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Chronic Graft-versus-host DiseasePhase 2NCT01366092

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

A Phase 2 study testing Interleukin-2 in people with Chronic Graft-versus-host Disease, run by Dana-Farber Cancer Institute at 3 sites in United States.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial summary

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. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Chronic Graft-versus-host Disease
What is being tested?
Interleukin-2
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
35 participants
Where
3 sitesUnited States
Study dates
Jul 2011to Dec 2026
Phase
Phase 2
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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

Who cannot join

  • 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
Show all 10Show fewer
  • Organ transplant (allograft) recipient
  • HIV-positive on combination antiretroviral therapy
  • Active hepatitis B or C
  • Pregnant or breast-feeding

Trial locations and contacts

3 sites in 1 country.

United States3 sites

  • Dana-Farber Cancer Institute

    Boston, Massachusetts, 02214

  • Massachusetts General Hospital

    Boston, Massachusetts, 02214

  • Beth Israel Deaconess Medical Center

    Boston, Massachusetts, 02215

Other trials in United States

About this study

Official title: A Phase II Trial of Daily Low-Dose Interleukin-2 (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.

Read the detailed 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.

Being tested
Interleukin-2
Keywords
stem cell transplant, GVHD, bone marrow transplant, cord blood transplant, regulatory T cell, interleukin
Medical subject headings
Bronchiolitis Obliterans Syndrome

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Interleukin-2Drug

    Also called 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

Study groups in NCT01366092
Study groupRoleWhat they receive
Interleukin-2ExperimentalEach 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.
Receives: Drug: Interleukin-2

What is being measured

The primary outcome is the main question the study is designed to answer.

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

    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.

    Measured: Baseline, 6 weeks, and 12 weeks

Secondary outcomes (5)

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

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

    Measured: 12 weeks

  2. Prednisone Taper With IL-2 Therapy

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

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

  3. Overall Survival and Progression-free Survival

    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.

    Measured: 2 years from start of IL-2

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

    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.

    Measured: 16 weeks of study follow-up

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

    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.

    Measured: 16 weeks of study follow-up

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted3 Jun 2011 (estimated)
  2. Study startJul 2011
  3. Primary completionOct 2014
  4. Study completionDec 2026 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
35 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01366092
Sponsor's protocol ID
11-149
Other IDs
P01CA142106
Status last verified
Feb 2026
First submitted
2 Jun 2011
Last updated
20 Feb 2026
Data monitoring committee
Yes

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Interleukin-2 in people with Chronic Graft-versus-host Disease, run by Dana-Farber Cancer Institute at 3 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 5 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: under way, not looking for participants, as of the sponsor's last update on 20 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 3 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 15 Jan 2015). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Overall Response Rate of Low-dose Daily SC IL-2 in Steroid-refractory cGVHD (time frame: Baseline, 6 weeks, and 12 weeks). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01366092.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Feb 2026.

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