Phase 1b Study of ION356 to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics in Patients with Pelizaeus-Merzbacher Disease

Phase 1

• Conditions: Pelizaeus-Merzbacher Disease; MedDRA version: 20.0Level: PTClassification code: 10067610Term: Pelizaeus-Merzbacher disease Class: 100000004850; Therapeutic area: Diseases [C] - Nervous System Diseases [C10]; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

• Registration Number: CTIS2022-502432-39-00
• Lead Sponsor: Ionis Pharmaceuticals Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2023-07-12
• Last Update Posted: 22 July 2024

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: Male
• Target Recruitment: 73

Inclusion Criteria

1. Previous diagnosis of PMD with genetic confirmation of PLP1 gene duplication with molecular confirmation by a CLIA, CE-marked, or equivalent lab 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

Exclusion Criteria

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, which, 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, 04. Any contraindication or unwillingness to undergo MRI (e.g., metal implants, claustrophobia, agitation, or motor symptoms of a severity that precludes 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

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified