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Sirolimus as Secondary Therapy in Chronic Graft-Versus-Host Disease Not Responding To Prior Treatment

23. mai 2017 oppdatert av: Paul Carpenter, Fred Hutchinson Cancer Center

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

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

Studieoversikt

Status

Fullført

Forhold

Intervensjon / Behandling

Detaljert beskrivelse

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.

Studietype

Intervensjonell

Registrering (Faktiske)

44

Fase

  • Fase 2

Kontakter og plasseringer

Denne delen inneholder kontaktinformasjon for de som utfører studien, og informasjon om hvor denne studien blir utført.

Studiesteder

  • Forente stater
    • Washington
      • Seattle, Washington, Forente stater, 98109
        • Fred Hutchinson Cancer Research Center/University of Washington Cancer Consortium

Deltakelseskriterier

Forskere ser etter personer som passer til en bestemt beskrivelse, kalt kvalifikasjonskriterier. Noen eksempler på disse kriteriene er en persons generelle helsetilstand eller tidligere behandlinger.

Kvalifikasjonskriterier

Alder som er kvalifisert for studier

  • Barn
  • Voksen
  • Eldre voksen

Tar imot friske frivillige

Nei

Kjønn som er kvalifisert for studier

Alle

Beskrivelse

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

Studieplan

Denne delen gir detaljer om studieplanen, inkludert hvordan studien er utformet og hva studien måler.

Hvordan er studiet utformet?

Designdetaljer

  • Primært formål: Støttende omsorg
  • Tildeling: N/A
  • Intervensjonsmodell: Enkeltgruppeoppdrag
  • Masking: Ingen (Open Label)

Våpen og intervensjoner

Deltakergruppe / Arm
Intervensjon / Behandling
Eksperimentell: Sirolimus
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.
Legemiddel: sirolimus
Gitt PO
Andre navn:
  • Rapamune
  • AY 22989
  • rapamycin
  • SLM

Hva måler studien?

Primære resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
Number of Participants Experiencing Treatment Success
Tidsramme: Approximately 7 years
Defined as the absence of any immunosuppressive treatment, including sirolimus, with resolution of all reversible manifestations of chronic GVHD and no additional systemic therapy.
Approximately 7 years
Number of Participants Experiencing Treatment Failure
Tidsramme: Approximately 7 years
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.
Approximately 7 years
Number of Participants Needing Additional Systemic Therapy
Tidsramme: Approximately 7 years
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.
Approximately 7 years
Number of Participants With Recurrent Malignancy
Tidsramme: Approximately 7 years
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.
Approximately 7 years

Sekundære resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
Proportion of Patients Who Discontinue Administration of Sirolimus Because of Toxicity
Tidsramme: Approximately 7 years
Approximately 7 years
Proportion With Infections Categorized by Organism
Tidsramme: Approximately 7 years
Approximately 7 years
Secondary Malignancies
Tidsramme: Up to 7 years
Proportion of participants who developed at least one secondary malignancy by 7 years
Up to 7 years
Duration of Treatment With Prednisone
Tidsramme: Approximately 7 years
Approximately 7 years
Probability of Survival Without Recurrent Malignancy
Tidsramme: Approximately 7 years
Kaplan-Meier estimate assessed at 7 years for probability of survival without recurrent malignancy.
Approximately 7 years
Probability of Overall Survival
Tidsramme: Approximately 7 years
Kaplan-Meier estimate assessed at 7 years
Approximately 7 years
Probability of Cumulative Incidence of Death Without Recurrent Malignancy
Tidsramme: Approximately 7 years
Analyzed with recurrent malignancy as a competing risk factor. Assessed at 7 years.
Approximately 7 years
Probability of Cumulative Incidence of Recurrent Malignancy
Tidsramme: Approximately 7 years
Analyzed with death as a competing risk factor. Assessed at 7 years.
Approximately 7 years

Samarbeidspartnere og etterforskere

Det er her du vil finne personer og organisasjoner som er involvert i denne studien.

Sponsor

Fred Hutchinson Cancer Center

Samarbeidspartnere

National Cancer Institute (NCI)

Studierekorddatoer

Disse datoene sporer fremdriften for innsending av studieposter og sammendragsresultater til ClinicalTrials.gov. Studieposter og rapporterte resultater gjennomgås av National Library of Medicine (NLM) for å sikre at de oppfyller spesifikke kvalitetskontrollstandarder før de legges ut på det offentlige nettstedet.

Studer hoveddatoer

Studiestart

1. april 2002

Primær fullføring (Faktiske)

1. juli 2009

Studiet fullført (Faktiske)

10. juni 2010

Datoer for studieregistrering

Først innsendt

8. mars 2004

Først innsendt som oppfylte QC-kriteriene

9. mars 2004

Først lagt ut (Anslag)

10. mars 2004

Oppdateringer av studieposter

Sist oppdatering lagt ut (Faktiske)

20. juni 2017

Siste oppdatering sendt inn som oppfylte QC-kriteriene

23. mai 2017

Sist bekreftet

1. mai 2017

Mer informasjon

Begreper knyttet til denne studien

Ytterligere relevante MeSH-vilkår

Andre studie-ID-numre

  • 1706.00
  • NCI-2011-01817 (Registeridentifikator: CTRP (Clinical Trial Reporting Program))

Plan for individuelle deltakerdata (IPD)

Planlegger du å dele individuelle deltakerdata (IPD)?

NEI

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