Alexander Disease
Conditions
Keywords
Alexander disease, leukodystrophy, expanded access
Brief summary
The purpose of the expanded access program (EAP) is to provide access to zilganersen for eligible individuals with Alexander disease (AxD).
Detailed description
This is an expanded access program (EAP) for eligible individuals diagnosed with Alexander disease residing in the United States, prior to approval of zilganersen by the local regulatory agency (FDA). A medical doctor must decide whether the potential benefits of zilganersen outweigh the potential risks based on the individual patient's medical history and program eligibility criteria. Access can be requested via the contact information below.
Interventions
DRUGzilganersen
zilganersen
Sponsors
Ionis Pharmaceuticals, Inc.
Eligibility
Sex/Gender
ALL
Age
2 Years to 99 Years
Inclusion criteria
Key Inclusion Criteria: 1. Approved drug or drugs available for treatment did not work for the patient, or the patient cannot tolerate the side effects of the FDA-approved drug or drugs for treatment of AxD. 2. Patients who are ≥ 2 years old. 3. Patients who have a clinical phenotype and brain imaging consistent with a diagnosis of AxD. 4. Patients who have a documented variant in the GFAP gene. 5. Patient resides in and is a resident of the US. Key
Exclusion criteria
1. Patients who have any medical history, physical exam findings, or clinically significant laboratory abnormalities that contraindicate performing an LP for ITB administration of zilganersen. 2. Patients who have current obstructive hydrocephalus. 3. Patients who have the presence of a functional ventriculoperitoneal shunt for the drainage of CSF. 4. Patients who have any other medical history or current conditions, which, in their Treating Physician's opinion, would make the patient unsuitable for inclusion (e.g., active Hepatitis B virus or hepatitis C virus infection, severe hepatic or renal disease, uncontrolled acute or chronic condition, etc.). 5. Patients who are pregnant or plan to become pregnant, or patients who are breastfeeding. Other inclusion/
Contacts
CONTACTIonis Pharmaceuticals, Inc.
Outcome results
None listed