Adjunctive cannabidiol therapy in patients with CDKL5 deficiency disorder. Interventional drug study on efficacy and safety with focus on seizure effects

Adjunctive cannabidiol therapy in patients with CDKL5 deficiency disorder. Interventional drug study on efficacy and safety with focus on seizure effects - EPICDKL5

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2021-001959-13-IT

Enrollment

Registered

2021-07-27

Start date

2021-12-02

Completion date

Unknown

Last updated

2025-01-27

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

patients with CDKL5 MedDRA version: 20.0 Level: PT Classification code 10015039 Term: Epilepsy congenital System Organ Class: 10010331 - Congenital, familial and genetic disorders

Interventions

Trade Name: Epidyolex Product Name: Epidyolex Product Code: [Epidyolex] Pharmaceutical Form: Oral solution INN or Proposed INN: Cannabidiolo CAS Number: 13956-29-1 Current Sponsor code: Epidyolex Conc

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: •Male and female patients aged =1 year and = 30 years •Patients with a confirmed diagnosis of CDKL5 deficiency disorder •Patients with = 4 motor seizures/month in the last 6 months at baseline • Patients with 12-week baseline seizure frequency data available either from seizure diaries or medical records • Based on the physician's clinical judgment, seizures not adequately controlled with the current treatment Are the trial subjects under 18? yes Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 3 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range

Exclusion criteria

Exclusion criteria: • Patients on an investigational drug or device within 30 days prior to the initiation of the present study •Patients on an artisanal cannabis derivative treatment •Patients with severe hepatic impairment • Patients contraindicated for cannabidiol use (according to SmPC), including suspected sesame seed allergy or history of hypersensitivity to cannabidiol or any ingredients in the product •Pregnant or lactating females

Design outcomes

Primary

Measure	Time frame
Main Objective: •effectiveness and safety of cannabidiol adjunctive therapy in patients with CDKL5 deficiency disorder presenting uncontrolled seizures.;Secondary Objective: •to estimate the change of pre-/on-CBD EEGs, analysed for the following criteria: background frequency, focal slowing, reactivity, frequency of interictal epileptiform discharges (IED) and total number of recorded seizures; •to estimate the incidence of the following on subjects receiving CBD: -use of rescue medication; -hospitalization to treat seizures; -status epilepticus (SE); •to assess the effect of CBD on the following measures: -mean changes versus baseline of CGI-Improvement Score-CGI-I at 3, 6, and 12 months and of CBCL, DBC-P, sleep questionnaire at 12 months.;Primary end point(s): Primary endpoint of this study: •to assess the effect of CBD using the following effectiveness measures: -median percentage change from baseline in motor seizures frequency at 6 and 12 months; -% withdrawal due to lack of efficacy; -mean change in the number of concomitant AEDs; -50% responder rates for motor generalized seizures (patients with =50% reduction of all generalized seizures) at each time point visit; -median percentage change from baseline in all seizures type frequency at 3 and 12 months; -seizure freedom rate considering the last three months of treatment for motor generalized seizures and for all seizure types; -% change of days free from seizures for absences and myoclonic seizures; - score change from baseline on National Hospital Seizure Severity Scale (NHS3) for each seizure type at 6 and 12 months; •to assess the safety of CBD using the following measures: -retention rate at 6 and 12 months; -% withdrawal due to adverse reactions; -percentage of patients experiencing adverse reactions (ADRs) and serious adverse reactions;Timepoint(s) of evaluation of this end point: 6 and 12 months	—

Measure

Time frame

Main Objective: •effectiveness and safety of cannabidiol adjunctive therapy in patients with CDKL5 deficiency disorder presenting uncontrolled seizures.;Secondary Objective: •to estimate the change of pre-/on-CBD EEGs, analysed for the following criteria: background frequency, focal slowing, reactivity, frequency of interictal epileptiform discharges (IED) and total number of recorded seizures; •to estimate the incidence of the following on subjects receiving CBD: -use of rescue medication; -hospitalization to treat seizures; -status epilepticus (SE); •to assess the effect of CBD on the following measures: -mean changes versus baseline of CGI-Improvement Score-CGI-I at 3, 6, and 12 months and of CBCL, DBC-P, sleep questionnaire at 12 months.;Primary end point(s): Primary endpoint of this study: •to assess the effect of CBD using the following effectiveness measures: -median percentage change from baseline in motor seizures frequency at 6 and 12 months; -% withdrawal due to lack of efficacy; -mean change in the number of concomitant AEDs; -50% responder rates for motor generalized seizures (patients with =50% reduction of all generalized seizures) at each time point visit; -median percentage change from baseline in all seizures type frequency at 3 and 12 months; -seizure freedom rate considering the last three months of treatment for motor generalized seizures and for all seizure types; -% change of days free from seizures for absences and myoclonic seizures; - score change from baseline on National Hospital Seizure Severity Scale (NHS3) for each seizure type at 6 and 12 months; •to assess the safety of CBD using the following measures: -retention rate at 6 and 12 months; -% withdrawal due to adverse reactions; -percentage of patients experiencing adverse reactions (ADRs) and serious adverse reactions;Timepoint(s) of evaluation of this end point: 6 and 12 months

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Secondary

Measure	Time frame
Secondary end point(s): •to estimate the change of pre-/on-CBD EEGs, analysed for the following criteria: background frequency, focal slowing, reactivity, frequency of interictal epileptiform discharges (IED) and total number of recorded seizures; •to estimate the incidence of the following on subjects receiving CBD: -use of rescue medication; -hospitalization to treat seizures; -status epilepticus (SE); •to assess the effect of CBD on the following measures: - mean changes versus baseline of CGI-Improvement Score-CGI-I at 3, 6, and 12 months and of CBCL, DBC-P, sleep questionnaire at 12 months.;Timepoint(s) of evaluation of this end point: 3, 6 e 12 mesi	—

Measure

Time frame

Secondary end point(s): •to estimate the change of pre-/on-CBD EEGs, analysed for the following criteria: background frequency, focal slowing, reactivity, frequency of interictal epileptiform discharges (IED) and total number of recorded seizures; •to estimate the incidence of the following on subjects receiving CBD: -use of rescue medication; -hospitalization to treat seizures; -status epilepticus (SE); •to assess the effect of CBD on the following measures: - mean changes versus baseline of CGI-Improvement Score-CGI-I at 3, 6, and 12 months and of CBCL, DBC-P, sleep questionnaire at 12 months.;Timepoint(s) of evaluation of this end point: 3, 6 e 12 mesi

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Countries

Italy

Contacts

Public ContactDIREZIONE SCIENTIFICA

FONDAZIONE POLICLINICO UNIV A.GEMELLI IRCCS

direzione.scientifica@policlinicogemelli.it0630155701

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026