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Phase 1b Study of ION356 to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics in Patients with Pelizaeus-Merzbacher Disease
EU CTIS ID: 2022-502432-39-00
What this study is testing
To evaluate the safety and tolerability of ION356
- Human Pharmacology (Phase I)- First administration to humans
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- 1. Diagnosis of PMD with genetic confirmation of PLP1 gene duplication with molecular confirmation by a CLIA, CE-marked, or equivalent laboratory provided by the Investigator at Screening.
- 2. Clinical phenotype and brain imaging consistent with a diagnosis of PMD
- 3. Male between the ages of 2 and 17 years, inclusive, at the time of informed consent
- 4. Able and willing to meet all study requirements (in the opinion of the Investigator), including travel to Study Center, procedures, measurements, and visits
You likely can't join if
- 01. > 2 copies of the PLP1 gene
- 10. Known brain or spinal disease or previous spinal surgery that would interfere with the LP process, CSF circulation, or safety assessment, including tumors or abnormalities by MRI or computed tomography, subarachnoid hemorrhage, spinal stenosis or curvature, Chiari malformation, syringomyelia, tethered spinal cord syndrome, and connective tissue disorders such as Ehlers-Danlos syndrome and Marfan syndrome
- 11. History of severe post-LP headache and/or blood patch
- 12. Hospitalization for any major medical or surgical procedure involving general anesthesia within 12 weeks prior to Screening or planned during the study
- 13. Have any other conditions, that, in the opinion of the Investigator, would make the participant unsuitable for inclusion, or could interfere with participation in or completion of the study
- 02. Clinically significant abnormalities in medical history (e.g., previous acute coronary syndrome or evidence of renal impairment within 6 months of Screening, major surgery within 3 months of Screening) or physical examination
See the full eligibility criteria
Who can join
- 1. Diagnosis of PMD with genetic confirmation of PLP1 gene duplication with molecular confirmation by a CLIA, CE-marked, or equivalent laboratory provided by the Investigator at Screening.
- 2. Clinical phenotype and brain imaging consistent with a diagnosis of PMD
- 3. Male between the ages of 2 and 17 years, inclusive, at the time of informed consent
- 4. Able and willing to meet all study requirements (in the opinion of the Investigator), including travel to Study Center, procedures, measurements, and visits
What rules you out
- 01. > 2 copies of the PLP1 gene
- 10. Known brain or spinal disease or previous spinal surgery that would interfere with the LP process, CSF circulation, or safety assessment, including tumors or abnormalities by MRI or computed tomography, subarachnoid hemorrhage, spinal stenosis or curvature, Chiari malformation, syringomyelia, tethered spinal cord syndrome, and connective tissue disorders such as Ehlers-Danlos syndrome and Marfan syndrome
- 11. History of severe post-LP headache and/or blood patch
- 12. Hospitalization for any major medical or surgical procedure involving general anesthesia within 12 weeks prior to Screening or planned during the study
- 13. Have any other conditions, that, in the opinion of the Investigator, would make the participant unsuitable for inclusion, or could interfere with participation in or completion of the study
- 02. Clinically significant abnormalities in medical history (e.g., previous acute coronary syndrome or evidence of renal impairment within 6 months of Screening, major surgery within 3 months of Screening) or physical examination
- 03. Unwillingness to comply with study procedures, including follow-up, as specified by this protocol, or unwillingness to cooperate fully with the Investigator
- 4. Any contraindication or unwillingness to undergo MRI (e.g., metal implants, claustrophobia, agitation, or motor symptoms of a severity to preclude MRI scans).
- 05. 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
- 06. 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; or history of hypersensitivity to ION356 or its excipients; or history of hypersensitivity to any ASO. This exclusion does not apply to vaccines (both mRNA and viral vector vaccines)
- 07. History of gene therapy or cell transplantation, or any experimental brain surgery
- 08. Current obstructive hydrocephalus
- 09. Presence of a functional ventriculoperitoneal shunt for the drainage of CSF or an implanted CNS catheter
The study team makes the final eligibility decision.
Where it's taking place
- United States
- Japan
- Australia
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling male, 0-17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include United States; Japan; Australia. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.