Trial Search Results
A Study to Evaluate the Safety and Efficacy of ION373 in Patients With Alexander Disease (AxD)
The purpose of this study is to evaluate the safety and efficacy of ION373 in improving or stabilizing gross motor function across the full range of affected domains in patients with AxD.
Stanford is currently accepting patients for this trial.
Ionis Pharmaceuticals, Inc.
- Drug: ION373
- Drug: Placebo
Key Inclusion Criteria:
1. Clinical phenotype and brain imaging consistent with a diagnosis of Alexander disease
2. Documented genetic mutation in the GFAP gene
3. Aged ≥ 2 to 65 years old at the time of informed consent
4. Able and willing to meet all study requirements, including travel to Study Center,
procedures, measurements and visits
5. Patients < 18 years old at Screening must have a trial partner (parent, caregiver or
Key Exclusion Criteria:
1. Clinically significant abnormalities in medical history or physical examination
2. Any clinically significant laboratory abnormalities that would render a patient
unsuitable for inclusion
3. Any contraindication or unwillingness to undergo MRI
4. 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
5. 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).
6. History of gene therapy or cell transplantation or any other experimental brain
7. Obstructive hydrocephalus
8. Presence of a functional ventriculoperitoneal shunt for the drainage of cerebrospinal
fluid (CSF) or an implanted central nervous system (CNS) catheter
9. Known brain or spinal disease that would interfere with the lumbar puncture (LP)
process, CSF circulation or safety assessment.
10. Hospitalization for any major medical or surgical procedure involving general
anesthesia within 12 weeks prior to Screening or planned during the study
11. 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
Ages Eligible for Study
2 Years - 65 Years
Genders Eligible for Study