A Phase 3, Placebo-Controlled Study to Investigate LP352 in Children and Adults With Dravet Syndrome (DS)

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Longboard Pharmaceuticals Inc.

Participant type

Pediatric, Patients

Age range

0-17 years, 18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Nervous System Diseases [C10]

Trial duration

20 May 2025 → ongoing

Decision date (initial)

2025-05-06

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Longboard Pharmaceuticals, Inc.

External identifiers

EU CT number

2024-514937-39-00

ClinicalTrials.gov

NCT06660394

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Efficacy, Others

To evaluate the efficacy of LP352 in Dravet Syndrome as assessed by countable motor seizures

Secondary objectives 1

1. To evaluate the efficacy of LP352 in Dravet Syndrome

Conditions and MedDRA coding

Dravet Syndrome

Study design 3 periods

Regulatory references

Scientific advice from competent authorities

European Medicines Agency, Food And Drug Administration

EMA paediatric investigation plan (PIP)

EMEA-003288-PIP01-22

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 7

1. Participant is ≥2 to ≤65 years of age at the time of Screening
2. Diagnosis of Dravet Syndrome according to International League Against Epilepsy definition and must fulfill all of the following criteria: a. Onset of seizures age >1 and <20 months in an otherwise healthy infant. b. History of at least 1 of the following seizure type(s): i. Prolonged generalized tonic-clonic ii. Hemiclonic iii. Myoclonic iv. Tonic v. Atonic vi. Atypical absence vii. Focal awareness viii. Nonconvulsive status epilepticus
3. The following countable motor seizure types: a. Generalized tonic-clonic b. Tonic (bilateral) c. Clonic (bilateral) d. Atonic (bilateral) with truncal/leg involvement e. Focal motor (including hemiclonic) f. Focal to bilateral tonic-clonic
4. Demonstrated an average of at least 4 countable motor seizures (as per inclusion criterion 3) per month for the 3 months prior to Screening
5. Has been taking 1 to 4 ASMs at a stable dose for at least 4 weeks prior to Screening
6. The participant must be willing and able to provide written informed consent.
7. The participant, parent, or caregiver is willing and able (in the judgment of the investigator) to comply with completion of the diaries throughout the study.

Exclusion criteria 8

1. The participant has a history of infantile/epileptic spasms.
2. The participant has been admitted to a medical facility for treatment of status epilepticus requiring mechanical ventilation within 3 months prior to Screening.
3. The participant has a neurodegenerative disorder as indicated by magnetic resonance imaging or genetic testing.
4. The participant has an acquired lesion/injury unrelated to the primary etiology that could contribute as a secondary cause of seizures.
5. The participant is receiving exclusionary medications, as described in the protocol.
6. The participant has used any cannabis product or cannabidiol that is not in oral solution/capsule/tablet form, not obtained from a government-approved dispensary, or contains ≥50% Delta-9-tetrahydrocannabinol (THC)
7. The participant has unstable, clinically significant neurologic (other than the disease being studied; e.g., recurrent strokes), psychiatric, cardiovascular (e.g., pulmonary arterial hypertension, cardiac valvulopathy, orthostatic hypotension/tachycardia), pulmonary, hepatic, renal, metabolic, gastrointestinal, urologic, immunologic, hematopoietic, or endocrine disease or other abnormality which may impact the ability of the participant to participate or potentially confound the study results.
8. The participant is unwilling to comply with any of the study requirements or timelines.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Frequency percent change in countable motor seizures during Maintenance compared to Baseline

Secondary endpoints 9

1. Safety laboratory parameters
2. Physical examination findings
3. Vital signs
4. Growth parameters (height and weight)
5. 12-lead Electrocardiogram (ECGs)
6. Columbia-Suicide Severity Rating Scale (C-SSRS) responses
7. Patient Health Questionnaire-9 (PHQ-9) total score and Question 9 score
8. 50% responder rate (the percentage of participants with a ≥50% reduction in countable motor seizures) during Maintenance compared to Baseline
9. Frequency percent change in countable motor seizures during Treatment compared to Baseline

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Longboard Pharmaceuticals Inc.

Sponsor organisation

Longboard Pharmaceuticals Inc.

Address

4275 Executive Square Suite 950

City

La Jolla

Postcode

92037-9208

Country

United States

Scientific contact point

Organisation

Longboard Pharmaceuticals Inc.

Contact name

Global Regulatory Lead

Public contact point

Organisation

Longboard Pharmaceuticals Inc.

Contact name

Global Regulatory Lead

Third parties 11

Locations

11 EU/EEA countries · 42 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 320 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

17 submissions — initial application plus substantial / non-substantial modifications