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Study to Investigate LP352 in Subjects With Developmental and Epileptic Encephalopathies (PACIFIC)

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ClinicalTrials.gov Identifier: NCT05364021
Recruitment Status : Completed
First Posted : May 6, 2022
Last Update Posted : January 19, 2024
Sponsor:
Information provided by (Responsible Party):
Longboard Pharmaceuticals

Brief Summary:
The objective of this study is to assess the safety, tolerability, efficacy, and pharmacokinetics of adjunctive therapy of LP352 in adults and adolescents with developmental and epileptic encephalopathies.

Condition or disease Intervention/treatment Phase
Developmental and Epileptic Encephalopathy Dravet Syndrome Lennox Gastaut Syndrome Drug: LP352 Drug: Placebo Phase 1 Phase 2

Detailed Description:

This is a randomized, double-blind, parallel-group, dose-escalation, placebo-controlled study of LP352 in adults and adolescents with developmental and epileptic encephalopathies (DEE) with an average of ≥ 4 observed/countable motor seizures per 4-week period during the 12 weeks before screening while on stable antiseizure medicine (ASM).

Subjects will be randomized 4:1 to LP352 or placebo. The study will have a baseline period of 28 days, followed by a 15 day up-titration period during which time subjects will titrate up to their highest tolerated doses, and a 60-day maintenance period. After Day 75, subjects will be tapered down over a period of up to 15 days, with a follow-up visit 30 days after last dose. Enrolled subjects will be allowed to continue treatment with up to 4 concomitant ASMs at a stable dose.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 52 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official 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
Actual Study Start Date : March 3, 2022
Actual Primary Completion Date : November 16, 2023
Actual Study Completion Date : November 20, 2023


Arm Intervention/treatment
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.
Drug: LP352
LP352 administered three times daily, orally or through G-tube

Placebo Comparator: Placebo
Placebo for LP352
Drug: Placebo
Matching placebo for LP352 administered three times daily, orally or through G-tube
Other Name: Placebo Comparator




Primary Outcome Measures :
  1. Treatment-emergent Adverse Events [ Time Frame: Baseline up to Day 75 ]
    Incidence and severity of 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

  2. Columbia-Suicide Severity Rating Scale (C-SSRS) Response [ Time Frame: Baseline up to Day 75 ]
    Type of Suicidal Ideation, Intensity (1 - 5, with 5 being most severe), Suicidal Behavior

  3. Patient Health Questionnaire-9 Total Score and Question 9 Score [ Time Frame: Baseline up to Day 75 ]
    Severity Rating Scale: 0 - 27; higher scores indicate greater severity of depressive disorder

  4. Percent Change from Baseline in Observed Countable Motor Seizure Frequency (per 28 Days) During the Treatment Period [ Time Frame: Baseline up to Day 75 ]
  5. Percent Change from Baseline in Observed Countable Motor Seizure Frequency (per 28 Days) During the Maintenance Period [ Time Frame: Baseline up to Day 75 ]

Secondary Outcome Measures :
  1. Observed Plasma Concentrations of LP352 by Time and Dose [ Time Frame: Baseline up to Day 75 ]
  2. Modeled Estimate of Average Plasma Concentration [ Time Frame: Baseline up to Day 75 ]
  3. Modeled Estimate of Observed Plasma Concentration Just Prior to Dosing [ Time Frame: Baseline up to Day 75 ]
  4. Correlation of Plasma Concentration with Incidence of Treatment-emergent Adverse Events [ Time Frame: Baseline up to Day 75 ]
  5. Correlation of Plasma Concentration with Seizure Frequency [ Time Frame: Baseline up to Day 75 ]
  6. Observed and Change from Baseline Prolactin Concentration During the Treatment Period [ Time Frame: Baseline up to Day 75 ]


Information from the National Library of Medicine

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Ages Eligible for Study:   12 Years to 65 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Key Inclusion Criteria:

  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 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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05364021


Locations
Show Show 34 study locations
Sponsors and Collaborators
Longboard Pharmaceuticals
Investigators
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Principal Investigator: Dennis J Dlugos, MD Children's Hospital of Philadelphia
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Responsible Party: Longboard Pharmaceuticals
ClinicalTrials.gov Identifier: NCT05364021    
Other Study ID Numbers: LP352-201
First Posted: May 6, 2022    Key Record Dates
Last Update Posted: January 19, 2024
Last Verified: January 2024
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Longboard Pharmaceuticals:
CDKL5 deficiency disorder
developmental and epileptic encephalopathy
Dravet Syndrome
epilepsy
Lennox-Gastaut Syndrome
treatment resistant epilepsy
tuberous sclerosis complex
Additional relevant MeSH terms:
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Brain Diseases
Epilepsy
Epilepsies, Myoclonic
Lennox Gastaut Syndrome
Syndrome
Disease
Pathologic Processes
Central Nervous System Diseases
Nervous System Diseases
Epilepsy, Generalized
Epileptic Syndromes
Genetic Diseases, Inborn