Canadian clinical trial registry

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Information is also accessible through the patient and families tab. Family friendly summaries are created and reviewed by our advocacy partners. The information is updated to the best of our knowledge but might not reflect the latest information. Note that most studies are only available at a limited number of sites, please click on ‘further information’ for details. Studies, particularly early phase trials, may also temporarily close to enrolment or not have slots available for all treatment groups. In all cases, study teams at individual C17 centres will have the most up-to-date information.

89 results found

Title
Status

 

MS100070-0306 - Open-label, Phase I/II Study to Evaluate Pharmacokinetics, Pharmacodynamics, Safety, and Anticancer Activity of Avelumab in Pediatric Subjects From Birth to Less Than 18 Years of Age With Refractory or Relapsed Solid Tumors and Lymphoma

Completed

MS100070-0306 - Open-label, Phase I/II Study to Evaluate Pharmacokinetics, Pharmacodynamics, Safety, and Anticancer Activity of Avelumab in Pediatric Subjects From Birth to Less Than 18 Years of Age With Refractory or Relapsed Solid Tumors and Lymphoma

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DiagnosisRefractory or relapsed solid tumors, Lymphoma, Non-Hodgkin lymphoma, Other solid tumoursStudy StatusCompleted
PhaseI/II
AgeChild, Adult - (up to 18 Years)RandomisationNO
Line of treatmentDisease relapse or progression
Routes of Treatment AdministrationIV
Last Posted Update2022-02-23
ClinicalTrials.gov #NCT03451825
International Sponsor
EMD Serono Research & Development Institute, Inc.
Principal Investigators for Canadian Sites
The Hospital for Sick Children - Dr. Daniel Morgenstern
CHU Ste Justine - Dr. Yvan Samson
Children's Hospital of Western Ontario - Dr. Alexandra Zorzi
Centres
Medical contact

Dr. Daniel Morgenstern

daniel.morgenstern@sickkids.ca

Social worker/patient navigator contact

Karen Fung 

karen.fung@sickkids.ca

Clinical research contact

New Agent and Innovative Therapies (NAIT) 

nait.info@sickkids.ca

 

Medical contact
Dr. Alexandra Zorzi
Dr. Shayna Zelcer
 
Social worker/patient navigator contact
Cindy Milne Wren
Jessica Mackenzie Harris
 
Clinical research contact
Mariam Mikhail
Medical contact
Dr. Henrique Bittencourt
Dr. Monia Marzouki
Dr. Sebastien Perreault (neuro-onc)
 
Social worker/patient navigator contact
Marie-Claude Charrette
 
Clinical research contact
Marie Saint-Jacques
 

 

 

Study Description

This is a study to evaluate the dose, safety, tolerability, antitumor activity and other pharmacologic characteristics of avelumab (an antibody that binds and blocks PDL-1 on tumour cells).

This study has two parts. Part 1 will find the recommended dose for avelumab therapy and evaluate its safety. Part 2 will further evaluate the efficacy of avelumab therapy.

Inclusion Criteria
  • Age between 0 and 18
  • Solid malignant tumors (including brain tumors) or lymphoma for which no standard therapy is available, or with confirmed progression, or refractory to standard therapy
  • Availability of tumor sample collected prior to avelumab treatment for subjects in Phase 2
  • Multiple other inclusion and exclusion criteria could apply and will be reviewed by your treating team.

OLIE - A Multicenter, Open-label, Randomized Phase 2 Study to Compare the Efficacy and Safety of Lenvatinib in Combination With Ifosfamide and Etoposide Versus Ifosfamide and Etoposide in Children, Adolescents and Young Adults With Relapsed or Refractory Osteosarcoma (OLIE)

Completed

OLIE - A Multicenter, Open-label, Randomized Phase 2 Study to Compare the Efficacy and Safety of Lenvatinib in Combination With Ifosfamide and Etoposide Versus Ifosfamide and Etoposide in Children, Adolescents and Young Adults With Relapsed or Refractory Osteosarcoma (OLIE)

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DiagnosisOsteosarcoma Study StatusCompleted
PhaseII
AgeChild, Adult - (2 Years to 25 Years) RandomisationYES
Line of treatmentDisease relapse or progression
Routes of Treatment AdministrationLevatinib taken by mouth (oral capsules or liquid) Ifosfamide and Etoposide administered through intravenous infusion
Last Posted Update2022-02-22
ClinicalTrials.gov #NCT04154189
International Sponsor
Eisai Inc.
Principal Investigators for Canadian Sites
The Hospital for Sick Children - Dr. Daniel Morgenstern
Centres
Medical contact

Dr. Daniel Morgenstern

daniel.morgenstern@sickkids.ca

Social worker/patient navigator contact

Karen Fung 

karen.fung@sickkids.ca

Clinical research contact

New Agent and Innovative Therapies (NAIT) 

nait.info@sickkids.ca

 

 

 

Study Description

This clinical trial studies whether adding an oral medication called lenvatinib to chemotherapy with ifosfamide/etoposide improves outcomes for patients with osteosarcoma that has come back (relapse). 

Lenvatinib blocks cell proteins and signals directed at blood vessels that help the cancer to survive.

Patients can be treated in two different arms: Arm A: chemotherapy + lenvatinib or  Arm B : chemotherapy. The participants will be divided by chance into those two separate groups through a computerised process. The treating team will NOT decide into which arm a given patient will be assigned. At this time, it is not known which treatment option is best.

 

Inclusion Criteria
  • Age between 2 and 25 years
  • Osteosarcoma that has come back (relapse) or is not improving despite treatments (refractory)
  • Multiple other inclusion and exclusion criteria could apply and will be reviewed by your treating team

E7080-A001-216 ADVL1711 - A Phase 1/2 Study of Lenvatinib in Combination With Everolimus in Recurrent and Refractory Pediatric Solid Tumors, Including CNS Tumors

Completed

E7080-A001-216 ADVL1711 - A Phase 1/2 Study of Lenvatinib in Combination With Everolimus in Recurrent and Refractory Pediatric Solid Tumors, Including CNS Tumors

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DiagnosisHigh Grade Glioma, Ewing sarcoma, RhabdomyosarcomaStudy StatusCompleted
PhaseI/II
AgeChild, Adult - (2 Years to 21 Years)RandomisationNO
Line of treatmentDisease relapse or progression
Routes of Treatment AdministrationOral hard tablets or suspension
Last Posted Update2022-02-14
ClinicalTrials.gov #NCT03245151
International Sponsor
Eisai Inc.
Principal Investigators for Canadian Sites
The Hospital for Sick Children - Dr. Daniel Morgenstern
IWK Health Centre - Dr. Craig Erker
Montreal Children's Hospital - Dr. Sharon Abish



Centres
Medical contact

Dr. Daniel Morgenstern

daniel.morgenstern@sickkids.ca

Social worker/patient navigator contact

Karen Fung 

karen.fung@sickkids.ca

Clinical research contact

New Agent and Innovative Therapies (NAIT) 

nait.info@sickkids.ca

 

Medical contact
Dr. Craig Erker
Dr. Conrad Fernandez 
Dr. Ketan Kulkarni 
 
Social worker/patient navigator contact
Rhonda Brophy
 
Clinical research contact
Tina Bocking
 
Medical contact
Clinical Research Unit
 
Social worker/patient navigator contact
Clinical Research Unit
 
Clinical research contact
Stephanie Badour
 

 

 

Study Description

This is a study of two medications taken by mouth, called lenvatinib and everolimus. Lenvatinib blocks cell proteins and signals directed at blood vessels that help the cancer to survive. Everolimus is a cancer medicine that interferes with the growth of cancer cells and slows their spread in the body. It is called an mTOR inhibitor.

The phase 1 of this study has been completed, and now the phase 2 is on-going to understand the anti-cancer activity of those two medications taken together in patients with Ewing sarcoma, rhabdomyosarcoma or high grade glioma that has come back (relapsed) or is refractory to treatment.

Inclusion Criteria
  • Age from 2 years to 21 years
  • Diagnosis of Ewing sarcoma, rhabdomyosarcoma or high grade glioma that has come back (relapsed) or is refractory to treatment
  • Multiple other inclusion and exclusion criteria could apply and will be reviewed by your treating team

OZM-077 - Phase I/Ib Trial of Combined 5'Azacitidine and Carboplatin for Recurrent/Refractory Pediatric Brain and Solid Tumors

Closed to enrollment

OZM-077 - Phase I/Ib Trial of Combined 5'Azacitidine and Carboplatin for Recurrent/Refractory Pediatric Brain and Solid Tumors

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DiagnosisRecurrent Childhood CNS Tumor, Ependymoma, Recurrent Childhood, Childhood Solid Tumor, Other solid tumors, Other brain tumoursStudy StatusClosed to enrollment
PhaseI
AgeChild, Adult - (1 Year to 18 Years)RandomisationNO
Line of treatmentDisease relapse or progression
Routes of Treatment AdministrationIV
Last Posted Update2021-12-23
ClinicalTrials.gov #NCT03206021
International Sponsor
The Hospital for Sick Children
Principal Investigators for Canadian Sites
The Hospital for Sick Children – Dr. Ramaswamy
Montreal Children's Hospital – Dr. Legault
Alberta Children's Hospital – Dr. Lafay-Cousin
BC Children's Hospital – Dr. Hukin
CHU Ste Justine – Dr. Samson
Children’s Hospital of Eastern Ontario (CHEO)– Dr. Johnston


Centres
Medical contact
Dr. Victor Lewis

 

Social worker/patient navigator contact
Wendy Pelletier
Clinical research contact
Debra Rich
Medical contact
Rebecca Deyell

 

Social worker/patient navigator contact
Ilana Katz 

 

Clinical research contact
Hem/Onc/BMT Clinical Trials Unit

 

Medical contact
Dr. Carol Portwine
 
Social worker/patient navigator contact
Jane Cassano 
 
Clinical research contact
Sabrina Millson
 
 
Medical contact
Dr. Alexandra Zorzi
Dr. Shayna Zelcer
 
Social worker/patient navigator contact
Cindy Milne Wren
Jessica Mackenzie Harris
 
Clinical research contact
Mariam Mikhail
Medical contact

Dr. Daniel Morgenstern

daniel.morgenstern@sickkids.ca

Social worker/patient navigator contact

Karen Fung 

karen.fung@sickkids.ca

Clinical research contact

New Agent and Innovative Therapies (NAIT) 

nait.info@sickkids.ca

 

Medical contact
Dr. Henrique Bittencourt
Dr. Monia Marzouki
Dr. Sebastien Perreault (neuro-onc)
 
Social worker/patient navigator contact
Marie-Claude Charrette
 
Clinical research contact
Marie Saint-Jacques
 
Medical contact
Clinical Research Unit
 
Social worker/patient navigator contact
Clinical Research Unit
 
Clinical research contact
Stephanie Badour
 
Medical contact
Dr. Donna Johnston
Dr. Lesleigh Abbott
Dr. Doaa Abdel Fattah
 
Social worker/patient navigator contact
Sherley Telisma
 
Clinical research contact
Carol Duchenne
 

 

 

Study Description

Many pediatric brain and solid tumors have genetic changes that do not involve changes to the underlying DNA sequence called epigenetic alterations.These dysfunctions can favor tumour developpment. 5'Azacitidine is a drug that has similarities with one of the components of DNA and RNA.

It can kill abnormal cells through incorporation into DNA and RNA. It is expected to slow or stop the growth of cancer cells.

The aim of this phase I/II study is to evaluate the dose, safety, tolerability, antitumor activity and other characteristics of 5'azacytidine in combination with carboplatin.

Inclusion Criteria
  • Greater than the age of 1 year and under age 18 at the time of study enrolment
  • Recurrent or refractory solid tumor (Phase I), or recurrent or refractory ependymoma (Phase Ib)
  • Tissue from diagnosis or resection prior to registration must be available (either flash frozen tissue or an FFPE (formalin fixed paraffin embedded) block)
  • Previous therapy with carboplatin will be permitted
  • Failed first line treatment (surgery, radiation therapy or chemotherapy) and should not be eligible for treatment with curative potential.

Multiple other inclusion and exclusion criteria could apply and will be reviewed by your treating team.

Publications

Mack SC, Witt H, Piro RM, Gu L, Zuyderduyn S, Stütz AM, Wang X, Gallo M, Garzia L, Zayne K, Zhang X, Ramaswamy V, Jäger N, Jones DT, Sill M, Pugh TJ, Ryzhova M, Wani KM, Shih DJ, Head R, Remke M, Bailey SD, Zichner T, Faria CC, Barszczyk M, Stark S, Seker-Cin H, Hutter S, Johann P, Bender S, Hovestadt V, Tzaridis T, Dubuc AM, Northcott PA, Peacock J, Bertrand KC, Agnihotri S, Cavalli FM, Clarke I, Nethery-Brokx K, Creasy CL, Verma SK, Koster J, Wu X, Yao Y, Milde T, Sin-Chan P, Zuccaro J, Lau L, Pereira S, Castelo-Branco P, Hirst M, Marra MA, Roberts SS, Fults D, Massimi L, Cho YJ, Van Meter T, Grajkowska W, Lach B, Kulozik AE, von Deimling A, Witt O, Scherer SW, Fan X, Muraszko KM, Kool M, Pomeroy SL, Gupta N, Phillips J, Huang A, Tabori U, Hawkins C, Malkin D, Kongkham PN, Weiss WA, Jabado N, Rutka JT, Bouffet E, Korbel JO, Lupien M, Aldape KD, Bader GD, Eils R, Lichter P, Dirks PB, Pfister SM, Korshunov A, Taylor MD. Epigenomic alterations define lethal CIMP-positive ependymomas of infancy. Nature. 2014 Feb 27;506(7489):445-50. doi: 10.1038/nature13108. Epub 2014 Feb 19.

Ramaswamy V, Hielscher T, Mack SC, Lassaletta A, Lin T, Pajtler KW, Jones DT, Luu B, Cavalli FM, Aldape K, Remke M, Mynarek M, Rutkowski S, Gururangan S, McLendon RE, Lipp ES, Dunham C, Hukin J, Eisenstat DD, Fulton D, van Landeghem FK, Santi M, van Veelen ML, Van Meir EG, Osuka S, Fan X, Muraszko KM, Tirapelli DP, Oba-Shinjo SM, Marie SK, Carlotti CG, Lee JY, Rao AA, Giannini C, Faria CC, Nunes S, Mora J, Hamilton RL, Hauser P, Jabado N, Petrecca K, Jung S, Massimi L, Zollo M, Cinalli G, Bognár L, Klekner A, Hortobágyi T, Leary S, Ermoian RP, Olson JM, Leonard JR, Gardner C, Grajkowska WA, Chambless LB, Cain J, Eberhart CG, Ahsan S, Massimino M, Giangaspero F, Buttarelli FR, Packer RJ, Emery L, Yong WH, Soto H, Liau LM, Everson R, Grossbach A, Shalaby T, Grotzer M, Karajannis MA, Zagzag D, Wheeler H, von Hoff K, Alonso MM, Tuñon T, Schüller U, Zitterbart K, Sterba J, Chan JA, Guzman M, Elbabaa SK, Colman H, Dhall G, Fisher PG, Fouladi M, Gajjar A, Goldman S, Hwang E, Kool M, Ladha H, Vera-Bolanos E, Wani K, Lieberman F, Mikkelsen T, Omuro AM, Pollack IF, Prados M, Robins HI, Soffietti R, Wu J, Metellus P, Tabori U, Bartels U, Bouffet E, Hawkins CE, Rutka JT, Dirks P, Pfister SM, Merchant TE, Gilbert MR, Armstrong TS, Korshunov A, Ellison DW, Taylor MD. Therapeutic Impact of Cytoreductive Surgery and Irradiation of Posterior Fossa Ependymoma in the Molecular Era: A Retrospective Multicohort Analysis. J Clin Oncol. 2016 Jul 20;34(21):2468-77. doi: 10.1200/JCO.2015.65.7825. Epub 2016 Jun 6.

116540 - An Open-Label, Dose-Escalation, Phase I/II Study to Investigate the Safety, Pharmacokinetics, Pharmacodynamics and Clinical Activity of the MEK Inhibitor Trametinib in Children and Adolescents Subjects With Cancer or Plexiform Neurofibromas and Trametinib in Combination With Dabrafenib in Children and Adolescents With Cancers Harboring V600 Mutations

Completed

116540 - An Open-Label, Dose-Escalation, Phase I/II Study to Investigate the Safety, Pharmacokinetics, Pharmacodynamics and Clinical Activity of the MEK Inhibitor Trametinib in Children and Adolescents Subjects With Cancer or Plexiform Neurofibromas and Trametinib in Combination With Dabrafenib in Children and Adolescents With Cancers Harboring V600 Mutations

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DiagnosisPlexiform Neurofibroma, Neurofibromatosis, LGG, Low Grade Glioma, LCH, Langerhans Cell Histiocytosis, other solid tumors and brain tumorsStudy StatusCompleted
PhaseI/II
AgeChild - (1 Month to 17 Years)RandomisationNO
Line of treatmentFirst line treatment, Disease relapse or progression
Routes of Treatment AdministrationTrametinib: oral (capsules, tablets or suspension) Dabrafenib: oral (capsules, tablets or suspension)
Last Posted Update2021-11-03
ClinicalTrials.gov #NCT02124772
International Sponsor
Novartis Pharmaceuticals
Principal Investigators for Canadian Sites
The Hospital for Sick Children – Dr. James Whitlock
Centres
Medical contact

Dr. Daniel Morgenstern

daniel.morgenstern@sickkids.ca

Social worker/patient navigator contact

Karen Fung 

karen.fung@sickkids.ca

Clinical research contact

New Agent and Innovative Therapies (NAIT) 

nait.info@sickkids.ca

 

 

 

Study Description

Some diseases such as cancer with BRAF mutations, low grade gliomas and plexiform neurofibroma in patients with neurofibromatosis type 1 [NF-1], or Langerhans cell histocytosis [LCH]) have been shown to have molecular dysfunction called MAPK pathway dysregulation which favors tumour development. The MAPK pathway is involved in cell growth, differentiation, inflammation and apoptosis.Trametinib is an inhibitor of components of the MAPK pathway called MEK1 and MEK2. Drabafenib is a small molecule that inhibits the MAPK pathway in BRAF mutated cells by blocking the BRAF serine-threonine kinase. Both are expected to slow or stop the growth of cancer cells.

The aim of this phase I/II study is to evaluate the dose, safety, tolerability, antitumor activity and other pharmacologic characteristics of Trametinib alone or with Dabrafenib in children with refractory or relapsed solid cancer or disease with BRAF mutations for which no standard therapy is available or for which the subject is not eligible for the existing therapy.

The study is currently open for LCH and neuroblastoma only.

Inclusion Criteria
  • Male or female between one month and <18 years of age (inclusive) at the time of signing consent
  • Must have a disease that is relapsed/refractory to all potentially curative standard treatment regimens or must have a current disease for which there is no known curative therapy, or therapy proven to prolong survival with an acceptable quality of life.
  • The subject's disease (i.e. cancer, neurofibromatosis type 1 [NF-1] with plexiform neurofibroma [PN], or Langerhans cell histocytosis [LCH]) must have relapsed after or failed to respond to frontline curative therapy or there must not be other potentially curative treatment options available. Curative therapy may include surgery, radiation therapy, chemotherapy, or any combination of these modalities
  • Able to swallow and retain enterally administered medication

Multiple other inclusion and exclusion criteria could apply and will be reviewed by your treating team.

EZH-102 - A Phase 1 Study of the EZH2 Inhibitor Tazemetostat in Pediatric Subjects With Relapsed or Refractory INI1-Negative Tumors or Synovial Sarcoma

Closed to enrollment

EZH-102 - A Phase 1 Study of the EZH2 Inhibitor Tazemetostat in Pediatric Subjects With Relapsed or Refractory INI1-Negative Tumors or Synovial Sarcoma

Go to Health Care Provider version

DiagnosisRhabdoid Tumor, INI1-negative Tumors, Synovial Sarcoma, Malignant Rhabdoid Tumor, Epithelioid sarcoma, Epithelioid malignant peripheral nerve sheah tumor, Extraskeletal myxoid chondrosarcoma, myoepithelial carcinoma, Renal medullary carcinoma, Study StatusClosed to enrollment
PhaseI
AgeChild - (6 Months to 17 Years)RandomisationNO
Line of treatmentDisease relapse or progression
Routes of Treatment Administrationoral (suspension and tablets)
Last Posted Update2021-10-19
ClinicalTrials.gov #NCT02601937
International Sponsor
Epizyme, Inc.
Principal Investigators for Canadian Sites
The Hospital for Sick Children - Dr. Daniel Morgenstern
Centres
Medical contact

Dr. Daniel Morgenstern

daniel.morgenstern@sickkids.ca

Social worker/patient navigator contact

Karen Fung 

karen.fung@sickkids.ca

Clinical research contact

New Agent and Innovative Therapies (NAIT) 

nait.info@sickkids.ca

 

 

 

Study Description

Some rare cancer such as rhabdoid tumors or epithelioid sarcoma have been shown to have a molecular dysfunction allowing a protein called EZH2 to favour tumour developpment. Tazemetostat is an EZH2 inhibitor and is expected to slow or stop the growth of cancer cells. The aim of this Phase I study is to evaluate the dose, safety, tolerability, antitumor activity and other pharmacologic characteristics of Tazemetostat in children with refractory or relapsed solid cancer  for which no standard therapy is available or for which the subject is not eligible for the existing therapy. 

Inclusion Criteria
  • Age ≥6 months to <18 years
  • Patient has one of the specific disease targeted in this study, histologically confirmed by a CLIA/CAP certified laboratory
  • Relapsed or refractory disease and no standard treatment options as determined by locally or regionally available standards of care and treating physician's discretion
  • Patient is able to swallow and retain orally administered medication

Multiple other inclusion and exclusion criteria could apply and will be reviewed by your treating team.

 

CFZ008 - Phase 1b Study of Carfilzomib in Combination With Induction Chemotherapy in Children With Relapsed or Refractory Acute Lymphoblastic Leukemia

Open

CFZ008 - Phase 1b Study of Carfilzomib in Combination With Induction Chemotherapy in Children With Relapsed or Refractory Acute Lymphoblastic Leukemia

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DiagnosisAcute Lymphoblastic LeukemiaStudy StatusOpen
PhaseI
AgeChild, Adult - (1 Year to 21 Years)RandomisationNO
Line of treatmentDisease relapse or progression
Routes of Treatment Administrationintravenous (carfilzomib); Other drugs as usually administered for leukemia therapy
Last Posted Update2021-09-03
ClinicalTrials.gov #NCT02303821
International Sponsor
Amgen
Principal Investigators for Canadian Sites
CHU Ste-Justine - Dr. Henrique Bittencourt

Centres
Medical contact
Dr. Henrique Bittencourt
Dr. Monia Marzouki
Dr. Sebastien Perreault (neuro-onc)
 
Social worker/patient navigator contact
Marie-Claude Charrette
 
Clinical research contact
Marie Saint-Jacques
 

 

 

Study Description

This phase I/II trial is trying to determine how much carfilzomib (an anti-cancer drug) should be used, alone and in combination with induction chemotherapy, to treat children with acute lymphoblastic leukemia that has come back (relapsed) or is refractory to treatment. Carfilzomib is an anti-cancer medication called proteasome inhibitor and is given intravenously.

 

Inclusion Criteria
  • Age 1 year to 21 years
  • Acute lymphoblastic leukaemia that has come back (relapsed) or is refractory to treatment
  • Multiple other inclusion and exclusion criteria could apply and will be reviewed by your treating team

N2011-01 - NANT 2011- 01: Randomized Phase II Pick the Winner Study of 131I-MIBG, 131I-MIBG With Vincristine and Irinotecan, or 131I-MIBG With Vorinostat for Resistant/Relapsed Neuroblastoma

Closed to enrollment

N2011-01 - NANT 2011- 01: Randomized Phase II Pick the Winner Study of 131I-MIBG, 131I-MIBG With Vincristine and Irinotecan, or 131I-MIBG With Vorinostat for Resistant/Relapsed Neuroblastoma

Go to Health Care Provider version

DiagnosisNeuroblastomaStudy StatusClosed to enrollment
PhaseII
AgeChild, Adult - (2 Years to 30 Years)RandomisationYES
Line of treatmentDisease relapse or progression
Routes of Treatment AdministrationVorinostat (oral); vincristine, irinotecan and 131I-MIBG are given intravenously.
Last Posted Update2021-09-03
ClinicalTrials.gov #NCT02035137
International Sponsor
New Approaches to Neuroblastoma Therapy Consortium
Principal Investigators for Canadian Sites
The Hospital for Sick Children – Dr. Meredith Irwin
Centres
Medical contact

Dr. Daniel Morgenstern

daniel.morgenstern@sickkids.ca

Social worker/patient navigator contact

Karen Fung 

karen.fung@sickkids.ca

Clinical research contact

New Agent and Innovative Therapies (NAIT) 

nait.info@sickkids.ca

 

 

 

Study Description

This study is specifically for patients diagnosed with relapsed or persistent neuroblastoma.

Metaiodobenzylguanidine (MIBG) is a compound that can be combined with radioactive iodine (I-131) to deliver targeted radiation therapy. Oncologists use to deliver targeted radiation to neuroblastoma. I-131 MIBG is administered to a child through an intravenous line and is absorbed by tumor cells, which are killed by radiation emitted by the radioactive I-131. 

The three treatment arms are the following:

  • 131 I-MIBG therapy alone
  • 131 I-MIBG therapy with Irinotecan and Vincristine: The two chemotherapy medications irinotecan and vincristine, which are known to be active agents for neuroblastoma, are given at the same time as the 131I-MIBG, and may make the 131I-MIBG more effective at treating neuroblastoma.
  • 131 I-MIBG with Vorinostat: Vorinostat is a drug that is FDA-approved to treat a certain type of cancer mainly seen in adults. Vorinostat affects the way the DNA that carries our genes is folded in cells. In the laboratory, vorinostat causes neuroblastoma cells to stop growing. This effect is even greater when vorinostat is combined with radiation. Giving vorinostat together with the 131I-MIBG may make the 131I-MIBG more effective at treating neuroblastoma.

Why is this study being done?

  • To find out which of the three 131I-MIBG treatment arms have a better tumor response rate
  • To compare the side effects seen with 131I-MIBG alone, compared with 131I-MIBG in combination with Vincristine and Irinotecan or  131I-MIBG with Vorinostat.
  • To describe the number of patients found to have tumor cells in the blood and bone marrow using a new sensitive test following each of the three 131I-MIBG treatment arms.
  • To compare the exposure of whole body radiation from 131I-MIBG received on the three treatment arms.
  • To learn about a new computerized way of reading MIBG scans.
Inclusion Criteria

(From NANT website - https://www.nant.org/n2011-01/)

  • Patients must be at least 24 months and no older than 30 years of age
  • Patients must have relapsed neuroblastoma, refractory neuroblastoma or persistent neuroblastoma 
  • Patients must have  at least one site of tumor with MIBG uptake based on an MIBG scan done within 4 weeks prior to entry on study
  • Patients must have an adequate number of  peripheral blood stem cells available.
  • Patients must have adequate organ function.
  • Multiple other inclusion and exclusion criteria could apply and will be reviewed by your treating team