Creutzfeldt-Jakob DiseasePhase 3NCT07482085
Efavirenz for the Treatment of Creutzfeldt-Jakob Disease
A Phase 3 study testing Efavirenz in people with Creutzfeldt-Jakob Disease, run by Xuanwu Hospital, Beijing at 1 site in China.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Creutzfeldt-Jakob Disease
- What is being tested?
- Efavirenz
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 246 participants
- Where
- 1 siteChina
- Study dates
- 14 Apr 2026to Dec 2028
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Xuanwu Hospital, Capital Medical University
Beijing, 100053
Yan Yin, 010-83199270, xwkxsc@163.com
Liyong Wu, MD
About this study
Official title: 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.
Read more
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.
- Conditions
- Creutzfeldt-Jakob Disease
- Being tested
- Efavirenz, Placebo
- Keywords
- Creutzfeldt-Jakob disease, efavirenz
- Medical subject headings
- Creutzfeldt-Jakob Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EfavirenzDrug
The study drug in this trial is Efavirenz, and the control is a matching placebo. Efavirenz is supplied as 200 mg film-coated tablets. The placebo tablets are identical in appearance, color, and size to the Efavirenz tablets but contain no active ingredient. The main excipients of the placebo include lactose, microcrystalline cellulose, cross-linked sodium carboxymethyl cellulose, hydroxypropyl cellulose, sodium lauryl sulfate, purified water, Opadry, and an enteric film-coating premix. Both Efavirenz and placebo tablets are manufactured and supplied uniformly by the sponsor and dispensed in identical packaging with matching label numbers. The distribution and administration of the study drugs follow a double-blind procedure to ensure that neither investigators nor participants can determine the group assignment based on the appearance of the tablets, thereby maintaining blinding throughout the study.
PlaceboDrug
The study drug in this trial is Efavirenz, and the control is a matching placebo. Efavirenz is supplied as 200 mg film-coated tablets. The placebo tablets are identical in appearance, color, and size to the Efavirenz tablets but contain no active ingredient. The main excipients of the placebo include lactose, microcrystalline cellulose, cross-linked sodium carboxymethyl cellulose, hydroxypropyl cellulose, sodium lauryl sulfate, purified water, Opadry, and an enteric film-coating premix. Both Efavirenz and placebo tablets are manufactured and supplied uniformly by the sponsor and dispensed in identical packaging with matching label numbers. The distribution and administration of the study drugs follow a double-blind procedure to ensure that neither investigators nor participants can determine the group assignment based on the appearance of the tablets, thereby maintaining blinding throughout the study.
| Study group | Role | What they receive |
|---|---|---|
| Efavirenz Treatment Arm | Experimental | Participants will receive oral efavirenz at a dose of 200 mg once daily (1 tablet per dose). After 1 week of continuous treatment, the dose will be increased to 400 mg once daily (2 tablets per dose). To reduce central nervous system-related adverse effects, administration at bedtime is recommended.
Participants will continue study treatment under double-blind conditions from the time of randomization until the primary study endpoint is reached or the study is completed. Receives: Drug: Efavirenz |
| Placebo Control Arm | Placebo comparator | Participants will receive oral placebo tablets that are identical in appearance, color, and size to efavirenz tablets at a dose of 200 mg once daily (1 tablet per dose). After 1 week of continuous treatment, the dose will be increased to 400 mg once daily (2 tablets per dose).
Participants will continue study treatment until the primary study endpoint is reached or the study is completed. All other treatment procedures and conditions will be the same as those applied in the efavirenz treatment arm. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Median survival time
The median number of days from randomization to death
Measured: From randomization to death, assessed up to 36 months
Secondary outcomes (4)
Time to Loss of Independent Feeding Ability
Time from randomization to the first occurrence of loss of independent feeding ability, defined as the first time the feeding item in the Barthel Index drops to 0 (days)
Measured: From randomization until the feeding item in the Barthel Index first reaches 0, assessed up to 36 months
Time to Loss of Bowel and Bladder Control
Time from randomization to the first occurrence of loss of bowel and bladder control, defined as the first time both "bowel control" and "bladder control" items in the Barthel Index drop to 0 (days)
Measured: From randomization until both bowel and bladder control items in the Barthel Index first reach 0, assessed up to 36 months
Time to Development of Akinetic Mutism
Time from randomization to the onset of akinetic mutism, defined as the time at which the patient is independently assessed and jointly confirmed by two investigators to have no spontaneous movements but maintains a sleep-wake cycle (days)
Measured: From randomization until the patient meets criteria for akinetic mutism, assessed up to 36 months
Changes in MRC-PDRS and Barthel Index Scores
Changes in functional assessment scores from randomization to each pre-specified follow-up time point, including MRC-PDRS scores and Barthel Index scores, assessed monthly via telephone follow-up interviews with the patient or caregiver
Measured: From randomization to each monthly follow-up assessment, assessed every month up to 36 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Mar 2026
- Study start14 Apr 2026
- Primary completionDec 2028 (estimated)
- Study completionDec 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Creutzfeldt-Jakob DiseaseStudies of the same condition, open and under way.
- More trials run by Xuanwu Hospital, BeijingThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 246 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Xuanwu Hospital, Beijing
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07482085
- Sponsor's protocol ID
- 临研审[2026]009号-002
- Status last verified
- Aug 2026
- First submitted
- 6 Mar 2026
- Last updated
- 3 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: The results of this study will be summarized promptly after study completion, with consideration given to publication in academic journals. Any publication will adhere to ICMJE guidelines regarding authorship and disclosure of conflicts of interest. Regardless of whether the study results are positive or negative, they will be reported on public platforms (e.g., updates on the Chinese Clinical Trial Registry). Data sharing will follow the policies of the Beijing Hospital Authority and the sponsor. Reasonable requests from researchers for data sharing will be considered, and anonymized data may be provided as appropriate, ensuring the confidentiality of participants' personal information. Before any paper or report is published, no participant may disclose interim results or unpublished data without the sponsor's permission, to avoid misinterpretation of the findings. Published content will present aggregate data only, without revealing individual identities.
Publications
Lerner AJ, Arnold SE, Maxfield E, Koenig A, Toth ME, Fortin B, Mast N, Trombetta BA, Denker J, Pieper AA, Tatsuoka C, Raghupathy S, Pikuleva IA. CYP46A1 activation by low-dose efavirenz enhances brain cholesterol metabolism in subjects with early Alzheimer's disease. Alzheimers Res Ther. 2022 Dec 29;14(1):198. doi: 10.1186/s13195-022-01151-z.
PubMed 36581878Ali T, Hannaoui S, Nemani S, Tahir W, Zemlyankina I, Cherry P, Shim SY, Sim V, Schaetzl HM, Gilch S. Oral administration of repurposed drug targeting Cyp46A1 increases survival times of prion infected mice. Acta Neuropathol Commun. 2021 Apr 1;9(1):58. doi: 10.1186/s40478-021-01162-1.
PubMed 33795005
Study at a glance
From the registry record, last updated by the sponsor on 3 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT07482085
- Acronym / sponsor ID
- 临研审[2026]009号-002
- Conditions
- Creutzfeldt-Jakob Disease
- Keywords
- Creutzfeldt-Jakob disease, efavirenz
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 246 participants (estimated)
- Lead sponsor
- Xuanwu Hospital, BeijingUniversity, hospital or other
- Study start
- 14 Apr 2026 (actual)
- Primary completion
- Dec 2028 (estimated)
- Study completion
- Dec 2028 (estimated)
- First posted
- 19 Mar 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Efavirenz Treatment Arm | Experimental | Drug: EfavirenzParticipants will receive oral efavirenz at a dose of 200 mg once daily (1 tablet per dose). After 1 week of continuous treatment, the dose will be increased to 400 mg once daily (2 tablets per dose). To reduce central nervous system-related adverse effects, administration at bedtime is recommended. Participants will continue study treatment under double-blind conditions from the time of randomization until the primary study endpoint is reached or the study is completed. |
| Placebo Control Arm | Placebo comparator | Drug: PlaceboParticipants will receive oral placebo tablets that are identical in appearance, color, and size to efavirenz tablets at a dose of 200 mg once daily (1 tablet per dose). After 1 week of continuous treatment, the dose will be increased to 400 mg once daily (2 tablets per dose). Participants will continue study treatment until the primary study endpoint is reached or the study is completed. All other treatment procedures and conditions will be the same as those applied in the efavirenz treatment arm. |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (4 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| EfavirenzThe study drug in this trial is Efavirenz, and the control is a matching placebo. Efavirenz is supplied as 200 mg film-coated tablets. The placebo tablets are identical in appearance, color, and size to the Efavirenz tablets but contain no active ingredient. The main excipients of the placebo include lactose, microcrystalline cellulose, cross-linked sodium carboxymethyl cellulose, hydroxypropyl cellulose, sodium lauryl sulfate, purified water, Opadry, and an enteric film-coating premix. Both Efavirenz and placebo tablets are manufactured and supplied uniformly by the sponsor and dispensed in identical packaging with matching label numbers. The distribution and administration of the study drugs follow a double-blind procedure to ensure that neither investigators nor participants can determine the group assignment based on the appearance of the tablets, thereby maintaining blinding throughout the study. | Drug | Efavirenz Treatment Arm |
| PlaceboThe study drug in this trial is Efavirenz, and the control is a matching placebo. Efavirenz is supplied as 200 mg film-coated tablets. The placebo tablets are identical in appearance, color, and size to the Efavirenz tablets but contain no active ingredient. The main excipients of the placebo include lactose, microcrystalline cellulose, cross-linked sodium carboxymethyl cellulose, hydroxypropyl cellulose, sodium lauryl sulfate, purified water, Opadry, and an enteric film-coating premix. Both Efavirenz and placebo tablets are manufactured and supplied uniformly by the sponsor and dispensed in identical packaging with matching label numbers. The distribution and administration of the study drugs follow a double-blind procedure to ensure that neither investigators nor participants can determine the group assignment based on the appearance of the tablets, thereby maintaining blinding throughout the study. | Drug | Placebo Control Arm |
Primary outcome
Median survival time
Time frame: From randomization to death, assessed up to 36 months
The median number of days from randomization to death
4 secondary outcomes
Time to Loss of Independent Feeding Ability
Time frame: From randomization until the feeding item in the Barthel Index first reaches 0, assessed up to 36 months
Time to Loss of Bowel and Bladder Control
Time frame: From randomization until both bowel and bladder control items in the Barthel Index first reach 0, assessed up to 36 months
Time to Development of Akinetic Mutism
Time frame: From randomization until the patient meets criteria for akinetic mutism, assessed up to 36 months
Changes in MRC-PDRS and Barthel Index Scores
Time frame: From randomization to each monthly follow-up assessment, assessed every month up to 36 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Sep 2026.
- Background: PubMed 36581878Lerner AJ, Arnold SE, Maxfield E, Koenig A, Toth ME, Fortin B, Mast N, Trombetta BA, Denker J, Pieper AA, Tatsuoka C, Raghupathy S, Pikuleva IA. CYP46A1 activation by low-dose efavirenz enhances brain cholesterol metabolism in subjects with early Alzheimer's disease. Alzheimers Res Ther. 2022 Dec 29;14(1):198. doi: 10.1186/s13195-022-01151-z.
- Background: PubMed 33795005Ali T, Hannaoui S, Nemani S, Tahir W, Zemlyankina I, Cherry P, Shim SY, Sim V, Schaetzl HM, Gilch S. Oral administration of repurposed drug targeting Cyp46A1 increases survival times of prion infected mice. Acta Neuropathol Commun. 2021 Apr 1;9(1):58. doi: 10.1186/s40478-021-01162-1.
- Individual participant dataSharing planned: Yes. The results of this study will be summarized promptly after study completion, with consideration given to publication in academic journals. Any publication will adhere to ICMJE guidelines regarding authorship and disclosure of conflicts of interest. Regardless of whether the study results are positive or negative, they will be reported on public platforms (e.g., updates on the Chinese Clinical Trial Registry). Data sharing will follow the policies of the Beijing Hospital Authority and the sponsor. Reasonable requests from researchers for data sharing will be considered, and anonymized data may be provided as appropriate, ensuring the confidentiality of participants' personal information. Before any paper or report is published, no participant may disclose interim results or unpublished data without the sponsor's permission, to avoid misinterpretation of the findings. Published content will present aggregate data only, without revealing individual identities.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Efavirenz in people with Creutzfeldt-Jakob Disease, run by Xuanwu Hospital, Beijing at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 3 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Zhongyun Chen, MD (+86-10-1365-100-6467, chenzhongyun3@163.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Median survival time (time frame: From randomization to death, assessed up to 36 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT07482085, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Sep 2026.