Phase 2
Completed N=44
Sirolimus as Secondary Therapy in Chronic Graft-Versus-Host Disease Not Responding To Prior Treatment
Source: ClinicalTrials.gov NCT00079183 ↗Enrolled (actual)
44
Serious AEs
50.0%
Results posted
May 2017
Primary outcomePrimary: Number of Participants Experiencing Treatment Success — 8 Participants
Summary
This phase II trial studies the side effects and how well sirolimus works as secondary therapy in treating patients with chronic graft-versus-host disease (GVHD) that did not respond to prior treatment. Sirolimus may be an effective treatment for chronic GVHD
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants Experiencing Treatment Success |
8 | — |
| PRIMARY Number of Participants Experiencing Treatment Failure |
9 | — |
| PRIMARY Number of Participants Needing Additional Systemic Therapy |
8; 9; 27 | — |
| PRIMARY Number of Participants With Recurrent Malignancy |
5 | — |
| SECONDARY Proportion of Patients Who Discontinue Administration of Sirolimus Because of Toxicity |
6 | — |
| SECONDARY Proportion With Infections Categorized by Organism |
21; 19; 6; 4; 1 | — |
| SECONDARY Secondary Malignancies |
15; 13; 3; 1; 1; 1 | — |
| SECONDARY Duration of Treatment With Prednisone |
45 | — |
| SECONDARY Probability of Survival Without Recurrent Malignancy |
0.54 | — |
| SECONDARY Probability of Overall Survival |
0.59 | — |
| SECONDARY Probability of Cumulative Incidence of Death Without Recurrent Malignancy |
0.25 | — |
| SECONDARY Probability of Cumulative Incidence of Recurrent Malignancy |
0.20 | — |
Eligibility 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
Data sourced from ClinicalTrials.gov (NCT00079183). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.