Trial Review

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Trial registered on ANZCTR


Registration number
ACTRN12624001140549p
Ethics application status
Submitted, not yet approved
Date submitted
20/08/2024
Date registered
20/09/2024
Date last updated
20/09/2024
Date data sharing statement initially provided
20/09/2024
Type of registration
Prospectively registered

Titles & IDs
Public title
A study to investigate small mobile stem cells (SMS cells) in participants aged 39 to 69 years with chronic obstructive pulmonary disease.
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] 312791 0
A005
Universal Trial Number (UTN)
Trial acronym
SORT-COPD
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Chronic Obstructive Pulmonary Disease 334851 0
Condition category
Condition code
Respiratory 331403 331403 0 0
Chronic obstructive pulmonary disease

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
This study is a phase 1, first-in-human, open-label, non-randomized, dose escalation safety study of 18 participants, between 39 and 69 years of age, with mild to moderate chronic obstructive pulmonary disease, treated with SORT-COPD (SMS cells).
The primary objective of this study is to determine the safety of SORT-COPD (SMS cells) at three doses in people with chronic obstructive pulmonary disease.

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.

18 participants are expected to be enrolled into this study. Participants will be divided into three groups of six volunteers, with the first group receiving the lowest dose of the study treatment, the second group receiving a higher dose, and the third group receiving the highest dose. Between each of the three groups, there will be a pause to permit detection of any short-term adverse effects of the treatment at a specific dose.

Participants will be treated with the study drug, SORT COPD (Small Mobile Stem Cells), delivered via a medical nebuliser on the 1st, 4th, and the 8th days of the study in-clinic. The nebuliser treatment involves inhaling mist from a mouthpiece attached to a standard medical nebuliser machine and takes about 10 to 15 minutes to complete.
The volume to be nebulised is 3 millilitres (less than one teaspoon).
Participants in the cohort 1 (low dose) will receive 1.2 billion cells/ml.
Participants in the cohort 2 (medium dose) will receive 2.4 billion cells/ml.
Participants in the cohort 3 (high dose) will receive 4.8 billion cells/ml.

The study treatment (SMS) cells will be given in three different concentrations with the lowest dose given to the 1st group of 6 participants, with the dose doubled for each group. The members of each group will receive the same dose at each treatment visit.

Participants will actively be in the study for up to 5 or 6 weeks, including the initial screening and testing period before the treatment is applied, during treatment, and about 4 weeks after the first treatment. Also, about 3 to 6 months after the study, participants will be contacted via telephone to answer some questions about their state of health, which will be the end of the study.
Individual participant duration will be up to a maximum of 15 months.
Intervention code [1] 329317 0
Treatment: Other
Comparator / control treatment
No control group
Control group
Uncontrolled

Outcomes
Primary outcome [1] 339151 0
To establish the safety and tolerability of SORT-COPD (SMS cells), in suspension, delivered to the lung via aerosolized mist produced by a vibrating mesh nebulizer.
Timepoint [1] 339151 0
Vital signs will be collected on screening, Day 0, Day 1, Day 2, Day 3, Day 4, Day 5, Day 8, Day 9, Day 21, Day 28.
Full physical examination will be performed on screening, Day 9, and Day 28. Focused physical examination will be performed on Day 1, Day 3, Day 5, Day 9, and Day 21.
High-resolution CT scan will be performed on screening, Day 28.
Adverse events will be recorded on Day 1, Day 2, Day 3, Day 4, Day 5, Day 8, Day 9, Day 21, Day 28, and at 3-6 months.
Clinical laboratory samples will be collected on Day 1, Day 3, Day 5, Day 9, and Day 21.
Spirometry will be performed on screening, Day 9, and Day 28.
Oscillometry and plethysmography will be performed on screening, Day 9, and Day 28.
Diffusing capacity of the lung for carbon monoxide will be performed on screening, Day 9, and Day 28.
Pulse oximetry will be performed on screening, Day 1, Day 3, Day 5, Day 9, Day 21, and Day 28.
6-minute walk test will be performed on screening, Day 9, and Day 28.
Fractional exhaled nitric oxide test will be performed on screening, Day 9, and Day 28.
Secondary outcome [1] 439502 0
To evaluate the preliminary efficacy of SORT-COPD (SMS cells), in suspension, delivered to the lung via aerosolized mist produced by a vibrating mesh nebulizer in participants' pulmonary function.
Timepoint [1] 439502 0
Spirometry will be performed on screening, Day 9, and Day 28.
Oscillometry and plethysmography will be performed on screening, Day 9, and Day 28.
Diffusing capacity of the lung for carbon monoxide will be performed on screening, Day 9, and Day 28.
Pulse oximetry will be performed on screening, Day 1, Day 3, Day 5, Day 9, Day 21, and Day 28.
6-minute walk test will be performed on screening, Day 9, and Day 28.
Secondary outcome [2] 439505 0
Any signs of change in lung structure consistent with regeneration of lung tissue, any other positive effects on airflow and circulation.
Timepoint [2] 439505 0
A Non-Contrast High-resolution CT(HRCT) of the lungs will be performed at baseline and at the follow-up visit 4 weeks after the first dose of the study intervention.
Secondary outcome [3] 439507 0
Quality of Life
Timepoint [3] 439507 0
St George’s Respiratory Questionnaire for COPD (SGRQ-C) will be completed on screening, Day 28, and 3-6 months.

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. If currently prescribed Inhaled Corticosteroids, must be willing and able to avoid intake of the morning dose on the days of IP administration, and limit taking the dose until late evening (>12 hours after administration of 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 renal insufficiency, defined as chronic kidney disease stage 2 to stage 5 as per KDIGO 2020 definitions.

7. Uncontrolled hypertension, or current hypertension controlled on more than two medications.

8. History 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).

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 or cannabis in the 28 days prior to screening, any use of nicotine containing products (vaping, nicotine patches, oral nicotine products or nicotine chewing gum), or as indicated by results of cotinine saliva testing, performed at screening.

15. History of alcohol or other substance abuse.

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
Dose Escalation
Phase
Not Applicable
Type of endpoint/s
Safety
Statistical methods / analysis

Recruitment
Recruitment status
Not yet recruiting
Date of first participant enrolment
Anticipated
21/10/2024
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
18
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
VIC

Funding & Sponsors
Funding source category [1] 317222 0
Commercial sector/Industry
Name [1] 317222 0
SMSbiotech
Country [1] 317222 0
Australia
Primary sponsor type
Commercial sector/Industry
Name
SMSbiotech Australia Pty Ltd
Address
Country
Australia
Secondary sponsor category [1] 319502 0
None
Name [1] 319502 0
Address [1] 319502 0
Country [1] 319502 0

Ethics approval
Ethics application status
Submitted, not yet approved
Ethics committee name [1] 315959 0
Bellberry Human Research Ethics Committee A
Ethics committee address [1] 315959 0
Ethics committee country [1] 315959 0
Australia
Date submitted for ethics approval [1] 315959 0
04/09/2024
Approval date [1] 315959 0
Ethics approval number [1] 315959 0

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 136346 0
Dr Kianoosh Noori Samie
Address 136346 0
Veritus Research, Building 21, 885 Mountain Highway Bayswater VIC 3153
Country 136346 0
Australia
Phone 136346 0
+610387361750
Fax 136346 0
Email 136346 0
[email protected]
Contact person for public queries
Name 136347 0
Kianoosh Noori Samie
Address 136347 0
Veritus Research, Building 21, 885 Mountain Highway Bayswater VIC 3153
Country 136347 0
Australia
Phone 136347 0
+610387361750
Fax 136347 0
Email 136347 0
[email protected]
Contact person for scientific queries
Name 136348 0
Dr Roger Schechter
Address 136348 0
SMSbiotech, 1825 Diamond St Ste 101 San Marcos, CA 92078
Country 136348 0
United States of America
Phone 136348 0
+17602903406
Fax 136348 0
Email 136348 0
[email protected]

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

Documents added manually
No documents have been uploaded by study researchers.

Documents added automatically
No additional documents have been identified.