Completed

A Phase II Clinical Trial to Evaluate the Safety and Efficacy of Sirolimus for Secondary Treatment of Chronic Graft-versus-Host Disease

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What is being tested

sirolimus

Drug
Who is being recruted

Bronchiolitis Obliterans Syndrome
+8

+ Organizing Pneumonia
+ Bronchial Diseases
See all eligibility criteria
How is the trial designed

Supportive Care Study

Phase 2
Interventional
Study Start: April 2002
See protocol details

Summary

Principal SponsorFred Hutchinson Cancer Center
Last updated: January 13, 2026
Sourced from a government-validated database.Claim as a partner
Study start date: April 1, 2002Actual date on which the first participant was enrolled.

PRIMARY OBJECTIVES: I. To assess the safety of sirolimus administered at a dose which provides steady-state, whole blood trough levels of 5-10 ng/mL in patients with chronic GVHD. II. To determine whether administration of sirolimus provides benefit for patients with chronic GVHD that has not responded adequately to previous systemic treatment. OUTLINE: Patients receive sirolimus orally (PO) once daily (QD). Patients continue to receive prednisone and cyclosporine or tacrolimus at the discretion of the managing physician. After completion of study treatment, patients are followed up periodically.

Official TitleA Phase II Clinical Trial to Evaluate the Safety and Efficacy of Sirolimus for Secondary Treatment of Chronic Graft-versus-Host Disease 
NCT00079183
Principal SponsorFred Hutchinson Cancer Center
Last updated: January 13, 2026
Sourced from a government-validated database.Claim as a partner

Protocol

This section provides details of the study plan, including how the study is designed and what the study is measuring.
Design Details
44 patients to be enrolledTotal number of participants that the clinical trial aims to recruit.
Supportive Care Study
These studies explore ways to improve comfort and daily life for people living with a condition. They may focus on easing symptoms, reducing treatment side effects, or supporting overall well-being.

How participants are assigned to different groups/arms
In this clinical study, all participants receive the same treatment. Since there is only one group, there is no need for randomization or assignment to different arms. This type of study is often used to test a new treatment without comparing it to another.

Other Ways to Assign Participants
Randomized allocation: Participants are assigned randomly, like flipping a coin, to ensure fairness and reduce bias.
Non-randomized allocation: Participants are assigned based on specific factors, such as their medical condition or a doctor's decision.

How treatments are given to participants
In this study, all participants receive the same treatment. This approach is often used to evaluate the effects of a single intervention without comparing it to another.

Other Ways to Assign Treatments
Parallel assignment: Participants are split into separate groups, each receiving a different treatment.
Cross-over assignment: Participants switch between treatments during the study.
Factorial assignment: Participants receive different combinations of treatments.
Sequential assignment: Participants receive treatments one after another in a specific order, possibly based on individual responses.
Other assignment: Treatment assignment does not follow a standard or predefined design.

How the effectiveness of the treatment is controlled
In a non placebo-controlled study, no participants receive an inert substance (placebo) to compare outcomes. Instead, all participants receive either the experimental treatment or an alternative treatment (often the Standard of Care). This method allows researchers to compare the effects of the experimental treatment with those of a different active intervention, rather than a placebo.

Other Options
Placebo-Controlled: A placebo is used to compare the effects of the experimental treatment with those of an inert substance, isolating the true treatment effect.

How the interventions assigned to participants is kept confidential
Everyone involved in the study knows which treatment is being given. This is typically used when it's not possible or necessary to hide the treatment details from participants or researchers.

Other Ways to Mask Information
Single-blind: Participants do not know which treatment they are receiving, but researchers do.
Double-blind: Neither participants nor researchers know which treatment is given.
Triple-blind: Participants, researchers, and outcome assessors do not know which treatment is given.
Quadruple-blind: Participants, researchers, outcome assessors, and care providers all do not know which treatment is given.

Eligibility

Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.
Conditions
Criteria
Any sexBiological sex of participants that are eligible to enroll.
Healthy volunteers not allowedIf individuals who are healthy and do not have the condition being studied can participate.
Conditions
Pathology
Bronchiolitis Obliterans Syndrome
Organizing Pneumonia
Bronchial Diseases
Bronchiolitis
Bronchiolitis Obliterans
Bronchitis
Graft vs Host Disease
Immune System Diseases
Lung Diseases
Lung Diseases, Obstructive
Respiratory Tract Diseases
Criteria

Inclusion Criteria: * Biopsy-confirmed diagnosis of clinical extensive chronic GVHD with inadequate response to previous treatment and where secondary systemic therapy is indicated because of * Clinical progression of signs and symptoms of chronic GVHD in a previously involved organ, or * Development of signs and symptoms of chronic GVHD in a previously uninvolved organ, or * Absence of improvement after 3 months of primary treatment, or * Continued need for treatment with prednisone at doses \>= 1.0 mg/kg/day for more than 2 months, without qualification for type of donor, graft or conditioning regimen * Patient or guardian able and willing to provide informed consent * Stated willingness to use contraception in women of child-bearing potential (Food and Drug Administration \[FDA\] requirement) * Stated willingness of the patient to comply with study procedures and reporting requirements * Stated willingness of the physician most involved in management of chronic GVHD (the "managing physician,") to comply with study procedures and reporting requirements Exclusion Criteria: * Fungal or viral infection with no radiographic evidence of improvement during continued appropriate antimicrobial therapy * Cytomegalovirus (CMV) antigenemia unresponsive to antiviral therapy * Active disseminated varicella zoster virus (VZV) infection with persistent non-crusted lesions * Inability to tolerate oral medications * Absolute neutrophil count (ANC) \< 1500/uL * Platelet count \< 50,000/uL * Persistent or recurrent malignancy, including histopathologic evidence of myeloma or lymphoma; patients with breakpoint cluster region-abelson (bcr/abl) detected by polymerase chain reaction (PCR) assay as the only evidence of persistent chronic myeloid leukemia may be enrolled * Pregnancy * Known history of hypersensitivity to sirolimus

Study Plan

Find out more about all the medication administered in this study, their detailed description and what they involve.
Treatment Groups
Study Objectives
One single intervention group 

is designated in this study

This study does not include a placebo group 

Treatment Groups
Group I
Experimental
Study participants receive sirolimus added once daily to their baseline combination therapy of prednisone plus either cyclosporine or tacrolimus at the discretion of the managing physician. Treatment other than cyclosporine (or tacrolimus) and prednisone must be discontinued when administration of sirolimus is started. Topical therapy, including psoralen and UVA irradiation (PUVA), glucocorticoid creams, topical tacrolimus, oral beclomethasone, topical azathioprine and ophthalmic glucocorticoids may be given at the discretion of the managing physician in consultation with the transplant center.

Given PO
Study Objectives
Primary Objectives

Defined as the absence of any immunosuppressive treatment, including sirolimus, with resolution of all reversible manifestations of chronic GVHD and no additional systemic therapy.

Defined as the initiation of additional systemic therapy, development of bronchiolitis obliterans, or death from causes other than recurrent malignancy during primary treatment for chronic GVHD, whichever occurs first.

Includes any intervention intended to control chronic GVHD through an immunosuppressive effect from oral or parenteral administration of any systemic medication not originally given under auspices of this protocol.

Defined as clinical or histopathologic evidence demonstrating the presence of any malignancy considered as the indication for transplant. Recurrent malignancy will also be defined as any post-transplant intervention not routinely used to prevent the development of overt recurrence, prompted by laboratory evidence of persisting malignant cells but without clinical or histopathologic evidence of recurrence.
Secondary Objectives

Proportion of participants who developed at least one secondary malignancy by 7 years

Kaplan-Meier estimate assessed at 7 years for probability of survival without recurrent malignancy.

Kaplan-Meier estimate assessed at 7 years

Analyzed with recurrent malignancy as a competing risk factor. Assessed at 7 years.

Analyzed with death as a competing risk factor. Assessed at 7 years.

Study Centers

These are the hospitals, clinics, or research facilities where the trial is being conducted. You can find the location closest to you and its status.
This study has 1 location
Suspended
Fred Hutchinson Cancer Research Center/University of Washington Cancer ConsortiumSeattle, United StatesSee the location
CompletedOne Study Center