A Study of the Safety of Joint Injected Sc-rAAV2.5IL-1Ra (a Virus Carried Gene Therapy) in Patients with Moderate Knee Osteoarthritis

Overview

  • Study type

    Interventional
  • Study phase

    I
  • Study IDs

  • Describes the nature of a clinical study. Types include:

    • Observational study — observes people and measures outcomes without affecting results.
    • Interventional study (clinical trial) — studies new tests, treatments, drugs, surgical procedures or devices.
    • Medical records research — uses historical information collected from medical records of large groups of people to study how diseases progress and which treatments and surgeries work best.
  • During the early phases (phases 1 and 2), researchers assess safety, side effects, optimal dosages and risks/benefits. In the later phase (phase 3), researchers study whether the treatment works better than the current standard therapy. They also compare the safety of the new treatment with that of current treatments. Phase 3 trials include large numbers of people to make sure that the result is valid. There are also less common very early (phase 0) and later (phase 4) phases. Phase 0 trials are small trials that help researchers decide if a new agent should be tested in a phase 1 trial. Phase 4 trials look at long-term safety and effectiveness, after a new treatment has been approved and is on the market.

  • Site IRB
    • Rochester, Minnesota: 15-007542
    NCT ID: NCT02790723
    Sponsor Protocol Number: 15-007542

About this study

The purpose of this study is to determine the safety of sc-rAAV2.5IL-1Ra, a gene therapy carried by a virus and delivered by injection into the knee joint of patients with moderate osteoarthritis of the knee.

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. If you need assistance understanding the eligibility criteria, please contact the study team.

See eligibility criteria

Inclusion Criteria

  • Age 18-65 years
  • Males and females
  • Has moderate Osteoarthritis of the knee
  • Persistent symptoms, despite standard NSAID use
  • Normal limits for the following
    • Complete blood count
    • Prothrombin Time
    • Activated partial thromboplastin time
    • Blood chemistry (Glucose, Na, K, Cl, CO2, BUN, creatinine, Ca, PO4, magnesium, uric acid)
    • Liver function tests (amylase, total bilirubin, alkaline phosphate, GGT, AST, ALT, total protein, albumin)
  • Able and willing to return to the Mayo Clinic for follow-up visits, as required by this study
  • Able to undergo MRI of the knee
  • Should be able to give appropriate consent or have an appropriate representative available to do so
  • Should have failed a three-month trial of a minimum of two conservative therapies before being considered for this trial, including
    • Activity modification
    • Weight loss
    • Physical therapy
    • Anti-inflammatory or injection therapy

Exclusion Criteria

  • Pregnant, or currently breast-feeding
  • Ongoing infectious disease including HIV, HTLV, hepatitis, syphilis or tuberculosis positive
  • Individuals who have OA as part of another syndrome (e.g. Ehler's Danlos, Stickler syndrome, etc.)
  • Has systemic, rheumatic or inflammatory disease of the knee or chondrocalcinosis, hemochromatosis, inflammatory arthritis, arthropathy of the knee associated with juxta-articular Paget's disease of the femur or tibia, ochronosis, hemophilic arthropathy, infectious arthritis, Charcot's knee joint, villonodular synovitis, and synovial chondromatosis
  • Has clinically significant cardiovascular, renal, hepatic, endocrine disease, diabetes, cancer, autoimmune diseases
  • Has had a serious infection or major operation within 30 days of enrollment
  • Has a history of psychiatric disease or recent history of alcoholism or drug addiction.
  • Currently taking immunosuppressant medications
  • Anticipated major surgery during the study period
  • Individuals involved in another protocol, or have been treated under one within the last 3 months
  • Intra-articular therapy in the index knee within the previous 6 months
  • Surgery to the target knee within 6 months prior to screening
  • Surgery to other weight bearing joints if it will interfere with knee assessments
  • Prior articular transplant procedures
  • Prior reconstruction surgery to the target knee within 12 months
  • X-ray findings of acute fractures
  • Known severe loss of bone density, and/or severe bone or joint deformity in the target knee
  • Knee pain associated with patella-femoral arthritis or chondromalacia in the target knee
  • Significant target knee infection or overlying skin disorder/infection within the previous 6 months prior to study enrollment
  • Requires a cane or other assistive device for walking
  • Symptomatic OA of the hips, spine or ankle if it would interfere with the evaluation of the target knee
  • History of documented nerve damage in the affected limb, or vascular insufficiency
  • Condition requiring use of systemic steroids
  • Coagulation disorder
  • Patients with unstable knees
  • Temperature above 99.5 F
  • Elevated liver transaminases
  • Identification as a member of a vulnerable population
  • BMI greater than 40
  • History of allergy to local anesthetics
  • Currently taking anti-rheumatic disease medication (including methotrexate or other antimetabolites) within the 3 months prior to entry in the study

Participating Mayo Clinic locations

Study statuses change often. Please contact us for help.

Mayo Clinic Location Status Contact

Rochester, Minn.

Mayo Clinic principal investigator

Christopher Evans, Ph.D.

Contact us for the latest status

Contact information:

Tyson Scrabeck

(507)538-1016

Scrabeck.Tyson@mayo.edu

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CLS-20258269

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