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NCT04421677 · ClinicalTrials.gov registry record · Phase 1
Safety and Tolerability of Phenylbutyrate in Inclusion Body Myositis
A Phase 1 study, sponsored by University of Kansas Medical Center.
- Completed
- Registry status
- Phase 1
- Development phase
- 10
- Enrollment target
NCT04421677: Completed Phase 1 study, sponsored by University of Kansas Medical Center.
NCT04421677 is a Phase 1 study that has completed, run by University of Kansas Medical Center. The registered enrollment target is 10 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (83% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04421677, a Phase 1 study, has completed, sponsored by University of Kansas Medical Center.
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 10 participants
- Enrollment target
Study Summary
This is a pilot study (phase 1 clinical trial) to evaluate the safety and tolerability of phenylbutyrate in IBM. In this open label study, 10 patients with sporadic inclusion body myositis will be treated with phenylbutyrate (3 gm twice daily) for 3 months. There will be a run-in period, during which certain biomarkers will be measured at baseline and at the end of the run-in period in addition to final measurement at the end of the treatment period.
Primary Outcome
Since this is a Phase I pilot trial, the primary outcome will be testing the safety and tolerability of phenylbutyrate in patients with Inclusion Body Myositis. This will be measured by the incidence of adverse events reported in subjects throughout the treatment period of the trial.
Interventions
- DRUG Phenylbutyrate Oral Tablet
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 10 participants |
| Start Date | 2020-08-20 |
| Est. Completion | 2022-01-20 |
| Phase | Phase 1 |
What the finished NCT04421677 record still lists
NCT04421677 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 10 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (83% lower).
The record links to 0 conditions, and to 1 intervention - of which Phenylbutyrate Oral Tablet is the first listed.
NCT04421677 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT04421677 about?
NCT04421677 is a clinical study titled "Safety and Tolerability of Phenylbutyrate in Inclusion Body Myositis". This is a pilot study (phase 1 clinical trial) to evaluate the safety and tolerability of phenylbutyrate in IBM. In this open label study, 10 patients with sporadic inclusion body myositis will be treated with phenylbutyrate (3 gm twice daily) for 3 months. There will be a run-in period, during whic...
What is the current status of trial NCT04421677?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 10 participants. The study started on 2020-08-20. Estimated completion is 2022-01-20.
What interventions are being tested in trial NCT04421677?
The interventions under investigation include: Phenylbutyrate Oral Tablet (DRUG).
Who is sponsoring clinical trial NCT04421677?
This trial is sponsored by University of Kansas Medical Center, which has 408 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT04421677's enrollment target sits among peer trials
10 1913th of 2000 higher than 46 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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