Study Of Palbociclib Combined With Chemotherapy In Pediatric Patients With Recurrent/Refractory Solid Tumors

  • End date
    Jan 21, 2025
  • participants needed
  • sponsor
Updated on 5 November 2020
Pfizer Call Center
Primary Contact
Children's National Medical Center (5.4 mi away) Contact
+59 other location
platelet count
renal function
measurable disease
bone marrow procedure
neutrophil count
blood transfusion
biological therapy
solid tumour
solid tumor
ewing's sarcoma
renal function test
diffuse intrinsic pontine glioma
kidney function test
rhabdoid tumor


This study will evaluate palbociclib in combination with chemotherapy (temozolomide with irinotecan and topotecan with cyclophosphamide) in children, adolescents and young adults with recurrent or refractory solid tumors. The main purpose of this study is to evaluate the safety of palbociclib in combination with chemotherapy in order to estimate the maximum tolerated dose. Pharmacokinetics and efficacy of palbociclib in combination with chemotherapy will be evaluated.

Treatment cyclophosphamide, Topotecan, Temozolomide, Irinotecan, Palbociclib
Clinical Study IdentifierNCT03709680
Last Modified on5 November 2020

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Inclusion Criteria

Is your age between 2 yrs and 21 yrs?
Gender: Male or Female
Do you have any of these conditions: Neuroblastoma or Rhabdomyosarcoma or Solid Tumors or Glioma or Medulloblastoma or Diffuse Intrinsic Pontine Glioma or Gliomas or Rhabdoid Tumor or Ewi...?
Histologically confirmed relapsed or refractory solid tumor as follows
For dose escalation and dose determination parts: Histologically confirmed relapsed or refractory solid tumor (including CNS tumors but not lymphomas). Patients with Diffuse Intrinsic Pontine Glioma do not require histological only radiographic confirmed relapse to enroll
For dose expansion and tumor specific cohorts: Histologically confirmed relapsed or refractory solid tumor including but not limited to EWS, rhabdoid tumor, rhabdomyosarcoma, neuroblastoma, and medulloblastoma. Patients with Diffuse Intrinsic Pontine Glioma do not require histological only radiographic confirmed relapse to enroll. EWS is not eligible for TOPO and CTX tumor-specific cohorts
Age 2 and <21 years at the time of study entry
Lansky performance status 50% for patients 16 years of age, or Eastern Cooperative Oncology Group (ECOG) 0, 1 or 2 for patients >16 years of age
Adequate bone marrow function
Absolute neutrophil count 1000/mm3
Platelet count 100,000/mm3 (transfusion independent)
Hemoglobin 8.5 g/dL (transfusion allowed)
Adequate renal function: Serum creatinine level based on age/gender must within protocol specified limits
Adequate liver function, including
Aspartate aminotransferase (AST) and alanine aminotransferase (ALT)
5 upper limit of normal (ULN) or 5 ULN for age, if attributable to disease involvement of the liver
Total bilirubin 1.5 ULN for age. 7. Measurable disease as defined by RECIST version 1.1 or modified RANO criteria for CNS disease or INRC for neuroblastoma. 8. Recovered to CTCAE Grade 1, or to baseline, from any non-hematological acute toxicities of prior surgery, chemotherapy, immunotherapy, radiotherapy, differentiation therapy or biologic therapy, with the exception of alopecia. 9. Serum/urine pregnancy test (for all girls 8 years of age) negative at screening and at the baseline visit. 10. Evidence of a personally signed and dated informed consent document indicating that the patient or a legally acceptable representative/parent(s)/legal guardian of minors, has been informed of all pertinent aspects of the study. Minor study patients also must provide age appropriate assent according to the local guidelines, where applicable. 11. Willing and able to comply with scheduled visits, treatment plan, laboratory tests, and other procedures

Exclusion Criteria

For palbociclib with IRN and TMZ combination, prior treatment with a CDK4/6 inhibitor or progression while on treatment with an IRN-containing regimen that includes TMZ. Patients who have received the combination of IRN and TMZ and did not progress while on these medications are eligible. For patients enrolling in the palbociclib with TOPO and CTX combination, prior treatment with a CDK4/6 inhibitor or progression while on treatment with a TOPO-containing regimen that includes CTX. Patients who have received the combination of TOPO and CTX and did not progress while on these medications are eligible
Prior intolerability to IRN and/or TMZ, for palbociclib with IRN and TMZ combination and prior intolerability to TOPO and/or CTX for palbociclib with TOPO and CTX combination
Use of strong cytochrome P450 (CYP) 3A inhibitors or inducers. Patients who are receiving strong uridine diphosphate-glucuronosyl transferase 1A1 (UGT1A1) inhibitors within 12 days of Cycle 1 Day 1 (C1D1) are not eligible for the palbociclib with IRN and TMZ combination. Patients who are receiving strong UGT1A1 inhibitors within 12 days of C1D1 are eligible for the palbociclib with TOPO and CTX combination (See Section 5.7.1 for list of products.)
Prior growth factors (including filgrastim) within 7 days before study entry or PEG-filgrastim within 14 days before study entry
Radiation therapy within 14 days before study entry
Systemic anti cancer therapy within 2 weeks prior to study entry and 6 weeks for nitrosoureas
Previous high dose chemotherapy requiring stem cell rescue within 90 days or persistent AE >Grade 1
Prior irradiation to >50% of the bone marrow (see Appendix 9)
Participation in other studies involving investigational drug(s) within 2 weeks or 5 half lives, whichever is longer, prior to study entry
Major surgery within 4 weeks prior to study entry. Surgical biopsies or central line placement are not considered major surgeries
Known or suspected hypersensitivity to palbociclib, IRN and/or TMZ
Patients with known symptomatic brain tumors or brain metastases and require steroids, unless they have been on a stable or on a decreasing steroid dose for >14 days
Patients with previously diagnosed brain metastases are eligible if they have completed their prior treatment and have recovered from the acute effects of radiation therapy or surgery prior to study entry for these metastases for at least 14 days post radiation and 4 weeks post-surgery and are neurologically stable
Hereditary bone marrow failure disorder
QTc >470 msec
History of clinically significant or uncontrolled cardiac disease, including
History of or active congestive heart failure; if patient had congestive heart failure resolve and >1 year from resolution, patient will be considered eligible
Clinically significant ventricular arrhythmia (such as ventricular tachycardia, ventricular fibrillation or Torsades de Pointes)
Diagnosed or suspected congenital or acquired prolonged QT syndrome
Need for medications known to prolong the QT interval
Uncorrected hypomagnesemia or hypokalemia because of potential effects on the QT interval
Left ventricular ejection fraction <50% or shortening fraction <28%. 17. Recent or ongoing clinically significant gastrointestinal disorder that may interfere with absorption of orally administered drugs (eg, gastrectomy). 18. Evidence of serious active or uncontrolled bacterial, fungal or viral infection or known history of hepatitis B virus, hepatitis C virus, or human immunodeficiency virus infection or acquired immunodeficiency syndrome-related illness. 19. Other severe acute or chronic medical or laboratory test abnormality that may increase the risk associated with study participation or investigational product administration or may interfere with the interpretation of study results, and in the judgment of the Investigator, would make the patient inappropriate for entry into this study. 20. Investigator site staff members directly involved in the conduct of the study and their family members, site staff members otherwise supervised by the investigator, or patients who are Pfizer employees, including their family members, directly involved in the conduct of the study. 21. Fertile male patients and female patients of childbearing potential who are unwilling or unable to use a highly effective method of contraception as outlined in this protocol for the duration of the study and for at least 90 after the last dose of investigational product
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