Clinical Trials Logo

Clinical Trial Details — Status: Recruiting

Administrative data

NCT number NCT06112275
Other study ID # ETX-DS-004
Secondary ID
Status Recruiting
Phase Phase 1/Phase 2
First received
Last updated
Start date March 2024
Est. completion date December 2029

Study information

Verified date February 2024
Source Encoded Therapeutics
Contact Encoded Patient Advocacy
Phone +1 (650) 398-4301
Email patientadvocacy@encoded.com
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

WAYFINDER is a Phase 1/2 study in Australia to evaluate the safety and efficacy of ETX101 in participants with SCN1A-positive Dravet syndrome aged 36 to <84 months (3 to <7 years). The study follows an open-label, dose-escalation design.


Recruitment information / eligibility

Status Recruiting
Enrollment 4
Est. completion date December 2029
Est. primary completion date December 2029
Accepts healthy volunteers No
Gender All
Age group 36 Months to 83 Months
Eligibility Inclusion Criteria: - Participant must have a predicted loss of function pathogenic or likely pathogenic SCN1A variant. - Participant must have experienced their first convulsive seizure between the ages of 3 and 15 months. - Participant must have a clinical diagnosis of Dravet syndrome or the treating clinician must have a high clinical suspicion of a diagnosis of Dravet syndrome. - Participant is receiving at least one prophylactic antiseizure medication. Exclusion Criteria: - Participant has another genetic mutation or clinical comorbidity which could potentially confound the typical Dravet phenotype. - Participant has a known central nervous system structural and/or vascular abnormality (indicated by an MRI or CT scan of the brain). - Participant has an abnormality that may interfere with CSF distribution and/or has an existing ventriculoperitoneal shunt. - Participant is currently taking or has taken antiseizure medications (ASMs) at a therapeutic dose that are contraindicated in Dravet syndrome, including sodium channel blockers. - Participant has experienced seizure freedom for a period of 4 consecutive weeks within the 6-month period prior to informed consent. - Participant has previously received gene or cell therapy. - Participant is currently enrolled in a clinical trial or receiving an investigational therapy. - Participant has clinically significant underlying liver disease.

Study Design


Related Conditions & MeSH terms


Intervention

Drug:
ETX101
ETX101 is composed of a non-replicating, recombinant adeno-associated viral serotype 9 (rAAV9) vector used to deliver a GABAergic regulatory element (reGABA) and an engineered transcription factor that increases transcription of the SCN1A gene (eTFSCN1A).

Locations

Country Name City State
Australia The Royal Children's Hospital Melbourne

Sponsors (1)

Lead Sponsor Collaborator
Encoded Therapeutics

Country where clinical trial is conducted

Australia, 

Outcome

Type Measure Description Time frame Safety issue
Primary Proportion of participants experiencing any treatment-emergent adverse events (AEs), serious adverse events (SAEs), related AEs, AEs with severity Grade = 3, AEs resulting in study discontinuation, and AEs with a fatal outcome. Day 1 through Study Completion, an average of 5 years
Primary Percent change from Baseline in monthly countable seizure frequency (MCSF) at Week 52, with countable seizures defined as generalized tonic-clonic/clonic, focal motor with clearly observable clinical signs, tonic bilateral, and atonic seizures. Between the 8-week baseline period and the 48-week post-dosing assessment period (defined as Week 5 to Week 52 following administration of ETX101)
Primary Proportion of participants free from episodes of prolonged seizures and/or status epilepticus at Week 52. Week 5 through Week 52
Secondary Proportion of participants with = 90% reduction in monthly countable seizure frequency (MCSF) from Baseline at Week 52. Between the 8-week baseline period and the 48-week post-dosing assessment period (defined as Week 5 to Week 52 following administration of ETX101)
Secondary Change from Baseline in the Vineland Adaptive Behavior Scales - Third Edition score at Week 52. Baseline through Week 52. Standard scores are normalized to a mean and SD of 100 and 15, respectively, and are not bounded by a range. Higher scores correspond to better outcomes.
See also
  Status Clinical Trial Phase
Recruiting NCT05651204 - GABA Biomarkers in Dravet Syndrome
Withdrawn NCT02910297 - The Pharmacokinetics of Cannabidiol (CBD) and Its Effects in Children With Severe Epilepsy
Recruiting NCT04462770 - EPX-100 (Clemizole Hydrochloride) as Add-on Therapy to Control Convulsive Seizures in Patients With Dravet Syndrome Phase 2
Completed NCT02896608 - Neuronal Excitability of HCN1 Channel Mutations in Dravet Syndrome
Withdrawn NCT05140122 - LEONIDaS Caregivers Study
Recruiting NCT05635266 - Tissue Repository Providing Annotated Biospecimens for Approved Investigator-directed Biomedical Research Initiatives
Completed NCT02091206 - A Dose-ranging Pharmacokinetics and Safety Study of GWP42003-P in Children With Dravet Syndrome (GWPCARE1) Phase 2
Enrolling by invitation NCT03655223 - Early Check: Expanded Screening in Newborns
Recruiting NCT05472389 - Neurodevelopmental Impact of Epilepsy on Autonomic Function in Dravet Syndrome N/A
Active, not recruiting NCT05626634 - Open-label, Long-term Safety Study of LP352 in Subjects With Developmental and Epileptic Encephalopathy Phase 2
Recruiting NCT01858285 - Genetics of Epilepsy and Related Disorders
Recruiting NCT04614506 - Transcranial Magnetic Stimulation to Measure Cortical Excitability in Dravet Syndrome
Recruiting NCT06118255 - A Study to Evaluate Safety, Tolerability, and Pharmacokinetics of Fenfluramine (Hydrochloride) in Infants 1 Year to Less Than 2 Years of Age With Dravet Syndrome Phase 3
Recruiting NCT04611438 - Research on Cognitive Effect of Cannabidiol on Dravet Syndrome and Lennox-Gastaut SyndromeGastaut Syndrome Phase 3
Completed NCT02091375 - Antiepileptic Efficacy Study of GWP42003-P in Children and Young Adults With Dravet Syndrome (GWPCARE1) Phase 3
Completed NCT05364021 - Study to Investigate LP352 in Subjects With Developmental and Epileptic Encephalopathies Phase 1/Phase 2
Withdrawn NCT03254680 - Turmeric as Treatment in Epilepsy N/A
Terminated NCT02187809 - Safety and Tolerability of Clobazam as Adjunctive Therapy in Paediatric Patients Aged ≥1 to ≤16 Years With Dravet Syndrome Phase 3
Withdrawn NCT02174094 - Clobazam as Adjunctive Therapy in Paediatric Patients Aged ≥1 to ≤16 Years With Dravet Syndrome Phase 3
Completed NCT02823145 - An Open-Label Extension Trial to Assess the Long-Term Safety of ZX008 (Fenfluramine Hydrochloride HCl) Oral Solution in Children and Young Adults With Dravet Syndrome Phase 3