An Open-Label Study to Investigate the Safety of Single and Multiple Ascending Doses in Children and Adolescents With Dravet Syndrome

PHASE1/PHASE2CompletedINTERVENTIONAL
Enrollment

62

Participants

Timeline

Start Date

June 29, 2020

Primary Completion Date

November 14, 2023

Study Completion Date

November 14, 2023

Conditions
Dravet Syndrome
Interventions
DRUG

STK-001 - Single Ascending Doses

Experimental : Single Ascending Doses - STK-001 drug product is an antisense oligonucleotide administered as an intrathecal injection. Four dose levels will be evaluated ( 10mg, 20mg,30mg, 45mg and 70mg ).

DRUG

STK-001 - Multiple Ascending Doses

Experimental : Multiple Ascending Doses - STK-001 drug product is an antisense oligonucleotide administered as an intrathecal injection. Three dose levels will be evaluated ( 20mg,30mg and 45mg ).

Trial Locations (18)

10016

NYU Comprehensive Epilepsy Center, New York

19104

Children's Hospital of Philadelphia, Philadelphia

20010

Children's National Medical Center, Washington D.C.

32803

AdventHealth Orlando, Orlando

33155

Nicklaus Children's Hospital, Miami

38105

Le Bonheur Children's Hospital, Memphis

48109

University of Michigan - Mott Children's Hospital, Ann Arbor

52242

University of Iowa Hospitals and Clinics; Pediatric Specialty Clinic, Iowa City

55905

Mayo Clinic, Rochester

60611

Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago

76104

Cook Children's Health Care System, Fort Worth

80045

Children's Hospital Colorado, Aurora

84108

Primary Children's Hospital, Salt Lake City

94158

UCSF Benioff Children's Hospital, San Francisco

97239

Oregon Health & Science University, Portland

98105

Seattle Children's Hospital, Seattle

98405

Multicare Institute for Research and Innovation, Tacoma

02114

Massachusetts General Hospital - Pediatric Epilepsy Program, Boston

All Listed Sponsors
lead

Stoke Therapeutics, Inc

INDUSTRY

NCT04442295 - An Open-Label Study to Investigate the Safety of Single and Multiple Ascending Doses in Children and Adolescents With Dravet Syndrome | Biotech Hunter | Biotech Hunter