Daily IL-2 for Steroid-Refractory Chronic Graft-versus-Host-Disease

Dana-Farber Cancer Institute

Status and phase: Active, not recruiting; Phase 2

Conditions: Chronic Graft-versus-host Disease

Treatments: Drug: Interleukin-2

Study type: Interventional

Funder types: Other; Industry; NIH

Identifiers

NCT01366092

P01CA142106 (U.S. NIH Grant/Contract)

11-149

Details and patient eligibility

About

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.

Full 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.

Enrollment: 35 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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35 participants in 1 patient group

Interleukin-2

Experimental group

Description:

Each study participant will receive daily subcutaneous IL-2 (1 x 106 IU/m2/day) for self-administration for 12 weeks, followed by a 4-week hiatus. IL-2 will be typically administered on an outpatient basis. After completing the 16 week study (12 weeks of IL-2 study treatment and a mandatory 4 weeks off-IL-2), patients experiencing clinical benefit (complete or partial response; as well as minor response not meeting NIH criteria for partial response) with an acceptable toxicity profile will be permitted to continue extended-duration treatment indefinitely at the discretion of the treating physician.

Treatment:

Drug: Interleukin-2