Orbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Participants With Pelizaeus Merzbacher Disease (PMD)
← catalyst calendarNCT06150716 · readout ≤ 680 d
Sponsored by Ionis Pharmaceuticals, Inc. (industry) · IONS — their whole pipeline →.
Phase
Phase 1
Status
Recruiting
Study type
Interventional
Design
Randomized · None · Treatment
Enrollment
24estimated
Sites
7
Countries
France, Germany, Japan +2
Every value on this page is a field the sponsor filed with ClinicalTrials.gov, reproduced. Dates are their own estimates, revised as a study runs, unless the registry marks them actual. sources →
Source: ClinicalTrials.gov, retrieved 2026-08-19
Dates
each axis at the precision it was filed, with the registry's own basis| Date | Filed | Basis | What it is |
|---|---|---|---|
| Start | 2024-04-10 | actual | When the study began enrolling |
| Primary completion | Jun 2028 | estimated | The date the last participant is measured for the primary outcome — the readout window |
| Study completion | Jun 2028 | estimated | The whole study's end, after follow-up |
| First posted | 2023-11-29 | actual | When this record first appeared on the registry |
| Results posted | — | When the sponsor posted results to the registry | |
| Record updated | 2025-12-12 | actual | The sponsor's own last edit to this record — every projection above is as current as this date |
What it studies
Condition
- Pelizaeus-Merzbacher Disease
Intervention
- Drug: ION356
Primary outcomes
what the primary-completion date above is the date OFNumber of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious TEAEs
measured Up to Week 145
Number of Participants With Clinically Significant Change from Baseline in Laboratory Assessments
measured Baseline up to Week 145
Number of Participants With Clinically Significant Change From Baseline in Neurological Examination Findings
measured Baseline up to Week 145
Number of Participants With Clinically Significant Change From Baseline in Vital Signs
measured Baseline up to Week 145
Number of Participants With Clinically Significant Change From Baseline in Electrocardiography (ECG)
measured Baseline up to Week 145
Number of Participants With Change From Baseline in Concomitant Medication Use
measured Baseline up to Week 145
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