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NCT03759665 · ClinicalTrials.gov registry record · Phase 2

N-Acetyl-L-Leucine for GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease)

A Phase 2 study, sponsored by IntraBio.

Completed
Registry status
Phase 2
Development phase
30
Enrollment target

NCT03759665 is a Phase 2 study that has completed, run by IntraBio. The registered enrollment target is 30 participants.

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The verdict

NCT03759665, a Phase 2 study, has completed, sponsored by IntraBio.

COMPLETED
Registry status
Phase 2
Development phase
30 participants
Enrollment target

Study Summary

This is a multinational, multicenter, open-label, rater-blinded prospective Phase II study which will assess the safety and efficacy of N-Acetyl-L-Leucine (IB1001) for the treatment of GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease). There are two phases to this study: the Parent Study, and the Extension Phase. The Parent Study evaluates the safety and efficacy of N-Acetyl-L-Leucine (IB1001) in the symptomatic treatment of GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease). The Extension Phase evaluates the long-term safety and efficacy of IB1001 for the neuroprotective, disease-modifying treatment of GM2 Gangliosidosis. The Extension Phase was considered exploratory.

Interventions

  • DRUG IB1001

Trial Details

FieldValue
Enrollment Target 30 participants
Start Date 2019-06-07
Est. Completion 2023-01-09
Phase Phase 2

Sponsor

IntraBio

6 total trials

What the Registry Record Tells You About NCT03759665

The ClinicalTrials.gov registry entry for NCT03759665 describes a study currently listed as completed, categorized as Phase 2. The registered enrollment target is 30 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is IntraBio, which has 6 total studies on file at ClinicalTrials.gov.

The record links to 0 conditions, and to 1 intervention - of which IB1001 is the first listed.

NCT03759665 reports 0 study locations.

Frequently Asked Questions

What is clinical trial NCT03759665 about?

NCT03759665 is a clinical study titled "N-Acetyl-L-Leucine for GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease)". This is a multinational, multicenter, open-label, rater-blinded prospective Phase II study which will assess the safety and efficacy of N-Acetyl-L-Leucine (IB1001) for the treatment of GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease). There are two phases to this study: the Parent Study, and the...

What is the current status of trial NCT03759665?

This trial is currently completed. It is a Phase 2 study. The enrollment target is 30 participants. The study started on 2019-06-07. Estimated completion is 2023-01-09.

What interventions are being tested in trial NCT03759665?

The interventions under investigation include: IB1001 (DRUG).

Who is sponsoring clinical trial NCT03759665?

This trial is sponsored by IntraBio, which has 6 total clinical trials registered on ClinicalTrials.gov.

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.