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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT06986070




Registration number
NCT06986070
Ethics application status
Date submitted
13/03/2025
Date registered
22/05/2025
Date last updated
30/05/2025

Titles & IDs
Public title
Investigation of Small Mobile Stem Cells (SMS Cells) in Participants With Chronic Obstructive Pulmonary Disease (COPD).
Scientific title
A Phase 1, First-in-Human, Clinical Trial to Evaluate the Safety of Small Mobile Stem Cells (SMS) Delivered Into the Lung as a Potential Organ Regeneration Therapy in Chronic Obstructive Pulmonary Disease (SORT-COPD Study
Secondary ID [1] 0 0
ACTRN12624001140549
Secondary ID [2] 0 0
A005
Universal Trial Number (UTN)
Trial acronym
SORT-COPD
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Chronic Obstructive Lung Disease 0 0
Condition category
Condition code
Respiratory 0 0 0 0
Chronic obstructive pulmonary disease

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Other - Stem cells

Experimental: Participants in the cohort 1 (low dose) will receive 1.2 billion cells/ml. - Dose escalating Cohort 1.

Experimental: Participants in the cohort 2 (medium dose) will receive 2.4 billion cells/ml. - Dose escalating Cohort 2.

Experimental: Participants in the cohort 3 (high dose) will receive 4.8 billion cells/m - Dose Escalating Cohort 3.


Treatment: Other: Stem cells
The suspension of stem cells will be put in a medical nebulizer machine, commonly used to deliver medicines into the lungs through inhalation. This means that the participants will breathe in the mist containing the SMS cells, produced by the nebulizer machine. This is a Phase I First-in-Human study, which indicates that this treatment is being studied for the first time in humans.
Intervention code [1] 0 0
Treatment: Other
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Adverse events.
Assessment method [1] 0 0
Treatment emergent adverse events and serious adverse events.
Timepoint [1] 0 0
Day 1, 2, 3, 4, 5, 8, 9, 21, 28, Month 3 and Month 6.
Primary outcome [2] 0 0
Dose limiting toxicity.
Assessment method [2] 0 0
Any treatment emergent adverse event that ii treatment related and classified as severe or the event is classified as a serious adverse event.
Timepoint [2] 0 0
Day 1, 2, 3, 4, 5, 8, 9, 21, 28, Month 3 and Month 6.
Primary outcome [3] 0 0
Safety laboratory testing
Assessment method [3] 0 0
All laboratory parameters will be summarized using descriptive statistics for each cohort and overall, for all time points assessed, including change from baseline for all post-dose assessments. Selected laboratory test results will be graded in accordance with the NCI CTCAE criteria.
Timepoint [3] 0 0
Screening, Day 2, 3, 5, 9, 21 and 28.
Secondary outcome [1] 0 0
Spirometry
Assessment method [1] 0 0
To be performed according to the American Thoracic Society/European Respiratory Society Standards. Forced Expiratory Volume in 1 second (FEV1) measured in liters (L).
Timepoint [1] 0 0
Screening, Day 1, 2, 3, 4, 5, 8, 9, 21 and 28.
Secondary outcome [2] 0 0
Spirometry
Assessment method [2] 0 0
To be performed according to the American Thoracic Society/European Respiratory Society Standards. Forced Vital Capacity (FVC) measured in liters (L).
Timepoint [2] 0 0
Screening, Day 1, 2, 3, 4, 5, 8, 9, 21 and 28.
Secondary outcome [3] 0 0
Oscillometry
Assessment method [3] 0 0
Resistance measured in cm H2O s/L .
Timepoint [3] 0 0
Screening, Day 1 and 28.
Secondary outcome [4] 0 0
Oscillometry
Assessment method [4] 0 0
Resistance change measured in cm H2O s/L..
Timepoint [4] 0 0
Screening, Day 1 and 28.
Secondary outcome [5] 0 0
Oscillometry
Assessment method [5] 0 0
Reactance of respiratory system measured in cm H2O s/L.
Timepoint [5] 0 0
Screening, Day 1 and 28.
Secondary outcome [6] 0 0
Plethysmograhy
Assessment method [6] 0 0
Functional Residual Capacity (FRC) measured in liters (L).
Timepoint [6] 0 0
Screening and Day 28.
Secondary outcome [7] 0 0
Plethysmograhy
Assessment method [7] 0 0
Total Lung Capacity (TLC) measured in liters (L).
Timepoint [7] 0 0
Screening and Day 28.
Secondary outcome [8] 0 0
Plethysmograhy
Assessment method [8] 0 0
Ratio of Residual Volume measured as a percentage (%).
Timepoint [8] 0 0
Screening and Day 28.
Secondary outcome [9] 0 0
Diffusing Capacity of the Lungs for Carbon Monoxide (DLCO)
Assessment method [9] 0 0
DLCO measured in milliliters per min per millimeter of Mercury (mL/min/mmHg).
Timepoint [9] 0 0
Screening and Day 28.
Secondary outcome [10] 0 0
Pulse oximetry
Assessment method [10] 0 0
Measuring oxygen saturation in the participants blood (SpO2)
Timepoint [10] 0 0
Screening, Day 1, 2, 3, 4, 5, 8, 9, 21 and 28.
Secondary outcome [11] 0 0
6-minute walk-test
Assessment method [11] 0 0
Assessment of exercise tolerance , distance walked in 6-minutes measured in meters (m).
Timepoint [11] 0 0
Screening, Day 1, 5, 9, 21 and 28.
Secondary outcome [12] 0 0
Exhaled Nitirc Oxide Test
Assessment method [12] 0 0
The fractional exhaled nitric oxide (FENO) test measures the amount of nitric oxide that is exhaled from a breath. It is a marker of lung inflammation, and is useful for differentiating asthma from COPD and other conditions presenting with similar symptoms \[26\]. FENO values of \< 25 parts per billion (ppb) are considered normal, 25-50 ppb as intermediate and \> 50 ppb as high.
Timepoint [12] 0 0
Screening, Day 1, 2, 3, 4, 5, 8, 9, 21 and 28.
Secondary outcome [13] 0 0
St George Respiratory Questionnaire for COPD
Assessment method [13] 0 0
Measurement of quality of life validated for participants with COPD. Each subscale and the total score range from 0 (no impact) to 100 (worst possible impact).
Timepoint [13] 0 0
Screening, Day 1, Day 28, Month 3 and Month 6.

Eligibility
Key inclusion criteria
1. Aged 39 to 69 years (inclusive).
2. Female participants:

A female participant is eligible to participate if she is not pregnant or breastfeeding, and one of the following conditions applies:

Is a woman of nonchildbearing potential (WONCBP) OR Is a woman of childbearing potential (WOCBP) and using an acceptable contraceptive method from screening until 30 days after the last dose of study intervention.

A WOCBP must have a negative highly sensitive pregnancy test (urine or serum as required by local regulations) at Screening and on Day 1, prior to administration of study intervention.

If a urine test cannot be confirmed as negative (e.g., an ambiguous result), a serum pregnancy test is required. In such cases, the participant must be excluded from participation if the serum pregnancy result is positive.

The investigator is responsible for review of medical history, menstrual history, and recent sexual activity to decrease the risk for inclusion of a woman with an early undetected pregnancy.
3. Male participants:

Male participants are eligible to participate if they agree to the following during the study intervention period and for at least 30 days after the last dose of study intervention:

Refrain from donating fresh unwashed semen. Be abstinent from heterosexual intercourse as their preferred and usual lifestyle (abstinent on a long-term and persistent basis) and agree to remain abstinent OR must agree to use contraception/barrier (male condom).
4. Has a diagnosis of mild or moderate COPD-C (cigarette smoking COPD) or COPD-P (biomass and pollution exposure COPD) according to the 2023 Global Initiative for Chronic Obstructive Lung Disease (GOLD) Criteria.

Lung impairment will be determined with post-bronchodilator FEV1 spirometry.
5. Has previously received treatment for COPD-C or COPD-P.
6. Has stable COPD disease state, defined as no exacerbations in the 12 weeks prior to screening, no hospitalizations in the 12 weeks prior to screening, and no changes in COPD medication in the 28 days prior to screening.
7. Agrees to comply with study specific procedures and visits, including non-smoking during the treatment and follow-up periods of the study.
8. Willing and able to provide written informed consent.
Minimum age
39 Years
Maximum age
69 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. Previous or current diagnosis of COPD-G, COPD-D, COPD-I, COPD-A or COPD-U; asthma, congestive heart failure, bronchiectasis, tuberculosis, obliterative bronchiolitis or diffuse panbronchiolitis.
2. Previous or current history of respiratory failure other than due to COPD (e.g. restrictive lung disease, sarcoidosis, tuberculosis, idiopathic pulmonary fibrosis, bronchiectasis, CMV pneumonitis, cystic fibrosis, asbestosis, silicosis or farmer's lung disease), or detection of any of these conditions through screening CT scanning.
3. History of COVID associated pneumonia with hypoxemic respiratory failure in the 12 weeks prior to screening.
4. Current use, or use within 21 days of screening, of systemic corticosteroids. Participants on inhaled corticosteroids or combined inhaled therapies can be included, however must schedule inhaled therapies 12 hours prior to dosing IP and 12-hours post dosing IP.
5. Any history of chronic liver disease, or abnormal liver function at screening, defined as:

Alanine transaminase (ALT) and/or aspartate aminotransferase (AST) more than 2.5 times the upper limit of normal (ULN) Total bilirubin more than two times the ULN Participants with documented Gilbert's syndrome are permitted to enter the study.
6. History of chronic renal insufficiency, defined as and eGFR < 90 mL/min/1.73 m2. Participants without a history of chronic renal insufficiency, who at screening have an eGFR detected between 60-89 mL/min/1.73 m2, should have their eGFR repeated as per standard clinical procedure.
7. Uncontrolled hypertension, or current hypertension controlled on more than two medications.
8. History, within the last two years, of coronary artery disease, coronary artery bypass graft surgery, percutaneous transluminal coronary angioplasty, severe peripheral arterial disease, cerebrovascular disease (including history of transient ischemic attack or cerebrovascular accident), or any such history that, in the opinion of the investigator, will impact participation in the study.
9. Type 1 or Type 2 diabetes mellitus with glycosylated hemoglobin (HbA1c > 7.5%) at screening.
10. No active malignancy in the last three years, including any potential neoplasm detected via high-resolution CT scan (nodule > 5 mm) detected as part of the screening process.

People with a history of basal cell or squamous cell carcinoma of the skin are eligible.
11. History of HIV or other immunosuppressed conditions, Hepatitis B or Hepatitis C, or use of immunosuppressive medications within 14 days prior to screening, and for 3 weeks after the last dose of study intervention is administered.
12. Active infection requiring systemic antibiotic treatment within 12 weeks prior to screening.
13. Body mass index (BMI) > 60 kg/m2.
14. Active smoking of tobacco, cannabis or vaping in the 28 days prior to screening as indicated by results of cotinine urine testing, performed at screening. Use of nicotine containing products such as nicotine patches, oral nicotine products or nicotine chewing gum is permitted, noting that cotinine results will be positive. Positive results will be documented by the investigator as "attributed to permitted nicotine products".
15. History of alcohol or other substance abuse, with sobriety less than one year, or that in the opinion of the investigator, will impact participation in the study.
16. Pregnant or breastfeeding.
17. Any severe medication allergy, including an allergy to bovine products.
18. Any other medical condition, psychiatric condition or illness, that according to the Principal Investigator might render the subject unlikely to tolerate the inhalation of SORT-COPD (SMS cells), or to complete the study.
19. Current participation in any other clinical trial or participation in the last six weeks or subject received an experimental therapy (drug or biologic) for any indication within 12 months prior to enrolment.
20. Unable or unwilling to comply with study specific schedules or procedures.

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Other
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
1/06/2025
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/12/2025
Actual
Sample size
Target
18
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
Veritas Research - Bayswater
Recruitment postcode(s) [1] 0 0
3153 - Bayswater

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
SMSbiotech
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Public notes

Contacts
Principal investigator
Name 0 0
Kianoosh Noori Samie, Medical Doctor
Address 0 0
Veritas Research
Country 0 0
Phone 0 0
Email 0 0
Contact person for public queries
Name 0 0
Kianoosh Noorie Samie, Medical Doctor
Address 0 0
Country 0 0
Phone 0 0
+61 3 8736 1750
Email 0 0
info@veritusresearch.com
Contact person for scientific queries

Data sharing statement


What supporting documents are/will be available?

No Supporting Document Provided


Results publications and other study-related documents

No documents have been uploaded by study researchers.

Results not provided in https://clinicaltrials.gov/study/NCT06986070