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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: Recruiting Phase 3 study of Glycogen Storage Disease Type II Infantile Onset, sponsored by Amicus Therapeutics.
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, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (95% lower). The trial reports 14 study locations across 7 states. According to ClinicalTrials.gov, the official US trial registry.
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 |
What NCT04808505 shows while recruiting
NCT04808505 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 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (95% lower).
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 lists 14 locations in 7 states (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.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT04808505's enrollment target sits among peer trials
36 1902nd of 2000 higher than 98 of 2,000 other Phase 3 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 3 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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