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


Phase 2
N/A
N/A
Not Enrolling
Both
Graft Versus Host Disease

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Trial Information

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


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.


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

Type of Study:

Interventional

Study Design:

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Supportive Care

Outcome Measure:

Treatment success

Outcome Description:

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

Outcome Time Frame:

Approximately 7 years

Safety Issue:

No

Principal Investigator

Paul Carpenter

Investigator Role:

Principal Investigator

Investigator Affiliation:

Fred Hutchinson Cancer Research Center/University of Washington Cancer Consortium

Authority:

United States: Federal Government

Study ID:

1706.00

NCT ID:

NCT00079183

Start Date:

April 2002

Completion Date:

Related Keywords:

  • Graft Versus Host Disease
  • Graft vs Host Disease

Name

Location

Fred Hutchinson Cancer Research Center/University of Washington Cancer ConsortiumSeattle, Washington  98109