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MonkeypoxPhase 1/2NCT05988203

A Clinical Study Investigating the Safety and Immune Responses After Immunization With Investigational Monkeypox Vaccines

A Phase 1/2 study testing BNT166a in people with Monkeypox, run by BioNTech SE at 9 sites in 2 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial summary

This was a dose-escalation, Phase I/II study evaluating the safety, tolerability, reactogenicity and immunogenicity of the investigational RNA-based multivalent vaccine candidate BNT166a for active immunization against monkeypox (mpox). Read more about this study

Who is it for?
Ages 18 to 65 yearsHealthy volunteers can take part
Conditions the study is for
Monkeypox
What is being tested?
BNT166a
Could I receive a placebo?
No, everyone receives an active treatment
How many people
96 participants
Where
9 sitesin 2 countries
Study dates
21 Sep 2023to 4 Mar 2026
Phase
Phase 1/2
Are there results?
Yes, posted in the registrySummary in the doctors' view

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 65 years
  • All sexes
  • Healthy volunteers accepted
  • Adult, Older adult

Inclusion Criteria (applicable to all substudies unless otherwise specified):

  • Had given informed consent by signing and dating the informed consent form (ICF) before initiation of any study-specific procedures.
  • Were willing and able to comply with scheduled visits, treatment schedule, laboratory tests, and other requirements of the study, including the prohibited concomitant medications. This included that they were able to understand and follow study-related instructions.
  • SSA and SSD only: Were 18 through 45 years of age (inclusive) at the time of informed consent.
  • SSB only: Were 50 through 65 years of age (inclusive) at the time of informed consent.
  • Had a body mass index over 18.5 kg/m^2 and under 30 kg/m^2 and weighed at least 50 kg at Visit 0.
  • Were healthy, in the clinical judgment of the investigator based on volunteer-reported medical history data, physical examination, 12-lead electrocardiogram (ECG), vital signs, and clinical laboratory test results.
  • SSA and SSD only: Had no prior history of known or suspected smallpox vaccination and no detectable smallpox vaccination characteristic scar (vaccinia-naïve participants).
  • SSB only: Had a history of prior smallpox vaccination (i.e., are vaccinia-experienced), determined based on medical records and/or presence of smallpox vaccination characteristic scar. The most recent smallpox vaccination was received before 1980.
  • Agreed not to enroll in another study with an investigational medicinal product starting from Visit 0 and until the end of this study.
  • Negative human immunodeficiency virus (HIV)-1 and HIV-2 antigen/antibody blood test result at Visit 0.
  • Negative Hepatitis B surface antigen and negative core antibodies test results and negative anti Hepatitis C virus antibodies (anti-HCV), or negative Hepatitis C virus (HCV) polymerase chain reaction test result if the anti-HCV was positive at Visit 0.
  • Volunteers of childbearing potential (VOCBP) must not have been pregnant. VOCBP and men who were sexually active with partners of childbearing potential and their sexual partners born female should have used a highly effective form of contraception from at least 28 days prior to Dose 1 up to at least 90 days after receiving the last dose of study treatment, and should have agreed not to donate eggs (ova, oocytes) or sperm.

Exclusion Criteria (applicable to all substudies unless otherwise specified):

  • History of mpox, smallpox or vaccinia infection based on volunteer-reported medical history.
  • Pregnant, breastfeeding, were planning pregnancy or were planning to father children starting from Visit 0 and continuously until 90 days after receiving Dose 2.
  • History of known or suspected severe adverse reaction including allergic reaction (e.g., anaphylaxis) to vaccines or to vaccine components such as lipids.
  • Current or history of the following medical conditions at Visit 0 or Visit 1:
  • Uncontrolled, moderate or severe asthma; asthma severity as defined in the US National Asthma Education and Prevention Program Expert Panel report
  • Chronic obstructive pulmonary disease.
  • Diabetes mellitus type 1 or type 2, including cases controlled with diet alone (Not excluded: history of isolated gestational diabetes).
  • Hypertension: If a person had hypertension, excluded for blood pressure that was not well controlled. Well controlled blood pressure was defined as consistently <=140 mm Hg systolic and <=90 mm Hg diastolic, with or without medication, with only isolated, brief instances of higher readings, which must have been <150 mm Hg systolic and <100 mm Hg.
  • Systolic blood pressure >=150 mm Hg or diastolic blood pressure >=100 mm Hg.
  • Malignancy, excluding localized basal or squamous cell cancer.
  • Cardiovascular diseases, (e.g., myocarditis, pericarditis, coronary heart disease, myocardial infarction, congestive heart failure, cardiomyopathy or clinically significant arrhythmias, stroke or transient ischemic attack).
  • Bleeding disorders (e.g., factor deficiency, coagulopathy, or platelet disorder).
  • Seizure disorder: History of seizure(s) within past 3 years; used medications in order to prevent or treat seizure(s) at any time within the past 3 years.
  • Estimated glomerular filtration rate <60 mL/min/1.73 m^2.
  • Chronic liver disease.
  • Schizophrenia, major depressive disorder, suicidal ideation. Such psychiatric illnesses, as bipolar disorder, autism and attention deficit-hyperactivity disorder that at the discretion of the investigator could interfere with participation and follow-up as outlined by the study.
  • Current or history of the following diseases associated with immune dysregulation:
  • Known or suspected immunodeficiency.
  • History of solid organ or bone marrow transplantation.
  • Asplenia: any condition resulting in the absence of a functional spleen.
  • Currently existing or history of any autoimmune disease.
  • SSA and SSB: At Visit 0, any screening hematology and/or blood chemistry laboratory value that met the definition of a Grade >=1 abnormality (according to the FDA toxicity grading scale; see separate exclusion criteria for bilirubin and troponin I). Individuals with any stable Grade 1 abnormalities had been considered eligible at the discretion of the investigator. A stable Grade 1 laboratory abnormality was defined as the value which was <= Grade 1 upon repeated testing on a second sample from this individual during the screening period (prior to Visit 1). Individuals with abnormal but not clinically significant parameters not included in the FDA toxicity guidance might have been considered eligible at discretion of investigator.
  • SSD only: At Visit 0, any screening hematology and/or blood chemistry laboratory value (according to the FDA toxicity grading scale) that met the definition of a Grade >=2 abnormality; individuals with clinically non-significant Grade 1 abnormalities may be considered eligible at the discretion of the investigator. Individuals with abnormal but clinically non-significant parameters not included in the FDA toxicity guidance might have been considered eligible at the discretion of the investigator. See separate exclusion criteria for bilirubin and troponin I.
  • Abnormal total bilirubin at Visit 0. Note: inclusion of volunteers with bilirubin <=1.25 upper limit of normal (ULN) if due to Gilbert's syndrome was allowed.
  • Any abnormal troponin I value at Visit 0.
  • A 12-lead ECG at Visit 0 which was consistent with probable or possible myocarditis/pericarditis or which demonstrated clinically relevant abnormalities that may have affected participant safety or are otherwise clinically significant findings (e.g., complete left bundle branch block, atrioventricular (AV) block, average corrected QT interval by Fridericia (QTcF interval) >450 msec, signs of myocardial infarction, sinus tachycardia (ST) elevation consistent with myocardial ischemia, or serious brady- or tachyarrhythmias).
  • Febrile illness (body temperature >=38.0°C) or other acute illness within 48 hours prior to Dose 1 and/or current (if presented at Visit 1, temporary deferral was allowed).
  • Participated or had planned participation in strenuous or endurance exercise within 7 days before or after each investigational medicinal product (IMP) administration.
  • SSA and SSD only: Vaccination with any Orthopoxvirus-based vaccine including vaccines for prevention of smallpox, disease caused by vaccinia virus or mpox, or vector Orthopoxvirus-based vaccines.
  • SSB only: Vaccination for prevention of mpox or disease caused by vaccinia virus, or with vector Orthopoxvirus-based vaccine. Vaccination for prevention of smallpox done in or after 1980.
  • Any vaccination within 28 days before Dose 1. Seasonal inactivated influenza vaccine was allowed, however, it should have been administered at least 14 days before IMP administration.
  • Any non-study IMP within 28 days or five half-lives (whichever was longer) before Dose 1.
  • Blood/plasma products and/or immunoglobulins within 120 days before Dose 1.
  • Allergy treatment with antigen injections within 14 days before Dose 1.
  • Immunosuppressive therapy, including corticosteroids, or radiotherapy within 6 months or five half-lives (whichever is longer) before Dose 1. If systemic corticosteroids were administered short term (<=14 days, at a dose of <=20 mg/day of prednisone or equivalent) for treatment of an acute illness, individuals should have been enrolled in the study only after corticosteroid therapy was discontinued for at least 28 days before Dose 1. Intraarticular, intrabursal, or topical (skin or eyes) corticosteroids were permitted.
  • Had a history of alcohol abuse within 1 year before Visit 0 or had a history of substance abuse within the past 5 years before Visit 0.
  • Were vulnerable individuals as per international council for harmonisation (ICH) E6 definition, i.e., were individuals whose willingness to volunteer in a clinical study might have been unduly influenced by the expectation, whether justified or not, of benefits associated with participation, or of a retaliatory response from senior members of a hierarchy in case of refusal to participate.

Trial locations and contacts

9 sites in 2 countries.

9 sites in 2 countries

United Kingdom5 sites
  • Addenbrooke's Hospital - Cambridge University Hospitals NHS Foundation Trust

    Cambridge, CB2 0QQ

  • Royal Surrey County Hospital Foundation Trust, NIHR Royal Surrey Clinical Research Facility

    Guildford, GU2 7XP

  • Guy's and St Thomas' NHS Foundation Trust of St Thomas' Hospital

    London, SE1 7EH

  • Medicine Evaluation Unit Ltd, The Langley Building, Wythenshawe Hospital

    Manchester, M239QZ

  • University Hospital Southampton

    Southampton, SO16 6YD

Other trials in United Kingdom

United States4 sites
  • Alliance for Multispecialty Research, LLC

    Kansas City, Missouri, 64114

  • Alliance for Multispecialty Research, LLC

    Knoxville, Tennessee, 37909

  • California Research Foundation

    San Diego, California, 92123

  • University of Washington Virology Research Clinic

    Seattle, Washington, 98104

Other trials in United States

About this study

Official title: A Randomized, Partially Observer-blind, Dose-escalation, Phase I/II Trial Evaluating the Safety and Immunogenicity of Investigational RNA-based Mpox Vaccine Candidates

This was a dose-escalation, Phase I/II study evaluating the safety, tolerability, reactogenicity and immunogenicity of the investigational RNA-based multivalent vaccine candidate BNT166a for active immunization against monkeypox (mpox).

This study was originally planned to include four substudies, i.e., substudy A (SSA), substudy B (SSB), substudy C (SSC), and substudy D (SSD). Sponsor decided not to conduct SSC, thus three substudies (SSA, SSB, and SSD) were conducted.

Read more

In SSA and SSB, dosing started with an initial sentinel group, followed by the expansion cohort. In SSD, dosing was initiated after the interim analysis of SSA and SSB 1-month post-Dose 2 safety, reactogenicity, and immunogenicity data was received.

This study was initially planned to investigate two vaccine candidates (the quadrivalent BNT166a and the trivalent BNT166c). The sponsor decided to not activate the groups with BNT166c.

Read the detailed description

Substudy A was an open-label, dose-escalation, Phase I substudy to assess the reactogenicity, safety, and immunogenicity of up to three dose levels of the multivalent vaccine candidate BNT166a in 48 healthy participants with no prior history of known or suspected smallpox vaccination (vaccinia-naïve participants).

Substudy B was a one group, open-label, Phase I substudy to assess the reactogenicity, safety and immunogenicity of the multivalent vaccine candidate BNT166a in 16 healthy participants with prior history of smallpox vaccination (vaccinia-experienced).

Substudy D was a one group, open-label, Phase IIa substudy to assess the reactogenicity, safety, and immunogenicity of one dose level of BNT166a in ~32 healthy participants with no prior history of known or suspected smallpox vaccination (i.e., vaccinia-naïve participants). SSD was initiated after the interim analysis of SSA and SSB safety, reactogenicity, and immunogenicity data.

The duration of study participation was ~ 14 months per participant in all of the substudies.

Conditions
Monkeypox
Being tested
BNT166a
Keywords
Prevention of mpox, Monkeypox virus, RNA vaccine, Vaccine, mpox, MPXV, Monkeypox
Medical subject headings
Mpox, Monkeypox

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • BNT166aBiological

    Multivalent ribonucleic acid (RNA)-based vaccine for active immunization against monkeypox administered as intramuscular injection.

Study groups in NCT05988203
Study groupRoleWhat they receive
BNT166a SSA: 10 mcgExperimentalParticipants received two intramuscular doses of the multivalent RNA-based vaccine BNT166a at Visit 1 (Day 1) and at Visit 5 (Day 31) for active immunization against monkeypox.
Receives: Biological: BNT166a
SSA: BNT166a 30 mcgExperimentalParticipants received two intramuscular doses of the multivalent RNA-based vaccine BNT166a at Visit 1 (Day 1) and at Visit 5 (Day 31) for active immunization against monkeypox.
Receives: Biological: BNT166a
SSA: BNT166a 60 mcgExperimentalParticipants received two intramuscular doses of the multivalent RNA-based vaccine BNT166a at Visit 1 (Day 1) and at Visit 5 (Day 31) for active immunization against monkeypox.
Receives: Biological: BNT166a
SSB: BNT166a 30 mcgExperimentalParticipants received two intramuscular doses of the multivalent RNA-based vaccine BNT166a at Visit 1 (Day 1) and at Visit 5 (Day 31) for active immunization against monkeypox.
Receives: Biological: BNT166a
SSD: BNT166a 60 mcgExperimentalParticipants received two intramuscular doses of the multivalent RNA-based vaccine BNT166a at Visit 1 (Day 1) and at Visit 6 (Day 31) for active immunization against monkeypox.
Receives: Biological: BNT166a

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Number of Participants Reporting Solicited Local Reactions At the Injection Site

    All reactogenicity events information recorded via the participant e-diaries or solicited by the investigator were considered as solicited events. Solicited local reactions were: pain at the injection site, erythema/redness, and induration/swelling. Any local reaction indicates participants with any reactions, including reactions that do not qualify for Grade 1 (<2.5 cm for erythema/redness or induration/swelling). The intensity of local reaction was assessed by the participant and may be confirmed or corrected by the investigator; grades were defined as Grade 1 = Mild; Grade 2 = Moderate; Grade 3 = Severe; Grade 4 = Potentially life-threatening.

    Measured: Up to 7 days post any vaccination, and up to 7 days post each vaccination dose (that is, post-dose 1 [up to Day 8]) and post-dose 2 [up to Day 38])

  2. Number of Participants Reporting Solicited Systemic Events

    All reactogenicity events information recorded via the participant e-diaries or solicited by the investigator were considered as solicited events. Solicited systemic reactions were: fever, chills, fatigue/tiredness, headache, muscle pain/myalgia, joint pain/arthralgia, vomiting, and diarrhea. Any systemic reaction indicated participants with any Grade >=1 reactions. The intensity of systemic events was assessed by the participants and may be confirmed or corrected by the investigator; grades were defined as Grade 1 = Mild; Grade 2 = Moderate; Grade 3 = Severe; Grade 4 = Potentially life-threatening.

    Measured: Up to 7 days post any vaccination, and up to 7 days post each vaccination dose (that is, post Dose 1 [up to Day 8] and post Dose 2 [up to Day 38])

  3. Number of Participants With At Least One Unsolicited Adverse Event (AE)

    An AE was defined as any untoward medical occurrence in a participant administered with a pharmaceutical product, and which did not necessarily have a causal relationship with this treatment. All AE information or safety data including solicited events persisting beyond 7 days that were voluntarily communicated by the participant or collected by the investigator (that is, ECG or laboratory results etc.) were considered unsolicited events. The intensity of AEs was graded by the investigator. Grades were defined as: Grade 1 - Mild; does not interfere with the trial participant's usual function; Grade 2 - Moderate; interferes to some extent with the trial participant's usual function Grade 3 - Severe; interferes significantly with the trial participant's usual function; and Grade 4 - Potentially life-threatening; life-threatening consequences, urgent intervention required.

    Measured: Up to 28 days post any vaccination, and up to 28 days post each vaccination dose (that is, post-dose 1 [up to Day 29] and post-Dose 2 [up to Day 59])

  4. Number of Participants With At Least One Serious Adverse Event (SAE)

    An SAE was defined as any untoward medical occurrence that, at any dose, resulted in death and was life-threatening. It also included any event requiring hospitalization or prolongation of existing hospitalization, resulted in persistent disability/incapacity, caused a congenital anomaly or birth defect, or any other event determined as SAE as per medical or scientific judgment.

    Measured: From Dose 1 up to Day 201

  5. Number of Participants With At Least One Adverse Event of Special Interest (AESI)

    An AESI, serious or non-serious, was one of scientific and medical concern specific to the sponsor's product or program, for which monitoring and rapid communication by the investigator to the sponsor was appropriate.

    Measured: From Dose 1 up to Day 201

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted14 Aug 2023
  2. Study start21 Sep 2023
  3. Primary completion25 Aug 2025
  4. Study completion4 Mar 2026

Resources

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 1/2
Primary purpose
Prevention
Allocation
Non-randomised
Intervention model
Sequential
Masking
None (open label)
Enrolment
96 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05988203
Sponsor's protocol ID
BNT166-01
Other IDs
1006947
Status last verified
Aug 2026
First submitted
4 Aug 2023
Last updated
15 Sep 2026
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Publications

  • Bahner F, Faust SN, Davies LRL, Blokhina O, Brandon DM, Smith WB, Chatterjee VKK, Hassanin H, Babu TM, Zuiani A, Steinhauser S, Poran A, Brittain C, Mucker E, Hooper JW, van der Most RG, Alonso PL, Tureci O, Mensa FJ, Sahin U. Safety and immunogenicity of mRNA-based mpox vaccine candidate BNT166a: an open-label, dose-escalation, first-in-human trial. Lancet Infect Dis. 2026 Jun 3:S1473-3099(26)00177-5. doi: 10.1016/S1473-3099(26)00177-5. Online ahead of print.

    PubMed 42235570
  • Zuiani A, Dulberger CL, De Silva NS, Marquette M, Lu YJ, Palowitch GM, Dokic A, Sanchez-Velazquez R, Schlatterer K, Sarkar S, Kar S, Chawla B, Galeev A, Lindemann C, Rothenberg DA, Diao H, Walls AC, Addona TA, Mensa F, Vogel AB, Stuart LM, van der Most R, Srouji JR, Tureci O, Gaynor RB, Sahin U, Poran A. A multivalent mRNA monkeypox virus vaccine (BNT166) protects mice and macaques from orthopoxvirus disease. Cell. 2024 Mar 14;187(6):1363-1373.e12. doi: 10.1016/j.cell.2024.01.017. Epub 2024 Feb 15.

    PubMed 38366591

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing BNT166a in people with Monkeypox, run by BioNTech SE at 9 sites in 2 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

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: finished, as of the sponsor's last update on 15 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 9 sites in United Kingdom, United States. 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. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 15 Sep 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of Participants Reporting Solicited Local Reactions At the Injection Site (time frame: Up to 7 days post any vaccination, and up to 7 days post each vaccination dose (that is, post-dose 1 [up to Day 8]) and post-dose 2 [up to Day 38])). The study lists 5 primary outcomes in all. 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 NCT05988203, 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 15 Sep 2026.

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