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

NCT01366092 | Active Not Recruiting Phase 2 Results DMC

• 2 other identifiers: 11-149P01CA142106
• Study Type: interventional
• Target: 35
• Locations: 1 country
• Sites: 3

Brief 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. 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.

Conditions

Chronic Graft-versus-host Disease

Keywords

stem cell transplant; GVHD; bone marrow transplant; cord blood transplant; regulatory T cell; interleukin

Outcome Measures

Primary Outcomes (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.

  Baseline, 6 weeks, and 12 weeks

Secondary Outcomes (5)

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

  12 weeks

• Prednisone Taper With IL-2 Therapy

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

• Overall Survival and Progression-free Survival

  2 years from start of IL-2

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

  16 weeks of study follow-up

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

  16 weeks of study follow-up

Study Arms (1)

Interleukin-2

EXPERIMENTAL

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.

Drug: Interleukin-2

Interventions

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

Also known as: IL-2

Interleukin-2

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Dana-Farber Cancer Institute: lead
• National Cancer Institute (NCI): collaborator
• Prometheus Laboratories: collaborator

Study Sites (3)

Dana-Farber Cancer Institute

Boston, Massachusetts, 02214, United States

Location

Massachusetts General Hospital

Boston, Massachusetts, 02214, United States

Location

Beth Israel Deaconess Medical Center

Boston, Massachusetts, 02215, United States

Location

MeSH Terms

Conditions

Bronchiolitis Obliterans Syndrome

Interventions

Interleukin-2

Condition Hierarchy (Ancestors)

Organizing Pneumonia; Bronchiolitis Obliterans; Bronchiolitis; Bronchitis; Bronchial Diseases; Respiratory Tract Diseases; Lung Diseases, Obstructive; Lung Diseases; Graft vs Host Disease; Immune System Diseases

Intervention Hierarchy (Ancestors)

Interleukins; Cytokines; Intercellular Signaling Peptides and Proteins; Peptides; Amino Acids, Peptides, and Proteins; Lymphokines; Proteins; Biological Factors

Results Point of Contact

• Title: John Koreth, MBBS, DPhil
• Organization: Dana-Farber Cancer Institute

JKoreth@partners.org

617-632-2949

Study Officials

• John Koreth, MBBS, DPhil

  Dana-Farber Cancer Institute

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: June 2, 2011
• First Posted: June 3, 2011
• Study Start: July 1, 2011
• Primary Completion: October 1, 2014
• Study Completion (Estimated): December 1, 2026
• Last Updated: February 20, 2026
• Results First Posted: January 15, 2015

Record last verified: 2026-02

Locations

US (3)