Thalidomide in Treating Patients With Myelofibrosis

National Cancer Institute (NCI)

Status and phase

Completed

Phase 2

Conditions

Primary Myelofibrosis

Treatments

Other: laboratory biomarker analysis

Drug: thalidomide

Study type

Interventional

Funder types

NIH

Identifiers

NCT00015821

NCI-2012-01853 (Registry Identifier)

NCCTG-N9982

U10CA025224 (U.S. NIH Grant/Contract)

CDR0000068367

N9982 (Other Identifier)

Details and patient eligibility

About

Phase II trial to study the effectiveness of thalidomide in treating patients who have myelofibrosis. Thalidomide may stop the growth of myelofibrosis by stopping blood flow to the cancer cells.

Full description

PRIMARY OBJECTIVES:

I. To investigate whether thalidomide, a potent inhibitor of angiogenic and fibrogenic growth factors, is an effective therapeutic agent in patients with MMM. Specifically, to assess whether thalidomide improves anemia and/or organomegaly in patients with MMM.

II. To assess the effects of thalidomide on the myelofibrotic stroma with respect to microvascular architecture and angiogenesis, collagen and reticulin deposition, and the expression of the mediating growth factors bFGF, TGF-b, and PDGF, and their respective receptors.

OUTLINE: This is a multicenter study.

Patients receive oral thalidomide once daily for 1 year in the absence of disease progression or unacceptable toxicity. Patients with stable or responding disease may receive 1 additional year of therapy.

Patients are followed every 6 months until 5 years from study entry.

Enrollment

43 estimated patients

Sex

All

Ages

18+ years old

Volunteers

No Healthy Volunteers

Inclusion and exclusion criteria

Inclusion Criteria:

Histologically confirmed myelofibrosis with myeloid metaplasia
- Agnogenic myeloid metaplasia
- Post-polycythemic myeloid metaplasia
- Post-thrombocythemic myeloid metaplasia
No metastatic carcinoma, lymphoma, myelodysplasia, hairy cell leukemia, mast cell disease, acute leukemia (including M7), or acute myelofibrosis
No chromosomal translocation t(9;22) or bcr/abl gene rearrangement
Presence of reticulin fibrosis in bone marrow and leukoerythroblastosis and dacrocytosis in peripheral blood
Presence of anemia (hemoglobin less than 10 g/dL), palpable splenomegaly, or hepatomegaly
Performance status - ECOG 0-2
Absolute neutrophil count greater than 750/mm^3
Platelet count less than 400,000/mm^3
WBC less than 50,000/mm^3
Bilirubin no greater than 2 mg/dL (if total bilirubin elevated, direct bilirubin must be normal)
AST no greater than 3 times upper limit of normal (ULN)
Alkaline phosphatase no greater than 3 times ULN
Creatinine no greater than 1.5 mg/dL
Creatinine clearance at least 60 mL/min
Not pregnant or nursing
Negative pregnancy test
Fertile women must use at least 1 highly active method AND 1 additional effective method of contraception for at least 4 weeks before study, during study, and for at least 4 weeks after study
Fertile men must use effective contraception during study and for at least 4 weeks after study
No uncontrolled infection
No concurrent condition that would preclude study
No peripheral neuropathy
At least 1 month since prior interferon, pirfenidone, anagrelide, or epoetin alfa
At least 1 month since prior hydroxyurea or other chemotherapy
At least 1 month since prior corticosteroids or androgen derivatives