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NCT02898753 · ClinicalTrials.gov registry record · Phase 1
VAL-1221 Delivered Intravenously in Ambulatory and Ventilator-free Participants With Late-Onset Pompe Disease
A Phase 1 study, sponsored by Valerion Therapeutics.
- Terminated
- Registry status
- Phase 1
- Development phase
- 12
- Enrollment target
NCT02898753: Clinical Trial Phase 1 study, sponsored by Valerion Therapeutics.
NCT02898753 is a Phase 1 study that was terminated before completion, run by Valerion Therapeutics. The registered enrollment target is 12 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (80% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT02898753, a Phase 1 study, was terminated before completion, sponsored by Valerion Therapeutics.
- TERMINATED
- Registry status
- Phase 1
- Development phase
- 12 participants
- Enrollment target
Study Summary
This Phase I/II open-label, randomized, dose-escalation study will assess the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficacy of VAL-1221 versus Myozyme®/Lumizyme® in participants with late-onset glycogen storage disease-II (GSD-II) (Pompe disease)
Interventions
- DRUG VAL-1221
- DRUG RhGAA
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 12 participants |
| Start Date | 2017-06-21 |
| Est. Completion | 2020-03-25 |
| Phase | Phase 1 |
Why NCT02898753 stopped before completion
NCT02898753 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 12 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (80% lower).
The record links to 0 conditions, and to 2 interventions - of which VAL-1221 is the first listed.
NCT02898753 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT02898753 about?
NCT02898753 is a clinical study titled "VAL-1221 Delivered Intravenously in Ambulatory and Ventilator-free Participants With Late-Onset Pompe Disease". This Phase I/II open-label, randomized, dose-escalation study will assess the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficacy of VAL-1221 versus Myozyme®/Lumizyme® in participants with late-onset glycogen storage disease-II (GSD-II) (Pompe disease)
What is the current status of trial NCT02898753?
This trial is currently terminated. It is a Phase 1 study. The enrollment target is 12 participants. The study started on 2017-06-21. Estimated completion is 2020-03-25.
What interventions are being tested in trial NCT02898753?
The interventions under investigation include: VAL-1221 (DRUG), RhGAA (DRUG).
Who is sponsoring clinical trial NCT02898753?
This trial is sponsored by Valerion Therapeutics, which has 2 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT02898753's enrollment target sits among peer trials
12 1852nd of 2000 higher than 90 of 2,000 other Phase 1 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 1 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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