Study to Evaluate Safety and PK of a Single IM Dose of G03-52-01 vs Placebo in Adult Subjects

NCT04171115 | Completed Phase 1 Results FDA Drug

• 1 other identifier: G03-52-01.001
• Study Type: interventional
• Target: 40
• Locations: 1 country
• Sites: 1

Brief Summary

A Phase 1, randomized, double-blind, placebo-controlled dose escalation trial of four dose cohorts of 10 subjects (1: 10mg, 2: 25mg, 3: 50mg, 4: 100mg).

Conditions

Healthy

Keywords

botulinum toxin; botulism

Outcome Measures

Primary Outcomes (6)

• The Occurrence of Serious Adverse Events (SAE) Following Administration of G03-52-01 to the Final Follow-up Visit.

  Determine number of SAEs after dosing (Cohorts 1-3)

  day 0 to day 120

• The Occurrence of Serious Adverse Events (SAE) Following Administration of G03-52-01 to the Final Follow-up Visit.

  Determine number of SAEs after dosing (Cohort 4)

  day 0 to day 180

• The Occurrence of Adverse Events (AE) Following Administration of G03-52-01 to the Final Follow-up Visit

  Determine number of AEs after dosing (Cohorts 1-3)

  day 0 to day 120

• The Occurrence of Adverse Events (AE) Following Administration of G03-52-01 to the Final Follow-up Visit

  Determine number of AEs after dosing (Cohort 4)

  day 0 to day 180

• The Occurrence of Changes From Baseline in Physical Examination, Vital Signs and Clinical Safety Laboratory Values Following Administration of G03-52-01 to the Final Follow-up Visit.

  Determine number of changes from baseline (Cohorts 1-3)

  day 0 to day 120

• The Occurrence of Changes From Baseline in Physical Examination, Vital Signs and Clinical Safety Laboratory Values Following Administration of G03-52-01 to the Final Follow-up Visit.

  Determine number of changes from baseline (Cohort 4)

  day 0 to day 180

Secondary Outcomes (26)

• Serotype A Peak Plasma Concentration (Cmax)

  pre-dose, days 4, 30, 60, 90, and 120

• Serotype A Peak Plasma Concentration (Cmax)

  pre-dose, 2 hours post-dose, days 4, 30, 45, 60, 90, and 120

• Serotype B Peak Plasma Concentration (Cmax)

  pre-dose, days 4, 30, 60, 90, and 120

• Serotype B Peak Plasma Concentration (Cmax)

  pre-dose, 2 hours post-dose, days 4, 30, 45, 60, 90, and 120

• Serotype A Time to Achieve Peak Concentration of the Drug After Administration (Tmax)

  pre-dose, days 4, 30, 60, 90, and 120

Study Arms (4)

10mg of G03-52-01

EXPERIMENTAL

8 subjects randomized to 10 mg of G03-52-01 and 2 subjects randomized to placebo

Drug: G03-52-01; Drug: Placebo

25mg of G03-52-01

EXPERIMENTAL

8 subjects randomized to 25 mg of G03-52-01 and 2 subjects randomized to placebo

Drug: G03-52-01; Drug: Placebo

50 mg of G03-52-01

EXPERIMENTAL

8 subjects randomized to 50 mg of G03-52-01 and 2 subjects randomized to placebo

Drug: G03-52-01; Drug: Placebo

100 mg of G03-52-01

EXPERIMENTAL

8 subjects randomized to 100 mg of G03-52-01 and 2 subjects randomized to placebo

Drug: G03-52-01; Drug: Placebo

Interventions

G03-52-01 DRUG

G03-52-01 administered intramuscularly

100 mg of G03-52-01; 10mg of G03-52-01; 25mg of G03-52-01; 50 mg of G03-52-01

Placebo DRUG

Placebo administered intramuscularly

100 mg of G03-52-01; 10mg of G03-52-01; 25mg of G03-52-01; 50 mg of G03-52-01

Eligibility Criteria

• Age: 18 Years - 45 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64)

You may qualify if:

• Informed consent understood and signed
• Healthy male or healthy, non-pregnant, non-lactating female
• Willingness to comply and be available for all protocol procedures
• Between 18 and 45 years of age on the day of IM injection
• Body Mass Index (BMI) of ≥18.5 and ≤35 kg/m2
• If the subject is female and of childbearing potential, she has a negative serum pregnancy test at screening and negative urine test within 24 hours prior to IM injection
• Note: 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 or successful Essure placement with documented confirmation test at least 3 months after the procedure.
• If the subject is female and of childbearing potential, she agrees to practice abstinence from sexual intercourse with men or use acceptable contraception during participation in the study
• Note: Acceptable contraception methods are restricted to effective devices (Intrauterine Contraceptive Devices)
• The hemoglobin, platelet count, white blood cell count and absolute neutrophil count are not below the LLN and ≤ULN x 10%
• The urine dipstick results on protein, glucose and blood are negative or trace
• Note: When a urine dipstick is more than trace positive for blood (whether a menstruating female or other subjects), that subject would not be excluded if the urine microscopic exam shows \<5 rbcs/hpf.
• Chemistry screening laboratory tests as outlined in Section 7.5.1.4 are in the normal reference range
• Note: The following exceptions to laboratory normal reference ranges are allowed: Creatinine, Blood Urea Nitrogen (BUN), total bilirubin, AST, ALT, lipase, amylase, Prothrombin Time (PT), Partial Thromboplastin Time (PTT) below the lower limit of normal (LLN); CK less than 400U/L; Glucose, potassium, total protein, and alkaline phosphatase with a toxicity grade of 1 is allowable; albumin above the upper limit of normal (ULN).
• 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.

You may not qualify if:

• 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.
• Note: Chronic medical conditions include but 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);
• History of severe allergic reaction of any type to medications, bee stings, food, or environmental factors or hypersensitivity or reaction to immunoglobulins.
• Note: Severe allergic reaction is defined as any of the following: anaphylaxis, urticaria, or angioedema
• A marked baseline prolongation of QT/QTc interval (e.g., repeated demonstration of a QTc interval \>450 milliseconds)
• Clinically significant abnormal electrocardiogram at screening.
• Note: Clinically significant abnormal ECG results include but not limited to: complete left or right bundle branch block; other ventricular conduction block; 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 HIV, HBsAg, or HCV antibodies
• Febrile illness with temperature ≥38°C within 7 days of dosing
• Pregnant or breastfeeding
• Donated blood within 56 days of enrollment
• Known allergic reactions to any of the study product components present in the formulation or in the processing, as listed in the Investigator Brochure
• Treatment with another investigational drug within 28 days of dosing
• Treatment with a monoclonal antibody within 3 months of enrollment.
• Receipt of antibody (e.g. TIG, VZIG, IVIG, IM gamma globulin) or blood transfusion within 6 months or within 5 half-lives of the specific product given

Sponsors & Collaborators

• Alachua Government Services, Inc.: lead
• United States Department of Defense: collaborator

Study Sites (1)

ICON Early Phase Services

San Antonio, Texas, 78209, United States

Location

MeSH Terms

Conditions

Botulism

Condition Hierarchy (Ancestors)

Clostridium Infections; Gram-Positive Bacterial Infections; Bacterial Infections; Bacterial Infections and Mycoses; Infections; Neuromuscular Junction Diseases; Neuromuscular Diseases; Nervous System Diseases; Neurotoxicity Syndromes; Foodborne Diseases; Poisoning; Chemically-Induced Disorders

Results Point of Contact

• Title: Angie Kimbler
• Organization: Resilience Government Services

angie.kimbler@resilience.com

386-418-8751

Study Officials

• Cassandra Key, MD

  ICON Early Phase Services, LLC

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: randomized, double-blind, placebo-controlled
• Purpose: PREVENTION
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: dose escalation trial of four dose cohorts of 10 subjects (A: 10mg, B: 25mg, C: 50mg, D: 100 mg)

Study Record Dates

• First Submitted: November 15, 2019
• First Posted: November 20, 2019
• Study Start: June 1, 2020
• Primary Completion: August 20, 2022
• Study Completion: April 5, 2023
• Last Updated: October 4, 2024
• Results First Posted: October 4, 2024

Record last verified: 2024-06

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)