NCT06150716 Orbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Participants With Pelizaeus Merzbacher Disease (PMD)
| NCT ID | NCT06150716 |
| Status | Recruiting |
| Phase | Phase 1 |
| Sponsor | Ionis Pharmaceuticals, Inc. |
| Condition | Pelizaeus-Merzbacher Disease |
| Study Type | INTERVENTIONAL |
| Enrollment | 24 participants |
| Start Date | 2024-04-10 |
| Primary Completion | 2028-06 |
Eligibility & Interventions
Eligibility Fast-Check
Enter your details for a quick preliminary check. This does not replace medical advice.
What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 1 is the earliest stage of human testing — safety and dosage are the primary focus. Visits are frequent and medical supervision is intensive. You will be among the first people to receive this treatment.
This trial targets 24 participants in total. It began in 2024-04-10 with a primary completion date of 2028-06.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
The primary purpose of this study is to evaluate the safety and tolerability of ION356.
Eligibility Criteria
Key Inclusion Criteria 1. 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. 2. Diagnosis of PMD with genetic confirmation of PLP1 gene duplication. 3. Clinical phenotype and brain imaging consistent with a diagnosis of PMD. 4. Male between the ages of 2 and 17 years, inclusive, at the time of informed consent. 5. 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 1. Clinically significant abnormalities in medical history, laboratory tests or physical examination. 2. Unwillingness to comply with study procedures, including follow-up, as specified by this protocol, or unwillingness to cooperate fully with the Investigator. 3. Any contraindication or unwillingness to undergo magnetic resonance imaging (MRI). 4. 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. 5. 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). 6. History of gene therapy or cell transplantation, or any experimental brain surgery. 7. Current obstructive hydrocephalus. 8. Known brain or spinal disease or previous spinal surgery that would interfere with the lumbar puncture (LP) process, CSF circulation, or safety assessment. 9. Hospitalization for any major medical or surgical procedure involving general anesthesia within 12 weeks prior to Screening or planned during the study. 10. 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.
Contact & Investigator
Ionis Pharmaceuticals, Inc.
✉ IonisPelizaeusMerzbacherStudy2@clinicaltrialmedia.com📞 (844) 387-9520
Frequently Asked Questions
Who can join the NCT06150716 clinical trial?
This trial is open to male participants only, aged 2 Years or older, up to 17 Years, studying Pelizaeus-Merzbacher Disease. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT06150716 trial and what does that mean for participants?
Phase 1 trials are the first stage of human testing. The primary goal is to assess safety and determine appropriate dosage levels. Participants are closely monitored. These trials typically involve a small number of volunteers.
Is NCT06150716 currently recruiting?
Yes, NCT06150716 is actively recruiting participants. Contact the research team at IonisPelizaeusMerzbacherStudy2@clinicaltrialmedia.com for enrollment information.
Where is the NCT06150716 trial being conducted?
This trial is being conducted at Atlanta, United States, Philadelphia, United States, Salt Lake City, United States, Le Kremlin-Bicêtre, France and 3 additional locations.
Who is sponsoring the NCT06150716 clinical trial?
NCT06150716 is sponsored by Ionis Pharmaceuticals, Inc.. The trial plans to enroll 24 participants.