Clinical Trial: Phase II Trial of Natalizumab (Tysabri®) Plus Prednisone for Initial Therapy of Acute Graft Versus Host Disease (aGVHD) of the Gastrointestinal Tract

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: Phase II Trial of Natalizumab (Tysabri®) Plus Prednisone for Initial Therapy of Acute Graft Versus Host Disease (aGVHD) of the Gastrointestinal Tract

Brief Summary: This research study is a Phase II clinical trial, which tests the safety and effectiveness of an investigational drug Natalizumab in treating Acute Graft versus Host Disease in the Gastrointestinal Tract.

Detailed Summary:

Natalizumab is a drug that was initially discovered as a treatment for autoimmune conditions. Natalizumab has been approved for use in patients with Multiple Sclerosis and Crohn's disease. In these diseases, the drug works to inhibit dysfunctional immune cells that are responsible for the symptoms seen in these diseases. Acute graft versus host disease is caused by a similar dysfunction of immune cells; Natalizumab is thought to inhibit these immune cells, similarly to how it does in Multiple Sclerosis and Crohn's disease. In this research study,the investigators are looking to see whether Natalizumab provides additional benefit to patients receiving standard treatment for acute graft versus host disease of the gastrointestinal tract.

Participants who fulfill eligibility criteria will be entered into the trial to receive Natalizumab.

  • Participant will receive a dose of the natalizumab through intravenous infusion. Participants may receive a second dose at Day 28 if they experience a partial response or very good partial response.
  • Scheduled Physical Examination at screening, during the week of first dose and at 28 days, 56 days, 100 days, 180 days and one year.

Sponsor: Dana-Farber Cancer Institute

Current Primary Outcome: GVHD-free survival rate [ Time Frame: Day 56 ]

GVHD free survival is defined as achieving complete response without death or relapse or requiring secondary immunosuppressive therapy . Proportions will be reported descriptively. Overall survival (OS) and GVHD-free survival will be assessed using the Kaplan-Meier method.


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • GVHD Response Rate [ Time Frame: 28 Days, 56 Days ]
    Proportions will be reported descriptively. Overall survival (OS) and GVHD-free survival will be assessed using the Kaplan-Meier method.
  • GI aGVHD Response Rate [ Time Frame: Day 28, Day 56 ]
    Proportions will be reported descriptively. Overall survival (OS) and GVHD-free survival will be assessed using the Kaplan-Meier method.
  • Overall Survival Rate [ Time Frame: 100 Days, 180 Days, and 1 year ]
  • Rate of GVHD flares [ Time Frame: by Day 28, ]
    Incidence of GVHD flares requiring therapy after initial CR or PR by day 28 after the first dose of natalizumab is administered.
  • Steroid dose at day 28, day 56, day 100, day 180 and one year after the first dose of Natalizumab is administered. [ Time Frame: Day 28, 56, 100, 180 and One year ]
    Steroid dose at day 28, day 56, day 100, day 180 and one year after the first dose of Natalizumab is administered.
  • Rate of systemic infections [ Time Frame: Day 56 and Day 180 ]
    Incidence of systemic infections at day 56 and day 180 after the first dose of Natalizumab is administered.


Original Secondary Outcome: Same as current

Information By: Dana-Farber Cancer Institute

Dates:
Date Received: June 25, 2014
Date Started: January 2015
Date Completion: March 2020
Last Updated: April 3, 2017
Last Verified: April 2017