NCT05485831

Brief Summary

This is a prospective, observational study on approximately 70-100 Real World participants affected by LGS, DS, or TSC treated with Epidyolex® as prescribed in the summary of product characteristics. The eligible participants are expected to participate in the study for a duration of 52 weeks of treatment.

Trial Health

77
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
100

participants targeted

Target at P50-P75 for all trials

Timeline
9mo left

Started Feb 2025

Geographic Reach
1 country

18 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress62%
Feb 2025Feb 2027

First Submitted

Initial submission to the registry

July 29, 2022

Completed
5 days until next milestone

First Posted

Study publicly available on registry

August 3, 2022

Completed
2.6 years until next milestone

Study Start

First participant enrolled

February 24, 2025

Completed
1.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

February 1, 2027

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

February 1, 2027

Last Updated

February 17, 2026

Status Verified

February 1, 2026

Enrollment Period

1.9 years

First QC Date

July 29, 2022

Last Update Submit

February 13, 2026

Conditions

Keywords

seizuresEpidyolexcannabidiol oral solution

Outcome Measures

Primary Outcomes (1)

  • Number of Participants Remaining on Therapy from Enrollment

    Treatment retention will be evaluated through the proportion of participants remaining on therapy from the enrollment visit (baseline, V0) to each study visit (Weeks 4 \[V1\], 16 \[V2\], 28 \[V3\], 40 \[V4\], 52 \[V5\]).

    Baseline up to Week 52 post-dose.

Secondary Outcomes (16)

  • Average Number of Seizure-Free Days in the Last 28 Days

    Weeks 4 (V1), 16 (V2), 28 (V3), 40 (V4) and 52 (V5)

  • Longest Duration of Seizure Free Days in the Last 28 Days

    Weeks 4 (V1), 16 (V2), 28 (V3), 40 (V4) and 52 (V5)

  • Average Maintenance Dose of Epidyolex®

    Weeks 4 (V1), 16 (V2), 28 (V3), 40 (V4) and 52 (V5)

  • Maximum Maintenance Dose of Epidyolex®

    Weeks 4 (V1), 16 (V2), 28 (V3), 40 (V4) and 52 (V5)

  • Type, Dosage, and Frequency of Concomitant Anti-Seizure Medications (ASMs)

    Weeks 4 (V1), 16 (V2), 28 (V3), 40 (V4) and 52 (V5)

  • +11 more secondary outcomes

Study Arms (1)

Lennox Gastaut, Dravet Syndrome, and Tuberous Sclerosis Complex

Participants ≥2 years of age diagnosed with LGS, DS, and TSC.

Drug: Epidiolex 100 mg/mL Oral Solution

Interventions

As prescribed in routine clinical practice in Italy.

Lennox Gastaut, Dravet Syndrome, and Tuberous Sclerosis Complex

Eligibility Criteria

Age2 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodProbability Sample
Study Population

The study will comprise of 70-100 male and female participants, aged ≥ 2 years, diagnosed with LGS, DS, and TSC. The decision to prescribe Epidyolex® as adjunctive therapy for seizures associated with LGS, DS, and TSC will be entirely independent of including the participant in the study.

You may qualify if:

  • Adult patients, and minor patients ≥ 2 years old diagnosed with LGS, DS or TSC.
  • Clinical decision, taken by the physician, to initiate Epidyolex®
  • Adult participants, parents or legal representatives must be willing and able to give informed consent/assent for participation in the study.

You may not qualify if:

  • Participants currently using or have used recreational, medicinal cannabis, or cannabinoid-based products within the three months prior to study entry and are unwilling to abstain from these products for the duration of the study.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (18)

Policlinico Sant'Orsola di Bologna

Bologna, 40138, Italy

RECRUITING

NPI AOU Mater Domini Università Magna Graecia

Catanzaro, 88100, Italy

RECRUITING

Università degli studi "G. D'annunzio" Chieti

Chieti, 66100, Italy

RECRUITING

Azienda Ospedaliera Universitaria Meyer Neurologia Pediatrica

Florence, 50139, Italy

RECRUITING

Istituto Gaslini Neurologia Pediatrica e Malattie Muscolari

Genova, 16147, Italy

RECRUITING

AOU Gaetano Martino

Messina, 98124, Italy

RECRUITING

Centro Regionale Epilessia - ASST Santi Paolo e Carlo

Milan, 20142, Italy

RECRUITING

ASST Grande Ospedale metropolitano Niguarda

Milan, 20162, Italy

RECRUITING

Azienda Ospedaliera Universitaria Federico II

Naples, 80131, Italy

RECRUITING

AOU Maggiore della Carità di Novara

Novara, 28100, Italy

RECRUITING

AOU di Padova

Padua, 35128, Italy

RECRUITING

Policlinico Tor Vergata

Roma, 00133, Italy

RECRUITING

IRCCS Ospedale Pediatrico Bambin Gesù

Roma, 00165, Italy

RECRUITING

Fondazione Policlinico Universitario Agostino Gemelli - IRCCS-NPI

Roma, 00168, Italy

RECRUITING

Azienda Ospedaliero Universitaria Sant'Andrea

Roma, 00189, Italy

RECRUITING

Ospedale Casa del sollievo e della sofferenza

San Giovanni Rotondo, 71013, Italy

RECRUITING

AOU Città della Salute PO Molinette

Torino, 10126, Italy

RECRUITING

Azienda Ospedaliero Universitaria delle Marche

Torrette, 60126, Italy

RECRUITING

MeSH Terms

Conditions

Lennox Gastaut SyndromeEpilepsies, MyoclonicTuberous SclerosisSeizures

Interventions

CannabidiolSolutions

Condition Hierarchy (Ancestors)

Epileptic SyndromesEpilepsyBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesEpilepsy, GeneralizedHamartomaNeoplasmsNeoplasms, Multiple PrimaryNeoplastic Syndromes, HereditaryMalformations of Cortical Development, Group IMalformations of Cortical DevelopmentNervous System MalformationsNeurocutaneous SyndromesHeredodegenerative Disorders, Nervous SystemNeurodegenerative DiseasesCongenital AbnormalitiesNeurologic ManifestationsSigns and SymptomsPathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

CannabinoidsTerpenesHydrocarbonsOrganic ChemicalsPharmaceutical Preparations

Central Study Contacts

Clinical Trial Disclosure & Transparency

CONTACT

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

July 29, 2022

First Posted

August 3, 2022

Study Start

February 24, 2025

Primary Completion (Estimated)

February 1, 2027

Study Completion (Estimated)

February 1, 2027

Last Updated

February 17, 2026

Record last verified: 2026-02

Data Sharing

IPD Sharing
Will not share

Locations