An Open-Label Extension Study of STK-001 for Patients With Dravet Syndrome

Part of paid clinical trials in San Francisco, California.

Sponsor
Stoke Therapeutics, Inc
Study ID
NCT04740476
Phase
PHASE2
Status
Active Not Recruiting

Conditions

  • Dravet Syndrome

Eligibility Criteria

Sex
ALL
Age
30 Months - N/A
Healthy Volunteers
Not accepted

Interventions

  • zorevunersen (STK-001) — DRUG
    zorevunersen drug product is an antisense oligonucleotide administered as an intrathecal injection.

Study Details

Stoke Therapeutics is evaluating the long-term safety \& tolerability of repeated doses of zorevunersen (STK-001) in patients with Dravet syndrome who previously participated in studies of zorevunersen. Change in seizure frequency and overall clinical status, and quality of life will be measured as secondary endpoints in this open-label study.

Key Dates

Start date
Jan 20, 2021
Status verified
Feb 2026
Primary completion
Mar 31, 2029
Completion
Mar 31, 2029

Study Design

Enrollment
60 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: zorevunersen (STK-001) multiple dose levels
    Enrollment of patients after completion of Study STK-001-DS-101 or Study STK-001-DS-102 if eligible. Patients will receive IT administration of study drug zorevunersen at the dose level they received while participating in Study STK-001-DS-101 or STK-001-DS-102, or at a dose level recommended by the Safety Monitoring Committee (SMC).The highest dose administered in this study may not exceed that which has already been evaluated in an zorevunersen Phase 1/2 study, and doses above 45 mg/dose in this study require approval from the Food and Drug Administration (FDA). Patients will initially receive 3 doses, one every approximately 4 months (16 weeks). Patients who are tolerating treatment may continue treatment with doses approximately every 4 months, with an End of Study/Follow-up Visit 24 weeks after the last dose of study drug. Patients who do not continue treatment after the third dose will have a Follow-up Visit (V5) at Week 48 and an End of Study Visit at Week 56.

Primary Outcome Measure

Safety of multiple doses of zorevunersen [ Time Frame: Screening (Day -1) until 6 months after multiple drug dosing ]

Locations (17)

FacilityCityStateZIPSite coordinators
University of California San Francisco Medical CenterSan FranciscoCalifornia94158-
Children's Hospital ColoradoAuroraColorado80045-
Children's National Medical CenterWashington D.C.District of Columbia20010-
Nicklaus Children's HospitalMiamiFlorida33155-
Florida Hospital for ChildrenOrlandoFlorida32803-
Ann & Robert H. Lurie Children's Hospital of ChicagoChicagoIllinois60611-
University of Iowa Children's HospitalIowa CityIowa52242-
Massachusetts General HospitalBostonMassachusetts02114-
Michigan MedicineAnn ArborMichigan48109-
Mayo ClinicRochesterMinnesota55905-
NYU Comprehensive Epilepsy CenterNew YorkNew York10016-
Oregon Health & Science UniversityPortlandOregon97239-
Children's Hospital of PhiladelphiaPhiladelphiaPennsylvania19104-
UT LeBonheur Pediatric Specialists, Inc.MemphisTennessee38103-
Cook Children's Medical CenterFort WorthTexas76104-
Seattle Children's HospitalSeattleWashington98105-
MultiCare Health System Institute for Research and InnovationTacomaWashington98405-

Find similar trials in San Francisco, CA

By research site
University of California San Francisco Medical Center· San Francisco, CAChildren's Hospital Colorado· Aurora, COChildren's National Medical Center· Washington D.C., DCNicklaus Children's Hospital· Miami, FLFlorida Hospital for Children· Orlando, FLAnn & Robert H. Lurie Children's Hospital of Chicago· Chicago, IL

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