gov.

Where is trial NCT04693598 being conducted?

This trial has 1 study location across Michigan. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.

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Data sourced from official public datasets. See our methodology for details. Retrieved and formatted by PlainTrial Editorial

Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page mirrors this trial's own ClinicalTrials.gov registry record, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.