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NCT Number: NCT01366092

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

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.

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This study is active but is not currently recruiting participants.

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Key information

Conditions

Chronic Graft-versus-host Disease Bronchial Diseases Bronchiolitis Bronchiolitis Obliterans Bronchiolitis Obliterans Syndrome Bronchitis Graft vs Host Disease Immune System Diseases Lung Diseases Lung Diseases, Obstructive Organizing Pneumonia Respiratory Tract Diseases

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Beth Israel Deaconess Medical Center, Boston, Massachusetts, United States

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Interleukin-2

Drug

Daily subcutaneous IL-2 (1 x 10^6 IU/m^2/day) for self-administration for 12 weeks followed by 4-week hiatus

Other names: IL-2

Primary outcomes

  1. Overall Response Rate of Low-dose Daily SC IL-2 in Steroid-refractory cGVHD

    Time frame: Baseline, 6 weeks, and 12 weeks

    Participants were evaluated according to the cGVHD NIH Consensus criteria at baseline, 6 weeks, and 12 weeks on study. Per cGVHD NIH Consensus criteria, cGVHD involved organ systems are given a grade 0-3 and an overall cGVHD score, from 0-10, is given. Complete Response is defined as resolution of all reversible manifestations in each organ or site of cGVHD. A partial response is defined as an improvement in measure at least one organ or site, or decrease in global ratings by at least a 2-point change on the 10-point scale, without progression measured at any other organ or site. Non-responders have no change in cGVHD meeting criteria for either partial response or disease progression. Progressive disease is defined as an increase in organ or site scales (1-point change on a 3-point scale) or 2- to 3-point increase on the global cGVHD ratings. Clinical worsening of cGVHD is not synonymous with progressive cGVHD per NIH criteria.

Secondary outcomes

  1. Toxicity of 12-week Course of Low-dose SC IL-2 Therapy

    Time frame: 12 weeks

    Participants were evaluated at clinical visits for toxicities related to IL-2 throughout their 12-week treatment course

  2. Prednisone Taper With IL-2 Therapy

    Time frame: End of treatment after 16 weeks or most recent follow-up date for patients on extended

    Participants had their steroid dose assessed at weeks 6, 12,16, and every 8 weeks while on extended duration IL-2 therapy.

  3. Overall Survival and Progression-free Survival

    Time frame: 2 years from start of IL-2

    Overall survival (OS) and progression-free survival (PFS) were calculated using the Kaplan-Meier method. OS was defined as from the study entry to death from any cause. Patients who were alive or lost to follow-up were censored at the time last seen alive. PFS was defined from the study entry to disease relapse or progression or death from any cause, whichever occurred first.

  4. Immunologic Effects of Low-dose Daily SC IL-2: Treg Cell Counts

    Time frame: 16 weeks of study follow-up

    Blood samples were collected throughout the patient's 12 weeks of IL-2 treatment and after the 4 week hiatus. The CD4+CD25+FOXP3+ regulatory T cells (Treg) counts were measured.

  5. Immunologic Effects of Low-dose Daily SC IL-2: Treg/Tcon Ratio

    Time frame: 16 weeks of study follow-up

    Blood samples were collected throughout the patient's 12 weeks of IL-2 treatment and after the 4 week hiatus. The ratio between CD4+CD25+FOXP3+ regulatory T cells (Treg) and CD4 conventional T cell (Tcon) counts were measured.

Sponsors and collaborators

Lead sponsor

Dana-Farber Cancer Institute

Other

Collaborators

  • National Cancer Institute (NCI)
  • Prometheus Laboratories

Registry information

Official study title

A Phase II Trial of Daily Low-Dose Interleukin-2 (IL-2) for Steroid-Refractory Chronic Graft-Versus-Host-Disease

Important dates

Study start
2011
Primary completion
2014
Study completion
2026
First posted
Jun 3, 2011
Registry last updated
Feb 20, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

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This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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