HealthyPhase 2NCT05348993
Single or Repeat Dose of G03-52-01 in Adult Subjects
A Phase 2 study testing G03-52-01 in people with Healthy and Botulinum Toxin, run by U.S. Army Medical Research and Development Command at 15 sites in United States.
Trial summary
A Phase 2, randomized, double-blind, placebo-controlled single or repeat dose trial Read more about this study
- Who is it for?
- Ages 18 to 65 yearsHealthy volunteers can take part
- Conditions the study is for
- Healthy, Botulinum Toxin
- What is being tested?
- G03-52-01
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 622 participants
- Where
- 15 sitesUnited States
- Study dates
- 15 Jun 2022to 18 Feb 2025
- Phase
- Phase 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
Who may be able to join
- Informed consent understood and signed prior to screening procedures.
- Assessed by the Investigator to be a healthy male or healthy, non-pregnant, non-lactating female between the ages of 18 and 65 inclusive on the day of dosing.
- Able and willing to comply and be available for all protocol procedures and follow-up for the duration of the study.
- Body Mass Index (BMI) of ≥18.5 and ≤35 kg/m2.
- Females of child-bearing potential must have a negative serum pregnancy test at screening and negative urine pregnancy test on Day 1 prior to dosing.
- A woman is considered of childbearing potential unless post-menopausal (≥ 1 year without menses) or surgically sterilized via bilateral oophorectomy, or hysterectomy or bilateral tubal ligation.
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- If the subject is female and of childbearing potential, she agrees to practice abstinence from sexual intercourse with men or use medically effective contraception (methods with a failure rate of < 1% per year when used consistently and correctly) during participation in the study. Acceptable methods include:
- Hormonal contraception including implants, injections or oral
- Two barrier methods, e.g., condom and cervical cap (with spermicide) or diaphragm (with spermicide)
- Intrauterine device (IUD) or intrauterine system
- Screening clinical laboratory results within normal ranges or are no greater than a Grade 1 and deemed not clinically significant by Medical Monitor (MM) and Principal Investigator (PI). Any subjects with results that are Grade 2 or above according to Appendix B will be excluded.
- Laboratory values that are outside the range of eligibility but are thought to be due to an acute condition or due to laboratory error may be repeated once.
- The urine drug screen is negative.
- For Cohorts 1-3, if a subject has a positive urine drug screen that the PI believes is caused by a currently prescribed medication, (except for THC), the PI may enroll the subject if they meet all inclusion criteria, and none of the exclusion criteria.
- For Cohort 4, if a subject has a positive urine drug screen that the PI believes is caused by a currently prescribed medication or positive for THC, the PI may enroll the subject if they meet all other inclusion criteria and none of the exclusion criteria.
- Breathalyzer test is negative.
- Available for follow-up for the duration of the study.
- Agrees not to participate in vigorous activity 2 days prior to dosing and 2 days post-dose Day 1 and Day 45 for Cohorts 1-3 and Day 1 for Cohort 4, per Investigator discretion.
Who cannot join
- History of a chronic medical condition that would either interfere with the accurate assessment of the objectives of the study or increase the risk profile of the subject.
- Chronic medical conditions include but are not limited to diabetes; Asthma requiring use of medication in the year before screening; Autoimmune disorder such as lupus, Wegener's, rheumatoid arthritis, thyroid disease; coronary artery disease; chronic hypertension; History of malignancy except low-grade (squamous and basal cell) skin cancer thought to be cured; chronic renal, hepatic, pulmonary, or endocrine disease (except previous asthma which has required no treatment for the past year).
- Known history of severe allergic reaction of any type to medications, bee stings, food, or environmental factors or hypersensitivity or reaction to immunoglobulins.
- Severe allergic reactions are defined as any of the following: anaphylaxis, urticaria, or angioedema.
- Known allergic reactions to any of the study product components present in the formulation or in the processing.
- A marked baseline prolongation of QT/QTc interval (e.g., repeated demonstration of a QTc interval >450 milliseconds).
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- Clinically significant abnormal electrocardiogram (ECG) at screening.
- Clinically significant abnormal ECG results include but are not limited to: complete left or right bundle branch block; other ventricular conduction block except for incomplete RBB; 2nd degree or 3rd degree atrioventricular (AV) block; sustained ventricular arrhythmia; sustained atrial arrhythmia; two Premature Ventricular Contractions in a row; pattern of ST elevation felt consistent with cardiac ischemia; or any condition deemed clinically significant by a study investigator.
- Positive serology results for human immunodeficiency virus (HIV), hepatitis B surface antigen (HBsAg), or hepatitis C virus (HCV) antibodies.
- Febrile illness with temperature ≥38°C within 7 days of dosing. Subjects with acute febrile illness within 7 days of dosing may be rescreened no earlier than 7 days following resolution of symptoms.
- Female subjects that are pregnant or breastfeeding or intending to become pregnant within the projected duration of the trial starting from the Screening visit until last dose.
- Donation of blood or blood product within 56 days of enrollment.
- Is currently participating or has participated in a study with an investigational product (IP) within 28 days preceding Day 1 (documented receipt of placebo in a previous trial would be permissible for trial eligibility)
- Plans to enroll in another clinical trial that could interfere with safety assessment of the IP at any time during the study period.
- Includes trials that have a study intervention such as a drug, biologic, or device only
- Treatment with a mAB within 3 months of Day 1.
- Receipt of antibody (e.g., tetanus immune globulin [TIG], varicella zoster immune globulin [VZIG], intravenous immunoglobulin [IVIG], IM gamma globulin) or blood transfusion within 6 months or within 5 half-lives of the specific product given.
- Reported active drug or alcohol or substance abuse/independence or illicit drug use that, in the opinion of the Investigator, would interfere with adherence to study requirements.
- Use of H1 antihistamines or beta-blockers within 5 days of dosing Day 1 and Day 45 for Cohorts 1-3 and Day 1 for Cohort 4 (PRN use could be allowed with MM approval).
- Use of any prohibited medication within 28 days prior to study entry or planned use during the study period.
- Note: Prohibited medications include immunosuppressives (except nonsteroidal anti-inflammatory drugs [NSAIDs]); immune modulators; oral corticosteroids (topical/intranasal steroids are acceptable); anti-neoplastic agents.
- Previous exposure to botulinum toxin, receipt of antibodies against botulinum toxin, or previous treatment with equine antitoxin.
- Any previous injection or any planned injection within 4 months after enrollment of botulinum toxin for cosmetic reasons, spastic dysphonia, torticollis, or any other reason.
- Any illness or condition that in the judgment of the Investigator may affect the safety of the subject or the evaluation of any study endpoint.
- Is a study site employee, staff, or close relative as defined.
- PIs and Sub-Investigators
- Staff who are supervised by the PI, Sub-Investigators
- Member of the team conducting this clinical trial
- Children, spouse, partners, siblings, and parents of site staff
Trial locations and contacts
15 sites in 1 country.
15 sites in 1 country
United States15 sites
AMR El Dorado
El Dorado, Kansas, 67042
AMR Fort Myers
Fort Myers, Florida, 33912
AMR Kansas City
Kansas City, Missouri, 64114
AMR Knoxville West
Knoxville, Tennessee, 37909
AMR Las Vegas
Las Vegas, Nevada, 89119
AMR Lexington
Lexington, Kentucky, 40509
AMR Miami
Miami, Florida, 33134
AMR Mobile
Mobile, Alabama, 36608
AMR New Orleans
New Orleans, Louisiana, 70119
AMR Newton
Newton, Kansas, 67114
AMR Norfolk
Norfolk, Virginia, 23502
AMR Norman
Norman, Oklahoma, 73069
AMR Tempe
Tempe, Arizona, 85281
AMR Wichita West
Wichita, Kansas, 67205
AMR Wichita East
Wichita, Kansas, 67207
About this study
Official title: A Phase 2, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety, Tolerability, Pharmacodynamics, and Immunogenicity of G03-52-01 in Adult Subjects
A Phase 2, randomized, double-blind, placebo-controlled single or repeat dose trial
Read the detailed description
A Phase 2, multicenter, randomized, double-blinded, placebo-controlled study to evaluate a single (100 mg) or repeat dose (50 mg and 100 mg) of G03-52-01 administered by IM injection(s) in adult subjects. Approximately 625 subjects will be enrolled in this study.
- Conditions
- Healthy, Botulinum Toxin
- Being tested
- G03-52-01, Placebo
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
G03-52-01Drug
G03-52-01 administered intramuscularly
PlaceboDrug
placebo
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1: 50 mg G03-52-01 | Experimental | Participants will receive a 50 mg dose of G03-52-01 on Days 1 and 45, administered by IM injection. Receives: Drug: G03-52-01 |
| Cohort 2: 100 mg G03-52-01 | Experimental | Participants will receive a 100 mg dose of G03-52-01 on Days 1 and 45, administered by IM injection. Receives: Drug: G03-52-01 |
| Cohort 3: Placebo | Placebo comparator | Participants will receive placebo on Days 1 and 45, administered by IM injection. Receives: Drug: Placebo |
| Cohort 4: 100 mg G03-52-01 | Experimental | Participants will receive a 100 mg dose of G03-52-01 on Day 1, administered by IM injection. Receives: Drug: G03-52-01 |
| Cohort 4: Placebo | Placebo comparator | Participants will receive placebo on Day 1, administered by IM injection. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
An AE was any unfavourable and unintended sign, symptom, or disease temporarily associated with the use of investigational product (IP), whether or not related to the IP.
A SAE was any untoward medical occurrence that at any dose: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in a congenital abnormality/birth defect, or caused any persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions.
Clinically significant changes in physical examination, vital signs, and clinical safety laboratory values were included as AEs.
Measured: Cohorts 1-3: 240 days; Cohort 4: 120 days
Cohorts 1-2 Only: Percentage of Participants With Target Protective Concentration Neutralizing Antibody Concentration (NAC) Value > 0.02 U/mL (Botulinum Neurotoxin [BoNT]/A) or > 0.03 U/mL (BoNT/B) at Day 45 and Day 90
Pharmacodynamic (PD) samples were tested using the Battelle Mouse Neutralization Assay (MNA) for serotypes A and B. The MNA was used to determine the concentration of functional antibodies in a sample capable of neutralizing BoNT (i.e., amount of BoNT/A or BoNT/B toxin neutralization afforded by the combination of antibodies). NAC, as measured by the MNA, is proposed to bridge efficacy between animal models and humans.
Measured: Day 45 and Day 90
Cohort 4 Only: Percentage of Participants With Target Protective Concentration NAC Value > 0.02 U/mL (BoNT/A) or > 0.03 U/mL (BoNT/B) at 4 and 8 Hours Post Dose
PD samples were tested using the MNA for serotypes A and B. The MNA was used to determine the concentration of functional antibodies in a sample capable of neutralizing BoNT (i.e., amount of BoNT/A or BoNT/B toxin neutralization afforded by the combination of antibodies). NAC, as measured by the MNA, is proposed to bridge efficacy between animal models and humans.
Measured: 4 and 8 hours post dose on Day 1
Secondary outcomes (19)
Cohorts 1-2 Only: Percentage of Participants With Target Protective Concentration NAC Value > 0.02 U/mL (BoNT/A) or > 0.03 U/mL (BoNT/B) at Day 120
PD samples were tested using the MNA for serotypes A and B. The MNA was used to determine the concentration of functional antibodies in a sample capable of neutralizing BoNT (i.e., amount of BoNT/A or BoNT/B toxin neutralization afforded by the combination of antibodies). NAC, as measured by the MNA, is proposed to bridge efficacy between animal models and humans.
Measured: Day 120
Cohorts 1-2 Only: Area Under the Concentration-time Curve to the Last Concentration Above the Lower Limit of Quantitation (AUC(0-t)) for Serotypes BoNT/A and BoNT/B
PD samples were tested by MNA for serotypes BoNT/A and BoNT/B. The MNA was used to determine the concentration of functional antibodies in a sample capable of neutralizing BoNT (i.e., amount of BoNT/A or BoNT/B toxin neutralization afforded by the combination of antibodies). NAC, as measured by the MNA, is proposed to bridge efficacy between animal models and humans.
If pre-dose concentration was inconclusive, not tested, or missing, or if there were fewer than two measurable concentrations in the participant's profile, the parameters were not determined and the participant was excluded from MNA analysis.
In the calculation of PD parameters, below limit of quantitation (BLQ) values were treated as zero prior to the first measurable concentration. After the first measurable concentration, subsequent BLQ values were treated as missing. Values that were above the upper limit of quantitation were set to the ULQ (upper limit of quantitation) of the assay for analysis.
Measured: Day 1 pre-dose, 24 hours post dose, and on Days 8, 15, 45 (prior to dosing), 49, 90, and 120
Cohorts 1-2 Only: Terminal Half-life (t1/2) for Serotypes BoNT/A and BoNT/B
PD samples were tested by MNA for serotypes BoNT/A and BoNT/B. The MNA was used to determine the concentration of functional antibodies in a sample capable of neutralizing BoNT (i.e., amount of BoNT/A or BoNT/B toxin neutralization afforded by the combination of antibodies). NAC, as measured by the MNA, is proposed to bridge efficacy between animal models and humans.
t1/2 was determined after the first dose on Day 1. This value was calculated as t1/2 = ln(2)/ λz where λz is the elimination rate constant.
Only samples up to Day 45 predose were used in these calculations, however the above equation allows a half life greater than the sampling interval.
Measured: Day 1 pre-dose, 24 hours post dose, and on Days 8, 15 and 45 (prior to dosing)
Cohorts 1-2 Only: Maximum Observed Concentration (Cmax) for Serotypes BoNT/A and BoNT/B
PD samples were tested by MNA for serotypes BoNT/A and BoNT/B. The MNA was used to determine the concentration of functional antibodies in a sample capable of neutralizing BoNT (i.e., amount of BoNT/A or BoNT/B toxin neutralization afforded by the combination of antibodies). NAC, as measured by the MNA, is proposed to bridge efficacy between animal models and humans.
If pre-dose concentration was inconclusive, not tested, or missing, or if there were fewer than two measurable concentrations in the participant's profile, the parameters were not determined and the participant was excluded from MNA analysis.
In the calculation of PD parameters, BLQ values were treated as zero prior to the first measurable concentration. After the first measurable concentration, subsequent BLQ values were treated as missing. Values that were above the upper limit of quantitation were set to the upper limit of quantitation of the assay for analysis.
Measured: Day 1 pre-dose, 24 hours post dose, and on Days 8, 15, 45 (prior to dosing), 49, 90, and 120
Cohorts 1-2 Only: Time of Maximum Observed Concentration (Tmax) for Serotypes BoNT/A and BoNT/B
PD samples were tested by MNA for serotypes BoNT/A and BoNT/B. The MNA was used to determine the concentration of functional antibodies in a sample capable of neutralizing BoNT (i.e., amount of BoNT/A or BoNT/B toxin neutralization afforded by the combination of antibodies). NAC, as measured by the MNA, is proposed to bridge efficacy between animal models and humans.
If pre-dose concentration was inconclusive, not tested, or missing, or if there were fewer than two measurable concentrations in the participant's profile, the parameters were not determined and the participant was excluded from MNA analysis.
In the calculation of PD parameters, BLQ values were treated as zero prior to the first measurable concentration. After the first measurable concentration, subsequent BLQ values were treated as missing. Values that were above the upper limit of quantitation were set to the upper limit of quantitation of the assay for analysis.
Measured: Day 1 pre-dose, 24 hours post dose, and on Days 8, 15, 45 (prior to dosing), 49, 90, and 120
Cohorts 1-2 Only: Number of Participants With Anti-drug Antibodies (ADA) to Each Component of the G03-52-01 Drug Product (DP) at Each Timepoint Tested
The presence of ADA was determined using a validated electrochemiluminescence assay (ECLA) that measured total ADA in serum.
Detected and Not Detected results correspond to samples that were potential positive during screening testing. No Recorded Result (NRR) results correspond to samples that were NRR during screening testing. Percentages are based on n, the number of participants in each treatment group with a non-missing result at each time point for the given analyte.
Measured: Day 1 pre-dose and Days 15, 45 (prior to dosing), 60, 90, 120, 150, 180, and 240
Cohort 4 Only: Percentage of Participants With Target Protective NAC Value > 0.02 U/mL (BoNT/A) or > 0.03 U/mL (BoNT/B) at 2 Hours Post Dose
PD samples were tested using the MNA for serotypes A and B. The MNA was used to determine the concentration of functional antibodies in a sample capable of neutralizing BoNT (i.e., amount of BoNT/A or BoNT/B toxin neutralization afforded by the combination of antibodies). NAC, as measured by the MNA, is proposed to bridge efficacy between animal models and humans.
Measured: 2 hours post dose on Day 1
Cohort 4 Only: Area Under the Concentration-time Curve Extrapolated to Infinity (AUC(0-inf)) of Each Component of the G03-52-01 DP
PK parameters were measured by enzyme-linked immunosorbent assay (ELISA) or ECLA for each of the monoclonal antibodies (mAb) of G03-52-01, and PK parameters were estimated for each of the six mAbs separately using noncompartmental methods in WinNonlin or a similar software package.
Measured: Day 1 pre-dose, 2 hours, 4 hours, 8 hours, 24 hours, 72 hours post dose, and on Days 8, 15, 30, 45, 90, and 120
Cohort 4 Only: AUC(0-t) of Each Component of the G03-52-01 DP
PK parameters were measured by ELISA or ECLA for each of the mAb of G03-52-01, and PK parameters were estimated for each of the six mAbs separately using noncompartmental methods in WinNonlin or a similar software package.
Measured: Day 1 pre-dose, 2 hours, 4 hours, 8 hours, 24 hours, 72 hours post dose, and on Days 8, 15, 30, 45, 90, and 120
Cohort 4 Only: Total Body Clearance (CL/F) of Each Component of the G03-52-01 DP
PK parameters were measured by ELISA or ECLA for each of the monoclonal antibodies (mAb) of G03-52-01, and PK parameters were estimated for each of the six mAbs separately using noncompartmental methods in WinNonlin or a similar software package.
Measured: Day 1 pre-dose, 2 hours, 4 hours, 8 hours, 24 hours, 72 hours post dose, and on Days 8, 15, 30, 45, 90, and 120
Cohort 4 Only: Cmax of Each Component of the G03-52-01 DP
PK parameters were measured by ELISA or ECLA for each of the mAb of G03-52-01, and PK parameters were estimated for each of the six mAbs separately using noncompartmental methods in WinNonlin or a similar software package.
Measured: Day 1 pre-dose, 2 hours, 4 hours, 8 hours, 24 hours, 72 hours post dose, and on Days 8, 15, 30, 45, 90, and 120
Cohort 4 Only: t1/2 of Each Component of the G03-52-01 DP
PK parameters were measured by ELISA or ECLA for each of the monoclonal antibodies (mAb) of G03-52-01, and PK parameters were estimated for each of the six mAbs separately using noncompartmental methods in WinNonlin or a similar software package.
Measured: Day 1 pre-dose, 2 hours, 4 hours, 8 hours, 24 hours, 72 hours post dose, and on Days 8, 15, 30, 45, 90, and 120
Cohort 4 Only: Tmax of Each Component of the G03-52-01 DP
PK parameters were measured by ELISA or ECLA for each of the mAb of G03-52-01, and PK parameters were estimated for each of the six mAbs separately using noncompartmental methods in WinNonlin or a similar software package.
Measured: Day 1 pre-dose, 2 hours, 4 hours, 8 hours, 24 hours, 72 hours post dose, and on Days 8, 15, 30, 45, 90, and 120
Cohort 4 Only: Volume of Distribution (Vz/F) of Each Component of the G03-52-01 DP
PK parameters were measured by ELISA or ECLA for each of the mAb of G03-52-01, and PK parameters were estimated for each of the six mAbs separately using noncompartmental methods in WinNonlin or a similar software package.
Measured: Day 1 pre-dose, 2 hours, 4 hours, 8 hours, 24 hours, 72 hours post dose, and on Days 8, 15, 30, 45, 90, and 120
Cohort 4 Only: AUC(0-t) for Serotypes BoNT/A and BoNT/B
PD samples were tested by MNA for serotypes BoNT/A and BoNT/B. The MNA was used to determine the concentration of functional antibodies in a sample capable of neutralizing BoNT (i.e., amount of BoNT/A or BoNT/B toxin neutralization afforded by the combination of antibodies). NAC, as measured by the MNA, is proposed to bridge efficacy between animal models and humans.
If pre-dose concentration was inconclusive, not tested, or missing, or if there were fewer than two measurable concentrations in the participant's profile, the parameters were not determined and the participant was excluded from MNA analysis.
In the calculation of PD parameters, BLQ values were treated as zero prior to the first measurable concentration. After the first measurable concentration, subsequent BLQ values were treated as missing. Values that were above the upper limit of quantitation were set to the upper limit of quantitation of the assay for analysis.
Measured: Day 1 pre-dose, 2, 4, 8 and 24 hours post-dose, and on Days 30, 45, and 90
Cohort 4 Only: t1/2 for Serotypes BoNT/A and BoNT/B
PD samples were tested by MNA for serotypes BoNT/A and BoNT/B. The MNA was used to determine the concentration of functional antibodies in a sample capable of neutralizing BoNT (i.e., amount of BoNT/A or BoNT/B toxin neutralization afforded by the combination of antibodies). NAC, as measured by the MNA, is proposed to bridge efficacy between animal models and humans.
t1/2 was determined after the first dose on Day 1. This value was calculated as t1/2 = ln(2)/ λz where λz is the elimination rate constant.
Only samples up to Day 45 were used in these calculations, however the above equation allows a half life greater than the sampling interval.
Measured: Day 1 pre-dose, 2, 4, 8 and 24 hours post-dose, and on Days 30 and 45
Cohort 4 Only: Cmax for Serotypes BoNT/A and BoNT/B
PD samples were tested by MNA for serotypes BoNT/A and BoNT/B. The MNA was used to determine the concentration of functional antibodies in a sample capable of neutralizing BoNT (i.e., amount of BoNT/A or BoNT/B toxin neutralization afforded by the combination of antibodies). NAC, as measured by the MNA, is proposed to bridge efficacy between animal models and humans.
If pre-dose concentration was inconclusive, not tested, or missing, or if there were fewer than two measurable concentrations in the participant's profile, the parameters were not determined and the participant was excluded from MNA analysis.
In the calculation of PD parameters, BLQ values were treated as zero prior to the first measurable concentration. After the first measurable concentration, subsequent BLQ values were treated as missing. Values that were above the upper limit of quantitation were set to the upper limit of quantitation of the assay for analysis.
Measured: Day 1 pre-dose, 2, 4, 8 and 24 hours post-dose, and on Days 30, 45, and 90
Cohort 4 Only: Tmax for Serotypes BoNT/A and BoNT/B
PD samples were tested by MNA for serotypes BoNT/A and BoNT/B. The MNA was used to determine the concentration of functional antibodies in a sample capable of neutralizing BoNT (i.e., amount of BoNT/A or BoNT/B toxin neutralization afforded by the combination of antibodies). NAC, as measured by the MNA, is proposed to bridge efficacy between animal models and humans.
If pre-dose concentration was inconclusive, not tested, or missing, or if there were fewer than two measurable concentrations in the participant's profile, the parameters were not determined and the participant was excluded from MNA analysis.
In the calculation of PD parameters, BLQ values were treated as zero prior to the first measurable concentration. After the first measurable concentration, subsequent BLQ values were treated as missing. Values that were above the upper limit of quantitation were set to the upper limit of quantitation of the assay for analysis.
Measured: Day 1 pre-dose, 2, 4, 8 and 24 hours post-dose, and on Days 30, 45, and 90
Cohort 4 Only: Number of Participants With ADA to Each Component of the G03-52-01 DP at Each Timepoint Tested
The presence of ADA was determined using a validated ECLA that measured total ADA in serum.
Detected and Not Detected results correspond to samples that were potential positive during screening testing. NRR results correspond to samples that were NRR during screening testing. Percentages are based on n, the number of participants in each treatment group with a non-missing result at each time point for the given analyte.
Measured: Day 1 pre-dose and Days 90 and 120
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Apr 2022
- Study start15 Jun 2022
- Primary completion18 Feb 2025
- Study completion18 Feb 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for HealthyStudies of the same condition, open and under way.
- 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 2
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Single dose of G03-52-01 100 mg or placebo or a repeat dose of G03-52-01 (50mg or 100mg) or placebo on days 1 and 45
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 622 participants (actual)
Sponsor and investigators
- Lead sponsor
- U.S. Army Medical Research and Development Command
- Sponsor type
- US federal agency
- Collaborators
- United States Department of Defense
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05348993
- Sponsor's protocol ID
- G03-52-01-002
- Status last verified
- Jan 2026
- First submitted
- 31 Mar 2022
- Last updated
- 17 Feb 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 17 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety, Tolerability, Pharmacodynamics, and Immunogenicity of G03-52-01 in Adult Subjects
- ClinicalTrials.gov ID
- NCT05348993
- Acronym / sponsor ID
- G03-52-01-002
- Conditions
- Healthy, Botulinum Toxin
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 622 participants (actual)
- Lead sponsor
- U.S. Army Medical Research and Development CommandUS federal agency
- Collaborators
- United States Department of Defense
- Study start
- 15 Jun 2022 (actual)
- Primary completion
- 18 Feb 2025 (actual)
- Study completion
- 18 Feb 2025 (actual)
- First posted
- 27 Apr 2022 (actual)
- Results
- Posted 17 Feb 2026
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
A Phase 2, randomized, double-blind, placebo-controlled single or repeat dose trial
Detailed description
A Phase 2, multicenter, randomized, double-blinded, placebo-controlled study to evaluate a single (100 mg) or repeat dose (50 mg and 100 mg) of G03-52-01 administered by IM injection(s) in adult subjects. Approximately 625 subjects will be enrolled in this study.
Study design
- Primary purpose
- Prevention
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 5
- Model description
- Single dose of G03-52-01 100 mg or placebo or a repeat dose of G03-52-01 (50mg or 100mg) or placebo on days 1 and 45
| Arm | Type | Receives |
|---|---|---|
| Cohort 1: 50 mg G03-52-01 | Experimental | Drug: G03-52-01Participants will receive a 50 mg dose of G03-52-01 on Days 1 and 45, administered by IM injection. |
| Cohort 2: 100 mg G03-52-01 | Experimental | Drug: G03-52-01Participants will receive a 100 mg dose of G03-52-01 on Days 1 and 45, administered by IM injection. |
| Cohort 3: Placebo | Placebo comparator | Drug: PlaceboParticipants will receive placebo on Days 1 and 45, administered by IM injection. |
| Cohort 4: 100 mg G03-52-01 | Experimental | Drug: G03-52-01Participants will receive a 100 mg dose of G03-52-01 on Day 1, administered by IM injection. |
| Cohort 4: Placebo | Placebo comparator | Drug: PlaceboParticipants will receive placebo on Day 1, administered by IM injection. |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (18 listed)
- Informed consent understood and signed prior to screening procedures.
- Assessed by the Investigator to be a healthy male or healthy, non-pregnant, non-lactating female between the ages of 18 and 65 inclusive on the day of dosing.
- Able and willing to comply and be available for all protocol procedures and follow-up for the duration of the study.
- Body Mass Index (BMI) of ≥18.5 and ≤35 kg/m2.
- Females of child-bearing potential must have a negative serum pregnancy test at screening and negative urine pregnancy test on Day 1 prior to dosing.
Key exclusion criteria (29 listed)
- History of a chronic medical condition that would either interfere with the accurate assessment of the objectives of the study or increase the risk profile of the subject.
- Known history of severe allergic reaction of any type to medications, bee stings, food, or environmental factors or hypersensitivity or reaction to immunoglobulins.
- Known allergic reactions to any of the study product components present in the formulation or in the processing.
- A marked baseline prolongation of QT/QTc interval (e.g., repeated demonstration of a QTc interval >450 milliseconds).
- Clinically significant abnormal electrocardiogram (ECG) at screening.
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 |
|---|---|---|
| G03-52-01G03-52-01 administered intramuscularly | Drug | Cohort 1: 50 mg G03-52-01, Cohort 2: 100 mg G03-52-01, Cohort 4: 100 mg G03-52-01 |
| Placeboplacebo | Drug | Cohort 3: Placebo, Cohort 4: Placebo |
Primary outcome
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time frame: Cohorts 1-3: 240 days; Cohort 4: 120 days
An AE was any unfavourable and unintended sign, symptom, or disease temporarily associated with the use of investigational product (IP), whether or not related to the IP. A SAE was any untoward medical occurrence that at any dose: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in a congenital abnormality/birth defect, or caused any persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions. Clinically significant changes in physical examination, vital signs, and clinical safety laboratory values were included as AEs.
Cohorts 1-2 Only: Percentage of Participants With Target Protective Concentration Neutralizing Antibody Concentration (NAC) Value > 0.02 U/mL (Botulinum Neurotoxin [BoNT]/A) or > 0.03 U/mL (BoNT/B) at Day 45 and Day 90
Time frame: Day 45 and Day 90
Pharmacodynamic (PD) samples were tested using the Battelle Mouse Neutralization Assay (MNA) for serotypes A and B. The MNA was used to determine the concentration of functional antibodies in a sample capable of neutralizing BoNT (i.e., amount of BoNT/A or BoNT/B toxin neutralization afforded by the combination of antibodies). NAC, as measured by the MNA, is proposed to bridge efficacy between animal models and humans.
Cohort 4 Only: Percentage of Participants With Target Protective Concentration NAC Value > 0.02 U/mL (BoNT/A) or > 0.03 U/mL (BoNT/B) at 4 and 8 Hours Post Dose
Time frame: 4 and 8 hours post dose on Day 1
PD samples were tested using the MNA for serotypes A and B. The MNA was used to determine the concentration of functional antibodies in a sample capable of neutralizing BoNT (i.e., amount of BoNT/A or BoNT/B toxin neutralization afforded by the combination of antibodies). NAC, as measured by the MNA, is proposed to bridge efficacy between animal models and humans.
19 secondary outcomes
Cohorts 1-2 Only: Percentage of Participants With Target Protective Concentration NAC Value > 0.02 U/mL (BoNT/A) or > 0.03 U/mL (BoNT/B) at Day 120
Time frame: Day 120
Cohorts 1-2 Only: Area Under the Concentration-time Curve to the Last Concentration Above the Lower Limit of Quantitation (AUC(0-t)) for Serotypes BoNT/A and BoNT/B
Time frame: Day 1 pre-dose, 24 hours post dose, and on Days 8, 15, 45 (prior to dosing), 49, 90, and 120
Cohorts 1-2 Only: Terminal Half-life (t1/2) for Serotypes BoNT/A and BoNT/B
Time frame: Day 1 pre-dose, 24 hours post dose, and on Days 8, 15 and 45 (prior to dosing)
Cohorts 1-2 Only: Maximum Observed Concentration (Cmax) for Serotypes BoNT/A and BoNT/B
Time frame: Day 1 pre-dose, 24 hours post dose, and on Days 8, 15, 45 (prior to dosing), 49, 90, and 120
Cohorts 1-2 Only: Time of Maximum Observed Concentration (Tmax) for Serotypes BoNT/A and BoNT/B
Time frame: Day 1 pre-dose, 24 hours post dose, and on Days 8, 15, 45 (prior to dosing), 49, 90, and 120
Cohorts 1-2 Only: Number of Participants With Anti-drug Antibodies (ADA) to Each Component of the G03-52-01 Drug Product (DP) at Each Timepoint Tested
Time frame: Day 1 pre-dose and Days 15, 45 (prior to dosing), 60, 90, 120, 150, 180, and 240
Cohort 4 Only: Percentage of Participants With Target Protective NAC Value > 0.02 U/mL (BoNT/A) or > 0.03 U/mL (BoNT/B) at 2 Hours Post Dose
Time frame: 2 hours post dose on Day 1
Cohort 4 Only: Area Under the Concentration-time Curve Extrapolated to Infinity (AUC(0-inf)) of Each Component of the G03-52-01 DP
Time frame: Day 1 pre-dose, 2 hours, 4 hours, 8 hours, 24 hours, 72 hours post dose, and on Days 8, 15, 30, 45, 90, and 120
Cohort 4 Only: AUC(0-t) of Each Component of the G03-52-01 DP
Time frame: Day 1 pre-dose, 2 hours, 4 hours, 8 hours, 24 hours, 72 hours post dose, and on Days 8, 15, 30, 45, 90, and 120
Cohort 4 Only: Total Body Clearance (CL/F) of Each Component of the G03-52-01 DP
Time frame: Day 1 pre-dose, 2 hours, 4 hours, 8 hours, 24 hours, 72 hours post dose, and on Days 8, 15, 30, 45, 90, and 120
Cohort 4 Only: Cmax of Each Component of the G03-52-01 DP
Time frame: Day 1 pre-dose, 2 hours, 4 hours, 8 hours, 24 hours, 72 hours post dose, and on Days 8, 15, 30, 45, 90, and 120
Cohort 4 Only: t1/2 of Each Component of the G03-52-01 DP
Time frame: Day 1 pre-dose, 2 hours, 4 hours, 8 hours, 24 hours, 72 hours post dose, and on Days 8, 15, 30, 45, 90, and 120
Cohort 4 Only: Tmax of Each Component of the G03-52-01 DP
Time frame: Day 1 pre-dose, 2 hours, 4 hours, 8 hours, 24 hours, 72 hours post dose, and on Days 8, 15, 30, 45, 90, and 120
Cohort 4 Only: Volume of Distribution (Vz/F) of Each Component of the G03-52-01 DP
Time frame: Day 1 pre-dose, 2 hours, 4 hours, 8 hours, 24 hours, 72 hours post dose, and on Days 8, 15, 30, 45, 90, and 120
Cohort 4 Only: AUC(0-t) for Serotypes BoNT/A and BoNT/B
Time frame: Day 1 pre-dose, 2, 4, 8 and 24 hours post-dose, and on Days 30, 45, and 90
Cohort 4 Only: t1/2 for Serotypes BoNT/A and BoNT/B
Time frame: Day 1 pre-dose, 2, 4, 8 and 24 hours post-dose, and on Days 30 and 45
Cohort 4 Only: Cmax for Serotypes BoNT/A and BoNT/B
Time frame: Day 1 pre-dose, 2, 4, 8 and 24 hours post-dose, and on Days 30, 45, and 90
Cohort 4 Only: Tmax for Serotypes BoNT/A and BoNT/B
Time frame: Day 1 pre-dose, 2, 4, 8 and 24 hours post-dose, and on Days 30, 45, and 90
Cohort 4 Only: Number of Participants With ADA to Each Component of the G03-52-01 DP at Each Timepoint Tested
Time frame: Day 1 pre-dose and Days 90 and 120
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Feb 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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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. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
15 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing G03-52-01 in people with Healthy and Botulinum Toxin, run by U.S. Army Medical Research and Development Command at 15 sites in United States.
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 18 inclusion and 29 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: finished, as of the sponsor's last update on 17 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 15 sites in 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. 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 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 17 Feb 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 With Adverse Events (AEs) and Serious Adverse Events (SAEs) (time frame: Cohorts 1-3: 240 days; Cohort 4: 120 days). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 17 Feb 2026.