Status:
ACTIVE_NOT_RECRUITING
A Study to Evaluate the Safety and Efficacy of Zilganersen (ION373) in Patients With Alexander Disease (AxD)
Lead Sponsor:
Ionis Pharmaceuticals, Inc.
Conditions:
Alexander Disease
Eligibility:
All Genders
2-65 years
Phase:
PHASE3
Brief Summary
The purpose of this study is to evaluate the safety and efficacy of zilganersen (ION373) in improving or stabilizing gross motor function across the full range of affected domains in patients with AxD...
Detailed Description
This is a Phase 1-3, multi-center, double-blind, placebo-controlled, multiple-ascending dose (MAD) study in approximately 73 patients with AxD. Participants will be randomized in a 2:1 ratio to receiv...
Eligibility Criteria
Inclusion
- Key
- Clinical phenotype and brain imaging consistent with a diagnosis of Alexander disease
- Documented genetic mutation in the GFAP gene
- Aged ≥ 2 to 65 years old at the time of informed consent
- Able and willing to meet all study requirements, including travel to Study Center, procedures, measurements and visits
- Patients \< 18 years old at Screening must have a trial partner (parent, caregiver or other)
- Key
Exclusion
- Clinically significant abnormalities in medical history or physical examination
- Any clinically significant laboratory abnormalities that would render a patient unsuitable for inclusion
- Any contraindication or unwillingness to undergo MRI
- Treatment with another investigational drug, biological agent, or device within 1 month of Screening, or 5 half-lives of investigational agent, whichever is longer; concurrent participation in any other clinical study (including observational and non-interventional studies)
- Previous treatment with an oligonucleotide (including small interfering ribonucleic acid \[siRNA\]) within 4 months of Screening if single dose received, or within 12 months of Screening if multiple doses 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 other experimental brain surgery \[ROW\]
- Obstructive hydrocephalus
- Presence of a functional ventriculoperitoneal shunt for the drainage of cerebrospinal fluid (CSF) or an implanted central nervous system (CNS) catheter
- Known brain or spinal disease 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 patient unsuitable for inclusion, or could interfere with the patient participating in or completing the study
Key Trial Info
Start Date :
June 1 2021
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
September 1 2029
Estimated Enrollment :
54 Patients enrolled
Trial Details
Trial ID
NCT04849741
Start Date
June 1 2021
End Date
September 1 2029
Last Update
November 28 2025
Active Locations (13)
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1
Lucile Packard Children's Hospital Stanford
Palo Alto, California, United States, 94304
2
Children's Hospital of Atlanta
Atlanta, Georgia, United States, 30329
3
Massachusetts General Hospital
Boston, Massachusetts, United States, 02114
4
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104