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NCT06150716 · ClinicalTrials.gov registry record · Phase 1
Orbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Participants With Pelizaeus Merzbacher Disease (PMD)
A Phase 1 study of Pelizaeus-Merzbacher Disease, sponsored by Ionis Pharmaceuticals.
- Recruiting
- Registry status
- Phase 1
- Development phase
- 24
- Enrollment target
- 7
- Study locations
NCT06150716: Recruiting Phase 1 study of Pelizaeus-Merzbacher Disease, sponsored by Ionis Pharmaceuticals.
NCT06150716 is a Phase 1 study of Pelizaeus-Merzbacher Disease that is actively recruiting participants, run by Ionis Pharmaceuticals. The registered enrollment target is 24 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (60% lower). The trial reports 7 study locations across 4 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT06150716, a Phase 1 study of Pelizaeus-Merzbacher Disease, is actively recruiting participants, sponsored by Ionis Pharmaceuticals.
- RECRUITING
- Registry status
- Phase 1
- Development phase
- 24 participants
- Enrollment target
- 7
- Study locations
Study Summary
The primary purpose of this study is to evaluate the safety and tolerability of ION356.
Conditions Studied
Interventions
- DRUG ION356
Study Locations (7)
Other
- Ionis Investigative Site - Le Kremlin-Bicêtre
- Ionis Investigative Site - Göttingen
- Ionis Investigative Site - Tokyo
- Ionis Investigative Site - Amsterdam
Georgia
- Ionis Investigative Site - Atlanta
Pennsylvania
- Ionis Investigative Site - Philadelphia
Utah
- Ionis Investigative Site - Salt Lake City
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 24 participants |
| Start Date | 2024-04-10 |
| Est. Completion | 2028-06 |
| Phase | Phase 1 |
What NCT06150716 shows while recruiting
NCT06150716 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 24 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (60% lower).
The record links to 1 condition, with Pelizaeus-Merzbacher Disease appearing as the primary indexed condition, and to 1 intervention - of which ION356 is the first listed.
NCT06150716 lists 7 locations in 4 states (Other, Georgia, Pennsylvania).
Frequently Asked Questions
What is clinical trial NCT06150716 about?
NCT06150716 is a clinical study titled "Orbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Participants With Pelizaeus Merzbacher Disease (PMD)". The primary purpose of this study is to evaluate the safety and tolerability of ION356.
What is the current status of trial NCT06150716?
This trial is currently recruiting. It is a Phase 1 study. The enrollment target is 24 participants. The study started on 2024-04-10. Estimated completion is 2028-06.
What conditions does trial NCT06150716 study?
This clinical trial studies the following conditions: Pelizaeus-Merzbacher Disease.
What interventions are being tested in trial NCT06150716?
The interventions under investigation include: ION356 (DRUG).
Who is sponsoring clinical trial NCT06150716?
This trial is sponsored by Ionis Pharmaceuticals, which has 76 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT06150716 being conducted?
This trial has 7 study locations across Georgia, Pennsylvania, Utah. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
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Where NCT06150716's enrollment target sits among peer trials
24 1520th of 2000 higher than 405 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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