Safety and Tolerability Study of MSC Exosome Ointment

NCT05523011 | Completed Phase 1 Results DMC

• 1 other identifier: PTD2021P001
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

Brief Summary

This phase 1 study aims to evaluate the safety and tolerability of the application of the MSC exosome ointment with repeated topical application on adult healthy subjects (TID from Day 1 to 20). The results of this study were to provide the first clinical information on the drug's safety and inform the selection of administration of exosome ointment to be evaluated in subsequent clinical studies. The endpoints included to assess the safety and tolerability of exosome ointment for topical application in healthy adult volunteers are:

• Frequency of treatment-emergent adverse events (TEAEs)
• The incidence rate of local skin responses (LSR, i.e., erythema, flaking/scaling, crusting, swelling, vesiculation/pustulation, erosion/ulceration, hyperpigmentation, hypopigmentation, and scarring) \[Time Frame: Days 1 and 21\]
• Changes from baseline in blood parameters (including hematology, chemistry, and other inflammation parameters such as C-reactive protein \[CRP\] and erythrocyte sedimentation rate \[ESR\]) \[Time Frame: Days 1 and 21\]

Conditions

Psoriasis

Keywords

Mesenchymal stem/stromal cell; Exosome; Exosome ointment; Topical application; Safety and Tolerability; Extracellular vesicle

Outcome Measures

Primary Outcomes (38)

• Number of Participants With Treatment-emergent Adverse Events (TEAEs)

  All through the treatment period, the subjects completed the Subject Diary to capture the frequency of TEAEs.

  Day 1 - 20

• Change From Baseline in the Systolic Blood Pressure After 20 Days of Treatment

  Systolic blood pressure (mmhg) was assessed on Day 1 (pre-treatment) and Day 21 (+3 days)

  Day 1 - 21+3

• Change From Baseline in the Diastolic Blood Pressure After 20 Days of Treatment

  Diastolic blood pressure (mmhg) was assessed on Day 1 (pre-treatment) and Day 21 (+3 days)

  Day 1 - 21+3

• Change From Baseline in the Heart Rate After 20 Days of Treatment

  Heart rate (beats/min) was assessed on Day 1 (pre-treatment) and Day 21 (+3 days)

  Day 1 - 21+3

• Change From Baseline in the Respiratory Rate After 20 Days of Treatment

  Respiratory rate (breaths/min) was assessed on Day 1 (pre-treatment) and Day 21 (+3 days)

  Day 1 - 21+3

• Change From Baseline in the Body Temperature After 20 Days of Treatment

  Body temperature (°C) was assessed on Day 1 (pre-treatment) and Day 21 (+3 days)

  Day 1 - 21+3

• Change From Baseline in the Basophils After 20 Days of Treatment

  Basophils (10\^9 Basophils /L) were assessed on Day 1 (pre-treatment) and Day 21 (+3 days)

  Day 1 - 21+3

• Change From Baseline in the Eosinophils After 20 Days of Treatment

  Eosinophils (10\^9 Eosinophils/L) were assessed on Day 1 (pre-treatment) and Day 21 (+3 days)

  Day 1 - 21+3

• Change From Baseline in the Hematocrit After 20 Days of Treatment

  hematocrit(%) assessed on Day 1 (pre-treatment) and Day 21 (+3 days)

  Day 1 - 21+3

• Change From Baseline in the Hemoglobin After 20 Days of Treatment

  Hemoglobin (g/dL) was assessed on Day 1 (pre-treatment) and Day 21 (+3 days)

  Day 1 - 21+3

• Change From Baseline in the Lymphocytes After 20 Days of Treatment

  Lymphocytes (10\^9 Lymphocytes/L) were assessed on Day 1 (pre-treatment) and Day 21 (+3 days)

  Day 1 - 21+3

• Change From Baseline in the Monocytes After 20 Days of Treatment

  Monocytes (10\^9 Monocytes/L) were assessed on Day 1 (pre-treatment) and Day 21 (+3 days)

  Day 1 - 21+3

• Change From Baseline in the Red Blood Cell After 20 Days of Treatment

  Red blood cell (10\^12 Red blood cells/L) was assessed on Day 1 (pre-treatment) and Day 21 (+3 days)

  Day 1 - 21+3

• Change From Baseline in the White Blood Cell After 20 Days of Treatment

  White Blood Cell (10\^9 White blood cells/L) was assessed on Day 1 (pre-treatment) and Day 21 (+3 days)

  Day 1 - 21+3

• Change From Baseline in the Bicarbonate After 20 Days of Treatment

  Bicarbonate (mmol/L) was assessed on Day 1 (pre-treatment) and Day 21 (+3 days)

  Day 1 - 21+3

• Change From Baseline in the Blood Urea Nitrogen After 20 Days of Treatment

  Blood urea nitrogen (mg/dL) was assessed on Day 1 (pre-treatment) and Day 21 (+3 days)

  Day 1 - 21+3

• Change From Baseline in the Calcium After 20 Days of Treatment

  Calcium (mmol/L) was assessed on Day 1 (pre-treatment) and Day 21 (+3 days)

  Day 1 - 21+3

• Change From Baseline in the Chloride After 20 Days of Treatment

  Chloride (mmol/L) was assessed on Day 1 (pre-treatment) and Day 21 (+3 days)

  Day 1 - 21+3

• Change From Baseline in the Creatinine After 20 Days of Treatment

  Creatinine (umol/L) was assessed on Day 1 (pre-treatment) and Day 21 (+3 days)

  Day 1 - 21+3

• Change From Baseline in the Glucose After 20 Days of Treatment

  Glucose (mmol/L) was assessed on Day 1 (pre-treatment) and Day 21 (+3 days)

  Day 1 - 21+3

• Change From Baseline in the Potassium After 20 Days of Treatment

  Potassium (mmol/L) was assessed on Day 1 (pre-treatment) and Day 21 (+3 days)

  Day 1 - 21+3

• Change From Baseline in the Sodium After 20 Days of Treatment

  Sodium (mmol/L) was assessed on Day 1 (pre-treatment) and Day 21 (+3 days)

  Day 1 - 21+3

• Change From Baseline in the Alanine Transaminase After 20 Days of Treatment

  Alanine transaminase (U/L) was assessed on Day 1 (pre-treatment) and Day 21 (+3 days)

  Day 1 - 21+3

• Change From Baseline in the Albumin After 20 Days of Treatment

  Albumin (g/L) was assessed on Day 1 (pre-treatment) and Day 21 (+3 days)

  Day 1 - 21+3

• Change From Baseline in the Alkaline Phosphatase After 20 Days of Treatment

  Alkaline phosphatase (U/L) was assessed on Day 1 (pre-treatment) and Day 21 (+3 days)

  Day 1 - 21+3

• Change From Baseline in the Aspartate Aminotransferase After 20 Days of Treatment

  Aspartate aminotransferase (U/L) was assessed on Day 1 (pre-treatment) and Day 21 (+3 days)

  Day 1 - 21+3

• Change From Baseline in the Direct Bilirubin After 20 Days of Treatment

  Direct bilirubin (umol/L) was assessed on Day 1 (pre-treatment) and Day 21 (+3 days)

  Day 1 - 21+3

• Change From Baseline in the Total Bilirubin After 20 Days of Treatment

  Total bilirubin (umol/L) was assessed on Day 1 (pre-treatment) and Day 21 (+3 days)

  Day 1 - 21+3

• Change From Baseline in the C-reactive Protein After 20 Days of Treatment

  C-reactive protein (mg/L) was assessed on Day 1 (pre-treatment) and Day 21 (+3 days)

  Day 1 - 21+3

• Change From Baseline in the Erythrocyte Sedimentation Rate After 20 Days of Treatment

  Erythrocyte sedimentation rate (mm/hr) was assessed on Day 1 (pre-treatment) and Day 21 (+3 days)

  Day 1 - 21+3

• Assessment of the Dryness of the Area of Application

  Visual assessments of the dryness of the area of application was carried out using an adapted version of SCORAD scale on Day 1 (pre-treatment) and Day 21 (+3 days). SCORAD scale assessment (range: 0-3) 0= none, 1= mild, 2= moderate, 3= severe

  Day 1 - 21+3

• Assessment of the Oozing/Crusting of the Area of Application

  Visual assessments of the oozing/crusting of the area of application was carried out using an adapted version of SCORAD scale on Day 1 (pre-treatment) and Day 21 (+3 days). SCORAD scale assessment (range: 0-3) 0= none, 1= mild, 2= moderate, 3= severe

  Day 1 - 21+3

• Assessment of the Redness of the Area of Application

  Visual assessments of the rednessof the area of application was carried out using an adapted version of SCORAD scale on Day 1 (pre-treatment) and Day 21 (+3 days). SCORAD scale assessment (range: 0-3) 0= none, 1= mild, 2= moderate, 3= severe

  Day 1 - 21+3

• Assessment of the Scratch Marks of the Area of Application

  Visual assessments of the scratch marks the area of application were carried out using an adapted version of SCORAD scale on Day 1 (pre-treatment) and Day 21 (+3 days). SCORAD scale assessment (range: 0-3) 0= none, 1= mild, 2= moderate, 3= severe

  Day 1 - 21+3

• Assessment of the Skin Thickening of the Area of Application

  Visual assessments of the skin thickening the area of application was carried out using an adapted version of SCORAD scale on Day 1 (pre-treatment) and Day 21 (+3 days). SCORAD scale assessment (range: 0-3) 0= none, 1= mild, 2= moderate, 3= severe

  Day 1 - 21+3

• Assessment of the Swelling of the Area of Application

  Visual assessments of the swelling of the area of application was carried out using an adapted version of SCORAD scale on Day 1 (pre-treatment) and Day 21 (+3 days).

  Day 1 - 21+3

• Assessment of the Itch of the Area of Application

  Subjective symptoms: The identified area of application was assessed for the subjective symptom itch, graded from 0 (no itch) to 10 (worst imaginable itch)

  Day 1 - 21+3

• Assessment of the Sleeplessness

  Subjective symptoms: The participant was assessed for the subjective symptom sleeplessness and graded from 0 (no sleeplessness) to 10 (worst imaginable sleeplessness)

  Day 1 - 21+3

Study Arms (1)

Exosome Ointment

EXPERIMENTAL

The subjects will apply exosome ointment along with Vesiderm liposome cream (TID per day, 20 days). Each day (from Day 1 to 20), the study product will be applied with a gap of 4 hours between three doses to a healthy area (one hand area) on the forearm using 1 fingertip unit (FTU). The Vesiderm liposome cream is meant to keep the application site moisturized.

Drug: Exosome ointment

Interventions

Exosome ointment DRUG

100 µg MSC exosomes/g ointment

Exosome Ointment

Eligibility Criteria

• Age: 21 Years+
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Signed ICF prior to entering the study or undergoing any study procedures.
• Male or female, \>21 years of age at the time of signing the informed consent.
• Subject who had been fully vaccinated for COVID-19 and was not under quarantine or Stay Home Notice or on medical leave.
• Subject who was willing and able to comply with scheduled visits, treatment plan, laboratory tests, and other study procedures.
• Subject was able to read and write in the English language.
• For women who were not postmenopausal (\> 12 months of non-therapy induced amenorrhea) or surgically sterile (absence of ovaries and/or uterus):
• had a negative pregnancy test at screening
• remained abstinent or used contraceptive methods during the treatment period and continued for at least 30 days (one menstrual cycle) after the last treatment dose.

You may not qualify if:

• Subject had any serious skin condition that was not well controlled.
• Subject was currently using topical treatments.
• Pregnant or breast-feeding women.
• Subject had received phototherapy, oral corticosteroids, oral retinoid, oral immunosuppressive / immune modulative drugs, cytostatic / cytotoxic drugs, cyclosporine, or methotrexate within 30 days prior to the first application of the study product.
• Subject was currently using any medications including biologics or undergoing treatment known to affect skin conditions.
• Subject had any other clinically significant laboratory abnormalities, co-morbidities or psychiatric conditions that would place the subject at increased risk or confound the objectives of the study.
• Subject had used any investigational drugs or biologics and/or participated in any clinical trial within the last 60 days before the day of the first dose of the study drug or was taking part in a non-medication study that would interfere with study compliance or outcome assessments.

Sponsors & Collaborators

• Paracrine Therapeutics Dermatology Pte. Ltd.: lead
• National University Hospital, Singapore: collaborator

Study Sites (1)

National University Hospital

Singapore, 119074, Singapore

Location

MeSH Terms

Conditions

Psoriasis

Condition Hierarchy (Ancestors)

Skin Diseases, Papulosquamous; Skin Diseases; Skin and Connective Tissue Diseases

Results Point of Contact

• Title: Lai Ruenn Chai
• Organization: Paracrine Therapeutics Private Limited

ruennchai.lai@paracrinetherapeutics.com

+6593375977

Study Officials

• Nisha Suyien Chandran, Dr.

  National University Hospital, Singapore

  PRINCIPAL INVESTIGATOR

Publication Agreements

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

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Masking Details: Open-label
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Single-center, single-arm, open-label study

Study Record Dates

• First Submitted: August 25, 2022
• First Posted: August 31, 2022
• Study Start: March 8, 2022
• Primary Completion: April 5, 2022
• Study Completion: April 5, 2022
• Last Updated: October 31, 2024
• Results First Posted: October 31, 2024

Record last verified: 2024-08

Data Sharing

• IPD Sharing: Will not share

Locations

SG (1)