ASCEND: Safety and Tolerability of ION337 for the Treatment of Dravet Syndrome
NCT07531745 · Status: RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 32
Last updated 2026-05-20
Summary
The primary purpose of this study is to evaluate the safety and tolerability of ION337 in participants with Dravet syndrome (DS).
Conditions
Interventions
- DRUG
-
ION337
ION337 will be administered by ITB injection.
Sponsors & Collaborators
-
Ionis Pharmaceuticals, Inc.
lead INDUSTRY
Study Design
- Allocation
- NON_RANDOMIZED
- Purpose
- TREATMENT
- Masking
- NONE
- Model
- SEQUENTIAL
Eligibility
- Min Age
- 2 Years
- Max Age
- 12 Years
- Sex
- ALL
- Healthy Volunteers
- No
Timeline & Regulatory
- Start
- 2026-04-30
- Primary Completion
- 2030-12-31
- Completion
- 2030-12-31
- FDA Drug
- Yes
Countries
- United States
Study Locations
More Related Trials
-
A Clinical Trial of Elsunersen in Pediatric SCN2A-DEE to Assess Efficacy and Safety
NCT07019922 ·Status: RECRUITING ·Phase: PHASE3
-
Evaluating The Efficacy And Safety Of Donepezil Hydrochloride (HCl) (Aricept) In Treating Cognitive Dysfunction Exhibited By Children With Down Syndrome
NCT00570128 ·Status: COMPLETED ·Phase: PHASE2
-
Multiple-dose,Double-blind,Placebo-controlled Study of Sodium Oxybate in Patients With Essential Tremor
NCT00598078 ·Status: COMPLETED ·Phase: PHASE2
-
Evaluating The Safety Of Donepezil Hydrochloride (Aricept) For Up To 1 Year In The Treatment Of The Cognitive Dysfunction Exhibited By Children With Down Syndrome - Follow-Up To A 10-Week, Double-Blind, Placebo-Controlled Trial
NCT00675025 ·Status: TERMINATED ·Phase: PHASE2
-
A Study Evaluating Soticlestat in Participants With Dravet Syndrome or Lennox-Gastaut Syndrome Who Have Been Exposed to Fenfluramine
NCT06422377 ·Status: TERMINATED ·Phase: PHASE3
-
Treatment Plan to Provide Expanded Access to Stiripentol for Patients With Dravet Syndrome
NCT01983722 ·Status: APPROVED_FOR_MARKETING
-
Phase 1b Study of Tizanidine in Pediatric Patients With Cerebral Palsy
NCT01405950 ·Status: TERMINATED ·Phase: PHASE1
-
Pharmacokinetics and Safety of Givinostat in DMD Patients Ages From at Least 2 Years to Less Then 6 Years Old
NCT06769633 ·Status: RECRUITING ·Phase: PHASE2
-
NB-001 in Children and Adolescents With 22q11 Deletion Syndrome
NCT05290493 ·Status: COMPLETED ·Phase: PHASE2
-
Carisbamate in Adult & Pediatric Subjects With Lennox-Gastaut Syndrome
NCT03731715 ·Status: COMPLETED ·Phase: PHASE1
-
Longitudinal Study of Phenotypic and Developmental Severity in Patients With Dravet Syndrome With SCN1A Gene Mutation
NCT07251673 ·Status: RECRUITING
-
Low and High Dose Zonisamide in Children as Monotherapy
NCT01127165 ·Status: COMPLETED ·Phase: PHASE4
-
A Phase 3, Placebo-Controlled Study to Investigate LP352 in Children and Adults With Dravet Syndrome (DS)
NCT06660394 ·Status: RECRUITING ·Phase: PHASE3
-
Evaluating The Efficacy And Safety Of Donepezil Hydrochloride (Aricept) In The Treatment Of The Cognitive Dysfunction Exhibited By Children With Down Syndrome, Aged 11 To 17
NCT00754052 ·Status: TERMINATED ·Phase: PHASE3
-
Opsoclonus Myoclonus Syndrome/Dancing Eye Syndrome (OMS/DES) in Children With and Without Neuroblastoma (NBpos and NBneg)Opsoclonus Myoclonus Syndrome/Dancing Eye Syndrome (OMS/DES) in Children With and Without Neuroblastoma (NBpos and NBneg)
NCT01868269 ·Status: COMPLETED ·Phase: PHASE3
-
Verapamil as Therapy for Children and Young Adults With Dravet Syndrome
NCT01607073 ·Status: COMPLETED ·Phase: PHASE2
-
Evaluating The Efficacy And Safety Of Donepezil Hydrochloride (Aricept) In The Treatment Of The Cognitive Dysfunction Exhibited By Children With Down Syndrome, Aged 6 To 10
NCT00754013 ·Status: TERMINATED ·Phase: PHASE3
-
BMB-101 in Absence Epilepsy and DEE
NCT06401538 ·Status: RECRUITING ·Phase: PHASE2
-
A 4-Week Safety Study of Oral ELND005 in Young Adults With Down Syndrome Without Dementia
NCT01791725 ·Status: COMPLETED ·Phase: PHASE2
-
A Double-blind, Placebo-controlled Comparative Study and Open-label Extension Study to Confirm the Efficacy and Safety of E2020 in Subjects With Down Syndrome Having Regression Symptoms and Disabled Activities of Daily Living.
NCT02094053 ·Status: COMPLETED ·Phase: PHASE2
-
A Phase 2 Study of JNT-517 in Adolescent Participants With Phenylketonuria
NCT06637514 ·Status: COMPLETED ·Phase: PHASE2
-
A Single-Stage, Adaptive, Open-label, Dose Escalation Safety and Efficacy Study of AADC Deficiency in Pediatric Patients
NCT02852213 ·Status: RECRUITING ·Phase: PHASE1
-
A Study of RO5186582 in Down Syndrome Among Children 6 to 11 Years of Age
NCT02484703 ·Status: TERMINATED ·Phase: PHASE2
-
Cardiomyopathy in DMD: Lisinopril vs. Losartan
NCT01982695 ·Status: COMPLETED ·Phase: NA
-
A Clinical Study to Evaluate the Safety and Efficacy of ETX101, an AAV9-Delivered Gene Therapy in Children With SCN1A-positive Dravet Syndrome
NCT06283212 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE1/PHASE2