Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
NCT04174105 · ClinicalTrials.gov registry record · Phase 1
Gene Transfer Study in Patients With Late Onset Pompe Disease
A Phase 1 study of Pompe Disease (Late-onset), sponsored by Astellas Gene Therapies.
- Active
- Registry status
- Phase 1
- Development phase
- 11
- Enrollment target
- 4
- Study locations
NCT04174105: Active Phase 1 study of Pompe Disease (Late-onset), sponsored by Astellas Gene Therapies.
NCT04174105 is a Phase 1 study of Pompe Disease (Late-onset) that is active but no longer recruiting, run by Astellas Gene Therapies. The registered enrollment target is 11 participants, below the 46-participant average among 8 other Pompe Disease (Late-onset) trials with a reported enrollment target (76% lower). The trial reports 4 study locations across 3 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04174105, a Phase 1 study of Pompe Disease (Late-onset), is active but no longer recruiting, sponsored by Astellas Gene Therapies.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 1
- Development phase
- 11 participants
- Enrollment target
- 4
- Study locations
Study Summary
This is a phase 1/2 open-label, ascending dose, multicenter clinical study to evaluate the safety and efficacy of AT845 in adult (aged ≥ 18 years) subjects, ambulatory or nonambulatory, with Late Onset Pompe Disease (LOPD).
Primary Outcome
Frequency of adverse events (AEs), serious adverse events (SAEs), and changes from baseline in relevant clinical laboratory tests
Conditions Studied
Interventions
- GENETIC zocaglusagene nuzaparvovec
Study Locations (4)
California
- University of California Irvine, Department of Neurology - Orange
- Stanford University - Palo Alto
Utah
- University of Utah, Division of Medical Genetics - Salt Lake City
Other
- Newcastle Upon Tyne Hospitals Foundation Trust Clinical Research Facility - Newcastle upon Tyne
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 11 participants |
| Start Date | 2020-10-27 |
| Est. Completion | 2035-02-28 |
| Phase | Phase 1 |
What the registry record for NCT04174105 still lists
NCT04174105 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 11 participants, a relatively small participant target, below the 46-participant average among 8 other Pompe Disease (Late-onset) trials with a reported enrollment target (76% lower).
The record links to 1 condition, with Pompe Disease (Late-onset) appearing as the primary indexed condition, and to 1 intervention - of which zocaglusagene nuzaparvovec is the first listed.
NCT04174105 reports a single indexed study location in California, Utah, Other.
Frequently Asked Questions
What is clinical trial NCT04174105 about?
NCT04174105 is a clinical study titled "Gene Transfer Study in Patients With Late Onset Pompe Disease". This is a phase 1/2 open-label, ascending dose, multicenter clinical study to evaluate the safety and efficacy of AT845 in adult (aged ≥ 18 years) subjects, ambulatory or nonambulatory, with Late Onset Pompe Disease (LOPD).
What is the current status of trial NCT04174105?
This trial is currently active not recruiting. It is a Phase 1 study. The enrollment target is 11 participants. The study started on 2020-10-27. Estimated completion is 2035-02-28.
What conditions does trial NCT04174105 study?
This clinical trial studies the following conditions: Pompe Disease (Late-onset).
What interventions are being tested in trial NCT04174105?
The interventions under investigation include: zocaglusagene nuzaparvovec (GENETIC).
Who is sponsoring clinical trial NCT04174105?
This trial is sponsored by Astellas Gene Therapies, which has 7 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT04174105 being conducted?
This trial has 4 study locations across California, Utah. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
Similar trials for Pompe Disease (Late-onset)
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT04174105's enrollment target sits among peer trials
11 8th of 8 higher than 1 of 8 other Pompe Disease (Late-onset) trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Pompe Disease (Late-onset) 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
-
A Study to Evaluate Safety, Tolerability, and Efficacy of AB-1009 Gene Therapy (GAA Gene) in Adult Participants With Late Onset Pompe Disease (PROGRESS-GT LOPD)
RECRUITING · Phase 1
-
A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)
ACTIVE NOT RECRUITING · Phase 1
-
Pompe Pregnancy Sub-Registry
RECRUITING
-
ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD
ACTIVE NOT RECRUITING · Phase 3
-
Developing a Management Approach for Patients With "Late-Onset" Pompe Disease
ACTIVE NOT RECRUITING
-
A Study About Antibody Levels and Biomarkers in the Blood in People With Late-onset Pompe Disease
ACTIVE NOT RECRUITING · NA
Nationwide trials with similar profiles
Cross-condition peers matched on enrollment target and registry start date, not the same-condition list above.
Similar enrollment target
- NCT03114462 · 11 participants · Phase 1
Trial of Stereotactic HYpofractionateD RadioAblative (HYDRA) Treatment of Laryngeal Cancer
- NCT06039709 · 11 participants · Phase 1
Sonodynamic Therapy in Patients With Recurrent GBM
- NCT06425380 · 11 participants · NA
Pilot Open-Label Trial of Resistant Potato Starch in Patients With Cirrhosis and Overt Hepatic Encephalopathy
- NCT06472661 · 11 participants · Early Phase 1
FUSA With Intratumoral PolyICLC in Regionally Advanced Resectable Melanoma
Similar registry start date
- NCT05962346 · started 2026-12 · NA
Fetal Endoscopic Tracheal Occlusion for Congenital Diaphragmatic Hernia
- NCT07125183 · started 2026-12 · Phase 2
Study on Efficacy and Tolerability of Weekly Doxorubicin in Elderly Patients With Advanced or Metastatic Leiomyosarcoma
- NCT07292298 · started 2026-11 · Phase 2
Phase 2 Single-Arm Rectal Cancer Brachytherapy for Patients With Low-Lying Residual Adenocarcinoma After Total Neoadjuvant Therapy to Improve Organ Preservation Rates
- NCT04263285 · started 2026-10 · NA
Treatment of Depression Post-SCI