Clinical Trial: Peginterferon Alfa-2b in Treating Younger Patients With Craniopharyngioma That is Recurrent or Cannot Be Removed By Surgery

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: Phase II Study of Peginterferon Alfa-2b (Sylatron) for Pediatric Patients With Unresectable or Recurrent Craniopharyngioma

Brief Summary: This phase II trial studies how well peginterferon alfa-2b works in treating younger patients with craniopharyngioma that is recurrent or cannot be removed by surgery. Peginterferon alfa-2b may interfere with the growth of tumor cells and slow the growth of craniopharyngioma.

Detailed Summary:

PRIMARY OBJECTIVES:

I. To estimate the 1-year disease stabilization rate associated with the use of Sylatron (peginterferon alfa-2b) in patients with progressive unresectable or recurrent craniopharyngiomas following surgery alone who have not received radiation therapy.

II. To estimate the sustained objective response rate (partial response [PR] + complete response [CR]) to Sylatron in patients with craniopharyngiomas which progress or recur following radiation therapy.

SECONDARY OBJECTIVES:

I. To estimate the response rate in patients with progressive unresectable or recurrent craniopharyngioma treated with Sylatron by study stratum.

II. To estimate the progression-free survival distribution for patients with unresectable or recurrent craniopharyngiomas treated with Sylatron by study stratum.

III. To evaluate the toxicity profile of Sylatron in children with unresectable or recurrent craniopharyngiomas.

IV. To compare the protocol specific disease assessment criteria to MacDonald criteria during the first year of treatment in stratum I and at the time of objective response and progressive disease in both strata.

V. To characterize evidence of WNT pathway activation by immunohistochemistry and MAPK pathway activation by pyrosequencing in resected tumor tissue in patients with craniopharyngiomas, and correlate these results with outcome and response data.

OUTLINE:

Patients receive peginterferon alfa-2b subcutaneously (SC) weekly f
Sponsor: Pediatric Brain Tumor Consortium

Current Primary Outcome:

  • Rate of disease stabilization for 1 year (i.e. 9 courses of treatment) (Stratum 1) [ Time Frame: Up to 1 year ]
    Exact confidence interval estimates will be provided for the true 1-year disease stabilization rate for stratum 1.
  • Sustained objective response (PR+CR) rate in the cystic and/or soft tissue component observed during the first year of treatment (Stratum 2) [ Time Frame: Up to 1 year ]
    Exact confidence interval estimates will be provided for the true sustained objective response rate for stratum 2.


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Sustained objective response rate (Stratum 1) [ Time Frame: Up to 2 years ]
    Exact confidence interval estimates will be provided for the true rate of sustained objective response.
  • Progression-free survival [ Time Frame: From the date of initial treatment to the earliest date of disease progression, second malignancy, or death, assessed up to 2 years ]
    Kaplan-Meier estimates of distributions of PFS for all eligible patients will be provided separately for each stratum.


Original Secondary Outcome: Same as current

Information By: Pediatric Brain Tumor Consortium

Dates:
Date Received: October 14, 2013
Date Started: September 2013
Date Completion:
Last Updated: October 26, 2016
Last Verified: October 2016