NCT07482085 · Phase 3 · RECRUITING
Efavirenz for the Treatment of Creutzfeldt-Jakob Disease
This trial tests whether efavirenz, an existing HIV medicine, helps people with Creutzfeldt-Jakob disease (CJD) live longer compared with a placebo. About 246 people will take it daily until death or the end of the study. It is a Phase 3 trial, a large comparison testing whether it works and is safe, not yet proven or approved for CJD.
You may qualify if
- 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.
You're excluded if
- Presence of severe somatic diseases or unstable clinical conditions that may affect study compliance or patient safety, including malignancy, advanced liver or kidney dysfunction, severe cardiac disease (including patients with a history of significant QTc prolongation).
- Current use of drugs that are known contraindications with efavirenz and cannot be discontinued or substituted.
- Female participants who are pregnant or breastfeeding.
- Other medical or psychiatric conditions, as judged by the investigator, that may interfere with the patient's participation in or completion of the study.
The sponsor's own eligibility wording, lightly reformatted. The study team makes the final eligibility decision — worth discussing with your doctor.
Eligibility criteria as of 2026-09-03