Clinical trial NCT00098865: Thalidomide and Temozolomide in Relapsed or Progressive CNS Disease or Neuroblastoma
Official title: A Phase II Pilot Study Of Thalidomide With Temozolomide In Patients With Relapsed Or Progressive Brain Tumors Or Neuroblastoma
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Central Nervous System Tumor, Pediatric
Neuroblastoma
Lead sponsor: Dana-Farber Cancer Institute
Sponsor class: OTHER
Collaborators: name: National Cancer Institute (NCI)
name: Boston Children's Hospital
name: Celgene Corporation
First posted: 2004-12-09
Enrollment: 15
Sex: ALL
Maximum age (years): 21
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: RATIONALE: Thalidomide may stop the growth of tumor cells by stopping blood flow to the tumor. Drugs used in chemotherapy, such as temozolomide, work in different ways to stop tumor cells from dividing so they stop growing or die. Combining thalidomide with temozolomide may kill more tumor cells.

PURPOSE: This phase II trial is studying the effectiveness of combining thalidomide with temozolomide in treating young patients who have relapsed or progressive brain tumors or recurrent neuroblastoma.

Detailed description: OBJECTIVES:

Primary

* Determine the feasibility of thalidomide and temozolomide in pediatric patients with relapsed or progressive poor prognosis brain tumors or recurrent neuroblastomas.

Secondary

* Determine preliminarily evidence of biologic activity of this regimen in these patients.
* Determine the toxic effects of this regimen in these patients.

STATISTICAL DESIGN: The primary data analysis will estimate the percentage of patients who can complete 6 months of therapy in the mixed population. With a target accrual of 20 patients the 90% confidence for the true feasibility rate will be no wider than 40%.

Eligibility criteria: DISEASE CHARACTERISTICS:

* Histologically confirmed\* diagnosis of 1 of the following:

  * Poor prognosis brain tumor

    * Relapsed or progressive disease
    * No curative therapy exists
  * Neuroblastoma

    * Recurrent disease NOTE: \*Histologic confirmation not required for brain stem glioma; patients with brain stem glioma must have clinical and radiographic evidence of disease
* Patients with brain stem glioma must have symptoms lasting \< 3 months comprising cranial nerve deficits (often VI or VII) and/or ataxia and/or long tract signs

PATIENT CHARACTERISTICS:

Age

* 21 and under

Performance status

* Karnofsky 50-100% OR
* Lansky 50-100%

Life expectancy

* More than 2 months

Hematopoietic

* Hemoglobin ≥ 9.0 g/dL
* Platelet count \> 75,000/mm\^3
* WBC \> 2,000/mm\^3
* Absolute neutrophil count \> 1,000/mm\^3

Hepatic

* Bilirubin ≤ 1.5 mg/dL
* SGOT and SGPT ≤ 2 times normal (SGOT ≤ 4 times normal for patients taking Zantac)
* Alkaline phosphatase ≤ 2 times normal
* No active hepatic disease ≥ grade 3

Renal

* Creatinine \< 1.5 mg/dL OR
* Creatinine clearance ≥ 70 mL/min
* No active renal disease ≥ grade 3

Cardiovascular

* No active cardiac disease ≥ grade 3

Pulmonary

* No active pulmonary disease ≥ grade 3

Other

* Not pregnant or nursing
* Fertile patients must use effective contraception during and for 4 weeks after study participation

  * Willing and able to participate in the System for Thalidomide Education and Prescription Safety (S.T.E.P.S.\^®) program
* No active psychiatric disease ≥ grade 3

PRIOR CONCURRENT THERAPY:

Biologic therapy

* Prior biologic therapy allowed

  * No prior thalidomide

Chemotherapy

* Prior chemotherapy allowed

  * No prior temozolomide

Endocrine therapy

* Concurrent steroids allowed

Radiotherapy

* Prior radiotherapy allowed

Surgery

* Prior surgery allowed

Other

* Concurrent antiseizure medications allowed
* No other concurrent investigational agents

Primary outcomes:
measure: Therapy Completion Rate
timeFrame: 6 months
Secondary outcomes:
measure: Overall Response
timeFrame: Assessed every 8 weeks while on treatment and every 3 months for one year off-study
measure: Overall Survival
timeFrame: Assessed after treatment discontinued every 3 months up to 2 years.
Other outcomes:
Locations:
city: Boston
country: United States
facility: Dana Farber Cancer Institute
state: Massachusetts
Study officials:
affiliation: Dana-Farber Cancer Institute
name: Mark W. Kieran, MD, PhD
role: STUDY_CHAIR
References: