Back to resultsOpen-label, Long-term Safety Study of LP352 in Subjects With Developmental and Epileptic Encephalopathy
Primary Purpose
Developmental and Epileptic Encephalopathy, Dravet Syndrome, Lennox-Gastaut Syndrome
Status
Recruiting
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
LP352
Sponsored by
About this trial
This is an interventional treatment trial for Developmental and Epileptic Encephalopathy focused on measuring CDKL5 deficiency disorder, developmental and epileptic encephalopathy, Dravet Syndrome, epilepsy, Lennox-Gastaut Syndrome, treatment resistant epilepsy, tuberous sclerosis complex
Eligibility Criteria
Inclusion Criteria: Male or non-pregnant, non-lactating female, age 12 to 65 years who have satisfactorily completed study LP352-201 Diagnosis of Dravet syndrome, Lennox-Gastaut syndrome, or other developmental and epileptic encephalopathy The patient/parent/caregiver is able and willing to attend study visits, complete the diary and take study drug as instructed Exclusion Criteria: Had an SAE in Study LP352-201 that was definitely, probably, or possibly related to exposure to study drug Current or past history of cardiovascular or cerebrovascular disease, such as cardiac valvulopathy, myocardial infarction, stroke, pulmonary arterial hypertension or abnormal blood pressure Has glaucoma, renal impairment, liver disease or any other medical condition that would affect study participation or pose a risk to the subject Current or recent history of moderate or severe depression, anorexia nervosa, bulimia or at risk of suicidal behavior Currently taking anorectic agents, monoamine oxidase inhibitors; serotonin agonists or antagonists including fenfluramine, atomoxetine, vortioxetine, or other medications for weight loss Positive test result on the drug screen, except tetrahydrocannabinol (THC) for patients taking prescribed cannabidiol
Sites / Locations
- Arkansas Children's HospitalRecruiting
- Rancho Los Amigos National Rehabilitation Center (RLANRC)Recruiting
- University of California San FranciscoRecruiting
- Northwest Florida Clinical Research GroupRecruiting
- University of Miami
- Advent Health Orlando
- Research Institute of OrlandoRecruiting
- University of South Florida
- Hawaii Pacific NeuroscienceRecruiting
- Northwestern University Feinberg School of MedicineRecruiting
- Mid-Atlantic Epilepsy and Sleep CenterRecruiting
- Spectrum HealthRecruiting
- Mayo Clinic
- Washington University School of MedicineRecruiting
- Boston Children's Health Physicians LLP
- Northwell HealthRecruiting
- Northeast Regional Epilepsy Group
- OnSite Clinical Solutions LLC
- Wake Forest University School of Medicine
- University Hospitals Cleveland Medical CenterRecruiting
- Providence Neurological Specialties-EastRecruiting
- Austin Epilepsy Care CenterRecruiting
- University of UtahRecruiting
- University of Washington Valley Medical Center
- Austin HealthRecruiting
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
LP352
Arm Description
Subjects will be titrated up to highest tolerated dose of LP352 during a 15-day period, followed by a 48-week maintenance period and a 15-day taper/down titration period.
Outcomes
Primary Outcome Measures
Treatment-emergent Adverse Events
Incidence and severity of treatment-emergent adverse events, including serious adverse events and adverse events leading to study discontinuation and clinically significant changes in vital signs, physical examination endpoints, clinical safety laboratory values and ECGs
Columbia-Suicide Severity Rating Scale (C-SSRS) Response
Type of Suicidal Ideation, Intensity (1 - 5, with 5 being most severe), Suicidal Behavior
Patient Health Questionnaire-9 Total Score and Question 9 Score
Severity Rating Scale: 0 - 27; higher scores indicate greater severity of depressive disorder
Secondary Outcome Measures
Percent Change from Baseline in Observed Countable Motor Seizure Frequency During the Treatment Period
Baseline Used for Seizure Frequency = Baseline from Study LP352-201 and Baseline from Study LP352-202
Proportion of Subjects with > 50% Reduction in Total Seizures During the Treatment Period
Percent Reduction in Individual Seizure Type During the Treatment Period
Proportion of Subjects Requiring Rescue Medication During the Treatment Period
Percent Change from Baseline in the Number of Episodes of Status Epilepticus During the Treatment Period
Percent of Subjects with Countable Motor Seizure-free Days During the Treatment Period
Percentage Change from Baseline in Non-motor and Difficult to Count Seizures
LGS: Percentage Change from Baseline in the Frequency of Observed Drop Seizures Over the Treatment Period
Full Information
NCT ID
NCT05626634
First Posted
November 15, 2022
Last Updated
September 26, 2023
Sponsor
Longboard Pharmaceuticals
1. Study Identification
Unique Protocol Identification Number
NCT05626634
Brief Title
Open-label, Long-term Safety Study of LP352 in Subjects With Developmental and Epileptic Encephalopathy
Official Title
A Phase 2, Multicenter, Open-label, Long-term Safety Study of LP352 in Subjects With Developmental and Epileptic Encephalopathy Who Completed Study LP352-201 and Are Candidates for Continuous Treatment for Up to 52 Weeks
Study Type
Interventional
2. Study Status
Record Verification Date
September 2023
Overall Recruitment Status
Recruiting
Study Start Date
November 8, 2022 (Actual)
Primary Completion Date
October 2024 (Anticipated)
Study Completion Date
October 2024 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Longboard Pharmaceuticals
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
5. Study Description
Brief Summary
The objective of this study is to assess the long-term safety, tolerability, and efficacy of adjunctive therapy of LP352 in subjects with developmental and epileptic encephalopathies who completed participation in Study LP352-201.
Detailed Description
This Phase 2, multicenter, open-label, multiple-dose extension clinical study is designed to evaluate long-term safety of LP352 in subjects with developmental and epileptic encephalopathy who completed Study LP352-201.
The study consists of a Screening Period (Day -1) and a 50-week open-label Treatment Period that includes a 15-day Up-titration Period (during which time subjects will titrate up to their highest tolerated doses) and an open-label Maintenance Period (48 weeks). The Treatment Period will be followed by a Down-titration/Taper Period (up to 15 days) and Follow-up Period (14 days after completion of down-titration). The starting dose of up-titration will be 6 mg TID. The target final maintenance doses are 6 mg TID, 9 mg TID, and 12 mg TID after a 15-day up-titration period, if tolerated.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Developmental and Epileptic Encephalopathy, Dravet Syndrome, Lennox-Gastaut Syndrome
Keywords
CDKL5 deficiency disorder, developmental and epileptic encephalopathy, Dravet Syndrome, epilepsy, Lennox-Gastaut Syndrome, treatment resistant epilepsy, tuberous sclerosis complex
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
50 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
LP352
Arm Type
Experimental
Arm Description
Subjects will be titrated up to highest tolerated dose of LP352 during a 15-day period, followed by a 48-week maintenance period and a 15-day taper/down titration period.
Intervention Type
Drug
Intervention Name(s)
LP352
Intervention Description
LP352 6 mg, 9 mg or 12 mg administered three times daily, orally or through G-tube
Primary Outcome Measure Information:
Title
Treatment-emergent Adverse Events
Description
Incidence and severity of treatment-emergent adverse events, including serious adverse events and adverse events leading to study discontinuation and clinically significant changes in vital signs, physical examination endpoints, clinical safety laboratory values and ECGs
Time Frame
Baseline up to Week 52
Title
Columbia-Suicide Severity Rating Scale (C-SSRS) Response
Description
Type of Suicidal Ideation, Intensity (1 - 5, with 5 being most severe), Suicidal Behavior
Time Frame
Baseline up to Week 52
Title
Patient Health Questionnaire-9 Total Score and Question 9 Score
Description
Severity Rating Scale: 0 - 27; higher scores indicate greater severity of depressive disorder
Time Frame
Baseline up to Week 52
Secondary Outcome Measure Information:
Title
Percent Change from Baseline in Observed Countable Motor Seizure Frequency During the Treatment Period
Description
Baseline Used for Seizure Frequency = Baseline from Study LP352-201 and Baseline from Study LP352-202
Time Frame
Baseline to Week 50
Title
Proportion of Subjects with > 50% Reduction in Total Seizures During the Treatment Period
Time Frame
Baseline to Week 50
Title
Percent Reduction in Individual Seizure Type During the Treatment Period
Time Frame
Baseline to Week 50
Title
Proportion of Subjects Requiring Rescue Medication During the Treatment Period
Time Frame
Baseline to Week 50
Title
Percent Change from Baseline in the Number of Episodes of Status Epilepticus During the Treatment Period
Time Frame
Baseline to Week 50
Title
Percent of Subjects with Countable Motor Seizure-free Days During the Treatment Period
Time Frame
Baseline to Week 50
Title
Percentage Change from Baseline in Non-motor and Difficult to Count Seizures
Time Frame
Baseline to Week 50
Title
LGS: Percentage Change from Baseline in the Frequency of Observed Drop Seizures Over the Treatment Period
Time Frame
Baseline to Week 50
10. Eligibility
Sex
All
Minimum Age & Unit of Time
12 Years
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Male or non-pregnant, non-lactating female, age 12 to 65 years who have satisfactorily completed study LP352-201
Diagnosis of Dravet syndrome, Lennox-Gastaut syndrome, or other developmental and epileptic encephalopathy
The patient/parent/caregiver is able and willing to attend study visits, complete the diary and take study drug as instructed
Exclusion Criteria:
Had an SAE in Study LP352-201 that was definitely, probably, or possibly related to exposure to study drug
Current or past history of cardiovascular or cerebrovascular disease, such as cardiac valvulopathy, myocardial infarction, stroke, pulmonary arterial hypertension or abnormal blood pressure
Has glaucoma, renal impairment, liver disease or any other medical condition that would affect study participation or pose a risk to the subject
Current or recent history of moderate or severe depression, anorexia nervosa, bulimia or at risk of suicidal behavior
Currently taking anorectic agents, monoamine oxidase inhibitors; serotonin agonists or antagonists including fenfluramine, atomoxetine, vortioxetine, or other medications for weight loss
Positive test result on the drug screen, except tetrahydrocannabinol (THC) for patients taking prescribed cannabidiol
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Longboard Study Contact
Phone
858-999-8858
Email
clinicalstudies@longboardpharma.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Dennis J Dlugos, MD
Organizational Affiliation
Children's Hospital of Philadelphia
Official's Role
Principal Investigator
Facility Information:
Facility Name
Arkansas Children's Hospital
City
Little Rock
State/Province
Arkansas
ZIP/Postal Code
72202
Country
United States
Individual Site Status
Recruiting
Facility Name
Rancho Los Amigos National Rehabilitation Center (RLANRC)
City
Downey
State/Province
California
ZIP/Postal Code
90242
Country
United States
Individual Site Status
Recruiting
Facility Name
University of California San Francisco
City
San Francisco
State/Province
California
ZIP/Postal Code
94158
Country
United States
Individual Site Status
Recruiting
Facility Name
Northwest Florida Clinical Research Group
City
Gulf Breeze
State/Province
Florida
ZIP/Postal Code
32561
Country
United States
Individual Site Status
Recruiting
Facility Name
University of Miami
City
Miami
State/Province
Florida
ZIP/Postal Code
33136
Country
United States
Individual Site Status
Not yet recruiting
Facility Name
Advent Health Orlando
City
Orlando
State/Province
Florida
ZIP/Postal Code
32803
Country
United States
Individual Site Status
Not yet recruiting
Facility Name
Research Institute of Orlando
City
Orlando
State/Province
Florida
ZIP/Postal Code
32806
Country
United States
Individual Site Status
Recruiting
Facility Name
University of South Florida
City
Tampa
State/Province
Florida
ZIP/Postal Code
33606
Country
United States
Individual Site Status
Not yet recruiting
Facility Name
Hawaii Pacific Neuroscience
City
Honolulu
State/Province
Hawaii
ZIP/Postal Code
96817
Country
United States
Individual Site Status
Recruiting
Facility Name
Northwestern University Feinberg School of Medicine
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60611
Country
United States
Individual Site Status
Recruiting
Facility Name
Mid-Atlantic Epilepsy and Sleep Center
City
Bethesda
State/Province
Maryland
ZIP/Postal Code
20817
Country
United States
Individual Site Status
Recruiting
Facility Name
Spectrum Health
City
Grand Rapids
State/Province
Michigan
ZIP/Postal Code
49503
Country
United States
Individual Site Status
Recruiting
Facility Name
Mayo Clinic
City
Rochester
State/Province
Minnesota
ZIP/Postal Code
55905
Country
United States
Individual Site Status
Not yet recruiting
Facility Name
Washington University School of Medicine
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63110
Country
United States
Individual Site Status
Recruiting
Facility Name
Boston Children's Health Physicians LLP
City
Hawthorne
State/Province
New York
ZIP/Postal Code
10532
Country
United States
Individual Site Status
Not yet recruiting
Facility Name
Northwell Health
City
New York
State/Province
New York
ZIP/Postal Code
10075
Country
United States
Individual Site Status
Recruiting
Facility Name
Northeast Regional Epilepsy Group
City
Staten Island
State/Province
New York
ZIP/Postal Code
10305
Country
United States
Individual Site Status
Not yet recruiting
Facility Name
OnSite Clinical Solutions LLC
City
Charlotte
State/Province
North Carolina
ZIP/Postal Code
98277
Country
United States
Individual Site Status
Not yet recruiting
Facility Name
Wake Forest University School of Medicine
City
Winston-Salem
State/Province
North Carolina
ZIP/Postal Code
27157
Country
United States
Individual Site Status
Not yet recruiting
Facility Name
University Hospitals Cleveland Medical Center
City
Cleveland
State/Province
Ohio
ZIP/Postal Code
44106
Country
United States
Individual Site Status
Recruiting
Facility Name
Providence Neurological Specialties-East
City
Portland
State/Province
Oregon
ZIP/Postal Code
97213
Country
United States
Individual Site Status
Recruiting
Facility Name
Austin Epilepsy Care Center
City
Austin
State/Province
Texas
ZIP/Postal Code
78758
Country
United States
Individual Site Status
Recruiting
Facility Name
University of Utah
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84132
Country
United States
Individual Site Status
Recruiting
Facility Name
University of Washington Valley Medical Center
City
Renton
State/Province
Washington
ZIP/Postal Code
98055
Country
United States
Individual Site Status
Not yet recruiting
Facility Name
Austin Health
City
Heidelberg
State/Province
Victoria
ZIP/Postal Code
3084
Country
Australia
Individual Site Status
Recruiting
12. IPD Sharing Statement
Learn more about this trial
Open-label, Long-term Safety Study of LP352 in Subjects With Developmental and Epileptic Encephalopathy
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