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Trial registered on ANZCTR


Registration number
ACTRN12615000439549
Ethics application status
Approved
Date submitted
22/04/2015
Date registered
7/05/2015
Date last updated
7/07/2017
Type of registration
Prospectively registered

Titles & IDs
Public title
The evaluation of allogeneic adipose derived mesenchymal stem cells as a treatment for symptomatic knee osteoarthritis, specifically evaluating safety, effects on knee pain, function and structure, and quality of life in knee osteoarthritis patients.
Scientific title
A Phase 1 Randomised, Double Blind, Placebo Controlled, Single Ascending Dose Study to Evaluate the Safety, Tolerability and Preliminary Efficacy of Intra-articular Progenza (PRG) in Adults with Symptomatic Knee Osteoarthritis
Secondary ID [1] 286520 0
Nil known
Universal Trial Number (UTN)
Trial acronym
The STEP Trial (Safety, Tolerability and Efficacy of Progenza)
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Osteoarthritis 294763 0
Condition category
Condition code
Musculoskeletal 295044 295044 0 0
Osteoarthritis

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Progenza (PRG) is composed of in vitro expanded mesenchymal stem cells (MSCs) derived from human donor adipose tissue, frozen in vials ready to inject. PRG will be administered in a single, once only, intra-articular injection into the knee of PRG. Two doses of PRG are being used in this trial. Patients will be randomised to receive a single injection into the knee of either 3.9 million cells or 6.7 million cells or placebo. Participants will have follow up assessments out to 12 months post injection.
Intervention code [1] 291627 0
Treatment: Other
Comparator / control treatment
The placebo is vehicle control of cell culture media, administered as a single, once only, intra-articular injection into the knee. Participants will have follow up assessments out to 12 months post injection.
Control group
Placebo

Outcomes
Primary outcome [1] 294809 0
To evaluate the safety of PRG administered via a single intra-articular injection in the knees adults with Kellgren-Lawrence grade 1, 2 or 3 knee osteoarthritis. The outcome will be measured by the incidence and nature of adverse events.
Timepoint [1] 294809 0
Adverse events are captured and assessed from the time of informed consent onwards, however post-treatment safety is specifically assessed at visits on Day 1, 7, 14, 28 and then at months 1, 3, 6, 9 and 12.
Primary outcome [2] 294917 0
The safety of PRG will be measured by clinical laboratory evaluations of routine biochemistry and haematology tests.
Timepoint [2] 294917 0
Laboratory evaluations are done at Day 1, and 28, then at Month 6, and 12.
Primary outcome [3] 294918 0
The safety of PRG will be measured by physical assessments, specifically physical examination and vital signs.
Timepoint [3] 294918 0
Physical examination and vital signs are done at all clinic visits, (Day 1, 7, and 28, then Months 3, 6, 9 and 12).
Secondary outcome [1] 314109 0
To investigate the effect of PRG on study knee pain and function using a VAS pain score and the Western Ontario McMaster Universities Arthritis Index (WOMAC)
Timepoint [1] 314109 0
Assessment of knee pain and function will be done at Days 1 and 28, then Months 3, 6, 9 and 12.
Secondary outcome [2] 314345 0
To investigate the effect of PRG on quality of life using the AQoL-4D questionnaire
Timepoint [2] 314345 0
Quality of life is assessed at Days 1 and 28, then Months 3, 6, 9 and 12.
Secondary outcome [3] 314346 0
To investigate the effect of PRG on study knee joint structures using magnetic resonance imaging (MRI)
Timepoint [3] 314346 0
Assessment of knee joint structures via MRI will be done at Screening and 12 months.
Secondary outcome [4] 314347 0
To investigate the effect of PRG on osteoarthritis biomarkers in serum and urine, specifically MIF, HA, CTX-I, C2C, CTX-II
Timepoint [4] 314347 0
Assessment of biomarkers will be done at Screening, Day 1 (pre-dose) and then at 3, 6, 9, and 12 months.
Secondary outcome [5] 314348 0
To investigate the effect of PRG on activity levels using a fitbit (optional participation)
Timepoint [5] 314348 0
The fitbit will be used to collect activity data for the 7 days immediately prior to each clinic visit.
Secondary outcome [6] 314556 0
Additional Primary Outcome:
The tolerability of PRG administered via a single intra-articular injection will be measured by the incidence and nature of adverse events.
Timepoint [6] 314556 0
Adverse events are captured and assessed from the time of informed consent onwards, however post-treatment safety is specifically assessed at visits on Day 1, 7, 14, 28 and then at months 1, 3, 6, 9 and 12.

Eligibility
Key inclusion criteria
Patients will be eligible to participate if they:
- are between 40 and 65 years of age inclusive
- have Kellgren-Lawrence grade 1, 2 or 3 symptomatic knee osteoarthritis.
- have moderate to severe knee pain associated with OA
Minimum age
40 Years
Maximum age
65 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
Patients will not be eligible to participate if they:
- have OA in the non-study knee that has worse pain than the study knee
- have had joint surgery in the past 3 years
- have major mechanical issues in the study knee
- have significant OA in other major joints

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled 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
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Date of first participant enrolment
Anticipated
8/05/2015
Actual
24/07/2015
Date of last participant enrolment
Anticipated
1/04/2016
Actual
29/03/2016
Date of last data collection
Anticipated
7/04/2017
Actual
24/03/2017
Sample size
Target
20
Accrual to date
Final
20
Recruitment in Australia
Recruitment state(s)
NSW

Funding & Sponsors
Funding source category [1] 291098 0
Commercial sector/Industry
Name [1] 291098 0
Regeneus Ltd
Country [1] 291098 0
Australia
Primary sponsor type
Commercial sector/Industry
Name
Regeneus Ltd
Address
25 Bridge Street
Pymble NSW 2073
Country
Australia
Secondary sponsor category [1] 289774 0
None
Name [1] 289774 0
Address [1] 289774 0
Country [1] 289774 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 293422 0
Bellberry HREC
Ethics committee address [1] 293422 0
129 Glen Osmond Rd
Eastwood SA 5063
Ethics committee country [1] 293422 0
Australia
Date submitted for ethics approval [1] 293422 0
11/03/2015
Approval date [1] 293422 0
16/04/2015
Ethics approval number [1] 293422 0
2015-03-133

Summary
Brief summary
This study examines the safety and tolerability of Progenza, a stem cell product derived from a single donor, for the treatment of knee osteoarthritis. Progenza is injected once into the knee. There is a 1 in 5 chance of receiving placebo, which contains no stem cells. The study will investigate the effect of Progenza on knee pain, function and cartilage integrity. Quality of life and activity level will also be examined over the course of the study.
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 56478 0
Dr Donald Kuah
Address 56478 0
Sydney Sportsmed Specialists
Park House
Level 3, 187 Macquarie Street
Sydney NSW 2000
Country 56478 0
Australia
Phone 56478 0
+61 2 9231 0102
Fax 56478 0
Email 56478 0
[email protected]
Contact person for public queries
Name 56479 0
Carolyn Koch
Address 56479 0
Sydney Sportsmed Specialists
Park House
Level 3, 187 Macquarie Street
Sydney NSW 2000
Country 56479 0
Australia
Phone 56479 0
+61 2 9231 0102
Fax 56479 0
Email 56479 0
[email protected]
Contact person for scientific queries
Name 56480 0
Janet Wilson
Address 56480 0
Regeneus Ltd
25 Bridge Street
Pymble NSW 2073
Country 56480 0
Australia
Phone 56480 0
+61 2 9499 8010
Fax 56480 0
Email 56480 0
[email protected]

No information has been provided regarding IPD availability


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
SourceTitleYear of PublicationDOI
EmbaseSafety, tolerability and efficacy of intra-articular Progenza in knee osteoarthritis: A randomized double-blind placebo-controlled single ascending dose study.2018https://dx.doi.org/10.1186/s12967-018-1420-z
EmbaseInsights into the present and future of cartilage regeneration and joint repair.2022https://dx.doi.org/10.1186/s13619-021-00104-5
N.B. These documents automatically identified may not have been verified by the study sponsor.