A Study to Evaluate the Safety and Efficacy of Zilganersen (ION373) in Patients With Alexander Di… (NCT04849741) | Clinical Trial Compass
Active — Not RecruitingPhase 3
A Study to Evaluate the Safety and Efficacy of Zilganersen (ION373) in Patients With Alexander Disease (AxD)
United States, Australia54 participantsStarted 2021-06-01
Plain-language 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.
Who can participate
Age range2 Years – 65 Years
SexALL
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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
✓. 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)
Exclusion criteria
✕. 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).
✕
What they're measuring
1
Percent Change from Baseline in the 10-Meter Walk Test (10MWT)
. 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