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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT01851070




Registration number
NCT01851070
Ethics application status
Date submitted
19/04/2013
Date registered
10/05/2013
Date last updated
26/06/2020

Titles & IDs
Public title
A Multi-center Study a Single IV Infusion of Allogeneic MPCs in Patients With Rheumatoid Arthritis and Incomplete Response to at Least One TNFa Inhibitor
Scientific title
A Double-blind, Randomized, Placebo-controlled, Dose-escalation, Multi-center Study a Single Intravenous Infusion of Allogeneic Mesenchymal Precursor Cells (MPCs) in Patients With Rheumatoid Arthritis and Incomplete Response to at Least One TNFa Inhibitor
Secondary ID [1] 0 0
MSB-RA001
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Rheumatoid Arthritis 0 0
Condition category
Condition code
Musculoskeletal 0 0 0 0
Osteoarthritis
Inflammatory and Immune System 0 0 0 0
Rheumatoid arthritis

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Allogeneic Mesenchymal Precursor Cells
Treatment: Drugs - Normal Saline

Placebo Comparator: Normal Saline Placebo - Placebo will be delivered in 100 mL normal saline administered intravenously over approximately 45 minutes.

Active Comparator: Allogeneic Mesenchymal Precursor Cells - Mesenchymal Precursor Cells (MPCs), either 1.0 or 2.0 million cells/kg, will be delivered in 100 mL normal saline administered intravenously over approximately 45 minutes.


Treatment: Drugs: Allogeneic Mesenchymal Precursor Cells


Treatment: Drugs: Normal Saline


Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Evaluation of the safety of a single IV infusion of allogeneic MPCs compared to placebo at 12 weeks post-infusion - To evaluate the safety, tolerability and feasibility of a single intravenous (IV) infusion of allogeneic mesenchymal precursor cells (MPCs) compared to placebo at 12 weeks post-infusion in the treatment of patients with active rheumatoid arthritis (RA) who have received methotrexate +/- other oral DMARDs for at least 4 months and who have had an incomplete response to at least one course of a TNFa inhibitor.
Overall safety will be based on the overall assessment of AE/SAEs, Vital Signs, Physical Examination, clinical laboratory tests, ECGs and Chest x-ray.
Timepoint [1] 0 0
12 weeks post IV Infusion
Secondary outcome [1] 0 0
Evaluation of the efficacy of a single intravenous infusion of allogeneic MPCs compared with placebo at 12 weeks post-infusion with MPCs or placebo in the treatment of patients with active RA - To assess the efficacy of a single intravenous infusion of allogeneic MPCs compared with placebo at 12 weeks post-infusion with MPCs or placebo in the treatment of patients with active RA who have received methotrexate +/- other DMARDs for at least 4 months and who have had an incomplete response to least one TNFa inhibitor.
Timepoint [1] 0 0
12 weeks post IV infusion with MPCs
Secondary outcome [2] 0 0
Evaluation of long-term safety and efficacy of a singly IV infusion of allogeneic MPCs in patients with active RA - To evaluate the long-term efficacy and safety of allogeneic MPCs over the entire study duration in the treatment of patients with active RA who have received methotrexate +/- other DMARDs for at least 4 months and who have had an incomplete response to at least one TNFa inhibitor
Safety will be assessed according to the following:
Adverse events/serious adverse events ("primary endpoint")
Vital signs
Physical examination
Clinical laboratory tests
Electrocardiogram
Chest x-ray (CXR)
Efficacy will be assessed according to the following:
ACR20/50/70
DAS28 (mean changes from baseline as measured by using hsCRP and ESR)
Mean changes from baseline in all components of the ACR core response criteria
Remissions (as defined in the 2011 Joint Statement of the American College of Rheumatology (ACR) and European League Against Rheumatism (EULAR)
Joint erosion of hands and wrists assessed via x-ray
Patient-reported outcomes
SF36v2
HAQ_DI
Timepoint [2] 0 0
52 weeks post IV Infusion

Eligibility
Key inclusion criteria
- Males and Females ages 18-80 years old

- Active rheumatoid arthritis (RA) disease as per 2010 ACR/EULAR classification criteria
for the diagnosis of RA.

- Must be positive for rheumatoid factor and/or anti-cyclic citrullinated peptide
(anti-CCP3) but without extra-articular disease or functional limitation

- Patient with active RA defined as:

- = 4 tender joint count (TJC) 28 joint count at screening and

- = 4 swollen joint count (SJC) count 28 joint count at screening

- ESR = 28 mm/hr or hsCRP >2.0 mg/L

- Patient has been taking MTX for at least 4 months with dose and route of
administration stable for at least 8 weeks prior to screening

- Patient has had an inadequate response to at least one TNFa inhibitor with last dose
at least 6 weeks prior to screening

- Use of oral DMARD (sulfasalazine, hydroxychloroquine, chloroquine and leflunomide) is
permitted but must be stable for at least 3 months prior to screening
Minimum age
18 Years
Maximum age
80 Years
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- Pregnant women or women who are breastfeeding.

- Other investigational therapy received within 8 weeks or five half-lives (whichever is
longer) prior to Screening (except as in exclusion #13).

- Known or suspected alcohol or drug abuse within three years preceding Screening.

- Autoimmune disease other than RA (such as systemic lupus erythematosus (SLE), mixed
connective tissue disease, scleroderma, polymyositis/dermatomyositis, vasculitis)

- History of or current inflammatory joint disease other than RA (such as tophaceous
gout, reactive arthritis, psoriatic arthritis, ankylosing spondylitis or other
spondyloarthropathy, Lyme disease). Patients primarily diagnosed with osteoarthritis
are excluded.

- Bedridden or confined to a wheelchair or patients with > 3 arthroplasties due to RA.

- History of diagnosed and/or treated malignancy with no evidence of recurrence in past
5 years

- Surgical procedures planned to occur during the trial (these patients may be
rescreened following completion of and recovery from the surgical procedure).

- Use of TNFa inhibitor for treatment of RA at time of screening or within the 6 weeks
prior to screening.

- Prior use of biologic agent for treatment of RA within 6 weeks prior to screening

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 assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,TAS,VIC
Recruitment hospital [1] 0 0
Royal Prince Alfred Hospital - Camperdown
Recruitment hospital [2] 0 0
Southern Clinical Research Pty Ltd - Hobart
Recruitment hospital [3] 0 0
Emeritus Research - Malvern
Recruitment postcode(s) [1] 0 0
2050 - Camperdown
Recruitment postcode(s) [2] 0 0
7000 - Hobart
Recruitment postcode(s) [3] 0 0
3145 - Malvern
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Alabama
Country [2] 0 0
United States of America
State/province [2] 0 0
Arizona
Country [3] 0 0
United States of America
State/province [3] 0 0
California
Country [4] 0 0
United States of America
State/province [4] 0 0
Florida
Country [5] 0 0
United States of America
State/province [5] 0 0
Maryland
Country [6] 0 0
United States of America
State/province [6] 0 0
Massachusetts
Country [7] 0 0
United States of America
State/province [7] 0 0
Minnesota
Country [8] 0 0
United States of America
State/province [8] 0 0
New Jersey
Country [9] 0 0
United States of America
State/province [9] 0 0
North Carolina
Country [10] 0 0
United States of America
State/province [10] 0 0
Oklahoma
Country [11] 0 0
United States of America
State/province [11] 0 0
Pennsylvania
Country [12] 0 0
United States of America
State/province [12] 0 0
Tennessee
Country [13] 0 0
United States of America
State/province [13] 0 0
Texas

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Mesoblast, Ltd.
Address
Country
Other collaborator category [1] 0 0
Commercial sector/Industry
Name [1] 0 0
PPD
Address [1] 0 0
Country [1] 0 0

Ethics approval
Ethics application status

Summary
Brief summary
Study is a double-blind, randomized, placebo controlled, dose escalating study. The primary
objective of this study is to evaluate the safety, tolerability and feasibility of a single
intravenous infusion of allogeneic mesenchymal precursor cells (MPCs) compared to placebo at
12 weeks post-infusion in the treatment of patients with active rheumatoid arthritis (RA) who
have received methotrexate +/- other DMARDs for at least 6 months prior to screening and who
have had an incomplete response to at least one TNF-alpha inhibitor.
Trial website
https://clinicaltrials.gov/show/NCT01851070
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Donna Skerrett, MD, MS
Address 0 0
Mesoblast, Ltd.
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Summary results
Other publications