This trial is active, not recruiting.

Condition chronic graft-versus-host disease
Treatment interleukin-2
Phase phase 2
Sponsor Dana-Farber Cancer Institute
Collaborator National Cancer Institute (NCI)
Start date July 2011
End date August 2014
Trial size 41 participants
Trial identifier NCT01366092, 11-149, P01CA142106


Chronic GVHD is a medical condition that may occur after a bone marrow, stem cell or cord blood transplant. The donor's immune system may recognize the your body (the host) as foreign and attempt to 'reject' it. This process is known as graft-versus-host-disease. It is thought that IL-2 may help control chronic GVHD by stopping the donor's immune system from 'rejecting' your body. In this research study, we are looking to see how IL-2 can be used in combination with steroids to treat cGVHD.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Endpoint classification safety/efficacy study
Intervention model single group assignment
Masking open label
Primary purpose treatment
interleukin-2 IL-2
Daily subcutaneous IL-2 (1 x 10^6 IU/m^2/day) for self-administration for 12 weeks followed by 4-week hiatus

Primary Outcomes

To determine the overall response rate of low-dose daily SC IL-2 in steroid-refractory cGVHD
time frame: 3 years

Secondary Outcomes

To determine toxicity of low-dose SC IL-2 therapy
time frame: 3 years
To determine ongoing prednisone use with IL-2 therapy
time frame: 3 years
To assess overall survival, progression-free survival, non-relapse mortality and relapse at 1 year after start of IL-2
time frame: 3 years
To assess the immunologic effects of low-dose daily SC IL-2
time frame: 3 years

Eligibility Criteria

Male or female participants at least 18 years old.

Inclusion Criteria: - Recipient of allogeneic stem cell transplantation with myeloablative or non-myeloablative conditioning regimens - Steroid refractory cGVHD with systemic therapy onset within the prior 6 months - No more than 2 prior lines of cGVHD therapy - Estimated life expectancy > 3 months - Adequate organ function Exclusion Criteria: - Ongoing prednisone requirement > 1 mg/kg/day (or equivalent) - Concurrent use of calcineurin-inhibitors plus sirolimus - History of thrombotic microangiopathy, hemolytic-uremic syndrome or thrombotic thrombocytopenic purpura - Active malignant relapse - Active uncontrolled infection - Uncontrolled cardiac angina or symptomatic congestive heart failure - Organ transplant (allograft) recipient - HIV-positive on combination antiretroviral therapy - Active hepatitis B or C - Pregnant or breast-feeding

Additional Information

Official title A Phase II Trial of Daily Low-Dose Interleukin-2 (IL-2) for Steroid-Refractory Chronic Graft-Versus-Host-Disease
Principal investigator John Koreth, MBBS, DPhil
Description You will give yourself or be given IL-2 daily through an injection under your skin. You should rotate the injection site, if possible. You will do this once every day for 12 weeks. You will then have 4 weeks off of IL-2. During the first 6 weeks of IL-2, you will continue to take steroids without changing the dose your doctor has set for you while you are on IL-2. After 6 weeks of IL-2 therapy, your doctor may reduce the amount of steroids you take. While you are on study, a member of the study team will examine you to evaluate your cGVHD. These assessments may include examination of your skin, joints/muscles, eyes, mouth, lungs and gastrointestinal system. You will have clinic visits for evaluation of toxicity and clinical benefit approximately every 4 weeks. You will also have immunologic assays approximately every 8 weeks. Immunologic assays will measure the effect of IL-2 on immune cells. You will be on the study for about 16 weeks. You may continue on study treatment for longer if you experience a clinical benefit.
Trial information was received from ClinicalTrials.gov and was last updated in June 2014.
Information provided to ClinicalTrials.gov by Dana-Farber Cancer Institute.