Peginterferon Alfa-2b in Treating Younger Patients With Craniopharyngioma That is Recurrent or Cannot Be Removed By Surgery
|ClinicalTrials.gov Identifier: NCT01964300|
Recruitment Status : Active, not recruiting
First Posted : October 17, 2013
Last Update Posted : February 28, 2018
|Condition or disease||Intervention/treatment||Phase|
|Childhood Craniopharyngioma||Biological: peginterferon alfa-2b Other: laboratory biomarker analysis||Phase 2|
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.
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.
Patients receive peginterferon alfa-2b subcutaneously (SC) weekly for 6 weeks. Treatment may repeat every 6 weeks for up to 18 courses in the absence of disease progression or unacceptable toxicity.
After completion of study treatment, patients are followed up for 30 days.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||52 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Phase II Study of Peginterferon Alfa-2b (Sylatron) for Pediatric Patients With Unresectable or Recurrent Craniopharyngioma|
|Study Start Date :||September 2013|
|Estimated Primary Completion Date :||January 2019|
Experimental: Treatment (peginterferon alfa-2b)
Patients receive peginterferon alfa-2b SC weekly for 6 weeks. Treatment may repeat every 6 weeks for up to 18 courses in the absence of disease progression or unacceptable toxicity.
Biological: peginterferon alfa-2b
Other: laboratory biomarker analysis
- 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.
- 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.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01964300
|United States, California|
|Children's Hospital Los Angeles|
|Los Angeles, California, United States, 90027|
|Stanford University and Lucile Packard Children Hospital|
|Palo Alto, California, United States, 94304|
|United States, District of Columbia|
|Children's National Medical Center|
|Washington, District of Columbia, United States, 20010|
|United States, Illinois|
|Lurie Children's Hospital|
|Chicago, Illinois, United States, 60614|
|United States, Maryland|
|National Cancer Institute Pediatric Oncology Branch|
|Bethesda, Maryland, United States, 20892|
|United States, New York|
|Memorial Sloan Kettering Cancer Center|
|New York, New York, United States, 10065|
|United States, Pennsylvania|
|Children Hospital of Pittsburgh of UPMC|
|Pittsburgh, Pennsylvania, United States, 15224|
|United States, Tennessee|
|St. Jude Children Research Hospital|
|Memphis, Tennessee, United States, 38105|
|United States, Texas|
|Texas Children's Hospital|
|Houston, Texas, United States, 77030|
|Principal Investigator:||Stewart Goldman||Ann & Robert H. Lurie Children Hospital of Chicago|