# Orbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Participants With Pelizaeus Merzbacher Disease (PMD)

> **NCT06150716** · PHASE1 · RECRUITING · sponsor: **Ionis Pharmaceuticals, Inc.** · enrollment: 24 (estimated)

## Conditions studied

- Pelizaeus-Merzbacher Disease

## Interventions

- **DRUG:** ION356

## Key facts

- **NCT ID:** NCT06150716
- **Lead sponsor:** Ionis Pharmaceuticals, Inc.
- **Sponsor class:** INDUSTRY
- **Phase:** PHASE1
- **Study type:** INTERVENTIONAL
- **Status:** RECRUITING
- **Start date:** 2024-04-10
- **Primary completion:** 2028-06
- **Final completion:** 2028-06
- **Target enrollment:** 24 (ESTIMATED)
- **Last updated:** 2025-12-12


## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT06150716

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT06150716, "Orbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Participants With Pelizaeus Merzbacher Disease (PMD)". Retrieved via AI Analytics 2026-06-23 from https://api.ai-analytics.org/clinical/NCT06150716. Licensed CC0.

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*[Clinical trials dataset](/datasets/clinical-trials) · CC0 1.0*