Clinical Trial: Thalidomide in Treating Patients With Myelofibrosis

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: A Pilot Study of Thalidomide as an Inhibitor of Angiogenesis in the Treatment of Myelofibrosis With Myeloid Metaplasia (MMM)

Brief Summary: 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.

Detailed Summary:

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.


Sponsor: National Cancer Institute (NCI)

Current Primary Outcome: Confirmed Response, i.e., an objective status of complete or partial response, recorded on 2 consecutive evaluations at least 4 weeks apart. [ Time Frame: Up to 5 years ]

The proportion of successes will be estimated using the Binomial point estimator (number of successes divided by the total number of evaluable patients) and 95% confidence intervals calculated using the Duffy-Santner algorithm for multi-stage designs.


Original Primary Outcome:

Current Secondary Outcome:

  • Survival [ Time Frame: Number of days from registration date to the date of death or last follow-up, assessed up to 5 years ]
    Kaplan-Meier survival curves will be generated to estimate survival.
  • Time to progression [ Time Frame: Number of days from registration date to the date of disease progression or last follow-up, assessed up to 5 years ]
    Kaplan-Meier survival curves will be generated to estimate time to progression.
  • Response duration [ Time Frame: Number of days from the first date that objective status = complete or partial response was recorded to the date of disease progression or date of death, whichever comes first, assessed up to 5 years ]
    Kaplan-Meier survival curves will be generated to estimate response duration.


Original Secondary Outcome:

Information By: National Cancer Institute (NCI)

Dates:
Date Received: May 6, 2001
Date Started: May 2000
Date Completion:
Last Updated: October 7, 2013
Last Verified: October 2013