ASCEND: Safety and Tolerability of ION337 for the Treatment of Dravet Syndrome
ConditionDravet Syndrome
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age2-12
SponsorIonis Pharmaceuticals, Inc.
About this trial
The primary purpose of this study is to evaluate the safety and tolerability of ION337 in participants with Dravet syndrome (DS).
Eligibility criteria
Qualifiers
Participant is aged ≥ 2 to ≤ 12 years old at the time of informed consent.
Participant has at least 1 parent or caregiver ≥ 18 years old who is willing and able to provide informed consent (signed and dated) and attend all scheduled study visits.
Has a documented diagnosis of DS according to the International League Against Epilepsy (ILAE) criteria and as agreed by the Epilepsy Study Consortium, Inc (ESCI).
Has confirmation of a pathogenic or likely pathogenic SCN1A variant.
Disqualifiers
Known brain or spinal disease that would interfere with the LP procedure or CSF circulation, or presence of other factors that would affect the safety of the LP procedure.
Pathogenic or likely pathogenic variant in another gene that causes epilepsy.
Has had prior treatment with or is currently enrolled in an interventional clinical trial for a gene therapy or for another antisense oligonucleotide (ASO) for the treatment of DS.
Has had treatment with or is currently enrolled in an interventional clinical trial of any other investigational drug, biological agent, or device within 30 days prior to Screening, or 5 half-lives of investigational agent, whichever is longer.
Trial design
Treatments tested in this trial
- ION337
Treatment groups
32 Participants
are divided into 5 treatment groupsSponsors and collaborators
Source ClinicalTrials.gov