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Erlotinib Hydrochloride and Temozolomide in Treating Younger Patients with Relapsed, Recurrent, or Refractory Solid Tumors

Trial Status: Complete

This phase II trial studies how well erlotinib hydrochloride (erlotinib) and temozolomide work in treating younger patients with solid tumors that have returned after a period of improvement or do not respond to treatment. Erlotinib hydrochloride may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Drugs used in chemotherapy, such as temozolomide, work in different ways to stop the growth of tumor cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Giving temozolomide and erlotinib hydrochloride may work better in treating patients with solid tumors.

Inclusion Criteria

  • Relapsed, recurrent, or refractory malignancy; all solid tumor diagnoses will be eligible * Pathologic confirmation of the diagnosis either at original diagnosis or recurrence * Known non-synonymous mutation in the following genes: EGFR, ERBB2, or JAK2V617F (JAK2); genomic sample preferably from relapse, but may be from other stage of treatment if relapse sample is not reasonably obtainable; genetic analysis for determination of eligibility occurs as part of routine care and is not being performed specifically for the purposes of this study
  • Measurable disease defined as lesions that can be accurately measured in at least one dimension (longest diameter to be recorded) as >= 10 mm with computed tomography (CT) or magnetic resonance (MRI) scan, as >= 20 mm by chest x-ray, or >= 10 mm with calipers by clinical exam
  • Patients must have fully recovered from the acute toxic effects of all prior chemotherapy, immunotherapy, or radiotherapy prior to entering this study
  • Patients must not have received myelosuppressive chemotherapy within 2 weeks of enrollment onto this study (6 weeks if prior nitrosourea)
  • At least 14 days must have elapsed after receiving pegfilgrastim and least 7 days must have elapsed since the completion of therapy with a non-pegylated growth factor
  • At least 7 days must have elapsed since completion of therapy with a biologic agent; for agents that have known adverse events occurring beyond 7 days after administration, this period prior to enrollment must be extended beyond the time during which adverse events are known to occur
  • At least 3 half-lives must have elapsed since prior therapy that included a monoclonal antibody
  • At least 2 weeks must have elapsed since local palliative radiation therapy (XRT) (small port); at least 6 weeks must have elapsed since treatment with therapeutic doses of metaiodobenzylguanidine (MIBG); at least 3 months must have elapsed if prior craniospinal XRT was received, if more than 50% of the pelvis was irradiated, or if total body irradiation (TBI) was received; at least 6 weeks must have elapsed if other substantial bone marrow irradiation was given
  • No evidence of active graft vs. host disease and at least 2 months must have elapsed since transplant
  • Patients must have a performance status corresponding to Karnofsky or Lansky greater than or equal to 50%; use Karnofsky for patients > 16 years of age and Lansky for patients =< 16 years of age; patients who are unable to walk because of paralysis, but who are up in a wheelchair, will be considered ambulatory for the purpose of assessing the performance score
  • Absolute neutrophil count >= 1,000/mcl
  • Platelets >= 100,000/mcl
  • Total bilirubin =< 1.5 x institutional upper limit of normal (IULN)
  • Aspartate aminotransferase (AST) (serum glutamic oxaloacetic transaminase [SGOT])/alanine aminotransferase (ALT) (serum glutamate pyruvate transaminase [SGPT]) =< 3.0 x IULN
  • Creatinine =< IULN OR creatinine clearance >= 60 mL/min/1.73 m^2 for patients with creatinine levels above institutional normal
  • International normalized ratio (INR) =< IULN or prothrombin time (PT) =< IULN
  • Women of childbearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control, abstinence) prior to study entry and for the duration of study participation; should a woman become pregnant or suspect she is pregnant while participating in this study, she must inform her treating physician immediately
  • Ability to understand and willingness to sign an Institutional Review Board (IRB) approved written informed consent document (or that of legally authorized representative, if applicable)

Exclusion Criteria

  • A history of other malignancy =< 5 years previous with the exception of basal cell or squamous cell carcinoma of the skin which were treated with local resection only or carcinoma in situ of the cervix
  • Currently receiving any other investigational agents
  • A history of allergic reactions attributed to compounds of similar chemical or biologic composition to erlotinib, temozolomide, or dacarbazine
  • Currently on the following concomitant medications or substances that have the potential to affect the activity or pharmacokinetics of the study drug(s); the use of the following medications should be discontinued prior to initiation of protocol therapy and should be avoided during protocol therapy if reasonable alternatives exist * Erlotinib * Temozolomide
  • Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, renal failure, cardiac arrhythmia, interstitial lung disease, hepatic failure / hepatorenal syndrome, gastrointestinal (GI) perforation, cerebrovascular event, microangiopathic hemolytic anemia, corneal perforation/ulceration, or documented hepatitis B virus infection
  • Pregnant and/or breastfeeding; women of childbearing potential must have a negative serum or urine pregnancy test within 7 days of study entry
  • Know human immunodeficiency virus (HIV) positivity on combination antiretroviral therapy


Saint Louis
Washington University School of Medicine
Contact: Alok Kirankumar Kothari
Phone: 314-454-6018


I. To determine the response rate of relapsed, recurrent, or refractory pediatric tumors possessing a non-synonymous mutation in epidermal growth factor receptor (EGFR), soluble receptor tyrosine-protein kinase (ERBB2), or janus kinase 2 (JAK2)V617F (JAK2) to the combination of erlotinib and temozolomide.


I. To determine the time to progression of patients receiving this treatment regimen.

II. To further define and describe the scope of toxicities of patients receiving multiple cycles of this regimen.


Patients receive erlotinib hydrochloride orally (PO) once daily (QD) on days 1-28 and temozolomide PO QD on days 1-5 approximately 1 hour after erlotinib hydrochloride. Treatment repeats every 28 days for up to 6 courses in the absence of disease progression or unacceptable toxicity.

After completion of study treatment, patients are followed up at 30 days, every month for 3 months, and then at 1 year.

Trial Phase Phase II

Trial Type Treatment

Lead Organization
Siteman Cancer Center at Washington University

Principal Investigator
Alok Kirankumar Kothari

  • Primary ID 201604002
  • Secondary IDs NCI-2016-00549
  • ID NCT02689336