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NCT03911505 · ClinicalTrials.gov registry record · Phase 3

ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD

A Phase 3 study of Pompe Disease (Late-onset), sponsored by Amicus Therapeutics.

Active
Registry status
Phase 3
Development phase
21
Enrollment target
17
Study locations

NCT03911505 is a Phase 3 study of Pompe Disease (Late-onset) that is active but no longer recruiting, run by Amicus Therapeutics. The registered enrollment target is 21 participants, below the 45-participant average among 8 other Pompe Disease (Late-onset) trials with a reported enrollment target (53% lower). The trial reports 17 study locations across 13 states.

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The verdict

NCT03911505, a Phase 3 study of Pompe Disease (Late-onset), is active but no longer recruiting, sponsored by Amicus Therapeutics.

ACTIVE NOT RECRUITING
Registry status
Phase 3
Development phase
21 participants
Enrollment target
17
Study locations

Study Summary

This is a Phase 3, open-label, multicenter study to evaluate the safety, PK, efficacy, PD, and immunogenicity of Cipaglucosidase Alfa/Miglustat treatment in enzyme replacement therapy (ERT)-experienced and ERT-naïve pediatric subjects with Pompe disease, aged 0 to \< 18 years

Conditions Studied

Pompe Disease (Late-onset)

Interventions

  • DRUG Miglustat
  • BIOLOGICAL Cipaglucosidase Alfa

Study Locations (17)

Other

  • San Gerardo Hospital - Monza
  • Gunma University Hospital - Gunma
  • Tohoku University Hospital - Miyagi
  • Tokyo Women's Medical University - Tokyo

Florida

  • University of Florida Clinical Research Center - Gainesville
  • Wolfson Children's Hospital - Jacksonville

Georgia

  • Woodruff Memorial Research Building - Atlanta

Missouri

  • St. Louis Children's Hospital - St Louis

North Carolina

  • Duke University Medical Center - Durham

Ohio

  • Cincinnati Children's Hospital Medical Center - Cincinnati

Pennsylvania

  • UPMC Children's Hospital of Pittsburgh - Pittsburgh

Utah

  • University of Utah, Clinical and Translational Sciences Institute - Salt Lake City

Trial Details

FieldValue
Enrollment Target 21 participants
Start Date 2020-02-13
Est. Completion 2026-06
Phase Phase 3

Sponsor

Amicus Therapeutics

34 total trials

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What the Registry Record Tells You About NCT03911505

The ClinicalTrials.gov registry entry for NCT03911505 describes a study currently listed as active not recruiting, categorized as Phase 3. The registered enrollment target is 21 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 45-participant average among 8 other Pompe Disease (Late-onset) trials with a reported enrollment target (53% lower). The listed sponsor is Amicus Therapeutics, which has 34 total studies on file at ClinicalTrials.gov.

The record links to 1 condition, with Pompe Disease (Late-onset) appearing as the primary indexed condition, and to 2 interventions - of which Miglustat is the first listed.

NCT03911505 reports 17 study locations spanning 13 distinct geographic areas - top geographies include Other, Florida, Georgia.

Frequently Asked Questions

What is clinical trial NCT03911505 about?

NCT03911505 is a clinical study titled "ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD". This is a Phase 3, open-label, multicenter study to evaluate the safety, PK, efficacy, PD, and immunogenicity of Cipaglucosidase Alfa/Miglustat treatment in enzyme replacement therapy (ERT)-experienced and ERT-naïve pediatric subjects with Pompe disease, aged 0 to \< 18 years

What is the current status of trial NCT03911505?

This trial is currently active not recruiting. It is a Phase 3 study. The enrollment target is 21 participants. The study started on 2020-02-13. Estimated completion is 2026-06.

What conditions does trial NCT03911505 study?

This clinical trial studies the following conditions: Pompe Disease (Late-onset).

What interventions are being tested in trial NCT03911505?

The interventions under investigation include: Miglustat (DRUG), Cipaglucosidase Alfa (BIOLOGICAL).

Who is sponsoring clinical trial NCT03911505?

This trial is sponsored by Amicus Therapeutics, which has 34 total clinical trials registered on ClinicalTrials.gov.

Where is trial NCT03911505 being conducted?

This trial has 17 study locations across Florida, Georgia, Missouri, North Carolina, Ohio. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.

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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.