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
This trial does not accept healthy volunteersQualifiers
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
Design model
Sequential
Treatments tested in this trial
ION337
DrugION337 will be administered by ITB injection.
Treatment groups
32 Participants
are divided into 5 treatment groupsGroup A: Part 1: Single Ascending Dose (SAD): Dose Level 1Experimental treatment 1 intervention
Group B: Part 1: SAD: Dose Level 2Experimental treatment 1 intervention
Group C: Part 1: SAD: Dose Level 3Experimental treatment 1 intervention
Group D: Part 1: SAD: Dose Level 4Experimental treatment 1 intervention
Group E: Part 2: Multiple Ascending Dose (MAD): Dose Level 1-4Experimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
Parts 1 and 2: Number of Participants with Treatment-emergent Adverse Events (TEAEs) and Serious TEAEs
Time frame
Part 1: up to 6 months; Part 2: up to 31 months2
Number of Participants With Clinically Significant Change From Baseline in Safety Laboratory Values
Time frame
Part 1: up to 6 months; Part 2: up to 31 months3
Parts 1 and 2: Number of Participants With Clinically Significant Change From Baseline in Vital Signs
Time frame
Part 1: up to 6 months; Part 2: up to 31 months4
Parts 1 and 2: Number of Participants With Clinically Significant Change From Baseline in Electrocardiogram (ECG)
Time frame
Part 1: up to 6 months; Part 2: up to 31 monthsSecondary outcomes
1
Pharmacokinetic (PK) Parameters Measure Description: Analysis of plasma concentrations of ION337
Time frame
Pre-Dose Day 1 (Dosing) until 6 months after dosing in Part 1 and up to 31 months in Part 22
Exposure of ION337 in Cerebrospinal Fluid (CSF) Measure Description: Measurement of ION337 concentrations
Time frame
Pre-dose Day 1 (Dosing) until 6 months after dosing in Part 1 and up to 31 months in Part 23
Parts 1 and 2: Percent Change From Baseline in 28-day Normalized Major Motor Seizure (MMS) Frequency
Time frame
Part 1: up to 6 months; Part 2: up to 31 monthsSponsors and contacts
Click on the lead sponsor to view all of their trials.
Source ClinicalTrials.gov