Orbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Participants With Pelizaeus Merzbacher Disease (PMD)
Public ClinicalTrials.gov record NCT06150716. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
A Phase 1b Study to Evaluate the Safety, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION356 in Patients With Pelizaeus Merzbacher Disease
Study identification
- NCT ID
- NCT06150716
- Recruitment status
- Recruiting
- Study type
- Interventional
- Phase
- Phase 1
- Lead sponsor
- Ionis Pharmaceuticals, Inc.
- Industry
- Enrollment
- 24 participants
Conditions and interventions
Conditions
Interventions
- ION356 Drug
Drug
Eligibility (public fields only)
- Age range
- 2 Years to 17 Years
- Sex
- Male
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Apr 9, 2024
- Primary completion
- May 31, 2028
- Completion
- May 31, 2028
- Last update posted
- Dec 11, 2025
2024 – 2028
United States locations
- U.S. sites
- 3
- U.S. states
- 3
- U.S. cities
- 3
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Ionis Investigative Site | Atlanta | Georgia | 30342 | Recruiting |
| Ionis Investigative Site | Philadelphia | Pennsylvania | 19104 | Recruiting |
| Ionis Investigative Site | Salt Lake City | Utah | 84113 | Recruiting |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 4 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT06150716, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Dec 11, 2025 · Synced Jul 21, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT06150716 live on ClinicalTrials.gov.