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A Multi-center Study a Single IV Infusion of Allogeneic MPCs in Patients With Rheumatoid Arthritis and Incomplete Response to at Least One TNFα Inhibitor

Phase 2
Completed
Conditions
Rheumatoid Arthritis
Interventions
Registration Number
NCT01851070
Lead Sponsor
Mesoblast, Ltd.
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.

Detailed Description

Not available

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
48
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 TNFα 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

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 TNFα 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

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Allogeneic Mesenchymal Precursor CellsNormal SalineMesenchymal 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.
Normal Saline PlaceboAllogeneic Mesenchymal Precursor CellsPlacebo will be delivered in 100 mL normal saline administered intravenously over approximately 45 minutes.
Primary Outcome Measures
NameTimeMethod
Evaluation of the safety of a single IV infusion of allogeneic MPCs compared to placebo at 12 weeks post-infusion12 weeks post IV 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 TNFα 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.

Secondary Outcome Measures
NameTimeMethod
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 RA12 weeks post IV infusion with MPCs

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 TNFα inhibitor.

Evaluation of long-term safety and efficacy of a singly IV infusion of allogeneic MPCs in patients with active RA52 weeks post IV Infusion

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 TNFα 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

Trial Locations

Locations (23)

University of Pittsburgh

🇺🇸

Pittsburgh, Pennsylvania, United States

Arthrocare Arthritis Care and Research PC

🇺🇸

Gilbert, Arizona, United States

UCLA

🇺🇸

Los Angeles, California, United States

Pinnacle Research Group

🇺🇸

Anniston, Alabama, United States

Inland Rheumatology Clinical Trials Incorporated

🇺🇸

Upland, California, United States

Triwest Research Associates

🇺🇸

El Cajon, California, United States

Sarasota Arthritis Research Center

🇺🇸

Sarasota, Florida, United States

McIlwain Medical Group

🇺🇸

Tampa, Florida, United States

Arthritis Center

🇺🇸

Palm Harbor, Florida, United States

Ocala Rheumatology Research Center

🇺🇸

Ocala, Florida, United States

JHU Arthritis Center Baltimore

🇺🇸

Baltimore, Maryland, United States

Reliant Medical Group

🇺🇸

Worcester, Massachusetts, United States

DJL Clinical Research

🇺🇸

Charlotte, North Carolina, United States

Office of Ramesh C. Gupta, MD

🇺🇸

Fair Lawn, New Jersey, United States

West Tennessee Research Institute

🇺🇸

Jackson, Tennessee, United States

Mayo Clinic

🇺🇸

Rochester, Minnesota, United States

Accurate Clinical Research

🇺🇸

Houston, Texas, United States

Health Research of Oklahoma

🇺🇸

Oklahoma City, Oklahoma, United States

Texas Arthritis Research Center

🇺🇸

San Antonio, Texas, United States

Royal Prince Alfred Hospital

🇦🇺

Camperdown, New South Wales, Australia

Emeritus Research

🇦🇺

Malvern, Victoria, Australia

Southern Clinical Research Pty Ltd

🇦🇺

Hobart, Tasmania, Australia

Arthritis Treatment Center

🇺🇸

Frederick, Maryland, United States

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