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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05364021




Registration number
NCT05364021
Ethics application status
Date submitted
22/03/2022
Date registered
6/05/2022

Titles & IDs
Public title
Study to Investigate LP352 in Subjects With Developmental and Epileptic Encephalopathies
Scientific title
Randomized, Double-blind, Placebo-controlled, Parallel-group, Dose-escalation Study to Investigate the Safety, Tolerability, PK, PD, and Exploratory Efficacy of LP352 in Subjects With Developmental and Epileptic Encephalopathies
Secondary ID [1] 0 0
LP352-201
Universal Trial Number (UTN)
Trial acronym
PACIFIC
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Developmental and Epileptic Encephalopathy 0 0
Dravet Syndrome 0 0
Lennox Gastaut Syndrome 0 0
Condition category
Condition code
Neurological 0 0 0 0
Epilepsy
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - LP352
Treatment: Drugs - Placebo

Experimental: LP352 - Subjects will be titrated up to highest tolerated dose of LP352 during a 15-day period, followed by a 60-day maintenance period and a 15-day taper/down titration period.

Placebo comparator: Placebo - Placebo for LP352


Treatment: Drugs: LP352
LP352 administered three times daily, orally or through G-tube

Treatment: Drugs: Placebo
Matching placebo for LP352 administered three times daily, orally or through G-tube
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Treatment-emergent Adverse Events
Timepoint [1] 0 0
Baseline up to Day 75
Primary outcome [2] 0 0
Columbia-Suicide Severity Rating Scale (C-SSRS) Response
Timepoint [2] 0 0
Baseline up to Day 75
Primary outcome [3] 0 0
Patient Health Questionnaire-9 Total Score and Question 9 Score
Timepoint [3] 0 0
Baseline up to Day 75
Primary outcome [4] 0 0
Percent Change from Baseline in Observed Countable Motor Seizure Frequency (per 28 Days) During the Treatment Period
Timepoint [4] 0 0
Baseline up to Day 75
Primary outcome [5] 0 0
Percent Change from Baseline in Observed Countable Motor Seizure Frequency (per 28 Days) During the Maintenance Period
Timepoint [5] 0 0
Baseline up to Day 75
Secondary outcome [1] 0 0
Observed Plasma Concentrations of LP352 by Time and Dose
Timepoint [1] 0 0
Baseline up to Day 75
Secondary outcome [2] 0 0
Modeled Estimate of Average Plasma Concentration
Timepoint [2] 0 0
Baseline up to Day 75
Secondary outcome [3] 0 0
Modeled Estimate of Observed Plasma Concentration Just Prior to Dosing
Timepoint [3] 0 0
Baseline up to Day 75
Secondary outcome [4] 0 0
Correlation of Plasma Concentration with Incidence of Treatment-emergent Adverse Events
Timepoint [4] 0 0
Baseline up to Day 75
Secondary outcome [5] 0 0
Correlation of Plasma Concentration with Seizure Frequency
Timepoint [5] 0 0
Baseline up to Day 75
Secondary outcome [6] 0 0
Observed and Change from Baseline Prolactin Concentration During the Treatment Period
Timepoint [6] 0 0
Baseline up to Day 75

Eligibility
Key inclusion criteria
Key

1. Male or non-pregnant, non-lactating female, age 12 to 65 years
2. Diagnosis of Dravet syndrome, Lennox-Gastaut syndrome, or other developmental and epileptic encephalopathy
3. Has a minimum number of seizures per 4-week period while taking 1 to 4 anti-seizure medications
4. All medications and epilepsy interventions must be stable for 4 weeks before screening and are expected to remain stable during the study
5. The patient/parent/caregiver is able and willing to attend study visits, complete the diary and take study drug as instructed

Key
Minimum age
12 Years
Maximum age
65 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. Current or past history of cardiovascular or cerebrovascular disease, such as cardiac valvulopathy, myocardial infarction, stroke, pulmonary arterial hypertension or abnormal blood pressure
2. Has glaucoma, renal impairment, liver disease or any other medical condition that would affect study participation or pose a risk to the subject
3. Current or recent history of moderate or severe depression, anorexia nervosa, bulimia or at risk of suicidal behavior
4. Currently taking anorectic agents, monoamine oxidase inhibitors; serotonin agonists or antagonists including fenfluramine, atomoxetine, vortioxetine, or other medications for weight loss
5. Positive test result on the drug screen, except tetrahydrocannabinol (THC) for patients taking prescribed cannabidiol

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
3/03/2022
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
20/11/2023
Sample size
Target
Accrual to date
Final
52
Recruitment in Australia
Recruitment state(s)
QLD,VIC
Recruitment hospital [1] 0 0
Royal Brisbane Women's Hospital - Herston
Recruitment hospital [2] 0 0
Queensland Children's Hospital - South Brisbane
Recruitment hospital [3] 0 0
Monash Children's Hospital, Monash Health - Clayton
Recruitment hospital [4] 0 0
Austin Health - Heidelberg
Recruitment hospital [5] 0 0
Alfred Health - Melbourne
Recruitment postcode(s) [1] 0 0
4029 - Herston
Recruitment postcode(s) [2] 0 0
4101 - South Brisbane
Recruitment postcode(s) [3] 0 0
3168 - Clayton
Recruitment postcode(s) [4] 0 0
3084 - Heidelberg
Recruitment postcode(s) [5] 0 0
3004 - Melbourne
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
Country [2] 0 0
United States of America
State/province [2] 0 0
Arkansas
Country [3] 0 0
United States of America
State/province [3] 0 0
California
Country [4] 0 0
United States of America
State/province [4] 0 0
Florida
Country [5] 0 0
United States of America
State/province [5] 0 0
Hawaii
Country [6] 0 0
United States of America
State/province [6] 0 0
Idaho
Country [7] 0 0
United States of America
State/province [7] 0 0
Illinois
Country [8] 0 0
United States of America
State/province [8] 0 0
Maryland
Country [9] 0 0
United States of America
State/province [9] 0 0
Michigan
Country [10] 0 0
United States of America
State/province [10] 0 0
Minnesota
Country [11] 0 0
United States of America
State/province [11] 0 0
Missouri
Country [12] 0 0
United States of America
State/province [12] 0 0
New York
Country [13] 0 0
United States of America
State/province [13] 0 0
North Carolina
Country [14] 0 0
United States of America
State/province [14] 0 0
Ohio
Country [15] 0 0
United States of America
State/province [15] 0 0
Oregon
Country [16] 0 0
United States of America
State/province [16] 0 0
Texas
Country [17] 0 0
United States of America
State/province [17] 0 0
Utah
Country [18] 0 0
United States of America
State/province [18] 0 0
Washington

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Longboard Pharmaceuticals
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Dennis J Dlugos, MD
Address 0 0
Children's Hospital of Philadelphia
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT05364021