Clinical trial NCT01851070: 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
Official 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 TNFα Inhibitor
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Rheumatoid Arthritis
Lead sponsor: Mesoblast, Ltd.
Sponsor class: INDUSTRY
Collaborators: name: PPD Development, LP
First posted: 2013-05-10
Enrollment: 48
Sex: ALL
Minimum age (years): 18
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


Eligibility criteria: 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

Primary outcomes:
measure: Evaluation of the safety of a single IV infusion of allogeneic MPCs compared to placebo at 12 weeks post-infusion
timeFrame: 12 weeks post IV Infusion
Secondary outcomes:
measure: 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
timeFrame: 12 weeks post IV infusion with MPCs
measure: Evaluation of long-term safety and efficacy of a singly IV infusion of allogeneic MPCs in patients with active RA
timeFrame: 52 weeks post IV Infusion
Other outcomes:
Locations:
city: Anniston
country: United States
facility: Pinnacle Research Group
state: Alabama
city: Gilbert
country: United States
facility: Arthrocare Arthritis Care and Research PC
state: Arizona
city: El Cajon
country: United States
facility: Triwest Research Associates
state: California
city: Los Angeles
country: United States
facility: UCLA
state: California
city: Upland
country: United States
facility: Inland Rheumatology Clinical Trials Incorporated
state: California
city: Ocala
country: United States
facility: Ocala Rheumatology Research Center
state: Florida
city: Palm Harbor
country: United States
facility: Arthritis Center
state: Florida
city: Sarasota
country: United States
facility: Sarasota Arthritis Research Center
state: Florida
city: Tampa
country: United States
facility: McIlwain Medical Group
state: Florida
city: Baltimore
country: United States
facility: JHU Arthritis Center Baltimore
state: Maryland
city: Frederick
country: United States
facility: Arthritis Treatment Center
state: Maryland
city: Worcester
country: United States
facility: Reliant Medical Group
state: Massachusetts
city: Rochester
country: United States
facility: Mayo Clinic
state: Minnesota
city: Fair Lawn
country: United States
facility: Office of Ramesh C. Gupta, MD
state: New Jersey
city: Charlotte
country: United States
facility: DJL Clinical Research
state: North Carolina
city: Oklahoma City
country: United States
facility: Health Research of Oklahoma
state: Oklahoma
city: Pittsburgh
country: United States
facility: University of Pittsburgh
state: Pennsylvania
city: Jackson
country: United States
facility: West Tennessee Research Institute
state: Tennessee
city: Houston
country: United States
facility: Accurate Clinical Research
state: Texas
city: San Antonio
country: United States
facility: Texas Arthritis Research Center
state: Texas
city: Camperdown
country: Australia
facility: Royal Prince Alfred Hospital
state: New South Wales
city: Hobart
country: Australia
facility: Southern Clinical Research Pty Ltd
state: Tasmania
city: Malvern
country: Australia
facility: Emeritus Research
state: Victoria
Study officials:
affiliation: Mesoblast, Ltd.
name: Donna Skerrett, MD, MS
role: STUDY_DIRECTOR
References: