Efalizumab in Treating Patients With Graft-Versus-Host Disease of the Skin That Did Not Respond to Previous Steroids
RATIONALE: Efalizumab may be an effective treatment for graft-versus-host disease of the skin caused by a donor stem cell transplant.
PURPOSE: This clinical trial is studying the side effects and how well efalizumab works in treating patients with graft-versus-host disease of the skin that did not respond to previous steroids.
|Study Design:||Endpoint Classification: Safety Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Supportive Care
|Official Title:||Weekly Subcutaneous Efalizumab for the Treatment of Steroid Refractory Graft-Versus-Host Disease of the Skin and Liver|
- Number of subjects experiencing adverse events [ Time Frame: 120 days ] [ Designated as safety issue: Yes ]
The primary objective of this exploratory study is to evaluate the general tolerability of efalizumab in patients suffering from steroid refractory GVHD
Subjects will be evaluated for drug toxicity each visit. Toxicity will be graded using the NCTCAE common criteria.
- Feasibility of digital imaging [ Time Frame: 120 days ] [ Designated as safety issue: No ]To study the feasibility of digital imaging to objectively quantify cutaneous GVHD.
- Feasibility of serial skin biopsies [ Time Frame: 57 days ] [ Designated as safety issue: No ]To evaluate the feasibility of serial skin biopsies to monitor disease response to efalizumab therapy in patients with cutaneous GVHD.
- Overall complete response rate [ Time Frame: 57 days ] [ Designated as safety issue: No ]To assess the overall complete response rate on study days 29 and 57 after 4 and 8 doses of weekly subcutaneous efalizumab.
|Study Start Date:||May 2007|
|Study Completion Date:||October 2008|
|Primary Completion Date:||June 2008 (Final data collection date for primary outcome measure)|
All patients on study will receive a total of 8 injections of efalizumab
Efalizumab will be administered as a subcutaneous injection once a week for 8 weeks (total of 8 doses). First efalizumab injection will be dosed at 0.7mg/kg. Subsequent weekly injections given on days 8-50 will be dosed at
Other Name: Raptiva
- Assess the general safety of efalizumab in patients with cutaneous graft-vs-host disease (GVHD).
- Study the feasibility of digital imaging to objectively quantify cutaneous GVHD.
- Evaluate the feasibility of serial skin biopsies to monitor disease response to efalizumab in patients with cutaneous GVHD.
- Assess the overall complete response rate in patients treated with this drug.
- Assess the overall cutaneous response rate (complete cutaneous response rate and partial cutaneous response rate) in patients treated with this drug.
- Assess the overall hepatic response rate (complete hepatic response rate and partial hepatic response rate) in patients treated with this drug.
- Assess the duration of any responses observed.
- Assess the effect of this drug on overall patient survival.
- Use the preliminary efficacy and toxicity data collected in this small exploratory study to decide on the appropriateness of a larger, subsequent phase II trial to more formally assess toxicity and efficacy of this drug in this patient population.
- Collect pharmacokinetic data on this drug in these patients.
OUTLINE: Patients receive efalizumab subcutaneously once weekly for 8 weeks (total of 8 doses).
Digital photographs of body regions are taken for determination of disease involved body surface area. Skin biopsies are obtained before and after treatment and analyzed for LFA-1, ICAM-1, CD4, CD8, and possibly CD20 by immunohistochemistry.
After completion of study therapy, patients are followed at 1 and 9 weeks.
|United States, North Carolina|
|Lineberger Comprehensive Cancer Center at University of North Carolina - Chapel Hill|
|Chapel Hill, North Carolina, United States, 27599-7295|
|Principal Investigator:||Thomas C. Shea, MD||UNC Lineberger Comprehensive Cancer Center|