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Clinical Trials by Cancer Site

Pediatric Cancer

The types of cancers that develop in children are very different from the types that develop in adults and tend to be more aggressive. Following is a list of the most common pediatric (childhood) cancers, listed from most to least common:

  • Leukemia (cancer of the blood and bone marrow)
  • Brain and other central nervous system tumors
  • Neuroblastoma (often begins in the nerve tissue of the adrenal glands)
  • Non-Hodgkin lymphoma and Hodgkin lymphoma (cancers of the lymph system that may spread into the bone marrow)
  • Wilms tumor (a type of kidney cancer)
  • Rhabdomyosarcoma (a soft tissue sarcoma that usually begins in muscles that are attached to bones)
  • Osteosarcoma (a type of bone cancer)
  • Retinoblastoma (a cancer that begins in the retina at the back of the eye)
  • Ewing sarcoma (a type of bone cancer)

Although pediatric cancers are rare, it is estimated that there will be about 11,630* children from newborn to age 14 newly diagnosed with some form of pediatric cancer in 2013.

Given that childhood cancer is relatively rare, it is important to seek treatment in centers that specialize in the treatment of children with cancer. Specialized cancer programs at comprehensive, multidisciplinary cancer centers follow established protocols (step-by-step guidelines for treatment). These protocols are carried out using a team approach. The team of health professionals is involved in designing the appropriate treatment and support program for your child and family.

*American Cancer Society. Cancer Facts and Figures 2013. Atlanta: American Cancer Society; 2013

Pediatric Cancer Clinical Trials

The NCI Center for Cancer Research (CCR) conducts clinical trials at the NIH Clinical Center in Bethesda, Maryland. CCR is currently conducting the following trials for children diagnosed with cancer. Click on the trials below for additional details, including a summary of key eligibility criteria, study outline, and information on how to contact the principal investigators and their staff directly. For information on a particular trial or to discuss a screening visit, contact a member of the research team listed on the trial’s summary.

If you need further assistance, call our toll-free number at 1-888-NCI-1937 (1-888-624-1937) to be connected to a referral coordinator appropriate for your diagnosis.


  
Trial and Protocol Number
Pediatric Cancers
Phase II
A Phase II Study of the Raf Kinase and Receptor Tyrosine Kinase Inhibitor Sorafenib (IND# 69896) in Children and Young Adults With Relapsed/Refractory Rhabdomyosarcoma, Wilms Tumor, Hepatocellular Carcinoma, and Papillary Thyroid Carcinoma
NCI-12-C-0098, NCT01502410

Principal Investigator:Referral Contact:
Brigitte WidemannPediatric Oncology
301-496-7387301-496-4256
1-877-624-4878 (Toll free)

Phase I/II
Phase I/II Trial of Mithramycin In Children and Adults With Refractory Extracranial Solid Tumors (Phase I) or Ewing Sarcoma and EWS-FLI1 Fusion Transcript
NCI-12-C-0135, NCT01610570

Principal Investigator:Referral Contact:
Brigitte WidemannPediatric Oncology
301-496-7387301-496-4256
1-877-624-4878 (Toll free)

Phase I
A Phase I Study of XL184 (Cabozantinib, IND# 116059) in Children and Adolescents With Recurrent or Refractory Solid Tumors, Including CNS Tumors
NCI-13-C-0042, NCT01709435

Principal Investigator:Referral Contact:
Brigitte WidemannPediatric Oncology
301-496-7387301-496-4256
1-877-624-4878 (Toll free)

A Phase I Study of Ridaforolimus in Pediatric Patients With Advanced Solid Tumors
NCI-12-C-0138 , NCT01431534

Principal Investigator:Referral Contact:
Lee J. HelmanPediatric Oncology
301-496-4257301-496-4256
1-877-624-4878 (Toll free)

A Phase I Dose Escalation Study of Sorafenib and Irinotecan Combination Therapy in Pediatric Patients With Relapsed or Refractory Solid Tumors
NCI-12-C-0208, NCT01518413

Principal Investigator:Referral Contact:
Brigitte WidemannPediatric Oncology
301-496-7387301-496-4256
1-877-624-4878 (Toll free)

A Phase I Study of Monotherapy Dalotuzumab and Ridaforolimus-Dalotuzumab Combination Treatment in Pediatric Patients With Advanced Solid Tumors
NCI-12-C-0140

Principal Investigator:Referral Contact:
Lee J. HelmanPediatric Oncology
301-496-4257301-496-4256
1-877-624-4878 (Toll free)

Phase I Study of T Cells Expressing an Anti-CD19 Chimeric Receptor in Children and Young Adults With B Cell Malignancies
NCI-12-C-0112, NCT01593696

Principal Investigator:Referral Contact:
Alan S. WaynePediatric Oncology
301-496-4256301-496-4256
1-877-624-4878 (Toll free)

A Phase I Study of NK Cell Infusion Following Allogeneic Peripheral Blood Stem Cell Transplantation From Related or Matched Unrelated Donors in Pediatric Patients With Solid Tumors and Leukemias
NCI-11-C-0073, NCT01287104

Principal Investigator:Referral Contact:
Kristin BairdPediatric Oncology
301-496-4256301-496-4256
1-877-624-4878 (Toll free)

Phase I Study of Temsirolimus in Combination With Irinotecan and Temozolomide in Children, Adolescents, and Young Adults With Relapsed or Refractory Solid Tumors
NCI-11-C-0053, NCT01141244

Principal Investigator:Referral Contact:
Brigitte WidemannPediatric Oncology
301-496-7387301-496-4256
1-877-624-4878 (Toll free)

Phase I Trial and Pharmacokinetic Study of the Oral Platinum Analog Satraplatin in Children and Young Adults With Refractory Solid Tumors Including Brain Tumors
NCI-11-C-0047, NCT01259479

Principal Investigator:Referral Contact:
Brigitte WidemannPediatric Oncology
301-496-7387301-496-4256
1-877-624-4878 (Toll free)

Phase I Trial to Evaluate the Safety, Activity and Pharmacokinetics of Marqibo® (Vincristine Sulfate Liposomes Injection) in Children and Adolescents With Refractory Cancer
NCI-10-C-0220, NCT01222780

Principal Investigator:Referral Contact:
Alan S. WaynePediatric Oncology
301-496-4256301-496-4256
1-877-624-4878 (Toll free)

A Phase 1/2 Study of PF-02341066, An Oral Small Molecule Inhibitor of Anaplastic Lymphoma Kinase (ALK) and C-Met, in Children With Relapsed/Refractory Solid Tumors and Anaplastic Large Cell Lymphoma
NCI-10-C-0178, NCT01182896

Principal Investigator:Referral Contact:
Brigitte WidemannPediatric Oncology
301-496-7387301-496-4256
1-877-624-4878 (Toll free)

A Phase I Study of Ipilimumab (Anti-CTLA-4) in Children, Adolescents, and Young Adults With Treatment Refractory Cancer
NCI-08-C-0007, NCT00556881

Principal Investigator:Referral Contact:
Melinda MerchantPediatric Oncology
301-443-7955 301-496-4256
1-877-624-4878 (Toll free)

No Phase
Understanding the Perceived Influence of Childhood Cancer on the Parents’ Marital/Partner Relationship: A Descriptive Study
NCI-12-C-0206, NCT01702922

Principal Investigator:Referral Contact:
Lori S. WienerPediatric Oncology
301-451-9148301-496-4256
1-877-624-4878 (Toll free)

Validation of the English Version of the Pain Interference Index and the Pain Rating Scale in Children, Adolescents, and Young Adults With Chronic Illness and Their Parents
NCI-12-C-0160, NCT01639950

Principal Investigator:Referral Contact:
Staci M. PeronPediatric Oncology
301-435-3964301-496-4256
1-877-624-4878 (Toll free)

Understanding the Psychosocial Needs of Parents Who Have Lost a Child to Cancer
NCI-11-C-0132, NCT01324154

Principal Investigator:Referral Contact:
Lori S. WienerPediatric Oncology
301-451-9148301-496-4256
1-877-624-4878 (Toll free)

A Comprehensive Omics Analysis of Pediatric Oncology Solid Tumors and Establishment of a Repository for Related Biological Studies
NCI-10-C-0086, NCT01109394

Principal Investigator:Referral Contact:
Javed KhanPediatric Oncology
301-435-2937301-496-4256
1-877-624-4878 (Toll free)



  
Trial and Protocol Number
Brain Tumor
Phase II
A Phase II Randomized Trial of Lenalidomide in Pediatric Patients With Recurrent, Refractory or Progressive Juvenile Pilocytic Astrocytomas and Optic Pathway Gliomas
NCI-12-C-0136, NCT01553149

Principal Investigator:Referral Contact:
Katherine E. WarrenLinda Ellison
301-496-4256301-496-8009
ellisonl@mail.nih.gov
Phase I/II
PBTC-033 A Phase I/II Study of ABT-888, an Oral Poly(ADP-ribose) Polymerase Inhibitor, and Concurrent Radiation Therapy, Followed by ABT-888 and Temozolomide, in Children With Newly Diagnosed Diffuse Pontine Gliomas (DIPG)
NCI-12-C-0213, NCT01514201

Principal Investigator:Referral Contact:
Katherine E. WarrenLinda Ellison
301-496-4256301-496-8009
ellisonl@mail.nih.gov
Phase I
Phase I and Pharmacokinetic Study of AZD6244 for Recurrent or Refractory Pediatric Low Grade Glioma
NCI-11-C-0191, NCT01386450

Principal Investigator:Referral Contact:
Katherine E. WarrenLinda Ellison
301-496-4256301-496-8009
ellisonl@mail.nih.gov
A Phase I Trial of Lenalidomide and Radiotherapy in Children With Diffuse Intrinsic Pontine Gliomas and High-grade Gliomas
NCI-10-C-0219, NCT01226940

Principal Investigator:Referral Contact:
Katherine E. WarrenLinda Ellison
301-496-4256301-496-8009
ellisonl@mail.nih.gov
No Phase
An Exploratory Study of Biologic and Pathophysiologic Effects of Radiation Therapy in Pediatric Patients With Central Nervous System Tumors
NCI-06-C-0219, NCT00401037

Principal Investigator:Referral Contact:
Katherine E. WarrenLinda Ellison
301-496-4256301-496-8009
ellisonl@mail.nih.gov
A Comparative Study of Pediatric CNS Tumor Activity as Assessed by 18F-FDG PET Imaging and Proton Magnetic Resonance Spectroscopic Imaging (1H-MRSI)
NCI-03-C-0278, NCT00070512

Principal Investigator:Referral Contact:
Katherine E. WarrenLinda Ellison
301-496-4256301-496-8009
ellisonl@mail.nih.gov


  
Trial and Protocol Number
Bronchiolitis Obliterans (BO)
Phase II
Multi-Institutional Prospective Phase II Study of Montelukast for the Treatment of Bronchiolitis Obliterans Following Allogeneic Stem Cell Transplantation in Children and Adults
NCI-08-C-0097, NCT00656058

Principal Investigator:Referral Contact:
Ronald E. GressZetta Blacklock
301-435-4654301-594-2056
bblacklock@mail.nih.gov


  
Trial and Protocol Number
Bronchiolitis Obliterans Syndrome (BOS)
Phase II
Targeted Therapy of Bronchiolitis Obliterans Syndrome (BOS)
NCI-11-C-0226, NCT01307462

Principal Investigator:Referral Contact:
Kirsten M. WilliamsZetta Blacklock
301-435-6456301-594-2056
bblacklock@mail.nih.gov


  
Trial and Protocol Number
DOCK8 Deficiency
Phase I
A Pilot Study of Reduced-Intensity Hematopoietic Stem Cell Transplant for DOCK8 Deficiency
NCI-10-C-0174, NCT01176006

Principal Investigator:Referral Contact:
Dennis HicksteinZetta Blacklock
301-594-1718301-594-2056
bblacklock@mail.nih.gov


  
Trial and Protocol Number
Hematologic/Blood Cancers
Phase I/II
A Pilot Trial of WT1 Peptide-Loaded Allogeneic Dendritic Cell Vaccine and Donor Lymphocyte Infusion for WT1-Expressing Hematologic Malignancies
NCI-08-C-0051, NCT00923910

Principal Investigator:Referral Contact:
Alan S. WaynePediatric Oncology
301-496-4256301-496-4256
1-877-624-4878 (Toll free)

Phase I
Study of the Biology and Natural History of Disease Outcomes in Patients Treated With Allogeneic Hematopoietic Stem Cell Transplantation for Hematologic Malignancies
NCI-11-C-0125, NCT01326728

Principal Investigator:Referral Contact:
Nancy M. HardyZetta Blacklock
301-451-1406301-594-2056
bblacklock@mail.nih.gov
A Phase I, Multi-Center, Dose Escalation Study of CAT-8015 in Pediatric Patients With Refractory CD22+ Acute Lymphoblastic Leukemia (ALL) or Non-Hodgkin’s Lymphoma (NHL)
NCI-08-C-0123, NCT00659425

Principal Investigator:Referral Contact:
Alan S. WaynePediatric Oncology
301-496-4256301-496-4256
1-877-624-4878 (Toll free)

No Phase
Hematologic Malignancy Biology Study
NCI-04-C-0102, NCT00923442

Principal Investigator:Referral Contact:
Alan S. WaynePediatric Oncology
301-496-4256301-496-4256
1-877-624-4878 (Toll free)



  
Trial and Protocol Number
Lymphoma
Phase II
Short-Course EPOCH-Rituximab in Untreated CD-20+ HIV-Associated Lymphomas
NCI-01-C-0030, NCT00006436

Principal Investigator:Referral Contact:
Wyndham H. WilsonPeggy Shovlin
301-435-2415301-594-6597
mshovlin@mail.nih.gov


  
Trial and Protocol Number
Malignant Peripheral Nerve Sheath Tumor
Phase II
SARC016: Phase II Study of the mTOR Inhibitor Everolimus in Combination With Bevacizumab in Patients With Sporadic and Neurofibromatosis Type 1 (NF1) Related Refractory Malignant Peripheral Nerve Sheath Tumors
NCI-12-C-0148

Principal Investigator:Referral Contact:
Brigitte WidemannPediatric Oncology
301-496-7387301-496-4256
1-877-624-4878 (Toll free)



  
Trial and Protocol Number
Multicentric Castleman Disease and Associated Conditions
No Phase
Targeted Oncolytic Virotherapy and Natural History Study of KSHV-Associated Multicentric Castleman’s Disease With Laboratory and Clinical Correlates of Disease Activity
NCI-04-C-0275, NCT00099073

Principal Investigator:Referral Contact:
Robert YarchoanKaren Aleman
301-496-8959301-496-8959
1-800-243-2732 ext. 4 (Toll Free)
alemank@mail.nih.gov


  
Trial and Protocol Number
Neurofibroma/Neurofibromatosis
Phase II
A Phase II Trial of Peginterferon Alpha-2b (Pegintron) for Neurofibromatosis Type 1 Related Unresectable, Symptomatic, or Life-Threatening Plexiform Neurofibromas
NCI-08-C-0130, NCT00678951

Principal Investigator:Referral Contact:
Brigitte WidemannPediatric Oncology
301-496-7387301-496-4256
1-877-624-4878 (Toll free)

Phase I
A Phase II Study of Everolimus (RAD001) for Children With Neurofibromatosis Type-1 and Chemotherapy-Refractory Radiographic Progressive Low-Grade Gliomas
NCI-12-C-0058, NCT00901849

Principal Investigator:Referral Contact:
Katherine E. WarrenLinda Ellison
301-496-4256301-496-8009
ellisonl@mail.nih.gov
Phase I Study of the Mitogen Activated Protein Kinase (MEK) 1 Inhibitor AZD6244 Hydrogen Sulfate in Children With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas (PN)
NCI-11-C-0161, NCT01362803

Principal Investigator:Referral Contact:
Brigitte WidemannPediatric Oncology
301-496-7387301-496-4256
1-877-624-4878 (Toll free)

Phase 0
Acceptance and Commitment Therapy for Adolescents and Adults With Neurofibromatosis Type 1 and Chronic Pain: A Pilot Study
NCI-12-C-0155, NCT01633008

Principal Investigator:Referral Contact:
Staci M. PeronPediatric Oncology
301-435-3964301-496-4256
1-877-624-4878 (Toll free)

No Phase
Natural History Study and Longitudinal Assessment of Children, Adolescents, and Adults with Neurofibromatosis
NCI-08-C-0079, NCT00924196

Principal Investigator:Referral Contact:
Brigitte WidemannPediatric Oncology
301-496-7387301-496-4256
1-877-624-4878 (Toll free)



  
Trial and Protocol Number
Osteosarcoma
Phase II
A Randomized, Double-Blinded, Placebo-Controlled, Multi-Institutional, Phase II.5 Study of Saracatinib (AZD0530), a Selective Src Kinase Inhibitor, in Patients With Recurrent Osteosarcoma Localized to the Lung
NCI-09-C-0104, NCT00752206

Principal Investigator:Referral Contact:
Kristin BairdPediatric Oncology
301-496-4256301-496-4256
1-877-624-4878 (Toll free)



  
Trial and Protocol Number
Sarcoma
Phase II
A Pilot Study of Genetically Engineered NY-ESO-1 Specific (c259) T cells in HLA-A2+ Patients With Synovial Sarcoma
NCI-11-C-0113, NCT01343043

Principal Investigator:Referral Contact:
Crystal MackallPediatric Oncology
301-402-5940301-496-4256
1-877-624-4878 (Toll free)



  
Trial and Protocol Number
Thyroid Cancer
Phase I/II
Phase I/II Trial of Vandetanib (ZD6474, ZACTIMA) in Children and Adolescents With Hereditary Medullary Thyroid Cancer
NCI-07-C-0189, NCT00514046

Principal Investigator:Referral Contact:
Brigitte WidemannPediatric Oncology
301-496-7387301-496-4256
1-877-624-4878 (Toll free)

No Phase
Longitudinal Assessment and Natural History Study of Children and Young Adults With Medullary Thyroid Carcinoma
NCI-12-C-0178 , NCT01660984

Principal Investigator:Referral Contact:
Brigitte WidemannPediatric Oncology
301-496-7387301-496-4256
1-877-624-4878 (Toll free)

Clinical and Genetic Studies in Familial Non-medullary Thyroid Cancer
NCI-10-C-0102, NCT01109420

Principal Investigator:Referral Contact:
Electron KebebewErinn M. Hopkins
301-496-5049301-496-6457
erinn.hopkins@nih.gov


  
Trial and Protocol Number
Xeroderma Pigmentosum
No Phase
Cancer Risk in Xeroderma Pigmentosum Heterozygotes
NCI-02-C-0313, NCT00049621

Principal Investigator:Referral Contact:
Kenneth H. KraemerDeborah Tamura
301-496-9033301-594-5030
tamurad@mail.nih.gov
Examination of Clinical and Laboratory Abnormalities in Patients With Defective DNA Repair: Xeroderma Pigmentosum, Cockayne Syndrome, or Trichothiodystrophy
NCI-99-C-0099, NCT00004044

Principal Investigator:Referral Contact:
Kenneth H. KraemerDeborah Tamura
301-496-9033301-594-5030
tamurad@mail.nih.gov


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