A Multicenter, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Clinical Study to Evaluate the Efficacy and Safety of Efavirenz in Patients With Creutzfeldt-Jakob Disease
This is a multicenter, randomized, double-blind, placebo-controlled, parallel-group clinical trial designed to evaluate the efficacy and safety of efavirenz in patients with Creutzfeldt-Jakob disease (CJD). A total of 246 eligible participants will be enrolled across 21 study centers nationwide. Participants will be randomly assigned in a 1:1 ratio to receive either efavirenz or placebo. Participants in the efavirenz group will receive 200 mg once daily at bedtime for the first week, followed by an increased dose of 400 mg once daily thereafter. Participants in the placebo group will receive matching placebo tablets using the same dosing schedule. Treatment will be administered under double-blind conditions and will continue until death or study completion. During the study, all participants will receive monthly telephone follow-up assessments starting from treatment initiation to evaluate long-term efficacy and safety, continuing until death or study termination. The primary objective of the study is to determine whether efavirenz can prolong survival in patients with CJD. The primary endpoint is median survival time from randomization to death. Secondary endpoints include assessment of the effect of efavirenz on the rate of functional decline and treatment tolerability. Adverse events (AEs) and serious adverse events (SAEs) will be recorded and evaluated for frequency, severity, outcomes, and their relationship to the study drug. Key inclusion criteria include adults aged 18 to 80 years of either sex with a baseline MRC-Prion Disease Rating Scale (MRC-PDRS) score greater than 10 and the availability of a reliable caregiver to support study participation. Key exclusion criteria include the presence of other serious or life-threatening illnesses, use of medications contraindicated with efavirenz that cannot be adjusted, and pregnancy or breastfeeding. Written informed consent will be obtained from all participants or their legally authorized representatives prior to enrollment.
• Patients who meet the World Health Organization (WHO) diagnostic criteria for probable sporadic CJD or have a genetically confirmed diagnosis of hereditary CJD.
• Age 18-80 years, any sex.
• Baseline score on the Medical Research Council Prion Disease Rating Scale (MRC-PDRS) \>10 (i.e., retaining some functional ability).
• The patient has a caregiver aged ≥18 years who can accompany the patient during the study and assist in providing relevant information.
• The patient or their legally authorized representative has signed the informed consent form.