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Trial registered on ANZCTR


Registration number
ACTRN12619000883112
Ethics application status
Approved
Date submitted
8/06/2019
Date registered
21/06/2019
Date last updated
21/06/2019
Date data sharing statement initially provided
21/06/2019
Type of registration
Prospectively registered

Titles & IDs
Public title
Evaluation of intra-venous mesenchymal stem cells for the treatment of small joint osteoarthritis
Scientific title
The evaluation of safety of intra-venous autologous adipose derived mesenchymal stem cell therapy in the treatment of small joint osteoarthritis. A pilot study.
Secondary ID [1] 298449 0
Nil
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Osteoarthritis 313193 0
Condition category
Condition code
Musculoskeletal 311652 311652 0 0
Osteoarthritis

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
This study will look to prospectively assess the response of small joint osteoarthritis to intravenous mesenchymal stem cell therapy using the following protocol :
- a single intra-venous infusion of mesenchymal stem cells (1.5million per kg of body weight) at commencement of treatment (baseline). Mesenchymal stem cells will be mixed with a plasmalyte-148 solution to a total of up to 50mls and will be infused over a 20minute period.
- participants will be routinely monitored on the day of infusion for a period of 3hours.
- prior to administration participants wil undergo a routine 12-lead ECG, and have blood samples taken for clinical laboratory testing.

This will be a single treatment group uncontrolled case series pilot study.

Autologous adipose derived mesenchymal stem cells will be used due to the ease of harvest (liposuction) and safety. The small abdominal liposuction will be performed by an appropriately qualified medical clinician. This will be a day procedure and will take up to 1 hour. Isolation and expansion of mesenchymal stem cells will be performed in a suitable certified clean room facility operated by Magellan Stem Cells.

Commencement of therapy will be performed no earlier than 8 weeks after liposuction due to time taken to isolate and expand the mesenchymal stem cells.

Intra-venous infusion will be performed under the supervision of a qualified medical clinician with a medical degree equivalent of Bachelor of Medicine & Bachelor of Surgery (MBBS).

Mesenchymal stem cell will be cryopreserved in clinical grade qualified MSC cryoprotectant media using a validated control rate freezing method and stored in liquid nitrogen until use.
Intervention code [1] 314695 0
Treatment: Other
Comparator / control treatment
No control group
Control group
Uncontrolled

Outcomes
Primary outcome [1] 320356 0
To evaluate the safety of autologous adipose-derived mesenchymal stem cells (AdMSC) administered intravenously for the treatment of small joint osteoarthritis.
- safety will be assessed using routine medical vital sign observations, ECG assessment, and blood biochemistry laboratory testing. These tests will be conducted at baseline, time of stem cell administration and throughout the follow-up period of 12 months.
- Adverse Events will be assessed by the principle investigator and clinical investigator and also by a Data and Safety Management Committee at regular intervals. Adverse events will be collected from medical records and participant self reporting of symptoms.
- No serious adverse events have been reported in previous studies.
Timepoint [1] 320356 0
Assessment at baseline, day 7 and months 1,3,6,9 and 12 post commencement of therapy. Formal primary end point will be at 12 months.
Secondary outcome [1] 371344 0
Pain using the validated questionnaire Numeric Pain Rating Scale
Timepoint [1] 371344 0
Outcome scores assessed at 0, 1, 3, 6, 9 and 12months
Secondary outcome [2] 371562 0
Function assessed using the validated questionnaire Disability of arm, shoulder and hand (DASH) score.
Timepoint [2] 371562 0
Outcome scores completed at 0,1,3,6,9 and 12 months
Secondary outcome [3] 371563 0
Structural outcome assessed using MRI including synovitis, sunchondral oedema, osteophyte formation and cartilage loss/
Timepoint [3] 371563 0
MRI performed at baseline and 12months
Secondary outcome [4] 371828 0
Function assessed using the Short Form Health Survey 36
Timepoint [4] 371828 0
Outcome scores completed at 0,1,3,6,9 and 12 months

Eligibility
Key inclusion criteria
Inclusion criteria:
1. Provision of written informed consent
2. Males and females aged 18 years or over
3. Documented radiological diagnosis of small joint osteoarthritis of the hand (> 2 joints).
4. Primary OA treatment already undertaken, defined as one or more of:
a) analgesia/anti-inflammatory medication, supplements approved by the treating clinician (e.g. glucosamine sulphate),
5. Female subjects of child bearing potential must be willing to comply with the contraceptive requirements of the study. Hormonal contraception by itself will be considered inadequate. All women of child bearing potential must return a negative serum pregnancy test result at Screening and a negative urine pregnancy test before injection of IP. Note, proof of postmenopausal status will be confirmed with the presence of amenorrhea for more than 12 months and a follicle stimulating hormone level of greater than 25 IU/L.
6. Male subjects (and their female partners) must be willing to comply with the contraceptive requirements of the study.
7. Sufficient English skills to communicate well with the PI and site personnel and agree to comply with all the study procedures and requirements.
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
Exclusion criteria:
1. Clinically significant abnormalities in the screening physical examination, vital signs, ECG, or clinical laboratory tests, as determined by the PI. Repeat testing may be performed at the PI’s discretion.
2. History of an atypical pain syndrome.
3. History of infective or inflammatory joint disorders, or suspected infective or inflammatory joint disease
4. History of other musculoskeletal or neurological condition that effects upper limb function.
5. History of significant drug allergies (including penicillin and streptomycin and to any excipients) including a history of anaphylactic reaction (particularly reactions to anesthetic agents).
6. Known allergy or intolerance to bovine meats, products containing bovine serum albumin and/or products derived from bovine sources.
7. Intake of prescribed anticoagulants including warfarin within 21 days prior to Day -1. Low dose (up to 100 mg daily) acetylsalicylic acid is permitted.
8. Female subjects who are pregnant or lactating.
9. Females who are planning a pregnancy at any time during the following 12 months.
10. Major surgery anticipated at any time during the following 12 months.
11. Any contraindication to an X-ray or MRI of the study hand.
12. Unlikely to comply with the clinical study protocol; e.g. uncooperative attitude, inability to return for follow-up visits, and improbability of completing the study.
13. Any other condition, which in the opinion of the PI precludes the subject’s participation in the clinical study.

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
Single group
Other design features
Phase
Not Applicable
Type of endpoint/s
Statistical methods / analysis
All data management and statistical analysis will be performed by a designated and independent biostatistician. All statistical analysis will be performed using the latest available version of statistical analysis software (i.e. SAS® (SAS Institute Inc., Cary, North Carolina, United States of America [USA]), version 9.2 or higher.

Recruitment
Recruitment status
Not yet recruiting
Date of first participant enrolment
Anticipated
22/06/2019
Actual
Date of last participant enrolment
Anticipated
1/03/2020
Actual
Date of last data collection
Anticipated
1/06/2021
Actual
Sample size
Target
10
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
VIC

Funding & Sponsors
Funding source category [1] 302996 0
Commercial sector/Industry
Name [1] 302996 0
Melbourne Stem Cell Centre
Country [1] 302996 0
Australia
Funding source category [2] 302997 0
Commercial sector/Industry
Name [2] 302997 0
Magellan Stem Cells
Country [2] 302997 0
Australia
Primary sponsor type
Commercial sector/Industry
Name
Melbourne Stem Cell Centre
Address
Level 2, 116-118 Thames St
Box Hill Nth 3128
Victoria
Country
Australia
Secondary sponsor category [1] 302964 0
Commercial sector/Industry
Name [1] 302964 0
Magellan Stem Cells
Address [1] 302964 0
Level 2, 116-118 Thames St
Box Hill Nth 3128
Victoria
Country [1] 302964 0
Australia

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 303545 0
Charles Sturt University Human Research Ethics Committee
Ethics committee address [1] 303545 0
Locked Bag 588
Boorooma St
Wagga Wagga
NSW 2678
Ethics committee country [1] 303545 0
Australia
Date submitted for ethics approval [1] 303545 0
30/05/2018
Approval date [1] 303545 0
21/06/2018
Ethics approval number [1] 303545 0
H18117

Summary
Brief summary
This is a proof of concept pilot study to evaluate the safety and tolerability of autologous adipose-derived mesenchymal stem cells (ADMSC) administered intravenously for the treatment of small joint osteoarthritis. A secondary objective will be to assess efficacy with pain and functional assessment over a 12month follow-up period.
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 94038 0
A/Prof Julien Freitag
Address 94038 0
Melbourne Stem Cell Centre
116-118 Thames St
Box Hill North
Vic 3128
Country 94038 0
Australia
Phone 94038 0
+61 392708000
Fax 94038 0
Email 94038 0
[email protected]
Contact person for public queries
Name 94039 0
Julien Freitag
Address 94039 0
Melbourne Stem Cell Centre
116-118 Thames St
Box Hill North
Vic 3128
Country 94039 0
Australia
Phone 94039 0
+61 392708000
Fax 94039 0
Email 94039 0
[email protected]
Contact person for scientific queries
Name 94040 0
Julien Freitag
Address 94040 0
Melbourne Stem Cell Centre
116-118 Thames St
Box Hill North
Vic 3128
Country 94040 0
Australia
Phone 94040 0
+61 392708000
Fax 94040 0
Email 94040 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?

Doc. No.TypeCitationLinkEmailOther DetailsAttachment
2293Informed consent form    377745-(Uploaded-08-06-2019-16-33-45)-Study-related document.pdf



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.