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NCT04808505 · ClinicalTrials.gov registry record · Phase 3
A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18
A Phase 3 study of Glycogen Storage Disease Type II Infantile Onset, sponsored by Amicus Therapeutics.
- Recruiting
- Registry status
- Phase 3
- Development phase
- 36
- Enrollment target
- 14
- Study locations
NCT04808505 is a Phase 3 study of Glycogen Storage Disease Type II Infantile Onset that is actively recruiting participants, run by Amicus Therapeutics. The registered enrollment target is 36 participants. The trial reports 14 study locations across 7 states.
The verdict
NCT04808505, a Phase 3 study of Glycogen Storage Disease Type II Infantile Onset, is actively recruiting participants, sponsored by Amicus Therapeutics.
- RECRUITING
- Registry status
- Phase 3
- Development phase
- 36 participants
- Enrollment target
- 14
- Study locations
Study Summary
This is a Phase 3, open-label, multicenter study to evaluate the safety, efficacy, PK, PD, and immunogenicity of cipaglucosidase alfa/miglustat treatment in ERT-experienced and ERT-naïve pediatric subjects with IOPD.
Conditions Studied
Interventions
- BIOLOGICAL Cipaglucosidase alfa
- DRUG Miglustat
Study Locations (14)
Other
- Universitätsklinikum Gießen und Marburg GmbH, Zentrum fur Kinderheilkunde und Jugendmedizin Abteilung fur Kinderneurologic, Sozialpadiatric und Epileptologie - Giessen
- Universitätsklinikum Heidelberg - Pädiatrisches Klinisch-Pharmakologisches Studienzentrum (paedKliPS) - Heidelberg
- SphinCS GmbH - Höchheim
- Universitätsklinikum Münster Klinik für Kinder- und Jugendmedizin Albert-Schweitzer-Campus 1 - Münster
- AOU Federico II - Naples
- Erasmus MC, Sophia Kinderziekenhuis - Rotterdam
- National Taiwan University Hospital - Taipei
- Great Ormond Street Hospital for Children NHS Foundation Trust - London
Florida
- University of Florida Clinical Research Center - Gainesville
Georgia
- The Emory Clinic - Atlanta
North Carolina
- Duke University Early Phase Research Unit - Durham
Ohio
- Cincinnati Children's Hospital Medical Center - Cincinnati
Pennsylvania
- UPMC Hospital of Pittsburgh - Pittsburgh
Utah
- University of Utah, Clinical and Translational Sciences Institute - Salt Lake City
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 36 participants |
| Start Date | 2023-07-18 |
| Est. Completion | 2027-07 |
| Phase | Phase 3 |
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT04808505
The ClinicalTrials.gov registry entry for NCT04808505 describes a study currently listed as recruiting, categorized as Phase 3. The registered enrollment target is 36 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Amicus Therapeutics, which has 34 total studies on file at ClinicalTrials.gov.
The record links to 1 condition, with Glycogen Storage Disease Type II Infantile Onset appearing as the primary indexed condition, and to 2 interventions - of which Cipaglucosidase alfa is the first listed.
NCT04808505 reports 14 study locations spanning 7 distinct geographic areas - top geographies include Other, Florida, Georgia.
Frequently Asked Questions
What is clinical trial NCT04808505 about?
NCT04808505 is a clinical study titled "A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18". This is a Phase 3, open-label, multicenter study to evaluate the safety, efficacy, PK, PD, and immunogenicity of cipaglucosidase alfa/miglustat treatment in ERT-experienced and ERT-naïve pediatric subjects with IOPD.
What is the current status of trial NCT04808505?
This trial is currently recruiting. It is a Phase 3 study. The enrollment target is 36 participants. The study started on 2023-07-18. Estimated completion is 2027-07.
What conditions does trial NCT04808505 study?
This clinical trial studies the following conditions: Glycogen Storage Disease Type II Infantile Onset.
What interventions are being tested in trial NCT04808505?
The interventions under investigation include: Cipaglucosidase alfa (BIOLOGICAL), Miglustat (DRUG).
Who is sponsoring clinical trial NCT04808505?
This trial is sponsored by Amicus Therapeutics, which has 34 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT04808505 being conducted?
This trial has 14 study locations across Florida, Georgia, North Carolina, Ohio, Pennsylvania. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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