Safety and Efficacy Study of GB001 Recombinant Peptide Spray in Subjects With Mild Recurrent Aphthous Ulcers

A Multicenter, Randomized, Double-blind, Placebo-controlled Phase IIa Clinical Trial to Evaluate the Efficacy and Safety of Oral Local Use of GB001 Recombinant Peptide Spray for the Treatment of Mild Recurrent Aphthous Ulcer

This trial is conducted in China. The purpose is to evaluate the efficacy, Pharmacokinetics (PK) profile, immunogenicity and safety of GB001 recombinant peptide spray in adults with mild recurrent aphthous ulcer.

Study Overview

• Status: Active, not recruiting
• Conditions: Healthy
• Intervention / Treatment: Drug: GB001 recombinant peptide spray low dose group、high dose group; Drug: GB001 recombinant peptide spray placebo

• Study Type: Interventional
• Enrollment (Actual): 120
• Phase: Phase 2

Contacts and Locations

Study Locations

• China
  • Hunan
    • Changsha, Hunan, China, 410000
      • The First Hospital of Hunan University of Chinese Medicine

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. It meets the diagnostic criteria for mild recurrent Aphthous ulcer in the fifth edition of Oral Mucosa published by People's Medical Publishing House in 2020.
2. 18≤ age ≤65 years old, gender is not limited.
3. Patients with untreated target ulcer onset ≤48 hours at the time of screening.
4. VAS score of target ulcer irritation pain ≥3 points, 2mm≤ length diameter of target ulcer ≤10mm.
5. Subjects and their sexual partners agree to use effective contraception during the study period and for at least 30 days after the study ends.
6. Sign a written informed consent, and be able to comply with the visit and related procedures stipulated in the program.

Exclusion criteria:

1. The diagnosis was severe aphthous ulcer, herpetic aphthous ulcer, acute herpetic gingivitis stomatitis, traumatic ulcer, cancerous ulcer, tuberculous ulcer, syphilitic ulcer, necrotic salivate metaplasia, Behcet disease and other diseases or drug induced ulcers.
2. Patients with suppurative tonsillitis or other painful lesions in the mouth, such as pericoronitis, pulpitis, periapical inflammation, etc., affecting the pain score of target ulcer.
3. Target ulcer is affected by residual root, residual crown, denture, prosthesis, orthodontic device and other stimulating factors in the corresponding parts of the target ulcer.
4. The ulcer is located in the lingual frenulum, the back wall of the pharynx and other parts, and its size is not easy to measure.
5. Those who plan to perform other oral treatments during the trial that affect the determination of drug effectiveness and safety.
6. Smokers > 20 cigarettes/day or betel nut lovers in the past 3 months.
7. People who have used painkillers or drugs that may affect the efficacy of pain observation within 24 hours before the first administration, such as sedatives, anti-allergy drugs, non-steroidal anti-inflammatory drugs, etc.
8. Patients who have used antibiotics or antiviral drugs locally or systematically within 1 week before screening.
9. Patients who had used immunosuppressive agents locally within 1 week before screening or systemic immunosuppressive agents within 2 weeks before screening.
10. Patients with oral local or systemic use of glucocorticosteroids within 4 weeks prior to screening.
11. Patients who have taken anticholinergic drugs to reduce salivary secretion within 2 weeks prior to screening.
12. Complicated with severe liver and kidney diseases, or abnormal liver and kidney function tests (ALT and AST≥ 1.5 times the upper limit of normal, SCr > the upper limit of normal).
13. Patients with severe anemia (Hb < 60g/L).
14. Complicated with severe heart and lung disease, uncontrolled diabetes (fasting blood glucose > 7.0mmol/L or random blood glucose ≥11.1mmol/L), advanced tumors, diseases of the blood and hematopoietic system, or other serious or progressive diseases of the system.
15. Known or suspected allergic history or serious adverse reactions to the experimental drug and its excipients.
16. Pregnant or lactating women and those with recent pregnancy plans.
17. Participants who had participated in other interventional clinical trials within 3 months prior to screening.
18. Other conditions deemed inappropriate by the investigator for participation in the study.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: GB001 recombinant peptide spray low dose group; Each subject will take oral GB001 recombinant peptide spray 4 times a day, 4 sprays each time, no more than 29 times，The dosage of the group is 0.108mg/spray.	Drug: GB001 recombinant peptide spray low dose group、high dose group; Administrated oral spray. Each subject will take oral GB001 recombinant peptide spray 4 times a day, 4 sprays each time, no more than 29 times，The dosage of the group is 0.108mg/spray for low dose group and 0.216mg/spray for high dose group.
Experimental: GB001 recombinant peptide spray high dose group; Each subject will take oral GB001 recombinant peptide spray 4 times a day, 4 sprays each time, no more than 29 times，The dosage of the group is 0.216mg/spray.	Drug: GB001 recombinant peptide spray low dose group、high dose group; Administrated oral spray. Each subject will take oral GB001 recombinant peptide spray 4 times a day, 4 sprays each time, no more than 29 times，The dosage of the group is 0.108mg/spray for low dose group and 0.216mg/spray for high dose group.
Experimental: Placebo group; Each subject will take oral GB001 recombinant peptide spray 4 times a day, 4 sprays each time, for 8 days.	Drug: GB001 recombinant peptide spray placebo; Administrated oral spray.Each subject will take oral GB001 recombinant peptide spray placebo 4 times a day, 4 sprays each time, no more than 29 times

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Target ulcer healing rate for patients in day 6	Ulcer healing was defined as disappearance of pain and zeroing of ulcer size. At the screening visit the ulcer area and pain severity of patient to clinical examination will be rated at baseline. The patients will be self-assessment pain severity in vas score sheet every day . And the patients will be re-examined in day 6 to assess the ulcer area by periodontal probe.	Day 6
Subjects self-rated the time it took for irritation pain to disappear		Day 8

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Time for target ulcer to heal	Ulcer healing was defined as disappearance of pain and zeroing of ulcer size.The patients will be self-assessment pain severity in vas score sheet every day . And the patients will be re-examined to assess the ulcer area by periodontal probe.	Day 8
Targeted ulcer healing rate for patients in day 4 and day 8	Ulcer healing was defined as disappearance of pain and zeroing of ulcer size. At the screening visit the ulcer area and pain severity of patient to clinical examination will be rated at baseline. The patients will be self-assessment pain severity in vas score sheet every day . And the patients will be re-examined in day 4 and day 8 to assess the ulcer area by periodontal probe.	Day 4,Day 8
Changes in target ulcer area relative to baseline	At the screening visit the ulcer area of patient to clinical examination will be rated at baseline. And the patients will be re-examined in day 4、day 6 and day 8 to assess the ulcer area by periodontal probe.	Day 4,Day 6,Day 8
Self evaluation of pain disappearance rate and relief rate		Day 4,Day 6,Day 8
Pharmacokinetics Characteristics， t½ of GB001 Recombinant Peptide	Apparent terminal phase half-life	Day 1,Day 4
Pharmacokinetics Characteristics，AUC of GB001 Recombinant Peptide	Area under the plasma concentration versus time curve	Day 1,Day 4
Pharmacokinetics Characteristics， Cmax of GB001 Recombinant Peptide	Maximum plasma concentration	Day 1,Day 4
Pharmacokinetics Characteristics， Tmax of GB001 Recombinant Peptide	Time of maximum plasma concentration	Day 1,Day 4
Immunogenicity(drug resistant antibody (ADA))		Day 1,Day 14,Day 28
Incidence of Adverse Events		First dose to last visit,an average of 1 month.

Collaborators and Investigators

• Sponsor: Zhejiang Echon Biopharm Limited

Study record dates

Study Major Dates

• Study Start (Actual): July 18, 2023
• Primary Completion (Actual): December 6, 2023
• Study Completion (Estimated): July 10, 2026

Study Registration Dates

• First Submitted: April 30, 2024
• First Submitted That Met QC Criteria: May 22, 2024
• First Posted (Actual): May 28, 2024

Study Record Updates

• Last Update Posted (Actual): April 9, 2025
• Last Update Submitted That Met QC Criteria: April 8, 2025
• Last Verified: April 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Mouth Diseases; Stomatognathic Diseases; Stomatitis; Stomatitis, Aphthous
• Other Study ID Numbers: YK2019L01P-IIa

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No