A Study of the Outcomes of Natalizumab plus Standard Steroid Treatment for High Risk Acute Graft-Versus-Host Disease

Overview

  • Study type

    Interventional
  • Study phase

    II
  • 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: 16-000650
    NCT ID: NCT02133924
    Sponsor Protocol Number: UMCC 2014.051

About this study

The purpose of this study is to assess the safety and effectiveness of combining the study drug Natalizumab (Tysabri®) with the standard use of steroid treatment, as a new treatment for acute graft versus host disease (acute GVHD). GVHD is the most common serious complication, after bone marrow transplant. GVHD occurs when the donor cells (the graft), treat the recipient's body as "foreign" and attack the cells in the recipient's body. During this immune system response, donor cells damage body tissues, such as the skin, liver, stomach, and/or intestines. Acute GVHD can be severe and potentially fatal to the transplant recipient.

 

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:

  • New onset acute GVHD Ann Arbor score 2 or 3 following allogeneic BMT. Any clinical severity (Glucksberg grade I-IV) is eligible. Patients with prior or existing diagnosis of GVHD without any treatment are eligible. Patients given only topical corticosteroids for skin GVHD are eligible.
  • Any donor type (e.g., related, unrelated) or stem cell source (bone marrow, peripheral blood, cord blood). Recipients of non-myeloablative and myeloablative transplants are eligible.
  • No prior systemic treatment for acute GVHD except for a maximum of 3 days of prednisone (or IV methylprednisolone) ≤2 mg/kg/day. Topical skin steroid treatment, non-absorbable oral steroid treatment for GI GVHD, and resumption of GVHD prophylaxis agents (e.g., calcineurin inhibitors) are permissible. Patients enrolled in BMT CTN 1501 who randomized to sirolimus are also eligible.
  • Age 18 years or older.
  • Direct bilirubin must be <2 mg/dL unless the elevation is known to be due to Gilbert syndrome or aGVHD within 3 days of enrollment.
  • ALT/SGPT and AST/SGOT must be <5 x the upper limit of the normal range within 3 days of enrollment unless the elevation is due to liver GVHD.
  • If the patient is a woman of child-bearing potential, the patient and their sexual partner must agree to practice effective contraception.
  • Written informed consent from patient or legal representative.
  • Biopsy of acute GVHD target organ is strongly recommended but not required. Enrollment should not be delayed for biopsy or pathology results. Patients who do not enroll within 3 days of initiation of systemic steroid treatment for acute GVHD are not permitted to participate.

Exclusion Criteria:

  • Patients with malignancy that is suspected or proven to have progressed, relapsed, or be persistent since BMT.
  • Uncontrolled active infection.
  • Patients with chronic GVHD only. Patients diagnosed with overlap syndrome are still eligible.
  • History of or current diagnosis of progressive multifocal leukoencephalopathy (PML).
  • Pregnant or nursing (lactating) women.
  • Use of other drugs for the treatment of acute GVHD.
  • Steroid therapy for indications other than GVHD at doses >0.5 mg/kg/d of methylprednisolone or equivalent within 7 days prior to initiation of GVHD treatment.
  • Patients on dialysis.
  • Patients requiring ventilator support.
  • Investigational agent within 30 days of enrollment without approval from the Sponsor-Investigator (PI).
  • History of allergic reaction to natalizumab.

 

Participating Mayo Clinic locations

Study statuses change often. Please contact us for help.

Mayo Clinic Location Status Contact

Rochester, Minn.

Mayo Clinic principal investigator

William Hogan, M.B., B.Ch.

Open for enrollment

Contact information:

Brenda Ingalls Hanson

(507)538-2918

IngallsHanson.Brenda@mayo.edu

More information

Publications

Publications are currently not available

Study Results Summary

Not yet available

Supplemental Study Information

Not yet available

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CLS-20266934

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