Alexander Disease| zilganersen
A Study to Evaluate the Safety and Efficacy of ION373 in Patients With Alexander Disease (AxD)
This is a Phase 1-3, multi-center, double-blind, placebo-controlled, multiple-ascending dose (MAD) study in up to 58 patients with AxD. Participants will be randomized in a 2:1 ratio to receive ION373 or matching placebo for a 60-week double-blind treatment period; then all participants will receive ION373 for a 60-week open-label treatment period.
zilganersen
Purpose of Study
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.
Eligibility
Age:
2 - 65Sex:


Key Inclusion / Exclusion Criteria
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).
- 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
Conditions:
Alexander Disease
Status:
Recruiting
Phase:
Phase 3
Clinical Trials ID
NCT04849741
Read Detailed Summary:
Start Date / End Date:
06 2021 - 03 2029
Intervention:
zilganersen
Enrollment:
73
Full Details:
14 Study Locations
United States
Lucile Packard Children’s Hospital Stanford
Palo Alto, California, 94304
United States
contact: Erika Shols
email: [email protected]
phone: 650-272-8986
Children’s Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
United States
contact: Amy T Waldman, MD, MSCE
email: [email protected]
phone: 267-425-2063
Australia
Murdoch Children’s Research Institute
Parkville, Victoria, 3052
Australia
contact: Eloise Uebergang
email: [email protected]
phone: +61383416382
Canada
McGill University Health Centre
Montreal, Quebec, H3A 0G4
Canada
email: [email protected]
phone: 514-412-4400 ext 23380
Israel
Pediatric Neurology Institute, Dana–Dwek Children’s Hospital, Tel Aviv Sourasky Medical Center
Tel Aviv, 6423906
Israel
contact: Ayelet Zerem
email: [email protected]
phone: +972-3-6974115
Italy
Japan
Netherlands
Amsterdam Universitair Medische Centra – Academisch Medisch Centrum
Amsterdam, Noord-Holland, 1105 AZ
Netherlands
contact: Marjo van der Knaap
email: [email protected]
phone: +31205667508