NCT06150716 · Ionis Pharmaceuticals, Inc.
Orbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Participants With Pelizaeus Merzbacher Disease (PMD)
What this study is about
The primary purpose of this study is to evaluate the safety and how well patients handle the treatment of ION356.
View original scientific description
The primary purpose of this study is to evaluate the safety and tolerability of ION356.
Interventions
DRUG
ION356
Administered as intrathecal (IT) injection.
Primary outcome measures
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious TEAEs
Time frame: Up to Week 145
Number of Participants With Clinically Significant Change from Baseline in Laboratory Assessments
Time frame: Baseline up to Week 145
Number of Participants With Clinically Significant Change From Baseline in Neurological Examination Findings
Time frame: Baseline up to Week 145
Number of Participants With Clinically Significant Change From Baseline in Vital Signs
Time frame: Baseline up to Week 145
Number of Participants With Clinically Significant Change From Baseline in Electrocardiography (ECG)
Time frame: Baseline up to Week 145
Number of Participants With Change From Baseline in Concomitant Medication Use
Time frame: Baseline up to Week 145
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- 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 PMD with genetic confirmation of PLP1 gene duplication.
- Clinical phenotype and brain imaging consistent with a diagnosis of PMD.
- Male between the ages of 2 and 17 years, inclusive, at the time of informed consent.
- Able and willing to meet all study requirements (in the opinion of the Investigator), including travel to Study Center, procedures, measurements, and visits.
Exclusion criteria
- 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
- Atlanta, Georgia
- Philadelphia, Pennsylvania
- Salt Lake City, Utah
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Dec 12, 2025 · Source of record for eligibility and locations