Status:
COMPLETED
An Open-Label Study to Investigate the Safety of Single and Multiple Ascending Doses in Children and Adolescents With Dravet Syndrome
Lead Sponsor:
Stoke Therapeutics, Inc
Conditions:
Dravet Syndrome
Eligibility:
All Genders
2-18 years
Phase:
PHASE1
PHASE2
Brief Summary
Stoke Therapeutics is evaluating the safety and tolerability of single and multiple ascending doses of STK-001 in patients with Dravet syndrome. Change in seizure frequency, overall clinical status, a...
Detailed Description
STK-001 is an investigational new medicine for the treatment of Dravet syndrome. STK-001 is an antisense oligonucleotide (ASO) that is intended to increase the level of productive SCN1A messenger RNA ...
Eligibility Criteria
Inclusion
- Diagnosis of Dravet Syndrome (DS) with onset of recurrent focal motor or hemiconvulsive or generalized tonic-clonic seizures prior to 12 months of age, which are often prolonged and triggered by hyperthermia.
- No history of causal MRI lesion
- No other known etiology
- Normal development at seizure onset.
- Documented pathogenic, likely pathogenic variant, or variant of uncertain significance in the SCN1A gene associated with DS.
- Use of at least 2 prior treatments for epilepsy that either had lack of adequate seizure control (requiring an additional AED) or had to be discontinued due to an AE(s).
- Currently taking at least one AED at a dose which has been stable for at least 4 weeks prior to Screening.
- Stable epilepsy medications or interventions for epilepsy (including ketogenic diet or vagal nerve stimulator) for at least 4 weeks prior to Screening.
Exclusion
- Known pathogenic mutation in another gene that causes epilepsy
- Currently treated with an AED acting primarily as a sodium channel blocker, as maintenance treatment, including: phenytoin, carbamazepine, oxcarbazepine, lamotrigine, lacosamide, or rufinamide.
- Clinically significant unstable medical conditions other than epilepsy.
- Clinically relevant symptoms or a clinically significant illness in the 4 weeks prior to Screening or prior to dosing on Day 1, other than epilepsy.
- History of brain or spinal cord disease (other than epilepsy or DS), or history of bacterial meningitis or brain malformation
- Spinal deformity or other condition that may alter the free flow of cerebrospinal fluid (CSF) or has an implanted CSF drainage shunt.
- Any other significant disease or disorder which, in the opinion of the Investigator, may either put the patient at risk because of participation in the study, may influence the results of the study, or may affect the patient's ability to participate in the study.
Key Trial Info
Start Date :
June 29 2020
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
November 14 2023
Estimated Enrollment :
62 Patients enrolled
Trial Details
Trial ID
NCT04442295
Start Date
June 29 2020
End Date
November 14 2023
Last Update
May 18 2025
Active Locations (18)
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1
UCSF Benioff Children's Hospital
San Francisco, California, United States, 94158
2
Children's Hospital Colorado
Aurora, Colorado, United States, 80045
3
Children's National Medical Center
Washington D.C., District of Columbia, United States, 20010
4
Nicklaus Children's Hospital
Miami, Florida, United States, 33155