Developmental and Epileptic EncephalopathyPhase 1/2PACIFICNCT05364021
Study to Investigate LP352 in Subjects With Developmental and Epileptic Encephalopathies
A Phase 1/2 study testing LP352 in people with Developmental and Epileptic Encephalopathy and Dravet Syndrome, run by Longboard Pharmaceuticals at 34 sites in 2 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 12 to 65 years
- Conditions the study is for
- Developmental and Epileptic Encephalopathy, Dravet Syndrome, Lennox Gastaut Syndrome
- What is being tested?
- LP352
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 52 participants
- Where
- 34 sitesin 2 countries
- Study dates
- 3 Mar 2022to 20 Nov 2023
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 to 65 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Male or non-pregnant, non-lactating female, age 12 to 65 years
- Diagnosis of Dravet syndrome, Lennox-Gastaut syndrome, or other developmental and epileptic encephalopathy
- Has a minimum number of seizures per 4-week period while taking 1 to 4 anti-seizure medications
- All medications and epilepsy interventions must be stable for 4 weeks before screening and are expected to remain stable during the study
- The patient/parent/caregiver is able and willing to attend study visits, complete the diary and take study drug as instructed
Who cannot join
- 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
Trial locations and contacts
34 sites in 2 countries.
34 sites in 2 countries
United States29 sites
Child Neurology Consultants of Austin
Austin, Texas, 78757
Austin Epilepsy Care Center
Austin, Texas, 78758
Mid-Atlantic Epilepsy and Sleep Center
Bethesda, Maryland, 20817
Consultants in Epilepsy and Neurology
Boise, Idaho, 83702
OnSite Clinical Solutions LLC
Charlotte, North Carolina, 98277
Northwestern University Feinberg School of Medicine
Chicago, Illinois, 60611
University Hospitals Cleveland Medical Center
Cleveland, Ohio, 44106
Rancho Los Amigos National Rehabilitation Center (RLANRC)
Downey, California, 90242
Spectrum Health
Grand Rapids, Michigan, 49503
Northwest Florida Clinical Research Group
Gulf Breeze, Florida, 32561
Boston Children's Health Physicians LLP
Hawthorne, New York, 10532
Hawaii Pacific Neuroscience
Honolulu, Hawaii, 96817
Arkansas Children's Hospital
Little Rock, Arkansas, 72202
University of Miami
Miami, Florida, 33136
New York University Langone Hospital - Long Island
Mineola, New York, 11501
Northwell Health
New York, New York, 10075
Children's Hospital of Orange County
Orange, California, 92868
Advent Health Orlando
Orlando, Florida, 32803
Research Institute of Orlando
Orlando, Florida, 32806
Providence Neurological Specialties-East
Portland, Oregon, 97213
University of Washington Valley Medical Center
Renton, Washington, 98055
Mayo Clinic
Rochester, Minnesota, 55905
University of Utah
Salt Lake City, Utah, 84132
University of California San Francisco
San Francisco, California, 94158
Washington University School of Medicine
St Louis, Missouri, 63110
Northeast Regional Epilepsy Group
Staten Island, New York, 10305
University of South Florida
Tampa, Florida, 33606
University of Arizona - Health Sciences Center
Tucson, Arizona, 85724
Wake Forest University School of Medicine
Winston-Salem, North Carolina, 27157
Australia5 sites
Monash Children's Hospital, Monash Health
Clayton, Victoria, 3168
Austin Health
Heidelberg, Victoria, 3084
Royal Brisbane Women's Hospital
Herston, Queensland, 4029
Alfred Health
Melbourne, Victoria, 3004
Queensland Children's Hospital
South Brisbane, Queensland, 4101
About this study
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
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.
Read the 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.
- Being tested
- LP352, Placebo
- Keywords
- CDKL5 deficiency disorder, developmental and epileptic encephalopathy, Dravet Syndrome, epilepsy, Lennox-Gastaut Syndrome, treatment resistant epilepsy, tuberous sclerosis complex
- Medical subject headings
- Epilepsies, Myoclonic, Lennox Gastaut Syndrome, CDKL5 deficiency disorder, Epilepsy, Tuberous Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LP352Drug
LP352 administered three times daily, orally or through G-tube
PlaceboDrug
Also called Placebo Comparator
Matching placebo for LP352 administered three times daily, orally or through G-tube
| Study group | Role | What they receive |
|---|---|---|
| LP352 | Experimental | 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. Receives: Drug: LP352 |
| Placebo | Placebo comparator | Placebo for LP352 Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Treatment-emergent Adverse Events
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
Measured: Baseline up to Day 75
Columbia-Suicide Severity Rating Scale (C-SSRS) Response
Type of Suicidal Ideation, Intensity (1 - 5, with 5 being most severe), Suicidal Behavior
Measured: Baseline up to Day 75
Patient Health Questionnaire-9 Total Score and Question 9 Score
Severity Rating Scale: 0 - 27; higher scores indicate greater severity of depressive disorder
Measured: Baseline up to Day 75
Percent Change from Baseline in Observed Countable Motor Seizure Frequency (per 28 Days) During the Treatment Period
Measured: Baseline up to Day 75
Percent Change from Baseline in Observed Countable Motor Seizure Frequency (per 28 Days) During the Maintenance Period
Measured: Baseline up to Day 75
Secondary outcomes (6)
Observed Plasma Concentrations of LP352 by Time and Dose
Measured: Baseline up to Day 75
Modeled Estimate of Average Plasma Concentration
Measured: Baseline up to Day 75
Modeled Estimate of Observed Plasma Concentration Just Prior to Dosing
Measured: Baseline up to Day 75
Correlation of Plasma Concentration with Incidence of Treatment-emergent Adverse Events
Measured: Baseline up to Day 75
Correlation of Plasma Concentration with Seizure Frequency
Measured: Baseline up to Day 75
Observed and Change from Baseline Prolactin Concentration During the Treatment Period
Measured: Baseline up to Day 75
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 May 2022
- Study start3 Mar 2022
- Primary completion16 Nov 2023
- Study completion20 Nov 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Developmental and Epileptic EncephalopathyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 52 participants (actual)
Sponsor and investigators
- Lead sponsor
- Longboard Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Dennis J Dlugos, MD, Principal investigator, Children's Hospital of Philadelphia
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05364021
- Sponsor's protocol ID
- LP352-201
- Acronym
- PACIFIC
- Status last verified
- Nov 2024
- First submitted
- 22 Mar 2022
- Last updated
- 8 Nov 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Nov 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT05364021
- Acronym / sponsor ID
- PACIFIC / LP352-201
- Keywords
- CDKL5 deficiency disorder, developmental and epileptic encephalopathy, Dravet Syndrome, epilepsy, Lennox-Gastaut Syndrome, treatment resistant epilepsy, tuberous sclerosis complex
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 52 participants (actual)
- Lead sponsor
- Longboard PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 3 Mar 2022 (actual)
- Primary completion
- 16 Nov 2023 (actual)
- Study completion
- 20 Nov 2023 (actual)
- First posted
- 6 May 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| LP352 | Experimental | Drug: LP352Subjects 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 | Placebo comparator | Drug: PlaceboPlacebo for LP352 |
Who may qualify
- Ages
- 12 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- Male or non-pregnant, non-lactating female, age 12 to 65 years
- Diagnosis of Dravet syndrome, Lennox-Gastaut syndrome, or other developmental and epileptic encephalopathy
- Has a minimum number of seizures per 4-week period while taking 1 to 4 anti-seizure medications
- All medications and epilepsy interventions must be stable for 4 weeks before screening and are expected to remain stable during the study
- The patient/parent/caregiver is able and willing to attend study visits, complete the diary and take study drug as instructed
Key exclusion criteria (5 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| LP352LP352 administered three times daily, orally or through G-tube | Drug | LP352 |
| PlaceboAlso: Placebo ComparatorMatching placebo for LP352 administered three times daily, orally or through G-tube | Drug | Placebo |
Primary outcome
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
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
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
Percent Change from Baseline in Observed Countable Motor Seizure Frequency (per 28 Days) During the Treatment Period
Time frame: Baseline up to Day 75
Percent Change from Baseline in Observed Countable Motor Seizure Frequency (per 28 Days) During the Maintenance Period
Time frame: Baseline up to Day 75
6 secondary outcomes
Observed Plasma Concentrations of LP352 by Time and Dose
Time frame: Baseline up to Day 75
Modeled Estimate of Average Plasma Concentration
Time frame: Baseline up to Day 75
Modeled Estimate of Observed Plasma Concentration Just Prior to Dosing
Time frame: Baseline up to Day 75
Correlation of Plasma Concentration with Incidence of Treatment-emergent Adverse Events
Time frame: Baseline up to Day 75
Correlation of Plasma Concentration with Seizure Frequency
Time frame: Baseline up to Day 75
Observed and Change from Baseline Prolactin Concentration During the Treatment Period
Time frame: Baseline up to Day 75
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Nov 2024.
- Individual participant dataSharing planned: No
Related trials for Developmental and Epileptic Encephalopathy
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RecruitingObservational study
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Developmental and Epileptic Encephalopathies (DEE)
RecruitingObservational study
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Rare Disorders, Undiagnosed Disorders
Active, not recruitingPhase 3
A Phase 3, Placebo-Controlled Study to Investigate LP352 in Children and Adults With Dravet Syndrome (DS)
Dravet Syndrome
RecruitingPhase 1/2
A Clinical Study to Evaluate the Safety and Efficacy of ETX101 in Infants and Children With SCN1A-Positive Dravet Syndrome
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Dennis J Dlugos, MD principal investigator
Children's Hospital of Philadelphia
34 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing LP352 in people with Developmental and Epileptic Encephalopathy and Dravet Syndrome, run by Longboard Pharmaceuticals at 34 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 8 Nov 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 34 sites in United States, Australia. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Treatment-emergent Adverse Events (time frame: Baseline up to Day 75). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Nov 2024.