Single or Repeat Dose of G03-52-01 in Adult Subjects

NCT05348993 | Completed Phase 2 Results DMC FDA Drug

• 1 other identifier: G03-52-01-002
• Study Type: interventional
• Target: 622
• Locations: 1 country
• Sites: 15

Brief Summary

A Phase 2, randomized, double-blind, placebo-controlled single or repeat dose trial

Conditions

Healthy; Botulinum Toxin

Outcome Measures

Primary Outcomes (3)

• 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.

  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.

  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.

  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

  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

  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

  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

  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

  Day 1 pre-dose, 24 hours post dose, and on Days 8, 15, 45 (prior to dosing), 49, 90, and 120

Study Arms (5)

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.

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.

Drug: G03-52-01

Cohort 3: Placebo

PLACEBO COMPARATOR

Participants will receive placebo on Days 1 and 45, administered by IM injection.

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.

Drug: G03-52-01

Cohort 4: Placebo

PLACEBO COMPARATOR

Participants will receive placebo on Day 1, administered by IM injection.

Drug: Placebo

Interventions

G03-52-01 DRUG

G03-52-01 administered intramuscularly

Cohort 1: 50 mg G03-52-01; Cohort 2: 100 mg G03-52-01; Cohort 4: 100 mg G03-52-01

Placebo DRUG

placebo

Cohort 3: Placebo; Cohort 4: Placebo

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.
• 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.

You may not qualify if:

• 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.
• 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).
• 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.

Sponsors & Collaborators

• U.S. Army Medical Research and Development Command: lead
• United States Department of Defense: collaborator

Study Sites (15)

AMR Mobile

Mobile, Alabama, 36608, United States

Location

AMR Tempe

Tempe, Arizona, 85281, United States

Location

AMR Fort Myers

Fort Myers, Florida, 33912, United States

Location

AMR Miami

Miami, Florida, 33134, United States

Location

AMR El Dorado

El Dorado, Kansas, 67042, United States

Location

AMR Newton

Newton, Kansas, 67114, United States

Location

AMR Wichita West

Wichita, Kansas, 67205, United States

Location

AMR Wichita East

Wichita, Kansas, 67207, United States

Location

AMR Lexington

Lexington, Kentucky, 40509, United States

Location

AMR New Orleans

New Orleans, Louisiana, 70119, United States

Location

AMR Kansas City

Kansas City, Missouri, 64114, United States

Location

AMR Las Vegas

Las Vegas, Nevada, 89119, United States

Location

AMR Norman

Norman, Oklahoma, 73069, United States

Location

AMR Knoxville West

Knoxville, Tennessee, 37909, United States

Location

AMR Norfolk

Norfolk, Virginia, 23502, United States

Location

Results Point of Contact

• Title: US Army Medical Research and Development Command
• Organization: Office of Regulated Activities (ORA)

usarmy.detrick.medcom-usamrmc.mbx.regulatory-affairs@health.mil

301-619-0317

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: LTE60
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: randomized, double-blind, placebo-controlled
• Purpose: PREVENTION
• Intervention Model: PARALLEL
• Sponsor Type: FED
• Responsible Party: SPONSOR

Model Details: 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

Study Record Dates

• First Submitted: March 31, 2022
• First Posted: April 27, 2022
• Study Start: June 15, 2022
• Primary Completion: February 18, 2025
• Study Completion: February 18, 2025
• Last Updated: February 17, 2026
• Results First Posted: February 17, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will not share

Locations

US (15)