Cancer drug may reveal HIV's secret brain hideout
NCT ID NCT03239899
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tested whether a single dose of pembrolizumab, a cancer drug, is safe for people with HIV who do not have cancer. The goal was to see if the drug could help reveal HIV hiding in the brain. Thirteen adults with well-controlled HIV participated. The study was not meant to treat HIV, but to learn more about where the virus may persist.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- pembrolizumab
- What this could lead to
- If successful, this could help researchers understand where HIV hides in the brain, potentially pointing toward future strategies to eliminate the virus.
- What could go wrong
- This is a very early, small safety study with only 13 participants and a single dose. It is not designed to treat HIV, and pembrolizumab can cause serious immune-related side effects.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
-
Phase 1
The first testing in people. Mainly checks safety and dose, usually in a small group.
- Participants
-
13 people
The number who actually took part.
- Started
-
Apr 2018
- Finished
-
Feb 2023
- Lead sponsor
-
A government research agency
The lead sponsor is the US National Institutes of Health.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
-
18 to 120 years
- Sex
-
Anyone
- Healthy volunteers
-
Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
* INCLUSION CRITERIA: * 18 years or older * Diagnosis of HIV-1 infection, with positive HIV 1 antibody testing * HIV RNA less than or equal to 40 copies/mL in plasma in the last 12 or greater months * CD4 count above 350 cells/uL * Antiretroviral therapy for 12 months prior to trial * Fully vaccinated against SARS-CoV-2. Fully vaccinated is defined as: * Two weeks out from the second dose of a two-dose vaccine series (Moderna, Pfizer-BioNTech); or * Two weeks out from a single-dose vaccine (Johnson \& Johnson/Janssen) * Patient must be willing and able to comply with all the aspects of trial design and follow-up. * Patients must be able to provide informed consent * Women of childbearing potential must agree to use contraception (defined as two forms of effective birth control), from the time of enrollment until 4 months after the last exposure to pembrolizumab * Participants who are physically able to father a child must agree to use 2 effective methods of contraception (birth control) from the time you enroll in the study until 4 months after your last exposure to pembrolizumab * Effective methods of contraception for this study include: * hormonal contraception (birth control pills, birth control patches, injected hormones, hormonal implants or vaginal ring), * Intrauterine device, * Barrier methods (condom or diaphragm) combined with spermicide, and * Surgical sterilization (hysterectomy, tubal ligation, or vasectomy). * If you have had a hysterectomy, tubal ligation, or vasectomy (or have a partner with a hysterectomy, tubal ligation or vasectomy), you do not have to use 2 methods of birth control. EXCLUSION CRITERIA: * Clinically significant medical disorders that might expose the patient to undue risk of harm confound study outcomes or prevent the patient from completing the study as identified on screening studies and by patient history. Examples of such conditions include known cardiac disease such as congestive heart failure, chronic obstructive pulmonary disease, uncontrolled hypertension, kidney disease, liver disease, endocrine disease, pulmonary disease, heart disease, progressive CNS disease such as Parkinson s disease, dementia, prior tuberculosis infection or ongoing CNS opportunistic infection. * Patient has received immunomodulatory/immunosuppressive therapy (including IV steroids but excluding local injections) in the preceding 6 months. * Patient with known autoimmunity that would include but is not limited to disorders such as hypo/hyperthyroidism, myasthenia gravis, diabetes mellitus type 1, hemolytic anemia, and immune mediated hepatitis (but excluding patients with hypothyroidism already on thyroid replacement therapy). * Prior history of cancer (excluding non-invasive squamous and basal cell carcinoma) * Any opportunistic infection in the prior 2 years (excluding thrush) including latent TB (or a positive TB Quantiferon Gold test) * Patient has received other investigational drugs within 3 months before enrollment * Positive serological or PCR evidence of active or prior infection with HTLV-1/II, Hepatitis B or C. Patients with hepatitis B core (+), surface antibody (+), surface antigen ( ) and hepatitis B DNA (-) eligible to participate in the study (provided they are on tenofovir, lamivudine or TAF). Participants with prior hepatitis C who are hepatitis C antibody (+) but hepatitis C RNA (-) with normal liver enzymes and no evidence of cirrhosis on clinical liver ultrasound are eligible to participate in the study. * Metal in the body which would make having an MRI scan unsafe, such as pacemakers, stimulators, pumps, aneurysm clips, metallic prostheses, artificial heart valves, cochlear implants or shrapnel fragments, or history of welding or metal worker * Claustrophobia * Inability to lie comfortably on the back for up to two hours. * Abnormal anti-thyroid panel (anti-TPO and anti-TG) test at screening visit. * Abnormal screening/baseline blood tests exceeding any of the limits defined below or as deemed exclusionary by the investigators on review: * AST and ALT values \>1.1 times ULN * Fasting triglyceride \> 300 mg/dL * Total bilirubin \>1.1 times ULN (unless participant is taking atazanavir or has Gilbert syndrome) * Creatinine Clearance or eGFR \<60 ml/minute (adjusted for race) * Hemoglobin \< 10 g/dL * Absolute neutrophil count \< 1000/microliter * Platelet count \<130,000/mm(3) (if platelet clumping is present on hematology slide review, platelet count \<100,000/mm3 is considered exclusionary to study) * Hemoglobin A1c greater than or equal to 6% * Thyroid-stimulating hormone (TSH) and adrenocorticotropic hormone (ACTH) within normal limits. If TSH is not within normal limits then the participant may be eligible if thyroxine (T4) is within normal limits. Participants are not excluded if they are on a stable dose of replacement thyroid medication; dose may be adjusted as needed. * An employee or staff of the NIH
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for HIV infections are added.
Genom att skicka in godkänner du våra Användarvillkor
Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
-
National Institutes of Health Clinical Center
Bethesda, Maryland, 20892, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can a Three-Session couples program cut drug use and HIV risk?
- Decades of HIV data could reveal which treatments last
- Can testing together again keep male couples HIV-Free?
- Can a rapid HIV test get people treated sooner?
- Can Same-Day HIV prevention reach people before infection takes hold?
- New injectable HIV prevention drug tested in healthy volunteers