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Thalidomide, Prednisone, and Cyclophosphamide in Treating Patients With Myelofibrosis and Myeloid Metaplasia

16 maart 2011 bijgewerkt door: Mayo Clinic

Phase II Study of the Combination of Low-Dose Thalidomide, Prednisone, and Oral Cyclophosphamide ("TPC") in the Therapy of Myelofibrosis With Myeloid Metaplasia (MMM)

RATIONALE: Giving thalidomide together with prednisone and cyclophosphamide may lessen symptoms caused by myelofibrosis and myeloid metaplasia.

PURPOSE: This phase II trial is studying the side effects and how well giving thalidomide together with prednisone and cyclophosphamide works in treating patients with myelofibrosis and myeloid metaplasia.

Studie Overzicht

Toestand

Voltooid

Conditie

Interventie / Behandeling

Gedetailleerde beschrijving

OBJECTIVES:

Primary

Determine the benefit of thalidomide, prednisone, and cyclophosphamide in alleviating disease-associated anemia, thrombocytopenia, and/or splenomegaly in patients with myelofibrosis with myeloid metaplasia (MMM).
Determine the benefit of this regimen in palliating four hypercatabolic constitutional symptoms (i.e., weight loss, fatigue, drenching night sweats, and unexplained fevers) in these patients.
Determine the toxicity profile of this regimen in these patients.

Secondary

Determine the effect of this regimen on leukocyte count.
Determine the effect of this regimen on bone marrow histology, including microvessel density and reticulin fibrosis.
Determine the effect of this regimen on intramedullary and urinary markers of angiogenesis.
Determine the effect of this regimen on circulating myeloid progenitor cells by quantifying CD34+ cells.

OUTLINE: Patients receive oral thalidomide, oral prednisone, and oral cyclophosphamide (TPC) once daily on days 1-28. Treatment repeats every 28 days for 3 courses. After 3 courses (3 months) of treatment, patients who respond to TPC therapy may receive oral thalidomide alone once daily for up to 3 months in the absence of disease progression or unacceptable toxicity.

Patients undergo bone marrow aspirate and biopsy prior to study entry, 6 months after starting therapy, and then every 6 months for up to 3 years. Samples are analyzed by microvessel density/angiogenesis studies (i.e., CD34 immunohistochemical and vascular endothelium-specific staining) to determine the effect of therapy on markers of bone marrow angiogenesis.

After completion of study therapy, patients are followed every 6 months for up to 3 years.

PROJECTED ACCRUAL: A total of 22 patients will be accrued for this study.

Studietype

Ingrijpend

Inschrijving (Verwacht)

Fase

Fase 2

Deelname Criteria

Onderzoekers zoeken naar mensen die aan een bepaalde beschrijving voldoen, de zogenaamde geschiktheidscriteria. Enkele voorbeelden van deze criteria zijn iemands algemene gezondheidstoestand of eerdere behandelingen.

Geschiktheidscriteria

Leeftijden die in aanmerking komen voor studie

18 jaar en ouder (Volwassen, Oudere volwassene)

Accepteert gezonde vrijwilligers

Nee

Geslachten die in aanmerking komen voor studie

Allemaal

Beschrijving

DISEASE CHARACTERISTICS:

Histologically confirmed myelofibrosis with myeloid metaplasia (MMM) of any of the following subtypes:
- Agnogenic myeloid metaplasia
- Post-polycythemic myeloid metaplasia
- Post-thrombocythemic myeloid metaplasia
Must have 1 of the following MMM-related conditions:
- Anemia, defined as hemoglobin < 10 g/dL
  - Iron deficiency must be excluded as cause
- Thrombocytopenia, defined as platelet count < 100,000/mm³
- Palpable hepatomegaly or splenomegaly
No evidence of myelofibrosis-associated conditions in the bone marrow, including any of the following:
- Metastatic carcinoma
- Lymphoma
- Myelodysplasia
- Hairy cell leukemia
- Mast cell disease
- Acute leukemia (including M7 type)
- Acute myelofibrosis
No chromosomal translocation t(9:22) or bcr-abl as determined by bone marrow chromosome analysis or peripheral blood fluorescent in situ hybridization (FISH) analysis

PATIENT CHARACTERISTICS:

ECOG performance status 0-3
Absolute neutrophil count ≥ 750/mm³
Bilirubin ≤ 2 times upper limit of normal (ULN), unless elevation due to MMM
AST ≤ 5 times ULN, unless elevation due to MMM
Creatinine ≤ 2.5 mg/dL
No uncontrolled infection, including tuberculosis
- No known history of positive purified protein derivative (PPD) untreated by isoniazid therapy
  - Positive PPD with normal chest X-ray and completion of full-course isoniazid therapy allowed
No federal medical center inmates or other incarcerated patients
No peripheral neuropathy ≥ grade 2
No comorbid condition in which the use of study therapy is felt to be potentially harmful
Not pregnant or nursing
Negative pregnancy test
Fertile patients must use 2 forms of effective contraception

PRIOR CONCURRENT THERAPY:

No chemotherapy (e.g., hydroxyurea, myelosuppressive therapy) within the past 14 days
Prior splenectomy for MMM allowed
No concurrent hematopoietic growth factors

Studie plan

Dit gedeelte bevat details van het studieplan, inclusief hoe de studie is opgezet en wat de studie meet.

Hoe is de studie opgezet?

Ontwerpdetails

Primair doel: Behandeling
Masker: Geen (open label)

Wat meet het onderzoek?

Primaire uitkomstmaten

Uitkomstmaat
Confirmed response, defined as a complete or partial response in ≥ 1 of 3 response categories (i.e., anemia, thrombocytopenia, or splenomegaly or hepatomegaly)

Secundaire uitkomstmaten

Uitkomstmaat
Tijd voor progressie
Progressievrije overleving
Algemeen overleven
Duur van de reactie
Constitutional symptom status and bone marrow morphology
Toxicity as measured by NCI CTC v 2.0

Medewerkers en onderzoekers

Hier vindt u mensen en organisaties die betrokken zijn bij dit onderzoek.

Sponsor

Mayo Clinic

Medewerkers

National Cancer Institute (NCI)

Onderzoekers

Studie stoel: Ruben A. Mesa, MD, Mayo Clinic

Studie record data

Deze datums volgen de voortgang van het onderzoeksdossier en de samenvatting van de ingediende resultaten bij ClinicalTrials.gov. Studieverslagen en gerapporteerde resultaten worden beoordeeld door de National Library of Medicine (NLM) om er zeker van te zijn dat ze voldoen aan specifieke kwaliteitscontrolenormen voordat ze op de openbare website worden geplaatst.

Bestudeer belangrijke data

Studie start

1 oktober 2004

Primaire voltooiing (Werkelijk)

1 oktober 2006

Studie voltooiing (Werkelijk)