Actively Recruiting
Orbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Participants With Pelizaeus Merzbacher Disease (PMD)
Led by Ionis Pharmaceuticals, Inc. · Updated on 2025-12-12
24
Participants Needed
7
Research Sites
216 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
The primary purpose of this study is to evaluate the safety and tolerability of ION356.
CONDITIONS
Official Title
Orbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Participants With Pelizaeus Merzbacher Disease (PMD)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Participant's parent or legally accepted representative can provide informed consent, attend all scheduled study visits, provide feedback regarding the participant's symptoms, and can comply with all study requirements.
- Diagnosis of Pelizaeus Merzbacher Disease with genetic confirmation of PLP1 gene duplication.
- Clinical phenotype and brain imaging consistent with a diagnosis of Pelizaeus Merzbacher Disease.
- Male between the ages of 2 and 17 years, inclusive, at the time of informed consent.
- Able and willing to meet all study requirements, including travel to Study Center, procedures, measurements, and visits.
You will not qualify if you...
- Clinically significant abnormalities in medical history, laboratory tests or physical examination.
- Unwillingness to comply with study procedures, including follow-up, 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 within 4 months of Screening if a single dose was received, or within 12 months if multiple doses were received (does not apply to vaccines).
- History of gene therapy, cell transplantation, or experimental brain surgery.
- Current obstructive hydrocephalus.
- Known brain or spinal disease or previous spinal surgery that would interfere with lumbar puncture, 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.
- Any other conditions that would make the participant unsuitable or interfere with study participation, in the opinion of the Investigator.
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 7 locations
1
Ionis Investigative Site
Atlanta, Georgia, United States, 30342
Actively Recruiting
2
Ionis Investigative Site
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
3
Ionis Investigative Site
Salt Lake City, Utah, United States, 84113
Actively Recruiting
4
Ionis Investigative Site
Le Kremlin-Bicêtre, France, 94270
Actively Recruiting
5
Ionis Investigative Site
Göttingen, Germany, 37075
Actively Recruiting
6
Ionis Investigative Site
Tokyo, Japan, 187-8551
Actively Recruiting
7
Ionis Investigative Site
Amsterdam, Netherlands, 1105AZ
Actively Recruiting
Research Team
I
Ionis Pharmaceuticals, Inc.
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
2
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here