Orbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Participants With Pelizaeus Merzbacher Disease (PMD)
Recruiting now · Phase 1
Conditions studied: Pelizaeus-Merzbacher Disease
In brief
The primary purpose of this study is to evaluate the safety and tolerability of ION356.
Key facts
- Study ID
- NCT06150716
- Run by
- Ionis Pharmaceuticals, Inc.
- People needed
- 24
- Starts
- 2024-04-10
- Expected to finish
- 2028-06-01
- Last updated by the study team
- 2025-12-12
Who can join
Age: 2 and older, up to 17. Sex: male. Healthy volunteers: not accepted.
You may not qualify if…
- Clinically significant abnormalities in medical history, laboratory tests or physical examination.
- Unwillingness to comply with study procedures, including follow-up, as specified by this protocol, or unwillingness to cooperate fully with the Investigator.
- Any contraindication or unwillingness to undergo magnetic resonance imaging (MRI).
- Treatment with another investigational drug, biological agent, or device within 1 month of Screening, or 5 half-lives of the investigational agent, whichever is longer.
- Previous treatment with an oligonucleotide (including small interfering ribonucleic acid) within 4 months of Screening if a single dose was received, or within 12 months of Screening if multiple doses were received. This exclusion does not apply to vaccines (both messenger ribonucleic acid [mRNA] and viral vector vaccines).
- History of gene therapy or cell transplantation, or any experimental brain surgery.
- Current obstructive hydrocephalus.
- Known brain or spinal disease or previous spinal surgery that would interfere with the lumbar puncture (LP) process, CSF circulation, or safety assessment.
- Hospitalization for any major medical or surgical procedure involving general anesthesia within 12 weeks prior to Screening or planned during the study.
- Have any other conditions, which, in the opinion of the Investigator, would make the participant unsuitable for inclusion, or could interfere with the participant participating in or completing the study.
Where it is running
- Ionis Investigative Site — Atlanta, Georgia, United States (enrolling)
- Ionis Investigative Site — Philadelphia, Pennsylvania, United States (enrolling)
- Ionis Investigative Site — Salt Lake City, Utah, United States (enrolling)
- Ionis Investigative Site — Le Kremlin-Bicêtre, France (enrolling)
- Ionis Investigative Site — Göttingen, Germany (enrolling)
- Ionis Investigative Site — Tokyo, Japan (enrolling)
- Ionis Investigative Site — Amsterdam, Netherlands (enrolling)
Full record on ClinicalTrials.gov
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