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NCT04910776 · ClinicalTrials.gov registry record · Phase 3
Clinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of ERT With Avalglucosidase Alfa
A Phase 3 study of Glycogen Storage Disease Type II, sponsored by Sanofi.
- Active
- Registry status
- Phase 3
- Development phase
- 17
- Enrollment target
- 16
- Study locations
NCT04910776: Active Phase 3 study of Glycogen Storage Disease Type II, sponsored by Sanofi.
NCT04910776 is a Phase 3 study of Glycogen Storage Disease Type II that is active but no longer recruiting, run by Sanofi. The registered enrollment target is 17 participants, below the 673-participant average among 3 other Glycogen Storage Disease Type II trials with a reported enrollment target (97% lower). The trial reports 16 study locations across 10 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04910776, a Phase 3 study of Glycogen Storage Disease Type II, is active but no longer recruiting, sponsored by Sanofi.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 3
- Development phase
- 17 participants
- Enrollment target
- 16
- Study locations
Study Summary
This is a single group, treatment, Phase 3, open-label study to assess efficacy, safety, pharmacokinetic (PK), pharmacodynamics (PD) of avalglucosidase alfa in treatment-naïve male and female participants with IOPD. Study details include: * Study duration: Screening - up to 4 weeks; * Primary Analysis Period (PAP) - 52 weeks; * Extended Treatment Period (ETP) - 52 weeks; * Extended Long term Treatment Period (ELTP) - 104 weeks; 4-week follow-up period for a total study duration - up to 4.08 years. * Treatment duration: Up to 4 years * Visit frequency: every other week and potentially every week
Conditions Studied
Interventions
- DRUG avalglucosidase alfa
Study Locations (16)
Other
- Investigational Site Number : 0560001 - Leuven
- Investigational Site Number : 1560002 - Qingdao
- Investigational Site Number : 1560001 - Shanghai
- Investigational Site Number : 2760001 - Bad Oeynhausen
- Investigational Site Number : 5280001 - Rotterdam
- Investigational Site Number : 1580001 - Taipei
- Investigational Site Number : 8260002 - Manchester
California
- Stanford Hospital- Site Number : 8400006 - Stanford
Minnesota
- Children's Hospitals and Clinics of Minnesota- Site Number : 8400008 - Minneapolis
New York
- Advanced Medical Genetics- Site Number : 8400002 - Hawthorne
North Carolina
- Duke University Medical Center- Site Number : 8400004 - Durham
Ohio
- Cincinnati Children's Hospital Medical Center- Site Number : 8400001 - Cincinnati
Washington
- Seattle Children's Hospital- Site Number : 8400003 - Seattle
Lombardy
- Investigational Site Number : 3800002 - Monza
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 17 participants |
| Start Date | 2021-09-01 |
| Est. Completion | 2027-08-10 |
| Phase | Phase 3 |
What the registry record for NCT04910776 still lists
NCT04910776 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 17 participants, a relatively small participant target, below the 673-participant average among 3 other Glycogen Storage Disease Type II trials with a reported enrollment target (97% lower).
The record links to 1 condition, with Glycogen Storage Disease Type II appearing as the primary indexed condition, and to 1 intervention - of which avalglucosidase alfa is the first listed.
NCT04910776 lists 16 locations in 10 states (Other, California, Minnesota).
Frequently Asked Questions
What is clinical trial NCT04910776 about?
NCT04910776 is a clinical study titled "Clinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of ERT With Avalglucosidase Alfa". This is a single group, treatment, Phase 3, open-label study to assess efficacy, safety, pharmacokinetic (PK), pharmacodynamics (PD) of avalglucosidase alfa in treatment-naïve male and female participants with IOPD. Study details include: * Study duration: Screening - up to 4 weeks; * Primary Anal...
What is the current status of trial NCT04910776?
This trial is currently active not recruiting. It is a Phase 3 study. The enrollment target is 17 participants. The study started on 2021-09-01. Estimated completion is 2027-08-10.
What conditions does trial NCT04910776 study?
This clinical trial studies the following conditions: Glycogen Storage Disease Type II.
What interventions are being tested in trial NCT04910776?
The interventions under investigation include: avalglucosidase alfa (DRUG).
Who is sponsoring clinical trial NCT04910776?
This trial is sponsored by Sanofi, which has 713 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT04910776 being conducted?
This trial has 16 study locations across California, Minnesota, New York, North Carolina, Ohio. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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