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Clinical Trials/2024-512589-32-00
2024-512589-32-00
Not yet recruiting
Phase 1/2

ION582-CS1: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION582 in Patients with Angelman Syndrome

Ionis Pharmaceuticals Inc.2 sites in 2 countries4 target enrollmentStarted: August 28, 2024Last updated:
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Overview

Phase
Phase 1/2
Status
Not yet recruiting
Enrollment
4
Locations
2
Primary Endpoint
To evaluate the safety and tolerability of single and multiple doses of ION582 (incidence, severity, and dose-relationship of adverse effects and changes in the laboratory parameters)
OverviewEligibility CriteriaOutcomesInvestigatorsSitesRecords & IdentifiersSimilar Trials

Overview

Brief Summary

To evaluate the safety and tolerability of ascending dose-levels of multiple intrathecal bolus administrations of ION582 in patients with Angelman syndrome based on incidence and severity of treatment-emergent adverse events and serious adverse events, changes in vital signs, and changes in clinical laboratory results

Eligibility Criteria

Ages
0 years to 64 years (0-17 Years, 18-64 Years)
Accepts Healthy Volunteers
Yes

Inclusion Criteria

  • •Participant has a documented and certified diagnosis of Angelman syndrome (ubiquitin-protein ligase E3A deletion or UBE3A mutation)
  • •Male or female between the ages of 0-50 years of age, with signed informed consent from parent(s) or legal guardian(s)
  • •Currently receiving stable standard of care treatments such as, stable doses of anti-epileptic medication, behavioral management medications, sleep medications, gabapentin, cannabidiol, and including special diets, supplements or nutritional support for at least 3 months prior to first dose.
  • •Follow good study practice and not participate in the sharing of personal or study information on social media platforms, such as any website or social media site (e.g., Facebook, Instagram, Twitter, YouTube, etc.) until notified that the study is completed.
  • •Other protocol-defined inclusion criteria apply

Exclusion Criteria

  • •Has documented molecular AS confirmation of paternal uniparental disomy or imprinting defect
  • •Any clinically significant cardiovascular, endocrine, hepatic, renal, pulmonary, gastrointestinal, neurologic, malignant, metabolic, psychiatric, or other condition that, in the judgment of the Investigator, will pose a safety risk, will make the patient unsuitable for participation in, and/or unable to complete the study procedures. Has poorly controlled seizures as determined by the Investigator or has documented Status Epilepticus in the past 6 months that could pose a safety risk while on study
  • •Known bone, spine, bleeding, or other disorder that exposes the patient to risk of injury or unsuccessful lumbar puncture. Previous treatment with an oligonucleotide (including small interfering ribonucleic acid, antisense oligonucleotide). COVID-19 vaccinations are allowed.
  • •Any prior use of gene therapy. Have any other conditions, which, in the opinion of the Investigator would make the participant unsuitable for inclusion or could interfere with the participant taking part in or completing the study.
  • •Other protocol-defined exclusion criteria apply

Outcomes

Primary Outcomes

To evaluate the safety and tolerability of single and multiple doses of ION582 (incidence, severity, and dose-relationship of adverse effects and changes in the laboratory parameters)

To evaluate the safety and tolerability of single and multiple doses of ION582 (incidence, severity, and dose-relationship of adverse effects and changes in the laboratory parameters)

Secondary Outcomes

  • Maximum Observed Plasma Concentration of ION582
  • Time to Reach Maximal Plasma Concentration of ION582
  • Plasma Elimination Half-Life of ION582
  • Concentration ION582 in cerebrospinal fluid

Investigators

Sponsor Class
Pharmaceutical company
Responsible Party
Principal Investigator
Principal Investigator

Global Regulatory Affairs

Scientific

Ionis Pharmaceuticals Inc.

Study Sites (2)

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