Extension Study to Evaluate NBI-827104 in Pediatric Participants With Epileptic Encephalopathy With Continuous Spike-and-Wave During Sleep (Steamboat 2)
Primary Purpose
Epileptic Encephalopathy, Continuous Spike and Wave During Sleep
Status
Enrolling by invitation
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
NBI-827104
Sponsored by
About this trial
This is an interventional treatment trial for Epileptic Encephalopathy focused on measuring Epilepsy, Epileptic Encephalopathy, Neurocrine, NBI-827104, Steamboat 2
Eligibility Criteria
Key Inclusion Criteria:
1. Completed 12 weeks of treatment in Study NBI-827104-CSWS2010
Key Exclusion Criteria:
- Have developed any other disorder for which the treatment takes priority over treatment of EECSWS or is likely to interfere with study treatment or impair treatment compliance.
- Body weight <10 kg at randomization Day 1.
- Clinically relevant findings related to cardiovascular or laboratory parameters at screening as determined by the investigator.
- Used any active investigational drug other than NBI-827104 in the context of a clinical study within 30 days or 5 half-lives (whichever is longer) before screening or plans to use such an investigational drug (other than NBI-827104) during the study.
Sites / Locations
- Neurocrine Clinical Site
- Neurocrine Clinical Site
- Neurocrine Clinical Site
- Neurocrine Clinical Site
- Neurocrine Clinical Site
- Neurocrine Clinical Site
- Neurocrine Clinical Site
- Neurocrine Clinical Site
- Neurocrine Clinical Site
- Neurocrine Clinical Site
- Neurocrine Clinical Site
- Neurocrine Clinical Site
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
NBI-827104
Arm Description
NBI-827104 administered orally
Outcomes
Primary Outcome Measures
The Occurrence of Serious Treatment-emergent Adverse Events (TEAEs)
Secondary Outcome Measures
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT05301894
Brief Title
Extension Study to Evaluate NBI-827104 in Pediatric Participants With Epileptic Encephalopathy With Continuous Spike-and-Wave During Sleep
Acronym
Steamboat 2
Official Title
Long-Term, Open-Label Extension Study to Evaluate the Safety and Tolerability of NBI-827104 in Pediatric Subjects With Epileptic Encephalopathy With Continuous Spike-and-Wave During Sleep
Study Type
Interventional
2. Study Status
Record Verification Date
August 2023
Overall Recruitment Status
Enrolling by invitation
Study Start Date
June 7, 2022 (Actual)
Primary Completion Date
February 2025 (Anticipated)
Study Completion Date
February 2025 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Neurocrine Biosciences
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
The primary objective for this study is to evaluate the long-term safety and tolerability of NBI-827104 in pediatric participants with epileptic encephalopathy with continuous spike-and-wave during sleep (EECSWS).
Detailed Description
This study will enroll participants who completed treatment in the Phase 2, multicenter, randomized, double-blind, placebo-controlled, parallel-group Study NBI-827104-CSWS2010 (NCT04625101). Participants who did not participate in Study NBI-827104-CSWS2010 may also be eligible for enrollment.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Epileptic Encephalopathy, Continuous Spike and Wave During Sleep
Keywords
Epilepsy, Epileptic Encephalopathy, Neurocrine, NBI-827104, Steamboat 2
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
24 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
NBI-827104
Arm Type
Experimental
Arm Description
NBI-827104 administered orally
Intervention Type
Drug
Intervention Name(s)
NBI-827104
Intervention Description
T-type calcium channel blocker.
Primary Outcome Measure Information:
Title
The Occurrence of Serious Treatment-emergent Adverse Events (TEAEs)
Time Frame
Day 1 up to 238 weeks
10. Eligibility
Sex
All
Minimum Age & Unit of Time
4 Years
Maximum Age & Unit of Time
12 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Key Inclusion Criteria:
For participants who enroll directly following the completion of the Study NBI-827104-CSWS2010:
Completed 12 weeks of treatment in Study NBI-827104-CSWS2010.
For Participants Who Do Not Enroll Directly from or Did Not Participate in Study NBI-827104-CSWS2010:
Have diagnosis of EECSWS confirmed by the Diagnosis Confirmation Panel (DCP).
Key Exclusion Criteria:
For participants who enroll directly following the completion of the Study NBI-827104-CSWS2010:
Have developed any other disorder for which the treatment takes priority over treatment of EECSWS or is likely to interfere with study treatment or impair treatment compliance.
For Participants Who Do Not Enroll Directly from or Did Not Participate in Study NBI-827104-CSWS2010:
Body weight <15 kg at Day 1.
Clinically relevant findings related to cardiovascular or laboratory parameters at screening as determined by the investigator.
Presence of relevant neurological disorders other than EECSWS and its underlying conditions as judged by the investigator. Symptomatic conditions underlying EECSWS (for example, neonatal strokes) have to be stable for at least 1 year prior to screening.
Planned surgical intervention related to structural abnormalities of the brain from screening through the Week 6 Visit.
Used any active investigational drug other than NBI-827104 in the context of a clinical study within 30 days or 5 half-lives (whichever is longer) before screening or plans to use such an investigational drug (other than NBI-827104) during the study.
Have developed any other disorder for which the treatment takes priority over treatment of EECSWS or is likely to interfere with study treatment or impair treatment compliance.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Clinical Development Lead
Organizational Affiliation
Neurocrine Biosciences
Official's Role
Study Director
Facility Information:
Facility Name
Neurocrine Clinical Site
City
Orange
State/Province
California
ZIP/Postal Code
92868
Country
United States
Facility Name
Neurocrine Clinical Site
City
Aurora
State/Province
Colorado
ZIP/Postal Code
80045
Country
United States
Facility Name
Neurocrine Clinical Site
City
Washington
State/Province
District of Columbia
ZIP/Postal Code
20010
Country
United States
Facility Name
Neurocrine Clinical Site
City
Miami
State/Province
Florida
ZIP/Postal Code
33155
Country
United States
Facility Name
Neurocrine Clinical Site
City
Rochester
State/Province
Minnesota
ZIP/Postal Code
55905
Country
United States
Facility Name
Neurocrine Clinical Site
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27705
Country
United States
Facility Name
Neurocrine Clinical Site
City
Cleveland
State/Province
Ohio
ZIP/Postal Code
44195
Country
United States
Facility Name
Neurocrine Clinical Site
City
Dianalund
ZIP/Postal Code
4293
Country
Denmark
Facility Name
Neurocrine Clinical Site
City
Barcelona
ZIP/Postal Code
08950
Country
Spain
Facility Name
Neurocrine Clinical Site
City
Madrid
ZIP/Postal Code
28034
Country
Spain
Facility Name
Neurocrine Clinical Site
City
Zürich
ZIP/Postal Code
8032
Country
Switzerland
Facility Name
Neurocrine Clinical Site
City
London
ZIP/Postal Code
WC1N 3JH
Country
United Kingdom
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
Extension Study to Evaluate NBI-827104 in Pediatric Participants With Epileptic Encephalopathy With Continuous Spike-and-Wave During Sleep
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