A Double-Blind Trial to Evaluate Efficacy and Safety of Cannabidiol as an add-on Therapy for Treatment in Refractory Epilepsy

NCT02783092 | Unknown Phase 3

• 1 other identifier: CBD-PRATI-USP_01
• Study Type: interventional
• Target: 126
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this study is to assess the efficacy of the adjuvant use of cannabidiol administered twice daily in doses of 5-25 mg/kg/day through the proportion of responsive patients; that is, participants with at least 50% decrease in the frequency of epileptic seizures in the last month of the trial relative to baseline (pretreatment with AEDs only). Primary end point(s): Rate of responsive patients; that is, participants with at least 50% decrease in the frequency of epileptic seizures in the last month of the trial relative to baseline (pretreatment with AEDs only).

Conditions

Epilepsy

Outcome Measures

Primary Outcomes (1)

• Frequency of epileptic seizures

  17 th week

Study Arms (2)

Cannabidiol

EXPERIMENTAL

Concentration unit: 200mg/ml; 5 - 25 mg/Kg/day ; Oral solution

Drug: Cannabidiol

Placebo

PLACEBO COMPARATOR

Oral solution

Drug: Placebo

Interventions

Cannabidiol DRUG

Oral solution of cannabidiol 200 mg / mL dissolved in corn oil. Titration period: 5 mg / kg / day up to 25 mg / kg / day. Maintenance period: highest dose obtained during the titration period (maximum of 25 mg/kg / day)

Also known as: CBD

Cannabidiol

Placebo DRUG

The placebo will be an oral solution of corn oil. Titration period: 5 mg / kg / day up to 25 mg / kg / day. Maintenance period: highest dose obtained during the titration period (maximum of 25 mg/kg / day)

Also known as: PLC

Placebo

Eligibility Criteria

• Age: 2 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Men and women aged 2 years to 18 years.
• Diagnosis of treatment-resistant epilepsy according to the criteria of the International League Against Epilepsy (ILAE) (Kwan et al., 2010).
• Participants with at least 4 epileptic seizures with intervals no longer than 21 days.
• In treatment with up to 3 AEDs concomitantly and at stable doses for at least 1 month before the baseline assessment and expected to remain stable during the period of the trial. Vagus nerve stimulation (VNS) will be considered as an AED.
• Availability of a legal guardian able to follow the protocol (e.g., understand and fill up diaries) and visitation and medication schemes, according to the decision of the investigator.
• Availability of brain neuroimaging exams (magnetic resonance or computed tomography) collected within the last 5 years.
• No significant comorbid conditions, according to medical decision, to other criteria in this Protocol, and to additional assessments: medical records, blood pressure, heart rate, and temperature measures, physical exam, ECG, EEG, and laboratory tests.
• Women in reproductive age may be included as long as they are sexually abstinent or using effective contraceptive methods.
• Participants and their legal guardians, when applicable, must sign an informed consent form approved by the local ethics committee.

You may not qualify if:

• Occurrence of simple partial seizures (preserved consciousness) only, with no motor symptomatology.
• History or presence of pseudoseizures.
• History of suicide attempt.
• History of major depression.
• Pregnancy.
• Drug use.
• Hypertension.
• Participants with severe dysphagia and no gastric or nasogastric tubes.
• Current treatment with drugs that may significantly affect the metabolism of CBD, except AEDs if stable for at least 1 month before the screening interview.
• Presence of any clinical or neuroimaging finding suggestive of brain disorders, brain tumors, or metabolic or neurodegenerative diseases of rapid progression.
• Presence of acute and clinically significant diseases as assessed by a medical investigator, such as kidney, liver, urinary, bowel, or respiratory infections.
• Presence of known chronic and clinically significant diseases as assessed by a medical investigator and which may interfere with participation in the trial or pose safety risks for the participant.
• History of liver, kidney, lung, hematological, heart, or psychiatric diseases that may affect the volunteers' health or participation in the trial.
• Hypotension or hypertension with any etiology and requiring pharmacological management.
• History of surgeries that may affect the volunteers' health and/or participation in the trial.

Sponsors & Collaborators

• Antonio Waldo Zuardi: lead
• Prati Donaduzzi & Cia Ltda: collaborator

Study Sites (1)

Unidade de Pesquisa Clínica HCRP-USP

Ribeirão Preto, São Paulo, 14048900, Brazil

Location

MeSH Terms

Conditions

Epilepsy

Interventions

Cannabidiol

Condition Hierarchy (Ancestors)

Brain Diseases; Central Nervous System Diseases; Nervous System Diseases

Intervention Hierarchy (Ancestors)

Cannabinoids; Terpenes; Hydrocarbons; Organic Chemicals

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: MD, PhD, Professor

Study Record Dates

• First Submitted: May 23, 2016
• First Posted: May 26, 2016
• Study Start: January 9, 2019
• Primary Completion: July 10, 2024
• Study Completion: September 25, 2024
• Last Updated: November 29, 2023

Record last verified: 2023-05

Locations

BR (1)