Pelizaeus-Merzbacher Disease Trials in the USA

Every industry-sponsored study on the ClinicalTrials.gov registry that lists a US site and is open, about to open, active-but-closed, or enrolling by invitation. This deliberately excludes academic and other non-industry sponsors — the full US registry across all sponsors runs to over 22,000 recruiting studies alone.

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Source: ClinicalTrials.gov. Complete coverage of the industry-sponsored slice — all 9094 matching records are in this view (non-industry sponsors are out of scope by design, not truncated). Synced 17 Sep 2026, 08:23 UTC

9094

Studies in scope

industry-sponsored, all four open statuses

4935

Recruiting now

open to new participants

2188

Lead sponsors

across recruiting studies

Top states

Georgia 2 Pennsylvania 2 Utah 1

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

NCT06150716Pelizaeus-Merzbacher Disease

Ionis Pharmaceuticals, Inc.

Phase 1

Georgia, Pennsylvania, Utah

2024-04-10

Recruiting

Rocket Study: A Study to Characterize Biomarkers and Disease Progression in Participants With Pelizaeus-Merzbacher Disease

NCT05659901Pelizaeus-Merzbacher Disease

Ionis Pharmaceuticals, Inc.

Observational

Georgia, Pennsylvania

2022-10-03

Cite this view

Priya Life Science (2026). Clinical trial register: industry-sponsored studies with a US investigative site, filtered. Derived from ClinicalTrials.gov. Retrieved 17 September 2026. https://priyalifescience.com/clinical-trials-usa/condition/pelizaeus-merzbacher-disease

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Listing here is informational and not medical advice; eligibility is determined by each study team. Compare pay for the teams running these studies in the US Pharma Salary Guide, see who's behind them in the US Company Directory, or track approvals on the FDA Approvals Tracker. For Irish, UK and EU sites, see the Ireland, UK and EU registers.