Safety of Topical Exosome-Containing Liquid in Healthy Volunteers

Phase 1Not Yet RecruitingNCT07703969

West Kazakhstan Marat Ospanov Medical University10 enrolled

Overview

This Phase 1 split-site pilot study will evaluate the safety and dermal tolerability of a topical exosome-containing liquid in 10 healthy adult volunteers. The investigational liquid will be applied to a small defined area of intact skin. A vehicle liquid without exosomes may be applied to a matched contralateral skin site as a control. The study will assess local skin reactions, systemic adverse events, vital signs, clinical laboratory parameters, and feasibility of topical administration. No burn wound or artificial skin wound will be induced in participants in this Phase 1 safety study.

The investigational product is a sterile topical exosome-containing liquid being developed for future evaluation in burn wound repair. Preclinical in vivo evaluation in murine wound models suggested improved wound closure and better histological organization of repaired skin after topical exosome-containing formulation administration, with the therapeutic effect being more pronounced in the thermal burn model. In the burn model, the exosome-containing formulation group showed faster wound-area reduction, improved epidermal and dermal organization, reduced inflammatory cellularity, and no significant body-weight differences compared with control groups. This first-in-human Phase 1 study is designed to evaluate safety and dermal tolerability in healthy adult volunteers before the product is tested in patients with burn wounds. No artificial burn wound or skin wound will be created. The investigational liquid will be applied to a small defined area of intact skin according to the protocol-defined dose and schedule. If a split-site design is used, vehicle liquid without exosomes will be applied to a matched contralateral intact skin site as a control. Participants will be monitored for local skin irritation, erythema, edema, pruritus, burning sensation, pain, rash, allergic reaction, systemic adverse events, vital signs, and clinical laboratory abnormalities. The first two participants will be enrolled as sentinel participants. Safety data through at least 72 hours after first application will be reviewed before enrollment of the remaining participants. The main purpose of the study is to determine whether topical administration of the exosome-containing liquid is safe and tolerable on intact human skin.

Study Type

INTERVENTIONAL

Allocation

Interventions

Exosome-Containing LiquidBIOLOGICAL

The investigational product is a sterile topical liquid containing exosome-enriched extracellular vesicles derived from \[insert cell source, e.g., mesenchymal stromal cells\]. The liquid will be applied topically to a small defined area of intact skin at a dose of \[insert exact dose, e.g., X mL/cm² containing Y particles/mL, equivalent to Z particles/cm²\] according to the protocol-defined schedule. The product is investigational and is being evaluated for safety and dermal tolerability before future testing in patients with burn wounds.

Vehicle LiquidOTHER

Vehicle liquid without exosomes will be applied topically to a matched contralateral intact skin site according to the same protocol-defined schedule as the exosome-containing liquid. The vehicle liquid is used as a within-participant control for local dermal tolerability assessment.

Eligibility

Sex: ALLMin age: 18 YearsMax age: 55 YearsHealthy volunteers:

Medical Language ↔ Plain English

Inclusion Criteria: 1. Healthy adult volunteers aged 18 to 55 years. 2. Able and willing to provide written informed consent. 3. No clinically significant abnormality based on medical history, physical examination, vital signs, and screening laboratory tests. 4. Intact healthy skin at the planned application sites. 5. Willingness to avoid applying other topical products, cosmetics, antiseptics, or irritant substances to the application sites during the study period. 6. Willingness to avoid excessive sun exposure, sauna, swimming pool use, and mechanical irritation of the application sites during the study period. 7. Willingness to comply with all study visits, topical application procedures, local skin assessments, skin photography, safety assessments, and follow-up. 8. For women of childbearing potential, a negative pregnancy test before first application and willingness to use an acceptable method of contraception during the study period. Exclusion Criteria: 1. Any active skin disease, dermatitis, eczema, psoriasis, urticaria, acneiform eruption, skin infection, open wound, scar, tattoo, burn scar, pigmentation disorder, or clinically significant skin abnormality at the planned application sites. 2. History of severe allergy, anaphylaxis, or hypersensitivity to topical liquids, dressings, biological products, exosome-containing products, or any component of the investigational product or vehicle liquid. 3. Current acute illness, fever, or active infection. 4. Known autoimmune disease, immunodeficiency, or current systemic immunosuppressive therapy. 5. Use of systemic corticosteroids, immunomodulatory drugs, biological agents, or investigational products within 30 days before enrollment. 6. Use of topical corticosteroids, topical immunomodulators, topical antibiotics, retinoids, keratolytic agents, or other medicated topical products on or near the planned application sites within 14 days before enrollment. 7. Clinically significant hepatic, renal, cardiovascular, hematologic, endocrine, neurologic, psychiatric, or systemic disease that may increase risk or interfere with interpretation of study results. 8. Known active malignancy or history of malignancy within the past 5 years. 9. Positive screening test for clinically relevant transmissible infection, if required by the protocol. 10. Pregnancy or breastfeeding. 11. Positive pregnancy test at screening or before first application in women of childbearing potential. 12. Participation in another interventional clinical trial within 30 days before enrollment. 13. Blood donation or major blood loss within 30 days before enrollment, if laboratory safety monitoring is included in the protocol. 14. Any condition that, in the investigator's judgment, would make participation unsafe or interfere with interpretation of study results.

Outcomes

Primary Outcomes

Number of Participants With Treatment-Emergent Adverse Events

Number of participants with any treatment-emergent adverse event after topical application of the exosome-containing liquid. Events include local application-site reactions such as erythema, edema, pruritus, burning sensation, pain, rash, vesicles, ulceration, allergic reaction, or infection, and systemic events such as fever, malaise, abnormal vital signs, clinically significant laboratory abnormalities, serious adverse events, or any other clinically significant adverse event judged by the investigator.

Time frame: From first application through Day 28

Secondary Outcomes

Maximum Total Score on the Draize Dermal Irritation Scale at the Exosome-Containing Liquid Application Site

The Draize Dermal Irritation Scale grades erythema and eschar formation from 0, indicating no erythema, to 4, indicating severe erythema or eschar formation, and edema from 0, indicating no edema, to 4, indicating severe edema. The total score is the sum of the erythema/eschar score and edema score and ranges from 0 to 8. Higher scores indicate worse local skin irritation. The maximum total score observed at the exosome-containing liquid application site will be reported.

Time frame: Baseline, 30 minutes, 24 hours, 48 hours, 72 hours, Day 7, Day 14, and Day 28

Within-Participant Difference in Total Draize Dermal Irritation Scale Score Between Exosome-Containing Liquid and Vehicle-Control Sites

The within-participant difference will be calculated as the total Draize Dermal Irritation Scale score at the exosome-containing liquid application site minus the total score at the vehicle-control liquid application site at each assessment time point. The total Draize score ranges from 0 to 8, with higher scores indicating worse local skin irritation. Positive values indicate higher irritation at the exosome-containing liquid site, and negative values indicate higher irritation at the vehicle-control site.

Time frame: Baseline, 30 minutes, 24 hours, 48 hours, 72 hours, Day 7, Day 14, and Day 28

Number of Participants With Clinically Significant Vital Sign Abnormalities

Number of participants with any clinically significant abnormality in vital signs after topical application of the exosome-containing liquid. Vital signs include systolic blood pressure, diastolic blood pressure, heart rate, respiratory rate, and body temperature. Clinical significance will be determined by the investigator using protocol-defined reference ranges and clinical judgment. The reporting unit is number of participants.

Time frame: From first application through Day 28

Number of Participants With Clinically Significant Clinical Laboratory Abnormalities

Number of participants with any clinically significant abnormality in clinical laboratory safety tests after topical application of the exosome-containing liquid. Laboratory safety tests include complete blood count, renal function tests, liver function tests, and inflammatory markers according to the protocol. Clinical significance will be determined by the investigator using protocol-defined reference ranges and clinical judgment. The reporting unit is number of participants.

Time frame: From first application through Day 28

Percentage of Planned Topical Applications Completed According to Protocol

For each participant, the topical application completion percentage will be calculated as the number of completed topical applications divided by the number of protocol-planned topical applications, multiplied by 100. The outcome will be reported as the mean percentage of planned topical applications completed across participants. A higher percentage indicates better feasibility of topical application.

Time frame: From Day 0 through Day 14

Number of Participants With Delayed Treatment-Emergent Adverse Events

Number of participants with delayed local or systemic treatment-emergent adverse events occurring after completion of the primary Day 28 safety assessment. Delayed events may include local skin reactions, allergic reactions, infection, abnormal vital signs, clinically significant laboratory abnormalities, serious adverse events, or any other clinically significant adverse event judged by the investigator.

Time frame: From Day 29 through Day 56

Safety of Topical Exosome-Containing Liquid in Healthy Volunteers

Overview

Conditions

Interventions

Eligibility

Locations (1)

Outcomes

Primary Outcomes

Secondary Outcomes

Central Contacts